Archive for the ‘Skin Stem Cells’ Category

If youve ever felt overwhelmed by the thousands of beauty products on the market, we hear you. Thats why we spent hundreds of hours doing the leg work and finding the best ones for you! We read through scientific studies to understand how ingredients work, did market research to find the best and newest products on the market, and asked our most-trusted experts what products they live and swear by. We take beauty seriously, because we understand the importance a product can play in our lives.

Because sometimes a $5 cheap thrill is as good (or *gasp* better) than its pricey counterpart, we decided to throw in a drugstore and luxe pick for almost every item, and we interviewed experts to get their picks, too. Each product deserves a place on this list for its unique features, and we cant wait to share them with you. Youll also find standout stars that were so great that we had to feature themeven though they didnt fit into any one product category.

The HelloGiggles team poured our hearts, souls, and most importantly, brainpower into this project and we found the best of the best. We stand behind each and every one of these products so that you can find the ones that speak to you. Consider the Beauty Crush Awards your shopping list for the yearmade by your best friends for the people they love.

Put your best face forward with these top-of-the class moisturizers, serums, face masks, and more. We tested hundreds of skincare products to find the crme de la crme. And, because we know skin can mean a hell of a lot of things to a lot of different people, we made sure that every winner blessed the face of women and men with different skin concerns, textures, and tones.

Best Drugstore Cleanser: Honest Beauty Gel-to-Milk Cleanser, $12.99 (orig. $14.99), amazon.com

This cleanser is a godsend for those with sensitive skin because of both the affordable price and the rosewater-infused formula. (Studies show that rose extract has anti-inflammatory benefits, which tackles irritation, making it perfect for those with skin prone to irritation.) The product starts as a gel-like formula and, upon contact with water, turns into a milky liquid that gently and effectively cleanses skin.

Best Luxury Cleanser: SkinCeuticals Glycolic Renewal Cleanser Gel, $38, dermstore.com

If your skin is looking dull or is congested, consider using an exfoliating cleanser two to three times a week to decongest and reveal bright, glowy skin. This cleanser uses glycolic acid, a chemical exfoliant that helps turn over dead skin cells to treat concerns such as uneven skin tone, rough skin, and fine lines.

Best Expert-Recommended Cleanser: Differin Daily Refreshing Cleanser, $8.49, amazon.com

I am a big fan of Differin Gel .1% adapalene, [and] I love this new addition to the line, says Dendy Engelman, M.D., a board-certified dermatologist. When cleansing, you dont want to strip the skin of natural oils. This can cause your skin to go into oil production overdrive. This cleanser uses vitamin B and glycerin to gently cleanse; vitamin B to calm the skin and provide relief to inflammation and redness, and glycerin to prevent water loss so the skins natural oils can nourish and repair.

Best Drugstore Makeup Remover: Almay Micellar Biodegradable Makeup Wipes, $4.99 (orig. $5.99), amazon.com

By now we all know and love micellar water for how effectively it removes makeup and cleanses skin in just one step. For those who need a refresh, micelles work like tiny magnets to attract impurities off the skin. We love these makeup wipes because they allow us to easily remove our makeup (even the stubborn waterproof kind) at the end of the day without irritating even the most sensitive of skin. This product won five extra points during our testing period for its biodegradable material, which is made up of plant fibers and wood pulp, and it will completely compost in just three months.

Best Luxury Makeup Remover: First Aid Beauty Conditioning Eye Makeup Remover, $18, ulta.com

Eye makeup can be a fickle foe when it comes to taking it off at the end of the night. Too often leftover mascara clings to our lashes, eyeshadow lingers in the creases of our lids, and eyeliner finds its way to our inner eye areano thank you. This oil-free formula works to remove even waterproof makeup without leaving a trace or any greasy residue. To use, apply some liquid onto a cotton ball, place over the eye area for five seconds, then gently pull down and away. Bonus pointit uses soothing chamomile and is fragrance-free, so its suitable for sensitive skin, too.

Best Expert-Recommended Makeup Remover: Simple Water Boost Micellar Make-up Remover Eye Pads, $8.49, cvs.com

This budget-friendly product uses micellar technologies to remove even the most longwear makeup without irritating skin. Anyone who knows me can attest to the fact that I go all out with both eyeshadow and mascara every day, so when it comes to taking it off, I need something thats going to really workand requires the least amount of elbow grease on my part, says Lisa DeSantis, Deputy Beauty Director of Real Simple and Health magazines. These rounds are saturated with just enough of the hardworking micellar formula to remove even the most stubborn eye makeup.

Best Drugstore Toner: Burts Bees Micellar Toning Water With Rose Water, $8.99, target.com

If youre looking for a wallet-friendly toner that wont sting your skin (no skincare product ever should) or dry you out, this product is for you. This alcohol-free formula uses calming rose petal extract, hydrating glycerin, and tone-balancing lactic acid.

Best Luxury Toner: belif Aqua BombHydrating Toner, $28, sephora.com

Imagine a facial toner that feels like a cup of water for your skinthats this one. It combines hydrating ingredients like glycerin with soothing ones like oat extract and calendula for a pleasant experience thats both effective in removing makeup and debris and gentle on the skin.

Best Expert-Recommended Toner: Peter Thomas Roth Water Drench Hyaluronic Cloud Hydrating Toner Mist, $28, dermstore.com

This product uses hyaluronic acid to hydrate and instantly reduce the appearance of fine lines and wrinkles, says Deanne Robinson, M.D., a dermatologist based in Connecticut.

Best Drugstore Serum: Honest Beauty Vitamin C Radiance Serum, $27.99, amazon.com

Vitamin C is a powerful antioxidant that neutralizes free radicals, which come from the sun, pollution, stress, and smokingfactors that can ultimately damage the cells DNAand gives your skin a radiant boost. Regularly using a vitamin C skincare product can boost collagen production and reverse cell damage, which in turn helps skin look firmer and looking younger, and even helps fade and prevent brown spots or pigmentation.

Best Luxury Serum: La Mer Regenerating Serum, $365, sephora.com

Environmental factors such as pollution, free radicals, and harmful UV rays can take a real toll on your skin, making it show signs of aging sooner. This super-charged serum uses antioxidants, seaweed extract, and a new proprietary blend that helps boost the skins natural collagen to reduce the appearance of fine lines, pores, and redness. One HG tester says that after using it for a few weeks, shed never seen her skin look healthier: I feel like I can see a whole new layer of skin on my face.

Best Expert-Recommended Serum: La Roche-Posay Hyalu B5 Serum, $29.99, dermstore.com

Kavita Mariwalla, M.D., a board-certified dermatologist based in West Islip, NY, adores this budget-friendly pick: I love this product for a few reasons: 1) Price point. For the quality of ingredients in it, it is priced really well. 2) B5 is a great ingredient for all skin types and madecassoside is a hero ingredient that acts as an antioxidant but also skin soother. Combine that with its hyaluronic acid and youve got a serum that has great workhouse ingredients in it.

Best Drugstore Moisturizer: Olay Regenerist Whip Face Moisturizer SPF 40, $38.99, ulta.com

To protect your skin from the sun, opt for a face moisturizer with SPF to streamline your routine. Testers loved that this Olay pick looked smooth on skin and acted as the perfect primer for makeup.

Best Luxury Moisturizer: Drunk Elephant Lala RetroWhipped Cream Moisturizer, $60, sephora.com

Testers liked that this cream instantly melted into their skin and made it feel healthy, hydrated, and glowy. It uses a mix of oils, ceramides, and antioxidants, which together provide moisture and help skin retain that moisture. Its also formulated at an ideal pH level of 5.2, so it wont throw your skin off. Oh, and did we mention it has a fun pump? The pump dispenses the perfect-sized amount of moisturizer onto your skin without any of the mess.

Best Expert-Recommended Moisturizer: Cetaphil Ultra Healing Lotion with Ceramides, $19.97, amazon.com

Although this moisturizing lotion is technically formulated for the body, Tiffany Libby, M.D., a board-certified dermatologist in Rhode Island, recommends it for the face as well. I use this on my body and face when I need to double down on my moisture, especially in the winter months, and I love that it is formulated with ceramides to help enhance my skin barrier and keep my skin smooth and hydrated, she says.

Best Drugstore Face Oil: e.l.f. All The Feels Facial Oil, $11.99, target.com

Squalane, hemp seed oil, and rosehip seed oil make this lightweight facial oil the perfect last step in your skincare routine. Simply apply a few drops onto your skin after your moisturizer, then pat it in to encourage absorption and help lock in your skincare.

Best Luxury Face Oil: Snow Fox Herbal Youth Oil, $40, saksfifthavenue.com

This silky-feeling serum combines hydrating and anti-aging properties into one product. It uses blueberry seed oil, which studies show contains high levels of antioxidant properties that protect skin from free radicals, and an exotic herb called Brazilian Paracressextract, which is a muscle relaxant that helps relieve micro-tension. HG testers raved that it gave their skin a healthy glow, one of them even saying it made her skin look like there was a ring light shining on her face at all times. Natural-looking radiance with no filterwere here for it.

Best Expert-Recommended Face Oil: Mary Kay Naturally Nourishing Oil, $48, marykay.com

[This blend] uses a delicious combination of almond, olive, and sesame oils which leave your skin super hydrated, says Ursula Carranza, Beauty and Fashion Director of People en Espaol. You can even use it on your cuticles and on the ends of your hair!

Best Drugstore Eye Cream: Inkey List Caffeine, $9.99, sephora.com

Consider this concentrate a cup of coffee for your under-eye area. Studies show that when applied topically, caffeine can temporarily plump the skin, so a jolt of java-infused product to your weary eyes will make you look instantly more awake.

Best Luxury Eye Cream: Cl de Peau Enhancing Eye Contour Cream Supreme, $270, nordstrom.com

It takes six years to extract the iris extract in this luxe cream (three to cultivate and three to dry), the key result being plumper, firmer, revived skin. Using the cold steel applicator is kind of like jade rolling, and I visibly notice it de-puffing my eyes, mentioned one HG tester, adding that the difference between eyes after applying on just one side was very drastic.

Best Expert-Recommended Eye Cream: Care Skincare Eye + Lip Nourishing Cream, $30, careskincare.com

Formulated to treat and repair the delicate area around the eyes and around the lips, this dense cream with a light diffusing finish softens the look of fine lines and dark circles, says Dr. Robinson. It absorbs quickly and wont drift into eyes or interfere with makeup.

Best Drugstore Exfoliator: Yes To Grapefruit AHA + BHA Exfoliating Tonic, $14.99, amazon.com

AHAs and beta hydroxy acids (BHAs) are known chemical exfoliants that help remove dead skin cells and excess sebum inside the pores. This product is packed with 10% AHA and BHA, making it a powerful exfoliator that more sensitive skin types should probably avoid. However, if your skin can tolerate it, using it two to three times a week will help you achieve a more even skin texture and a newfound radiance.

Best Luxury Exfoliator: Pestle & Mortar Exfoliate Glycolic Acid Toner, $38, neimanmarcus.com

Brighten, tone, and reveal a more radiant face with the help of this glycolic-based exfoliator that will gently slough away dead skin cells. This non-irritating, vegan, and fragrance-free formula is suitable for all skin types.

Best Expert-Recommended Exfoliator: SkinBetter AlphaRet Peel Pads, $95, skinbetter.com

This new peel is an exfoliation powerhouse. Using lactic, glycolic, and salicylic acids, it exfoliates using AHAs and BHAs, targeting discoloration and age spots, boosting collagen and elastin production, and penetrating deep into the cell to dissolve dead skin buildup, explains Dr. Engelman. It contains a patented retinoid, AlphaRet and it also helps treat acne.

Best Drugstore Face Mask: AveenoOat Face Mask with Pomegranate Seed Extract, $8.38, amazon.com

This brightening face mask is a great option for people with dry skinit uses colloidal oatmeal to maintain the skins natural moisture barrier while treating and calming, dried skin. Studies show that pomegranate, the products key ingredient promotes skin cell turnover, which will help users achieve a more radiant appearance. The texture is rich and velvety, and youll get the best results if used two to three times a week.

Best Luxury Face Mask: Glow Recipe Avocado Melt Retinol Sleeping Mask, $49, sephora.com

Face masks dont have to be rinse-and-go. This Glow Recipe mask can be used overnight for added benefits or can work in as little as 20 minutes as a quick skin-boost if thats your preference. Its a good introduction to retinol, the gold-standard of anti-aging ingredients, since its gentle enough that even sensitive skin types can use it without irritation. One HG tester commented that her skin looked less congested the day after using it, and adding that she didnt notice any dryness sometimes associated with retinol products. Additionally, our testers were impressed by how cooling it felt on skin, how soft it felt after washing off, and how it didnt transfer onto their pillows when using overnight.

Best Expert-Recommended Face Mask: SkinBetter Refresh Detoxifying Scrub Mask, $55, skinbetter.com

The clay minerals [in this mask] absorb unwanted oil and remove toxins, while biodegradable spherical beads to lightly resurface the skin, explains Dr. Robinson of her top pick. It can even be used as a spot treatment on areas of focus!

Best Drugstore Sunscreen: La Roche-Posay Anthelios SPF 50 Mineral Sunscreen, $22.99, amazon.com

This mineral sunscreen is oil-free (so non-comedogenic), non-greasy, and easily blends into all skin shades without leaving a white cast. If youre a minimalist who prefers multi-purpose products, this SPF 50 made for face and body should be your go-to. Its TSA-approved too, so you can throw it into your carry-on next time you travel to the beach.

Best Luxury Sunscreen: Coola Mineral Sun Silk Crme SPF 30, $42, amazon.com

The silky feel this mineral sunscreen leaves on the skin will make you feel like a million bucks. And while yes, it makes you feel like you have a Chinchilla-soft face, what we love most about this sunscreen is its formulabecause science. It has UVA and UVB protection, which means that it will help protect your skin from both the suns harmful rays and also from the blue-light emitted from your smart-phone and laptop.

Best Expert-Recommended Sunscreen: Paulas Choice On-The-Go Shielding Powder SPF 30, $29, dermstore.com

Dermatologists recommend reapplying your sunscreen every two hours, but realistically, not too many of us are down for applying a lotion over our makeup throughout the day. Thats why this powder formula is so greatit comes in what looks like a makeup brush, and all you have to do is swipe it across your face to achieve your recommended SPF dose. Plus, it gets rid of excess shine, as Carranza points out.

Best Drugstore Lip Treatment: Awake BeautyMoisture Balm Daytime Lip Mask, $14, ulta.com

This hydrating lip mask smells a little like a watermelon Jelly Rancher and works like a charm thanks to its concentrated blend of vitamin E, olive, argan, rosehip, and raspberry seed oil. Testers unanimously gave it a high score for seeing noticeably healthier lips the day after using it. For best results, use overnight since it is on the thicker side.

Best Luxury Lip Treatment: Biossance Squalane + Rose Vegan Lip Balm, $18, sephora.com

A recent poll showed that 3 percent of the US population say they are vegan, which may not seem like a lot, but that amounts to nearly 10 million people (or nearly the entire population of the state of Michigan). For those 10 million, it may be important to pick a vegan lip treatment that aligns with their dietand this is the best one. It uses plant-derived squalane, rose oil, ceramides, and algae to nourish and plump skin. Apply at night and wake up to smoother and visibly healthier lips.

Best Expert-Recommended Lip Treatment: Hourglass No 28 Tinted Lip Treatment Oil, $49, sephora.com

Treat your mouth to a luxurious anti-aging treatment that rivals even the best high-shine lip glosses on the market. It uses top-notch ingredients that work to hydrate and plump the lips, comes in four colors, and has Carranzas stamp of approval: Its the perfect lip oilits deeply hydrating without being sticky, gives your lips an amazing shine, and the gold-plated applicator is to die for!

Best Self-Tanning Face Towelettes: Haute Bronze Face Towel, $35, haute-bronze.com

For those hoping to get a sun-kissed glow without having to spend time in the sun, these bronzing face towels are a holy grail, and theyre so easy to use! Just unfold the towel, and using circular motions apply it to dry, clean skinthats it! Plus, since it is a towel, its carry-on approved.

Best Self-Tanning Face Serum: Tan-Luxe Super Glow Hyaluronic Self-Tan Serum, $49, sephora.com

My goal: maintaining a year-round healthy glow without the harmful sun exposure. The answer: this magic fluid, shares DeSantis. Its an anti-aging serum and tanning formula rolled into one, so it helps me cut down on my skincare steps every morning and night, and Im left with a believable bronze that scores me tons of compliments.

Best Face Roller: MDNA Skincare The Beauty Roller, $150 (orig. $200), nordstrom.com

This tool is great for de-puffing and encouraging lymphatic drainage, since the grooves and rolling action stimulates circulation and relieves tension, says Dr. Engelman. It can also help drive your serum deep into the skin which allows your products to work better for younot to mention, it feels great! Regular lymphatic drainage can help reduce puffiness and can temporarily help sculpt the face for a more lifted look.

Best Gua Sha: Wildling Empress Stone, $65, wildling.com

If youre more of a Gua Sha person, clean beauty expert and HelloGiggles columnist Jessica Yarbrough recommends this product, saying its at the top of her list. I have an extensive collection of what I like to call non-skincare skincare products, things that support my skin without technically absorbing into my skin, she explains. With this in hand, [giving myself a] mindful facial massage has become a daily morning ritual, and my skin is healthier and glowier for it.

Best Device: Finishing Touch Flawless Dermaplane Glo, $19.88, amazon.com

Dermaplaning is a treatment primarily found at a dermatologist or estheticians office, wherein a sharp blade sloughs away the peach fuzz and dead skin cells from the uppermost layer of the skin, leaving your face radiant and baby-soft. This at-home device isnt as sharp as a professionals would be, for obvious safety reasons, but its effective in removing fuzz and excessive dead skin cell buildup. It also comes with a built-in light that improves visibility and in turn, helps you get better results.

Best Tweezers: Tweezerman Rose Gold Point Tweezer, $20.32, amazon.com

Designed for precision, this pointed-tip tweezer is the answer to effectively removing stubble, pesky ingrown hairs, and super-fine facial strands.

Skincare products arent, and shouldnt, be reserved for only the face. Advancements in skincare technology have allowed for the creation of body care products that blew our minds this year. From those that protect our skin from environmental aggressors and reverse signs of aging by altering our genetic code to innovative fuss-free packaging and bath products that are vagina-friendly, here are the body products were currently crushing on.

Best Drugstore Body Wash: Dove Body Wash Mousse with Rose Oil, $5.99, target.com

Body wash, but in a mousse. This gentle body wash has a subtle rose scent that you can actually scratch-and-sniff on the bottle to test out before purchasing. A little goes a long way for head-to-toe moisture.

Best Luxury Body Wash: LOccitane Almond Shower Shake, $25, amazon.com

Because we clearly love a fun body wash, its no wonder we loved this shower shake. Studies show that when applied topically, almond oil improves skin tone and prevents UV damage, and its the key ingredient in this product. To use, shake the bottle to mix the ingredients into a milky formula and apply onto wet skin. It will leave your body feeling hydrated and soft.. Now, Alexa, play OutKasts Hey Ya!.

Best Expert-Recommended Body Wash: Olay Moisture Ribbons Plus Shea Butter + Blue Lotus Body Wash, $6.99, walgreens.com

Dr. Mariwalla recommends this moisturizing body wash for its shea butterwhich studies show is anti-inflammatory, rich in antioxidants, and is a rich emollientperfect for sensitive skin types.

Best Drugstore Body Lotion: Curl Dry Skin Therapy Itch Defense, $10.94, amazon.com

Tackle dry, itchy skin with this fast-absorbing body lotion that almost instantly soothes and calms skin. This moisturizer, has approval from the National Psoriasis Foundation and holds a seal from the National Eczema Association, is packed with ceramides, vitamin E, and oatmeal extractand its moisturizing effects last all day.

Best Luxury Body Lotion: Augustinus Bader The Body Cream, $165, neimanmarcus.com

The advanced skincare technology in this cream makes this splurge worth the while. It uses epigenetic technology, which essentially tells the skin cells to be healthy overall and can over time alter your genetic code, and helping treat stretch marks, bumpy skin, and cellulite.

Best Expert-Recommended Body Lotion: CetaphilRestoring Lotion with Antioxidants, $19.97, amazon.com

Cetaphil took their classic lotion and supercharged it, says Dr. Engelman. This product is safe for sensitive skin and the added antioxidants and niacinamides will neutralize free radical damage and repair the skin barrier. Niacinamides have a similar effect to retinol, strengthening the skin barrier. However, unlike retinol, it fortifies from the get-go without sensitivity or irritation.

Best Drugstore Hand Cream: eos Shea Better Coconut Hand Cream, $2.98, amazon.com

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The best beauty products from the 2020 Beauty Crush Awards - Yahoo Lifestyle

Numerous revelations about the world have been made in various forms throughout history. Researchers and scientists have continuously managed to discover new ways we can understand the world around us.

Major scientific breakthroughs have been made that helped improve our way of life and will make it easier for us to achieve even more amazing innovation eventually. The 21st century was especially fruitful for the advancements in technology and science. Many of the essential revelations in history were made during the last 100 years. This article will attempt to name the most important ones.

In 2015, the National Aeronautics and Space Administration managed to find evidence that proves that there is liquid water on Mars. Scientists were aided by the first spectrometer provided by NASA, called the Mars Reconnaissance Orbiter.

With its help, they were able to detect hydrated salts all over the distant planet. The hydrated salts are more prevalent during the warmer seasons, which means that water is a crucial ingredient in their development.

Many scientists believe this to be the most crucial discovery of the 21st century. Since Albert Einsteins theory of relativity was published, the thought of time travel has excited scientific minds all over the world.

The LIGO (Laser Interferometer Gravitational-Wave Observatory) project in the United States is responsible for the detection of gravitational waves, which would imply that with enough research time travel could be possible. Journey to the earliest parts of our universe does not seem as impossible as before since this discovery.

Proof of the existence of Dark Matter was found in 2006 by a team of researchers, led by Maxim Markevitch of the Harvard-Smithsonian Center for Astrophysics in Cambridge. They proved its existence by measuring the location of mass that gets created when galaxies collide. Specific clusters of mass get disconnected, and a large amount of visible matter is what makes up dark matter. While this sounds overly complex, the important thing to remember is that this proves that dark matter makes up for 68% of the universe.

Through stem cell research, we can provide better access to organs for patients, meaning that patients no longer need to wait for donors, making it easier to cure certain conditions. Stem cells make it possible to grow an indefinite number of cells of the same type, but other types of cells also arise from that process. This means that it is possible to regenerate organs using skin cells. In the future, the discovery could make it so that the organs needed for treatment are created in a laboratory.

The process of face transplantation uses tissues of a dead person to replace another persons face. It is a complicated process that was first successfully performed in France in 2005. This transplant was only partial, but the first full-face transplant happened only five years later in Spain.

Since then, people with significant congenital disabilities or facial disfigures caused by diseases or burns have had this procedure performed on them in multiple countries. What seemed like science fiction in the 1990s is a reality now.

HIV is considered to be one of the deadliest viruses in the history of humankind. While we still have not found a cure for AIDS, the disease that is a result of an HIV that has advanced too much, we did manage to improve the treatment of HIV.

With the new methods, some of which were developed in Germany, patients with HIV can live longer, almost being able to lead healthy lives. With these exciting advancements, millions of lives have been saved, and it is only a matter of time before we find a complete cure.

Scientists atthe Massachusetts Institute of Technology (MIT)managed to find a way to create false memories and plant them into the brains of mice. While this enormous scientific breakthrough can help us better understand the concept of mind and help us with dealing with mental health issues, it can still be problematic.

It does seem like a scene out of an old science fiction movie, but with this advancement, it could be possible to manipulate a persons memory, and who knows what consequences that could have.

Scientists at the University of Twente have developed robotic body parts using biomechanics. They managed to create robotic arms that can make life easier for people with severe muscular dystrophy. They also implemented previous research on prosthetics in creating these limbs. These robotic body parts could prove to be extremely helpful for wounded soldiers, people suffering from disabilities or older people.

A photon was teleported into space with the help of a laser beam by Canadian scientists. This process is called quantum teleportation, and it can be used to transport information about something, not its physical state. However, it is a step in the right direction of possibly making teleportation of physical matter possible someday.

The process itself was incredibly hard and required extreme precision. Photons are tiny, meaning that it will be a while until we can successfully teleport larger items.

Although the World Wide Web originated much earlier, it was not until the 21st century that we saw everything the internet is capable of. And it is a fitting way to end this list because it impacted more lives than anything else. Thanks to the internet, we can do things we could only imagine 20 years ago, basically from anywhere in the world. We can access any information, watch every movie in existence, or talk to people halfway around the world. It is the discovery of all the possibilities of the internet that made this century what it is, at least for now.

Where and when was the first face transplant procedure performed?

The first face transplant procedure was performed in France in 2005.

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10 Of The Biggest World Revelations In The 21st Century - World Atlas

The most emotional issue my patients have is hair loss, says New York dermatologist Cheryl Karcher, MD below a jaw-dropping before-and-after photo shared to her Instagram page. On the left half of the photo shared is a young womans exposed hairlinethe hair is so thin and sparse, the entire scalp is visible wherever your eye is drawn. On the right side of the photo, the same woman, but with an almost unbelievable amount of thicker hair, and, somehow, a sense of renewed confidence.

The secret? A little thing called nanofat.

In the past we only had PRP to offer that had to be done three times or more. Sometimes it would work, sometimes it didnt. Now we have nanofat hair restoration, which needs to be done just once, and is much more effective way to treat hair loss and grow hair, explains Dr. Karcher.

You May Also Like: How Low Level Laser Therapy Actually Works to Thicken Hair

So what is nanofat? According to Dr. Karcher, its derived from our own adipose tissue, whereas the ever popular PRP is derived from our blood. Nanofat includes adipose-derived stromal vascular fraction, which contains stem cells as well as growth factors. PRP contains the growth factors released from platelets in the blood, she adds. The procedure itself involves extracting anywhere from 20 to 40 millilitersof fat, usually from the abdomen, then processing it through mechanical filters, before injecting.

Like PRP, the possibilities of what nanofat can help with doesnt stop at the hairline. After the nanofat is processed to the point where there is no fat left, only stem cells and growth factors, it is injected into the scalp, the face, the neck, the decollete, or to improve sun damage, skin pigmentation, decrease wrinkles, and of course grow hair, says Dr. Karcher.

When nanofat is used for hair restoration, Dr. Karcher says she first injects the nanofat, then injects the patients PRP on top of it to act as a fertilizer for the nanofat. Perhaps the best part? Theres little to no painDr. Karcher says the most pain patients feel is during the PRP injections, so the scalp is numbed topicallyand no downtime. When nanofat is used on the face, chest or other areas, Dr. Karcher warns there may be some downtime of erythema and swelling or bruising. If injected for [skin] rejuvenation via microneedling the downtime is only about 48 hours.

While Dr. Karcher has seen unparalleled results from nanofat hair restoration, it is only ideal for patients who have some hair still present on the scalppatients who are completely bald may not be ideal candidates for the procedure. The only time I ever use PRP for hair restoration now is in a patient that doesnt have enough fat to harvest. The nanofat is just one treatment and the results seem to be superior. However, as La Jolla, CA plastic surgeon Robert Singer, MD notes, there is no safety or efficacy data surrounding nanofat treatment as of press time.

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Doctors Are Injecting This Naturally-Derived Substance to Restore Hair Thicknessand Its Not PRP - NewBeauty Magazine

Treatment for sickle cell disease has come a long way since the 1970s when the life expectancy of people living with it was less than 20 years.

People with sickle cell disease are not only living longer life expectancy is now 42 to 47 years of age but are enjoying a better quality of life, too.

"In the Philadelphia area, there has been great pediatric care for sickle cells disease and because of that people who have it are living very well," said Dr. Farzana Sayani, a hematologist at Penn Medicine.

Sayani is the director of a comprehensive sickle cell program focusing on adults living with the disease. Penn also has an active transition program for youth transitioning from a pediatric institution to adult care.

Sickle cell disease is an inherited red blood cell disorder that affects about 100,000 Americans.It is most often found in people of African or Hispanic descent.About 1 in 365 African-American babies are born with sickle cell disease, according to Sayani.

People who have the disease inherit an abnormal type of hemoglobin in their red blood cells, called Hemoglobin S, from both their mother and father.When only one parent has the hemoglobin S gene, a child will have the sickle cell trait, but usually does not develop the disease. But they may pass it on to their children.

Hemoglobin is the protein in the blood responsible for carrying oxygen to the rest of the body. Hemoglobin S causes red blood cells to become stiff and sickle-shaped. Instead of being round in shape, they look like crescent moons.

Sickle cells are sticky and can bind together, blocking the flow of blood and preventing oxygen from getting where it needs to go in the body. This causes sudden attacks of pain referred to as a pain crisis.

There are severaldifferent types of sickle cell disease.Hemoglobin SS, also known as sickle cell anemia, is the most common and most severe type of sickle cell disease.

Anemia occurs when red blood cells die at a rate faster than the body can replace them. Normal red blood cells generally live for 90 to 120 days. Sickled cells only live for 10 to 20 days. This shorter life-to-death cycle is harder for the body to sustain.

Another form,Hemoglobin SC, is not as severe as sickle cell anemia, but it can still cause significant complications, Sayani said.Other forms include Hemoglobin S0 thalassemia, Hemoglobin S+ thalassemia, Hemoglobin SD and Hemoglobin SE.

Sickle cell disease screening is a mandatory part of newborn screenings in Pennsylvania.

If the screening is positive, the family is informed and plugged into the health care system in order to receive the proper care.

If the disease is not diagnosed at birth, a blood test can confirm it at any age in which symptoms start to surface.

The severity of sickle cell disease can vary.

Each individual is affected differently, making it difficult to predict who will get what complications, Sayani said. That is why a comprehensive sickle cell program is so important.

Early signs include a yellowish tint to the skin or jaundice, fatigue and a painful swelling of the hands and feet.

"Young children with sickle cell disease may be tired, not eat very well and have delayed growth," Sayani said. "They may also develop anemia, be at greater risk of infection and start to experience pain crises."

Acute pain crises, also known as vaso-occlusive crises, can lead to long stays in the hospital to manage the crippling pain. Children with sickle cell disease also tend to experience delayed growth and puberty.

As a person with sickle cell disease grows older, the sickled red blood cells start to affect various organs, bones and joints.

This can lead to acute chest syndrome, which occurs when damaged lung tissues makes it difficult to breathe. Brain complications, including stroke, are possible.People with sickle cell disease are also prone to heart damage, eye problems, and infections like chlamydia, salmonella and staphylococcus. Chronic and acute pain is common.

There are different types of medicine that can help manage sickle cell disease.

Last year, an oral medicine was approved that makes sickle cells less likely to sickle. So was an intravenous medicine that has been shown to reduce pain crises and hospitalizations by 50%. Some people living with sickle cell disease also may need regular blood transfusions.

Hydroxyurea has also been used successfully for many years to reduce pain crises and the need for blood transfusions and hospitalizations.

Currently, blood and bone marrow transplant is the only way to cure the disease. But it is not an option for everyone because of the difficulty of finding a well-matched stem cell donor.

A related donor is best but only about a third of sickle cell patients have a donor that is related and fully-matched, Sayani said.

While these transplants have a 85% or more success rate, they also are associated with significant risks, including organ dysfunction, infection and graft vs. host disease which can be quite debilitating.

Transplants completed in children have the best results, Sayani said. But because of the risks involved, doctors only suggest it for patients with severe forms of the disease.

Early clinical trials with gene therapy are also showing promise, she added.

More:
New sickle cell disease treatments are helping people live longer and giving them a higher quality of life - PhillyVoice.com

Medical ResearchThe belief that acute stress can turn hair gray is a popular one, but until now it hasnt been scientifically proven.

But findings that appeared in the publication Nature indicate that the belief may be more than a myth. The study, which used mice as models, was funded in part by the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other NIH components.

Hair color is determined by cells called melanocytes, which produce the pigment melanin. New melanocytes are made from melanocyte stem cells that live within the hair follicle at the base of the hair strand. As we age, these stem cells gradually disappear. The hair that regrows from hair follicles that have lost melanocyte stem cells has less pigment and appears gray.

A research team, led by Dr. Ya-Chieh Hsu of Harvard University, used mice to examine stress and hair graying. The mice were exposed to three types of stress involving mild, short-term pain, psychological stress, and restricted movement. All caused noticeable loss of melanocyte stem cells and hair graying.

Having established a link between stress and graying, the scientists then explored several potential causes, including the role of the stress hormone corticosterone, but altering its levels didnt affect stress-related graying.

The researchers eventually turned to the neurotransmitter noradrenaline, which, along with corticosterone, was elevated in the stressed mice. They found that noradrenaline, also known as norepinephrine, was key to stress-induced hair graying. By injecting noradrenaline under the skin of unstressed mice, the researchers were able to cause melanocyte stem cell loss and hair graying.

Noradrenaline is produced mostly by the adrenal glands. However, mice without adrenal glands still showed stress-related graying. Noradrenaline is also the main neurotransmitter of the sympathetic nervous system, which is responsible for the fight-or-flight reaction in response to stress.

Ultimately, the team discovered that signaling from the sympathetic nervous system plays a critical role in stress-induced graying. Sympathetic nerves extend into each hair follicle and release noradrenaline in response to stress. Normally, the melanocyte stem cells in the follicle are dormant until a new hair is grown. Noradrenaline causes the stem cells to activate.

Using fluorescent labelling, the researchers observed the stem cells change to melanocytes and migrate away from their reserve in the hair follicle. With no remaining stem cells, no new pigment cells can be made, and any new hair becomes gray, then white.

When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu says. After just a few days, all of the melanocyte stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

The authors highlight the need to further study the interactions between the nervous system and stem cells in different tissues and organs. A news release from the NIH said that the knowledge gained in this work will be useful in future investigations into the impact of stress on the body and the development of new interventions.

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Stress and Gray Hair - ThirdAge

Patient Analyses and Safety Data Continue to Underscore Positive Impact of KD025 in cGVHD

Pre-NDA Meeting with FDA Planned for March 2020; Topline Results of Primary Analysis to be Announced in Q2 2020

NEW YORK, NY / ACCESSWIRE / February 23, 2020 / Kadmon Holdings, Inc. (KDMN) today announced expanded results from the previously reported interim analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD). The data were presented today in the oral latebreaker session at the 2020 Transplantation & Cellular Therapy (TCT) Meetings.

As announced in November 2019, KD025 met the primary endpoint of Overall Response Rate (ORR) at the study's planned interim analysis, two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). In the expanded KD025-213 dataset presented today, ORRs were consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%). Three patients achieved a Complete Response. Responses were observed in all affected organ systems, including in organs with fibrotic disease. KD025 has been well tolerated: adverse events were consistent overall with those expected to be observed in cGVHD patients receiving corticosteroids, and no apparent increased risk of infection was observed. Additional secondary endpoints, including duration of response, corticosteroid dose reductions, Failure-Free Survival, Overall Survival and Lee Symptom Scale reductions continue to mature and will be available later in 2020.

"KD025 has been well tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member.

"We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."

At the TCT Meetings, Kadmon also presented long-term follow-up data from KD025-208, its ongoing Phase 2 study of KD025 in cGVHD (Abstract #15205). These data were recently presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in December 2019.

About the ROCKstar (KD025-213) Trial

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg once daily or KD025 200 mg twice daily, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%.

While the ORR endpoint was met at the interim analysis, which was conducted as scheduled two months after completion of enrollment, topline data from the primary analysis of the KD025-213 study, six months after completion of enrollment, will be reported in Q2 2020. Full data from the primary analysis will be submitted for presentation at an upcoming scientific meeting.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

Story continues

About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, China, Japan and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel (xx) the potential benefits from any of our product candidates being granted orphan drug or breakthrough designation; (xxi) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxii) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (the "SEC"), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/577466/Kadmon-Announces-Expanded-Results-of-Interim-Analysis-of-Pivotal-Trial-of-KD025-in-cGVHD

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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD - Yahoo Finance

Stress may play a key role in just how quickly hair goes from colored to ashen.

Scientists have long understood some link is possible between stress and grey hair.

But this new research more deeply probes the exact mechanisms at play.

The researchers initial tests looked closely at cortisol, the stress hormone that surges in the body when a person experiences a fight or flight response.

Its an important bodily function, but the long-term presence of heightened cortisol links to a host of negative health outcomes.

But the culprit ended up being a different part of the bodys fight or flight response the sympathetic nervous system.

These nerves are all over the body, including making inroads to each hair follicle, the researchers reported.

Chemicals released during the stress response causes pigment producing stem cells to activate prematurely, depleting the hairs reserves of color.

The detrimental impact of stress that we discovered was beyond what I imagined, a lead study author said.

After just a few days, all of the pigment-regenerating stem cells were lost.

Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

But stress isnt the only reason that most people get grey hair.

In most cases, its simple genetics.

Gray hair caused by loss of melanocytes (pigment cells) in the hair follicle.

This happens as we age and, unfortunately, there is no treatment that can restore these cells and the pigment they produce, melanin, a dermatologist told.

Genetic factors determine when you go grey.

There is nothing that can be done medically to prevent this from happening when it is genetically predetermined to happen.

That doesnt mean environmental factors such as stress dont play a role.

Smoking, for instance, is a known risk factor for premature graying.

So kick the habit if you want to keep that color a little longer.

Other contributing factors to premature graying include deficiencies in protein, vitamin B-12, copper, and iron as well as aging due in part to an accumulation of oxidative stress.

That stress prompted by an imbalance between free radicals and antioxidants in your body that can damage tissue, proteins, and DNA.

And some degree of oxidative stress is a natural part of life.

Changes you can pursue to delay premature grays include eating a diet high in omega-3 fatty acids such as walnuts and fatty fish.

It doesnt spend too much time in the skin-damaging and hair-damaging ultraviolet light of the sun, and taking vitamin B-12 and vitamin B-6 supplements.

That said, if you are going gray prematurely, it wouldnt hurt to go have a checkup just in case natural genetic factors arent the sole culprit.

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Stress could be a major cause of grey hair - BOL News

The first time Bill Cronin Googled his own cancer diagnosis in 2016, his heart sank. He had Szary syndrome, a rare and aggressive form of cutaneous T-cell lymphoma and staring back at him were countless articles predicting a negative prognosis.

However, after receiving a stem-cell transplant at Dana-Farber/Brigham and Womens Cancer Center, Cronin is returning to the life he enjoyed before cancer.

Im at a place I never thought Id get to, Cronin says.

In 2015, Cronin, then 60, started feeling incredibly itchy and developed an accompanying rash. He went to his dermatologist, who diagnosed him with eczema and told him to return in five months. The rash continued to grow, however, and at the five month mark, Cronins dermatologist encouraged him to undergo further testing at Dana-Farber.

A blood test revealed that Cronins T-cells a type ofwhite blood cells that make up part of the immune system had becomecancerous. In the case of Szary syndrome, lymphoma cells will circulatethrough the blood stream and deposit in different areas of the skin. This willgenerally lead to a full-body rash and intense itchiness.

Cronin would need a stem cell transplant to combat the disease, but before he could receive one, his care team had to get him into remission. Patients who do not achieve remission prior to transplant have a high chance of relapsing.

When they first told me everything, I was really scared, says Cronin. But I knew I was in one of the best places in the world to figure out and treat this rare disease.

Cronins pre-transplant care was spearheaded by oncologists David Fisher, MD, and Nicole LeBoeuf, MD, MPH, clinical director of Cutaneous Oncology at Dana-Farber, with his transplant conducted by Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. Initially, Cronins disease was incredibly resistant; for nearly three years, mainstay drugs including steroids, monoclonal antibodies, and enzyme blockers all failed to put his disease into remission.

Ultimately, it would take a new drug, mogamulizumab (a type of immunotherapy that directly kills T-cells involved with Sezary Syndrome) to get Cronins disease into remission.

In May 2019, Cronin was cleared to undergo an allogeneic transplant, a type of transplant that uses a donors stem cells, in this case, Cronins brother. Since his transplant Cronin has remained in remission.

We had to use all of our big guns to get him totransplant, but Im pleased with where we are now, says Cutler.

I know the situation can always change, but it was great tobe able to share some good news with my family and friends, adds Cronin.

Patients like Cronin serve as a reminder of how stem cell transplants have improved and continue to impact patient outcomes, Dana-Farber experts note. Initially offered to only an incredibly small patient population when first performed at Dana-Farber in the 1970s, research advancements have, and continue to, broaden who is eligible for a transplant. In 2019, Dana-Farber/Brigham and Womens Cancer Center (DF/BWCC) surpassed 10,000 total adult transplants.

This milestone indicates our success as a program and our volume has allowed us to do the research to help move the field forward rather impressively, says Joseph Antin, MD, chief emeritus of Adult Stem Cell Transplantation at DF/BWCC.

In 1996, Dana-Farber Cancer Institute and Brigham and Womens Hospital merged their then separate transplant centers. By pooling together physical and intellectual resources, the new combined program was able to more than double the number of transplants each hospital could perform individually.

We always felt collaboration was better than competition, explains Robert Soiffer, MD, vice chair of Medical Oncology for Hematological Malignancies and chief of the Division of Hematologic Malignancies, who oversaw the merger with Antin. Each side could learn from the other, and that helped to catapult us into the leadership position we have today.

The Stem Cell Transplantation Program is also bolstered by the Connell and OReilly Families Cell Manipulation Core Facility (CMCF), which was established in 1996. The state-of-the-art center, led by Jerome Ritz, MD, not only processes the stem cells for transplant; it also assists researchers in developing new cell-based therapies for patients.

Another key component to the programs success has been the creation of the Ted and Eileen Pasquarello Tissue Bank. The Pasquarello Tissue Bank receives, processes, banks, and distributes research samplesof blood, bone marrow, and other tissues. Through a database overseen by Vincent Ho, MD, the Institute is able to log, assess, and later review every patients disease, including all complications and mutations. This technology allows researchers to explore the genetic makeup of past donors and better understand why a transplant was or was not successful.

Were still learning from biological specimens we collected 20 years ago, and it will continue to impact care 20 years from now, Soiffer says.

Today, there is a continuous push to develop new and more precise therapies to complement and improve stem cell transplants. The hope is to bring new treatment options to patients like Cronin who are facing rare and difficult diseases.

Before his diagnosis, Bill, and Barbara Finney, his partner ofnearly 30 years, were avid English Country dancers. English Country dancingevolved from the court dances of Europe in the early 17th century, and Croninand Barbara have friends from all over the country who share their passion forit.

While Cronin isnt dancing just yet, as hes stillrecovering from his transplant, he says he couldnt have gotten through thiswithout his partner on the dance floor and in life.

Barbara has been amazing and has helped take care ofeverything I couldnt do, he adds. Ive been fortunate and privileged to notonly have her, but to have been able to come to Dana-Farber.

Read more:
Transplant for Szary Syndrome is Patient's First Step in Returning to the Dance Floor - Dana-Farber Cancer Institute

Researchers from the group of Vlad Cojocaru together with colleagues the Max Planck Institute in Mnster (Germany) have revealed how an essential protein helps to activate genomic DNA during the conversion of regular adult human cells into stem cells.A cells identity is driven by which DNA is read or not read at any point in time. Signaling in the cell to start or stop reading DNA happens through proteins called transcription factors. Identity changes happen naturally during development as cells transition from an undesignated cell to a specific cell type. As it turns out, these transitions can also be reversed. In 2012, Japanese researchers were awarded the Nobel prize for being the first to push a regular skin cell backwards to a stem cell.A fuller understanding of molecular processes towards stem cell therapiesUntil now, it is unknown how the conversion of a skin cell into a stem cell happens exactly, on a molecular scale. Fully understanding the processes with atomic details is essential if we want to produce such cells for individual patients in the future in a reliable and efficient manner, says research leader Vlad Cojocaru of the Hubrecht Institute. It is believed that such engineered cell types may in the future be part of the solution to diseases like Alzheimers and Parkinsons, but the production process would have to become more efficient and predictable.Pioneer transcription factorOne of the main proteins involved in the stem cell generation is a transcription factor called Oct4. It induces gene expression, or activity, of the proteins that reset the adult cell into a stem cell. Those genes induced are inactive in the adult cells and reside in tightly packed, closed states of chromatin, the structure that stores the DNA in the cell nucleus. Oct4 contributes to the opening of chromatin to allow for the expression of the genes. For this, Oct4 is known as a pioneer transcription factor.

The data from Cojocaru and his PhD candidate and first author of the publication Jan Huertas show how Oct4 binds to DNA on the so-called nucleosomes, the repetitive nuclear structures in chromatin. Cojocaru: We modelled Oct4 in different configurations. The molecule consists of two domains, only one of which is able to bind to a specific DNA sequence on the nucleosome in this phase of the process. With our simulations, we discovered which of those configurations are stable and how the dynamics of nucleosomes influence Oct4 binding. The models were validated by experiments performed by our colleagues Caitlin MacCarthy and Hans Schler in Mnster.One step closer to engineered factorsThis is the first time computer simulations show how a pioneer transcription factor binds to nucleosomes to open chromatin and regulate gene expression. Our computational approach for obtaining the Oct4 models can also be used to screen other transcription factors and to find out how they bind to nucleosomes, Cojocaru says.

Moreover, Cojocaru wants to refine the current Oct4 models to propose a final structure for the Oct4-nucleosome complex. For already almost 15 years now, we know that Oct4 together with three other pioneer factors transforms adult cells into stem cells. However, we still do not know how they go about. Experimental structure determination for such a system is very costly and time consuming. We aim to obtain one final model for the binding of Oct4 to the nucleosome by combining computer simulations with different lab experiments. Hopefully, our final model will give us the opportunity to engineer pioneer transcription factors for efficient and reliable production of stem cells and other cells needed in regenerative medicine.ReferenceHuertas et al. (2020) Nucleosomal DNA Dynamics Mediate Oct4 Pioneer Factor Binding. Biophysical Journal. DOI: https://doi.org/10.1016/j.bpj.2019.12.038

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Visualizing the Conversion of Adult Cells to Stem Cells - Technology Networks

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R01DK081113, U01DK103152, P50CA127003; Cell Stem Cell

A case of reverse development: Dana-Farber scientists solve long-debated puzzle of how the intestine heals itself

Newswise BOSTON Deep within the lining of the human intestine lies the source of the organs ability to renew itself and recover from damage: intestinal stem cells (ISCs), lodged in pockets of tissue called crypts, generate the cells that continuously repopulate the intestinal lining. Even the stem cells themselves have a safety net: when theyre damaged, healthy replacements appear in less than a week.

For years, scientists have debated how the ISCs re-emergence occurs. Some have held that the intestine keeps a pool of ISCs on reserve a kind of backup-backup supply to replenish the cache of front-line ISCs that have been lost. Others have maintained that something more involuted is as work: The ISCs, like queen bees, give rise to more specialized, or differentiated, progeny in this case, daughter cells that form the inner lining of the intestine. When the ISCs are damaged, this school of thought held, the daughter cells reverse course and de-differentiate reverting into the ISCs from which they arose.

A new study by Dana-Farber Cancer Institute scientists comes down solidly on the latter option.

Published online today by the journalCell Stem Cell, the researchers found that ISCs and their daughter cells have a strikingly reciprocal relationship: under normal conditions, ISCs differentiate into daughter cells, and, if the ISCs are lost, the daughter cells simply reverse course and become ISCs. Our findings suggest that the restoration of intestinal stem cells occurs entirely by the process of de-differentiation, says the studys senior author, Ramesh Shivdasani, MD, PhD, of Dana-Farber, Brigham and Womens Hospital (BWH), and the Harvard Stem Cell Institute. We showed theres no need for a reserve set of ISCs.

Bolstering their findings, the researchers were also able to capture the de-differentiation process in real time. When cells begin to de-differentiate, they switch on a gene that that allows them to be isolated and collected with laboratory techniques, Shivdasani explains. Through this process, researchers were able to capture the cells along a continuum of de-differentiation. Shivdasani likens it to a baseball play in which a runner is tagged out between first and second base.

Heavy turnover

The intestine is one of just three tissues in the body, along with the skin and blood, in which cells are constantly turning over dying and being replaced by freshly made cells. They share this quality because they are the tissues most intimately in contact with material from the environment, and therefore with potentially harmful substances. The constant turnover, its thought, is a way to prevent toxic substances from having lasting effects on cells and their offspring.

The crypts that hold ISCs are, in a sense, misnamed. Far from being enclosures where dead cells are entombed, they are the sites where ISCs daily generate the billions of daughter cells that take the place of defunct intestinal cells.

One of the chief characteristics of ISCs is that they are extremely radiosensitive, or vulnerable to radiation. People exposed to high levels of radioactivity, in the form of nuclear fallout, for example, can suffer severe intestinal damage because the loss of ISCs halts production of cells to regenerate the damaged tissue. But if ISCs succumb easily to radiation, they also make a rapid return. Patients with radiation-induced intestinal damage who can be kept alive for a week often recover as their ISC levels bounce back.

To determine whether this rebound is due to a reserve stockpile of ISCs or to de-differentiation of daughter cells, Shivdasani and his collaborators performed a kind of time-lapse experiment. They treated a collection of ISC cells with the drug tamoxifen, which caused the cells and their offspring to become fluorescent. They waited 48 hours for the label to take hold, then killed the ISC cells. If the daughter cells were indeed de-differentiating, any ISC cells produced after that point would be fluorescent.Thats exactly what researchers found.

While scientists have been able to convert many kinds of differentiated cells into stem cells using laboratory techniques, Shivdasani and his colleagues discovery demonstrates that de-differentiation ismore than a curious act of nature; it is the principal means to restore damaged stem cell in the intestine. Its not known whether cells in other organs and tissues have this capability, but it remains an open avenue of investigation.

It also isnt clear how the crypt knows that stem cells have died and need to be replaced, Shivdasani remarks, or how the daughter cells receive the signal to de-differentiate. This is a subject were currently exploring.

The lead author of the new paper is Kazutaka Murata, PhD of Dana-Farber and BWH. Co-authors are Unmesh Jadhav, PhD, and Alessia Cavazza, PhD, of Dana-Farber and BWH; Shariq Madha, Justin Dean, Kai Wucherpfennig, MD, PhD, and Franziska Michor, PhD, of Dana-Farber; and Johan van Es, PhD, and Hans Clevers, MD, PhD, of Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences and University Medical Centre, Utrecht, the Netherlands. The research was supported by the National Institutes of Health (grants R01DK081113, U01DK103152, and P50CA127003) and gifts from the Lind family.

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Dana-Farber Cancer Institute is one of the worlds leading centers of cancer research and treatment. It is the only center ranked in the top 5 of U.S. News and World Reports Best Hospitals for both adult and pediatric cancer care.

Dana-Farbers mission is to reduce the burden of cancer through scientific inquiry, clinical care, education, community engagement, and advocacy. We provide the latest in cancer for adults through Dana-Farber/Brigham and Women's Cancer Care and for children through Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

Dana-Farber is dedicated to a unique and equal balance between cancer research and care, translating the results of discovery into new treatments for patients locally and around the world.

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A case of reverse development: Dana-Farber scientists solve long-debated puzzle of how the intestine heals itself - Newswise

He asked her for a list of dream projects she would love to investigate. What followed was a year of challenges, stresses and the ultimate reward guided intellectual freedom toward scientific discovery.

Ashley Kramer, a student at the Wayne State University School of Medicine, is enrolled in the schools M.D.-Ph.D. program, an eight-year commitment broken down into three parts the first two years of medical school, four years of graduate school, then the final two years of medical school. Like all M.D./Ph.D. students at the medical school, Kramer had to complete research rotations with faculty she thought would make good dissertation advisors.

Because I have always loved stem cell biology and had experience working with zebrafish in the past, I decided to do an eight-week rotation in Dr. Thummels lab between my medical year one and medical year two, and made the decision that this was absolutely the perfect lab for me, she said.

Ryan Thummel, Ph.D., is an associate professor of Ophthalmology, Visual and Anatomical Sciences. His lab focuses on retinal development and regeneration in zebrafish, an attractive model to study neurodegenerative diseases because of its ability to regenerate neuronal tissues. Zebrafish fully regenerate their retinas in just a matter of weeks, an ability mammals lack.

Zebrafish and mammals both have a cell called Mller glia that supports retinal neurons. In zebrafish, however, these cells convert to stem cells and are responsible for retinal regeneration.

At the end of the rotation, Dr. Thummel floated the crazy idea of starting to work on this grant, a 70-plus page monster undertaking, during my M2 year, and I immediately jumped at the opportunity. I was excited at the idea of having a four-year research project completely planned out by the time I started my Ph.D. after M2 so I could hit the ground running after the dreaded STEP 1, Kramer said.

I came to him two days later with a nine-page document of project ideas. We sat down for three hours discussing projects and came up with a top-two list of cohesive projects for me to move forward with as a grant and thesis, she said. From there, it was a nearly yearlong process of writing, meeting, revising and repeating for each of the many sections of the grant.

The effort was worth it. Kramer secured a five-year, $294,102 grant from the National Eye Institute of the National Institutes of Health last year to study the molecular mechanisms of retinal regeneration in zebrafish, an organism that exhibits a remarkable capacity for regeneration.

"Ashley is a dedicated young scientist and worked very hard on this grant application," Dr. Thummel said.

The grant is one of the NIHs Ruth L. Kirschstein National Research Service awards, also known as an F30. The project, Elucidating the role of DNA methyltransferases in epigenetic regulation of retinal regeneration in the zebrafish, started last month. She is the principal investigator.

This was an incredibly challenging experience that allowed me to grow immensely as a scientist. Grant writing, planning effective and novel longitudinal scientific investigations, and time management will all be critical skills for me moving forward in my career as a physician scientist, she said. I cannot thank Dr. Thummel and my past advisors enough for all of their mentoring and support in the last ten years who have gotten me to where I am today, and I am looking forward to the rest of my training here at Wayne State and beyond.

Kramer earned her bachelors degree in Genetics, Cell Biology and Development from the University of Minnesota in 2014. Her love of research and stem cell biology started when she was an undergraduate research assistant there.

Nearly a decade later, she is studying how epigenetic marks are added to, and removed from, genes in zebrafish retinal stem cells during the process of retinal regeneration. The role of epigenetics in the body is akin to traffic signs on the road.

If roads had no traffic lights, stop signs or barricades, it would be complete chaos. The same is true for your cells. If you used every single gene encoded in your DNA 100% of the time, your cells would be chaos. Epigenetics is what is responsible for telling your skin cell to be a skin cell and your liver cell to be a liver cell, while they both have the exact same underlying DNA sequence, Kramer said. There are various different epigenetic marks that decorate the DNA without actually changing the sequence. These marks come in many forms and can act to either start, stop or change the amount that a particular gene is used, similar to how a green light, road block or stop sign direct traffic rules.

The process is critical for normal embryonic development and everyday cell processes.

If we can gain a deeper understanding of how species like the zebrafish are able to regenerate tissues when mammals cannot, despite having the same cell types, we may be able to start working to translate those mechanisms to mammals, she said. It is possible that certain regeneration pathways have been epigenetically silenced through evolution and we may be able to use modern advances in gene therapy techniques to unlock regenerative capacity in mammals.

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Anatomy of a grant: Ashley Kramer's yearlong journey to finding her doctoral thesis - The South End

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

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Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Therapy Market Competitive Analysis and Forecast 2017-2025 - News Parents

The global Collagen and gelatin market for regenerative medicine will grow at a CAGR of 8.5% from 2017 to 2022 to reach USD 709.9 million by 2022, according to the latest publication from Meticulous Research. The global collagen and gelatin market for regenerative medicine is driven by rising prevalence of chronic diseases, rapid growth in aging population, and increasing funding for R&D of regenerative medicines. However, growing use of alternate biomaterials inhibits the growth of this market to some extent.

The global collagen and gelatin market for regenerative medicine is mainly segmented by type (collagen and gelatin), by source (porcine, bovine, marine, and other), by application (orthopedics, cardiovascular, wound care, and other), and geography. Based on source, bovine collagen and gelatin held the largest share of the market in 2016, owing to their abundant availability and wide range of applications in the tendon reinforcement, hernia repair, skin & wound healing, and plastic & reconstructive surgery. Further, on the basis of application, orthopedics accounted for the major share of the global collagen and gelatin market for regenerative medicines in 2016, owing to the high prevalence of osteoporosis across the globe due to aging population, growing obesity, and a poor level of physical activity.

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Geographically, this market is segmented into North America (U.S. and Canada), Europe (Germany, France, U.K., Italy, Spain, and RoE), Asia Pacific (China, India, Japan, and RoAPAC), Latin America, and Middle East & Africa. North America commanded the largest share in the global collagen and gelatin market for regenerative medicines in 2016, followed by Europe and Asia-Pacific. The large share of this region is mainly attributed to the increasing prevalence of osteoporosis, chronic wounds, heart diseases; growing meat processing; availability of funding; and presence of many key players in this market. However, Asia Pacific region is expected to witness significant growth during the forecast period due to increasing burden of chronic diseases such as osteoporosis, diabetes, and heart diseases; and growing meat processing.

The key players operating in the global collagen and gelatin market for regenerative medicines are Collagen Solutions Plc (U.S.), Royal DSM (Netherlands), Symatese (France), NuCollagen LLC (U.S.), GELITA AG (Germany), Nitta Gelatin Inc. (Japan), Tessenderlo Group (Belgium), Vornia Biomaterials (Ireland), Advanced BioMatrix (U.S.), Jellagen Pty Ltd (U.K.), EnColl Corporation (U.S.), and XIAMEN HYFINE GELATIN CO., LTD. These vendors have employed various strategies to expand their product and application offerings, global footprint, and augment their market share.

TOP 10 COMPANIES IN COLLAGEN AND GELATIN MARKET FOR REGENERATIVE MEDICINE MARKET

Key questions answered in the report-

Which are the high growth market segments in terms of type, source, application, and regions/countries?

What is the historical market for collagen and gelatin for regenerative medicine across the globe?

What are the market forecasts and estimates from the period 2015-2022?

What are the major drivers, restraints, and opportunities in the global collagen and gelatin market for regenerative medicine?

Who are the major players in the global collagen and gelatin market for regenerative medicineand what share of the market do they hold?

Who are the major players in various countries and what share of the market do they hold?

What are the competitive landscapes and who are the market leaders by sub-region in the global collagen and gelatin market for regenerative medicine?

What are the recent developments in the global collagen and gelatin market for regenerative medicine?

What are the different strategies adopted by the major players in the global collagen and gelatin market for regenerative medicine?

What are the geographical trends and high growth regions/ countries?

Who are the local emerging players in the global collagen and gelatin market for regenerative medicine and how do they compete with the global players?

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Microbubbles/Ultrasound Contrast Agents Market by Therapeutic Area (Cardiovascular Diseases, Renal and Associated Diseases, and others), by Application (Molecular Imaging, Gene Therapy, Drug Delivery, and Stem Cells Delivery) Global Forecasts to 2024

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Collagen and Gelatin Market for Regenerative Medicine is expected to reach USD 709.9 million by 2022 - PharmiWeb.com

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Dubai: It's an industry on the rise. And it's accelerating the speed by which Dubai is fast becoming the "new Beverly Hills of the Middle East."

Already, cosmetic surgery has topped the list in medical tourism in Dubai. According to a Dubai Health Authority (DHA) report, the emirate has the highest number of cosmetic surgeons per capita in the region about 50 specialists for a million people.

Globally, the cosmetic surgery market size is projected to reach $21.97 billion with 7.8% annual growth rate by 2023, according to a Medgadget report.

These are the top surgery options:

To put this in perspective, we talked to several people.

Myra J., a belly dancer working at a Dubai hotel. She was unhappy. Saddle bags on her thighs were making her performance less graceful. She honestly feared she would be jobless soon.

On a weekend, she checked into a leading aesthetic clinic and the surgeon trimmed the pockets of fat around her thighs, sculpting her body back into perfect shape.

It took her a week to recover and get back to work, but she is delighted with her shapely thighs and feels happier than ever before.

'Ageing gracefully'

Nina M., (52), always loved how beautiful her eyes were and how youthful her cheeks looked.

Last year, nowever, as she got busy with her sons marriage preparations, she started to experience bouts of anxiety at the sight of slowly encroaching bags under her eyes and a slight droop in her cheeks.

I wanted to look and feel my best and thought a filler and a round of botox were harmless indulgences. It took me just an hour at the clinic and the results were amazing. I am glad I was able to stop the onslaught of aging in time.

"Its beautiful to age gracefully, but we all do use creams and gels to delay the process. I think of these minor injectibles as tools in our make-up bag.

"I have used fillers and Botox twice since last year, combined with my beauty regimen. I feel it has given me not just confidence but also psychological boost. I would recommend it to everyone.

Anna M was a physical trainer but vexed with her body shape as she had a masculine build.

- Anna M, a physical trainer

This was affecting her work. Most women were intimidated by her personality and she was losing self-confidence.

Four years ago, she went in for breast implants and her life changed.

Addiction to surgery?

People talk about getting addicted to cosmetic surgery. But that is not so. Most people approach a cosmetic surgeon only when they cannot deal with a physical issue themselves and just like one needs medicine when one is ill, cosmetic surgery acts as a solution to boost confidence and self-esteem.

"Look at how people have reclaimed their health with gastric bypass!

- Dr Sanjay Parashar, chairman, Scientific of the Emirates Plastic Surgery Society

Changing the world one person at a time and providing them with an incredible burst of confidence, aesthetic and cosmetic surgeons in the UAE have built a practice of reliability that can take as little as one hour to a day to transform your personality.

Welcome to the multi-million dirham cosmetic surgery industry in the UAE.

It is the toast of medical tourism in the region, with a high footfall of Gulf and Asian medical tourists as well as resident expatriates.

The industry has accelerated at a speed that is making Dubai be hailed as the new Beverly Hills of the Middle East.

Cosmetic or Plastic Surgery?

These are two different concepts. Cosmetic surgery refers to aesthetic surgery and revolves around enhancement of physical features of an individual and is elective.

This includes procedures such as:

Plastic surgery, on the other hand, is a surgical speciality dealing with life-saving procedures of re-construction of the face and body owing to congenital defects, disfigurement due to accident, trauma, burns, tumour removal due to diseases such as cancer.

In most cases, plastic surgery is not elective.

Dh12b Medical tourism sales in 2018

Since the time pop stars began to inundate Instagram with images of their perfect bodies, dazzling smiles, flawless skin and enviable hair volume, elective procedures have become commonplace with teenagers as young as 13 who are going in for instant fixes.

While some procedures require a couple of days of hospital stay and being out of circulation for a while, many quick fixes are carried out during lunch breaks in one-hour durations.

Highest per capita cosmetic surgeons in UAE

Dr Sanjay Parashar, chairman, Scientific of the Emirates Plastic Surgery Society, told Gulf News: Cosmetic surgery tops the list in medical tourism in Dubai and according to a 2015 report of Dubai Health Authority (DHA), Dubai has the highest number of cosmetic surgeons per capita in the region about 50 specialists for a million people.

- Dr Zuhair Al Fardan, President of the Emirates Plastic Surgery

Dr Parashar added: The field has grown beyond expectations and much of the credit goes to the development of a world-class infrastructure in this field and the corresponding health regulations.

"Today, in Dubai, most Day Care Surgery centres where most of the plastic surgery procedures are carried out have the best international accreditations.

Dr Zuhair Al Fardan, President of the Emirates Plastic Surgery, said: Much of the advancement in plastic and cosmetic surgery is work in progress as surgeons are constantly upgrading themselves with techniques, technologies.

The UAE is keeping abreast of the best that is taking place in the world. In the last five years or so, there have been tremendous advancements in cosmetic and plastic surgery in the UAE.

"We have the top plastic surgeons of the world come here to do surgeries and the UAE hosts two major international plastic surgery conferences each year.

Soaring revenues

With greater acceptance and broadening of scope for the discipline, it is evident that plastic surgery is a major revenue earner and places UAE as one of the leading medical tourism destinations in the region.

From a price range of Dh150-250 for a filler to Dh40,000 for a detailed body sculpting procedure, these procedures are money-spinners.

Together, the plastic and cosmetic surgery is a multimillion dirham business in the UAE. While there are no exact figures available, safe estimates can be made, say surgeons.

MULTIMILLION BUSINESS

There are 30 hospitals in Dubai, of which 70 per cent are internationally accredited. The emirate aims to build 22 hospitals by 2020 18 private and 4 public hospitals.

In Dubai, at least 50 per cent of its 30 hospital offer cosmetic and plastic surgery options.

30 number of hospitals in Dubai, of which 70 per cent are internationally accredited

Besides that there are about 150 Day Care Surgery centres and 400 aesthetic clinics in Dubai.

All of them offer a bouquet of cosmetic surgery procedures and their average annual revenue is between Dh4-6 million a year.

400 Number of aesthetic clinics in Dubai

If one were to compute that with the numbers of facilities including hospitals, the annual revenue from cosmetic surgery would run into many millions of dirhams annually.

High on medical tourism

Currently, Dubai aims to attract 500,000 medical tourists a year by end 2020.

In a short priod of time, Dubai has managed to be ranked 17 among the top 25 global destinations for medical tourism and cosmetic surgery, along with fertility, orthopaedic, dental and wellness disciplines in the list of most-billed medical procedures.

40% percentage of tourists who come to Dubai come only for medical tourism. Medical tourism sales topped Dh12 billion in 2018, a 5.5% jump in the number of medical tourists

Based on official data, about 46 per cent of the current medical tourists in Dubai come from Asian countries. About a quarter (25 per cent), come from GCC and Arab countries and 13 per cent from African countries, and the remaining 16 per cent from other countries, mainly the UK and Commonwealth of Independent States (CIS) countries.

In fact, 40 per cent of tourists who come to Dubai come only for medical tourism.

- Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai

Medical tourism sales topped Dh12 billion in 2018, with a 5.5 per cent overall increase in medical tourists.

Medical tourists

Dubai attracted a total of 640,542 international and domestic medical tourists in 2018 (51 per cent were international patients).

European tourists consisting mostly of UK, French and Italian citizens, share 16 per cent of health and wellness tourists. A substantial medical tourism revenue, it is evident, is earned through plastic and cosmetic surgery.

Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai, remarked: American Hospital Dubai, is one of the few facilities offering both comprehensive reconstructive and cosmetic surgery services.

"Our plastic surgeons are fully trained in both reconstructive and cosmetic surgery so we see a wide-ranging case mix, from severe trauma cases to cancer cases and of course, those opting for cosmetic surgery."

Most popular cosmetic surgery

Typically, these patients would be female, who done with having children and raising them, now wish to address the changes in their body. I also see a large number of male patients who want to correct problems associated with their chest with the help of liposuction.

Source: Dr Francis Conroy, consultant plastic, cosmetic and reconstructive surgeon at the American Hospital, Dubai

Most popular cosmetic surgery

"Typically, these patients would be female, who done with having children and raising them, now wish to address the changes in their body. I also see a large number of male patients who want to correct problems associated with their chest with the help of liposuction, said Dr Conroy.

Non-surgical treatments (neuro-modulators, fillers, etc) are still very popular and I have seen a trend in that patients are starting with such treatments at an earlier age.

Given the prestigious reputation of the hospital and the Dubai governments plan to promote medical tourism, I have noticed a huge influx of patients from Africa, Nigeria and Ghana in particular.

"These patients come mainly for cosmetic surgery, knowing that they are in the hands of a highly qualified surgeon, in a safe, luxurious facility, with standards second to none, said Dr Conroy.

Top six cosmetic surgery procedures in town

The procedures can be divided into categories:

Does health insurance cover plastic surgery?

"Reconstructive surgery such as correction of birth deformities such as a tuberous breast, cleft lip, hand deformities, skin transplant following burns, road trauma and breast augmentation and reconstruction following a mastectomy and rebuilding after a tumour resection is all covered under all leading health insurances. There is also new kinds of stem cell therapy being used to regenerate tissues and nerves especially in diabetic patients.

Know the rules:

The DHA has made it mandatory for all Day Care Surgery Centres, most of who carry out aesthetic procedures, to have one leading international accreditation from Canada, US, UK or Australia.

These accreditations were earlier mandatory for hospitals only, but from 2020, all Day Care Surgery centres compulsorily must have an international accreditation.

This ensures that an independent, international medical body enforces global health standards to grant them certification and in case of a sentinel event, conducts its independent inquiry and downgrades these places in case of a serious lapse.

DHA on its own has issued a 25-page manual on quality and regulations that is to be followed at all centres.

A close examination of the Day Care Surgery centres indicates several layers of quality control.

Pre surgery quality

This involves free consultation, especially in case of a second opinion or a first time patient seeking to enquire about a procedure based on his/her requirement. When a patient uploads a request on the website of a centre from anywhere in the world, the centre has to provide a detailed consultation free of charge.

Services available to a patient

Once the patient is convinced and comes in person to consult the doctor, quality is upheld in the pre-diagnostic tests that the patient has to undergo.

Infrastructure quality

DHA has graded Day Care Centres into A, B and C categories based on the level of medical facilities that can be accessed by a patient. Anaesthesia methods such as oral, epidural and general also help classify centres. For instance, hair transplant procedures can only be carried out in B and above grade clinics. Day Care Centres that conduct surgeries under general anaesthesia much be equipped with the Advance Cardiac Life Support (ACLS) with their surgeons and registered nurses being certified as trained in administering ACLS to a patient.

Patient safety protocol

There are very specific guidelines for patient safety and the doctor/surgeon must explain the procedure in detail to the patient and his/her family and obtain a written consent to go ahead after ascertaining that all risks and side-effects have been clearly explained to the patient.

Post-operative regulation

There are specific protocols for discharge of patients undergoing cosmetic surgery.

Although ambulatory care means the patient has to be discharged within the same day, there is a specification about asking the patient to desist from long-distance travel, specific rehabilitative work to be carried out from the next day for which the patient has to be within Dubai and also specific instructions when an overseas patient is declared fit to fly out of the country.

When a patient flies out, he or she is provided with a proper review and notes, with instructions for overseas rehabilitation protocol with reference notes for the rehabilitation instructor and instructions for medication and periodic reviews.

Accountability

The law is clear, said Dr Al Fardan, The DHA regulations constitutes an accountability committee and holds an enquiry to fix the liability. If the centre is found guilty, its licence can be suspended or cancelled depending on the extent of guilt. If the surgeon, anaesthesiologists, nurse and technicians are found guilty, their license to practice is suspended or cancelled. If the crime is lighter, then both the centre and the team are let off with serious warnings.

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Dubai is the 'new Beverly Hills of the Middle East': Here's why - Gulf News

]]> Understanding the genetic causes of rare diseases supports drug development. Credit: Shutterstock.

Developing drugs to treat rare diseases is fraught with challenges; these range from trying to recruit from tiny patient populations to fill much-need clinical trials to the complex reimbursement landscape for these innovative, and often bespoke, therapies. However, as scientists improve their understanding of the genetic causes of many rare conditions and regulators explore new reimbursement options, pharma companies and smaller biotech firms are increasingly being empowered to address more of these tricky indications.

In this context, could 2020 be a breakthrough year for patients with rare diseases? Here are three case studies of companies on the verge of having treatments for rare diseases approved Rocket and Fanconi anaemia, PTC Therapeutics and aromatic l-amino acid decarboxylase (AADC) deficiency and, finally, Amryt and epidermolysis bullosa.

Fanconi anaemia (FA) is a rare paediatric inherited diseasecharacterised by bone marrow failure and predisposition to cancer, in the words of Rocket Pharmas CEO Gaurav Shah. Caused by a mutation in the FANC genes, patients with Fanconi experience bone marrow failure as they are unable to create new blood cells.

The current standard of care for Fanconi is a stem cell transplant, but Shah explains the risks involved with these pioneering procedures.

While these transplants do prolong patients lives, the procedure is incredibly difficult and is associated with a high potential for graft-versus-host disease, he says. Stem cell transplants can also lead to an even higher risk of head and neck cancer risk for Fanconi patients; almost everyone with FA who undergoes this procedure dies in their 30s.

Rocket wants to change this situation with its lentiviral vector gene therapy, RP-L102. It is specifically for Fanconi-A, which Shah explains is the most common form of the disease. He adds that the therapy contains patient-derived haematopoietic stem cells that have been generally modified to contain a functional copy of FANCA gene, a mutation which causes Fanconi-A.

RP-L102 is currently in a global registrational Phase IIA study, which has been efficacious and safe in patients so far. The data demonstrate that a single dose of RP-L102 leads to both genetic and functional correction as measured by a progressive increase in corrected peripheral blood and bone marrow cells, says Shah. Most importantly, this treatment can be administered without a conditioning regimen [of chemotherapy and radiation]. [This] means we may be able to treat patients as a preventative measure before bone marrow failure occurs, like a vaccine, with a single dose administration early in life.

Based on these promising signals, RP-L102 has received all accelerated regulatory tools from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The company is hoping to complete its biologics license applications and marketing authorisation applications (MAA) to the two regulators within the next few years.

To overcome challenges facing Rocket in the development of RP-L102, Shah explains the company worked to improve upon its own expertise in rare diseases by working with world-class research and development partners, as well immersing itself within patient communities to learn more about their treatment needs.

Slightly further along the drug approval journey is PTC Therapeutics AADC deficiency drug, PTC-AADC, for which the company recently submitted an MAA to the EMA. The company expects full EMA approval towards the end of 2020 and to treat the first patients either in the first or second quarter of 2021.

PTC acquired PTC-AADC, alongside other gene therapy assets, when it bought rare central nervous system-focused Agilis Biotherapeutics in July 2018, PTCs EMEA and Asia Pacific senior vice-president and general manager Adrian Haigh explains.

AADC deficiency is a rare condition caused by a mutation in the DDC gene, which leads to issues with the AADC enzyme and subsequent reductions in the production of dopamine. Children suffering with AADC deficiency fail to reach neurological and development milestones and have a high risk of death early in life. The only current approach to treating the condition is through dopamine agonists, which Haigh notes are largely ineffective.

The particular approach developed by Agilis, [which is] unlike other forms of gene therapy, involves delivering a very small dose of gene therapy directly into the affected, post-mitotic cells, Haigh says. The rationale is that once youve delivered the drug to post-mitotic cells, which are not dividing, it is going to stay there for a long time.

Other advantages include a reduced chance of significant immune reaction and since the dose is smaller, the treatment could overcome some of the manufacturing issues facing other gene therapies. PTC has decided to bring PTC-AADCs manufacturing in house so they are not reliant on third parties schedules and capacities.

PTCs MAA for its AADC deficiency gene therapy is based on two clinical trials of 26 patients in total. Haigh explains the company has mapped motor milestones, and he noted that in advisory boards with payers theyve been incredibly impressed by our videos showing children progressing from lying flat on their backs to walking around.

He notes that in this case, it is certainly not ethical to drill a hole in a patients head and inject a virus containing a placebo and instead PTC has successfully completed a single-arm trial by comparing with patients natural history. Regulators need to be open to novel clinical trial design, particularly in rare diseases where you have ethical problems, Haigh argues.

The company had to abandon a previous drug in development because they could not agree an economic and deliverable clinical trial design with the FDA.

One of the main challenges that faced PTC in the development of PTC-AADC was diagnosis. Haigh explains they found a lot of patients have been misdiagnosed with either cerebral palsy or epilepsy so the company launched a free genetic testing programme. This also allowed them to find patients to recruit into the trial and estimate the number of patients with AADC deficiency who might be able to benefit from this gene therapy.

Epidermolysis bullosa (EB) is a group of rare skin conditions caused by genetic mutations in the genes that encode for the proteins of the skin, particularly in collagen VII.

There are currently no approved treatments for this condition, EB charity DEBRAs UK branch director of research Caroline Collins notes the condition is managed by regular changing of dressings and the lancing of blisters.

EB is characterised by blisters and wounds on the skin; these wounds are extremely painful and can cover huge areas of the patients body, such as their whole back or entire legs. However, Collins explains these are not like the kinds of wounds you get with ulcers or burns, and they move continuously.

As well as making it incredibly challenging for patients to deal with these never-healing wounds, it also makes it difficult for drug developers to find and establish accepted clinical trial endpoints centred on wound healing. DEBRA is therefore advocating for natural history to be considered in clinical trial designs, Collins explains.

Despite these challenges, UK drug company Amryt is hoping to submit authorisation applications to the FDA and EMA by the end of 2021 for its EB drug, AP101. The company has repurposed the topical gel created for burns wounds to treat EB. It is made from a combination of an extract from the bark of the birch tree and pure sunflower oil, the companys chief medical officer Dr Mark Sumeray explains.

AP101 is currently being studied in a Phase III study Amryt claim this is the biggest global EB trial ever undertaken and has been granted rare paediatric disease designation from the FDA.

Although the current results are blinded, Sumeray explains a recent analysis by an independent data monitoring board found that the firm only needed to increase the number of patients slightly, suggesting that at this point in time, the data would have looked encouraging. Too small a patient population makes it hard for efficacy to be statistically significant.

Since Amryts AP101 may be the first drug approved for EB, Collins emphasises it is important that the company has productive conversations with regulators about the specific challenges of EB. This will help to set the ground for others to follow and further transform the lives of EB patients.

It is clear that Amryt is committed to EB because the company in-licensed a second EB candidate, a topical gene therapy called AP103 in 2018.

Sumeray explains: We have invested a lot of time and effort in the development, not only of the lead product, but also of relationships with physicians and scientists working in EB. If we can figure out how to successfully bring products to the market and have them reimbursed, then all of that knowledge can applied again.

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Rare disease outlook 2020: three therapies set to make waves this year - pharmaceutical-technology.com

The road into Batlow is littered with the dead.

In the smoky, gray haze of the morning, it's hard to make out exactly what Matt Roberts' camera is capturing. Roberts, a photojournalist with the Australian Broadcasting Corporation, keeps his lens focused on the road as he rolls into the fire-ravaged town 55 miles west of Canberra, Australia's capital. At the asphalt's edge, blackened livestock carcasses lie motionless.

The grim scene, widely shared on social media, is emblematic of the impact the 2019-20 bushfire season has had on Australia's animal life. Some estimates suggest "many, many billions" of animals have been killed, populations of endemic insects could be crippled and, as ash washes into riverways, marine life will be severely impacted. The scale of the bushfires is so massive, scientists are unlikely to know the impact on wildlife for many years.

But even before bushfires roared across the country, Australia's unique native animals were in a dire fight for survival. Habitat destruction, invasive species, hunting and climate change have conspired against them. Populations of native fauna are plummeting or disappearing altogether, leaving Australia with an unenviable record: It has the highest rate of mammal extinctions in the world.

A large share of Australia's extinctions have involved marsupials -- the class of mammals that includes the nation's iconic kangaroos, wallabies, koalas and wombats. A century ago, the Tasmanian tiger still padded quietly through Australia's forests. The desert rat-kangaroo hopped across the clay pans of the outback, sheltering from the sun in dug-out nests.

Now they're gone.

Australia's 2019-20 bushfire season has been devastating for wildlife.

In a search for answers to the extinction crisis, researchers are turning to one lesser-known species, small enough to fit in the palm of your hand: the fat-tailed dunnart. The carnivorous mouse-like marsupial, no bigger than a golf ball and about as heavy as a toothbrush, has a tiny snout, dark, bulbous eyes and, unsurprisingly, a fat tail. It's Baby Yoda levels of adorable -- and it may be just as influential.

Mapping the dunnart's genome could help this little animal become the marsupial equivalent of the lab mouse -- a model organism scientists use to better understand biological processes, manipulate genes and test new approaches to treating disease. The ambitious project, driven by marsupial geneticist Andrew Pask and his team at the University of Melbourne over the last two years, will see scientists take advantage of incredible feats of genetic engineering, reprogramming cells at will.

It could even aid the creation of a frozen Noah's Ark of samples: a doomsday vault of marsupial cells, suspended in time, to preserve genetic diversity and help prevent further decline, bringing species back from the brink of extinction.

If that sounds far-fetched, it isn't. In fact, it's already happening.

Creating a reliable marsupial model organism is a long-held dream for Australian geneticists, stretching back to research pioneered by famed statistician Ronald Fisher in the mid-20th century. To understand why the model is so important, we need to look at the lab mouse, a staple of science laboratories for centuries.

"A lot of what we know about how genes work, and how genes work with each other, comes from the mouse," says Jenny Graves, a geneticist at La Trobe University in Victoria, Australia, who has worked with marsupials for five decades.

The mouse is an indispensable model organism that shares many genetic similarities with humans. It has been key in understanding basic human biology, testing new medicines and unraveling the mysteries of how our brains work. Mice form such a critical part of the scientific endeavor because they breed quickly, have large litters, and are cheap to house, feed and maintain.

The lab mouse has been indispensable in understanding physiology, biology and genetics.

In the 1970s, scientists developed a method to insert new genes into mice. After a decade of refinement, these genetically modified mice (known as "transgenic mice") provided novel ways to study how genes function. You could add a gene, turning its expression up to 11, or delete a gene entirely, shutting it off. Scientists had a powerful tool to discover which genes performed the critical work in reproduction, development and maturation.

The same capability does not exist for marsupials. "At the moment, we don't have any way of manipulating genes in a devil or a kangaroo or a possum," says Graves. Without this capability, it's difficult to answer more pointed questions about marsupial genes and how they compare with mammal genes, like those of mice and humans.

So far, two marsupial species -- the Tammar wallaby and the American opossum -- have been front and center of research efforts to create a reliable model organism, but they both pose problems. The wallaby breeds slowly, with only one baby every 18 months, and it requires vast swaths of land to maintain.

The short-tailed opossum might prove an even more complicated case. Pask, the marsupial geneticist, says the small South American marsupial is prone to eating its young, and breeding requires researchers to sift through hours of video footage, looking for who impregnated whom. Pask also makes a patriotic jab ("they're American so we don't like them") and says their differences from Australian marsupials make them less useful for the problems Australian species face.

But the dunnart boasts all the features that make the mouse such an attractive organism for study: It is small and easy to house, breeds well in captivity and has large litters.

"Our little guys are just like having a mouse basically, except they have a pouch," Pask says.

Pask (front) and Frankenberg inspect some of their dunnarts at the University of Melbourne.

A stern warning precedes my first meeting with Pask's colony of fat-tailed dunnarts.

"It smells like shit," he says. "They shit everywhere."

I quickly discover he's right. Upon entering the colony's dwellings on the third floor of the University of Melbourne's utilitarian BioSciences building, you're punched in the face by a musty, fecal smell.

Pask, a laid-back researcher whose face is almost permanently fixed with a smile, and one of his colleagues, researcher Stephen Frankenberg, appear unfazed by the odor. They've adapted to it. Inside the small room that houses the colony, storage-box-cages are stacked three shelves high. They're filled with upturned egg cartons and empty buckets, which work as makeshift nests for the critters to hide in.

Andrew Pask

Frankenberg reaches in without hesitation and plucks one from a cage -- nameless but numbered "29" -- and it hides in his enclosed fist before peeking out of the gap between his thumb and forefinger, snout pulsing. As I watch Frankenberg cradle it, the dunnart seems curious, and Pask warns me it's more than agile enough to manufacture a great escape.

In the wild, fat-tailed dunnarts are just as inquisitive and fleet-footed. Their range extends across most of southern and central Australia, and the most recent assessment of their population numbers shows they aren't suffering population declines in the same way many of Australia's bigger marsupial species are.

Move over, Baby Yoda.

As I watch 29 scamper up Frankenberg's arm, the physical similarities between it and a mouse are obvious. Pask explains that the dunnart's DNA is much more closely related to the Tasmanian devil, an endangered cat-sized carnivore native to Australia, than the mouse. But from a research perspective, Pask notes the similarities between mouse and dunnart run deep -- and that's why it's such an important critter.

"The dunnart is going to be our marsupial workhorse like the mouse is for placental mammals," Pask says.

For that to happen, Pask's team has to perfect an incredible feat of genetic engineering: They have to learn how to reprogram its cells.

To do so, they collect skin cells from the dunnart's ear or footpad and drop them in a flask where scientists can introduce new genes into the skin cell. The introduced genes are able to trick the adult cell, convincing it to become a "younger," specialized cell with almost unlimited potential.

The reprogrammed cells are known as "induced pluripotent stem cells," or iPS cells, and since Japanese scientists unraveled how to perform this incredible feat in 2006, they have proven to be indispensable for researchers because they can become any cell in the body.

"You can grow them in culture and put different sorts of differentiation factors on them and see if they can turn into nerve cells, muscle cells, brain cells, blood vessels," Pask explains. That means these special cells could even be programmed to become a sperm or an egg, in turn allowing embryos to be made.

Implanting the embryo in a surrogate mother could create a whole animal.

It took about 15 minutes to get this dunnart to sit still.

Although such a technological leap has been made in mice, it's still a long way from fruition for marsupials. At present, only the Tasmanian devil has had iPS cells created from skin, and no sperm or egg cells were produced.

Pask's team has been able to dupe the dunnart's cells into reverting to stem cells -- and they've even made some slight genetic tweaks in the lab. But that's just the first step.

He believes there are likely to be small differences between species, but if the methodology remains consistent and reproducible in other marsupials, scientists could begin to create iPS cells from Australia's array of unique fauna. They could even sample skin cells from wild marsupials and reprogram those.

Doing so would be indispensable in the creation of a biobank, where the cells would be frozen down to -196 degrees Celsius (-273F) and stored until they're needed. It would act as a safeguard -- a backup copy of genetic material that could, in some distant future, be used to bring species back from the edge of oblivion, helping repopulate them and restoring their genetic diversity.

Underneath San Diego Zoo's Beckman Center for Conservation Research lies the Frozen Zoo, a repository of test tubes containing the genetic material of over 10,000 species. Stacked in towers and chilled inside giant metal vats, the tubes contain the DNA of threatened species from around the world, suspended in time.

It's the largest wildlife biobank in the world.

"Our goal is to opportunistically collect cells ... on multiple individuals of as many species as we can, to provide a vast genetic resource for research and conservation efforts," explains Marlys Houck, curator at the Frozen Zoo.

The Zoo's efforts to save the northern white rhino from extinction have been well publicized. Other research groups have been able to create a northern white rhino embryo in the lab, combining eggs of the last two remaining females with frozen sperm from departed males. Scientists propose implanting those embryos in a surrogate mother of a closely related species, the southern white rhino, to help drag the species back from the edge of oblivion.

For the better part of a decade, conservationists have been focused on this goal, and now their work is paying off: In the "coming months," the lab-created northern white rhino embryo will be implanted in a surrogate.

Sudan, the last male northern white rhinoceros, was euthanized in 2018.

Marisa Korody, a conservation geneticist at the Frozen Zoo, stresses that this type of intervention was really the last hope for the rhino, a species whose population had already diminished to just eight individuals a decade ago.

"We only turn to these methods when more traditional conservation methods have failed," she says.

In Australia, researchers are telling whoever will listen that traditional conservation methods are failing.

"We've been saying for decades and decades, many of our species are on a slippery slope," says John Rodger, a marsupial conservationist at the University of Newcastle, Australia, and CEO of the Fauna Research Alliance, which has long advocated for the banking of genetic material of species in Australia and New Zealand.

In October, 240 of Australia's top scientists delivered a letter to the government detailing the country's woeful record on protecting species, citing the 1,800 plants and animals in danger of extinction, and the "weak" environmental laws which have been ineffective at keeping Australian fauna alive.

Institutions around Australia, such as Taronga Zoo and Monash University, have been biobanking samples since the '90s, reliant on philanthropic donations to stay online, but researchers say this is not enough. For at least a decade, they've been calling for the establishment of a national biobank to support Australia's threatened species.

John Rodger

"Our real problem in Australia ... is underinvestment," Rodger says. "You've got to accept this is not a short-term investment."

The current government installed a threatened-species commissioner in 2017 and committed $255 million ($171 million in US dollars) in funding to improve the prospects of 20 mammal species by 2020. In the most recent progress report, released in 2019, only eight of those 20 were identified as having an "improved trajectory," meaning populations were either increasing faster or declining slower compared to 2015.

A spokesperson for the commissioner outlined the $50 million investment to support immediate work to protect wildlife following the bushfires, speaking to monitoring programs, establishment of "insurance populations" and feral cat traps. No future strategies regarding biobanking were referenced.

Researchers believe we need to act now to preserve iconic Australian species like the koala.

In the wake of the catastrophic bushfire season and the challenges posed by climate change, Australia's extinction crisis is again in the spotlight. Koalas are plastered over social media with charred noses and bandaged skin. On the front page of newspapers, kangaroos bound in front of towering walls of flame.

Houck notes that San Diego's Frozen Zoo currently stores cell lines "from nearly 30 marsupial species, including koala, Tasmanian devil and kangaroo," but that's only one-tenth of the known marsupial species living in Australia today.

"Nobody in the world is seriously working on marsupials but us," Rodger says. "We've got a huge interest in maintaining these guys for tourism, national icons... you name it."

There's a creeping sense of dread in the researchers I talk to that perhaps we've passed a tipping point, not just in Australia, but across the world. "We are losing species at an alarming rate," says Korody from the Frozen Zoo. "Some species are going extinct before we even know they are there."

With such high stakes, Pask and his dunnarts are in a race against time. Perfecting the techniques to genetically engineer the tiny marsupial's cells will help enable the preservation of all marsupial species for generations to come, future-proofing them against natural disasters, disease, land-clearing and threats we may not even be able to predict right now.

Pask reasons "we owe it" to marsupials to develop these tools and, at the very least, biobank their cells if we can't prevent extinction. "We really should be investing in this stuff now," he says. He's optimistic.

In some distant future, years from now, a bundle of frozen stem cells might just bring the koala or the kangaroo back from the brink of extinction.

And for that, we'll have the dunnart to thank.

Originally published Feb. 18, 5 a.m. PT.

Originally posted here:
Building a 'doomsday vault' to save the kangaroo and koala from extinction - CNET

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(BPT) - For many people with cancer and other life-threatening diseases, stem cell transplants provide hope and can impact the course of the disease, but they also come with risks. One of those risks is graftversushost disease (GVHD).

What is GVHD?

GVHD is a potentially life-threatening condition that can occur after an allogeneic stem cell transplant from a donor, in which the donated cells initiate an immune response and attack the recipient's organs and tissues. There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.

Although the exact incidence of GVHD is unknown, it is estimated that up to 70% of stem cell transplant recipients will develop either acute or chronic GVHD, resulting in significant morbidity and mortality. Due to these concerning statistics, health care experts and the entire GVHD community are calling for additional research and support.

People with GVHD and their caregivers face a multitude of challenges, often including limited support, minimal information and few treatment options. Its time to change the future for those living with GVHD.

New award inspires the GVHD community

The Incyte Ingenuity Award aims to encourage innovation in GVHD care and other serious diseases. As part of the award, one unique proposal that addresses a critical unmet need in the GVHD community will be awarded up to $100,000 for the proposed initiative to be developed and executed. Specific initiatives may include patient and/or professional educational programs, policy-focused activities as well as awareness and support campaigns.

Incyte wanted to create a community driven program dedicated to improving the lives of patients with serious diseases, such as GVHD, which can be difficult to treat and have a devastating impact on the lives of patients, says Barry Flannelly, Pharm.D., Executive Vice President and General Manager, U.S., Incyte. Through this award, we hope to spark creativity and innovation, resulting in impactful and actionable initiatives for the GVHD community.

Get involved to make a difference

Submissions are accepted from nonprofit 501(c)(3), patient, policy and caregiver organizations, as well as health care providers and midlevel or junior faculty who submit under their health care organizations. To apply, visit http://www.IncyteIngenuityAward.com and submit an online application featuring a summary of the proposed initiative. The application window is now open and will close April 30, 2020.

All applications will be reviewed and evaluated by an independent judging panel that will select the top three entries, who will then be asked to submit a more detailed proposal of their initiative. The final award recipient will be announced in August of 2020. Apply now!

MAT-INC-00717 02/20

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Health insights: What is GVHD and why is innovation so critical? - Eagle & Times

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to enfortumab vedotin-ejfv (Padcev; Astellas Pharma and Seattle Genetics) in combination with Mercks (known as MSD outside the United States and Canada) anti-PD-1 therapy pembrolizumab (Keytruda) for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting.

It is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020. [1] Urothelial cancer accounts for 90% of all bladder cancers and can also be found in the renal pelvis, ureter, and urethra. [2] Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide. [3]

The recommended first-line treatment for patients with advanced urothelial cancer is cisplatin-based chemotherapy. For patients who are unable to receive cisplatin, such as people with kidney impairment, a carboplatin-based regimen is recommended. However, fewer than half of patients respond to carboplatin-based regimens and outcomes are typically poorer compared to cisplatin-based regimens. [4]

Conditionally approvedEnfortumab vedotin-ejfv, a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer, was conditionally approved by the FDA in December 2019 based on the Accelerated Approval Program. [5][6]

Antibody-drug Conjugates or ADCs are highly targeted biopharmaceutical drugs that combine monoclonal antibodies specific to surface antigens present on particular tumor cells with highly potent anti-cancer agents linked via a chemical linker.

With seven approved drugs on the market, ADCs have become a powerful class of therapeutic agents in oncology and hematology.

Continued approval for enfortumab vedotin-ejfv in combination with pembrolizumab for the treatment of patients with advanced or metastatic urothelial cancer may be contingent upon verification and description of clinical benefit in confirmatory trials. [5]

The drug is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting.

Nonclinical data suggest the anticancer activity of enfortumab vedotin is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis). [5]

Breakthrough therapyThe Breakthrough Therapy process is designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition. The designation is based upon preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints. In the case of enfortumab vedotin, the designation was based on the initial results from Phase Ib/II EV-103 Clinical Trial.

The FDAs Breakthrough Therapy designation reflects the encouraging preliminary evidence for the combination of enfortumab vedotin and pembrolizumab in previously untreated advanced urothelial cancer to benefit patients who are in need of effective treatment options, said Andrew Krivoshik, M.D., Ph.D., Senior Vice President, and Oncology Therapeutic Area Head, Astellas.

We look forward to continuing our work with the FDA as we progress our clinical development program as quickly as possible.

This is an important step in our investigation of enfortumab vedotin in combination with pembrolizumab as first-line therapy for patients with advanced urothelial cancer who are unable to receive cisplatin-based chemotherapy, said Roger Dansey, M.D., Chief Medical Officer, Seattle Genetics.

Based on encouraging early clinical activity, we recently initiated a phase III trial of this platinum-free combination and look forward to potentially addressing an unmet need for patients.

Clinical trialThe Breakthrough Therapy designation was granted based on results from the dose-escalation cohort and expansion cohort A of the Phase Ib/II trial, EV-103 (NCT03288545), evaluating patients with locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy-treated in the first-line setting with enfortumab vedotin-ejfv in combination with pembrolizumab.

The initial results from the trial were presented at the European Society of Medical Oncology (ESMO) 2019 Congress, and updated findings at the 2020 Genitourinary Cancers Symposium.

EV-103 is an ongoing, multi-cohort, open-label, multicenter phase Ib/II trial of PADCEV alone or in combination, evaluating the safety, tolerability, and efficacy in muscle-invasive, locally advanced and first- and second-line metastatic urothelial cancer.

Adverse eventsSerious adverse reactions occurred in 46% of patients treated with enfortumab vedotin-ejfv. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Discontinuing treatmentAdverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Specific recommendations

HyperglycemiaHyperglycemia occurred in patients treated with enfortumab vedotin-ejfv, including death and diabetic ketoacidosis (DKA), in patients with and without pre-existing diabetes mellitus. The incidence of Grade 3-4 hyperglycemia increased consistently in patients with higher body mass index and in patients with higher baseline A1C. In one clinical trial, 8% of patients developed Grade 3-4 hyperglycemia. Patients with baseline hemoglobin A1C 8% were excluded.

Physicians are recommended to closely monitor blood glucose levels in patients with, or at risk for, diabetes mellitus or hyperglycemia and, if blood glucose is elevated (>250 mg/dL), withhold the drug.

Peripheral neuropathyPeripheral neuropathy (PN), predominantly sensory, occurred in 49% of the 310 patients treated with enfortumab vedotin-ejf in clinical trials. Two percent (2%) of patients experienced Grade 3 reactions. In one clinical trial, peripheral neuropathy occurred in patients treated with enfortumab vedotin-ejf with or without preexisting peripheral neuropathy.

The median time to onset of Grade 2 was 3.8 months (range: 0.6 to 9.2). Neuropathy led to treatment discontinuation in 6% of patients. At the time of their last evaluation, 19% had complete resolution, and 26% had partial improvement.

Physicians should:

Occular disordersOcular disorders occurred in 46% of the 310 patients treated with enfortumab vedotin-ejf. The majority of these events involved the cornea and included keratitis, blurred vision, limbal stem cell deficiency and other events associated with dry eyes. Dry eye symptoms occurred in 36% of patients, and blurred vision occurred in 14% of patients, during treatment with enfortumab vedotin-ejf.

The median time to onset to symptomatic ocular disorder was 1.9 months (range: 0.3 to 6.2).

Physicians should monitor patients for ocular disorders and consider:

Skin reactionsSkin reactions occurred in 54% of the 310 patients treated with enfortumab vedotin-ejf in clinical trials. Twenty-six percent (26%) of patients had a maculopapular rash and 30% had pruritus. Grade 3-4 skin reactions occurred in 10% of patients and included symmetrical drug-related intertriginous and flexural exanthema (SDRIFE), bullous dermatitis, exfoliative dermatitis, and palmar-plantar erythrodysesthesia. In one clinical trial, the median time to onset of severe skin reactions was 0.8 months (range: 0.2 to 5.3).

Of the patients who experienced rash, 65% had complete resolution and 22% had partial improvement.

Physicians should monitor patients for skin reactions, and consider:

Infusion site extravasationSkin and soft tissue reactions secondary to extravasation have been observed after the administration of enfortumab vedotin-ejf. Of the 310 patients, 1.3% of patients experienced skin and soft tissue reactions. Reactions may be delayed.

Erythema, swelling, increased temperature, and pain worsened until 2-7 days after extravasation and resolved within 1-4 weeks of peak. One percent (1%) of patients developed extravasation reactions with secondary cellulitis, bullae, or exfoliation.

Physicians should ensure adequate venous access prior to starting enfortumab vedotin-ejf and monitor for possible extravasation during administration. If extravasation occurs, stop the infusion and monitor for adverse reactions.

Embryo-fetal toxicityEnfortumab vedotin-ejf can cause fetal harm when administered to a pregnant woman.

Physicians should advise patients of the potential risk to the fetus and advise female patients of reproductive potential to use effective contraception during enfortumab vedotin-ejf treatment and for 2 months after the last dose. At the same time, they should advise male patients with female partners of reproductive potential to use effective contraception during treatment with enfortumab vedotin-ejf and for 4 months after the last dose.

Clinical trialA Study of Enfortumab Vedotin Alone or With Other Therapies for Treatment of Urothelial Cancer (EV-103) NCT03288545

References[1] American Cancer Society. Cancer Facts & Figures 2020. Online. Last accessed on January 23, 2020.[2] American Society of Clinical Oncology. Bladder cancer: introduction (10-2017). Online. Last accessed on January 23, 2020.[3] International Agency for Research on Cancer. Cancer Tomorrow: Bladder. Online. Last accessed on January 23, 2020.[4] National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Bladder Cancer. Version 4; July 10, 2019. Online. Last accessed on January 23, 2020.[5] Enfortumab vedotin-ejfv (Padcev; Astellas Pharma [package insert]. Northbrook, IL)[6] Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.

A version of this article was first published in ADC Review | Journal of Antibody-drug Conjugates.

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Combination Enfortumab Vedotin + Pembrolizumab Granted Breakthrough Therapy in Bladder Cancer - OncoZine

Global Stem Cell Banking Market Report 2020comprises of data that can be quite essential when it comes to dominate the market or making a mark in the market as a new emergent. The statistics are represented in graphical format in this business research report for a clear understanding on facts and figures. The report provides market insights which help to have a more precise understanding of the market landscape, issues that may impinge on the Stem Cell Banking industry in the future, and how to position specific brands in the best way. Analysis and discussion of important industry trends, market size, and market share estimates are mentioned in the Tissue-Engineered Products Market analysis report.

For More Info, Get a Sample[emailprotected]https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-stem-cell-banking-market

The Increased Market Growth can be identified by the increasing procedures of hematopoietic stem cell transplantation (hsct), emerging technologies for stem cell processing, storage and preservation. Increasing birth rates, awareness of stem cell therapies and higher treatment done viva stem cell technology. Data Bridge Market Research has recently announced publishing of a report, titled Global Stem Cell Banking Market Industry Trends and Forecast to 2026 As per the report, Global stem cell banking market is set to witness a substantial CAGR of 11.03% in the forecast period of 2019- 2026. The Stem Cell Banking Market report provides the details related to fundamental overview, development status, latest advancements, market dominance and market dynamics. The report also presents the evaluation of the competitive landscape of the market.

Few Of The Major Competitors Currently Working In The Global Stem Cell Banking Market AreNSPERITE N.V, Caladrius, ViaCord, CBR Systems, Inc, SMART CELLS PLUS, LifeCell International, Global Cord Blood Corporation, Cryo-Cell International, Inc., StemCyte India Therapeutics Pvt. Ltd, Cordvida, ViaCord, Cryoviva India, Vita34 AG, CryoHoldco, PromoCell GmbH, Celgene Corporation, BIOTIME, Inc., BrainStorm Cell Therapeutics and others.

Market Definition: Global Stem Cell Banking Market

Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Explore Key Industry Insights In 60 Tables And 220 Figures From The 350 Pages Of Report, Global Stem Cell Banking MarketBy Source (Placental Stem Cells (PSCs), Human Embryo-Derived Stem Cells (hESCs), Bone Marrow-Derived Stem Cells (BMSCs), o Dental Pulp-Derived Stem Cells (DPSCS), Adipose Tissue-Derived Stem Cells (ADSCs) and Other Stem Cell Sources), By Application (Personalized Storage, Clinical, Research), Service Type (Sample Collection and Transportation, Sample Processing, Sample Analysis, Sample Preservation and Storage), Geography (North America, Europe, Asia Pacific, Latin America and The Middle East and Africa) Industry Trends and Forecast to 2026.

The global Stem Cell Banking market report covers scope and product overview to define key terms and offers detailed information about market dynamics to the readers. This is followed by a regional outlook and segmental analysis. The report also consists of the facts and key values of the global Stem Cell Banking market, in terms of sales and volume, revenue and its growth rate.

One of the important factors in the global Stem Cell Banking market report is competitive analysis. The report covers all of the key parameters, such as product innovation, market strategies of the key players, market share, revenue generation, the latest research and development and market experts views.

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The report focusses on weaknesses and strengths of the global Stem Cell Banking market with a competitive landscape that includes information on some market vendors. Information presented in the report is gathered from primary and secondary research methods. The report also presents recent trends and opportunities of the market helping players strive for the lions share in the market.

Segmentation: Global Stem Cell Banking Market

By Source

By Application

Research

By Service Type

Competitive Analysis: Global Stem Cell Banking Market

The global Stem Cell Banking market is highly fragmented and the major players have used various strategies such as product (software) launches, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of Stem Cell Banking market for global, Europe, North America, Asia Pacific and South America.

Research Methodology: Global Stem Cell Banking Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more please request an analyst call or can drop down your enquiry.

The key research methodology used by DBMR research team is data triangulation which involves data mining, analysis of the impact of data variables on the market, and primary (industry expert) validation. Apart from this, other data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Company Market Share Analysis, Standards of Measurement, Top to Bottom Analysis and Vendor Share Analysis. To know more about the research methodology, drop in an inquiry to speak to our industry experts.

Primary Respondents

Demand Side: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

Supply Side: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, and Regulatory Affairs Managers among others.

Reasons to Purchase this Report

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Research Methodology: Global Stem Cell Banking Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more please request an analyst call or can drop down your enquiry.

The key research methodology used by DBMR research team is data triangulation which involves data mining, analysis of the impact of data variables on the market, and primary (industry expert) validation. Apart from this, other data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Company Market Share Analysis, Standards of Measurement, Top to Bottom Analysis and Vendor Share Analysis. To know more about the research methodology, drop in an inquiry to speak to our industry experts.

Customization of the Report

All segmentation provided above in this report is represented at country level.

All products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

Table of Content: Global Stem Cell Banking Market

Continued..

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Stem Cell Banking Market 2020 Climbs on Positive Outlook of Booming Sales|NSPERITE NV, Caladrius, ViaCord, CBR Systems, SMART CELLS PLUS, LifeCell...

On August 23, 2019, the FamilieSCN2A Foundation held their biennial SCN2A Professional and Family meeting, in Seattle, Washington. The gathering brought together 37 families of individuals with mutations in the SCN2A gene, 60 investigators, eight clinicians and five industry groups that conduct research and/or clinical work on conditions related to this genetic change. A number of SFARI scientists and staff also attended the event.

The SCN2A family meeting was one of many events that family organizations of rare, neurodevelopmental disorders organized last summer. These meetings help families connect with others similarly affected as well as professionals working to better understand these conditions and develop new therapeutics. SFARI often attends and facilitates research opportunities carried on at these events.

SCN2A is a high-confidence autism risk gene, which encodes a subunit of a sodium channel in the brain called Nav1.2. When the channel malfunctions, conditions like epilepsy and autism follow. As part of its mission to understand the genetics and neurobiological underpinnings of autism, SFARI has awarded about $3 million for research on SCN2A, and some of this research was presented at the meeting. SFARI also supports a genetics first initiative called Simons Searchlight (formerly known as Simons VIP), which enrolls people with a genetic diagnosis showing rare genetic changes associated with autism and related neurodevelopmental conditions, such as SCN2A.

Many stories that may reflect the different ways SCN2A can be disabled were told at the meeting. One child had his first seizure when he was days old, and now spends many of his days irritable and immobilized by dystonia. Another developed normally until his first seizure as a toddler, which seemed to wipe out all of his skills; his milestones are now hard won in the face of continuing seizures and an autism diagnosis. Another had a sudden regression at 1 year of age, and after a misdiagnosis and seizure medication, she goes to a school for children with autism. Still another suffered from relentless seizures, which robbed her of speech; she died last year at the age of 12.

So far, about 300 different variants of the SCN2A gene have beendocumented, and the functional consequences of many are unclear. Some researchers have developed high-throughput experiments to systematically test each of thesevariants, and to screen compounds that could normalize their function2. Another approach may use genetherapy to boostexpression of the remaining good copy of SCN2A. Either way, finding appropriate in vitro testing grounds for these SCN2A variants is essential and may help personalize treatment approaches or identify more homogeneous patient groups for drug trials.

The meeting also underscored the power of family gatherings to push the science ahead. Investigators could see multiple examples of a rare genetic condition and engage new participants in research studies such as The Investigation of Genetic Exome Research (TIGER), a project of the University of Washington that compares phenotypes of single-gene conditions. In turn, families had the opportunity to express their concerns to scientists and infuse the research proceedings with urgency.

My biggest takeaway from this years conference was the mutual inspiration between the scientists and the families, says Leah Schust, meeting organizer and executive director of the FamilieSCN2A Foundation. Her son has a mutation in SCN2A.

Meeting the researchers working on a cure for our kids motivates us to fight on, Schust says. Then the scientists all say that meeting the families inspires them to go back to their labs and work even harder.

Family focus. The family meeting helped researchers reconsider what would be meaningful clinical endpoints for potential treatments. Schust says that most researchers and industry groups had thought seizure control was the most important issue. After listening to us, they realized that quality of life, movement disorders and autonomic dysfunction are higher on our list of where we would like to see improvement, she says.

When SCN2A mutations were first linked to autism, the gene stood out because it encodes a relatively well-understood protein, unlike many of the other identified genes. Nav1.2 is a voltage-gated channel found exclusively on excitatory neurons in the brain, where it controls the flow of sodium ions into the neuron, and thus its propensity for firing action potential. Experiments have revealed detailed pictures of Nav1.2s structure3, and known drugs alter its function4.

SCN2A also stands out because of its high recurrence rate in autism: unlike other autism genes, SCN2A is mutated with somewhat regular frequency5 (Figure 1).

Just as understanding why a car wont start is critical to fixing it, researchers need to understand how these SCN2A mutations alter the Nav1.2 channel. A current model1 posits that some mutations are gain-of-function, rendering the channel too active and the brain hyperexcitable, leading to infantile epilepsy; conversely, loss-of-function mutations reduce excitability and seem associated with autism and/or intellectual disability, as well as childhood-onset (as opposed to neonatal) seizures.

Yet the functional consequences of most SCN2A mutations remain unknown, and some may not fall neatly into a loss-of-function or gain-of-function category. A way of making sense of these mutations may come from looking at the working parts of Nav1.2, said Arthur Campbell of the Broad Institute of MIT and Harvard. For example, missense SCN2A variants linked to epilepsy seem to hit the channel randomly. But when marking their location on a crystal structure model of the channel, the missense variants cluster in several places: on the voltage sensor, on the linker helix responsible for conveying voltage sensor movement to the channel pore, on an area thought to interact with the beta-subunits involved in chaperoning the channel to the right place, and on the inactivation gate, which closes the pore off from sodium ion flow. He suggested that this knowledge, combined with the structural similarities between all sodium channels, may help drug development for SCN2A-related conditions.

High-throughput systems that can assay hundreds of cells at a time are helping researchers systematically explore SCN2A mutation, explained SFARI Investigator Al George of Northwestern University. While conventional electrophysiology would require weeks of work to characterize a single SCN2A variant, Georges group uses an automated patch-clamp system that can characterize multiple variants transfected into non-neuronal cell lines in a day. Using this system, two variants associated with neonatal seizures both exhibited an exceptional willingness to activate and a slowness to inactivate, which are properties consistent with a gain-of-function interpretation.

The high-throughput set up also promises to expedite the hunt for drugs to normalize SCN2A function: George described a 384-well plate design that allows measurement of the effects of two different drugs, at four different concentrations, on the SCN2A variant and control channels simultaneously. A known drug (carbamazepine) and an experimental drug (PRX-330) shifted channel inactivation to more hyperpolarized voltages, which could help quiet channels with gain-of-function mutations.

To narrow in on potentially therapeutic compounds, Jeff Cottrell and colleagues at the Broad Institute of MIT and Harvard have come up with a two-stage screen to find small molecule activators or inhibitors of Nav1.2 channels. First, compounds are initially tested on non-neural cells transfected with Nav1.2 sodium channels and potassium channels, which enables them to spike. The cells in 384-well plates are stimulated in parallel, and voltage-sensitive dyes give a readout of spiking activity; remarkably, Cottrells system allows data collection from up to 96 wells simultaneously. Any compounds that modulate spiking would then be subjected to the second stage, in a high-throughput electrophysiology assay similar to that described by George. Compounds with helpful mechanisms would then be tested for selectivity for Nav1.2 versus other sodium channels. A selective compound would then be tested in neurons, first in vitro then in vivo. This step-wise process has identified an activating compound that makes Nav1.2 more likely to open at rest and has potent effects on action potentials in brain slices and on electroencephalogram (EEG) traces from mice engineered to carry a disabled copy of SCN2A; however, Cottrell said this particular compound is not a therapeutic candidate in part because it broadens the action potential in a way that could promote seizures. A full screen is underway, and so far has identified 378 modulators from a library of 77,000 compounds.

Beyond academia, J.P. Johnson Jr. of Xenon in Burnaby, British Columbia, discussed the companys work to create sodium channel inhibitors for treating epilepsy. To obtain selective compounds, the group targets the voltage-sensing domain because its structure is the most diverse region of sodium channels. Xenon uses a trial-and-error method to optimize sodium channel inhibitor potency and selectivity. The methodical process has yielded some interesting compounds, including both selective Nav1.6 inhibitors and dual Nav1.6 and Nav1.2 inhibitors. Both quashed spiking in mouse excitatory pyramidal neurons, which contain only Nav1.2 and Nav1.6, but they did not alter spiking in Nav1.1-containing inhibitory neurons. A Nav1.6 selective inhibitor, XEN901, is currently undergoing safety trials in humans.

Kathrin Meyer of Nationwide Childrens Hospital in Columbus, Ohio, addressed the possibility of using gene therapy to normalize malfunctioning Nav1.2 channels. Meyer has been involved in several gene-therapy trials for neuromuscular disorders, including a successful one for infant-onset spinal muscular atrophy type6. Gene therapy for brain diseases was spurred by the discovery of adeno-associated virus 9 (AAV9), which can cross the bloodbrain barrier to deliver genetic material to the central nervous system. AAV9 is small, cannot replicate, does not integrate into host DNA and seems not to cause disease in humans. In considering gene therapy for SCN2A-related conditions, Meyer emphasized an approach that adds back a working copy of the gene, thus sidestepping the need for gene editing to make mutation-specific corrections. Such a treatment would only apply to those with loss-of-function mutations.

The large size of the SCN2A gene precludes its delivery by AAV9, however. As a workaround, Meyer suggested that SCN2As mRNA transcript could be targeted in an attempt to replace only the affected area of the mRNA. So far, such strategies have not been very efficient, but there are new ideas that might address some of the difficulties. Because access to tissue samples of patients with neurological disorders is limited, the development and testing of new therapies is complicated. Meyer suggested developing gene therapies in vitro using neurons reprogrammed from skin cells of patients. This might help identify which patients would react best to a certain treatment. There is likely not a one-fit-for-all situation, she said.

SFARI deputy scientific director John Spiro underscored the need for in vitro systems, citing the organizations initiative to bank blood cells to systematically generate induced pluripotent stem cells from individuals with autism. Simons Searchlight is also a resource of many different biospecimens for researchers. So far, 186 families with SCN2A-related changes have registered, and 83 of these have completed consent, lab reports and medical histories with a large number of blood samples as well. (On the sidelines of the meeting, 18 parents, 11 of their children with SCN2A mutations, and three unaffected siblings donated blood toward this initiative.) Spiro also stressed a need to come up with more quantitative methods of phenotyping, such as wearable electronics that can monitor sleep and circadian rhythms. Data that can be collected longitudinally and at home might provide sensitive outcome measures for clinical trials.

A new role for Nav1.2 has been revealed in recent work described by SFARI Investigator Kevin Bender of the University of California, San Francisco: the channels mediate back-propagating action potentials, which travel into the dendritic trees of neurons. Mice engineered to lack one copy of SCN2A a situation that mimics people with truncating SCN2A mutations that render the resulting Nav1.2 channels useless had cortical neurons with slower action potentials, reduced dendritic excitability and immature synapses based on their shape and function7. This role for Nav1.2 was particularly important later in development: when conditional knockout mice lost an SCN2A copy later in life, their cortical neurons exhibited immature synapses, though their density remained normal. Preliminary experiments suggest that adding back a working copy of SCN2A later in life through transgenic methods or by upregulating transcription of the remaining good copy of SCN2A via CRISPR techniques can restore action potential velocity and synaptic maturity.

Bender stressed how interacting with the SCN2A family group helped focus his research on important aspects of their childrens conditions. For example, parents have noted sensory hypersensitivity in their children, leading Bender to collaborate with colleague Evan Feinberg to use an eye-tracking assay in mice to measure their visual responses. He noted that SCN2A haploinsufficient mice were more sensitive to certain visual stimuli than control mice; if the assay is robust, it could help bridge the gap between SCN2A-related phenotypes in humans and behaviors measured in mice.

As meeting attendees sorted through the new findings, therapeutic questions lingered. An important issue for any therapy, whether drug or gene, will be how early in development one will have to intervene to help someone with an SCN2A mutation. Bender noted that synaptic properties could be rescued in his mice when they were 30 days old equivalent to a 10-year-old human but these and other experiments will have to probe the time periods during which therapies will be maximally effective. To find good measures of efficacy also means understanding the full complement of conditions that beset people with SCN2A mutations. For example, though seizures afflict many, Keith Coffman of Childrens Mercy Hospital in Kansas City, Missouri, suggested that, in some cases, these represent a movement disorder rather than epilepsy. Basic descriptive knowledge like this is imperative for guiding future treatment approaches.

Another smaller SCN2A meeting is planned for this year from July 30 to August 2, in Columbus, Ohio. This will be more family focused, says Schust, and there will be opportunities to participate in research.

There is clearly a lot more work to do before all the terrific basic research that was discussed at this meeting produces meaningful results for families, but it is extremely gratifying to see how much progress has been made on so many fronts and how many new good ideas are emerging, Spiro says. And its terrific to witness firsthand the positive cycle of how families drive researchers and vice versa.

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Seeing through a forest of SCN2A gene variation - SFARI News

FEBRUARY 19, 2020 When Lt. Col. Jason Barnhill traveled to Africa last summer, he took with him not only the normal gear of an Army officer, but also a 3D printer.

Barnhill, who is the life science program director at the U.S. Military Academy, traveled to Africa to study how 3D printers could be used for field medical care. Barnhills printer was not set up to print objects made out of plastics as the printers are frequently known for. Instead, his printer makes bioprinted items that could one day be used to save Soldiers injured in combat.

The 3D bioprinting research has not reached the point where a printed organ or meniscus can be implanted into the body, but Barnhill and a team of cadets are working to advance the research in the field.

Twenty-six firsties are doing bioprinting research across seven different projects as their capstone this year. Two teams are working on biobandages for burn and field care. Two teams are working on how to bioengineer blood vessels to enable other bioprinted items that require a blood source, such as organs, to be viable. One team is working on printing a viable meniscus and the final team is working on printing a liver.

The basic process of printing biomaterial is the same as what is used to print a plastic figurine. A model of what will be printed is created on the computer, it is digitally sliced into layers and then the printer builds it layer by layer. The difference is the ink that is used.

Instead of heating plastic, 3D bioprinting uses a bioink that includes collagen, a major part of human tissue, and cells, typically stem cells.

A lot of this has to do with the bioink that we want to use, exactly what material were using as our printer ink, if you will, Class of 2020 Cadet Allen Gong, a life science major working on the meniscus project, said. Once we have that 3D model where we want it, then its just a matter of being able to stack the ink on top of each other properly.

Cadets are researching how to use that ink to create a meniscus to be implanted into a Soldiers injured knee or print a liver that could be used to test medicine and maybe one day eliminate the shortage of transplantable organs.

The research at West Point is funded by the Uniformed Services University of Health Science and is focused on increasing Soldier survivability in the field and treating wounded warriors.

Right now, cadets on each of the teams are in the beginning stages of their research before starting the actual printing process. The first stage includes reading the research already available in their area of focus and learning how to use the printers. After spring break, they will have their first chance to start printing with cells.

For the biobandage, meniscus and liver teams, the goal is to print a tangible product by the end of the semester, though neither the meniscus or liver will be something that could be implanted and used.

There are definitely some leaps before we can get to that point, Class of 2020 Cadet Thatcher Shepard, a life science major working on the meniscus project, said of actually implanting what they print. (We have to) make sure the body doesnt reject the new bioprinted meniscus and also the emplacement. There can be difficulties with that. Right now, were trying to just make a viable meniscus. Then, well look into further research to be able to work on methods of actually placing it into the body.

The blood vessel teams are further away from printing something concrete because the field has so many unanswered questions. Their initial step will be looking at what has already been done in the field and what questions still need to be answered. They will then decide on the scope and direction of their projects. Their research will be key to allowing other areas of the field to move forward, though. Organs such as livers and pancreases have been printed, so far, they can only be produced at the micro level because they have no blood flow.

Its kind of like putting the cart before the horse, Class of 2020 Cadet Michael Deegan, a life science major working on one of the blood vessel projects, said. Youve printed it, great, but whats the point of printing it if its not going to survive inside your body? Being able to work on that fundamental step thats actually going to make these organs viable is what drew me and my teammates to be able to do this.

While the blood vessel, liver and meniscus projects have the potential to impact long-term care, the work being done by the biobandage teams will potentially have direct uses in the field during combat. The goal is to be able to take cells from an injured Soldier, specifically one who suffers burns, and print a bandage with built in biomaterial on it to jumpstart the healing process.

Medics would potentially be deployed with a 3D printer in their Humvee to enable bandages to be printed on site to meet the needs of the specific Soldier and his or her exact wound. The projects are building on existing research on printing sterile bandages and then adding a bioengineering element. The bandages would be printed with specialized skin and stem cells necessary to the healing process, jumpstarting healing faster.

Were researching how the body actually heals from burns, Class of 2020 Cadet Channah Mills, a life science major working on one of the biobandage projects, said. So, what are some things we can do to speed along that process? Introducing a bandage could kickstart that healing process. The faster you start healing, the less scarring and the more likely youre going to recover.

The meniscus team is starting with MRI images of knees and working to build a 3D model of a meniscus, which they will eventually be able to print. Unlike a liver, the meniscus doesnt need a blood flow. It does still have a complex cellular structure, though, and a large part of the teams research will be figuring out how and when to implant those cells into what theyre printing.

Of the 26 cadets working on bioprinting projects, 17 will be attending medical school following graduation from West Point. The research they are doing gives them hands-on experience in a cutting-edge area of the medical field. It also enabled them to play a role in improving the care for Soldiers in the future, which will be their jobs as Army doctors.

Being on the forefront of it and just seeing the potential in bioengineering, its pretty astounding, Gong said. But it has also been sobering just to see how much more complicated it is to 3D print biomaterials than plastic.

The bioprinting projects will be presented during the academys annual Projects Day April 30.

By Brandon OConnor

More:
Cadets Research Bioprinting to Improve Soldier Care - MilitarySpot

Heres the truth about serum: Its the biggest secret to having younger-looking, radiant skin.

You know all the other steps to staying healthyand showing it: wearing SPF, and applying retinol. You stay hydrated and eat healthy foods. You get lots of exercise and plenty of sleep. But, are you applying serums? They can give your skin some of the most immediate and impactful benefits.

Serum seeps into all three layers of your skin to work its magic at deeper levels than moisturizer ever could. Those moisturizing creams merely sit atop your skin. Theyre equally important, and shield skin from toxins, but they do little to reverse dark spots and smooth wrinkles. Serums, on the other hand, can correct hyperpigmentation and puffy eyes.

Serum brightens your skin complexion, and can preserve moisture levels in ways that moisturizers never could (funny, given its name). No two serums are alike in terms of ingredients and overall benefits, but almost all of them brighten your skin and smooth complexion. Here are seven of our favorite serums; apply them to clean, dried skin before moisturizers, SPFs, retinol or night creams.

If your skin suffers from redness or discoloration, then look for a corrective serum. SkinCeuticals gel serum uses thyme and cucumber extract to soothe irritation, as well as mulberry extract to even skin tone and gently hydrate the skin. If most moisturizers make your skin break out, then this is a gentle and top-notch moisturizing alternative.

Vitamin C is one of the best ingredients you can apply to skin for brighter, more even complexion. Glossiers serum is packed with it, along with magnesium, for an instant pick-me-up for tired or dull skin. Use it daily to preserve a fresh face, and to get ahead of the inevitable hangovers or early mornings.

The skin around your eyes is more sensitive than the rest of your face, and is much thinner, too hence why you quickly show signs of tiredness. (Its fluid buildup from a lack of rest, shining through your skin.) So, target the eyes with a special-blend serum that tightens and brightens at the same time. In OSEAs eye serum, hyaluronic acid preserves firmness and moisture levels, grape-seed-derived resveratrol shields this thin skin from toxins, and algae extract plumps and nourishes tired eyes (and alert eyes, if youre plenty rested already everyone benefits). It also prevents and helps reverse fine lines and other signs of aging.

Some serums are best worn during the day those that focus on hydration and pure brightening. Others, like corrective and exfoliating serums, are often best suited for bedtime so that they can sync with your bodys circadian rhythm to boost cellular turnover (and in turn guarantee that you wake up looking rested and more youthful). However, some serums cover both bases: Brickells night serum is ultra-hydrating so much so that its impact is maximized by wearing it overnight. Its loaded with plant stem cells, hyaluronic acid and vitamin C, and promises to magnify the benefits of a full nights sleep. Your friends and colleagues will notice the difference within days. (Assuming you arent pairing it with a lovely binge-drinking or chain-smoking habit.)

Theres an entire family of serums that focuses on exfoliation, by using ingredients that dissolve dead skin cells and reduce hyperpigmentation (dark spots and discoloration). Herbivores serum does so with alpha- and beta-hydroxy acids, all of which are naturally derived, despite their sciencey-sounding names. Theyre extracted from things like willow bark and sugar maple, and in turn leave skin bright, while minimizing pore size and smoothing skin texture.

Its hard to categorize Caudelies skin-brightening serum, since it uses the brands proprietary blend of ingredients (Viniferine, derived from grape vines) but they seem to deliver on their promise to brighten skin 62 times better than Vitamin C. All around the globe, this is what Caudelies spa customers pay top dollar for, and you can enjoy the same complexion-smoothing effects for less than a buck a day (assuming you just use a couple drops daily).

If your skin texture needs attention just as much as your skin tone, then look for a corrective and smoothing serum. This one from PCA Skin is pumped with peptides, and it waxes poetic on your skin to undo signs of aging (like the appearance of fine lines, wrinkles, dark spots and acne spots). It also helps skin retain moisture thanks to the inclusion of hyaluronic acid, rice bran extract, squalene, ensuring firmness and brightness.

See the rest here:
The Best Serums to Brighten and Refresh Dull, Tired Skin - gearpatrol.com

TOKYO and BOTHELL, Wash., Feb. 19, 2020 /PRNewswire/ --Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Seattle Genetics, Inc. (Nasdaq:SGEN) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for PADCEV (enfortumab vedotin-ejfv) in combination with Merck's (known as MSD outside the United States and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting.

The FDA's Breakthrough Therapy process is designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition. Designation is based upon preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

"The FDA's Breakthrough Therapydesignation reflects the encouraging preliminary evidence for the combination of PADCEV and pembrolizumab in previously untreated advanced urothelial cancer to benefit patients who are in need of effective treatment options," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas. "We look forward to continuing our work with the FDA as we progress our clinical development program as quickly as possible."

"This is an important step in our investigation of PADCEV in combination with pembrolizumab as a first-line therapy for patients with advanced urothelial cancer who are unable to receive cisplatin-based chemotherapy," said Roger Dansey, M.D., Chief Medical Officer, Seattle Genetics. "Based on encouraging early clinical activity, we recently initiated a phase 3 trial of this platinum-free combination and look forward to potentially addressing an unmet need for patients."

The Breakthrough Therapy designation was granted based on results from the dose-escalation cohort and expansion cohort A of the phase 1b/2 trial,EV-103 (NCT03288545), evaluating patients with locally advanced or metastatic urothelialcancer who are unable to receive cisplatin-based chemotherapytreated in the first-line setting with PADCEV in combination with pembrolizumab. Initial results from the trial were presented at the European Society of Medical Oncology (ESMO) 2019 Congress, and updated findings at the 2020 Genitourinary Cancers Symposium. EV-103 is an ongoing, multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive, locally advanced and first- and second-line metastatic urothelial cancer.

About Bladder and Urothelial CancerIt is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020.1 Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.2

Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide.3

The recommended first-line treatment for patients withadvanced urothelial cancer is a cisplatin-based chemotherapy. For patients who are unable to receive cisplatin, such as people with kidney impairment, a carboplatin-based regimen is recommended. However, fewer than half of patients respond to carboplatin-based regimens and outcomes are typically poorer compared to cisplatin-based regimens.4

About PADCEV PADCEV (enfortumab vedotin-ejfv) was approved by the U.S. Food and Drug Administration (FDA) in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDA's Accelerated Approval Program based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.5

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.5,6 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).5PADCEV is co-developed by Astellas and Seattle Genetics.

Important Safety Information

Warnings and Precautions

Adverse ReactionsSerious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab AbnormalitiesIn one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased, hemoglobin decreased, phosphate decreased, lipase increased, sodium decreased, glucose increased, urate increased, neutrophils decreased.

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Astellas Astellas Pharma Inc., based in Tokyo, Japan, is a company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. For more information, please visit our website at https://www.astellas.com/en.

About Seattle Genetics Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in people's lives. The company is headquartered in Bothell, Washington, and has offices in California, Switzerland and the European Union. For more information on our robust pipeline, visit http://www.seattlegenetics.com and follow @SeattleGenetics on Twitter.

About the Astellas and Seattle Genetics CollaborationSeattle Genetics and Astellas are co-developing PADCEV (enfortumab vedotin-ejfv) under a collaboration that was entered into in 2007 and expanded in 2009. Under the collaboration, the companies are sharing costs and profits on a 50:50 basis worldwide.

About the Astellas, Seattle Genetics and Merck CollaborationSeattle Genetics and Astellas entered a clinical collaboration agreement with Merck to evaluate the combination of Seattle Genetics' and Astellas' PADCEV (enfortumab vedotin-ejfv) and Merck's KEYTRUDA (pembrolizumab), in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Astellas Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development), which is included in this press release is not intended to constitute an advertisement or medical advice.

Seattle Genetics Forward Looking StatementsCertain statements made in this press release are forward looking, such as those, among others, relating to the development of PADCEV in combination with pembrolizumab as a first-line therapyfor patients with advanced urothelial cancerwho are unable to receive cisplatin-based chemotherapy, and the therapeutic potential of PADCEVincluding its efficacy, safety and therapeutic uses. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the possibility that ongoing and subsequent clinical trials may fail to establish sufficient efficacy, that adverse events or safety signals may occur and that adverse regulatory actions may occur. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption "Risk Factors" included in the company's Annual Report on Form 10-K for the year ended December 31, 2019 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

1American Cancer Society. Cancer Facts & Figures 2020.https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2020/cancer-facts-and-figures-2020.pdf.Accessed 01-23-2020.2American Society of Clinical Oncology. Bladder cancer: introduction (10-2017). https://www.cancer.net/cancer-types/bladder-cancer/introduction. Accessed 05-09-2019.3International Agency for Research on Cancer. Cancer Tomorrow: Bladder.http://gco.iarc.fr/tomorrow 4National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Bladder Cancer. Version 4; July 10, 2019. https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf.5PADCEV [package insert]. Northbrook, IL: Astellas, Inc.6Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.

SOURCE Astellas Pharma Inc.

http://www.seattlegenetics.com

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Astellas and Seattle Genetics Receive FDA Breakthrough Therapy Designation for PADCEV (enfortumab vedotin-ejfv) in Combination with Pembrolizumab in...

New therapeutic approaches for chronic inflammatory diseases such as inflammatory bowel disease, rheumatoid arthritis, and psoriasis are needed because current treatments are often suboptimal in terms of both efficacy and the risks of serious adverse events. Inhibitor of apoptosis proteins (IAPs) are E3 ubiquitin ligases that inhibit cell death pathways and are themselves inhibited by second mitochondria-derived activator of caspases (SMAC). SMAC mimetics (SMs), small-molecule antagonists of IAPs, are being evaluated as cancer therapies in clinical trials. IAPs are also crucial regulators of inflammatory pathways because they influence both the activation of inflammatory genes and the induction of cell death through the receptor-interacting serine-threonine protein kinases (RIPKs), nuclear factor B (NF-B)inducing kinase, and mitogen-activated protein kinases (MAPKs). Furthermore, there is an increasing interest in specifically targeting the substrates of IAP-mediated ubiquitylation, especially RIPK1, RIPK2, and RIPK3, as druggable nodes in inflammation control. Several studies have revealed an anti-inflammatory potential of RIPK inhibitors that either block inflammatory signaling or block the form of inflammatory cell death known as necroptosis. Expanding research on innate immune signaling through pattern recognition receptors that stimulate proinflammatory NF-B and MAPK signaling may further contribute to uncovering the complex molecular roles used by IAPs and downstream RIPKs in inflammatory signaling. This may benefit and guide the development of SMs or selective RIPK inhibitors as anti-inflammatory therapeutics for various chronic inflammatory conditions.

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SMAC mimetics and RIPK inhibitors as therapeutics for chronic inflammatory diseases - Science

I have this really annoying habit which I know I need to work on. Despite the fact that I'm naturally a very early riser (I'm talking, *wakes up at 6:30 a.m. 365 days a yeareven on the weekends* kind of early riser), I pretty much refuse to adjust my bedtime a few hours earlierto counteract the impact of my early bird identity. I love to stay up late after a long day and binge-watch my favorite shows on Hulu, I love to read 100-page chunks of a novel, and without fail, I also love to fool around on my phone doing who even knows what. You know that feeling when you look at the clock and it's a perfectly respectable 10 p.m. and then the next time you blink it's verging on 1 a.m.?! Well, it happens to me all the time, and while I know I'd be the better for shutting down my phone, burning a candle, switching on my diffuser, and being the well-behaved beauty and wellness editor I know I should be, I really struggle to do that. And, yes, it definitely shows up on my face the next day.

I have notoriously shady under-eyes just naturally, (check out our A-list celebrity guide to dark circles, here), but my lack of sleep and consequential coffee dependence the following morning has exacerbated the situation. Luckily, my job allows me access to the newest and coolest eye creams and treatments which tout all sorts of under-eye miraclesfrom the banishment of dark circles to the erasing of puff, fine lines, and wrinkles. Spoiler: most fall flat. As in, that literally did nothing, kind of flat. However, during my 3 years as a beauty editor, I've collecteda glorified possession of creams and gels and balms that truly do work wonders on my dark circles. Regardless of whether your dark circles are sheerly caused by genetics, allergies, or not-so-stellar sleep habits (me), these shadow-targetingeye creams can lend a helping hand. Keep scrolling! I'm sharing the 15 best eye creams for dark circles I've tried since becoming a beauty editor.

Hot off of L'Occitane's French presses (it literally launched two days ago, but I received ahead of time to test-drive!), this serum has stolen my heart. It's the kid sister to the brand's best-sellingOvernight Reset Oil-in-Serum ($59) (one unit is purchased every minute!) and it uses the formula's same coveted moisture magic to do battle against telltale symptoms of fatigue like dark circles, puffiness, and expression lines. Plus, I have to say I love that you can literally see the 1,600 golden bubbles of precious Immortelle Essential Oil sitting in the stunning bottle. So stunning, right?!

Peter Thomas Roth Vital-E Microbiome Age Defense Eye Cream ($55)

Y'all, brands have really been upping the anty and cadence of eye cream launches because this is another formula for dark circles that JUST hit my vanity. (And stores everywhere.) Say goodbye to fine lines and wrinkles, dullness, uneven texture, and dark circles thanks to this protective solution designed to improve the delicate skin barrier around the eyes and support the skin's natural microbiome as well.

iS Clinical Youth Eye Complex ($105)

iS Clinical is one of the top brands chosen by the top estheticians in the industry, and this anti-aging eye cream is one of their choice formulas. Not only is it insanely creamy and hydrating (but not at all sticky or slimy like some eye creams can be!) it's a huge help in the dark circle departmentI'll apply it before bed and then again in the morning to finish off my morning skin routine. My circles are still there (obviously), but they're a lot less glaring. The key is in the mix of hyaluronic acid, peptides, and antioxidants.

111Skin Space Defence Bright Eye Lift Gel NAC Y2 ($190)

Once I found out that makeup artists use this formula from 111Skin to prep celebs' skin pre-red carpet, I was 88.5% on board. And, after a few applications when I started to notice that my under-eyes looked less shadowy and significantly firmer, tighter, and all-around less fatigued, I lept to a full 120%. Categorically, it's an eye cream, but it's formulated as a soothing, lightweight gel anyone who's against heavy, cloying creams will absolutely adore. Plus, there's the whole eye lifting effect which is pretty dang wonderful in and of itself.

EIGHTH DAY Eye Renewal Cream ($225)

If you're down to feel a little tingle (it's worth it, I promise!) this eye cream has some bite. I mean that literally because one of the reasons it quickly tightens, firms, and de-puffs is thanks to the MVP ingredient: snake venom. This has been one of my favorite formulas for a couple of years now, and it's what I reach for when my under-eyes are in a state of emergency.

RVive Skincare Intensit Complete Anti-Aging Eye Serum ($285)

Not only does the featherlight serum texture feel great underneath your eyes, but this favorite eye cream also features strategic brightening agents to help diffuse any purple circles that like to lurk. (You'll get what I mean once you see the gorgeous pearly finish.) It also looks AMAZING under makeup and concealer. It's a 10/10 if you can stomach the splurge.

By Terry Hydra-clat Eye Contour ($75)

If you have a weakness for anything lovely, French, and downright effective when it comes to your beauty product investments, you should definitely consider eye contour-improving cream from By Terry. Not only does application feel cooling and luxe thanks to the silver applicator, but it also hosts delicate ingredients like Black Rose, plant stem cells, and vitamin E to not ONLY reduce dark circles but to de-puff and soften the look of any fine lines and wrinkles. This is another one that thanks to its lightweight texture is superb under your foundation and/or concealer.

Sunday Riley Auto Correct Brightening and Depuffing Eye Contour Cream ($65)

One of my favorite eye creams of all time, this is probably the one I recommend to friends most often since it's a bit more affordable and so instantaneously affect. One swipe across your under-eyes and the mica-, caffeine-, and antioxidant-rich potion practically blurs away the look of lingering darkness. I honestly think this is Sunday Riley's absolute best product. But, that's just me.

La Prairie Skin Caviar Eye Lift Serum ($485)

Now, I wouldn't include a nearly $500 eye cream if it didn't work true sorcery against dark circles now would I? The sticker shock might be a bit gut-wrenching but this is probably THE most luxurious option for dark circles on the market. Plus, if you're consistent with use, the combination of La Prairie's iconic encapsulated caviar beads and an innovative gel-cream emulsion is probably just as effective as a pricey in-office treatment. The effect is a visual lift to the eye and brow area, less puffiness, lines, shadows, you get the idea.

Ole Henriksen Banana Bright Eye Crme ($39)

If you have dark circles, the yellow tint to this brightening elixir can definitely help with that. It's rich in vitamin C, it's anti-aging, and it even makes your concealer and foundation apply smoother. Wins all-around!

GlamGlow BRIGHTEYES Illuminating Anti-Fatigue Eye Cream ($39)

For glowing skin and under-eyes that mirror a full ten hours of sleep, try GlamGlow's new formula. It features illuminating spheres to brighten up dullness and darkness, caffeine to tighten and lift, hyaluronic acid to plump and moisturize, and last but not least peptides to diminish the appearance of fine lines and wrinkles.

Tula Skincare Glow & Get It Cooling & Brightening Eye Balm ($28)

Technically, this circle-eraser is a balm and not a cream, but please bear with meit works! Like, so, so good. You know those mornings when for whatever reason you wake up feeling like you've been hit by a freight train? Sweeping this cooling, aloe-, and caffeine-infused stick all around your eye area feels almost as good as a deep tissue marathon post-Barry's Bootcamp. (Oh, and there's also a radiance-enhancingRose Glow version FYI!)

SkinMedica Instant Bright Eye Cream ($88)

Did you know Hailey Bieber (and a slew of other celebs) is a huge fan of this brand? I don't know if miss HB has tried this brightening eye cream from SkinMedica, but if not, I personally recommend she get on that. It's brimming with tons of great ingredients like phenylethyl resorcinol (which helps to support melanin balance and correct hyperpigmentation), a hyaluronic acid blend for hydration, marine extracts to supporta healthy skin barrier, plus all the other good stuff for blue and purple under-eyes like caffeine, Persian silk tree extract, and St. Pauls Wort which aidvascular and lymphatic function for a firmer, more radiant result.

Versed Zero-G Smoothing Eye Cream ($18)

My 73-year-old mom and I agreethis affordably priced eye cream from clean skincare brand Versed, is seriously spectacular. I immediately noticed how it diminished the severity of my dark circles post-flight when I visited home recently, and my mom agreed that it reduced her puff, wrinkles, so on and so forth.

Belif Moisturizing Eye Bomb ($48)

This eye cream isn't actually touted as an eye cream for dark circles. That said, it's one of the most deeply hydrating formulas I've ever tested and due that insane moisture, I notice that my under-eys look less dried and skeletal and brighter, dewier, and plumper instead. It's a lightweight, very soothing formula that boasts a whopping 26-hour span of hydration endurance. It's one to throw in your basket the next time you find yourself wandering the aisles of Sephora.

Up next,How to Get Rid of Dark Circles Like an A-List Esthetician

This article originally appeared on Who What Wear

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I Average 5 Hours of Sleep, But No One Knows Thanks to These 15 Eye Creams - Yahoo Lifestyle