The great stem cell dilemma

By Jeffrey M. O'Brien, contributor

Stem cells stored in liquid nitrogen at Advanced Cell Technology in Marlborough, Mass.

FORTUNE -- Imagine yourself the proud but rueful owner of an ancient Jaguar. Every day you dread the uncertainty that comes with trying to get from here to there -- there, more often than not, being the shop. No sooner does one ailment find repair than another appears. At best, it's a slow, uncomfortable ride. Lonely too. There's really no one around who fully understands your plight.

That is how Patricia Riley describes life in a 95-year-old body. Riley, who reached that milestone birthday last St. Patrick's Day, lives alone in the same 1,100-square-foot house in Plainfield, Conn., that she's called home for 64 years, having survived her husband (heart disease), a daughter (breast cancer), and every friend she ever had. "All the people I knew have all gone, Jeffrey," she says in a quivering voice laced with melancholy. "They've all died. I go to church and I never see people my age." Her remaining family includes two daughters, five grandchildren, and eight great-grandchildren, including my two young sons. In a nod to her French-Canadian heritage, we call her Mme.

Mme attributes her longevity to good genes, but she clearly owes a debt to modern medicine. Over the years she's had a cholecystectomy, a hysterectomy, esophageal surgery, a stroke, and ulcerative colitis. Lately she relies on a cane and a walker, and her daily regimen includes pain pills for arthritis, two inhalers for asthma, high-blood-pressure meds, a statin, vitamins, digestion aids, and an anti-anxiety drug that she calls "my nerve pill." Her vision also comes courtesy of medical science. Three years ago Mme was diagnosed with a form of age-related macular degeneration, or AMD, a disease of the back of the retina that is the leading cause of vision loss in the developed world. The ophthalmologist gave her a choice: a needle into her eyeballs every six weeks, or blindness. Mme opted for the injections and now receives shots of an off-label cancer drug called Avastin, which has demonstrated efficacy in halting the progress of her type of AMD. Holding the ailment at bay is all she can hope for. "I'll have to go for as long as I live," she says. "It's just a treatment -- it's not a cure."

Treatments, not cures. This, in a nutshell, is the MO of our health care system, and it's precisely the reason that regenerative medicine -- and stem cell therapy in particular -- has been the subject of so much hope and hype over the past decade or so. Stem cell therapies promise to empower a body to fight ailments by enabling it to build new parts. Think about growing new neurons or heart tissue. Think about the difference between perpetually slathering that old Jag with Bondo and having it heal itself overnight in the garage.

MORE:Stem cell dollars: California leads the way

While stem cells have ignited plenty of religious outrage and political grandstanding, behind the headlines the underlying science has been advancing the way science often does -- by turns slowly and dramatically. To be clear, the earliest stem cell therapies are almost certainly years from distribution. But so much progress has been made at venerable research institutions that it now seems possible to honestly discuss the possibility of a new medical paradigm emerging within a generation. Working primarily with rodents in preclinical trials, MDs and Ph.D.s are making the paralyzed walk and the impotent virile. A stem cell therapy for two types of macular degeneration recently restored the vision of two women. Once they were blind. Now they see! Some experts assert that AMD could be eradicated within a decade. Other scientists are heralding a drug-free fix for HIV/AIDS. Various forms of cancer, Parkinson's, diabetes, heart disease, stroke, and ALS have already been eradicated in mice. If such work translates to humans, it will represent the type of platform advancement that comes along in medicine only once in a lifetime or two. The effect on the economy would be substantial. Champions of stem cell research say it would be on the order of the Internet or even the transistor.

The obstacles along the road from lab rat to human patients are many, of course, but the biggest by far is money. With the dramatic events in the lab, you might think that a gold rush would be under way. That's far from true. Long time horizons, regulatory hurdles, huge R&D costs, public sentiment, and political headwinds have all scared financiers. Wall Street isn't interested in financing this particular dream. Most stem cell companies that have dared go public are trading down 90% or more from their IPOs. Sand Hill Road is AWOL. The National Venture Capital Association doesn't even have a category to track stem cell investments.

Big Pharma would seem to be the most obvious benefactor. The drug companies understand the complexities (and billion-dollar outlays) involved in bringing therapies to market. A few drug companies have kicked the tires on stem cells over the years, but waiting for them to undo the current model is akin to banking on Big Oil to rethink energy. They may do it, but it's unlikely to be by choice. Which leaves stem cell researchers begging for state and federal grants at a time scientific funding is under siege.

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The great stem cell dilemma

Cytori’s Stem Cell Therapy for Burns Wins U.S. Contract

By Ryan Flinn - 2012-09-28T20:12:41Z

Cytori Therapeutics Inc. (CYTX), a biotechnology company with $10 million in annual revenue, rose the most in about a year after the company won a $4.7 million U.S. government contract to develop a stem cell therapy to treat burns caused by thermal or radioactive bombs.

Cytori jumped 14 percent to $4.41 at the close in New York, the biggest single-day increase since October 2011. The shares of the San Diego-based company have doubled this year.

Were seeing a lot of momentum, Chief Executive Officer Christopher Calhoun said today in an interview with Bloomberg Television. This contract is one more major thing that we are delivering on, and there is more to come.

The two-year contract with the Department of Health and Human Services Biomedical Advanced Research and Development Authority may be worth $106 million over five years if certain milestones are met, Cytori said today in a statement. The company had a net loss last year of $32 million, according to data compiled by Bloomberg.

Cytoris experimental therapy takes adipose tissue, or body fat, from a patient and through its device separates the adult stem and regenerative cells before transferring them to a burn wound. Money from the contract will be used to develop the device and take it through the U.S. regulatory approval process with the Food and Drug Administration, Calhoun said.

These cells help to facilitate the healing of the injury, he said in a telephone interview earlier this week. They release growth factors that stimulate new blood flow.

Testing the technology in a clinical trial and getting approval may take five years, Calhoun said. The company is currently testing its therapy for other soft tissue damage, as well as cardiovascular disease.

Once approved, the device will be deployed in hospitals across the country, and can be used for routine burns as well as a treatment for patients in wake of a mass casualty event that could injure 10,000 people, Cytori said in the statement.

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

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Cytori’s Stem Cell Therapy for Burns Wins U.S. Contract

StemCells, Inc. Achieves Spinal Cord Injury Milestone With First Neural Stem Cell Transplant Into Patient With Sensory …

NEWARK, Calif., Sept. 27, 2012 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) today announced that the first patient with an incomplete spinal cord injury has been enrolled in the Company's Phase I/II clinical trial in chronic spinal cord injury and transplanted with the Company's proprietary HuCNS-SC(R) neural stem cells. The patient, a Canadian man who suffered a thoracic spinal cord injury from a sports-related accident, was administered the cells yesterday at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation. This is the first patient in the second cohort of the trial, which will be comprised of four patients who retain some sensory function below the level of trauma and are therefore considered to have an incomplete injury.

"This is an important milestone for StemCells and the spinal cord injury community as it is the first time anyone has ever transplanted neural stem cells into a patient with an incomplete injury," said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. "Given the encouraging interim data from the most severely injured patient cohort that we reported earlier this month, testing patients with less severe injury should afford us an even better opportunity to continue to test safety and to detect and assess clinical changes. Unlike the patients in the first cohort, patients with incomplete injuries have retained a degree of spinal cord function that might be even further augmented by transplantation with neural stem cells."

Earlier this month, the Company reported that interim six-month data from the first patient cohort in the Phase I/II clinical trial continued to demonstrate a favorable safety profile, and showed considerable gains in sensory function in two of the three patients compared to pre-transplant baselines. Patients in the first cohort all suffered a complete injury to their spinal cord, leaving them with no neurological function below the level of injury. Following transplantation with HuCNS-SC cells, there were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first six months after transplantation. Changes in sensitivity to touch, heat and electrical stimuli were observed in well-defined and consistent areas below the level of injury in two of the patients, while the third patient remained stable. Importantly, the changes in sensory function were confirmed objectively by measures of electrical impulse transmission across the site of injury, each of which correlated with the clinical examination.

About the Spinal Cord Injury Clinical Trial

The Phase I/II clinical trial of StemCells, Inc.'s HuCNS-SC(R) purified human adult neural stem cells is designed to assess both safety and preliminary efficacy. Twelve patients with thoracic (chest-level) neurological injuries at the T2-T11 level are planned for enrollment, and their injuries must have occurred within three to twelve months prior to transplantation of the cells. In addition to assessing safety, the trial will assess preliminary efficacy based on defined clinical endpoints, such as changes in sensation, motor function and bowel/bladder function. The Company has dosed the first patient cohort, all of whom have injuries classified as AIS A according to the American Spinal Injury Association Impairment Scale (AIS). In AIS A injuries, there is no neurological function below the injury level. The second cohort will be patients classified as AIS B, in which there is some preservation of sensory or motor function below the injury level. The third cohort will be patients classified as AIS C, in which there is some preservation of both sensory and motor function.

All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord and will be temporarily immunosuppressed. Patients will be evaluated regularly in the post-transplant period in order to monitor and assess the safety of the HuCNS-SC cells, the surgery and the immunosuppression, as well as to measure any recovery of neurological function below the injury site. The Company intends to follow the effects of this therapy long-term, and each of the patients will be invited to enroll into a separate four year observational study after completing the Phase I/II study.

The trial is being conducted at Balgrist University Hospital, University of Zurich, a world leading medical center for spinal cord injury and rehabilitation, and is open for enrollment to patients in Europe, Canada and the United States. Enrollment for the second cohort is now underway. If you believe you may qualify and are interested in participating in the study, please contact the study nurse either by phone at +41 44 386 39 01 or by email at stemcells.pz@balgrist.ch.

Additional information about the Company's spinal cord injury program can be found on the StemCells, Inc. website at http://www.stemcellsinc.com/Therapeutic-Programs/Clinical-Trials.htm and at http://www.stemcellsinc.com/Therapeutic-Programs/Spinal-Cord-Injury.htm, including video interviews with Company executives and independent collaborators.

About Balgrist University Hospital

Balgrist University Hospital, University of Zurich is recognized worldwide as a highly specialized center of excellence providing examination, treatment and rehabilitation opportunities to patients with serious musculoskeletal conditions. The clinic owes its leading international reputation to its unique combination of specialized medical services. The hospital's carefully-balanced, interdisciplinary network brings together under one roof medical specialties including orthopedics, paraplegiology, radiology, anesthesiology, rheumatology, and physical medicine. More information about Balgrist University Hospital is available at http://www.balgrist.ch.

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Mayo Clinic finds way to weed out problem stem cells, making therapy safer

Public release date: 27-Sep-2012 [ | E-mail | Share ]

Contact: Jennifer Schutz newsbureau@mayo.edu 507-284-5005 Mayo Clinic

ROCHESTER, Minn. -- Mayo Clinic researchers have found a way to detect and eliminate potentially troublemaking stem cells to make stem cell therapy safer. Induced Pluripotent Stem cells, also known as iPS cells, are bioengineered from adult tissues to have properties of embryonic stem cells, which have the unlimited capacity to differentiate and grow into any desired types of cells, such as skin, brain, lung and heart cells. However, during the differentiation process, some residual pluripotent or embryonic-like cells may remain and cause them to grow into tumors.

"Pluripotent stem cells show great promise in the field of regenerative medicine; however, the risk of uncontrolled cell growth will continue to prevent their use as a therapeutic treatment," says Timothy Nelson, Ph.D., M.D., lead author on the study, which appears in the October issue of STEM CELLS Translational Medicine.

Using mouse models, Mayo scientists overcame this drawback by pretreated stem cells with a chemotherapeutic agent that selectively damages the DNA of the stem cells, efficiently killing the tumor-forming cells. The contaminated cells died off, and the chemotherapy didn't affect the healthy cells, Dr. Nelson says.

"The goal of creating new therapies is twofold: to improve disease outcome with stem cell-based regenerative medicine while also ensuring safety. This research outlines a strategy to make stem cell therapies safer for our patients while preserving their therapeutic efficacy, thereby removing a barrier to translation of these treatments to the clinic," says co-author Alyson Smith, Ph.D.

Stem cell therapies continue to be refined and improved. Researchers are finding that stem cells may be more versatile than originally thought, which means they may be able to treat a wider variety of diseases, injuries and congenital anomalies. Stem cell therapy is an emerging regenerative strategy being studied at Mayo Clinic.

"By harnessing the potential of regenerative medicine, we'll be able to provide more definitive solutions to patients," says Andre Terzic, M.D., Ph.D., co-author and director of Mayo Clinic's Center for Regenerative Medicine.

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Other members of the Mayo research team included Clifford Folmes, Ph.D., Katherine Hartjes, Natalie Nelson and Saji Oommen, Ph.D. The research was supported by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, National Institutes of Health New Innovator Award OD007015-01, and a Mayo Clinic Center for Regenerative Medicine accelerated research grant.

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Deadly complication of stem cell transplants reduced in mice

ScienceDaily (Sep. 27, 2012) Studying leukemia in mice, researchers at Washington University School of Medicine in St. Louis have reduced a life-threatening complication of stem cell transplants, the only curative treatment when leukemia returns.

About 50 percent of leukemia patients who receive stem cells from another person develop graft-versus-host disease, a condition where donor immune cells attack the patient's own body. The main organs affected are the skin, liver and gut. Now, the scientists have shown they can redirect donor immune cells away from these vital organs. Steering immune cells away from healthy tissue also leaves more of them available for their intended purpose -- killing cancer cells.

"This is the first example of reducing graft-versus-host disease not by killing the T- cells, but simply by altering how they circulate and traffic," says John F. DiPersio, MD, PhD, the Virginia E. and Sam J. Golman Professor of Medicine. "Donor T-cells do good things in terms of eliminating the recipient's leukemia, but they can also attack normal tissues leading to death in a number of patients. The goal is to minimize graft-versus-host disease, while maintaining the therapeutic graft-versus-leukemia effect."

The study is now available online in Blood.

Working with mice, Jaebok Choi, PhD, research assistant professor of medicine, showed that eliminating or blocking a particular protein -- the interferon gamma receptor -- on donor T-cells makes them unable to migrate to critical organs such as the intestines but still leaves them completely capable of killing leukemia cells.

"The fact that blocking the interferon gamma receptor can redirect donor T-cells away from the gastrointestinal tract, at least in mice, is very exciting because graft-versus-host disease in the gut results in most of the deaths after stem cell transplant," DiPersio says. "People can tolerate graft-versus-host disease of the skin. But in the GI tract, it causes relentless diarrhea and severe infections due to gut bacteria leaking into the blood, which can result in severe toxicity, reduction in the quality of life or even death in some patients."

Long known to be involved in inflammation, the roles of interferon gamma, its receptor and their downstream signaling molecules are just beginning to be described in the context of graft-versus-host disease, says DiPersio, who treats patients at Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine.

The cascade begins when interferon gamma activates its receptor. The interferon gamma receptor then activates molecules known as JAK kinases, followed by STAT, and finally CXCR3. CXCR3 mediates the trafficking of donor T-cells to the GI tract and other target organs.

Since deleting the interferon gamma receptor from donor T-cells directs them away from target organs, the researchers asked whether they could produce the same beneficial effects by inhibiting some of the receptor's downstream signaling molecules. Indeed, Choi also found that knocking out CXCR3 reduces graft-versus- host disease, but not completely.

"There are probably additional downstream targets of interferon gamma receptor signaling other than JAKs, STATs and CXCR3 that are responsible for T-cell trafficking to the GI tract and other target organs," DiPersio says. "We're trying to figure out what those are."

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Deadly complication of stem cell transplants reduced in mice

Study Shows Stem Cells May Prevent And Cure Alzheimer's

SEOUL, South Korea, Sept. 26, 2012 /PRNewswire/ --In the first study of its kind, researchers at Korea's leading university and the RNL Bio Stem Cell Technology Institute announced this week the results of a study that suggests an astounding possibility: adult stem cells may not only have a positive effect on those suffering from Alzheimer's disease, theycanprevent the disease.Using fat-derived adultstem cells from humans [scientific term:adMSCs, orhuman, adipose-derived mesenchymal stem cells], researchers were able to cause Alzheimer's disease brains in animal models to regenerate. The researchers, for the first time in history, used stem cells toidentify the mechanism that is key to treatment of Alzheimer's disease, and demonstrated how to achieve efficacy as well as prevention of the symptoms of Alzheimer's with adult stem cells, a "holy grail" of biomedical scientists for decades.

Alzheimer's disease, the most common form of dementia (loss of brain function), is the 6th leading cause of death, and affects 1 in 8 people -- more than breast cancer. As of 2010, there were 35.6 million people with Alzheimer's disease in the world, but this number is expected to double every 20 years. It is estimated that the total cost of Alzheimer's is US $604 billion worldwide, with 70% of this cost in the US and Europe. To put that in perspective, Alzheimer's care costs more than the revenues of Wal-Mart (US$414 billion) and Exxon Mobil (US$311 billion), according to the British World Alzheimer's Report of ADI. The cost of Alzheimer's is at the top of health economists' list of the disorders of aging that could topple nations' entire economies, and that regularly ruin not only the lives of patients but of their relatives.

According to the results of this first major study, Alzheimer's may soon be a preventable disease, or even a thing of the past. Equally important, the safety human administration of the kind of adult stem cells used in this experiment has been established in multiple articles and government-approved clinical trials.

THE RESEARCH:

The study was jointly led by Seoul National University Professor Yoo-Hun Suh and RNL Bio Stem Cell Technology Institute (SCTI) director Dr. Jeong-Chan Ra.

The researchers and their teams injected stem cells into mice genetically designed to have the core symptoms and physiology of Alzheimer's disease. They were able to identify that these human stem cells, derived from adipose tissue, behave in a very special way when injected into the tail vein of mice subjects. The cells migrated through the blood brain barrier, thought by many to be impossible for adult stem cells to cross, and went into the brain. In fact,fluorescent labeled cells were monitored for distribution in subjects and the team identified that the infused cells migrated throughout the bodiesincluding brainexcept the olfactory organ, and therefore confirmed that IV infused stem cell can reach to the brain across the blood brain barrier.

The team infused human adipose stem cells intravenously in Alzheimer model mice multiple times two weeks apart from three month to 10 month.Once there, the mice who received cells improved in every relevant way: ability to learn, ability to remember, and neuropathological signs. More important, for the first time ever, Alzheimer model mice showed the mediation of IL-10, which is known for anti-inflammation and neurological protection.

The team also found that stem cell restored special learning ability from Alzheimer model subjects with great reduction of neuropathy lesions.This was found using tests used for Alzheimer's disease: behavioral assessment. In assessment it was found, amazingly, that stem cells' therapeutic effect on Alzheimer's disease was tremendous. This was also found in pathological analysis. The key though was prevention: the scientists showed that stem cells, when infused into Alzheimer's mice, decreased beta amyloid and APP-CT, known to cause brain cell destruction, leading to dementia and Alzheimer's disease. In the lab it was clear that stem cells increased neprilysin, which hydrolyzes toxic proteins. No other compound or treatment has ever suggested so strongly the potential to prevent, as well as stop, this epidemic of incurable dementia sweeping across suffering patients and their families.

Stopping Alzheimer's disease, let alone preventing it, is the focus of thousands of researchers worldwide. Speaking of their breakthrough discovery,Professor Yoo-Hun Suh, who led the study, said, "It is a ground breaking discovery that such a simple method as IV injection of the safest autologous adipose stem cells, without causing any immune rejection, or any ethical issues, opened a new door to conquering Alzheimer's disease, one of the most horrible, expensive and incurablediseases of our time." Joining him, leader of the RNL Bio Stem Cell Technology InstituteDr. Jeong-Chan Ra said, "It has never been more clear that it is an ethical imperative for governments to provide patients with incurable diseases with their right to participate not only in studies like this but in therapies with such obvious potential, once they have been tested as many times for safety as has our technology." Both scientists stressed that the real breakthrough in their complex research is the prevention of the onset of symptoms.

Specifically, stem cells grafted in the brain, in another part of the study, were identified to induce cell division and neuro differentiation of endogenous neuro progenitor cells around the hippocampus and its surrounding cells and increase in great deal the stability of dendrites and synapses. Stem cell also contributed various anti-inflammatory and neuro growth factors, especially increased the expression of IL-10. This again suppressed apoptosis of brain neurons, the prevention effect against Alzheimer's disease.

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Study Shows Stem Cells May Prevent And Cure Alzheimer's

Three-dimensional fiber scaffold promotes large-scale stem cell proliferation and differentiation

Pluripotent embryonic stem cells encapsulated in the fiber scaffold developed at IBN. Credit: A*STAR Institute of Bioengineering and Nanotechnology

Thanks to the ability of pluripotent stem cells to self-renew and differentiate into a wide variety of specialized cell types, they are expected to revolutionize the treatment of illnesses such as type I diabetes and Parkinson's disease. Before this becomes a reality, however, scientists must develop culture systems to mass-produce these cells. To overcome the limitations of previous single-layer-substrate systems, a research team in Singapore has developed three-dimensional scaffolds that stimulate stem cell proliferation and differentiation under defined chemical conditions. Importantly, the system can be scaled up. The scaffolds consist of microscopic fibers obtained by weaving together polymer strands bearing opposite charges.

Hongfang Lu and Andrew Wan from the A*STAR Institute of Bioengineering and Nanotechnology led the research. Wan notes that the fiber-based scaffold not only avoids the need to consume large quantities of key growth factors, but it would also shield the cells from the shear stresses generated in large-scale bioreactors.

To manufacture the scaffold, the researchers opted for a positively charged biopolymer called chitin, which they extracted from crab shell, and a negatively charged polymer called sodium alginate. After depositing one droplet of each of these water-soluble polymers onto a sterile substrate, they brought the droplet interfaces into contact using forceps; this formed a chitinalginate complex. Held together by intermolecular electrostatic interactions, the complex extended into a continuous fiber. The team reeled the fiber onto a holder to complete the three-dimensional system.

By suspending the stem cells in the alginate solution, Lu, Wan and co-workers incorporated the cells into the scaffold during fiber formation, resulting in a network of uniformly distributed cells (see image). Preliminary tests showed that when the researchers destroyed the scaffold with enzymes, they could recover a high number of the cells.

Lu explains that their system provided a 'micro-environment' in which cells could grow in aggregates. When sub-cultured over many generations, the encapsulated stem cells remained pluripotent and did not undergo any genetic mutations. Moreover, the cells displayed excellent viability when frozen in the fiber for storage; in addition, they could either self-renew or differentiate, depending on the media available to them. "The small dimensions of the fibers are useful because they allow nutrients and growth factors to efficiently diffuse towards the cells within the scaffold," she adds.

The team is now planning to exploit their approach to produce transplantable tissue for cell-based therapy. "Our system allows us to generate large numbers of cells for tissue-engineering applications," says Wan.

More information: Lu, H. F., Narayanan, K., Lim, S.-X., Gao, S., Leong, M. F. & Wan, A. C. A. A 3D microfibrous scaffold for long-term human pluripotent stem cell self-renewal under chemically defined conditions. Biomaterials 33, 24192430 (2012): article

Provided by Agency for Science, Technology and Research (A*STAR), Singapore

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Three-dimensional fiber scaffold promotes large-scale stem cell proliferation and differentiation

Stem cell transplantation for boy with thalassaemia

A 10-year-old boy has been infused with stem cells harvested from the bone marrow of his brother to treat him for thalassaemia a disorder caused by destruction of red blood cells. Called allogeneic transplantation of stem cells, this was done at Kovai Medical Center and Hospital.

D. Dhanush may not have to undergo expensive and excruciating blood transfusion anymore if his body accepts the donor cells. But his condition will have to be evaluated very minutely for the next two years to confirm that the cells donated by his brother have been received well and adapted him.

Presenting the boy before media persons, Clinical Haematologist and Head of the Bone Marrow Transplant Unit T. Rajasekar explained that transplantation was of two types autologous and allogeneic.

The autologous procedure involves harvesting of stem cells from the patients themselves (those suffering from thalassaemia or leukaemia). The extracted cells are frozen and stored for high dose treatment.

After being treated, these are infused into the patient through a vein. This procedure was done for one person suffering from myeloma (cancer of plasma cells or white blood cells that produce anti-bodies that help fight infections/diseases) and another with a relapsed lymphoma (cancer of the lymphocytes cells that are part of immune system).

Under the allogeneic procedure, matching stem cells from a donor are used. Mostly, these cells are from siblings or a close relative as they need to pass the human leukocyte antigen (HLA) matching test. HLA matching is required, or the cells will be rejected by the recipient. Ideally, it is sibling whose cells will match because he or she will have the HLA from both parents. It is the combination of HLAs from both parents that are found in the children.

The cells can be harvested from the marrow or from the blood. In the case presented on Tuesday, Dr. Rajasekar said the cells were brought out of the bone marrow in Dhanushs brother and into his blood, from where these were harvested.

Chairman of the hospital Nalla G. Palaniswami said the tough procedure was performed by the new Comprehensive Cancer Centre, which was gradually bringing in specialists of all sub-specialities of cancer care. Only then can this be called a comprehensive centre, he said.

The hospital would form a KMCH Foundation, which would use funds from donors to treat poor children suffering from cancer and some other disorders that required expensive treatment.

The stem cell transplantation that Dhanush, the son of a police head constable, underwent cost Rs.12 lakh. Of this, Rs.9 lakh was provided by a donor, Dr. Palaniswami said. Dean of the hospital V. Kumaran and Head of Department of Interventional Radiology Mathew Cherian spoke on how the cancer centre was established and how developments were being made.

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Stem cell transplantation for boy with thalassaemia

Pacemaker from Stem Cells Receives Research Funding

(SACRAMENTO, Calif.) - Deborah K. Lieu, a stem cell scientist in cardiovascular medicine at UC Davis Health System, has received a $1.3 million research grant from the California Institute for Regenerative Medicine (CIRM) to develop stem cells that could serve as a biological alternative to the electronic pacemakers that people now use to regulate heart rhythm.

According to Lieu, each year 350,000 cardiology patients with abnormal heart rhythms receive electronic pacemakers to maintain a normal heart beat. The devices, while effective, have several disadvantages, including limited battery life and poor response to changing heart rates, such as when a person is exercising. Lieu, who is working with colleague Nipavan Chiamvimonvat, the Roger Tatarian Endowed Professor of Cardiovascular Medicine at UC Davis, plans to examine ways to improve the generation of pacemaking cells using human-induced pluripotent stem cells (hiPSCs), potentially creating what she calls a "biopacemaker."

"There are more than 3 million patients around the country who are dependent on electronic pacemakers," said Lieu. "Each one costs about $58,000 to implant and requires follow-up surgery about every 5 to 10 years to change batteries. Creating a biopacemaker from stem cells would avoid the burden of battery replacement and provide the physiological benefit of enabling a person's heart to naturally adapt to a rising heart rate during activities such as exercise."

Lieu's grant was among more than two dozen projects that received support from state stem cell agency's governing board last week as part of CIRM's Basic Biology awards program. The funding focuses on basic research projects that can provide a better understanding about the fundamental mechanisms of stem cell biology and move researchers closer to knowing how best to use stem cells to help patients.

To create the pacemaking cells, Lieu and her colleagues plan to manipulate an ion channel (the SK channels in cardiac myocytes) to alter the calcium signaling mechanisms during hiPSC differentiation. Stem cell scientists create hiPSCs - typically from an adult cell such as a skin cell - by inducing a "forced" expression of specific genes. Once reprogrammed, the cells take on a variety of capabilities (becoming pluripotent) and offer a range of stem cell treatment possibilities.

Development of a biopacemaker could also benefit the one-in-20,000 infants and premature babies suffering from congenital heart-rhythm dysfunction who currently are not suitable candidates for electronic pacemakers. Infants are physically too small for the device. A biological pacemaker could fit with their small stature and then grow as the infant grows.

Collaborating with Lieu and Chiamvimonvat on the research project will be Jan Nolta, director of the UC Davis Institute for Regenerative Cures; Donald Bers, chair of the UC Davis Department of Pharmacology; and James Chan, assistant professor in the Department of Pathology and affiliated with the NSF Center for Biophotonics Science and Technology at UC Davis.

UC Davis is playing a leading role in regenerative medicine, with nearly 150 scientists working on a variety of stem cell-related research projects at campus locations in both Davis and Sacramento. The UC Davis Institute for Regenerative Cures, a facility supported by the California Institute for Regenerative Medicine (CIRM), opened in 2010 on the Sacramento campus. This $62 million facility is the university's hub for stem cell science. It includes Northern California's largest academic Good Manufacturing Practice laboratory, with state-of-the-art equipment and manufacturing rooms for cellular and gene therapies. UC Davis also has a Translational Human Embryonic Stem Cell Shared Research Facility in Davis and a collaborative partnership with the Institute for Pediatric Regenerative Medicine at Shriners Hospital for Children Northern California. All of the programs and facilities complement the university's Clinical and Translational Science Center, and focus on turning stem cells into cures. For more information, visit http://www.ucdmc.ucdavis.edu/stemcellresearch.

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BioRestorative Therapies Announces the Appointment of Harvard Medical School's Wayne Marasco, M.D., Ph.D., as Chairman …

JUPITER, Fla., Sept. 25, 2012 /PRNewswire/ --BioRestorative Therapies, Inc. ("BRT" or the "Company") (BRTX), a life sciences company focused on stem cell based cellular therapies for various personal medical applications, announced today that Wayne A. Marasco, M.D., Ph.D. has been appointed as Chairman of the Company's Scientific Advisory Board.

Dr. Marasco currently serves as an Associate Professor of Medicine at Harvard Medical School and Dana-Farber Cancer Institute. He is head of the Marasco Labs, an accomplished research laboratory at Dana-Farber Cancer Institute known for discovery and therapeutic human monoclonal antibody development and for constructing and validating various Human-Mouse chimeric models in the area of cancer, infectious disease immunotherapy, regenerative medicine and tissue engineering. Dr. Marasco is an Affiliated Faculty Member of the Harvard Stem Cell Institute and has extensive experience in the field of stem cells.

In 2003, Dr. Marasco founded the National Foundation of Cancer Research Center for Therapeutic Antibody Engineering to expand the use of human monoclonal antibodies in the treatment of cancer. In 2009, he was listed among 13 top scientists in their field as the 21st century medicine "Pioneers of Medicine Progress" by US News & World Report. Dr. Marasco's recent work in the field of influenza has led to a new discovery in which a human antibody attacks a crucial non-mutating part of the influenza virus that can potentially lead to a vaccination of the virus, including the swine flu.

Dr. Marasco commented on his appointment, "I am very excited to be joining the BioRestorative Therapies team and look forward to adding my expertise to the organization and leading the Scientific Advisory Board. The Company has developed substantial scientific and medical programs and I am thrilled to be part of its continued development."

Mark Weinreb, Chief Executive Officer of BRT, stated, "Dr. Marasco is a highly regarded medical researcher and clinician and we are extremely pleased to have him join our Scientific Advisory Board as its Chairman. He will be an integral part in adding new members to the advisory board, and his participation will further the advances we are making with our science. BRT will benefit from Dr. Marasco's extraordinary experience in stem cell science and his unique insight and abilities in the field of cellular medicine."

Dr. Marasco received his PhD in 1980 from the University of Connecticut School of Medicine and postdoctoral training at the University of Michigan Medical School, where he also earned an MD in 1986 and completed training in internal medicine. He received his subspecialty training in infectious diseases at Harvard Medical School, and joined Dana-Farber Cancer Institute in 1989.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc.'s ("BRT") goal is to become a leader in developing medical procedures using cell and tissue protocols, primarily involving adult stem cells (non-embryonic), and allowing patients to undergo minimally invasive cellular-based treatments. BRT is developing the following scientific initiatives: OurbrtxDISCProgram (Disc Implanted Stem Cells) offers a non-surgical treatment for bulging and herniated discs and addresses the gap between non-invasive and invasive back procedures. OurThermoStem Program focuses on treatments for metabolic disorders (diabetes, heart disease, etc.) and obesity and uses brown fat stem cells, which initial research indicates increased caloric burning and reduced glucose and lipid levels in the body. The Company also offers plant stem cell-based facial creams and products under the Stem Pearls brand atwww.stempearls.com.

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

Investor Contacts: KCSA Strategic Communications Philip Carlson / Josh Dver +1 212.896.1233 / +1 212.896.1239 pcarlson@kcsa.com / jdver@kcsa.com

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BioRestorative Therapies Announces the Appointment of Harvard Medical School's Wayne Marasco, M.D., Ph.D., as Chairman ...