Archive for September, 2019

Dr. Freda Miller, a senior scientist at the Hospital for Sick Children, has discovered that stem cells found in adult skin can produce nervous system Schwann cells.Fred Thornhill / Toronto Sun

A new elementary school in the citys southwest is being named after a scientist who is an alumna of the Calgary public school system.

The school that is under construction in the community of Evergreen is called Dr. Freda Miller School. The Board of Trustees of the Calgary Board of Education approved the name for the school, which is expected to open in September 2020.

Miller is a cell and molecular developmental neurobiologist at Torontos Hospital for Sick Children and a professor at the University of Toronto. She is also a fellow of theRoyal Society of Canada and the American Association for the Advancement of Science.

Miller has made seminal scientific discoveries over the course of her career, the Calgary Board of Education said in a news release Monday.

Her discovery of stem cells in the second layer of the skin has provided the conceptual basis for using skin as a major source for genesis of human stem cells. The stem cells she discovered are critical for the repair of injured skin, the release stated.

At the same time, Dr. Miller discovered new mechanisms determining whether nerve cells live or die, findings that initiated new fields of research and that have major implications for our understanding of neurodegenerative disorders.

Miller attended schools in Montgomery and Bowness and was part of the gifted program at Queen Elizabeth High School in her junior high years. She has a PhD in medical sciences from the University of Calgary.

The school board said Miller maintains strong ties to Calgary and lives part time in Canmore.

When Dr. Freda Miller School opens, it will serve students from kindergarten to Grade 4.

The rest is here:
New elementary school in Calgary named after scientist - Calgary Herald

Currently, there is no licensed treatment to slow or stop the progression of Parkinsons disease. However, a team at Sheffield University in the UK are currently working to identify compounds that target the dopaminergic brain cells affected by the disease. Nikki Withers speaks to Dr Heather Mortiboys to hear how their work could potentially slow disease progression.

Presenting as the second most common neurodegenerative condition after Alzheimers, Parkinsons disease affects 148,000 people in the UK alone. Most people present to their doctor with movement symptoms, such as a tremor, which is a classical symptom and what people often associate with Parkinsons, explains Dr Heather Mortiboys, from the University of Sheffields Institute for Translational Neuroscience (SITraN) and the Universitys new Neuroscience Institute. However, there are other symptoms that occur prior to the movement symptoms, such as loss of sense of smell, autonomic disturbances such as constipation and disordered sleep, particularly in REM sleep.

Most patients with Parkinsons are termed sporadic or idiopathic; meaning the cause of the disease is unknown. Only about 10 percent of patients develop Parkinsons through a known gene mutation, although some genetic association studies have identified several genes that might increase a persons risk of developing Parkinsons, according to Dr Mortiboys, who adds that this does not mean that the person will develop the disease.

Dr Mortiboys has been working in Parkinsons research for over 18 years and currently holds several senior positions, including Senior Research Fellow for Parkinsons UK. When asked about the aetiology of the disease, she explains: We know the type of brain cells that are lost in Parkinsons. They are in a specific brain region the midbrain and they contain the neurotransmitter dopamine. However, we dont know why these cells fail to function properly.

The team used a reprogramming technique utilising the patients skin cells to generate induced neuronal progenitor cells

Dr Mortiboys main research focus of late has been understanding what causes these dopaminergic cells to die in Parkinsons patients. If we can identify what is happening at the cellular level, then we can try to target them with drug therapy to stop them dying, or at least slow it down, she explains. Her team has focused their research on the cells powerhouse the mitochondria. Dopaminergic brain cells are heavily reliant upon their mitochondria. We know that in people with Parkinsons, the mitochondria dont function properly, but we dont really understand the reason why. We also dont know whether we can increase that function and if so, whether that will stop or slow the brain cells from dying.

To study the function of the mitochondria in patients with Parkinsons, the team took skin biopsies from the forearms of patients and cultured the cells in the laboratory. We took a large number of skin cells from a variety of different people with Parkinsons; some with a genetic type that causes early-onset Parkinsons, some with a genetic type that causes later-onset Parkinsons and then the largest group of patients was those with an unknown cause, explains Dr Mortiboys. Once the skin cells had been obtained, the researchers were able to investigate how well the mitochondria, and the associated pathways, functioned. As expected, we found that in all the patients, the mitochondria were not functioning properly in their skin cells. We also noticed that there were problems with the cells recycling pathways.

The next stage of their project was to identify a compound that would have a beneficial effect on boosting mitochondrial and lysosomal function. We started off with all of the drugs that are currently in clinical use, which was around 5,000, says Dr Mortiboys, noting that when developing a drug for neurodegenerative conditions, it needs to pass through the blood-brain barrier. Instead of screening all 5,000 drugs in the laboratory, we performed in silico computer screening to rank the compounds in their ability to pass through the blood-brain barrier.

Once the initial computer-based screening had been performed, the team ran a secondary screen to rank the compounds on their clinical characteristics. We looked at their known side effects, metabolism and dose range, says Dr Mortiboys. We ended up with a rank list of all the clinically in-use compounds and then screened the top ranked of these compounds in the patients cells for their beneficial effect on the mitochondria in the skin cell.

Since their initial work focused on patients skin cells, the team needed to validate their findings in dopaminergic brain cells, which are lost in Parkinsons. This can be particularly challenging because we cant easily take a brain biopsy from a patient, says Dr Mortiboys. The team therefore used a reprogramming technique utilising the patients skin cells to generate induced neuronal progenitor cells. We used a slightly modified protocol, which doesnt take the cells all the way back to being stem cells, explains Dr Mortiboys. Our method takes them to an intermediate, which can only become brain cell types. Crucially for us, it doesnt take them back to the embryonic state. The reason for this is that age is one of the biggest risk factors for Parkinsons and many other neurodegenerative conditions. We didnt want to wipe all the age-associated changes in the cell; so, with this reprogramming technique, we retained the changes that had happened throughout the cells lifetime while still producing a high percentage of dopaminergic cells.

Once these cells had been cultured, the team studied their mitochondrial function and observed that they were far more defective in the patients brain cells than in their skin cells. This showed us that it did matter which cells we were looking at it really was a problem with the mitochondria in the dopaminergic brain cells, explained Dr Mortiboys.

Building on their previous work, the team then looked at the beneficial effects of the previously identified compounds on the dopaminergic brain cells. We had identified several compounds that boosted mitochondrial function in the skin cells and we wanted to see their effects on the brain cells. For example, would they stop them dying off? Are they healthier when they have been treated with the compound? Do their neuronal features change?

Dr Mortiboys explains that they were able to identify compounds that had a beneficial effect, and this will be the focus of their research over the next year. With funding through the Virtual Biotech Programme the drug development arm of charity Parkinsons UK our aim is to take several of these lead compounds and chemically modify them. Well then test these chemicallymodified compounds in the dopaminergic patientderived system.

The aim is for the team to identify a lead molecule that retains mitochondrial function and can be progressed through the drug discovery pipeline.

As with all drug discovery research, there are several challenges the team must address. I think, going forward, the challenge for this project will be to fine tune the chemical lead compounds to dial up the beneficial effects and dial out the less desirable effects. Then, of course, there is the challenge of being able to test something in the laboratory and have confidence that it will translate into a beneficial effect in the clinic, says Dr Mortiboys.

Commenting on Dr Mortiboys research, Richard Morphy, Drug Discovery Manager at Parkinsons UK, said: This is an exciting new approach that could rescue defective mitochondria inside neurons to prevent dysfunction and degeneration of dopamineproducing brain cells. We hope the project will identify a superior group of molecules that could one day deliver a life-changing drug for people living with the condition.

The main symptoms of Parkinsons disease are:

A person with Parkinsons disease can also experience a wide range of other physical and psychological symptoms. These include: depression and anxiety, balance problems, loss of sense of smell (anosmia), problems sleeping (insomnia), memory problems.

Parkinsons disease is caused by a loss of nerve cells in part of the brain called the substantia nigra, which leads to a reduction of dopamine in the brain. Dopamine plays a vital role in regulating the movement of the body. The exact cause of the loss of nerve cells is unclear. Most researchers think that a combination of genetic and environmental factors is responsible.

Parkinsons disease affects around one in 500 people. Most people with Parkinsons start to develop symptoms when theyre over 50, although around one in 20 people with the condition first present with symptoms when theyre under 40. Men are slightly more likely to get Parkinsons disease than women.

Dr Heather Mortiboys gained her PhD from the International Max Planck PhD Program in Dresden, Germany in 2006. She then worked in the Neurology department at the University Hospital Dresden as a research associate on an EU funded project investigating coenzyme Q deficiency in patient tissue. Heather joined the Neuroscience department at the University of Sheffield to set up mitochondrial investigations in models of Parkinsons disease working as a postdoctoral research associate with Professor Oliver Bandmann. She recently became a Parkinsons UK Senior Research Fellow based within the Sheffield Institute for Translational Neuroscience (SITraN) to continue and expand this work setting up her own laboratory.

Original post:
Slowing progression in Parkinson's - avenues to explore - Drug Target Review

Since its launch in 2014, MONAT has taken the beauty world by storm, launching vegan, efficacious hair products and developing a massive global fanbase. Today, the brand is launching its highly anticipated skin-care collection, which has had 1 million VIP customers eagerly awaiting its arrival. Luckily for me, I was able to get my hands on the products early, and the complexion-perfecting formulas did not disappoint! Here's the scoop.

You May Also Like: Everything to Know About Stem Cells in Your Skin Care

Designed for busy people who need to glow on the go, the products are split into two collections: Be Gentle (Creamy Cleanser and Nourishing Moisturizer for dry and sensitive skin types)and Be Balanced (Foamy Cleanser and Lightweight Moisturizer for normal and combination skin types). Both duos are paired with the Skin Revitalizing Essence ($45) and Rewind Age Control Nectar ($120).There are also two special treatmentsa Berry Refined Scrub ($58), which is my personal favorite in the roundup that also doubles as a mask, and Eye Smooth ($75), which is literally one of the smoothest eye creams I've ever tried. A common thread throughout the line is the dreamy textures: think whipped butters and silky creams that make tedious skin-care routines feel luxurious.

And the creation of two collections adds that extra thoughtful touch we all look for these days. "How a product absorbs into the skin varies based upon an individualsskin type. Therefore, different skin types need different care," says Marlene Garcia, licensed medical aesthetician and the brand's senior manager of skin care education. "We formulated theMONAT Skincare line taking into consideration the science behind productabsorption into the skin because absorption plays an important role in how wellproducts can help optimize skin texture and elasticity while the minimizing theappearance of fine lines, wrinkles and age spots.

One big claim to fame for MONAT is that its formulas are "clean," and these new skin-care products are no exception. "MONAT literally means Modern Nature," says Jamie Ross, senior vice president of technical services and the brand's head of R&D."We use ingredients that arenaturally based, safe, pure and sustainablethe best nature has to offerbut we combine them into unique complexes that make thesenaturally based ingredients work in harmony with each other to pump up their existing properties. This is what takes MONATformulations to the next level. Weare also a global brand, and our skin care meets the rigorous cleaningredient standards of the EU. We have a go-to list of potentiallytoxic ingredients we will never use, and we're also cruelty-free and vegan. Aboveall, anything we bring to market must perform. Its one thing to be clean, but another to be clean andachieve the highest performance possible."

You May Also Like: How Easy Is It to Make the Switch to 'Cleaner' Living?

In regards to performance, one of MONAT's cult-favorite productsREJUVENIQE Oil Intensiveinspired its skin care. "We are known for our intensive hair oil, which has a base of Abyssinian oil and an invigorating, proprietary blend of 13 natural plant extracts and essential oils that are rich in omega fatty acids, antioxidants and nutrients," says Alan Meyers, the brand's chief science officer."The products primary role is a hair oil, but we had thousands of customers using it on their skin, too. When we officially launched into skin care, we took this same REJUVENIQE blend and carried it into our star products, including Rewind Age Control Nectar($120)."

Fans had been requesting skin care from the brand for years, but MONAT wanted the formulas to bring something new to the market rather than launching a line because everyone else was. That's one reason why brand presidentStuart MacMillan stressed the importance of doing clinical trials (a step many brands don't take due to the expense and time required). "We wanted to be naturally based AND efficacious," he says. "We wanted to be a disruptorwith key elements that made us unique. As such, the credibility of the productwas essential to us. Clinical trials reinforce the efficacy of our products andprovide credibility and confidence for our distributors." The results of such trials revealed 24 hours of skin hydration, and after14 days, 100 percent of users saw improvements to their skinsbalance, appearance and texture, and a more even skin tone.

You May Also Like: 22 Beauty Gifts Dermatologists Give Their Friends and Family

My personal favorite from the line is the Berry Refined Scrub, which smells like a Capri Sun, feels super gentle on my skin but is powerful enough to exfoliate away grime, and leaves my skin feeling SO soft. It also contains the brand's signatureREJUVENIQE blend, and it'sthe perfect skin prep for the Skin Revitalizing Essence, which has asubtle peach scent and watery texture that sinks right in.

You can buy the two collections (four products each) as sets for $278, or pick and choose, as each product is sold separately as well. Which one are you excited to try first?

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MONAT Just Launched a Skin-Care Line and It's Everything You Hoped For - NewBeauty Magazine

Add this to your skin care kit

We're fortunate to be in an age where there's a solution for every skin problem that exists. Under the massive umbrella that is skin aging, there are many concerns and so many proposed solutions that it can befuddle the mind. We suggest you start off with an anti-aging serum. Serums contain higher proportion of active ingredients and penetrate deeper into the skin, which means better chances to improve texture, reduce lines and better elasticity. These 9 anti-aging serums are ideal for those with mature skin to target concerns of wrinkles, fine lines, age spots and sagging.

(Also Read:7 Sheet Masks For Every Anti-Aging Skin Concern)

The Plum Bright Years Age Specialist Cell Renewal Serum is powered by plant stem cell extracts that promote renewal, fight wrinkles and tighten skin in a 100% vegan and cruelty-free formula that comes in recyclable packaging.

The Biotique Bio Dandelion Visibly Ageless Serum has a preservative free blend of pure dandelion and nutmeg oil which are rich in vitamin E and minerals to brighten its tone, reduce spots as well as wrinkles.

The L'Oreal Paris Revitalift Laser X3 Renewing Anti-Aging Serum is inspired by dermatological procedures with concentrated actives like 3% Pro-Xylane and Adenosine. They work rapidly to provide triple action by smoothening skin, refining its texture and reducing wrinkles and lines.

The Lotus Professional Phyto-RX Intenstive Repair Anti-Aging Serum boosts production of collagen which lends skin with firmness and elasticity.

The Olay Regenerist Micro-Sculpting Serum is formulated with hyaluronic acid pentapeptides which penetrates the surface to regenerate cells from within which makes the face firmer and more lifted.

The Dot & Key Time Reverse Retinol Serum has peptide technology without parabens, sulphates or alcohol to minimize lines, fade spots and tighten skin.

The VLCC Vitalift Serum contains comfrey and soy to tighten skin and fade wrinkles in all skin types.

The La Roche-Posay Hyalu B5 Serum targets skin that is riddled with wrinkles, fine lines and sagginess. With hyaluronic acid and vitamin B5 as its heroes, it replenishes the skin's moisture barrier and activates renewal of cells.

The Sage Apothecary Face Serum has a host of ingredients like vitamin C and E, goji berry and hyaluronic acid which firms skin, reduces lines and shrinks pores.

Shop for similar serums here.

Add this to your skin care kit.

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9 Anti-Aging Serums That Will Turn Back The Clock On Your Skin - NDTV

By TNH Staff September 20, 2019

(NASA, ESA, S. Beckwith (STScI), HUDF Team via AP)

A 31-year-old Greek woman who has racked up international acclaim as a renowned scientist isnt one, academics said, and the US space agency NASA said she never worked there as she claimed.

Eleni Antoniadou, 31, has won praise and awards around the world for her supposed work in a wide range of fields, including regenerative medicine, artificial organ bioengineering and space medicine at NASA.

The British newspaper The Telegraph said it was told by NASA she had not been on the staff there and university professors also disputed her assertions, undercutting her frequent appearances n the media and her claims for international achievements and as she was just presented an achievement award by Greek Education Minister Niki Kerameus who said, Her passion for science inspires us and fills us with optimism.

A Facebook post by Costas Bouyioukos, assistant professor of bioinformatics at Paris Diderot University in France that went viral went even further in discrediting her as he said She is not even fit to be called a scientist for most people.

Bouyioukos said she only and only completed the space agencys Frontier Development Lab, an eight-week educational program.

Antoniadou, the inspiration for Greeces first Barbie doll, has been described as a specialist in the fields of regenerative medicine, artificial organ bioengineering and space medicine, as well as training astronauts at NASA, and working as CEO of Transplants Without Donors, which creates artificial organs for transplants, the paper said.

She has been called a Greek scientist of global calibre by Greek media and was voted 2013 Woman of the Year at the annual British FDM Everywoman in Technology Awards, winning the NASA-ESA Outstanding Researcher Award in 2012 which doesnt appear to be real and presiding over the European Health Parliament.

Greek Hoaxes, a team which debunks fake news, also dismissed her claim to have worked on a team that built the first trachea implant to be successfully used on a patient at University College London, saying the patient died afterwards, the paper reported.

She issued a statement on Facebook saying she was working on a project on artificial intelligence for NASA but would not comment to the paper while a spokesperson for the agency said she was not an employee there but couldnt say if she had worked as a sub-contractor on projects.

The National Herald earlier wrote of her achievements as well, saying that he had said of her motivation: Love another person, even when they lose themselves, when their hygiene is failing, when they dont eat, when they dont care if they are in the light or the darkness. When they have given up and you want to give them a kick and put them to bed. Love, even when youre not sure its worth it. Admire them, even if they look at you without actually seeing you.

She had said she was a researcher in the interdisciplinary fields of regenerative medicine and bioastronautics, specializing in the regeneration of artificial organs from stem cells as an alternative therapeutic pathway for transplants and worked on the creation of cerebral implants, artificial skin, muscles, ears, nerves and the esophagus.

She said she designed a series of bioreactors and tissue engineering tools and has created the worlds first amniotic fluid stem cell bank, conducted experimental studies on the development of bio-nanotubes as drug carriers for targeted cancer therapies, as well as clinical trials for stem cell therapies for lung cancer.

Antoniadou also said she was on the Advisory Committee of the Research and Analysis Organization, DIANEOSIS, in Greece and had been honored by the European Patent Organization in Germany but there were no reports in the wake of others challenging her background whether any of what she said she had done was true.

Read the original here:
Greek Woman's Claim to Be Scientist Refuted by NASA - The National Herald

MedicalResearch.com Interview with:

Dr. Ke Cheng, PhDProfessor, Department of Molecular Biomedical Sciences, NCSUProfessor, UNC/NCSU joint Department of Biomedical Engineering

MedicalResearch.com: What is the background for this study? What are exosomes?

Response: People are developing lots cosmetic products to keep a healthy and young appearance, like antioxidants, growth factors, peptides and more recently, stem cell products. Also, people are seeking more effective solutions for better absorption, like lotion, mask, laser and fillers. What is used for the treatment and how to deliver it are vitally important to the final effect and lasting time.

Exosomes are nano-sized small vehicles containing proteins, nucleic acids, and they are messengers for cell communication and regulation. Here we use skin cell-secreted exosomes to fight skin aging.

MedicalResearch.com: What are the main findings?

Response: We are proposing a cell-free, needle-free treatment to treat aged skin. Exosomes-treated skin was significantly refined and improved compared to commercial retinoids. Whats more, needle-free jet injector is excellent for the delivery of exosomes. The exosomes can reach deep dermis and still maintain their biological activity.

MedicalResearch.com: What should readers take away from your report?

Response: Exosomes play an important role in cell-free treatment and clinical translation. I think skin cell-derived exosomes hold great potential in rejuvenating skin cells, also, they can penetrate dermis well via jet injection methods. For lotions and mask, bioactive factors are hard to penetrate through stratum corneum, you must apply them daily to keep skin hydrated and smooth. This needle-free injection could achieve a long-lasting effect.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Cells are communicating with each other by secreting messengers. It is important to realize the messengers could be different when cells are at different state, like 2D culture, 3D spheroids, treated with drugs and cocultured with different types of cells.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: The limitation of this study is the lack of immunological study. Even though it is cell-free treatment, we would like to do more assessment in the future.

Citation:

Needle-Free Injection of Exosomes Derived from Human Dermal Fibroblast Spheroids Ameliorates Skin Photoaging. Shiqi Hu, Zhenhua Li, Jhon Cores, Ke Huang, Teng Su, Phuong-Uyen Dinh, Ke Cheng*.

ACS Nano. 26 Aug 2019.https://doi.org/10.1021/acsnano.9b04384

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Last Modified: Sep 19, 2019 @ 6:21 pm

The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.

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Needle-free injection of exosomes enable skin repair after UVB damage - MedicalResearch.com

Stem cells are biological cells which have the ability to distinguish into specialized cells, which are capable of cell division through mitosis. Amniotic fluid stem cells are a collective mixture of stem cells obtained from amniotic tissues and fluid. Amniotic fluid is clear, slightly yellowish liquid which surrounds the fetus during pregnancy and is discarded as medical waste during caesarean section deliveries. Amniotic fluid is a source of valuable biological material which includes stem cells which can be potentially used in cell therapy and regenerative therapies. Amniotic fluid stem cells can be developed into a different type of tissues such as cartilage, skin, cardiac nerves, bone, and muscles. Amniotic fluid stem cells are able to find the damaged joint caused by rheumatoid arthritis and differentiate tissues which are damaged. Medical conditions where no drug is able to lessen the symptoms and begin the healing process are the major target for amniotic fluid stem cell therapy. Amniotic fluid stem cells therapy is a solution to those patients who do not want to undergo surgery. Amniotic fluid has a high concentration of stem cells, cytokines, proteins and other important components. Amniotic fluid stem cell therapy is safe and effective treatment which contain growth factor helps to stimulate tissue growth, naturally reduce inflammation. Amniotic fluid also contains hyaluronic acid which acts as a lubricant and promotes cartilage growth.

With increasing technological advancement in the healthcare, amniotic fluid stem cell therapy has more advantage over the other therapy. Amniotic fluid stem cell therapy eliminates the chances of surgery and organs are regenerated, without causing any damage. These are some of the factors driving the growth of amniotic fluid stem cell therapy market over the forecast period. Increasing prevalence of chronic diseases which can be treated with the amniotic fluid stem cell therapy propel the market growth for amniotic fluid stem cell therapy, globally. Increasing funding by the government in research and development of stem cell therapy may drive the amniotic fluid stem cell therapy market growth. But, high procedure cost, difficulties in collecting the amniotic fluid and lack of reimbursement policies hinder the growth of amniotic fluid stem cell therapy market.

Get Sample Copy of this report at https://www.persistencemarketresearch.com/samples/23101?source=atm

The global amniotic fluid stem cell therapy market is segmented on basis of treatment, application, end user and geography: Segmentation by Treatment Allogeneic Amniotic Fluid stem cell therapy Autologous Amniotic Fluid stem cell therapy Segmentation by Application Regenerative medicines Skin Orthopedics Oncology Fetal tissue reconstruction Kidney regeneration Regeneration of neural tissue Cardiac regeneration Lung epithelial regeneration Others Drug research and development Segmentation by End User Hospital Ambulatory Surgical Centers Specialty Clinics Academic and Research Institutes Segmentation by Geography North America Latin America Europe Asia-Pacific Excluding China China Middle East & Africa

Rapid technological advancement in healthcare, and favorable results of the amniotic fluid stem cells therapy will increase the market for amniotic fluid stem cell therapy over the forecast period. Increasing public-private investment for stem cells in managing disease and improving healthcare infrastructure are expected to propel the growth of the amniotic fluid stem cell therapy market.

However, on the basis of geography, global Amniotic Fluid Stem Cell Therapy Market is segmented into six key regions viz. North America, Latin America, Europe, Asia Pacific Excluding China, China and Middle East & Africa. North America captured the largest shares in global Amniotic Fluid Stem Cell Therapy Market and is projected to continue over the forecast period owing to technological advancement in the healthcare and growing awareness among the population towards the new research and development in the stem cell therapy. Europe is expected to account for the second largest revenue share in the amniotic fluid stem cell therapy market. The Asia Pacific is anticipated to have rapid growth in near future owing to increasing healthcare set up and improving healthcare expenditure. Latin America and the Middle East and Africa account for slow growth in the market of amniotic fluid stem cell therapy due to lack of medical facilities and technical knowledge.

Request Report Methodology at https://www.persistencemarketresearch.com/methodology/23101?source=atm

Some of the key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

The report covers exhaustive analysis on: Amniotic Fluid Stem Cell Therapy Market Segments Amniotic Fluid Stem Cell Therapy Market Dynamics Historical Actual Market Size, 2012 2016 Amniotic Fluid Stem Cell Therapy Market Size & Forecast 2016 to 2024 Amniotic Fluid Stem Cell Therapy Market Current Trends/Issues/Challenges Competition & Companies involved Amniotic Fluid Stem Cell Therapy Market Drivers and Restraints

Regional analysis includes North America Latin America Europe Asia Pacific Excluding China China The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

Request to view TOC at https://www.persistencemarketresearch.com/toc/23101?source=atm

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Amniotic Fluid Stem Cell Therapy Market New Growth Opportunities By2018 2026 - My Health Reporter

The Kapamilya stars were among the first to try Belo Medical Group's latest facial treatment, the Salt Facial.

Leila Alcasid, as part of her preparations for the recently held ABS-CBN Ball 2019, had it at the Belo Beauty Suite.

Did you know she went to the ball without any make-up foundation right after she got the treatment?

Continue reading below

Other Kapamilya stars that tried this treatment before the ball were Piolo Pascual, Enrique Gil, Sue Ramirez, Heaven Peralejo, Mccoy De Leon, Barbie Imperial, and Daniella Stranner.

This three-step Salt Facial that helps "restore, replenish, and rejuvenate" the skin in an hour or less.

Dra. Vicki Belo said this treatment is perfect for those who have oily, acne-prone skin.

Continue reading below

It also has great results on minimizing wrinkles and even erasing stretch marks.

She said, "It goes to the dermal level. It uses salt, which has so many benefits for the skin, and we're bringing that machine. It's a brand new machine that we're bringing in, and we've seen amazing results for stretch marks."

Dra. Vicki and her team particularty timed the introduction of the treatment with the ABS-CBN Ball precisely to demonstrate to the stars its immediate results without skin irritation or downtime.

We chose it for the ball because, of course, we have to have calm skin for the night that will catch the light so it will reflect back and they will keep glowing.

Continue reading below

The treatment actually starts with the aesthetician cleaning the face with a cold facial cleanser that is very soothing to the skin.

This is followed by the natural sea salt exfoliation that removes dead skin cells.

Sea salt is a natural product that has anti-bacterial and anti-microbial properties, which kill pimple-causing bacteria.

The process uses a wand-like tool connected to the exfoliating machine that uses closed-loop pressure with microfine medical-grade sea salt.

The result is tighter and healthier skin that better absorbs skincare products.

Continue reading below

After this, a Mandelic or Glycolic acid will be applied on the face, depending on the client's needs. This stem cell-rich gel are massaged and infused on the skin using ultrasound technology.

The last part is the LED light therapy, which is adjusted according to the condition of the skin.

Continue reading below

Blue light is used to minimize inflammation and kill acne-causing bacteria. Meanwhile, red light promotes collagen production and also helps promote healing.

Dra. Vicki said that it also comes in green for pigmentation.

Immediate results can be noticed after the treatment. The skin feels softer, and glowing in an instant.

The Salf Facial will soon be available at Belo Clinics nationwide.

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Leila Alcasid's facial can dry acne fast in just one session - Philippine Entertainment Portal

Stem cells are biological cells which have the ability to distinguish into specialized cells, which are capable of cell division through mitosis. Amniotic fluid stem cells are a collective mixture of stem cells obtained from amniotic tissues and fluid. Amniotic fluid is clear, slightly yellowish liquid which surrounds the fetus during pregnancy and is discarded as medical waste during caesarean section deliveries. Amniotic fluid is a source of valuable biological material which includes stem cells which can be potentially used in cell therapy and regenerative therapies. Amniotic fluid stem cells can be developed into a different type of tissues such as cartilage, skin, cardiac nerves, bone, and muscles. Amniotic fluid stem cells are able to find the damaged joint caused by rheumatoid arthritis and differentiate tissues which are damaged. Medical conditions where no drug is able to lessen the symptoms and begin the healing process are the major target for amniotic fluid stem cell therapy. Amniotic fluid stem cells therapy is a solution to those patients who do not want to undergo surgery. Amniotic fluid has a high concentration of stem cells, cytokines, proteins and other important components. Amniotic fluid stem cell therapy is safe and effective treatment which contain growth factor helps to stimulate tissue growth, naturally reduce inflammation. Amniotic fluid also contains hyaluronic acid which acts as a lubricant and promotes cartilage growth.

With increasing technological advancement in the healthcare, amniotic fluid stem cell therapy has more advantage over the other therapy. Amniotic fluid stem cell therapy eliminates the chances of surgery and organs are regenerated, without causing any damage. These are some of the factors driving the growth of amniotic fluid stem cell therapy market over the forecast period. Increasing prevalence of chronic diseases which can be treated with the amniotic fluid stem cell therapy propel the market growth for amniotic fluid stem cell therapy, globally. Increasing funding by the government in research and development of stem cell therapy may drive the amniotic fluid stem cell therapy market growth. But, high procedure cost, difficulties in collecting the amniotic fluid and lack of reimbursement policies hinder the growth of amniotic fluid stem cell therapy market.

Get Full Report Overview at https://www.persistencemarketresearch.com/market-research/amniotic-fluid-stem-cell-therapy-market.asp

The global amniotic fluid stem cell therapy market is segmented on basis of treatment, application, end user and geography: Segmentation by Treatment Allogeneic Amniotic Fluid stem cell therapy Autologous Amniotic Fluid stem cell therapy Segmentation by Application Regenerative medicines Skin Orthopedics Oncology Fetal tissue reconstruction Kidney regeneration Regeneration of neural tissue Cardiac regeneration Lung epithelial regeneration Others Drug research and development Segmentation by End User Hospital Ambulatory Surgical Centers Specialty Clinics Academic and Research Institutes Segmentation by Geography North America Latin America Europe Asia-Pacific Excluding China China Middle East & Africa

Rapid technological advancement in healthcare, and favorable results of the amniotic fluid stem cells therapy will increase the market for amniotic fluid stem cell therapy over the forecast period. Increasing public-private investment for stem cells in managing disease and improving healthcare infrastructure are expected to propel the growth of the amniotic fluid stem cell therapy market.

However, on the basis of geography, global Amniotic Fluid Stem Cell Therapy Market is segmented into six key regions viz. North America, Latin America, Europe, Asia Pacific Excluding China, China and Middle East & Africa. North America captured the largest shares in global Amniotic Fluid Stem Cell Therapy Market and is projected to continue over the forecast period owing to technological advancement in the healthcare and growing awareness among the population towards the new research and development in the stem cell therapy. Europe is expected to account for the second largest revenue share in the amniotic fluid stem cell therapy market. The Asia Pacific is anticipated to have rapid growth in near future owing to increasing healthcare set up and improving healthcare expenditure. Latin America and the Middle East and Africa account for slow growth in the market of amniotic fluid stem cell therapy due to lack of medical facilities and technical knowledge.

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Some of the key players operating in global amniotic fluid stem cell therapy market are Stem Shot, Provia Laboratories LLC, Thermo Fisher Scientific Inc. Mesoblast Ltd., Roslin Cells, Regeneus Ltd. etc. among others.

The report covers exhaustive analysis on: Amniotic Fluid Stem Cell Therapy Market Segments Amniotic Fluid Stem Cell Therapy Market Dynamics Historical Actual Market Size, 2012 2016 Amniotic Fluid Stem Cell Therapy Market Size & Forecast 2016 to 2024 Amniotic Fluid Stem Cell Therapy Market Current Trends/Issues/Challenges Competition & Companies involved Amniotic Fluid Stem Cell Therapy Market Drivers and Restraints

Regional analysis includes North America Latin America Europe Asia Pacific Excluding China China The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

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Amniotic Fluid Stem Cell Therapy Market to Create Lucrative Opportunities for Existing Companies as Well as New Players - Herald Space

Stromal vascular fraction skin treatment is a type of stem cell therapy based on isolation of adipose tissue during liposuction or lipo-aspiration procedures of patients own body. In stromal vascular fraction treatment isolation of tissue contains fat cells, blood cells, and endothelial cells, as well as a large fraction of adipose-derived mesenchymal stem cells which provides regenerative properties and have positive anti-aging properties. A stromal vascular fraction is considered as a personalized stem cell therapy and effective tropical or injectable treatment.

With increasing age, regenerative and repair properties of skin are less effective due to decrease in stem cell count, and therefore, stromal vascular fraction treatment contains stem cell provides a boost in repair and maintenance mechanism of the skin leaving smooth, healthy, radiant skin. Stromal vascular fraction is a naturally occurring stem cell found in bundles of adipose tissues and are the primary source of growth factors along with macrophages and other cells. Due to the presence of growth factors, the stromal vascular fraction is utilized to decrease inflammation present in many diseases. A stromal vascular fraction is adopted in the treatment of rheumatoid arthritis, joint replacement, osteoarthritis, diabetes, Crohn's disease, and others.

Stromal Vascular Fraction Market: Overview

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Stromal vascular fraction is a combination of adipose-derived stromal cells (ADSCs), endothelial cells (ECs), endothelial precursor cells (EPCs), smooth muscle cells, macrophages, pericytes, and pre-adipocytes in the aqueous state. Stromal vascular fraction is advantageous over alternative medical treatments as SVF has the ability to regulate patients own system with the main focus on cell repair and regulation of defective cells. Stromal vascular fraction is a promising field for disease prophylaxis and currently are in clinical trials.

The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.

The report covers exhaustive analysis on: Market Segments Market Dynamics Market Size Supply & Demand Current Trends/Issues/Challenges Competition & Companies involved Technology Value Chain

Stromal Vascular Fraction Market: Segmentation

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The globalstromal vascular fraction marketcan be segmented on the basis of type of therapy, end-user, and region.

By Therapy Type SVF Isolation Products Enzymatic Isolation Non-enzymatic Isolation Automated POC Devices SVF Aspirate Purification Products SVF Transfer Products

By End-user Hospitals Specialty Clinics Stem Cell Banks/Laboratories Others

By Application Cosmetic Soft-tissue Orthopedic Others

By Region North America Latin America Europe Asia Pacific (APAC) South Korea Middle East and Africa (MEA)

In its last part, the report offers insights on the key players competing in the global market for stromal vascular fraction. With detailed profiling of each of the key companies active on the competitive landscape, the report provides information about their current financial scenario, revenue share at a global level, development strategies, and future plans for expansion. Strategic collaborations, mergers, and acquisitions have also been considered as a key strategy among a majority of leading companies in the market.

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Originally posted here:
Stromal Vascular Fraction Market by Top Key Players, Size, Subdivision & Market Dynamics Forces - Commerce Gazette

Everyone has heard of the seven wonders of the ancient world. Today I present my verdict on seven wonder discoveries in biological research, listed in rough chronological order.

Wonder 1: Cell TheoryCell theory, formulated in 1839, made three assertions: (a) All organisms are composed of basic units called cells; (b) The cell is the basic unit of structure and organisation in all living organisms; (c) New cells arise only from pre-existing cells.

Cell theory rationalised basic biology and contradicted the common notion that life can arise spontaneously from non-living matter.

Wonder 2: The Theory of Evolution Through Natural Selection.This is the most significant-ever insight into biology and was jointly proposed in 1858 by Charles Darwin (1809-1882) and Alfred Russell Wallace (1823-1913). It was already known that life on earth changes over long time periods. The theory of evolution explained the mechanism underpinning these changes natural selection. This theory draws all biology together into one unified framework. In its absence, biology would be reduced to a vast catalogue of unrelated observations.

Wonder 3: The Chemistry of LifeLife, unlike mechanical machines, works close to room temperature and without the assistance of significant temperature or pressure gradients. How then do cells grow, divide and create local order in a world that otherwise moves towards increasing disorder? This question was answered by biochemistry the chemistry of life.

A living cell is the end product of co-ordinated reactions between its innumerable chemical constituents metabolism. These reactions must proceed fast enough at room temperature to sustain life, and this is enabled by protein catalysts called enzymes, discovered in the 1800s. Cell metabolism is driven by energy, ultimately supplied by sunlight.

Wonder 4: Antibiotics and VaccinationsAntibiotics, discovered in 1928, are used to kill pathogenic microbes, and vaccination to provide immunity against disease has been in widespread use since 1900. Vaccination has eliminated many deadly diseases, including smallpox and polio.

Average life expectancy in 1900 was 50 years; today that figure is 82 years. Antibiotics and immunisation are one significant cause of this improvement. If we lost antibiotics and vaccinations today, life expectancy would gradually revert to 50-60 years.

Wonder 5: Discovering the Structure of DNAFaithful inheritance of parental characteristics by offspring is essential for biological evolution. The nature of the cells hereditary material was identified as nucleic acid (DNA) in 1944. In 1953, James Watson (born in 1928) and Francis Crick (1916-2004) made the greatest biological discovery of the 20th century when they solved the structure of DNA, a discovery that also indicated how DNA replicates and transmits genetic information from generation to generation.

DNA is a long molecule made of four sub-units A,T,G,C. Genetic information is encoded in the linear sequence (genes) of these subunits, dictating the structure/activity of all cell proteins, including all enzymes, thereby controlling the activities of the cell. Understanding DNA unlocked the secret of the molecular logic of life.

Wonder 6: CloningEvery animal body cell, including cells of the early embryo, contains a full set of the animals genes. Therefore, inducing any body cell into developing as embryonic cells develop should produce a genetic copy of the animal a clone. In 1996 Dolly the sheep was cloned in this manner.

Cloning offers many huge potential benefits, eg cloning animals, genetically modified to secrete a human hormone, to produce herds secreting large quantities of the hormone for use by patients deficient in the hormone. Cloning could also rebuild endangered animal populations or revive extinct animals.

Wonder 7: Induced Pluripotent Stem CellsThe human body is composed of over 200 tissues/organs skin, liver etc. Each tissue cell is differentiated to do a particular job. But all tissue cells develop from embryonic undifferentiated pluripotent stem cells.

In 2006 Shinya Yamanaka discovered how to transform differentiated body cells into pluripotent stem cells. These induced pluripotent stem cells (IPSC) can be coaxed to develop into specific adult tissues.

IPSCs have huge potential in many areas, eg regenerative medicine. New organs could be grown from patients own IPSCs to replace failing organs and without fear of immune rejection.

IPSCs are extremely useful for many research purposes, eg testing new drugs, and can substitute for stem cells extracted from human embryos, thereby avoiding ethical problems.

William Reville is an emeritus professor of biochemistry at UCC

See more here:
Story of life: Seven wonders of biological research - The Irish Times

Cancer stem cells (CSCs) refer to the cells obtained from tumor that posses potential to reproduce all types of cancer cells found in a cancer sample. Cancer stem cells are planned to grow in tumors as a separate population and thereby cause deterioration and metastasis of existing tumor through generation of new tumor. Thus, with advancement in technology especially in cancer stem cells research area, therapies specific to targeting cancer stem cells are expected to improve quality of life and survival cases of cancer patients with metastatic diseases.

Morbidity and mortality rate of cancer is rising at a faster speed worldwide and thus prevention of cancer and cancer treatment is grabbing attention of cancer researchers globally. Stem cells and cell therapy have shown significant potential to treat cancer effectively. Cancer stem cells (CSCs) have been tested on animal models and have also shown satisfactory results. However, human testing of cancer stem cells is still in its developing stage owing to stringent regulations and ethical issues associated with the same.

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Worldwide cancer research activities are increasing rapidly owing to rising burden of mortality rate of cancer. Cancer stem cells are under research for various types of cancers such as lung cancer, breast cancer, colorectal cancer, skin cancer, brain cancer and bone cancer. Government initiative to boost the cancer research activities and availability of funds are some of the factors that are driving the global cancer stem cells (CSCs) market towards growth. While on the other hand, ethical issues involved in the stem cells research and stringent regulations to perform human trials are some of the factors that are restraining the growth of the global cancer stem cells (CSCs) market.

Geographically, global cancer stem cells market is segmented into North America, Europe, Asia Pacific and Rest of the world (RoW) regions. Currently, North America is leading the global cancer stem cells (CSCs) market and is followed by Europe. Factors such as highly developed research infrastructure, well defined regulatory norms, availability of research funds, availability of skilled research and healthcare professionals and supportive economy are driving the North American cancer stem cells market towards growth. Asia Pacific is lucrative market for cancer stem cells. Governments in the Asia Pacific countries mainly, India and China are taking initiative to boost the healthcare and biotechnology industry in the respective countries and thus, research and development activities in these countries are swiftly increasing.

Apart from India and China, Japan will play a significant role in the cancer stem cells market. Japanese government is heavily investing in healthcare industry in order to improve the healthcare facilities in the country and thus rising cancer treatment are expected to escalate the cancer stem cells treatment market in Japan. Latin American countries namely, Brazil, Mexico and Argentina are expected to contribute more to cancer stem cells market than other countries in the rest of the world region. While on the other hand, African countries and Middle Eastern countries are expected to show slow or no growth rate in the global cancer stem cells (CSCs) market.

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Some of the major players in the global cancer stem cells market are AdnaGen GmbH, Advanced Cell Diagnostics, Inc., AVIVA Biosciences Corporation, Celula, Inc., Epic Sciences, Inc., Fluxion Biosciences, Inc., Rarecells USA, Inc. and Silicon Biosystems, S.p.A.

This research report analyzes this market on the basis of its market segments, major geographies, and current market trends. Geographies analyzed under this research report include North America Asia Pacific Europe Rest of the World

This report provides comprehensive analysis of Market growth drivers Factors limiting market growth Current market trends Market structure Market projections for upcoming years

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

Reasons for Buying this Report This report provides pin-point analysis for changing competitive dynamics It provides a forward looking perspective on different factors driving or restraining market growth It provides a six-year forecast assessed on the basis of how the market is predicted to grow It helps in understanding the key product segments and their future It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments It provides distinctive graphics and exemplified SWOT analysis of major market segments

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Cancer Stem Cells Market to Increase at Steady Growth Rate 2016-2024 - The Check Chronicle

Updated as of September 19, 2019

With one in 10 Americans over 65 currently living with symptomatic Alzheimers disease, you probably know someone affected by this disease. Worldwide, 50 million people live with symptomatic Alzheimers, making it the most common form of dementia. It commonly affects people over 65, but less than 4 percent of the estimated 5.7 million Americans affected have early-onset Alzheimers with symptoms beginning before age 65.

By 2050, nearly 14 million Americans are projected to suffer from this disease. Alzheimers is 6th leading cause of death in the US, making Alzheimers disease a top health concern. Unfortunately, there is no cure, but current medications and management strategies may improve symptoms, prolonging patient independence. In honor of November being Alzheimers Awareness month, we evaluated the current therapies, drugs in the pipeline and disease outlook.

Overview

Alzheimers disease is a degenerative brain disease that typically begins in late middle age or old age. Degeneration of brain cells, called neurons, cause the symptoms of progressive memory loss, impaired thinking, disorientation and mood and personality changes.

Risk factors: The greatest risk factors are old age, having a family history of Alzheimers and carrying a mutation in a certain gene called apolipoprotein E 4 (APOE4). Environmental and lifestyle factors, such as diet and exercise, also contribute to disease development.

The risk of Alzheimers doubles every five years after the age of 65, with nearly 1 in 3 people age 85 or older developing the disease. People with the APOE4 gene variant are thought to have an increased risk for developing late-onset Alzheimers, but thats not a steadfast rule: inheriting the gene variant does not mean the person will definitely get Alzheimers and some Alzheimers patients do not have the APOE4 gene variant.

Causes: Alzheimers develops as a result of a complex interaction between many risk factors, all resulting in neuron damage and death. The buildup of misfolded proteins, such as the tau protein and -amyloid, create the hallmark protein clumps called tangles and plaques seen in Alzheimers brains. While these protein clumps are thought to cause neuron death by blocking nerve cell communication and function, the exact relationship between the protein clump formation and neuron death is still unclear.

The four stages of Alzheimers: Based on the severity of dementia symptoms, Alzheimers can be characterized into four stages: preclinical, mild (early-stage), moderate (middle-stage) and severe (late-stage). The preclinical stage encompasses all the unseen changes in the brain, such as plaque accumulations, happening years before symptoms arise. Mild Alzheimers patients can still function independently, although they begin forgetting familiar words or locations of objects. As the dementia progresses to become moderate, the patient becomes more forgetful, has greater difficulty doing daily tasks and experiences personality and behavioral changes. At this stage, they may still remember significant life events. The moderate disease stage is the longest, often lasting for many years. Finally, severe Alzheimers patients can no longer respond to their environment, carry a conversation and control their movement or bowels. As the disease progresses, patients require an increasing level of care for daily activities.

Life expectancy: The earlier the diagnosis, the longer the life expectancy is: people diagnosed in their 60s to early 70s can live as long as 7 to 10 years, whereas those diagnosed in their 90s only average a 3-year life expectancy. Alzheimers patients live an average of four to eight years after their diagnosis, but can live as long as 20 years post-diagnosis. However, its difficult to link one disease to life expectancy, especially as you age, due to the many variables that influence life expectancy.

Cost: The cost burden of Alzheimers is as high as its prevalence: Alzheimers medications can range from $177 to $400 monthly, adding up to an annual prescription drugs estimated cost of $3,000. It will cost Americans an estimated $277 billion, including $186 billion in Medicare and Medicaid payments, to care for Alzheimers patients by the end of 2018. By 2050, this cost is projected to be more than $1.1 trillion, accounting for over four-fold increases in government spending through Medicare and Medicaid, as well as out-of-pocket expenses. Up to $7.9 trillion in medical and care costs could be saved by diagnosing earlier and more accurately.

Diagnosis Strategies

Although there is no specific test for Alzheimers disease, doctors use a variety of exams, imaging and lab testing to diagnose the disease.

Physical and neurological exams can test reflexes, coordination and memory. Brain imaging is used to rule out other physical abnormalities, such as tumors, stroke or other traumas, that can cause Alzheimers-like symptoms. Imaging can now be used to detect the specific changes that occur in the brains of living Alzheimers patients, not just in post-mortem analysis. Structural imaging techniques, such as magnetic resonance imaging (MRI) and computed tomography (CT), are used to rule out other physical injuries as well as assess Alzheimers-related brain shrinkage. Functional imaging, such as functional MRI (fMRI) and positron emission tomography (PET), can measure brain cell function by tracking the cells sugar and oxygen use.

Specific radioactive molecules, called radiotracers, can be used to detect -amyloid plaques via PET imaging. Three radiotracers have been approved by the U.S. Food and Drug Administration (FDA) since 2012: Amyvid (18F-florbetapir), Vizamyl (18F-flutametamol) and Neuraceq (18F-florbetaben).

Genetic testing can reveal if someone has a mutation, such as the APOE4 gene variant, that may increase their risk for developing Alzheimers. However, it is generally not recommended for Alzheimers diagnosis due to the lower accuracy, as many factors contribute to disease development. The exception is early-onset Alzheimers: Anyone with a family history of early Alzheimers can be screened for certain gene mutations, such as amyloid precursor protein (APP), presenilin-1 (PS-1) and presenilin-2 (PS-2).

Developing better diagnostic testing could facilitate earlier diagnoses, possibly leading to better outcomes. Future testing includes more sensitive mental ability exams and measuring key disease-associated proteins, called biomarkers, in the blood or spinal fluid.

Current Therapies

While there is no cure for Alzheimers disease, a handful of drugs have been approved by the FDA and shown to somewhat slow symptom progression. They can be broken down into two categories: cholinesterase inhibitors, which increase the amount of the neurotransmitter acetylcholine in the brain, resulting in more cell-to-cell communication; and NMDA receptor antagonists, which also alter how brain cells communicate.

Cholinesterase inhibitors include Eisais Aricept (donepezil) and Novartis Exelon (rivastigmine), both approved for all stages of Alzheimers, as well as Janssen Pharmaceuticals Razdyne (galantamine), which is approved for mild to moderate Alzheimers. Allerganhas two NMDA receptor antagonist-based drugs, Namenda (memantine) and the combination drug Namzaric (donepezil and memantine), which are both approved for moderate to severe Alzheimers. Antidepressants and anti-anxiety medications are sometimes prescribed as well to help control behavioral symptoms.

Unfortunately, these drugs can cause potentially severe side effects and arent overwhelmingly effective compared to placebo, although they have helped stave off mental decline for a while in some patients. However, the need for more effective drugs is clear.

Drug Pipeline

A variety of targeted therapies are currently being explored through clinical trials, including drugs against the tau protein, which forms distinctive tangles in Alzheimers brains; the -amyloid protein, which forms plaques in the Alzheimers brain; -secretase (BACE), an enzyme that cuts amyloid precursor protein (APP) into -amyloid; and the 5-HT2A serotonin receptor, which is involved in cognition and memory by mediating neurotransmitters, such as acetylcholine and glutamate.

The Alzheimers drug development market includes many large players, including Eli Lillywith six drugs (two in Phase 1, two in Phase 2 and two in Phase 3);Biogen with five drugs (two in Phase 1, one in Phase 2 and two in Phase 3); Roche, in collaboration with Genentech, AC Immune, and MorphoSys, with three drugs (two in Phase 2 and one in Phase 3); Eisai, in collaboration with Biogen, with one drug in Phase 3; and Eisai alone with one drug in Phase 2 (as of September 13, 2019).

As of September 13, 2019, there are over 670 active/recruiting/not yet recruiting clinical trials for Alzheimers listed on clinicaltrials.gov. According to a paper published in July 2019, there were 132 drugs in development for Alzheimers: 28 drugs in 42 Phase 3 trials, 74 drugs in 83 Phase 2 trials, and 30 drugs in 31 Phase 1 trials. The figure and legend below, taken from the July 2019 paper, shows all the drugs in clinical trials for Alzheimers as of February 2019.

UsAgainstAlzheimers released their 2019 Alzheimers Drug Pipeline report also in July 2019, where they focused on 98 late-stage Alzheimers drugs in development that could potentially reach the market in the next 5-10 years: 26 drugs in Phase 3 trials, and 72 drugs in Phase 2 trials. Their report shows that, despite some large Phase 3 failures this year, the Alzheimers pipeline is still robust.

The following analysis of some Alzheimers drugs in the pipeline will briefly discuss how each drug works and where it is in clinical trials. This information was up to date as of September 13, 2019. Any text in italics represents failed or terminated trials.

Note: This article is not meant to be completely comprehensive and may unintentionally exclude some drugs in development or clinical trials, especially those trials outside of the United States.

Phase 1

Biogen is exploring multiple antibody drugs against the -amyloid and tau proteins, including a Phase 1 trial studying the anti-tau antibody BIIB076 in 48 healthy and Alzheimers patients; a Phase 2 trial (TANGO) examining the anti-tau antibody BIIB092 (gosuranemab) in 528 early-stage Alzheimers patients; a Phase 2 trial in collaboration with Eisai studying the anti--amyloid antibody BAN2401 in 800 early-stage Alzheimers patients; and a Phase 3 trial (Clarity AD) studying BAN2401 in 1566 early Alzheimers patients.

Unfortunately, in March 2019, Biogen and its partner Eisai decided to end all studies involving another one of its anti--amyloid antibodies called aducanumab (previously called BIIB037), including their two Phase 3 trials (ENGAGE and EMERGE) each studying 1605 early-stage Alzheimers patients, a Phase 2 trial (EVOLVE) in 500 Alzheimers patients with mild cognitive impairment (MCI) or mild dementia due to Alzheimers, and a Phase 1 trial (PRIME) in 197 very mild (prodromal) or mild Alzheimers patients. The studies were stopped because they did not meet their clinical endpoints of slowing cognitive and functional impairment, not due to any safety concerns of the drug.

Eli Lilly is pursuing two chemical entities, a Tau Morphomer and an O-GlcNAcase Inhibitor, in Phase 1 clinical trials for Alzheimers.

Proclara Biosciencescombined a part of the human immunoglobulin protein with their unique protein technology, called General Amyloid Interaction Motif (GAIM), to create their fusion protein drug NPT088, which targets both -amyloid and tau proteins. Their Phase 1a safety trial showed that intravenous NPT088 is safe and well-tolerated in 40 healthy patients. Data from their Phase 1b dosing trial in Alzheimers patients is expected in 2019.

Cognition Therapeutics drug candidate CT1812 is a small molecule pill that disrupts -amyloid binding to a receptor called sigma-2 on brain cells, which is thought to prevent the proteins toxicity. CT1812 has been or is being studied in six clinical trials, including one recruiting Phase 1 trial with 18 mild to moderate Alzheimers patients, one recruiting Phase 1/2 trial with 21 mild to moderate Alzheimers patients, and one recruiting Phase 2 trial with 120 mild to moderate Alzheimers patients. CT1812 was well-tolerated and penetrated the brain very well in 80 healthy patients and 19 mild to moderate Alzheimers patients with mild to moderate side effects. Although the treated Alzheimers patients had lower levels of Alzheimers-related proteins (such as neurogranin and synaptotagmin-1, markers of synaptic damage) in their cerebrospinal fluid, they didnt show significantly different cognitive functioning compared with the placebo group after 28 days of treatment.

Samus Therapeuticsis developing a positron-emitting molecule, called 124I-PU-AD, that inhibits a certain protein complex called epichaperone complex, which reduced tau proteins in the brain, restored long-term memory and increased survival in preclinical animal models. 124I-PU-AD is also being used as a PET imaging agent to study the epichaperone complex in the brains of Alzheimers patients. They have completed an early Phase 1 trial in 5 Alzheimers and certain cancer patients to evaluate the molecules metabolism. Another Phase 1 study is currently recruiting 24 healthy volunteers to evaluate the safety and tolerance of the drug.

Janssen Research & Development is examining the ability of a radioactive PET imaging agent, called [18F]MNI-1020, to bind to the tau protein in Alzheimers patients. An early Phase 1 trial studied the safety and brain uptake efficacy of a single injection of the imaging agent in 15 Alzheimers and healthy age-matched patients. That study also compared the location of tau (using [18F]MNI-1020) and -amyloid (using Amyvid (florbetapir)) in patients with suspected Alzheimers.

Longeveron collects stem cells from healthy adult donors to create their own Longeveron mesenchymal stem cells (LMSCs), which have been shown to reduce inflammation and promote cell regeneration. Their Phase 1 clinical trial is currently recruiting 30 Alzheimers patients to evaluate the safety and efficacy of LMSCs.

Athira Pharmas small molecule drug NDX-1017 designed to restore lost or build new connections in the brain. Their Phase 1 trial is currently recruiting to evaluate the drugs safety in two parts, with Part A involving up to 56 healthy young and elderly participants and Part B involving 44 healthy, mild cognitive impairment or mild to moderate Alzheimers patients.

Cortexyme, Inc.is developing COR388, a first-in-class bacterial protease inhibitor that targets the bacteria Porphyromonas gingivalis, which is present in Alzheimers patients brains and cerebrospinal fluid and thought to contribute to the disease. Two completed Phase 1 trials have shown that COR388 is safe and well-tolerated in 58 healthy and nine Alzheimers patients. A Phase 2/3 trial is currently enrolling 573 mild to moderate Alzheimers patients to assess the drugs efficacy, safety, and tolerability.

Allergan was pursuing a small molecule drug called AGN-242071 that selectively targeted certain receptors in the brain, called muscarinic receptors, which may treat symptomatic cognitive deficits and behavioral symptoms in Alzheimers.

Unfortunately, Allergan decided to withdraw their Phase 1 trial evaluating the safety and tolerability of the drug prior to patient recruitment in November 2018.

Corium Internationalhas developed a novel delivery method for an approved drug, a once-weekly skin patch (the Corplex Donepezil Transdermal System) that delivers a sustained dose of donepezil. The patchs safety and drug profile were examined in multiple Phase 1 trials, which showed great skin tolerability and comparable dosages between the weekly patch and the currently prescribed daily donepezil pills. Corium is also developing a once-weekly skin patch to deliver memantine

Cognoptix has taken a different approach, developing an eye test called Sapphire II to catch and diagnose Alzheimers much earlier by detecting -amyloid deposits in their eyes. A fluorescent drug that binds to the -amyloid protein (Aftobetin-HCl) is administered to the eye as an ointment and binding is detected with the Sapphire II laser device. Their Phase 1 study determined the optimal dosing of the fluorescent drug in 15 participants and is currently recruiting 10 normal and 20 mild cognitive impairment (MCI) or mild Alzheimers patients for dose testing. If the dosing is optimal, then 30 more MCI and 30 more mild Alzheimers patients will be recruited, totaling 105 participants.

Phase 1/2

Ionis Pharmaceuticalsis collaborating with Biogen to study their antisense oligonucleotide drug IONIS-MAPTRx (also called BIIB080), which may reduce tau protein production and its accumulation in brain cells, in a Phase 1/2 trial in 44 mild Alzheimers patients.

QR Pharma, Inc.s small molecule drug Posiphen inhibits APP, tau and -synuclein (involved with Parkinsons disease) protein synthesis. They are currently recruiting 24 Alzheimers patients for their Phase 1/2 dosage study (DISCOVER).

Following their successful Phase 1 trial (SEAD) in 15 Alzheimers patients, Ausio Pharmaceuticalsbrought their estrogen receptor activating drug S-equol (also called AUS-131) to a Phase 1/2 trial (SEAD2), which is currently recruiting 40 Alzheimers patients to test the drugs tolerability and whether or not it affects cognitive abilities. Activating the estrogen receptors on mitochondria is thought to promote mitochondrial functioning, which could restore the reduced mitochondrial activity seen in Alzheimers patients. Less mitochondrial activity is thought to contribute to -amyloid protein build-up in the brain.

Nature Cell Co. is studying a fat cell-derived mesenchymal stem cell (MSC) therapy called AstroStem in an active Phase 1/2 study involving 21 mild to moderate Alzheimers patients.

Phase 2

Eli Lilly has two ongoing Phase 2 trials studying antibody drugs: one active trial (TRAILBLAZER-ALZ) evaluating the tolerability and efficacy of a humanized anti--amyloid antibody, called donanemab (LY3002813 or N3pG-A MAb), in 266 early symptomatic Alzheimers patients; and another currently recruiting trial evaluating the safety and efficacy of a humanized anti-tau antibody, called zagotenemab (LY3303560), in 285 early symptomatic Alzheimers patients.

Roche, in partnership with AC Immune, is studying crenezumab (RG7412), an anti--amyloid antibody drug that binds to -amyloid similar to Eli Lillys solanezumab. Crenezumab is being investigated in an active Phase 2 trial involving 252 non-symptomatic adults with a family history of Alzheimers who have a particular genetic mutation (autosomal-dominant PSEN1 E280A). Baseline data for 242 of the enrolled patients were presented at the Alzheimers Association International Conference in August 2019. Another not yet recruiting Phase 2 trial is in the works to study the effect of crenezumab on the longitudinal tau burden via PET imaging of 150 patients enrolled in the active Phase 2 trial (NCT01998841).

Crenezumab was being investigated in three Phase 3 trials: CREAD 1 evaluating the drugs safety and efficacy in 813 mild Alzheimers patients; CREAD 2 studying the drugs safety and efficacy in 750 mild Alzheimers patients; and an open-label extension trial (CREAD OLE) examining long-term drug treatment in 149 Alzheimers patients. Unfortunately, in January 2019, Roche discontinued all CREAD trials due to the interim analysis showing crenezumab was unlikely to meet the primary endpoint of improving cognition.

Genentech (a subsidiary of Roche) is partnering with AC Immune to develop the anti-tau antibody drug RO7105705 (also called RG6100 and MTAU9937A), which recognizes tau tangles and is meant to block their spread between cells. An active Phase 2 trial involving 457 prodromal to mild Alzheimers patients is studying the drugs safety and effect on cognitive function.

AbbVies humanized antibody drug ABBV-8E12, which targets the tau protein, is being evaluated for its safety and efficacy in an active Phase 2 trial involving 400 early-stage Alzheimers patients. An extension study to study the drugs long-term safety and tolerability is currently enrolling patients from the Phase 2 study (NCT02880956) by invitation.

Avid Radiopharmaceuticals, a wholly-owned subsidiary of Eli Lilly, is developing the PET imaging agent 18F-AV-1451 (also called Flortaucipir F 18 or F 18 T807), a molecule that binds to the tau protein, allowing researchers to study tau in living patients. There are multiple Phase 2 or Phase 2/3 trials studying the imaging agents safety and efficacy, with five Phase 2 trials currently recruiting or not yet recruiting: one to evaluate the agents safety and tau binding via PET imaging in 250 healthy, Alzheimers, traumatic brain injury and depression patients; one (ADRC proj 1) to compare tau tangles in the brain with cerebrospinal fluid CSF biomarkers and cognitive status in 80 Alzheimers patients; one (DIAN Project, AV ADAD) to study the presence of tau tangles in the brain and cognitive status in 130 adults; one to study the uptake and binding in 80 older HIV-positive adults with and without HIV-associated neurocognitive disorders and HIV-negative age-matched controls; and one (Protocol Z) to study tau and amyloid lesions in the brains of 80 APOE4+ adults with normal cognition or early-stage symptomatic Alzheimers.

Neurotrope Bioscience is developing bryostatin-1, a small molecule that activates protein kinase C (PKC), a protein that is important for learning and memory. This drug stimulates synapse repair and growth, activates -amyloid degrading enzymes and prevents tau tangle formation and neuron death. A Phase 2 trial evaluating the safety and efficacy of bryostatin-1 in 147 moderate to severe Alzheimers patients showed positive results: the lower (20 g) dose improved cognition and the ability to care for oneself. This prompted a second Phase 2 trial to study the drugs safety and efficacy at the lower dose in 108 moderately severe to severe Alzheimers patients.

Unfortunately, Neurotrope announced that the second Phase 2 trial did not show statistically significant improvement in memory, indicating it did not meet its primary endpoint of a change in the Severe Impairment Battery (SIB) test total score from baseline to week 13.

EIP Pharma is pursuing a small molecule called neflamapimod (VX-745) that inhibits an enzyme, called p38 MAPK, found in the neurons that is involved in inflammation and possibly -amyloid toxicity. Neflamapimod previously showed clinical activity in rheumatoid arthritis patients before being licensed to EIP Pharma. They are currently conducting a Phase 2b efficacy study (REVERSE-SD) in 161 participants with mild Alzheimers. A Phase 3 study is scheduled to start in the third quarter of 2020. Another Phase 2 trial is recruiting 40 Alzheimers patients to study neflamapimod on brain inflammation.

Actinogen Medicalis studying a drug called xanamem, which inhibits a cortisol-producing enzyme in the brain, ultimately blocking local production of cortisol, known as the stress hormone. While blood cortisol levels tend to rise with age, its particularly raised in patient with certain diseases, such as Alzheimers. Long-term high cortisol levels can be toxic to brain neurons, so preventing cortisol production in the brain may help slow cognitive decline and -amyloid plaque formation. After assessing xanamems safety and dosing in two Phase 1 trials, a Phase 2 trial (XanADu) assessed the drugs safety and efficacy in 186 early-stage Alzheimers patients.

Boehringer Ingelheims drug BI 425809 is a glycine transport inhibitor designed to regulate signaling in the brain that contributes to cognitive impairment. An active Phase 2 trial is studying the safety and effect on cognition of multiple dosages of the drug in 611 Alzheimers patients.

Neurocentriais developing a dietary supplement called MMFS, which contains a molecule called L-threonic acid magnesium salt (L-TAMS) that increases synapse density in portions of the brain needed for memory and executive functioning, such as the prefrontal cortex and hippocampus. Two previous studies showed improved cognition in mild to moderate Alzheimers patients, prompting the active Phase 2 trial that is recruiting 12 mild Alzheimers patients to examine the drugs safety and effect on cognition.

Alkahestis studying intravenously administered plasma-derived product called GRF6019, which is isolated from human plasma (a component of the blood) that has been shown to enhance neurogenesis and improve learning and memory in animals. Matching donor and patients blood types is not needed because the donor-specific antibodies (called immunoglobulins) are removed. A Phase 2 trial in 40 mild to moderate Alzheimers patients studied the safety and feasibility of GRF6019. Another Phase 2 trial is currently recruiting 20 severe Alzheimers patients to study the safety, tolerability, and cognitive benefits of the drug.

Suven Life Sciences drug SUVN-502 specifically inhibits a certain serotonin receptor (called 5-HT6), which is thought to improve cognition and memory. SUVN-502 in combination with donepezil and memantine was shown to increase the concentration of neurotransmitters, like acetylcholine. An active Phase 2 trial is testing the effect of this triple combination therapy on cognition in 563 moderate Alzheimers patients. An expanded access program is also available for eligible patients to receive the drug without being evaluated for safety and efficacy.

Neurim Pharmaceuticalsis taking a different approach by developing a drug, called piromelatine, that binds to and activates melatonin and serotonin receptors in the brain, promoting sleep and therefore neuroprotective effects. This drug was safe and promoted deeper and more REM sleep in a Phase 2 clinical trial in adults with insomnia. Given the link between sleep and Alzheimers, Neurim decided to study piromelatines effects on cognition in 500 mild Alzheimers patients in an active dose-ranging Phase 2 trial.

Eisai, in collaboration withPurdue Pharma, is studying their orexin receptor antagonist drug lemborexant in a Phase 2 trial involving 62 mild to moderate Alzheimers patients. The orexin receptor is involved in the regulation of sleep. Lemborexant binds to the orexin receptor, preventing orexin from binding, which should decrease wakefulness and promote falling and staying asleep naturally. Sleep, especially at appropriate hours, is troublesome for Alzheimers patients whose circadian rhythms tend to be dysregulated.

Phase 2/3

Novartis has partnered with Amgen and the Banner Alzheimer's Instituteto pursue Novartis drug umibacestat (CNP520), which inhibits BACE1, an enzyme involved in -amyloid production. After a successful Phase 2 trial safety study in 124 healthy elderly patients, there were two Phase 2/3 trials: one (Generation S1) to test the efficacy of CNP520 against an investigational immunotherapy drug (CAD106, a vaccine against a fragment of the -amyloid protein) in 481 non-symptomatic older patients with two copies of the APOE4 gene; and one (Generation S2) to test the drugs effect on cognition and underlying Alzheimers pathology in 1145 non-symptomatic older patients with at least one APOE4 allele and elevated brain -amyloid levels.

Unfortunately, both Phase 2/3 trials were discontinued in July 2019 due to worsening cognitive function seen during interim data analysis. As umibacestat was meant to delay the onset of symptoms, participants in the study will discontinue the investigational treatment and discuss further treatment options with their doctors.

TauRx Therapeutics drug TRx0237 (also called LMTX) is their second-generation tau protein aggregation inhibitor, which aims to both dissolve existing tau tangles and prevent new tangles from forming. Two previous Phase 3 clinical trials studied the safety and efficacy of high doses (150-250 mg/day) and a low dose control (8 mg/day) of the drug in 800 mild and 891 mild to moderate Alzheimers patients. Surprisingly, they found that the low dose was as beneficial as the higher doses, prompting a current Phase 2/3 trial (LUCIDITY) recruiting 375 early Alzheimers patients studying TRx0237 at low doses (8 and 16 mg/day). An expanded access program is also available to provide the drug to patients who have previously participated in a TauRx clinical trial but do not qualify for an ongoing trial.

Axsome Therapeutics is pursuing a treatment for agitation associated with Alzheimers and have been granted fast track status for their drug AXS-05, which combines dextromethorphan and bupropion. Dextromethorphan (called DM and commonly known as an over-the-counter cough suppressant) inhibits serotonin and norepinephrine transporters and the NMDA receptor at high doses. Bupropion increases the bioavailability of dextromethorphan and inhibits norepinephrine and dopamine reuptake. A Phase 2/3 trial (ADVANCE) is currently recruiting 435 Alzheimers patients to study the safety of AXS-05 and its effect on agitation.

Phase 3

Eli Lilly has a Phase 3 anti--amyloid antibody drug called solanezumab (LY2062430), which binds to soluble -amyloid monomers. The primary endpoints of trials involving this drug is to slow memory and cognitive decline. The drug is associated with 11 listed trials, including an active Phase 3 trial (A4) involving 1150 not yet symptomatic adults with evidence of amyloid plaque build-up in their brains, and a currently recruiting Phase 2/3 large collaboration trial (DIAN-TU) comparing solanezumab and gantenerumab in 490 non-symptomatic adults known to have an Alzheimers disease-causing mutation. This collaboration includes Eli Lilly, Roche, Avid Radiopharmaceuticals, Janssen, Alzheimers Association, National Institute on Aging (NIA), Accelerating Medicines Partnership (AMP), and Washington University School of Medicine.

Two Phase 3 trials (EXPEDITION and EXPEDITION 2) were completed previously and involved 1040 Alzheimers patients each. Although there was no difference in cognition between the treated and placebo groups, patients with mild Alzheimers did show slower cognitive decline compared to placebo, prompting further studies.

Unfortunately, the next three Phase 3 trials (EXPEDITION 3, EXPEDITION EXT and EXPEDITION PRO) were terminated due to lack of meeting primary endpoints, including slowing cognitive decline, and insufficient evidence that solanezumab would likely demonstrate a meaningful benefit to patients with prodromal Alzheimers.

Roche is currently investigating gantenerumab, an anti--amyloid antibody drug that binds and neutralizes -amyloid plaques. Gantenerumab, brought back after failing in previous clinical trials, is involved in four Phase 3 trials: two active trials studying the drugs effect on cognitive function in 799 prodromal and 389 mild Alzheimers patients; and two currently recruiting trials studying the drugs effect on cognition in 760 early Alzheimers patients each.

Eisai, in collaboration with Biogen, is studying their small molecule BACE1 inhibitor elenbecestat (also called E2609) in two Phase 3 trials (MISSION AD1 and MISSION AD2) currently recruiting 950 early-stage Alzheimers patients each. Inhibiting BACE1 is thought to interfere with -amyloid production.

Unfortunately, the companies announced that they were discontinuing their MISSION AD1 and AD2 Phase 3 trials on September 13, 2019. The decision was made based on results from a safety review that showed an unfavorable risk-benefit ratio of elenbecestat.

Avid Radiopharmaceuticals and Eli Lilly reported positive results earlier this year from their Phase 3 trial on the tau-binding PET imaging agent flortaucipir F 18 (18F-AV-1451 or Tau imaging agent) in Alzheimers patients. The trial met its two primary endpoints, successfully predicting both the disease-related role of tau in the brain and an Alzheimers diagnosis. PET imaging was performed on 156 end-of-life patients with cognition ranging from normal to dementia, with 67 of these patients being evaluated post-mortem. Flortaucipir could significantly detect Alzheimers-related changes in the brain, including both tau and -amyloid plaque densities. Being able to accurately image and diagnose Alzheimers patients is a critical component in understanding the disease and being able to manage it. There are currently 33 studies listed on clinicaltrials.gov for flortaucipir and Alzheimers.

AZTherapies, Inc. is studying the combination drug ALZT-OP1, which consists of the inhaled drug cromolyn and oral drug ibuprofen, both of which are anti-inflammatory. Inflammation in the brain is thought to trigger neuronal death, which causes progressive brain damage. Cromolyn was also shown to prevent -amyloid aggregation in one study. A Phase 3 trial (COGNITE) is currently studying the effect of this combination drug on cognitive decline in 620 early-stage Alzheimers patients.

ACADIA Pharmaceuticals drug pimavanserin (previously called ACP 103) is a selective serotonin inverse agonist (SSIA), meaning it both binds to serotonin receptor subtype 5-HT2A and blocks serotonin signaling. Following a few Phase 2 trials specifically in Alzheimers patients, there are currently three recruiting Phase 3 trials for a broader range of dementia patients: an efficacy study examining pimavanserins ability to prevent relapse of dementia-related psychosis symptoms in 356 dementia patients, a safety study in 300 patients with neurodegenerative disease, and an open-label extension study examining the drugs long-term safety in 300 patients with neurodegenerative disease who previously participated in another pimavanserin clinical trial by ACADIA.

Intra-Cellular Therapiesis developing lumateperone (ITI-007), a molecule that simultaneously affects serotonin, dopamine and glutamate signaling, which play important roles in multiple mental illnesses. Following a Phase 1b/2 study, they recruited 177 dementia patients, including Alzheimers patients, for a Phase 3 trial studying the safety and efficacy of the drug for reducing agitation.

However, the Phase 3 trial was terminated early due to interim data analysis indicating lumateperones lack of efficacy.

AVANIR Pharmaceuticals drug AVP-786 combines two approved drugs: deuterated dextromethorphan (d6-DM), which has better bioavailability and less side effects than regular DM, and an ultra-low dose of quinidine, which slows the metabolism of d6-DM by inhibiting an enzyme (CYP 2D6) that breaks down d6-DM. AVP-786 is a second-generation version of Nuedexta (formerly AVP-923), which is currently approved to treat pseudobulbar affect (PBA). Currently, there are four recruiting or active Phase 3 trials studying the safety and efficacy of AVP-786 in treating agitation in Alzheimers patients: one recruiting 412 Alzheimers patients with moderate to severe agitation worldwide, one active study involving 522 Alzheimers patients in the US, one completed study involving 410 Alzheimers patients in the US and a long-term extension study recruiting 700 patients who have completed previous clinical trials of AVP-786 by Avanir.

Otsuka Pharmaceutical Co.and Lundbeck are collaborating to develop brexpiprazole (brand name Rexulti) for treating agitation and behavioral symptoms in Alzheimers patients. Rexulti, which binds to and activates a particular dopamine receptor (D2), is currently FDA approved to treat schizophrenia and as an add-on treatment for major depression disorder. Two Phase 3 trials examining brexpiprazole at either fixed or flexible doses in a total of 703 Alzheimers patients showed reduced agitation compared to the placebo. They are currently recruiting for three Phase 3 trials: one evaluating the safety, efficacy, and tolerability in 225 Alzheimers patients with dementia-associated agitation in the US; one studying long-term treatment in 157 Alzheimers patients with dementia-associated agitation in Japan; and a 12-week extension study for 250 Alzheimers patients with dementia-associated agitation who were previously enrolled in other Otsuka trials studying brexpiprazole. They are also recruiting for a Phase 2/3 study in 407 Alzheimers patients with dementia-associated agitation in Japan.

Merck Sharp & Dohme Corp., a subsidiary of Merck, is studying their FDA approved drug suvorexant (previously called MK-4305, brand name Belsomra) to treat insomnia in Alzheimers patients. Currently approved for insomnia patients, the small molecule drug works by inhibiting the orexin receptor in the signaling system involved in wakefulness. Their Phase 3 trial studying suvorexants safety and efficacy at improving sleep in 285 Alzheimers patients and patients with insomnia concluded in October 2018, but results have not been posted yet.

Visit link:
Alzheimer's Disease Insight Report: Current Therapies, Drug Pipeline and Outlook - BioSpace

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Anti-Ageing Drugs Market 2019 In-depth Analysis by Leading Players: Nu Skin, BIOTIME, Elysium Health - Daily Research Chronicle

Once weve conquered our diets, instituted a regimen of exercise and saunas and cold plunges, doused ourselves in NMN and resveratrol and Metformin and benign viruses, quit smoking and cut down our drinking and remembered to wear our seatbelts, theres one main obstacle remaining in the way of an extra-long and healthy life: our guilt.

Whether its hard-wired or a result of societal expectations, we tend to feel that old farts should not outstay their welcome. Leave some room for future generations, we grumble under our breath, out of earshot of elderly relatives. Youre already taking up too much of the housing stock, making it near-impossible for millennials to buy homes. You want to bankrupt Social Security and Medicare too?

Just last month, Ezekiel Emanuel, the chair of the University of Pennsylvanias department of medical ethics (and a chief architect of Obamacare) confirmed that he stood by his controversial 2014 essay: Why I hope to die at 75. Despite the onslaught of anti-aging research, Emmannuel (now 62) said his main arguments still held water: That people in their 80s who were still vigorous were not doing meaningful work; that authors above 75 were not producing brand-new books but simply re-ploughing old furrows.

Let's leave aside the fact that's a pretty weird metric to judge the worth of a life -- sorry, grandma, time to go, you're not doing meaningful work or writing new books! Emanuel's argument ignores what biologists like Sinclair are telling us. The more we age in good health, the more useful we will be.

Sinclair, as you might expect, could not disagree with Emanuel more. First of all, he says, lets assume everyone stopped dying of age-related causes tomorrow and they wont, even under the most extreme anti-aging regimen. But if they do, thats only 100,000 extra people per day sticking around. (Around 150,000 people die every day, roughly two-thirds of them from age-related causes.)

Compare that to the worlds current growth rate. More than 350,000 babies arrive every 24 hours. Earth's population is growing because of the size of the average family in the developing world, not because more people are living longer. The main way to bring it down is to educate more women and move more families into cities where, by the way, we shouldnt blame Baby Boomers for the lack of housing. We simply need to build more.

Total human population should level off at around 11 billion around the time your century dawns, whether or not the aged continue to die. And as for the threat of climate change well, perhaps the older generation will start to pay more attention when theyre actually going to live with the effects themselves. Or when they have to look their great-great-grandchildren in the eyes and explain their inaction.

Secondly, a healthy longevity boom would actually take an enormous burden off the healthcare system. Reducing just one of the major killers like heart disease, even by 10 percent, could savetrillions of dollars, money that can then be reinvested in medical research or just returned to patients in the form of lower costs. And thats the whole point of treating aging as the ultimate disease, the one that effectively produces all the others. (For example, Sinclair writes, smoking makes lung cancer five times more likely, but just living from 20 to 70 increases your chances of getting the disease a thousandfold, even if youve never sucked on a cancer stick.)

Aging is by far the biggest risk factor in any disease, by an order of magnitude, Sinclair says; having volunteered in nursing homes with his wife, he knows whereof he speaks. Dont delude yourself: Getting old and getting sick is not fun, for you or for your family. So I believe we have an obligation to preserve our health for as long as possible.

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The end of aging: Are you ready to live to 150? - Mashable

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Rocket Pharmaceuticals (RCKT) is a best in class gene therapy company with five shots on goal and strong data to support its current valuation. The two largest assets, RP-L102, a lentiviral gene therapy for Fanconi Anemia and RP-A501, an AAV gene therapy for Danon Disease are each worth multiples of the current share price, if successfully commercialized. The management team is highly experienced and have successfully commercialized many products at predecessor companies. The board of directors are both experienced and proven money makers on wall street in the world of biotech. The shareholder base is strong with top quality investors and the company has sufficient cash on the balance sheet for at least two years, during which multiple value drivers will report out. Commercialization of the most advanced products could occur in the 2021 timeframe. While never an investment attribute alone, I would note that there have been multiple acquisitions in gene therapy during the last 18 months (AVXS, ONCE) at eye-watering valuations and large cap pharma is struggling to find pipeline assets and return on productivity for internal pipeline assets remains at a multi decade low.

This report provides an overview of the company and details of the most advanced product in development, RP-L102 for Fanconi Anemia, as this is the primary focus for investors currently. The company's largest pipeline asset, RP-A501 for Danon Disease will become a focus for investors during 2020.

RCKT has Five Programs. Four will be in the Clinic in 2019

Source: Company data

Pipeline has > $1bn in Revenue Potential

Source: Company data, my estimates

Plenty of Catalysts Anticipated During Next 12 months

With five assets either in, or almost in the clinic, there are multiple catalysts expected during the next twelve months.

Source: Company data, my estimates

The company finished 2Q 2019 with $257 million of cash on its balance sheet and during the last 12 months the company burnt $66.5 million of cash. This is expected to increase during 2020 and 2021 as multiple pivotal trials start and consensus forecasts suggest that the company will spend $99 million in 2020 and $98.5 million in 2021. Therefore the company has sufficient cash on its balance sheet for approximately 2.5 years during which time, there will be multiple clinical catalysts that will hopefully drive the share price higher, allowing the company to raise additional equity in late 2020 to fund the company to break even in the 2023 timeframe. In the current environment, investors need to avoid any company that requires substantial financing.

Rocket Pharmaceuticals trades with a market capitalization of just $546 million. As of June 30, 2019, the company had cash of $ 258 million and debt of $ 46 million. Compared to other companies in the gene therapy space, RCKT trades at a significant discount. The company is well capitalized with approximately two years of cash on the balance sheet and there are a number of value creating catalysts during the next 12 months. Additionally, whilst never a reason to solely own a biotech company, I would note that there have been a number of acquisitions in the gene therapy space during the past few years. Large-cap pharma and biotech is short on products and long on cash and they need to make acquisitions.

Selected M&A in the Gene Therapy Sector: 2016-2019

Source: Bloomberg, Company data

RCKT is currently covered by 8 Wall Street Sell Side analysts, as shown below. Notably, Large banks including Goldman Sachs, Jefferies, JP Morgan, Morgan Stanley, Citi and Barclays Capital are all missing. As the company evolves into a commercial company during the next several years, it is likely that some of these brokers will initiate coverage of the stock, thereby improving liquidity.

Source: Bloomberg

As with all biotechnology stocks, there are significant risks associated with this investment and under a worst case outcome, there is 100% downside. The most obvious risk is that the pipeline products fail in clinical development. While Rocket has five assets in its pipeline, and success in any one of these is likely enough to justify the current valuation, negative clinical trial data would clearly have a negative impact on the company's share price. Under the outcome that all five pipeline assets fail in development, the stock is likely worth zero.

We are also in an uncertain political environment with an election looming in 2020. It is unlikely that either party will be arguing for higher drug prices and biotech stocks often underperform during these periods. Investors can mitigate this risk by being short a number of lower quality biotech companies and long a number of higher quality biotech companies. In my opinion, investors need to be long biotech stocks that are financed through 2021 and have multiple catalysts during the next 12 months. Being short companies in the opposite camp likely generates a good return as well.

Currently this company is not really exposed to foreign exchange rate or interest rate risks but these factors may become relevant in years to come.

This report will start with a primer on exactly what gene therapy is and then a detailed analysis of Rocket's lead asset where clinical data has been evolving during the last 24 months.

Gene therapy refers to technologies that can insert genes into cells, thereby expressing the proteins encoded by the genes. Gene therapies consist of two key elements - the gene of interest, and a vector that carries the gene into the host's target cells. Over the years a number of vectors have been used, although most efforts now employ viruses to carry the target genes. In creating a gene therapy, most of the viral genome is replaced by the therapeutic gene of interest. This eliminates the ability of the virus to replicate and cause disease, and permits relatively large target genes to be carried. The manipulated genome is inserted into a viral vector and when the virus is given to a patient, it is taken up by the patient's cells where it delivers its DNA to the nucleus. The cell then makes the target protein using the new gene as if it were encoded by the cell's own genetic material. Importantly, this process of gene transfer can be conducted ex vivo or in vivo depending upon the application.

Although gene therapy has the potential to treat a wide range of conditions, orphan monogenic diseases are particularly well suited for this approach. There are a number of scientific, economic, and logistical attributes of severe, monogenic orphan diseases that make them ideal candidates for the development of gene therapies by small biotechnology companies. First, by their nature as monogenic diseases, their causes are defects in a single gene. The pathogenesis of the disease is often well understood, and its treatment can be straightforward: by placing a functional copy of the gene in affected tissues, the disease process can be functionally cured/halted. Second, as orphan disorders affect a relatively small number of patients, on the order of several thousand individuals, the clinical trial programs can be conducted in tens of patients, rather than thousands. Such trials are less expensive to run and the logistics are within the capabilities of even small biotech companies. Third, most monogenic orphan diseases have no currently available disease altering therapies. Therefore the unmet need is high and any safe and effective therapy will likely be embraced. Fourth, the FDA has been flexible in its requirements for licensure in severe orphan diseases, routinely granting accelerated approvals based on surrogate markers that are reasonably likely to predict clinical benefit. Finally, innovative, and effective orphan therapies still have pricing flexibility in most worldwide markets such that companies can achieve attractive risk-adjusted returns on their research and development investment. Therefore, the orphan business model is well established and has repeatedly generated high returns for small cap biotechnology companies.

Rocket is building a comprehensive gene therapy technology platform to address serious, rare diseases. Rocket is developing both ex vivo lentiviral-based gene therapy technologies as well as adeno-associated virus (AAV) technologies to be used in vivo. Rocket also has early preclinical efforts in gene editing such as CRISPR/Cas9 (Clustered Regularly Interspaced Short palindromic Repeat/CRISPR-associated protein-9 nuclease) in its pipeline.

RCKT is Focusing on both In Vivo and Ex Vivo Gene Therapies

Source: Company data

What is a Lentiviral Vector?

Lentiviruses are a genus of retroviruses that includes the human pathogen human immunodeficiency virus (HIV). Like all retroviruses, lentiviruses are RNA viruses that encode reverse transcriptase (RT). Once a virion infects a cell, RT converts the virus' RNA genome into a DNA copy. This DNA copy is then integrated into the host genome using the virally encoded integrase. Once integrated into the host genome, the virally encoded genes are expressed and copied alongside host genes using the normal host gene expression and replication machinery. Lentivirus-based gene therapy approaches seek to co-opt the viral integration process to stably introduce genes of therapeutic interest into the human genome. Unfortunately, every insertion event is associated with a theoretical risk of causing disease (insertional mutagenesis) due to disruption of the host genome at the site of integration. As a result, lentiviral gene therapy programs take several steps to limit the ability of the virus to generate unnecessary insertion events.

Lentiviral (and retroviral more generally) gene therapy is most often deployed in an ex vivo process whereby cells are removed from the body, transfected with a lentivirus encoding the gene of interest, and then reintroduced into the patient. In Rocket's programs, it is transducing hematopoietic stem cells (HSCs) isolated from patients with defined monogenic diseases in order to insert a normal copy of the gene that is defective in these patients. The transduced HSCs are then infused back into the patient so that they will engraft. Although historically the patient's native hematopoietic system is ablated to improve engraftment, Rocket and its academic collaborators have pioneered a lentiviral approach that requires no or minimal chemotherapy.

HSCs are a self-renewing cell type that reconstitutes the patient's hematopoietic system, thus providing permanent, life-long expression of the normal gene from this one-time treatment. Because HSCs differentiate to form a variety of terminal cell types, this general approach is potentially applicable to a variety of genetic diseases in a modular, repeatable fashion. The ex vivo use of HSCs rather than in vivo treatment of all cells dramatically reduces the number of insertion events required to generate a therapeutic effect thereby reducing the risk of insertional mutagenesis. In addition, Rocket's use of the patient's own cells (an autologous transplant) is an important attribute of lentiviral gene therapy, as this should avoid some of the serious immune complications associated with allogeneic transplants such as graft-versus host disease (GVHD), which require management with harsh immunosuppressive therapies and can be fatal.

The lentiviral vector Rocket uses is based on the HIV virus. The vector takes advantage of the virus' natural ability to integrate into the host genome in both dividing and nondividing cells in order to efficiently deliver the chosen genetic payload. However the vector has been modified in a number of ways to render it nonpathogenic. Virtually all the viral genes have been removed to make room for the transgene and eliminate the virus' ability to replicate. The infectious viral particles are generated by co-transfecting producer cells with separate plasmids containing the "gutted" viral backbone and transgene, the viral capsid proteins and viral polymerase to make viral RNA from the DNA plasmid, reverse transcriptase to make DNA from the virus' RNA, and VSV - a pantropic envelope protein that allows infection of a variety of human cell types (not just CD4+ T cells). This results in the production of infectious viral particles carrying the viral RNA, reverse transcriptase protein, and viral integrase protein. When the virus infects target cells, it is thus able to undergo the process of reverse transcription and integration into the genome, but because the natural viral genes are not present, it can only undergo this single cycle of transduction and cannot replicate or infect other cells. To make doubly sure of this, the terminal ends of the viral genome are also modified to be "self-inactivating," so that they would no longer be recognized for excision even if the necessary viral proteins were to become present in the cell. Thus, the transgene is stably inserted into the host genome. For those readers who would like additional information on lentival gene therapy I recommend you reed this report available on PubMed. Kenneth Lundstrom does a great job discussing the pros and cons of each approach.

AAV is a naturally occurring non-pathogenic virus that is not known to cause any disease in humans. AAV has a number of advantages as a delivery vehicle for in vivo applications of gene therapy. AAV vectors do not replicate inside the host cell, preventing their spread to unintended tissues, and they typically integrate at a very low level into the host cell's genome, reducing the risk of insertional mutagenesis. Moreover, cellular tropism can be effectively modulated by using the natural tropism of different AAV serotypes, synthetically engineering the AAV capsid, and/or altering the transgene's promoter sequence. AAV vectors are also able to transduce non-dividing cells (such as RPE cells in the retina), and once incorporated into a host cell, they can drive the expression of a therapeutic protein for years. Last, AAV vectors can carry a good amount of genetic material, up to 4.5kb permitting them to target a range of indications. Since AAVs are non-replicating and generally non-integrating, the viral genome is typically not copied when an infected cell divides. Therefore, there is a theoretical risk that the efficacy of AAV based therapy in dividing cells could wane as an increasing number of divisions occurs.

A large number of clinical trials of AAV gene therapy are either under way, or have been completed. Applications have been diverse, ranging from hemophilia to REP65-mediated blindness and Parkinson's disease. AAV is versatile, and can be delivered through a number of routes of administration including intravenous, intramuscular, intrapleural, intravitreal, subretinal, and intracranial. For example, in lysosmal storage disorder (LSD) and hemophilia, AAV gene therapies are delivered systemically via intravenous (i.v.) route of administration and liver cells are transduced. In more localized diseases such as retinal dystrophy, choroideremia, X-linked retinoschisis (XLRS), the gene therapies are directly injected into the eye. In advanced Parkinson's disease, the gene therapy candidate is injected intracranially.

Fanconi Anemia - A rare disease with limited treatment options and a median survival of 29 years

Fanconi Anemia (FA) is a rare autosomal recessive DNA repair-deficiency syndrome characterized by aplastic anemia and progressive bone marrow failure. Though FA is a blood disorder, broad complications across a number of organ systems are associated with the syndrome such as defects of the eyes, ears, bones, kidneys and the heart. Perhaps most important, up to 30% of patients with FA develop leukemia, myelodysplastic syndrome (MDS), and or solid tumors at ages between 5 and 15. The median life span for FA patients is approximately 29 years.

Disease Progression: Unmet need for a treatment for FA

Source: Kutler et al, Blood 101:1249, 2003

FA is a complex disease with abnormalities in at least 18 genes associated with the disorder. These genes typically belong in the FANC gene family (FANC A-G, FANC CJ, FANC CL, and FANC M). The FANC gene family is associated with the DNA repair pathway. A mutation in any of these genes renders cells unable to properly repair damaged DNA. FANC A, B, C, E, F, G, L and M) form a nuclear complex termed the FA core complex. The FA core complex is required for monoubiquitination of the FANCD2 protein. Monoubiquintination of the FANCD2 protein allows for FANCD2 to translocate to sites of DNA damage to facilitate BRCA2/FAN CD1 and FANC E function in homologous recombination for DNA repair. Due to mutations in this DNA repair machinery, FA patients are simply unable to repair DNA damage that occurs naturally as cells divide, are exposed to mutagens, etc. Depending upon the exact DNA insult that occurs, unrepaired DNA can lead to abnormal cell death (most commonly) or uncontrolled cell growth. The abnormal cell death in turn creates FA's characteristic anemia and other organ defects. In other cases unrepaired DNA damage leads to uncontrolled cell growth and the development of a leukemia, tumor, or MDS. While it is extremely uncommon for any one DNA insult to generate cancer rather than cell death, DNA damage is occurring constantly within millions of cells in any human. Therefore, with millions of potentially oncogenic unrepaired mutations occurring it is unsurprising that FA patients have a significantly increased risk of developing cancer.

Approximately 60% of FA cases are due to mutations in the FANC A gene (the specific genetic abnormality that Rocket's lead program addresses). Approximately half of FA patients are diagnosed prior to age 10 while about 10% are diagnosed during adulthood. The remaining ~40% of FA patients are diagnosed during their teenage years. Birth defects such as undeveloped skull, eyes, or abnormalities in radial bones, kidney, skeleton, or skin pigmentation often facilitate early diagnosis. The definitive test for FA is a chromosome breakage test using crosslinking agents (dieposxybutane or mitomycin C) in isolated patient blood cells. While blood cells from healthy volunteers are able to correct most of the crosslinking agent induced DNA damage, FA patients' cells are incapable of correcting the damage from DEB or MMC treatment. Other methods of diagnosis include the use of molecular genetic testing on the 18 genes associated with FA such as sequencing analysis. The only curative therapy for FA is hematopoietic stem cell transplantation (HSCT) (there is good information on this here).

However, HSCT has a number of notable difficulties and complications. For one, it can be difficult to find a matched donor so that the transplant can be performed with a reasonable likelihood of success. Even when a suitable match is found, HSCT confers a high degree of morbidity and mortality, particularly in FA patients. Recent advances in conditioning regimens and supportive care have reduced treatment-related mortality from 38% or higher to 5-10% at most centers; nonetheless, such rates of death due to the procedure are notable. Moreover, HSCT can have major short and long-term complications including veno-occlusive disease, infections, infertility, secondary malignancies and graft-versus-host disease. GvHD can be particularly problematic and can evolve into a life-long condition causing serious damage to the lung, skin and mucosa. In severe cases GvHD can also be deadly. Conditioning chemotherapy is also inherently mutagenic and is therefore associated with additional risk of tumors developing post-transplant (secondary malignancy). FA patients are unable to repair these mutations that occur throughout the body during conditioning. Therefore HSCT confers a particularly high risk of secondary malignancy to FA patients. For example, the chance of an FA patient developing a new malignancy such as squamous cell carcinoma is estimated to be ~4x higher post HSCT. Thus, while HSCT is curative of FA's characteristic hematological manifestations, "cured" patients remain at an elevated risk of experiencing morbidity/mortality.

There can be spontaneous improvement in a small fraction of FA patients due to somatic mosaicism. Somatic mosaicism results from the spontaneous, random mutations that occur during normal cell division and proliferation. The cells clonally derived from the initial mutant cell have a different genotype than their neighbors. Somatic mosaicism has been reported in patients with FA. In cells of FA patients, the reversion of a pathogenic FA allele to a functional wild type allele confers a survival advantage on the cell vs. its non-reverted sibling cells. The cell(s) with the wild type reversion exploit this survival advantage to gradually populate the bone marrow. Up to 10-15% of FA patients develop somatic mosaicism resulting in disease stabilization or even improvement in bone marrow function for a prolonged period of time. This observation supports the theory that a very small percentage of corrected cells is sufficient to change the clinical course of FA. Somatic mosaicism therefore provides a rational as to why gene therapy may be successful in the treatment of FA patients and RCKT refer to somatic mosaicism as natural gene therapy.

Somatic mosaicism in FA leads to stabilization/correction of blood counts, in some cases for decades. This uncommon variant results from a reverse mutation and demonstrates that a modest number of gene-corrected hematopoietic stem cells can repopulate a patient's blood and bone marrow with corrected (non-FA) cells.

Source: Soulier, J., et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

Commercial launch likely in 2021/22 with >$1bn potential.

RP-L102 is a lentiviral vector that employs the phosphoglycerate kinase (PGK) promoter to express the FANCA gene. Expression is further facilitated by inclusion of the Woodchuck Hepatitis virus posttranscriptional regulatory element (WPRE). RP-L102 was licensed from the Centro de Investigaciones Energeticas, Medioambientales Y Technologicas (CIEMAT) in Madrid, Spain. CIEMAT is the Investigational Medicinal Product Dossier (IMPD) sponsor of the ongoing Phase I/II FANCOLEN-1 study of RP-L102 in patients with FA. Rocket is entitled to the data and commercial rights to the drug product generated under the CIEMAT sponsored IMPD.

RP-L102 gene therapy could have significant advantages over HSCT for FA patients. Perhaps the most notable advantage is that RP-L102 is being developed by Rocket and its academic collaborators without the use of bone marrow conditioning with chemotherapy agents. In contrast, all HSCT protocols require chemotherapy conditioning. The lack of conditioning confers a number of advantages. For example, without the use of chemotherapy agents, patients do not need to be hospitalized, and treatment can occur outside of a transplant-unit. Most important, FA patients have a diminished ability to correct damage to genetic material like that typically caused by chemotherapeutic agents. Therefore, by avoiding chemotherapy conditioning, the FA patients should not have an increased risk of head and neck cancer or leukemia. Moreover, because of their toxicities in FA bone marrow transplants are indicated specifically for patients with signs of bone marrow failure. RPL102 should enable treatment earlier in the disease course, well before bone marrow failure. This will allow patients to avoid the risks associated with the low blood counts of bone marrow failure, including anemia, infections and hemorrhages.

Gene Therapy Value Proposition: Early, Low-toxicity Intervention to Prevent Hematologic Failure

Source: Company data

RCKT recently presented data at the American Society of Hematology of the first four patients treated with RCKT's lentivial gene therapy for FA.

Bone Marrow Engraftment: Increasing Levels Provide Evidence of Potential Survival Advantage of Gene-Corrected FA Cells

Source: Company data

Increases of Corrected Leukocytes Support Restoration of Normal Bone Marrow Function Consistent with Mosaic Phenotype

Source: ASH 2018

Functional Correction of Bone Marrow

Source: ASGCT 2018

RCKT is a best in class gene therapy company with multiple shots on goal. During the next 12 months, data will likely emerge on many of these assets and if successful, should lead to considerable upside. This report focuses on the company's lead asset and data that has been presented to date is extremely supportive of a likely successful outcome, which would lead to considerable upside. As with all biotech investments, there are obviously significant downside risks and the worst case outcome for this stock is that it ends up at zero. However, with 5 pipeline assets in development, this risk is lower than biotech companies that are reliant upon a single driver of value.

Disclosure: I am/we are long RCKT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

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Blast Off With Rocket Pharmaceuticals - Seeking Alpha

A new study has shown the power of genetic testing to pick out the best drugs for children with cancer to extend and improve their lives signalling a new era of precision medicine for young patients.

The pilot including more than 200 children found that half had gene mutations that are targetable by adult cancer drugs that are either available as standard treatment or via clinical trials.

Although few children on the study went on to receive adult drugs, those who did receive targeted therapies had significant benefits.

But the study also laid bare the regulatory and funding barriers to children receiving the newest drugs, as only 7 per cent of those with targetable mutations were able to access the appropriate adult drug.

The study was led by The Institute of Cancer Research, London, andThe Royal Marsden NHS Foundation Trust, and offered genetic testing of tumours to children as part of a clinical trial. Some 20 additional hospitals around the UK participated by sending childrens biopsies in for testing.

The research ispublished in theEuropean Journal of Cancertoday (Thursday) and was primarily funded by the parent-led charityChristophers Smileand theNIHR Biomedical Research Centreat The Royal Marsden and The Institute of Cancer Research (ICR).

Researchers used a gene panel test to read the DNA sequence of 91 genes that drive cancers growth and spread from 223 childrens tumour biopsies looking for potentially targetable mutations.

Solid tumours such as those of the brain, central nervous system, bone and muscle are rare but have much worse survival rates than childrens blood cancers such as leukaemia. Surgery is often not possible and treatment is limited to blunt instrument chemotherapies.

The researchers first validated the panel test, showing it to be than more than 99 per cent sensitive at picking up the 91 mutations, even with just 50 nanograms of DNA which is around 1,000 times less than the weight of a grain of table salt.

Using the test, they found 51 per cent of tumour samples tested had mutations that could be targeted by adult cancer drugs.

The most common potentially treatable mutations were in the genes ATRX, CDKN2A and CTNNB1 which were each found in 12 childrens tumours. MYCN mutations were found in 11 tumours and PI3K3CA mutations in 10 tumours.

Three children had BRAF gene mutations which are common in melanoma skin cancers and can be treated using a combination of the drugs dabrafenib and trametinib.

Using these melanoma drugs, one of the children had their brain tumour held in check for 13 months before developing resistance. Another was on the drug for nine months with no progression of disease. The third child couldnt tolerate the combination but had a response to dabrafenib for 15 months.

But there are still challenges to overcome, since the majority of children with targetable mutations didnt receive adult drugs because there was no trial available for the drug in children, they were unable to access the drug on the NHS or they were too ill to receive an experimental treatment by the time they were tested.

For eight of the patients, there were samples available at diagnosis and after treatment and in six of those, testing revealed that the cancer had acquired new mutations as it evolved in response to treatment. That highlights the need to take an additional biopsy at relapse to search for targetable mutations.

For 12 of the children, the researchers were also able to test for cancer gene mutations in DNA released from tumours into the bloodstream from a blood sample. They found blood tests picked up almost all of the mutations found in the tumour, and in some cases they also found extra mutations which were not detected in the tumour region biopsied.

In future work the researchers will use serial blood tests to monitor how tumours evolve in response to therapies which will be particularly useful in hard-to-biopsy tumours.

Additionally, for children with brain tumours, the researchers are now looking at using samples of cerebral-spinal fluid to find drug targets. Although lumbar punctures are invasive, they are less so than a brain biopsy.

Study author Dr Sally George, Clinical Research Fellow at the ICR and Consultant Paediatric Oncologist atThe Royal Marsden, said:

Children deserve the very best cancer treatments, so they can live as long as possible and as well as possible. We desperately need better, more intelligently designed treatments which can give children longer with their families with fewer side effects.

By testing tumours for specific gene mutations, we have shown its possible to identify new smarter, kinder treatment options for children, which may potentially give these patients much longer with their families after conventional therapies have failed.

But our study also exposes the desperately frustrating barriers that children still face in receiving new treatments barriers which lie in the regulations controlling how drugs for children are developed and approved.

Study leaderProfessor Louis Chesler, Professor of Paediatric Cancer Biology at the ICR, and Consultant at The Royal Marsden, said:

Our study has demonstrated that we have the scientific knowledge and technology to get children access to state-of-the-art testing and treatments. And because our testing currently only assesses a focused set of well-known and clinically meaningful mutations, it is more practical, faster and more cost-effective than looking at the whole genome.

In future, I want to be able to treat more children whose tumours have these targetable mutations with better drugs, as currently not all children have access. But gathering the molecular data is the first practical step to making this possible. This data, and more that we are continuing to collect, will be good evidence to more clearly guide use of the most appropriate drug for each child.

It is also very important that we extend very robust and detailed testing to children at time of diagnosis, so we can more accurately classify and treat these cancers in the first place. We will also be looking at the utility of the approaches for detecting cancer relapse, a very important area where we currently have few tools to anticipate what treatments may be required with adequate time to do so.

Dr Mike Hubank, Head of Clinical Genomics at The Royal Marsden and Reader in Translational Genomics at the ICR, said:

The next steps for testing will be to look at using liquid biopsies to detect targetable tumour mutations without having to rely on invasive biopsies to get the information.

Our early results, presented here, show that we can detect more mutations in blood than we do in conventional biopsies. It is probably in the blood that we get a more complete picture of the whole tumour, and not just the small part of the tumour that was removed for testing. Blood-based testing will also allow us to monitor tumour response to treatment and may be able to detect relapses early, offering the possibility of finely tuned, personalised treatments in the future.

Karen Capel is the founder and trustee of UK childrens cancer charityChristophers Smile, who funded the development of the test. Karen and her husband Kevin have campaigned tirelessly to improve the treatment for children with cancer after their son Christopher died from medulloblastoma in 2008. Karen said:

When our son died there was no biological information available to doctors about individual childrens tumours. There is an urgent unmet need to provide new treatments for those children diagnosed with the most aggressive and hard-to-treat cancers.

This test Professor Chesler and colleagues at the ICR developed is a first for children. We believe gene sequencing is the key foundation stone in enabling personalised medicine, and it will help to bring new treatments for children a step closer.

Building on the foundations of the sequencing test, blood tests could provide critical information for any child from diagnosis throughout their treatment and into remission opening the door for additional, continued or changed treatments. We are determined to fight for these liquid biopsies to become standard of care at the earliest opportunity.

Reference: George, et al. (2019) A tailored molecular profiling programme for children with cancer to identify clinically actionable genetic alterations. EJC. DOI: https://doi.org/10.1016/j.ejca.2019.07.027

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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The Power of Genetic Testing Picking Out the Best Drugs for Children With Cancer - Technology Networks

If you found out today you were at a high risk of being diagnosed with Alzheimers disease, what would you do? Crawl under the covers? Take that exotic vacation? Wish youd never found out?

Or would you change the way you live?

When a genetic test revealed that Jamie TenNapel Tyrone had a 91 percent chance of developing Alzheimers, she slipped into a deep depression. She thought about suicide.

Tyrone had been worried about multiple sclerosis. Thats why she took the test, to see if she had any of the genes associated with MS following some neurological symptoms. At 49 years old, Alzheimers disease wasnt anywhere on her radar.

I was devastated, Tyrone said. She was also critical about the way the 2009 study was designed, without genetic counseling before or after, leaving her completely unprepared for the shock of her results.

Then she surprised herself. She wrote a book about her experience to guide others through the pros, cons and pitfalls associated with genetic testing. It was co-written with geriatric neurologist Marwan Noel Sabbagh.

I think people now can get commercial genetic testing (services) like 23andMe and not understand the consequences of what theyre getting tested for, Sabbagh said.

Fighting for my Life: How to Thrive in the Shadow of Alzheimers describes the emotional journey Tyrone went through after participating in genetic testing as part of a research study 10 years ago.

The basis for the study was to find out if, or how people would change their lifestyle if they knew they had a high risk of disease by using genetic testing. The research showed that knowing the risk of disease did not lead to significant lifestyle changes.

Without the counseling, Tyrone said she felt abandoned by researchers when she found out she has two copies of the apolipoprotein E4 gene, which is linked to an increased risk for Alzheimers.

Afraid of the stigma associated with the disease, she avoided discussing her results at first, fearing that shed be discriminated against or ostracized.

There are laws that protect people from certain forms of discrimination related to genetic information, but theyre not all-inclusive.

The Genetic Information Nondiscrimination Act protects individuals from being denied health insurance or charged higher premiums based only on the genetic predisposition to a disease. It also means that employers cant make decisions to hire, fire, segregate or otherwise mistreat employees based on the results of genetic testing.

But it doesnt protect access to long-term care, life or disability insurance.

For three years I didnt talk about it and I became very depressed, said Tyrone, who lives with her husband in Ramona. I contemplated not being in the world.

After years of depression, suicidal thoughts, therapy and being diagnosed with post-traumatic stress disorder, Tyrone now volunteers as a research collaborator, or what she calls a lab rat. She wants to help scientists find a cure for the disease and hopes that participating might help her to prevent the onset of the disease, or cure her someday if needed.

The first person cured of Alzheimers disease is going to be a research participant, Tyrone said, paraphrasing UsAgainstAlzheimers founder George Vradenburg.

If she had it to do over again, would she take the test? Tyrone said she probably wouldnt.

I truly believe that if I had genetic counseling during the consent process, I would have chosen not to be tested, she said.

Even still, Tyrone said finding out about her risk has been a gift in some ways because it prompted her to help others through public speaking and writing her book. She hopes her story can help people to decide if and when to take genetic tests in search of potential health risks.

Co-author Sabbagh said he generally advises that genetic testing only be done if there are already symptoms present. He also suggests working to increase brain health early on before memory loss symptoms begin.

The fact is that by the time you come into the office to get seen for your memory issues, the changes in the brain have been accumulating for a long time, he said.

Now 59, Tyrone said shes cognitively healthy, but still searching for answers to the mysterious neurological issues that led her to the testing in the first place.

Read the rest here:
When genetic testing goes sideways: One woman's cautionary tale - The San Diego Union-Tribune

The agency has expressed worry that unsupported claims about gene-drug links could be dangerous if they spur patients to start, stop, or switch medications in inappropriate ways. But the American Clinical Laboratory Association says the warnings could stifle a burgeoning industry.

Stat:Groups Push Back Against Troubling FDA Crackdown On Genetic TestsIn recent weeks, the genetic testing world has been rattled by the Food and Drug Administrations efforts to quietly pressure a number of companies to stop reporting results to patients about how their genes may interact with specific drugs. Now, increasingly, the industry is pushing back. On Wednesday, a trade group that represents clinical laboratories sent a sharply worded letter to the agency calling its enforcement actions troubling and inappropriate. The letter, from the American Clinical Laboratory Association, warned that the agencys actions could stifle the growth of a burgeoning industry and make it harder for patients to get the right types and doses of their medications. (Robbins, 9/18)

In other news on the FDA

Stat:FDA To Modernize Its IT And Make More Efficient Use Of DataDr. Amy Abernethy, the principal deputy commissioner at the Food and Drug Administration, is unveiling a three-point plan to radically redirect the agencys efforts at using computer technology. At a Wednesday meeting held by Friends of Cancer Research, a patient advocacy group, Abernethy, who is also the FDAs acting chief information officer, plans to outline how regulators can move more of their work to the cloud virtual servers and automate work that is now done by hand. This, she said, could change the way that the agency interacts with the companies it regulates. (Herper, 9/18)

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FDA's Warnings Over What Genetic Testing Companies Can Report To Patients Deemed 'Troubling' By Industry Group - Kaiser Health News

Federal authorities warn of a telemarketing scam that offers "free" cancer screenings that lead to Medicare fraud. Federal authorities warn of a telemarketing scam that offers "free" cancer screenings that lead to Medicare fraud. Related stories

FORT MYERS, Fla. - Federal investigators call it the latest scheme targeting consumers on Medicare and it all begins with claims of free screening for cancer.

Tammy Rose fears her 83-year-old mom, who lives in Sebastian, recently fell for the scam after receiving a call from a telemarketer.

"They started asking her questions like what did your mother die from? What did your father died from? What's your family history?"

Tammy's mom has heart issues, cancer runs in the family so when the telemarketer pitched free cancer screening, her mom bought in. A package of q tip swabs and a dispenser arrived a few days later.

"She was to swab one cheek corner and then swab the other cheek corner," Tammy said. Her mom was directed by the company to place the swabs in separate bags and send it back to the facility, which she did. A few days later, Tammy received a frantic call from her mom.

"My mom called me crying saying she just saw something on TV saying it was a fraud," she said.

On the west coast of Florida, complaints about fraudulent DNA cancer screening is now the number one call at the Area Agency on Aging for Southwest Florida says senior advocate Camalita Aldridge.

"We're seeing more and more of these cases. There are various forms of the scam. Folks can receive these kits at home whether they're solicited or not," Aldridge said.

Aldridge says companies who do this fraudulently are targeting Medicare patients by telling them each screening is free. These companies are screen patients for which cancer or genetic testing is not medically necessary and/or not ordered by the beneficiary's doctor. But every DNA screening test represents an opportunity to cash in since Medicare (taxpayers) will pay for it. Each screening can range from $10,000 per test to upwards of $30,000 says Aldridge.

"They work on someone fear," she said. "If a consumer thinks that if I can know ahead, diagnose and then treat, then I might prevent [cancer], scammers know this so that's what they do," she said.

While legitimate DNA cancer screening can be helpful for some people who qualify, experts advise any health screening should take place at your doctor's office and be approved by your own doctor not a stranger.

Cole Buckley of Fort Myers showed us several flags and fliers he had made for what he thought would be a lucrative DNA cancer screening business.

"I was going to be the man on the ground," he said. "I was going to go to different things like health fairs, flea markets and set up a booth where we can pre-screen people," he explained about the pitch he heard about in January.

Buckley said the company, which no longer offers DNA screening, told him to only target seniors on Medicare and a company doctor, who Buckley never met, would approve each test. Buckley said he spent $200 to sign on with the company and another $1000 in marketing for the flags, fliers and business cards he had made which all tout "free DNA cancer screening." But in the end, he said, the company pulled out and never sent him any cancer screening kits. Buckley said he felt duped.

"I thought I was going to make a lot of money," he said. When asked how much, Buckley said he was told by the company it would be "thousands per month."

Tammy Rose's mother doesn't know yet if Medicare was charged for her screening. She hasn't received any results from her so-called "free" screening but she did file a police report and Tammy reported the company to Medicare knowing if her mother fell for it, chances are, thousands of others are doing it too.

"I'm angry that my mother was targeted. Someone's getting rich off this and they need to be in prison," she said.

How to protect yourself from falling victim to "free" cancer screening - If a genetic testing kit is mailed to you, don't accept it unless it was ordered by your doctor or return it to the sender. - Keep a record of the sender's name and date of your returned items. - Be suspicious of anyone who offers you "free" genetic or cancer screening and then requests your Medicare number. - Be cautious of unsolicited requests for your Medicare numbers. Don't provide your Medicare information to anyone you don't know.

- If you suspect Medicare fraud, contact the Office of the Inspector General.

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Genetic testing latest target for fraud - KTVZ

DENVER Sisters Marcy and Elissa Newman have always known they had strong familial ties to breast and ovarian cancer.

Our grandmother was a two-time breast cancer survivor, and we had several aunts that had breast cancer and had passed away, Marcy said.

Even so, the sisters only got tested for the BRCA gene after Marcy was diagnosed with ovarian cancer at the age of 47. The gene, often known as the breast cancer gene, actually raises the risk for multiple types of cancer.

When Marcy found out she was BRCA1 positive, she called her sister.

As we were hanging up the phone she said, 'By the way, Im BRCA1 positive, and you have a one in two, or 50 percent, chance of having the gene,' Elissa said.

She said she was encouraged to get tested even earlier in life because of her family history, but at the time, insurance laws allowed companies to discriminate based on a pre-existing condition.

Thats no longer the case, but genetic counselor Lisa Mullineaux said people are still fearful of getting genetic testing for other reasons.

They may not be ready to know that they have an increased risk, or theyre not ready to take action, said Mullineaux.

Some people who find out they have the BRCA gene may simply opt for additional screenings or may take birth control pills to reduce their ovarian cancer risk. But Elissa decided to take more extreme action. She had her ovaries, Fallopian tubes, uterus and breasts removed.

To reduce my risk to that of the natural population, was well worth it, she said.

Elissas 20-year-old will soon get tested for the BRCA gene, because men who have the gene face an increased risk for prostate, pancreatic and other cancers.

The United States Preventative Services Task Force recently updated the recommendations for BRCA1 and BRCA2 testing to include more of the population. And since its now considered preventative, the testing may be covered by insurance.

Marcy is celebrating 5 years as an ovarian cancer survivor, but wishes shed been told about all her options earlier.

I would have expected an expert who knew my family history would have had me take additional steps and screenings, she said. Now, I know we have to advocate for ourselves and educate others.

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Sisters encourage women, men to get tested for cancer gene - The Denver Channel

CHICAGO (CBS) The price of DNA testing to see if we are genetically more likely to develop certain health complications is dropping, but the benefits are still out of reach for many who cant afford it. One area hospital is now offering the tests free of charge to help patients better prepare for the health risks ahead.

CBS 2 Morning Insider Vince Gerasole took the test and shares the changes he now has to make in his life.

The world of genetic testing can save lives, helping patients detect mutations in their DNA that could lead to certain types of cancers and other medical complications.

The benefits, they can be many, said Dr. Peter Hulick, medical director of the Mark Neaman Center for Personalized Medicine at NorthShore University HealthSystem.

Youve seen the commercials; once costing thousands of dollars, DNA tests are now marketed to the masses at prices a bit more reasonable, most near $200. Its still a price not all can afford; minorities, especially, have been under-represented in the populations taking the tests.

Thats part of the reason why we opened this to our primary care network, so anyone of any ancestry can have access to this information, Hulick said.

NorthShore University HealthSystem is now providing DNA screening free of charge through a program called DNA 10K.

It aims to make interpreting the results and taking action if necessary part of standard health care.

What it allows us to do is to make a better plan, so that you can be proactive with this information so that we can get you on the right screening and prevention path, Hulick said.

CBS 2s Vince Gerasole participated in the process. Following a quick blood draw, his sample was sent to the labs at health technology firm Color. In a few weeks, a genetic counselor reviewed his results by phone.

The results included a significant red flag: several women in Gerasoles immediate family have died from breast cancer, so it was not much of a surprise that he tested positive for the BRACA 1 mutation, making him more likely to develop male breast cancer, as well as pancreatic and prostate cancer.

Genetics is not a crystal ball. It doesnt predict your life. It doesnt tell you when or exactly if something is going to occur, Hulick said.

However, the results can help patients consult with their doctors and make choices ranging from lifestyle to more frequent medical screenings to preventative surgeries like breast removal.

Its not destiny. Genetics isnt destiny, Hulick said.

But if available to more patients, it could be destined to help.

Call 847-570 GENE (4363) to learn how you can participate.

Originally posted here:
DNA Testing Can Help Detect Possible Health Risks; Genetics Is Not A Crystal Ball - CBS Chicago

16 September 2019

Seventeen former employees of a genetic testing company, Orig3n, have accused it of not meeting scientific standards and returning inaccurate results.

In an interview with Bloomberg Businessweek, the employees, who had previously been managers, lab technicians, software engineers, marketers, and salespeople, said that the company's test results could not be replicated for the same person. The company, based in Boston, Massachusetts, allegedly built software to automatically return the initial outcome if a repeat analysis did not match. A technician has reported a document of 407 such errors over a period of three months from fewer than 2000 tests.

'Accurate science didn't seem to be a priority,' a former lab technician told Bloomberg. 'Marketing was the priority.'

Orig3n's genetic tests, costing between US$28 and $298, are used to advise customers on lifestyle choices and help them to identify their genetic predispositions and the 'superhero traits' that they are most likely to have.

The employees claim that the advice provided to customers was often generic good health practice collected from the internet or had limited scientific evidence. Examples included eating kale, wearing sunglasses, or eating sugar and almond oilto reduce stretch marks.

Co-founder and CEO of Orig3n, Robin Smith, said the company 'wholeheartedly' disputed these assertations. He said the company has operated under the Clinical Laboratory Improvement Amendments (CLIA) - US federal testing regulations to ensure proper practice, since November 2017 and that many of the claims concern a period before this.

'Orig3n had to change many of its laboratory staff precisely because they simply wanted to "do things their way", rather than in compliance with CLIA,' he said. He also offered to review any tests if customers have concerns. The former employees were at the company between the summer of 2015 and autumn 2018.

Orig3n was previously singled out as part of an NBC Chicago investigation into genetic testing in 2018. When the reporter submitted genetic material from a Labrador retriever called Bailey, the company produced a seven-page report about her genetic traits, stating that she had strong muscle force and cardiac output for long endurance, while failing to note that she was not a human. Other companies said that the genetic material was 'unreadable'.

Read more from the original source:
DNA company accused of fudging people's genetic test results - BioNews

--(BUSINESS WIRE)--Wolters Kluwer Tax & Accounting:

What: In recent years when presented with the opportunity, the Internal Revenue Service (IRS) has identified a number of expenses as qualifying medical expenses for purposes of the itemized deduction for medical expenses or for qualified distributions from health savings accounts or flexible spending accounts. These have included smoking cessation programs, weight loss programs, and gluten-free products for celiac disease. Now, in a private letter ruling, the IRS has spelled out the circumstances under which genetic testing might qualify as a medical expense.

Why: While private letter rulings cannot be relied upon by taxpayers other than the taxpayer to whom it was issued, the ruling on genetic testing does indicate the IRS thinking on the matter and how they might treat similar situations:

Who: Federal tax expert Mark Luscombe, JD, LL.M, CPA, Principal Federal Tax Analyst at Wolters Kluwer Tax & Accounting, is available to discuss these developments with respect to genetic testing and qualified medical expenses in general.

PLEASE NOTE: The content of this article is designed to provide accurate and authoritative information in regard to the subject matter covered. The information is provided with the understanding that Wolters Kluwer Tax & Accounting is not engaged in rendering legal, accounting, or other professional services.

Contact: To arrange interviews with Mark Luscombe, other federal and state tax experts from Wolters Kluwer Tax & Accounting on this or any other tax-related topic, please contact:

MARISA WESTCOTT212-771-0853marisa.westcott@wolterskluwer.com

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MEDIA ALERT: IRS Approves Medical Expense Deduction for Genetic Testing - Business Wire

The ConversationSep 21, 2019 16:50:49 IST

Dramatic developments in genetics research and the availability of commercial genetics tests have put us in a very modern predicament we can now find out (quickly, easily and cheaply) whether we personally hold genetic risk factors that put us at a substantially increased risk of Alzheimers disease. In addition, we have recently shown that brain changes can be identified in people holding these genetic risk variants as early as 20 years old.

Should we be testing ourselves? Should we worry? No. Heres why:

Representational image credit: StunningArt/Shutterstock

Genetic research has revealed that some individuals carry variants of specific genes that confer an increased risk of developing Alzheimers disease in later life. For example, carriers of the 4 variant of the APOE gene are approximately three to eight times more likely to be diagnosed with Alzheimers disease after age 60 than individuals without this variant. The more variants, the greater the risk with a maximum of one inherited from each parent.

In our recent research, we looked at these genetic factors in young people (around 20 years old, on average). We split them into higher-risk and lower-risk groups depending on whether they did or did not carry the APOE-4 gene variant, respectively.

Using advanced brain imaging techniques, we were able to show that it is possible to detect subtle differences in particular brain networks for the higher-risk young adults, several decades before any clinical symptoms of Alzheimers emerge.

While brain structure and function were significantly different between the risk groups on average, it is very important to point out that not all higher-risk individuals go on to develop Alzheimers disease. (Note that we say higher not high risk.)

The brains of many of these individuals were comparable to those at lower risk. This means carrying a higher-risk gene variant does not necessarily lead to early brain changes, or an Alzheimers diagnosis later in life.

Public interest in genetics and gene testing is booming. Recent times have also seen highly publicised incidences of people responding to their own genetic risk with drastic interventions. For instance, Angelina Jolie, who has a faulty copy of the BRCA1 gene, associated with breast cancer and had elective surgery to remove both breasts and some of her reproductive organs.

Direct to consumer genetic testing kits sold by companies now provide people with convenient and affordable access to their personal genetic information, including their genetic risk for specific diseases, including Alzheimers.

But the relatively low cost of these tests reflects the fact that they typically only cover a fraction of the genome. The results, therefore, neglect the contribution of the rest of the consumers genetic code. This will include other genes with protective, as well as negative, effects.

Oral swaps and saliva samples are used by Direct To Consumer commercial genetic tests. image credit: B-DSPiotrMarcinsk/Shutterstock

Of other concern, these tests have been shown to frequently generate false positive results: indeed, one study found approximately 40% of variants in a variety of genes reported in raw commercial genetic test data were false positives. This could lead to unnecessary distress, treatment and lifestyle adjustments. These tests also come with privacy and social concerns.

On the upside, the popularity of commercial genetic testing partly reflects consumers positive desire to be proactive about their health. Consumers concerned about commercial genetic test findings should, however, request confirmatory tests from their clinician. These consumers should also understand that the disease risk reports they have purchased at best provide a partial answer to the question they are trying to address, because disease risk is about much more than genetics alone.

The next step for our research is to find out what leads some people at higher-risk to go on to develop these early brain changes, but not others. Do these people exercise or sleep less, maintain a poorer diet, or have poorer social relationships? Many possible answers involve lifestyle factors that could potentially be altered to buffer individuals against their genetic risk.

The only way to properly understand which lifestyle factors may have such a protective effect is to study large numbers of people with varying degrees of genetic risk over several decades.

We are part of an international team of scientists undertaking one such study, led by Professors Kim Graham and Andrew Lawrence at Cardiff University. The project involves collecting advanced brain imaging and cognitive test data from a large group of approximately 240 young adults. These individuals are part of a cohort that has been studied since birth, so we can access a wealth of retrospective health and lifestyle data.

Can lifestyle factors like reading, exercise and socialising protect us from our genetic risks as we age? image credit: RomanSamborskyi/Shutterstock

Smaller, isolated studies looking at lifestyle factors might currently be missing the big picture. Brain differences have been found in these high-risk groups between people who do and dont exercise regularly. This could suggest exercise has a protective effect on the brain, and may, in turn, mitigate Alzheimers risk. It could also be that exercisers engage in other protective behaviours like eating a healthier diet. It is only with large-scale cohort studies that we can begin to disentangle the genetic and lifestyle contributions to cognitive performance, the brain and Alzheimers risk.

Finally, if you are considering making lifestyle changes to offset your genetic risk for Alzheimers, taking regular exercise and maintaining a healthy lifestyle is seldom bad advice. Other drastic lifestyle changes, however, are likely unjustified.

Mark Postans, Postdoctoral research associate, Cardiff University and Carl Hodgetts, Research Fellow in Cognitive Neuroscience, Cardiff University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Alzheimer's Day: Carriers of risky gene show brain changes in its 20s but we don't need to worry - Firstpost