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Archive for the ‘Bone Marrow Stem Cells’ Category

Stem cell therapy is for animals too – SciTech Europa

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa

More awareness needed on stem cell donation: expert – The Hindu

Hematopoietic stem cell transplantation (HSCT), popularly known as bone marrow transplantation (BMT), is a curative modality for a number of benign and malignant blood disorders, said Dr. Murali Krishna Voonna, surgical oncologist and managing director of Mahatma Gandhi Cancer Hospital and Research Institute.

Speaking at an awareness programme on stem cell donation organised here by the hospital, in association with Datri Blood Stem Cell Donor Registry, he said hematopoietic stem cells are immature cells that can develop into all types of blood cellswhite blood cells, red blood cells, and platelets. They are found in the peripheral blood and bone marrow.

A sizeable population are diagnosed to have benign diseases such as thalassemia major, sickle cell anaemia and aplastic anaemia, and the HSCT is among the efficient curative measures. Acute leukaemia and other blood cancers also need this procedure, he said.

Highlighting that stem cell donation and a registry are vital, Dr. Muralikrishna explained for a successful hematopoietic stem cell transplant, the patients genetic typing (HLA typing) needs a close match with that of the donor. Every patient has 25% chance of finding a match within the family, he said.

Dr. Muralikrishna stated that in such cases, finding a donor is a pressing need. There are over 80 donor registries and more than 30 million registered donors across the globe, with a very few Indians being a part of it. This reduces the chances of finding a possible match for patients of Indian origin. Patients are more likely to find a possible match within their ethnicity, which means people sharing the same cultural linguistic and biological traits, he explained.

The problem can be solved if the donors enroll themselves with a registry which will store the stem cell details and the details. Pledging to donate stem cells is easy like swabbing the inner-cheek. The donors are contacted if patients have HLA matching, he said, adding that the stem cell donation was carried out only when a match was found for a patient, not when one pledge to donate.

A blood stem cell collection centre was inaugurated at the hospitals premises on the occasion. Earlier, to avail of such service and for HLA-typing, one has travel to Hyderabad and Chennai, Dr. Muralikrishna said.

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More awareness needed on stem cell donation: expert - The Hindu

Stem cells treatment gives hope in fighting Autism, blood disorders – OrissaPOST

Bhubaneswar: The advanced treatment of using stem cells for treating Autism and other neurological ailments have come as a ray of hope for the people living with some of these ailments. Medical experts working in the sector claim that the use of the technology improved the lives of many.

According to experts who practice stem cell therapy, the results have been overwhelming. Many of the patients have either been able to fight a deadly disease with the help of stem cells while many have been able to improve their quality of lives by using it. However, the technology is still not used widely in state hospitals.

Medical experts claim that stem cells could be used to treat neurological disorders like Autism, cerebral palsy, mental retardation, brain stroke, muscular dystrophy, spinal cord injury, head injury, cerebellar ataxia, dementia, motor neurone disease, multiple sclerosis while it has also been used to treat cancers like blood cancer with the help of bone marrow transplant when assisted by stem cell therapy.

However, treatment of Autism with stem cells is a new developing sector where visible changes are said to have been reported among children treated with this technology. However, the advanced technology which is now confined to only private sector is a bit expensive.

Autistic kids are usually treated with drugs for symptomatic relief, special education, occupational speech and behavioural therapies. In Autism, despite the best available medical and rehabilitative treatments satisfactory relief is still a far cry, said Dr Nandini Gokulchandran, Head Medical Services, NeuroGen Brain and Spine Institute, Mumbai.

Dr Gokulchandran claims that she has treated many cases of Autism in kids with stem cells which helped in overcoming their limited abilities. Under the treatment regime, an insertion procedure is undertaken followed by training to improve the skills and abilities of autistic kids.

Another neurologist, Dr Richa Bansod said that in India it has been reported that 1 in every 250 children have Autism and this number in increasing with better recognition and awareness of the condition. On the other hand, stem cells are now been used to fight deadly diseases.

Dr Joydeep Chakaborty, an oncologist and stem cell expert from HCG Cancer Hospital, Kolkata said, Stem cells and bone marrow transplants are now being used to cure blood cancer in many cases. It is also widely used to treat blood disorders like Thalassemia, Sickle Cell Anaemia and others.

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Stem cells treatment gives hope in fighting Autism, blood disorders - OrissaPOST

Dr. Raj & Stem Cell Therapy Innovation – LATF USA

For anyone who has had hip replacement surgery, Im sure they will agree that it is better to get hit by a bus than to undergo another one. Last year after several years of suffering, I decided to take the leap and go for the hip replacement that my specialist recommended. I was told that it was a common surgery and that it was the best solution for me. Between us; it was probably the most painful thing I have ever gone through. So much so, that at the time, I just wanted to die. Not only did the pain persist for several weeks after the operation, but I was on painkillers for days, which eventually added to my suffering. I had to use a walker for the first 2 weeks and then depended on a cane for over 2 months before I could walk on my own.

My entire demeanor changed, as well as the way I dealt with what once were minor things in life. I feared slipping in the shower, going down the stairs or walking my dogs. No one had prepared me for this. Ive had my share of surgeries including a double mastectomy when I was diagnosed with breast cancer but pain wise; this one was by far the worse. I was hoping after a very long recovery that I would never have to face this situation again. Unfortunately, a year later, I am starting to feel pain on the other side and dread the re-experience of my nightmare.

Although, I heard about Stem Cell, I did not know much about it. So I started to investigate for myself, speak to people, enquire about the procedure and look for a doctor in my area who specialized in Stem Cell. I was willing to do just about anything before considering another hip replacement. After extensive research, I came across Dr. Raj, a Double-Board Certified Orthopedic doctor in Beverly Hills, CA. Going to his website; I learned that he has been in private practice for 10 years. He has been named as one of Americas Top Orthopedists, been featured on the Best of LA and has received numerous other accolades and awards as one of the Top Orthopedic doctors. Providing the ultimate in state-of-the-art orthopedic care, Dr. Rajs practice is always on the cutting-edge of surgical and nonsurgical technologies, such as PRP (Platelet Rich Plasma) injections, stem cell injections for tendinitis and arthritis, minimally invasive surgery and more.

He is Board Certified as a Medical Legal Specialist in America, as well as, Canada and Dubai (Trial, Testimony, Deposition, IME) with a Subspecialty in Hip and Knee Surgery in Los Angeles, including Sports Surgeries.

He is also an Undergraduate from Dalhousie University in Halifax and Canada. He pursued his medical education at Memorial University PGME, before doing his internship and residency in the Department of Orthopedic Surgery. Now that I had found Dr. Raj, all I needed was to get myself educated. So lets start by what are stem cells? This is what I read: Mesenchymal stem cells (MSCs), commonly called stem cells, are precursor cells that havent decided yet what they are going to be in the body. They can differentiate into multiple forms including bone, cartilage, fat and other connective tissues. They play a significant role in the reparative processes throughout the human body.

Where do we find stem cells?

They may be harnessed from fat tissue, bone marrow, synovial tissue or umbilical cord tissue. While stem cell therapy is a promising technology, there is much we are still learning about the causes and pathways that lead to symptomatic osteoarthritis. We have not optimized the factors found in stem cell therapies. To be sure, only the good cells and growth factors are injected into a specific joint. And that is why further research is necessary before being approved by the FDA.

My next move would be to consult with Dr. Raj who would tell me the medical truth, beginning with this question:

What is the current state of Stem Cells and its success rate?

It's relatively new. It's been popular for about 20 years, internationally. In areas like Germany and Korea, it was utilized a lot more. It became popular here when athletes like Kobe Bryant started going to Germany for modified versions of PRP, which led on to regenerative technologies. We have a stigma correlating stem cells with abortions and issues like that. This in itself is completely different. We are not utilizing amniotic stem cells or placenta stem cells. We're utilizing your own stem cells. For issues such as a hip replacement, the most powerful stem cells are the ones in your body. Bone marrow stem cells work well on joints. Joints have zero blood supply. So, if God or the higher power created us where we had blood supply going through our joints, like a cut in our skin - we would constantly replenish or repair. A break in our bone would repair. If you get stem cells and you're in decent enough shape, you will heal no matter what because these stem cells will deposit. Will you heal straight? Probably not - that's where we come into play.

The reason why joints; hips, knees and shoulders degenerate is because there is no blood supply. So, if you have a cut or a loss of cartilage, it stays like that and accumulates overtime. The only way you can control it is externally. You get stronger, you lose weight and you increase your range of motion. But you can't control anything internally.

So regenerative technology is basically utilizing these cells to regenerate cartilage and repair. These are the same cells that flow through our body - and upon signal of an injury will heal skin to skin, bone to bone, tendon to tendon, muscle to muscle. Our joints are just an alcove of joint fluid and no blood supply. The whole concept is - throughout the years, we did steroid injections - they're like band aids. Basically they mask pain. What does masking pain do? It propagates injury. Because we put the band aid on, we don't feel it and we do more. We take this little cut or loss of cartilage and we make it even more over time.

Why is it that specialists do not recommend seeing a surgeon at a certain stage?

There are a lot of people who think one way and everyone is entitled to their own opinions. You can't change opinions.

Are people afraid of stem cells?

Some people are afraid because of stem cells causing cancer. But that's embryonic stem cells.

What is the process?

Bone marrow stem cells are the best because there is a higher chance of live stem cells. Less manipulation, meaning that - in a Mayo Clinic study 4 or 5 years ago, which has a two year follow through on people who are ready to get replacements for joint or knee - they had an 80% success rate where they didn't need it. I do replacements and I do stem cells.

How do you determine what's better for the patient?

My knowledge and years of experience. Also, my knowledge with fitness and being athletic myself. Understanding at a certain point, someone is mechanically compromised. Bone on bone is a term that's been used for years. There are a lot of people who think they are 'bone on bone." Coming from Canada, the US is notorious for doing unnecessary surgeries and replacements. It's the highest rate of replacements in the world. I do not like the term 'bone on bone' because a surgeon will look at an x-ray and say you're bone on bone because that's all they do: replacements. They become a 7-11 or 99 Cents store, lining up 21 people a day. That's not the right way to do things. You don't want to be one of those 21 people getting a replacement because you're not getting that surgeon's full attention. The reality is - you have a PA or an old plastic surgeon who's doing most of your surgery and there is more likelihood of issues. Amongst every specialty there is a lot of ignorance. The whole concept is - you preserve what you have for as long as you can. You have beauty on the outside; you need beauty on the inside too. What's beauty on the inside? Feeling good, you're less inflamed and your joints are healthy.

How does it work with a stem cell procedure?

I extract bone marrow from your pelvis. Take approximately 6 ccs. Under slight sedation, it takes about 5 minutes to take it. Then we separate it via an FDA approved technique. Per FDA, we cannot add anything to it, nor would I want to. We cannot harvest it because the longer it's outside of the body, the better it is. Basically, we then inject those pure cells right away into the joint. It's a four month process for an 80% of regeneration. So, it's not just reduction of inflammation, it's regeneration. It will be a year for a 100% effect. I've had probably about 20% of patients who have taken 6 months+. I've had over a 95% success rate with this technology.

Are you one of the only doctors doing this in LA?

I'm one of them. There are some family and pain management doctors who are doing it. I'm the only Orthopedic surgeon doing it. I'm sure different practitioners are starting to.

Dr. Raj and patient Paula Abdul

How often do you do the stem cell procedure?

You do it one time. It's a powerful injection and there are people Ihave 6 years out who are doing well.

Does it hurt after the fact?

No, not at all. You can walk and move. For example, with your hip - I would combine it with physical therapy to increase your range of motion. Once you have the anti-inflammatory effect, you have to take advantage of it. If you don't increase your range of motion - what happens is - you're walking on one nail vs. 100 nails. You want to dissipate the force over a greater area so that there's a higher chance of external success. Then you strengthen the muscles.

Are there people who are not good candidates for it?

Yes, when it's too far gone. Like I said, people are told they're bone on bone when they're not. They show you different views. It's a marketing gimmick. That person is lined up and ready to sell. Age is relative. There's physiologic age. It really depends on the person. Hypothetically, if you're an inflamed mess, a drinker and abusive to your body, then nothing is going to work. If you take care of yourself and you're motivated with the right protoplasm, then it's going to work.

What about the skeptics or the ones who think it's bad for you?

Don't get me wrong; amniotic stem cells are good for certain situations. Embryonic is bad. It means that it's too far gone. You want live stem cells in an area that does not have blood supply. The data is out there. How can you argue against a Mayo Clinic study with an 80% success rate? How can you argue against the hospitals for special surgery in New York that's doing it, or the Steadman Hawkins Clinic, I'm doing it. Top facilities in the world are doing it and a number of top athletes who are getting it done with success rates. Who's ignorant? Is it that one surgeon or everyone else?

Does insurance cover it?

No, not yet. Insurances are very backwards in terms of their understanding. They would rather cover a replacement.

Is it expensive?

If you break it down par and par and avoid a replacement, not really. On average, you're talking about $7,000, versus hospital, surgeon, facility fees+++,which can be about $25,000.

You're very progressive.

There are a lot of things that I do to try and reduce pain significantly.When I use screws, I use screws that are made out of calcium so they dissolve in your body. Some of my colleagues use tourniquet, I don't use one. I control bleeding and do it in less than an hour. The whole concept is, you don't have atourniquetsqueezing your leg and toxins causing significant pain.

And there you have it. Everything is a risk in life, we do not know if we will wake up tomorrow or if you will get hit by a car and so on so why not try this procedure. I believe that I am lucky enough to have met Dr. Raj. I have taken the decision to undergo the stem cells therapy FDA approved or not, anything before going under the knife one more time. Stay tuned, I will give you a report on the progress.

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Dr. Raj & Stem Cell Therapy Innovation - LATF USA

How Young India is fuelling the future of stem cell therapy and signing up to save lives – YourStory

Eighteen-year-old Aisha Choudhary was just like any other adolescent eyes filled with dreams and a heart brimming with energy. The only difference was she was battling a rare genetic disease, Severe Combined Immune Deficiency (SCID). Diagnosed when she was six months old and undergoing medical treatment for years, she was iron-willed in playing the cards she was dealt.

Since one of the most effective cures for SCID is a stem cell transplant (grafting of the parent cells from which all blood cells develop), Aishas parents, Niren and Aditi, decided to opt for that treatment mode. But their cells were not a complete match with their daughters, and they had to look at external donors. However, due to a low number of voluntary, registered stem cell donors, Aisha could not get a compatible donor whose genetic markers were a close enough match to hers. With no other alternative treatment available, Aisha had a bone marrow transplant. But, it came with a side-effect that cost her life Pulmonary Fibrosis, a disease known to damage the lung tissues.

Aishas Choudhary's role has been played by Zaira Wasim in The Sky is Pink.

Aishas journey has been captured in The Sky is Pink, a recent Bollywood movie starring Priyanka Chopra, Farhan Akhtar, Zaira Wasim, and Rohit Saraf.

The 18-year-olds life story is mirrored in the experiences of many who await stem cell donation as treatment for blood-related illnesses likeleukemia, lymphoma, and sickle cell anemia every year. With very few individuals signing up as donors and the probability of finding a match being a dismal 0.0008 percent in India (against a lean 16 percent abroad), fatalities are mounting year on year.

However, in recent times, there has been one small break in the clouds a number of youngsters, non-governmental organisations, and medical professionals have come forward and are working to spread awareness about stem cell donation and motivate a larger number of people to register as donors.

The stem cells in a human body mainly comprise red blood cells, platelets, and white blood cells. These are found in the umbilical cord of newborns and in the peripheral or circulating blood and bone marrow.

A stem cell donation is as simple and painless as a blood donation.

Certain diseases like blood cancer and leukemia tend to destroy the bone marrow or affect its functioning.For these, treatments like chemotherapy and radiotherapy are tried initially. However, in some cases, they do not prove effective for a cure. The only recourse then is replacing the patients stem cells with those of a healthy person.

One of the main criteria for a successful transplant is a good match between the stem cells of the donor and those of the patient. Therefore, a donor registry will administer a cheek swab test (tissue samples extracted from the cheek) on all potential donors to match cell characteristics. This procedure of pairing generic markers is called Human Leukocyte Antigen (HLA) in medical terms.

A cheek swab test in progress.

Each potential donors tissue is entered in the registry and given an identification number after the test is done. If the registry finds a match at any point in time, the donor is contacted to initiate the transplant.

There are many organisations today that are leading the charge in saving the lives of people suffering from serious blood disorders like cancer, thalassemia, and anaemia.

For instance, Datri, an Ahmedabad-based NGO, is working to create a wide and diverse database of potential stem cell donors by organising donation drives. Founded in 2009 by two doctors and an engineer, the organisation focuses on conducting awareness campaigns and helping individuals sign up on its registry as a committed and voluntary benefactor.

The team of the NGO Datri.

The idea for Datri was initially born in the minds of doctors Nezih Cereb and Soo Young Yang, who run a laboratory, Histogenetics, for determining tissue matches between patients and donors. Since pairing tissue types is imperative for any stem cell transplant, and confronting a severe shortage of donors, the doctor duo would run from pillar to post to meet hospitals requirements. Working with a number of the hospitals in India, they realised just how acute the shortfall was in people willing to donate stem cells. They recognised the immediate need to create a donor registry here.

Soon after, Raghu Rajagopal, an engineer from BITS Pilani and Director of ready-to-eat venture Millets and More, connected with them and they decided to start Datri.

Today, the functioning of the registry, its maintenance, and even the substantial costs involved in conducting the HLA matching are taken care of by the lab. In the last 10 years, Datri has gotten over four lakh people to register as donors and has saved around 600 lives through successful transplantation.

Every day, about 40 people are diagnosed with blood disorders in India. Though these can be cured through a stem cell transplant from a genetically matched donor, there is only a 25 percent chance of finding a match from within the family. Others have no option but to rely on unrelated donors. But the chances of getting a match is anywhere between one in 10,000 and one in two million. There is an urgent need to rope in as many potential donors as possible, which is precisely what Datri is trying to do, Raghu explains.

Another organisation that is dedicated to fighting blood disorders with stem cell treatment is DKMS-BMST. It was formed through a joint venture between two renowned NGOs DKMS, which is one of the largest international blood stem cell donor centres globally, and the Bangalore Medical Services Trust (BMST).

The team of DKMS-BMST.

DKMS was founded in Germany in 1991 by businessman Dr Peter Harf, after he lost his wife to leukemia. BMST was born in 1984 from the vision of Dr Latha Jagannathan, a medical director and managing trustee. Since both organisations had a common goal to find a matching donor for every patient with a blood disorder, they decided to come together to achieve it.

A group of youngsters registering to be stem cell donors.

So far, more than 37,000 people in India have registered as potential donors after attending DKMS-BMSTs donor drives.

In highly populous countries like India, thousands of people are in need of stem cell transplants every year to survive. Though donating stem cells is a painless and non-invasive process, it remains a lesser-known medical concept in India, with only 3.6 lakh people willing to play a part in it. Besides, the chances of stem cells of people of the same ethnicity matching are higher than those of individuals from different ethnic backgrounds. But, it is due to sheer lack of awareness that India lags severely in stem cell donations, say experts.

Students taking a cheek swab test at one of the colleges in Bengaluru.

Dr Govind Eriat, a reputed hematologist and bone marrow transplant specialist, says,

With a major hurdle to stem cell donation in India proving to be the myths surrounding the subject, the youth are coming forward to deconstruct common misconceptions.

For instance, 21-year-old Tejaswini Patel, a student of Information Science at New Horizon College of Engineering, Bengaluru, has been busting the false ideas on stem cell donation, starting among her family and friends. She says,

She adds, with a notable sense of pride, In the last two years alone, around 400 students from my college have registered themselves as donors.

(Edited by Athirupa Geetha Manichandar)

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How Young India is fuelling the future of stem cell therapy and signing up to save lives - YourStory

The Week That Wasn’t: Viagra BMTs, Pregnancy Stress, Breast Cancer Vaccine – Medscape

Stories of using the little blue pill for bone marrow transplants, how pregnancy stress is related to the baby's sex, and a vaccine for breast cancer proliferated on the Internet this week. Here's why you didn't read about them on Medscape.

Researchers at the University of California, Santa Cruz, seem to think Viagra has more to offer in medicine. In a recent study of mice, they tested whether the vasodilator couldspeed up the migration of hematopoietic stem cells and progenitor stem cells from the bone to the blood, where the cells could be harvested noninvasively.

The standard protocol for preparing bone marrow donors for the harvesting procedure, a 5-day regimen of granulocyte-colony stimulating factor (G-CSF),is "complex, costly, unsuccessful in a significant proportion of donors," the study authors write, and typically results in fatigue, nausea, and bone pain. Using a two-drug strategy, oral Viagra and a single injection of the CXCR4 antagonist AMD3100 (plerixafor), elicited the same mobilization of stem cells in 2 hours.

We didn't cover the study because it's still too early to say whether this strategy might be effective in people. After this mouse study, the next step is testing the approach in larger animals before human clinical trials.

A study of 187 healthy pregnant women age 18 to 45 years suggests that preterm mental and physical stress may be related to the baby's sex and increase the risk for preterm birth. In the study, 16% of women were physically stressed, as measured by higher blood pressure and calorie intake; and 17% were mentally stressed with high levels of depressionand anxiety; 66% of the women were in the healthy (nonstressed) group.

Women who were stressed during pregnancy were more likely to give birth to a girl. Typically, 105 males are born for every 100 females, but the study authors found that the male-to-female ratio decreased to 2:3 in psychologically stressed patients and 4:9 in physically stressed patients. Physically stressed mothers also gave birth an average of 1.5 weeks earlier than mothers in the healthy group, with 22% giving birth preterm compared with 5% in the healthy group.

The study authors say the findings demonstrate the importance of maternal mental health. Medscape has covered the consequences of maternal stress extensively, including preterm birth, neurobehavioral risks, and potential links to hyperactivity during the offspring's teen years. However, the sample size in this study was small: the mentally and physically stressed groups combined only included about 60 women. That's not sufficient to inform clinical practice in counseling women who want to get pregnant about how stress may affect the sex of their baby, so we didn't cover it.

News spread this week that Floridian Lee Mercker became the first woman to "beat" breast cancer with the help of a new vaccine. The vaccine, which stimulates the immune system to fight off early-stage breast cancer, was developed and administered by researchers at the Mayo Clinic in Jacksonville, Florida. The vaccine is currently in an early trial.

Reports of Mercker's success raise hopes, but she's reportedly the first participant in the trial. The news report also says she underwent a double mastectomy after her diagnosis in March, so it's unclear what evidence of the vaccine's efficacy the researchers measured. Before this experimental vaccine is relevant to Medscape readers, we need to see additional detailed data from more patients in the clinical trial published in a peer-reviewed journal.

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The Week That Wasn't: Viagra BMTs, Pregnancy Stress, Breast Cancer Vaccine - Medscape

Students from over 50 universities across the UK help blood cancer charity – FE News

150,000 POTENTIAL STEM CELL DONORS ARE GIVING BLOOD CANCER PATIENTS HOPE THANKS TO ONE UNIVERSITY SOCIETY -1 in 4 stem cell donors are now recruited by Marrow university societies

Students from over 50 universities across the UK have helped blood cancer charity Anthony Nolan recruit an incredible 150,000 students to the Anthony Nolan stem cell register, since the first Marrow group was created 21 years ago.

Marrow is the name given to blood cancer charity Anthony Nolans network of student volunteer groups.

The first Marrow society was created at the University of Nottingham, with the aim of recruiting students to the Anthony Nolan stem cell register. For many people with blood cancers or blood disorders, receiving stem cells from a stranger is their best chance of survival.

Research has found that younger donors are more likely to save the lives of patients, so the work done by Marrow is invaluable. Over a quarter of all stem cell donations that have occurred in the last two years were from donors recruited by Marrow. University students across the country are continually giving people with blood cancer and blood disorders a second chance of life.

Liam Du Ross, 24, from North Wales is a research chemist and signed up to the Anthony Nolan register in September 2014, while at Bangor University.

Liam said: I was at my university freshers fair and stopped to talk to the volunteers running the Marrow stall. I wanted to help someone in need, and I had already signed up to donate blood at this point, so the Anthony Nolan stem cell register seemed like the next step.

Earlier this year Liam received a call to say that he had been found to be a match for someone in desperate need of a stem cell transplant.

When I found out that I was a match for someone, I felt really lucky. I had absolutely no doubts about going through with the donation at all, the whole experience was a pleasure. The nurses involved in the process were exceptional, and they helped to put me at ease. I donated via PBSC (peripheral blood stem cell collection) so I was able to lie there and catch up on podcasts and TV shows!

I thought about my recipient a lot during my donation and how I would feel if I were in their situation. I would love to meet them one day and I hope they feel the same.

To anyone thinking of signing up to the register, I would say that you should absolutely sign up. If someone you knew was that person who needed a transplant, you'd want to doeverythingin your power to help them.

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Shaswath Ganapathi, 21, is a 4th year medical student at Birmingham University and the secretary of Birmingham Marrow. He decided to volunteer with Marrow after his friend, Rohan, sadly died from leukaemia last year. Shaswath and the other committee members hold events across their university, where they encourage students to sign up to the Anthony Nolan stem cell register, any of whom could go on to donate their stem cells in the future.

Shaswath said: The donors I have spoken to have said that its the most life changing thing they have ever done, and they would never have thought that spending a few minutes signing up at a stand and doing a quick cheek swab could lead to potentially saving someones life.

Aisling Cohn, Youth Programmes Manager at Anthony Nolan, said: Marrow really are the unsung heroes helping Anthony Nolan give hope to patients with blood cancer, by signing up an incredible number of potential donors to the stem cell register. Any one of these people could save the life of someone with blood cancer.

It costs 40 to add each new person to the Anthony Nolan register, any money raised by Marrow will directly help save lives. They really are lifesavers!

If a patient has a condition that affects their bone marrow or blood, then a stem cell transplant may be their best chance of survival. Doctors will give new, healthy stem cells to the patient via their bloodstream, where they begin to grow and create healthy red blood cells, white blood cells and platelets.

Marrow

Key statistics

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Students from over 50 universities across the UK help blood cancer charity - FE News

Bone marrow recipient comes face-to-face with CT donor for the first time – WTNH.com

BRIDGEPORT, Conn. (WTNH) The Gift of Life Marrow Registry organized the meeting Thursday between a bone marrow donor from Connecticut and the recipient whose life was saved by the donation.

Jennie Bunce, 25, of Redding donated her marrow. According to a representative for Gift of Life, Bunce was studying physical therapy and joined Gift of Life through a sorority event at North Carolinas High Point University in 2016.

I never win or get picked for anything, but it just felt like the right thing to do, Bunce told Gift of Life. Im just incredibly happy and grateful to be part of something so special. Its similar to holding the door open for someone or helping a friend in a time of need.

Across the country in Mesa, Arizona, father-of-6, Mark Roser, 33, was battling Acute Lymphoblastic Leukemia. He found out about the diagnosis after he broke a hip in 2018 and had continued weakness. Roser was told he needed a bone marrow transplant to survive.

The hardest part was knowing, no matter how hard I worked, that what I did would not be a deciding factor in my ability to receive this gift, said Roser.

The match was made by Gift of Life in about six months, and the transplant took place in Phoenix.

She is a hero to all the people in my life, said Roser.

She gave me life, she gave my children a future with their dad, she gave my wife a chance to hold her husband, to have someone hold her back. She allowed me to go to work, to play, to see things from a different perspective. I am grateful for every moment I have, and its because of her.

According to Gift a Life, medical privacy laws dictate that recipients and donors must remain anonymous and wait at least a year before meeting.

The two came face-to-face for the first time Thursday in Bridgeport at the Boca Oyster Bar.

Since its start in 1991, the Gift of Life Registry 349,000 individuals who have donated blood stem cells or bone marrow to save a life. The program has facilitated 16,800 matches and over 3,500 transplants.

To learn more about the organization and/or how to donate: https://www.giftoflife.org/.

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Bone marrow recipient comes face-to-face with CT donor for the first time - WTNH.com

Stem Cell Banking Market was valued at $1986 million in 2016 – Markets Gazette

A fresh report titled Stem Cell Banking Market has been presented by KD market insights. It evaluates the key market trends, advantages, and factors that are pushing the overall growth of the market. The report also analyzes the different segments along with major geographies that have more demand for Stem Cell Banking Market. The competition analysis is also a major part of the report.

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

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Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

KEY MARKET BENEFITS

This report offers a detailed quantitative analysis of the current market trends from 2016 to 2023 to identify the prevailing opportunities.

The market estimations provided in this report are based on comprehensive analysis of the key developments in the industry.

In-depth analysis based on geography facilitates in analyzing the regional market to assist in strategic business planning.

The development strategies adopted by key manufacturers are enlisted in the report to understand the competitive scenario of the market.

KEY MARKET SEGMENTS

By Cell Type

Umbilical Cord Stem Cell

Cord Blood

Cord Tissue

Placenta

Adult Stem Cell

Embryonic Stem Cell

By Bank Type

Public

Private

By Service Type

Collection & Transportation

Processing

Analysis

Storage

By Utilization

Used

Unused

By Region

North America

U.S.

Canada

Mexico

Europe

Germany

UK

France

Spain

Italy

Rest of Europe

Asia-Pacific

Japan

China

Singapore

India

South Korea

Rest of Asia-Pacific

LAMEA

Brazil

Saudi Arabia

South Africa

Rest of LAMEA

KEY PLAYERS PROFILED

Cord Blood Registry

ViaCord

Cryo-Cell

China Cord Blood Corporation

Cryo-Save

New York Cord Blood Program

CordVida

Americord

CryoHoldco

Vita34

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Table of Content

CHAPTER 1: INTRODUCTION

1.1. Report description1.2. Key benefits for stakeholders1.3. Key market segments1.4. Research methodology

1.4.1. Secondary research1.4.2. Primary research1.4.3. Analyst tools and models

CHAPTER 2: EXECUTIVE SUMMARY

2.1. CXO perspective

CHAPTER 3: MARKET OVERVIEW

3.1. Market definition and scope3.2. Key findings

3.2.1. Top investment pockets3.2.2. Top winning strategies

3.3. Porters five forces analysis3.4. Top Player Positioning3.5. Market dynamics

3.5.1. Drivers

3.5.1.1. Large number of newborns3.5.1.2. Increase in R&D activities for application of stem cells3.5.1.3. Increase in prevalence of fatal chronic diseases3.5.1.4. Growth in GDP and disposable income

3.5.2. Restraints

3.5.2.1. Legal and ethical issues during collection of stem cells3.5.2.2. High processing and storage cost3.5.2.3. Lack of acceptance and awareness

3.5.3. Opportunities

3.5.3.1. Initiatives to spread awareness3.5.3.2. Untapped market in developing regions

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

4.1. Overview

4.1.1. Market size and forecast

4.2. Umbilical Cord Stem Cells

4.2.1. Key market trends and growth opportunities4.2.2. Market size and forecast4.2.3. Market analysis, by country4.2.4. Cord Blood

4.2.4.1. Market size and forecast

4.2.5. Cord Tissue

4.2.5.1. Market size and forecast

4.2.6. Placenta

4.2.6.1. Market size and forecast

4.3. Adult stem cells

4.3.1. Key market trends and growth opportunities4.3.2. Market size and forecast4.3.3. Market analysis, by country

4.4. Embryonic stem cells

4.4.1. Key market trends and opportunities4.4.2. Market size and forecast4.4.3. Market analysis, by country

Continue

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About KD Market Insights

KD Market Insights offers a comprehensive database of syndicated research studies, customized reports, and consulting services. These reports are created to help in making smart, instant and crucial decisions based on extensive and in-depth quantitative information, supported by extensive analysis and industry insights.

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Stem Cell Banking Market was valued at $1986 million in 2016 - Markets Gazette

The Afterlife of Afterbirth – MedPage Today

Laura Wilson didn't know about placenta donation until she was on the operating table.

"I had a cesarean, so they came in while I was being prepped for surgery," Wilson says in a YouTube video. "It was something I hadn't heard of before."

But when she was told placental tissue could be used to help people suffering from serious wounds, the mother of three boys quickly signed off on the consent forms.

"It was fun to think, ok, this could go to some good use instead of just going with everything else" in the medical waste bag, she says.

Wilson isn't the only mother to suddenly face a decision about placenta donation in the operating room. In an article on the motherhood website Scary Mommy, Caila Smith said she was in the hospital about to have an emergency C-section when a nurse asked her if she wanted to donate her twins' placentas. Another woman described getting a call from her doctor's office after scheduling her C-section and being asked to donate.

While most pregnant women have heard about cord blood donation, far fewer are familiar with placenta donation which can actually refer to the placenta, umbilical cord, and amniotic fluid, or "birth tissue." All are now sought as rich sources of stem cells. The eleventh-hour decision raises questions about just how informed patients' consent actually is, and what their donations are ultimately used for, particularly given the explosion in stem cell clinics offering questionable therapies.

"If women are being approached when they're in labor, about to go into a C-section, it's not the optimal time to be making decisions like that," Leigh Turner, PhD, a bioethicist at the University of Minnesota who has tracked the rise in stem cell clinics, told MedPage Today.

"Maybe they think it's for altruistic purposes," he added. "They may not realize there's a large for-profit marketplace out there."

It's difficult to track what donations are actually used for, although placental material does have seemingly legitimate applications in wound care and research. Still, those markets can be lucrative information that isn't usually offered to a mother, who is not compensated for the organ, when she is making her decision.

As more hospitals launch placenta donation programs, more mothers may be faced with the choice at an emotional, vulnerable time in their lives.

"I wanted to do this in case one day my boys would need this," Wilson says in the video, which was posted to YouTube by a company called Birth Tissue Recovery to promote its placenta donation program.

Rise in Placenta Donations

St. David's Medical Center in Austin, Texas, launched its placenta donation program about a year and a half ago in partnership with GenCure, a company that describes itself as non-profit and says it "applies advances in regenerative medicine to help heal patients suffering from trauma, disease or the aging process" on its website.

GenCure's placenta donation program involves collecting the placenta and the umbilical cord, according to spokesperson Ashley Frolick. The company also collects cord blood through the Texas Cord Blood Bank, which it owns.

In most cases, mothers are told about the option to donate when they arrive at the hospital, Frolick said, although they are only eligible if they are having a C-section, which is often an emergency situation.

Michelle Kocks, RN, coordinator of the placenta donation program at St. David's, said patients are persuaded by the fact that they're helping others, and because the placenta will otherwise be discarded as medical waste -- a potential incentive for hospitals to run programs, as they would otherwise have to pay for medical waste disposal. (Hospitals are uniformly mum about why, exactly, they agree to facilitate donations that go to outside entities, or whether money changes hands.)

"Once [women] learn about the benefits ... they love being able to be a part of the program," Kocks told MedPage Today, adding that about 20% of eligible women who undergo C-section deliveries become donors. Frolick said nurses and doctors are not paid for referrals, and neither are the women who are giving birth.

Other hospitals are launching placenta donation programs. Rochester General Hospital added theirs in July, and Mount Carmel St. Ann's Hospital in Westerville, Ohio, had its first donation in April. Both are working with MTF Biologics, which also claims non-profit status and says it focuses on bone and skin grafts.

St. Joseph Mercy in Ann Arbor, Michigan, launched its program this summer in partnership with Gift of Life Michigan, the first hospital in the state to do so.

Other hospitals with new programs include Sisters of Charity Hospital in Buffalo, New York, which works with ConnectLife (formerly UNYTS); and Maple Grove Hospital in Minnesota, which works with for-profit MiMedx.

MiMedx has been plagued by scandals including charges involving its marketing and accounting practices, and allegations that it shipped more product than had been ordered and booked that as sales. This summer, the Department of Veterans Affairs advised its facilities not to use injectable amniotic tissue products from the company for not meeting purity and sterility standards. The VA also warned against using such products for managing plantar fasciitis, tendonitis, or osteoarthritis "since evidence is currently limited or lacking" for their efficacy.

MiMedx runs placentadonation.com through its subsidiary MiMedx Tissue Services.

Loralei Thornburg, MD, an ob/gyn at the University of Rochester Medical Center, said her institution discussed a placenta donation program with ConnectLife, but didn't create one because they treat too many high-risk cases.

"Most of the hospitals where you see it being done tend to [have] lower-risk, more normal populations," Thornburg said. "One of our lower-risk hospitals started looking into it ... because that's who's eligible. I do primarily high risk. They don't want a 28-week placenta with preeclampsia."

GenCure partners with six hospitals in Texas on placenta donation. Frolick said the birth tissue that it collects is provided to "partners who will then create therapeutic products that may be used to help treat burns, diabetic ulcers, traumatic injuries, and can go on to develop other FDA-approved therapies."

That could include stem cell therapies, Frolick said. These treatments have long taken advantage of a grey area in FDA regulation, which stipulates that human cell and tissue products are exempt from the extensive and expensive drug or biologic approval process as long as they meet certain criteria.

'It's Going to Be Marked Up and Sold Again'

Summa Health, a health system in northern Ohio, offers "placenta tissue injections" for "damaged tissue found in tendons, ligaments, inflamed tissue and degenerated joints."

A PubMed search reveals no studies of the therapy for any indication.

"I'm not aware of anything in this whole sector being shown to be effective or safe with good, solid data," stem cell researcher Paul Knoepfler, PhD, of the University of California Davis, told MedPage Today.

The rise in placenta and birth tissue donation parallels an increase in unregulated stem cell clinics around the country, with well over 1,000 today, up from 570 when Turner first started tracking them in 2016. The FDA has long waged war with these clinics, which purport to treat everything from pain to Parkinson's disease with stem cells. A federal judge handed the agency a victory in June when it ruled that FDA could block Florida-based U.S. Stem Cell from injecting patients with a stem-cell "extract" made from their own fat cells.

Given the ruling, Turner said clinics may shift from using fat or bone marrow to procure stem cells, to placental or birth tissue products -- also referred to as amniotic stem cell treatments -- especially since they can just be bought and injected. Also, they don't require collecting or processing material from patients.

But the amniotic stem cell supply chain isn't easy to trace.

"It's the part of the marketplace that's most obscured, and that we know the least about," Turner said.

Summa Health did not return a request for comment as to where it gets the material for its placenta tissue injections.

An investigation by ProPublica found that one company, R3 Stem Cell, sourced its amniotic stem cell products from Utah Cord Bank, which began as a private bank where parents stored cord blood and other birth tissue.

Apart from stem cell therapies, other uses of placental material such as wound care can also be lucrative, especially since these products also fall under FDA's "minimally manipulated" and "homologous use" requirements for human cell and tissue products, side-stepping the agency's costly and time-consuming approval process.

John Lantis, MD, a wound care expert at Mount Sinai Hospital in New York, said he's skeptical of claims that amniotic and placental skin grafts provide superior healing compared with regular skin grafts.

"The number of products out there delivering real stem cells is highly questionable," Lantis told MedPage Today. "No one knows if active placental tissues are better. No one has studied it in a meaningful fashion, so a regular skin graft may work just as well."

One of the most widely used products for lower extremity wounds, Lantis said, is EpiFix, which is made by MiMedx. It is synthesized from "dehydrated human amnion/chorion membrane" -- placental material -- according to the company's website. In 2013, the FDA warned the company about marketing the product without approval, which the firm eventually obtained.

If women knew more about the money, power, and scandal behind their donations, they might see donation differently, Turner said.

"What if women were given the full account, that some biobank is going to acquire it, process it, assign it value, and then it's going to be marked up and sold again," Turner said. "Some women might think twice."

2019-10-15T12:00:00-0400

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The Afterlife of Afterbirth - MedPage Today

Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia – Cancer Therapy Advisor

A case study published in Leukemia and Lymphoma described a patient with a diagnosis of Waldenstrm Macroglobulinemia (WM) that had subsequently undergone histological transformation to refractory high grade B-cell lymphoma and was successfully treated with CD19-targeted chimeric antigen receptor (CAR)-T cell therapy.1

WMis a type of B-cell non-Hodgkin lymphoma(NHL), typically characterized by overproduction of monoclonal immunoglobulinM, as well as infiltration of malignant lymphoplasmacytic cells into the bonemarrow.

Although considered incurable, WM often follows an indolent course andsome patients can be asymptomatic for long periods. Rarely, the diseasetransforms into a more aggressive form of NHL that has been associated with apoor prognosis.

The patient described in the case studywas a 71-year-old man who was first diagnosed with WM in 1998. The patient wasmonitored without undergoing active treatment for a period of 12 years, atwhich time he developed anemia and splenomegaly. At that time, he underwenttreatment with 6 cycles of fludarabine and rituximab and achieved a partial response totreatment. Following a worsening of symptoms 4 years later, the patient wastreated with 6 cycles of bendamustine and rituximab.

Biopsyof an enlarged cervical lymph node performed at that time revealed high-gradeB-cell lymphoma that was clonally related to the previously seenlymphoplasmacytic infiltrate, consistent with transformation.

Thepatient subsequently achieved a complete response to 6 cycles of rituximab,cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) plus ibrutinibfollowed by 6 months of ibrutinib maintenance therapy that lasted for 18months.

Salvagetherapy included 2 cycles of rituximab, dexamethasone, cytarabine, cisplatin(R-DHAP) followed by 1 cycle of rituximab plus high-dose cytarabine, followedby autologous stem cell transplantation several months later.

Asthe patients disease was considered to be chemorefractory based on subsequent imagingand pathological analyses, he was treated with axicabtagene ciloleucel, aCD19-targeted CAR-T cell therapy currently approved for the treatment of adultpatients with relapsed or refractory large B-cell lymphoma, including high gradeB-cell lymphoma, after 2 or more lines of systemic therapy.2

Althoughthe patient experienced pancytopenia, grade 1 cytokine release syndrome, andgrade 1 neurotoxicity following CAR-T therapy, he achieved a complete response1 month following treatment.

Notably,there was no evidence of either underlying WM or transformed disease at 6 and 12months follow-up.

Thestudy authors noted that longer term follow up in this patient will beinformative, as late relapses have occurred even in patients who achieve a deepresponse after transplant. CAR-T cell therapy may be an effective treatment forrelapsed or refractory WM that has not yet undergone histologicaltransformation, as CD19 is almost universally expressed on lymphoplasmacyticlymphoma cells. The researchers concluded that further analysis of this iswarranted in the context of clinical trials.

References

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Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia - Cancer Therapy Advisor

WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow…

BALTIMOREand PHILADELPHIA and IRVINE, Calif., Oct. 15, 2019 (GLOBE NEWSWIRE) -- WindMIL Therapeutics and the University of California, Irvine (UCI) today announced that the first patients have been identified in an investigator-sponsored study for the collection of bone marrow from patients with gliomas. The study will evaluate generating marrow infiltrating lymphocytes (MILs) for these patients through WindMILs proprietary cellular activation and expansion process. The study is being conducted at UCI.

Patients suffering with glioblastoma are in great need of new, promising treatments that might advance the current standard of care, said Daniela A. Bota, MD, PhD, director of theUCI Health Comprehensive Brain Tumor Program,seniorassociate dean for clinical research, UCI School of Medicine and clinical director, UCI Sue & Bill Gross Stem Cell ResearchCenter. The University of California, Irvine is excited toplay a key role in research that may lead to a clinical trial that enlists the immune system in novel ways to fight this terrible disease.

Gliomas are the most common of the malignant brain tumors. Glioblastoma, the most common glioma, has a five-year survival of less than 5 percent. Additional treatment options are urgently needed for these patients. Adoptive immunotherapy is a possible approach for gliomas and the use of MILs, a cell therapy that is naturally tumor-specific, is one such treatment option.

The bone marrow is a unique niche in the immune system to which antigen-experienced memory T cells traffic and are then maintained. WindMIL has developed a proprietary process to select, activate and expand these memory T cells into MILs. Because memory T cells in bone marrow occur as a result of the immune systems recognition of tumor antigens, MILs are specifically suited for adoptive cellular immunotherapy and are able to directly eradicate or facilitate eradication of each patients unique cancer. WindMIL is currently studying MILs in multiple myeloma, non-small cell lung cancer and squamous cell carcinoma of the head and neck, and plans to expand into other solid tumors.

WindMIL is looking forward to working with the University of California, Irvine on this exciting project and is optimistic that MILs may offer the potential to help patients with these hard-to-treat diseases, said Monil Shah, PharmD, MBA, Chief Development Officer at WindMIL.

About WindMIL Therapeutics

WindMIL Therapeutics is a clinical-stage company developing a novel class of autologous cell therapies based on marrow infiltrating lymphocytes (MILs) for cancer immunotherapy. As the leader in cellular therapeutics emanating from bone marrow, WindMIL translates novel insights in bone marrow immunology into potentially life-saving cancer immunotherapeutics for patients. WindMIL believes that Cell Source Matters and the companys proprietary process to extract, activate and expand these cells offers unique immunotherapeutic advantages, including inherent poly-antigen specificity, high cytotoxic potential and long persistence. For more information, please visit: http://www.windmiltx.com.

About UCI Health

UCI Healthcomprises the clinical enterprise of the University of California, Irvine. Patients can access UCI Health at primary and specialty care offices across Orange County and at its main campus, UCI Medical Center in Orange, California. The 417-bed acute care hospital provides tertiary and quaternary care, ambulatory and specialty medical clinics and behavioral health and rehabilitation services. UCI Medical Center features Orange Countys only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us onFacebookandTwitter.

About the University of California, Irvine

Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visitwww.uci.edu.

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WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow...

Light the Night Oct. 19 – Westlock News

Organizers of this years Light the Night, which is held in support of blood cancer research, hope members of the community can join them Oct. 19 as they take to the Rotary Trail to raise awareness and funds for the eradication of various blood cancers.

Leukemia, lymphoma, Hodgkins lymphoma and myeloma can all be beat, but for every inspirational tale of perseverance and survival, there are even more about those who could not hang on long enough and died as a result of the fast moving diseases that affect blood cells, bone marrow and lymph nodes to name a few.

I lost my niece to leukemia 11 years ago, she was 19, said Westlock organizer Joanne Rimmer, with tears in her eyes as fresh as the day she lost her niece.

I had positive thoughts the whole time, I thought she was going to get over it and everything was going to be back to normal and it didnt work out. I didnt want other families to go through that, so I thought it was a good thing to help raise money.

It has been 11 years and Im still crying. Its one way we can do something to say we really miss her.

Rimmer also has a close friend who was able to survive leukemia after a donation of stem cells from her brother saved her life. She also has another friend in Manitoba who is currently fighting off leukemia, with some success. These are her reasons for lighting up the night, so called because participants often hold lanterns of different colours that denote how thatindividual has been affected.

White lanterns are carried by survivors, gold is in remembrance and red is in support, which together makes for quite a sea of colour moving through the town.

Rimmer, who has been taking part in the event in one form or another for 11 years and helping to organize the Westlock event for the last six years is a proponent of having a bare-bones event with little flash or overhead to make sure as much money as possible is donated to the Leukemia and Lymphoma Society of Canada.

Any sponsors or potential contributors are asked to donate directly to the cause instead of providing other supports, which are appreciated, but ultimately unneeded.

I want every dollar that gets raised to actually go towards what were actually raising money for. I dont want to waste it on silly things.

Rimmer is urging those who want to participate to start collecting sponsors, and to register at http://www.lightthenight.ca. Participants should meet at the Rotary Spirit Centre after 6:30 p.m. Oct. 19 for the walk that will begin at 7 p.m., winding it way east on the Rotary Trail to the healthcare centre, then eventually on to the pool. All are welcome to join, from babies in strollers to their great-great parents, said Rimmer, who also noted the event will take place rain or shine.

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Light the Night Oct. 19 - Westlock News

Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests – Gaucher Disease News

Gaucher disease can predispose patients to rare types of blood cancer, and doctors should be vigilant for the development of these malignancies, a case report suggests.

The study, A case of bony lytic lesions in a patient with Gaucher disease, was published in the journal Clinical Case Reports.

The most common symptoms of Gaucher disease are reduced platelet count, enlarged liver and spleen, and lesions in the bones. This disease also has been associated with an increased risk ofblood disorders.

Researchers in Canada reported the case of a 57-year-old man who developed a rare type of blood cancer cell probably related to Gaucher disease, as he had Gaucher cells cells that accumulate abnormal amounts of a fat molecule (glucocerebroside) characteristic of the condition in his bone marrow.

The man had a scalp lesion that did not heal and progressively increased in size. Doctors performed a biopsy and discovered it was caused by a plasmacytoma, a rare form of blood cancer in which myeloma cells form a tumor in the bones or soft tissues.

At the time of biopsy, the patient had mild anemia, low levels of platelets, and a family of blood proteins called gamma globulins characteristic of myeloma. His kidney function and calcium levels were normal.

Further tests showed that the man had several bony lytic lesions spots of bone damage caused by cancerous myeloma cells and abone marrow biopsy showed infiltration of both plasma cells and Gaucher cells. That led to a diagnosis of plasma cell myeloma probably associated with Gaucher disease.

Gaucher cells infiltrating the bone marrow may mask the extent of abnormal plasma cell infiltrates, and immunohistochemical staining [a method that identifies abnormal cells in biopsies] can be invaluable in identifying the true burden of plasma cells for appropriate classification of suspected plasma cell neoplasia, the researchers said.

The investigators also noted that the man had a history of abnormal spleen size and reduced blood cell count. A bone marrow biopsy performed years earlier showed the presence of possible Gaucher cells.

Gaucher disease should be considered in the differential diagnosis of unexplained hepatomegaly [abnormal liver size], splenomegaly [abnormal spleen size], or cytopenias [reduced blood cell count], the investigators said. They added that further research of the previous symptoms might have allowed diagnosing Gaucher disease before the cancer appeared.

The patient received a combination of chemotherapy, cyclophosphamide, and Velcade (bortezomib), followed by high doses of melphalan and autologous stem cell transplantation. He tolerated the transplant well and was discharged with the recommendation of long-term follow-up.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 20

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Gaucher Might Be LInked to Rare Types of Blood Cancer, Report Suggests - Gaucher Disease News

Stem cell therapy helped Owen Franks but there’s still plenty to prove – Stuff.co.nz

Stem cell therapy, which All Blacks prop Owen Franks used to help fix a damaged shoulder, is raising hopes of a whole range of medical breakthroughs.

But there's a way to go before the medical establishment is convinced.

In late 2017, US Food and Drug Administration (FDA) Commissioner ScottGottliebhad this to say:"We're at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease."

REGEN CELLULAR

Dr Hassan Mubark takes blood from All Blacks prop Owen Franks.

Yet, as an indication of how far there is still to go, the FDA has also warnedpeople in the USagainst "unscrupulous providers" offering stem cell products that were unapproved and unproven.

READ MORE:*Rugby World Cup 2019: All Black Owen Franks thrown a stem cell lifeline*Owen Franks hits back at critics following omission from Rugby World Cup squad*Stem cell therapy for All Black Israel Dagg as he hits comeback trail with Crusaders*Experimental stem cell treatment shows results for Waikato woman with MSA Cerebella*Stem cell clinics accused of taking advantage of patients*Reported stem cell treatment could give hope to Michael Schumacher

"Researchers hope stem cells will one day be effective in the treatment of many medical conditions and diseases," it said, thenadded: "Stem cells have been called everything from cure-alls to miracle treatments. But don't believe the hype."

Looking at just the area of deteriorating joints, it's easy to see how stem cell therapies, if they deliver on the promise,could make life much better for many people with osteoarthritis who are in pain and have restricted movement.

Last week, Otago University researchers predictedthe number of knee replacement surgeries needed for osteoarthritis would increase from around 5000 a year in 2013 to abut9000 in 2038.

AP

Former Formula One champion Michael Schumacher received devastating head injuries in a ski accident six years ago. Last month it was reported he has undergone stem cell treatment in Paris.

Osteoarthritis is the area where ReGen Cellular,the clinic where Franks had the therapy, has done most of its work in the past two to three years, although ithas recently expanded its services to include a range of diagnosed auto-immune conditions, among them rheumatoid arthritis, multiple sclerosis, and type 1 diabetes.

ReGensaid 55 per cent of its patients were aged over 60, 35 per cent were 40-60 and 10 per cent were sports-based.

Theclinic usesPure Expanded Stem Cell (PESC) therapy, which involves taking 40 grams - about a teaspoon - of fat from around a patient's stomach. Mesenchymal stem cells (MSCs)in that sample are then multiplied in the clinic's Queenstown laboratory for about eight weeks. At the end of that process 100 million to 200 million cells have been produced.

Otago University

Otago University, Christchurch regenerative medicine research team have invented a bio-ink - a gel-like substance mixed with human stem cells - to be used with a bio-printer to make human body parts. Video shows the printer using bio-ink to make a body part.

For the treatment of osteoarthritis, between 50m and 100m stem cells are injected into larger joints, with 25m to 50m into smaller joints. ReGen said the therapy provided immediate pain reduction and increased mobility. MRI scans showed cartilage could and did regenerate.

ReGendescribedMSCs as the cells that "wake up damaged or lazy cells". Slightly more technically, Nature.com said MSCs wereadult stem cells present in multiple tissues, including the umbilical cord, bone marrow and fat.MSCscan self-renew by dividing and can differentiate into multiple tissues including bone, cartilage, muscle and fat cells, and connective tissue.

ReGen director of patient care Marcelle Noble said the clinic believed its treatments, if offered early enough, would save the public health system hundreds of millions of dollars through lessened replacement surgeries, and would save ACC millions of dollars in lengthy rehabilitation programmes.

The treatment for two knees was half the price of one knee replacement surgery within the public health system, she said. ReGen advertises osteoarthritis treatment for a single joint at $12,500 and for two joints at $15,000.

GETTY IMAGES

Former All Black Israel Dagg had stem cell therapy for an injured knee, but in the end had to give the game away because of the injury.

So far mainstream funding hadnot been offered for the therapy, Noble said. But the clinic had a "big breakthrough" earlier this year when two insurers in New Zealand accepted patients'PESC therapy claims. In July, ACC accepted consultation by ReGen's chief medical officer Dr Hassan Mubark.

ReGen only had data for the past five years on the success of its therapy, but the fact patients were returning to have other areas of their body treated was an indication of how people feltthe therapy was improving their quality of life, Noble said.

Globally, "massive" R&D spending was going into stem cell research. More therapies would become available and stem cell treatment would become "commonplace".

At any one time ReGen had 50-75 patients' cells growing in its incubators, Noble said. Of the patients treated, 40 per cent hadailments in therknees, 30 per cent in their hips, 20 per cent in their shoulders. The final 10 per cent were for sports and other issues, including problems with tendons, muscles, cartilage tears, fingers, elbows, ankles and hands.

SUPPLIED

Dr Ron Lopert undergoing part of the PESC treatment.

The first patient to undertake ReGen's PESC therapy was retired GP Dr Ron Lopert, who lives in Tauranga.

For five to 10 years, he had beengetting aches and pains in his hips after playing sport, and the problem was becoming more noticeable, he said. In 2013 he had an x-ray that showed he had moderate to severe osteoarthritis in both hips,more severein his right hip.

He stopped playing all sports and started researching different forms of treatment. Ideally, he wanted to be able to get some of his own cartilage back and reverse the osteoarthritis. It seemedPESCshould do that.

In 2015, aged 61, he had the therapy, with stem cells being injected into each hip joint.Within weeks henoticed an improvement in the range of motion and a decrease in pain, Lopert said.Some of that was just the anti-inflammatory component of stem cell injection, but he thought he also received a longer term benefit from cartilage regeneration.

SUPPLIED

Dr Lopert on his recent travels. He says he has much less hip pain.

He put the success of the procedure at75 per centin terms of symptoms and function, and100 per cent when it came to avoiding invasive surgery."I opted for a much more natural treatment where my own tissue is regenerating, instead of a metal prosthesis," Lopert said.

He was not sure all the improvement came from the stem cell treatment. As well as avoiding overuse of the joints, which meant he hadn't returned to playing sport, he had also switched to an anti-inflammatory diet.

His left hip continued to have hardly any symptomsbut he had started noticing the "odd twinge now and then" in his right hip.

"The vast majority of days it's fine provided I'm just walking and doing ordinary things. On the odd occasion I might carry something heavy, then I would notice it the next day and it (right hip) would stay painfulintermittentlyfor the next couple of days," Lopert said.

Sean Gallup

In this picture from February, German Chancellor Angela Merkel looks through a microscope at brain organoids grown from stem cells.

Some of his stem cells had been retained after the treatment, and he was booked in for a follow-up injection for his right hip at the end of October.

He expected the therapy would become a "go to" treatment, and would become an early intervention for osteoarthritis. But more independent research was needed to confirm the success of the treatment. "The evidence is slowly building up but there needs to be more before the Government will accept it," Lopert said.

In his case, he thought there had been cartilage regeneration in his hips, but that was based on his symptoms. "It would have been nice had I had MRI scans before and after the injection for objective evidence," he said.

From the perspective of the medical establishment, the New Zealand Orthopaedic Association said it supported a position statement on stem cell therapy produced by the Royal Australian College of Surgeons.

That paper, approved in mid-2018,noted stem cell therapy was a "rapidly advancing" area, but many proposed stem cell therapies were experimental and not yet proven. It did not support surgeons administering stem cell therapy outside of an ethically approved registered clinical trial.

"Whilst there may be scope for innovative treatment in the future, currently, the clinical effectiveness and safety of stem cell therapies remain scientifically unproven," RACS said.

In this country, an ACC spokesperson said ACC did not have an official position on stem cell therapy for the treatment of injuries. An internationally standardised evidence-based healthcare approach was used to help ACC decide how it covered injuries and funded treatments.

Dr HassanMubark, ReGen's chief medical officer, was a healthcare provider contracted to ACC in the specialty of rheumatology, and ACC had funded consultation fees with Mubark, the spokesperson said. Those consultations were for diagnostic and treatment planning purposes and did not need prior approval from ACC.

ACC had to consider legislative criteria when deciding whether to fund any particular treatment. There would be many reasons why ACC might decide to fund a client to see a rheumatologist for an opinion on the diagnosis and possible management of their condition. That would not commit ACC to funding any proposed treatment but would provide the client and ACC with information to help decision-making.

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Stem cell therapy helped Owen Franks but there's still plenty to prove - Stuff.co.nz

Global Cell Therapy Technologies, Companies & Markets During the Forecast Period, 2018-2028 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. The role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. The current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

The cell-based markets was analyzed for 2018 and projected to 2028. The markets are analyzed according to therapeutic categories, technologies, and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer, and cardiovascular disorders. Skin and soft tissue repair, as well as diabetes mellitus, will be other major markets.

The report contains information on the following:

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer.

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit https://www.researchandmarkets.com/r/9q5tz1

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Global Cell Therapy Technologies, Companies & Markets During the Forecast Period, 2018-2028 - ResearchAndMarkets.com - Business Wire

Drexel on the Road: Stem cell study for osteoarthritis – WKRG News 5

PENSACOLA, Fla. (WKRG) Osteoarthritis affects millions of people in the US. Symptoms range from minor pain to crippling pain that compromises quality of life. A groundbreaking study is underway at four prestigious research facilities in the United States. One of those is right here on the Gulf Coast. Tonight, Drexel Gilbert is on the road in Gulf Breeze.

Lori Jamison is a Pensacola native who, as a teenager, played basketball at Pine Forest High School. Today, she suffers from osteoarthritis in her knee. She believes its a result of basketball injuries.

I get stiffness, it interferes with my mobility. Sometimes its like a sharp needle going down your leg. When I go to the movie theater, I have to sit on the back row so I can stretch it out, Jamison said. She is participating in a clinical trial at Andrews Research and Education Foundation in Gulf Breeze.

The research is studying stem cell treatment for osteoarthritis in the knee. AREF is one of only four facilities in the country participating in the study. The others are Emory Orthopedics & Spine Center, Duke University and Sanford Health. Researchers hope it leads to FDA approval for the treatment. If that happens, it could be life-changing for patients.

Hopefully reduce their pain if not actually get rid of their pain. That is our goal. We want to delay, if not prevent, total knee replacement, said Dr. Josh Hackel, who is the primary investigator for the Andrews phase of the study. Were comparing three different stem cell sources. Bone marrow from their pelvis, adipose- thats tissue from their belly fat- and the third is umbilical cord tissue donated from pregnant mothers.

The bone marrow and belly fat stem cells are harvested from the study participants, under local anesthesia. The stem cells are later implanted into the knee joint using ultrasound guidance to implant the cells into the knee joint.

Jamison has already undergone stem cell harvesting.

It was very easy, very convenient, no downtime after the procedure was done, Jamison said

This $13 million clinical trial is being funded entirely by a grant from Bernie Marcus, founder of the Marcus Foundation and co-founder of Home Depot. Osteoarthritis is an issue that is close to the philanthropists heart because his mother was left disabled by the illness at a young age.

There will be around 120 participants at each of the four sites. There are plenty of openings. If youd like to be considered for the study, call AREF at 850-916-8591.

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BEYOND LOCAL: Expert recommends ‘path of cautious optimism’ about the future of stem cell treatment – ElliotLakeToday.com

This article, written byKatharine Sedivy-Haley, University of British Columbia, originally appeared on The Conversation and is republished here with permission:

When I was applying to graduate school in 2012, it felt like stem cells were about to revolutionize medicine.

Stem cells have the ability to renew themselves, and mature into specialized cells like heart or brain cells. This allows them to multiply and repair damage.

If stem cell genes are edited to fix defects causing diseases like anemia or immune deficiency, healthy cells can theoretically be reintroduced into a patient, thereby eliminating or preventing a disease. If these stem cells are taken or made from the patient themselves, they are a perfect genetic match for that individual, which means their body will not reject the tissue transplant.

Because of this potential, I was excited that my PhD project at the University of British Columbia gave me the opportunity to work with stem cells.

However, stem cell hype has led some to pay thousands of dollars on advertised stem cell treatments that promise to cure ailments from arthritis to Parkinsons disease. These treatments often dont help and may harm patients.

Despite the potential for stem cells to improve medicine, there are many challenges as they move from lab to clinic. In general, stem cell treatment requires we have a good understanding of stem cell types and how they mature. We also need stem cell culturing methods that will reliably produce large quantities of pure cells. And we need to figure out the correct cell dose and deliver it to the right part of the body.

Embryonic, 'induced and pluripotent

Stem cells come in multiple types. Embryonic stem cells come from embryos which makes them controversial to obtain.

A newly discovered stem cell type is the induced pluripotent stem cell. These cells are created by collecting adult cells, such as skin cells, and reprogramming them by inserting control genes which activate or induce a state similar to embryonic stem cells. This embryo-like state of having the versatile potential to turn into any adult cell type, is called being pluripotent.

However, induced pluripotent and embryonic stem cells can form tumours. Induced pluripotent stem cells carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming.

Genetic damage could be avoided by using younger tissues such as umbilical cord blood, avoiding tissues that might contain pre-existing mutations (like sun-damaged skin cells), and using better methods for reprogramming.

Stem cells used to test drugs

For now, safety concerns mean pluripotent cells have barely made it to the clinic, but they have been used to test drugs.

For drug research, it is valuable yet often difficult to get research samples with specific disease-causing mutations; for example, brain cells from people with amyotrophic lateral sclerosis (ALS).

Researchers can, however, take a skin cell sample from a patient, create an induced pluripotent stem-cell line with their mutation and then make neurons out of those stem cells. This provides a renewable source of cells affected by the disease.

This approach could also be used for personalized medicine, testing how a particular patient will respond to different drugs for conditions like heart disease.

Vision loss from fat stem cells

Stem cells can also be found in adults. While embryonic stem cells can turn into any cell in the body, aside from rare newly discovered exceptions, adult stem cells mostly turn into a subset of mature adult cells.

For example, hematopoietic stem cells in blood and bone marrow can turn into any blood cell and are widely used in treating certain cancers and blood disorders.

A major challenge with adult stem cells is getting the right kind of stem cell in useful quantities. This is particularly difficult with eye and nerve cells. Most research is done with accessible stem cell types, like stem cells from fat.

Fat stem cells are also used in stem cell clinics without proper oversight or safety testing. Three patients experienced severe vision loss after having these cells injected into their eyes. There is little evidence that fat stem cells can turn into retinal cells.

Clinical complications

Currently, stem cell based treatments are still mostly experimental, and while some results are encouraging, several clinical trials have failed.

In the brain, despite progress in developing treatment for genetic disorders and spinal cord injury, treatments for stroke have been unsuccessful. Results might depend on method of stem cell delivery, timing of treatment and age and health of the patient. Frustratingly, older and sicker tissues may be more resistant to treatment.

For eye conditions, a treatment using adult stem cells to treat corneal injuries has recently been approved. A treatment for macular degeneration using cells derived from induced pluripotent stem cells is in progress, though it had to be redesigned due to concerns about cancer-causing mutations.

A path of cautious optimism

While scientists have good reason to be interested in stem cells, miracle cures are not right around the corner. There are many questions about how to implement treatments to provide benefit safely.

In some cases, advertised stem cell treatments may not actually use stem cells. Recent research suggests mesenchymal stem cells, which are commonly isolated from fat, are really a mixture of cells. These cells have regenerative properties, but may or may not include actual stem cells. Calling something a stem cell treatment is great marketing, but without regulation patients dont know what theyre getting.

Members of the public (and grad students) are advised to moderate their excitement in favour of cautious optimism.

Katharine Sedivy-Haley, PhD Candidate in Microbiology and Immunology, University of British Columbia

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

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BEYOND LOCAL: Expert recommends 'path of cautious optimism' about the future of stem cell treatment - ElliotLakeToday.com

Fred Hutch scientist on how gold nanoparticles could bring CRISPR to the developing world – GeekWire

Jennifer Adair, a senior scientist at Fred Hutch, speaks at the 2019 GeekWire Summit. (GeekWire Photo / Kevin Lisota)

Genetically editing cells using CRISPR could be the answer to curing genetic disorders such as sickle cell anemia. But in order for the technology to be available for people in countries like Nigeria where around a quarter of the population carries the sickle cell trait the technology will need to become substantially cheaper and less invasive.

Thats where gold nanoparticles come in.

Scientists at the Fred Hutchinson Cancer Research Center are devising an approach that vastly simplifies how CRISPR is applied. Their goal is to create a safe process for gene editing that takes place entirely within the body of a patient.

In order to edit human stem cells using CRISPR today, scientists have to follow a process that involves removing the cells from a patients bone marrow, electrocuting those cells, and modifying them with engineered virus particles.

The process gets even more invasive from there. We actually have to treat these patients with chemotherapy, radiation or other agents in order for these cells that were genetically manipulated to be taken up, Jennifer Adair, a senior scientist at Fred Hutch, said during a talk at the 2019 GeekWire Summit.

The researchers think theyve figured out the first step, which is delivering CRISPR to blood stem cells inside the body. Theyre doing that using gold nanoparticles that are about a billionth the size of a grain of table salt and able to smuggle in RNA, DNA and a protein.

Weve been able to show that not only can we make these, but they passively deliver all of those components to blood stem cells, then we do get genetic editing. And weve been able to go on to show that we can correct the sickle cell defect using this approach, said Adair.

The nanoparticles are big enough to carry the CRISPR payload but small enough to infiltrate cell membranes. Gold is a useful medium since it isnt harmful to humans.

The Fred Hutch team published their work with gold nanoparticles earlier this year in the journal Nature Materials. The system safely edited 10 to 20 percent of the target cells, which the researchers hope will increase as the method is refined.

In an ideal world, clinicians would be able to deliver gene therapy through a syringe, a process that might be accomplished in a single office visit. Adair previously published research on agene therapy in a box concept, a table-top device that could provide gene therapy treatments without the need for expensive medical infrastructure.

We need to develop technologies that make gene editing simpler, more affordable and more accessible to patients around the world, Adair said.

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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2026 – Weekly Spy

Stem Cell Therapy Market is expected to reach 202.77 billion by 2026 from 12.25 billion in 2017 at CAGR of 42.02%.(Detailed analysis of the market CAGR is provided in the report) stands for use of stem cells to treat or prevent disease or condition.

Bone marrow transplant and some therapies derived from umbilical cord blood are mainly used in stem cell therapy. Advancement, in order to establish new sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes, heart disease, and other conditions, are increased in recent years. Stem Cell Therapy Market Researchers are making efforts to discover novel methods to create human stem cells. This will increase the demand as well as supply for stem cell production and potential investigation in disease management. Increasing investment & research grants for developing safe and effective stem cell therapy products, the growing patient base for target diseases, concentrated product pipelines, increasing approval of the new clinical trials, rapid technological advancement in genomics, and the rising awareness about the stem cell are expected to drive the growth of the Stem Cell Therapy solutions market during the forecast period.

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However, improper infrastructure, insufficient storage systems, nascent technology in underdeveloped economies, Ethical issues related to an embryonic stem cell, low patient acceptance rate, Difficulty in the preservation of stem cell are expected to restrain the market growth. North America is expected to be the largest growing region by 2026; the reason behind that is extensive funding by Government. However, Emerging countries like India, china, Korea have low growth rate as compared to Developed regions in 2017 but increase in awareness about stem cell therapy will lead the Asia Pacific to generate a significant level of revenue by 2026.Key Highlights of Stem Cell Therapy Market report

Detailed quantitative analysis of the current and future trends from 2017 to 2026, which helps to identify the prevailing market opportunities.Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, core competencies in terms of market development, growth strategies and identification of key companies that can influence this market on a global and regional scale.Assessment of Market definition along with the identification of key drivers, restraints opportunities and challenges for this market during the forecast period.Complete analysis of micro-markets with respect to individual growth trends, prospects, and contributions to the overall Stem Cell Therapy Solutions market.Stem Cell Therapy market analysis and comprehensive segmentation with respect to the Application, End users, Treatment, and geography to assist in strategic business planning.Stem Cell Therapy market analysis and forecast for five major geographies-North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key regions.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

Research Methodology:

The market is estimated by triangulation of data points obtained from various sources and feeding them into a simulation model created individually for each market. The data points are obtained from paid and unpaid sources along with paid primary interviews with key opinion leaders (KOLs) in the market. KOLs from both, demand and supply side were considered while conducting interviews to get an unbiased idea of the market. This exercise was done at a country level to get a fair idea of the market in countries considered for this study. Later this country-specific data was accumulated to come up with regional numbers and then arrive at a global market value for the stem cell therapy market.

Key Players in the Stem Cell Therapy Market are:

Chiesi Farmaceutici S.P.A Are:Gamida CellReNeuron Group, plcOsiris Therapeutics, Inc.Stem Cells, Inc.Vericel Corporation.Mesoblast, Ltd.

Key Target Audience:

Stem Cell Associations and OrganizationsGovernment Research Boards and OrganizationsResearch and consulting firmsStem Cell Therapy Market InvestorsHealthcare Service Providers (including Hospitals and Diagnostic Centers)Stem Cell Therapeutic Product Manufacturing OrganizationsResearch LabsClinical research organizations (CROs)Stem Cell Therapy Marketing PlayersPharmaceutical Product Manufacturing Companies

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Scope of the Stem Cell Therapy Market Report:

Stem Cell Therapy market research report categorizes the Stem Cell Therapy market based on Application, End users, Treatment, and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Stem Cell Therapy market with key developments in companies and market trends.Stem Cell Therapy Market, By Treatments:

Allogeneic Stem Cell TherapyAutologous Stem Cell Therapy

Stem Cell Therapy Market, By End Users:HospitalsAmbulatory Surgical CentersStem Cell Therapy Market, By Application:OncologyCentral Nervous System DiseasesEye DiseasesMusculoskeletal DiseasesWound & InjuriesMetabolic DisordersCardiovascular DisordersImmune System DisordersStem Cell Therapy Market, By Geography:

North AmericaEuropeAsia PacificMiddle East & AfricaLatin America

Available Customization:

With the given market data, Maximize Market Research offers customization of report and scope of the report as per the requirement

Regional Analysis:

Breakdown of the North America stem cell therapy marketBreakdown of the Europe stem cell therapy marketBreakdown of the Asia Pacific stem cell therapy marketBreakdown of the Middle East & Africa stem cell therapy marketBreakdown of the Latin America stem cell therapy market

MAJOR TOC OF THE REPORT

Chapter One: Stem Cell Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Stem Cell Therapy Market Competition, by Players

Chapter Four: Global Stem Cell Therapy Market Size by Regions

Chapter Five: North America Stem Cell Therapy Revenue by Countries

Chapter Six: Europe Stem Cell Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Stem Cell Therapy Revenue by Countries

Chapter Eight: South America Stem Cell Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Stem Cell Therapy by Countries

Chapter Ten: Global Stem Cell Therapy Market Segment by Type

Chapter Eleven: Global Stem Cell Therapy Market Segment by Application

Chapter Twelve: Global Stem Cell Therapy Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Stem Cell Therapy Market Report at: https://www.maximizemarketresearch.com/market-report/stem-cell-therapy-market/522/

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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2026 - Weekly Spy

The Connection Deeper Than Blood – Jewish Link of New Jersey

By JLNJ Staff | October 10, 2019

(Courtesy of Ezer Mizion) Flying 35,000 feet above the Atlantic Ocean is not an easy job! But Ofer had already spent 17 years as a fighter pilot in the IDF defending the State of Israel. In 2003 he left the reserves and joined El-Al full time. Most people dont realize that being a pilot is a very dangerous profession. When you know it is dangerous you are safe but when you think it is easy, when youre a cowboy, you are unsafe! A pilots job is to always be alert in case something happens. Ofer always remained alert with hundreds of travelers under his wing, quite literally!

But after 16 years of flying for El-Al, Ofer started to feel fatigued. It became difficult for me to walk up with steps to the plane from the tarmac. I thought I was starting to get old or out of shape. But the truth was far more devastating: after routine blood tests, Ofer was diagnosed with leukemia!

I was immediately rushed to the hospital. When I arrived they couldnt even find bone marrow inside my body for a biopsy. I had very little bone marrow left in my body.

Ofer started to think about his future. He thought, Will I ever be able to fly again? Will I be able to see my children again? Will I get to meet my grandchildren?

It was a very difficult time in my life. I was very lucky to have the best doctors in Israel. Shortly after Jan 1, 2017, Ofer was told that Ezer Mizion had a perfect bone marrow match for him! He was thrilled, but still very hesitant. I knew I was not yet out of the woods. I was on a new medication and I was starting to feel better. I did not know if I wanted to risk a transplant with possible complications. Ofer decided to take a vacation to Moscow. He had always traveled the world and Moscow was one place he had never visited but had always wanted to see. The doctors told me if I get even a small virus I can forget about the whole transplant. I put my faith in God and said, if it is meant to be, then I will return and have the transplant.

On Feb. 28, Ofer landed back in Tel Aviv, and March 1 started his preparations for a transplant.

Pushing through all the negative thoughts, Ofer decided to fight. He was absolutely determined to overcome this illness and would go to any lengths to get better.

A short six weeks later Ofer was released from the hospital and returned to his family.

David Bugoslavski was in the middle of his military service on Mt. Hermon when he received a call from Ezer Mizion that he is a perfect match for a cancer patient. Ironically, David wasnt supposed to have his phone on him while he was in the middle of active duty. Yet, as he explains, fate thought otherwise. He knew that Ezer Mizion needed him, and while he did not know Ofer personally, he jumped at the opportunity to save the pilots life.

Thanks to Davids transplant, Ofer is alive today. While the recovery process is slow and there has been some turbulence along the way, Ofer has his life back. One of Ofers dreams had always been to fly a Boeing Dreamliner. Unfortunately, due to his medical history, this dream will never come to fruition in his capacity as a pilot but he still loves to travel the world, even if hes sitting in the back of the plane.

David was able to jump on a once-in-a-lifetime opportunity to save a life. Ofer was able to be the recipient of a special and unique kindness, having his life literally saved by someone else. As Ofer explained so beautifully, David: without you, I wouldnt be here... For me, you are part of the family.

Ezer Mizions bone marrow registry has close to 1 million registrants, with over 550,000 of them IDF soldiers. At Ezer Mizion, no matter who you are or where you come from, your life matters. Ofer and David are just one example of the lifesaving mission of Ezer Mizion taking flight. At Ezer Mizion, unconditional love is not just a term thrown around, but a philosophy that is in the very DNA of the organization. As Dr. Bracha Zisser, director and founder of Ezer Mizions National Bone Marrow Registry says, We have created a true connection of blood between two people who did not know each other at all up to that point. A connection that would not have happened without the unconditional immediate enlistment of David or, as Ofer called him, my angel.

Join Ezer Mizion on November 9 at Congregation Keter Torah in Teaneck at 7:30 p.m. for an Evening of Heroes: a beautiful musical Havdalah by Shulem Lemmer, meet real IDF heroes who have saved lives by donating their stem cells, and a fireside chat with Bret Stephens and Nachum Segal. Learn more about Ezer Mizion and RSVP for the Evening of Hereos by going to http://www.eveningofheroes.com, or contact Ryan Hyman, national director of development, at [emailprotected] or 718-853-8400 ext.109.

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The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen – World Socialist Web Site

The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen By Benjamin Mateus 10 October 2019

In the course of a lifetime, the human heart will beat more than three billion times. We will have taken more than 670 million breaths before we reach the end of our lives. Yet, these critical events remain unconscious and imperceptible in everyday life, unless we exert ourselves, such as running up several flights of stairs. We quickly tire, stop to take deep breaths and become flushed.

With the deepening comprehension by medical science of how our bodies work, we have come to better understand the fundamental importance of oxygen to life. Every living organism relies on it in one form or another. However, how cells and tissues can monitor and respond to oxygen levels remained difficult to elucidate. It has only been late in the 20th century with advances in cellular biology and scientific instrumentation that these processes have finally been explained.

On Monday, the 2019 Nobel Prize in Physiology or Medicine was awarded jointly to three individuals: William G. Kaelin, Jr., Sir Peter J. Ratcliffe, and Gregg L. Semenza. Specifically, their discoveries helped elucidate the mechanisms for lifes most basic physiologic processes.

They were able to discover how oxygen levels directly affect cellular metabolism, which ultimately controls physiological functions. More importantly, their findings have significant implications for the treatments of conditions as varied as chronic low blood counts, kidney disease, patients with heart attacks or stroke and cancers. One of the hallmarks of cancer is its ability to generate new blood vessels to help sustain its growth. It also uses these oxygen cellular mechanisms to survive in low oxygen environments.

Dr. William G. Kaelin Jr. is a professor of medicine at Harvard University and the Dana-Farber Cancer Institute. The main focus of his work is on studying how mutations in what are called tumor suppressor genes lead to cancer development. Tumor suppressor genes are special segments of the DNA whose function is to check the integrity of the DNA before allowing a copy of itself to be made and undergo cell division, which prevents cells from propagating errors. Cellular mechanisms are then recruited to fix these errors or drive the cell to destroy itself if the damage is too severe or irreparable.

His interest in a rare genetic disorder called Von Hippel-Lindau disease (VHL) led him to discover that cancer cells that lacked the VHL gene expressed abnormally high levels of hypoxia-regulated genes. The protein called the Hypoxia-Inducible Factor (HIF) complex was first discovered in 1995 by Gregg L. Semenza, a co-recipient of the Nobel Prize. This complex is nearly ubiquitous to all oxygen-breathing species.

The function of the HIF complex in a condition of low oxygen concentration is to keep cells from dividing and growing, placing them in a state of rest. However, it also signals the formation of blood vessels, which is important in wound healing as well as promoting the growth of blood vessels in developing embryos. In cancer cells, the HIF complex helps stimulate a process called angiogenesis, the formation of new blood vessels, which allows the cancer cells to access nutrition and process their metabolic waste, aiding in their growth. When the VHL gene is reintroduced back into the cancer cells, the activity of the hypoxia-regulated genes returns to normal.

Dr. Gregg L. Semenza is the founding director of the vascular program at the Johns Hopkins Institute for Cell Engineering. He completed his residency in pediatrics at Duke University Hospital and followed this with a postdoctoral fellowship at Johns Hopkins. His research in biologic adaptations to low oxygen levels led him to study how the production of erythropoietin (EPO) was controlled by oxygen. EPO is a hormone secreted by our kidneys in response to anemia. The secretion of EPO signals our bone marrow to produce more red blood cells.

His cellular and mouse model studies identified a specific DNA segment located next to the EPO gene that seemed to mediate the production of EPO under conditions of low oxygen concentration. He called this DNA segment HIF.

Sir Peter J. Ratcliffe, a physician and scientist, trained as a nephrologist, was head of the Nuffield Department of Clinical Medicine at the University of Oxford until 2016, when he became Clinical Research Director at the Francis Crick Institute. Through his research on the cellular mechanisms of EPO and its interaction between the kidneys and red cell production, he found that these mechanisms for cellular detection of hypoxia, a state of low oxygen concentration, were also present in several other organs such as the spleen and brain. Virtually all tissues could sense oxygen in their micro-environment, and they could be modified to give them oxygen-sensing capabilities.

Dr. Kaelins findings had shown that the protein made by the VHL gene was somehow involved in controlling the response to low oxygen concentrations. Dr. Ratcliffe and his group made the connection through their discovery that the protein made by the VHL gene physically interacts with HIF complex, marking it for degradation at normal oxygen levels.

In 2001, both groups published similar findings that demonstrated cells under normal oxygen levels will attach a small molecular tag to the HIF complex that allows the VHL protein to recognize and bind HIF, marking it for degradation by enzymes. If the oxygen concentration is low, the HIF complex is protected from destruction. It begins to accumulate in the nucleus where it binds to a specific section of the DNA called hypoxia-regulating genes, which sets into motion the necessary mechanisms to respond to the low oxygen concentration.

The ability to sense oxygen plays a vital role in health and various disease states. Patients who suffer from chronic kidney failure also suffer from severe anemia because their ability to produce EPO is limited. This hormone is necessary for the stem cells in our bone marrow to produce red blood cells. Understanding how cancer cells utilize oxygen-sensing mechanisms has led to a variety of treatments that targets these pathways. The ability to elucidate these mechanisms offers insight into directions scientists and researchers can take to design or create novel treatments.

The WSWS recently published its 75,000th article. Become a monthly donor today and keep up this vital work. It only takes a minute. Thank you.

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The 2019 Nobel Prize in Medicine awarded for research in cellular responses to oxygen - World Socialist Web Site

Novartis completes certification of initial sites in Quebec for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)(i) – Canada NewsWire

DORVAL, QC, Oct. 9, 2019 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that sites in Quebec have been certified in accordance with applicable requirements to treat eligible patients with Kymriah (tisagenlecleucel), the first chimeric antigen receptor T cell (CAR-T) therapy that received regulatory approval in Canada. Patients with relapsed/refractory (r/r) pediatric and young adult B-cell acute lymphoblastic leukemia (ALL) and adult r/r diffuse large B-cell lymphoma (DLBCL) may be eligible to be treated with Kymriah at one of the initially certified Canadian treatment sites. This news coincides with the Quebec government announcement that Kymriah is now reimbursed for eligible patients under the Rgie de l'assurance maladie du Qubec (RAMQ)ii.

Eligible patients in Quebec are now able to access Kymriah from the Centre hospitalier universitaire (CHU) Sainte-Justine and Maisonneuve-Rosemont Hospital (HMR) in Montreal.

"Novartis feels it is important to acknowledge the collaborative effort by all stakeholders involved to ensure Canadians have access to the first approved CAR-T therapy for patients with B-cell ALL and DLBCL who historically have poor outcomes. With treatment centers certified in Quebec, this allows patients with these two life-threatening cancers the opportunity to be treated with CAR-T therapy," said Daniel Hbert, Medical Director, Novartis Pharmaceuticals Canada Inc. "Novartis is committed to bringing additional qualified treatment centers from other parts of the country into the network to give Canadians the opportunity to be treated closer to home."

Due to the sophisticated and individualized nature of Kymriah, treatment sites that are part of the network are required to be FACT-accredited (Foundation for the Accreditation of Cellular Therapy), qualified to perform intravenous infusion of stem cells collected from the bone marrow of a donor, also referred to as allogeneic hematopoietic stem cell transplantation (alloSCT) and have experience with cell therapies, leukemia and lymphoma to facilitate safe and seamless delivery of Kymriah to eligible patients.

"We are thrilled with this news because we will now be able to treat patients at our institution with the knowledge that their therapy will be publicly funded. We see this as a significant step forward. The young patients we see who have refractory or relapsed B-cell ALL are desperately in need of a new treatment option. Kymriah brings hope to patients who are literally in a fight for their life." said Dr. Henrique Bittencourt, hematologist at the CHU Sainte-Justine in Montreal and Associate Professor, Department of Pediatrics, Universit de Montral.

"The expertise at HMR has raised the profile of our organization, which is a major Quebec, Canadian and worldwide pole for health innovation. Thanks to the dedicated work of our care, research and teaching teams, patients can now access this new treatment with demonstrated effectiveness and impact on quality of life," said Sylvain Lemieux, President and CEO, Centre intgr universitaire de sant et de services sociaux (CIUSSS) de l'Est-de-l'le-de-Montral.

About Kymriah Kymriah (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunocellular therapy, is approved to treat two life-threatening cancers that have limited treatment options and historically poor outcomes, demonstrating the critical need for new therapies for these patients.

Kymriah is approved by Health Canada for use in pediatric and young adult patients 3 to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) who are refractory, have relapsed after allogenic stem cell transplant (SCT) or are otherwise ineligible for SCT, or have experienced second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphomai.

Kymriah is a one-time treatment that uses a patient's own T cells to fight and kill cancer cells. Bringing this innovative therapy to Canadian patients requires collaboration among many health system stakeholders.

Kymriah (tisagenlecleucel) Important Safety InformationThe full prescribing information for Kymriah can be found at: http://www.novartis.ca

Novartis Leadership in Cell and Gene TherapyNovartis is at the forefront of investigational immunocellular therapy and was the first pharmaceutical company to significantly invest in CAR-T research, work with pioneers in CAR-T and initiate global CAR-T trials. Kymriah, the first approved CAR-T cell therapy in Canada, is the cornerstone of this strategy. Active research programs are underway targeting other hematologic malignancies and solid tumors, and include efforts focused on next generation CAR-Ts that involve simplified manufacturing schemes and gene edited cells.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2018, the company invested $52 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,000 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

Kymriah is a registered trademark.

References_____________________________________________i Novartis Pharmaceuticals Canada Inc., Kymriah Product Monograph. January 7, 2019.ii Quebec Ministry of Health and Social Services press release. October 8, 2019. Available at: https://www.newswire.ca/fr/news-releases/la-therapie-car-t-cell-maintenant-disponible-au-quebec-821953237.html

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Daphne Weatherby, Novartis Corporate Communications, +1 514 633 7873, E-mail: camlph.communications@novartis.com

http://www.novartis.ca

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Novartis completes certification of initial sites in Quebec for first approved Canadian CAR-T therapy, Kymriah (tisagenlecleucel)(i) - Canada NewsWire

New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says – Sickle Cell Anemia News

A newly designed viral vector the vehicle that delivers a gene therapyto a patients cells for use insickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models show.

The study Development of a forward-orientated therapeutic lentiviral vector for hemoglobin disorders was published in the journal Nature Communications.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, causessickle cell.

Gene therapies involve either altering the mutated gene or introducing a healthy version of that gene to the body. Still under development for sickle cell, an estimated 27 patients have undergone experimental gene therapy. One strategy involves removing hematopoietic stem cells (which function to produce blood cells) from a patients bone marrow. A healthy copy of the HBB gene is then introduced into the cells using a modified, harmless virus known as a viral vector. The cells are then transplanted back into the patient where they will produce healthy red blood cells.

Traditionally, viral vectors for sickle cell have been designed in a way known as reverse structural orientation. This means that the HBB gene is translated or read from right to left, like reading an English sentence backwards. The reverse structural orientation design ensures that a key section of the gene (known as intron 2), which is necessary for the production of high levels of the HBB gene, is retained during viral vector preparation.

However, this design makes preparing the viral vectors more difficult, and decreases the efficiency of introducing the gene into the stem cells.

Researchersat the National Institutes of Healthdesigned a new viral vector, one in which the HBB gene is forward orientated and read from left to right. Genes essential for the virus were inserted into intron 2, meaning that only vectors that retained intron 2 would be produced (a type of positive selection).

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, John Tisdale, MD, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI) and the studys senior author, said in a press release.

Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder, he added.

The researchers compared the new vectors to traditional reverse-orientated vectors in mouse and monkey models. The new vectors were four to 10 times more efficient at introducing the healthy HBBgene into the stem cells, and could carry up to six times more HBB genes compared to the conventional vectors.

Furthermore, the new vectors remained incorporated into the cells of monkeys up to four years after a transplant. These vectors could also be produced in greater amounts, which may lessen the time and costs required for large-scale vector production.

The researchers hope that these characteristics will make gene therapy for sickle cell disease more effective and increase its use. The new vector design still needs to be tested in clinical trials in patients.

Our lab has been working on improving beta-globin vectors for almost a decade and finally decided to try something radically different and it worked, Tisdale said.

These findings bring us closer to a curative gene therapy approach for hemoglobin disorders, he added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says - Sickle Cell Anemia News

Penny Lancaster is reduced to tears as she gives bone marrow donor Ronnie Musselwhite, 5, The Suns Young H – The Sun

PENNY Lancaster was reduced to tears as she handed five-year-old Ronnie Musselwhite the Young Hero gong at The Suns Who Cares Wins health awards last night.

Spurs fan Ronnie bravely offered to give his older sister Ebonie a bone marrow transplant last year.

Eight-year-old Ebonie had leukaemia and it was her only hope.

Mum Christine Jenkins, 40, said: Ronnies stem cells worked perfectly. They did what they were supposed to do but the leukaemia came back somewhere new.

Ebonie, of Crawley, West Sussex, nominated her younger brother before her death in June.

Rod Stewarts partner Penny chatted with Ronnie about his love of football and Spurs.

She said: To say I am humbled to be here is an understatement. Sometimes you think life has turned a corner on you, but then someone else turns up to give you some inspiration.

"The courage this little man has shown is absolutely incredible. Hes only five years old, hes lost a sister.

"He was incredibly shy to stand up in front of everyone to collect his award, but he again was so brave.

Christine said: We want Ronnie to know that what he did still worked, was still brave, even though he lost his sister.

PM Boris Johnson was also at the awards held at The Suns London HQ near The Shard and paid tribute to our NHS heroes.

He presented an award to a pair of quick-thinking hospital porters who saved the life of a seven-week-old baby boy.

Nick Evans, 48, and Ruth Lowe, 47, sprang into action after Logan Clifford stopped breathing.

His parents, Sarah and Mike were visiting a relative at the Princess Royal Hospital in Telford, Shrops, when they noticed Logans lips had turned blue.

Sarahs screams alerted Ruth, who shouted for Nick. He grabbed Logan and performed CPR as he ran half a mile down the corridor to A&E.

Nick continued CPR until the resuscitation team took over and the porters stayed by Logans parents side until they knew he was going to be OK. Sarah, 30, has called the two porters my heroes.

As he handed the pair the Ultimate Lifesaver trophy, the PM said: The NHS is revered around the world, and in no small part due to the heroes working in it every day.

He added: My experience of the NHS is like everybody else in the NHS - one of admiration and love.

"It is the most extraordinary institution in the world. If our country was an omelette then the NHS is the egg white that holds the great British cake together.

Virgin Radio DJ Chris Evans presented the Best Nurse gong to Liz Monaghan, 53. She set up the widely praised Purple Rose initiative, which aims to improve the care for patients and their loved ones in the last days of their life.

Liz, who works at the Florence Nightingale Hospice, based at the Stoke Mandeville Hospital in Aylesbury, Bucks, said: Im a little embarrassed to have won. Im a small part of a big team.

DJ Chris said: Youve got to prepare yourself for nights like this because otherwise they hit you like an express train.

Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

Other nominees: Superhero Foundation and Team Domenica

BEST NEONATAL SPECIALIST

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

Other nominees: Dr Vesna Pavasovic and Professor Massimo Caputo

UNSUNG HERO

Winner: Therapeutic Care Volunteers at South Tees NHS Foundation Trust

30 therapeutic care volunteers, who all have a learning or physical disability, give up their time to support patients with spinal injuries at The James Cook University Hospital in Middlesbrough. They include Ify Nwokoro.

Other nominees: Ben Slack and Rob Allen

GROUNDBREAKING PIONEER

Winner: Guys and St Thomas London Auditory Brainstem Implant (ABI) Service

Leia Armitage, eight, was born with a rare form of deafness and was never expected to speak. But she now can thanks to pioneering brain surgery and speech therapy carried out by Guys and St Thomas London Auditory Brainstem Implant (ABI) Service.

Other nominees: Dr Helen Spencer and Girish Vajramani

BEST DOCTOR

Winner: Dr Matthew Boulter

Dr Boulter served in Afghanistan, teaches wild trauma to army medics and his surgery became the first in Cornwall to be given veteran friendly accreditation.

Other nominees: Margaret France and Dr Bijay Sinha

BEST MIDWIFE

Winner: Jane Parke

Jane helped deliver the youngest surviving twin boys in Britain when they were born at 22 weeks last year. She flew 190 miles with their mum Jennie Powell to a specialist neonatal unit.

Other nominees: Charlotte Day and Nagmeh Teymourian

ULTIMATE LIFESAVER

Winner: Ruth Lowe and Nick Evans

Porters Ruth and Nick saved the life of Sarah and Mike Cliffords seven-week-old baby Logan. He stopped breathing as they walked through the main entrance of The Princess Royal Hospital in Telford to visit a sick relative.

Other nominees: Dr Mark Forrest and Mike Merrett

BEST NURSE

Winner: Liz Monaghan

Liz is the Matron of the Florence Nightingale Hospice in Aylesbury, Bucks, and came up with the idea for the widely praised Purple Rose initiative to improve the care for patients in the last days of their lives.

Other nominees: Margaret Ballard and Carlton DeCosta

MENTAL HEALTH HERO

Winner: Ben West

Ben lost his brother Sam, 15, to suicide last year and since his death, has campaigned tirelessly to raise awareness for mental health.

Other nominees: Beth Gregan and Catherine Benfield

YOUNG HERO

Winner: Ronnie Musselwhite

Ronnie offered to help his sister Ebonie by giving her a bone marrow transplant when she was diagnosed with a rare form of leukaemia. Ebonie nominated her brother for his bravery before she died in June.

Other nominees: Bella Field and Kaitlyn Wright

I only walked ten metres into the room tonight and I already nearly burst into tears three times.

TV star Christine Lampard gave the Best Neonatal Specialist award to Prof Kypros Nicolaides, 66.

He was nominated by Sherrie Sharp, 29, of Horsham, West Sussex, for saving the life of her unborn baby son and her own.

After scans revealed Jaxson had spina bifida, Sherrie was offered a termination. But she contacted Prof Nicolaides, a surgeon at Kings College Hospital, London.

He had saved her life 30 years earlier when she developed a rare blood disorder in her mums womb.

He agreed to perform ground-breaking surgery on Jaxson while he was in Sherries womb.

Prof Nicolaides said: I was delighted to be able to help. Sherrie said: He has saved so many generations of my family. Hes our guardian angel.

The Who Cares Wins Awards were set up in 2017 by The Sun to honour the nations heroic doctors, nurses, midwives, other NHS staff and volunteers.

The Duchess of York presented an award to the parents of Natasha Ednan-Laperouse, 15, who died of an allergic reaction to a sandwich from Pret.

The duchess said: Can I just say to The Sun, I think youre incredible. Every minute Im sitting there and thinking Im so lucky. The NHS, The Sun and all of you, this is what makes Britain so great.

Lorraine Kelly, who presented the awards, said: Earlier on this year my dad was very sick and we honestly thought we were going to lose him.

"It was really difficult and it was only because of the efforts of the NHS hes still here. Its fantastic.

Who Cares Wins Awards: The winners

BEST HEALTH CHARITY

Nominees: Superhero Foundation

Team Domenica

Winner: Matt Hampson Foundation

Former English rugby union player Matt Hampson set up a charity to help others after being left paralysed in a scrum in 2005.

BEST NEONATAL SPECIALIST

Nominees: Dr Vesna Pavasovic

Professor Massimo Caputo

Winner: Professor Kypros Nicolaides

Professor Nicolaides performed pioneering keyhole surgeon on Sherrie Sharps unborn son Jaxon. By extraordinary coincidence, as a young surgeon, he also operated on Sherries mother when she was in the womb.

UNSUNG HERO

Nominees: Ben Slack

Rob Allen

Winner: Therapeutic Care Volunteers at South Tees NHS Foundation Trust

30 therapeutic care volunteers, who all have a learning or physical disability, give up their time to support patients with spinal injuries at The James Cook University Hospital in Middlesbrough. They include Ify Nwokoro.

GROUNDBREAKING PIONEER

Nominees: Dr Helen Spencer

Girish Vajramani

Winner: Guys and St Thomas London Auditory Brainstem Implant (ABI) Service

Leia Armitage, eight, was born with a rare form of deafness and was never expected to speak. But she now can thanks to pioneering brain surgery and speech therapy carried out by Guys and St Thomas London Auditory Brainstem Implant (ABI) Service.

BEST DOCTOR

Nominees: Margaret France

Dr Bijay Sinha

Winner: Dr Matthew Boulter

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Penny Lancaster is reduced to tears as she gives bone marrow donor Ronnie Musselwhite, 5, The Suns Young H - The Sun

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