Cell Medica Announces Treatment of First Pediatric Patient in Early Stage Clinical Trial of Cytovir ADV

LONDON--(BUSINESS WIRE)--

Cell Medica today announced the treatment of the first patient in the ASPIRE Trial, an early stage Phase I/II clinical study investigating the safety and efficacy of CytovirTM ADV for the treatment of adenovirus infections in immunosuppressed pediatric patients following bone marrow transplantation.

The ASPIRE Trial represents a collaborative R&D project among Cell Medica, UCL Institute of Child Health and the Great Ormond Street Hospital for Children. The project is funded in part by a grant from the Technology Strategy Board, the UKs innovation agency.

Cytovir ADV is under development as a new way to treat adenovirus infections in pediatric patients following allogeneic hematopoietic stem cell (bone marrow) transplantation. These patients are profoundly immunosuppressed for a period of three to six months after the procedure and therefore highly vulnerable to serious infections. In certain high risk pediatric groups following bone marrow transplantation, there is a mortality risk of up to 30% for patients developing adenovirus infections. No drug is currently approved for the treatment of adenovirus infections in this patient group.

Cytovir ADV is comprised of naturally occurring T lymphocyte cells (T cells) which demonstrate immune response functions when exposed to adenovirus antigens. The ASPIRE Trial will explore whether adenovirus-specific T cells can be infused in pediatric patients to reconstitute immediate and long-lasting immunity against the virus, thereby potentially avoiding the medical costs, hospitalization and mortality associated with adenovirus infections in this patient group.

The ASPIRE Trial will include up to 15 patients and is expected to complete in early 2015. The Chief Investigator is Dr. Waseem Qasim of the UCL Institute of Child Health.

Extending the pipeline of patient-specific T cell therapies for immune reconstitution

Cytovir ADV is an extension of Cell Medicas T cell products being investigated for infections in patients following bone marrow transplantation. Cell Medicas lead product, Cytovir CMV, is currently being tested in two randomized controlled studies across 15 transplantation centres in the UK.

Gregg Sando, CEO of Cell Medica, commented: The ASPIRE Trial initiates the clinical development of Cytovir ADV in pediatric patients following bone marrow transplantation. Along with Cytovir CMV, we are developing the adenovirus treatment to expand our cell therapy solutions to include two of the most important viral infections in patients following bone marrow transplantation. The clinical research partnership with the UCL Institute of Child Health and the Great Ormond Street Hospital has been very productive in allowing us to identify a high risk group of pediatric patients who could gain particular benefit from an effective and non-toxic antiviral treatment. The support of the Technology Strategy Board was instrumental to provide the platform for this successful collaboration.

Notes to Editors

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Cell Medica Announces Treatment of First Pediatric Patient in Early Stage Clinical Trial of Cytovir ADV

Osiris Therapeutics Announces First Quarter 2013 Financial Results

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics, Inc. (OSIR), the leading stem cell company focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets, announced today its results for the first quarter of 2013.

Highlights and Recent Developments

Our team's execution during the first quarter has given us a very strong start to 2013, said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. While we are pleased with our accomplishments, we are focused solely on the future.

First Quarter Financial Results

Product revenues during the first quarter of 2013 were $4.1 million, compared to $1.1 million during the first quarter of 2012, an increase of 257%. Gross margin during the first quarter was 72% compared to 66% during the first quarter of 2012. Gross profit was $2.9 million during the first quarter of 2013 and $0.75 million during the same period of 2012. We reported a loss of $2.7 million in the first quarter of 2013. As of March 31, 2013, Osiris had $34.9 million in cash, receivables and short-term investments.

Research and development expenses for the first quarter of 2013 were $3.0 million, compared to $4.0 million incurred in the first quarter of 2012. Selling, general and administrative expenses were $2.9 million for the first quarter of 2013, compared to $1.5 million for the same period of the prior year, reflecting our increased commercial activity.

Webcast and Conference Call

A webcast and conference call to discuss the financial results is scheduled for today, May 7, 2013, at 9:00 a.m. ET. To access the webcast, visit the Investor Relations section of the company's website at http://investor.osiris.com/events.cfm. Alternatively, callers may participate in the conference call by dialing (877) 303-6133 (U.S. participants) or (970) 315-0493 (international participants). Note that a presentation will accompany the webcast.

An archive of the webcast will be available approximately two hours after the completion of the call. To access the archived webcast, visit the Investor Relations section of the company's website at http://investor.osiris.com/events.cfm.

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Osiris Therapeutics Announces First Quarter 2013 Financial Results

The Alliance for the Advancement of Adult Stem Cell Therapy and Research Recently Has Proven Stem Cells To Be …

Bonita Springs, Florida (PRWEB) May 07, 2013

The Alliance for the Advancement of Stem Cell Therapy and Research monetarily supports some of the treatments provided by Intercellular Sciences. The Alliance is a non-profit organization dedicated to promoting research and treatment of life altering diseases with adult stem cells.

Howie Linderman, founding board member of The Alliance, says, The Alliance is very proud to be a part of helping people obtain this breakthrough treatment. When I had my stem cell treatment in 2008, the process was much more complex. This new protocol will change the way the world views cardiac problems and we are excited to participate.

To become a supporting member, please visit http://www.thestemcellalliance.org

John C. and Eddie W. have had Dilated Cardiomyopathy for many years and as time has gone on their heart muscle has continued to weaken. The deterioration of their hearts function deteriorated to the point that walking across the room was impossible without shortness of breath. Eddie had become so bad that he was restricted to a wheelchair. Modern science has not been able to stop the progression of this devastating disease. All the medications and defibrillators can do is treat the symptoms. Both men were facing a very grim prognosis. In March they each underwent a treatment using their own stem cells to regenerate the heart muscle. Within several weeks they were both feeling better and able to do much more activity than before. Eddie was out of his wheel chair. John went back to bowling.

John C. (age 62, Dickson, TN), has had Cardiomyopathy since he was a young man. First diagnosed while in college he has had to adjust his life style to adapt to his weak heart function. His doctors recommended a defibrillator a few years ago and he has been dependent on it since. His symptoms became rapidly progressive in the last couple of months. He had to stop many of his activities including cardiac rehab. It had become just too difficult for him. Johns ejection fraction (the percent of pumping of the heart) had dropped to under 10%. His doctors were not optimistic. He and his wife Karen refused to accept such a grim fate and searched for alternative treatment. They soon found an option: adult stem cell therapy. I have much more energy now, said John after his treatment. Im back to walking the perimeter of my 6 acre property and Im back at cardiac rehab. I have even started bowling again which I had stopped. Karen says that the change has been Amazing! An echocardiogram done 3 weeks after his treatment showed an increase in his ejection fraction of 50%. It is expected that his heart function will continue to improve for up to 6 months.

Eddie W. (age 41, Americus, GA) has battled heart disease for the last 14 years. He went through several years with just mild symptoms but, the last year has been devastating. His heart function deteriorated to such a degree that he has been hospitalized twice recently for severe swelling and shortness of breath. The doctors told his wife Leslie to get his affairs in order. While he was in the hospital this last time his family searched for other options. Coming across adult stem cell treatment for heart disease they took a leap of faith and made arrangements for treatment that has to be done outside the United States. Eddie was so weak that he could only get around being pushed in a wheelchair. He also had a pump attached that delivered a medication called Primacor to help his heart. Leslie had to fill the pump once a day with this life sustaining medicine. Eddie was treated in March. The treatment was a success. Within one week the pump was discontinued. Within two weeks he was getting around without the wheelchair. We hosted a church function and Eddie was able to keep up the whole time. Leslie says He could barely get out of his wheelchair before and now to be hosting a party, I cant believe it. Im so grateful. My boys have their father back. Eddies treatment was in part supported by The Alliance for The Advancement of Stem Cell Therapy and Research.

Zannos G. Grekos, MD, MAAC, FACC, chief science officer of Intercellular Sciences and a Florida-based pioneer in the field of adult stem cell therapy explains how, in this groundbreaking treatment, cardiologists insert a catheter into the patients heart similar to a heart cath. In 20 minutes, about 30 separate injections of Regenocytes (activated stem cells) are introduced into the damaged part of the heart. The process of tissue repair begins almost immediately.

The activation provides a key step in the process, Dr. Zannos Grekos explains. The lab extracts the stem cells, concentrates and activates them into over a billion of regenerative adult stem cells while educating them to assist the specific organ that needs regeneration. These cells we call Regenocytes.

Patients remain in the hospital overnight for observation, and are typically discharged the next day. Patients are scheduled for regular subsequent visits after the stem cell treatment to monitor their progress and measure their results.

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NYSCF scientists create personalized bone substitutes from skin cells

Public release date: 6-May-2013 [ | E-mail | Share ]

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (May 6, 2013) A team of New York Stem Cell Foundation (NYSCF) Research Institute scientists report today the generation of patient-specific bone substitutes from skin cells for repair of large bone defects. The study, led by Darja Marolt, PhD, a NYSCF-Helmsley Investigator and Giuseppe Maria de Peppo, PhD, a NYSCF Research Fellow, and published in the Proceedings of the National Academy of Sciences of the USA, represents a major advance in personalized reconstructive treatments for patients with bone defects resulting from disease or trauma.

This advance will facilitate the development of customizable, three-dimensional bone grafts on-demand, matched to fit the exact needs and immune profile of a patient. Taking skin cells, the NYSCF scientists utilized an advanced technique called "reprogramming" to revert adult cells into an embryonic-like state. These induced pluripotent stem (iPS) cells carry the same genetic information as the patient and they can become any of the body's cell types.

The NYSCF team guided these iPS cells to become bone-forming progenitors and seeded the cells onto a scaffold for three-dimensional bone formation. They then placed the constructs into a device called a bioreactor, which provides nutrients, removes waste, and stimulates maturation, mimicking a natural developmental environment.

"Bone is more than a hard mineral composite, it is an active organ that constantly remodels. Blood vessels shuttle important nutrients to healthy cells and remove waste; nerves provide connection to the brain; and, bone marrow cells form new blood and immune cells," said Marolt.

Previous studies have demonstrated the bone-forming potential from other cell sources, yet serious caveats for clinical translation remain. A patient's own bone marrow stem cells can form bone and cartilaginous tissue, not the underlying vasculature and nerve compartments; and, embryonic stem cell derived bone may prompt an immune rejection. The NYSCF scientists chose to work with iPS cells to overcome these limitations, comparing iPS sources with embryonic stem cells and bone marrow derived cells.

"No other research group has published work on creating fully-viable, functional, three-dimensional bone substitutes from human iPS cells. These results bring us closer to achieving our ultimate goal, to develop the most promising treatments for patients," said de Peppo.

While severity varies, bone defects and injuries are currently treated with bone grafts, taken either from another part of the patient's body or a donor bone bank, or with synthetic substitutes. None of these permit complex reconstruction, and they may elicit immune rejection or fail to integrate with surrounding connective tissues. For trauma patients, suffering from shrapnel wounds or vehicular injury, these traditional treatments provide limited functional and cosmetic improvement.

After a comprehensive in vitro analysis of the generated bone, the NYSCF team assessed stability when transplanted in an animal model to address a major concern for iPS-based cell therapies. Undifferentiated iPS cells can form teratomas, a type of tumor. The iPS cell-derived bone substitutes were implanted under the skin of immunocompromised mice. After 12 weeks, the explanted constructs matured and showed no malignancies but complete maturation of bone tissue, while blood vessel cells began to integrate along the grafts. These results indicate the stability of the bone substitutes.

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NYSCF scientists create personalized bone substitutes from skin cells

VistaGen Therapeutics and Duke University Publish Results on Production of Functional 3D Human Heart Tissue

SOUTH SAN FRANCISCO, CA--(Marketwired - May 7, 2013) - VistaGen Therapeutics, Inc. (OTCQB: VSTA), a biotechnology company applying stem cell technology for drug rescue, predictive toxicology and drug metabolism assays, announced that its high-quality, human pluripotent stem cell-derived cardiomyocytes (heart cells) were used by collaboration partner Duke University to grow a revolutionary three-dimensional (3D) human heart muscle. An abstract of the original research article published in Biomaterials, an international journal covering the science and clinical application of biomaterials, can be found online at: http://www.sciencedirect.com/science/article/pii/S0142961213004705.

Researchers at Duke University combined VistaGen's human stem cell-derived heart cells with innovative tissue engineering and cardiac electrophysiology technologies to grow what is being called a "heart patch," which mimics the natural functions of native human heart tissue. This heart patch technology is being developed to aid in a better understanding of the biology critical to cardiac tissue engineering, for applications in regenerative cell therapy for heart disease, and as predictive in vitro assays for drug rescue and development.

H. Ralph Snodgrass, PhD, VistaGen's President and Chief Scientific Officer, stated, "The developed contractile forces and other functional properties of these cardiac tissues are remarkable and are significantly higher than any previous reports. The achievement of successfully growing a human heart muscle from cardiomyocytes derived from human pluripotent stem cells not only expands the scope of our drug rescue capabilities, but also reflects the advanced nature and potential of our collaboration with the skilled biomedical engineers at Duke Medical Center."

"VistaGen's human cardiomyocytes produced engineered cardiac tissues that exhibited structural and functional properties superior to those previously reported," said Dr. Nenad Bursac, Associate Professor in the Departments of Cardiology and Biomedical Engineering at Duke University. "This is the closest man-made approximation of natural human heart muscle to date."

Achieving this capability represents a significant breakthrough in heart cell-based therapies and in testing new medicines for potential heart toxicity and potential therapeutic benefits impacting heart disease. The following are among several key development points from the study:

The original research article also will be published in print in Biomaterials.

About VistaGen Therapeutics

VistaGen is a biotechnology company applying human pluripotent stem cell technology for drug rescue, predictive toxicology and drug metabolism screening. VistaGen's drug rescue activities combine its human pluripotent stem cell technology platform, Human Clinical Trials in a Test Tube, with modern medicinal chemistry to generate novel, safer chemical variants (Drug Rescue Variants) of once-promising small molecule drug candidates. These are drug candidates discontinued by pharmaceutical companies, the U.S. National Institutes of Health (NIH) or university laboratories, after substantial investment in discovery and development, due to heart or liver toxicity or metabolism issues. VistaGen uses its pluripotent stem cell technology to generate early indications, or predictions, of how humans will ultimately respond to new drug candidates before they are ever tested in humans, bringing human biology to the front end of the drug development process.

VistaGen's small molecule prodrug candidate, AV-101, has completed Phase 1 development for treatment of neuropathic pain. Neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, affects millions of people worldwide.

Visit VistaGen at http://www.VistaGen.com, follow VistaGen at http://www.twitter.com/VistaGen or view VistaGen's Facebook page at http://www.facebook.com/VistaGen.

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VistaGen Therapeutics and Duke University Publish Results on Production of Functional 3D Human Heart Tissue

Dr. Ernesto Gutierrez is Named President of World Stem Cells Clinic, a Stem Cell Research and Stem Cell Treatment …

Tampa, FL (PRWEB) May 04, 2013

World Stem Cells Clinic in Cancun is pleased to announce the appointment of Dr. Ernesto Gutierrez to the position of President of their medical lab and treatment center. Dr. Gutierrez brings experience and intensity to his new position at World Stem Cells Clinic and promises he and his team of experts will provide the best medical care available in the world to improve their patients quality of life.

Dr. Ernesto Gutierrez is an extensively educated physician. He is a graduate from the Universidad Anhuac Norte, School of Medicine in Mexico City and additionally holds a Post-Graduate Degree in Aesthetic and Anti-Aging Medicine from the Instituto Mexicano de Medicina Antienvejecimiento y Esttica, Guadalajara, Mexico. He holds active membership of both the Mexican College of Aesthetic and Anti-Aging Medicine and the Age Management Medicine Group. Dr. Gutierrez has spent considerable time in additional training in the US, with rotations in both Florida and Nevada for two specialty organizations and is certified in Age Management Medicine by the Cenegenics Education and Research Foundation part of Cenegenics Medical Institute in Las Vegas, Nevada. His bilingual English and Spanish skills, both in medical and conversational applications, are excellent. Dr. Gutierrez is the perfect addition to the team given his thorough understanding of both the North and South American medical systems.

Dr. Ernesto Gutierrez explains how the clinic and laboratory at World Stem Cells Clinic in Cancun were designed, built and are operated under the stringent guidelines as established by The International Cellular Medical Society (ICMS) and the US Food and Drug Administrations Good Tissue Practice (cGTP) regulations for pharmaceutical, biologics and clinical laboratories. The strict adherence to these established guidelines and policies guarantees the highest quality of clinical care and stem cell treatment safety for the patient. At World Stem Cells Clinic they continually strive to improve the patients outcomes with breakthrough research and by creating new cells and protocols.

Both the US patient management team at World Stem Cells, LLC (http://worldstemcells.com/) and their Cancun patient management team at World Stem Cells Clinic (http://worldstemcellsclinic.com/) offer medical information on the stem cell treatments provided by the doctors, help patients arrange transportation to Cancun and within the city, hotels, medical records procurement, and basically ensure that their every need is met.

Dr. Gutierrez says, "we will treat you like family!"

Working under the guidelines set forth by ICMS, World Stem Cells Clinic (http://worldstemcellsclinic.com/) provides Stem Cell Treatment for Ankylosing Spondylitis, Autism, Cerebral Palsy, Charcot-Marie-Tooth Disease (CMT), Crohns Diseases, COPD, Fuchs disease, Guillain-Barre Syndrome, Hashimotos Thryroiditis, ITP, Kidney Diseases, Macular Degeneration, Lupus (SLE), Multiple Sclerosis, PAD, Parkinsons disease, Rheumatoid Arthritis, Scleroderma, Stroke, Ulcerative Colitis at its contract Clinics, GLP laboratory, doctors and hospitals in the beautiful resort area of Cancun. They endeavor to provide the best care possible at a competitive price while providing documentation of all treatments that can be used to provide better future care and scientific data to the medical industry. World Stem Cells Clinic is participating in ICMS (International Cellular Medical Society) Stem Cell Reimplantation Registry. They provide Stem Cell Treatments to patients in need.

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Dr. Ernesto Gutierrez is Named President of World Stem Cells Clinic, a Stem Cell Research and Stem Cell Treatment ...

RNL BIO Co. Ltd. Files IND To Commence Phase II Clinical Trial Of RNL-JointStem For Osteoarthritis In The United States

GERMANTOWN, Md., May 2, 2013 /PRNewswire/ -- RNL BIO, a stem cell biotechnology company dedicated to the commercialization of autologous cell therapy products for a variety of degenerative, ischemic and other indications, has announced that it has filed an Investigational New Drug application (IND) with the Food and Drug Administration (FDA) to begin clinical trials with its adipose-derived stem cell product, termed RNL-JointStem, for the treatment of osteoarthritis (OA). Assuming approval of the IND by the Center for Biologics Evaluation and Research (CBER) at the FDA, RNL BIO plans to initiate its double-blinded, randomized, positive-control Phase II clinical trial during the third quarter of 2013 in Sugarland, Texas. Dr. Jason Dragoo of Stanford University and Dr. David Alan Fisher of Indiana University acted as reviewers and completed revision of the protocol now under evaluation by the FDA. Phase I and Phase II clinical trials of RNL-JointStem have already been completed under the authority of the Korean Food and Drug Administration (KFDA).

"We are excited about filing an IND for RNL-JointStem because it brings us closer to a clinical trial with RNL-JointStem in the United States," said Jeong-Chan Ra, CEO and Chairman of RNL BIO. "Our goal for this trial is to achieve global demonstration that RNL-JointStem is efficacious. If our trial is successful it can lead to a paradigm shift in the treatment of osteoarthritis, using stem cell products derived from a patient's own fat tissue." Among the key advantages demonstrated in previous trials of RNL-JointStem is that it is administered in a single injection, unlike the many invasive treatments currently used in the treatment of OA. "Treatments of this painful chronic condition should relieve pain, not add to it," said Dr. Ra. "We hope to confirm the efficacy of RNL-JointStem for cartilage regeneration, pain reduction and joint function improvement for OA patients, and that it will see market approval in due course."

The clinical trial will compare RNL-JointStem to existing modalities utilizing hyaluronic acid (HA). The patient population for this initial clinical trial will include patients diagnosed with osteoarthritis and whose clinicians judge that they meet candidacy requirements. The intended market for RNL-JointStem is patients under 60 years of age for whom there is no efficacious therapy at present.

About Osteoarthritis

Degenerative arthritis is the most common type of arthritis. It is estimated that 26.9 million Americans 25 years old or older have clinical degenerative arthritis of some joints, with a higher percentage of affliction in the older population. Its clinical manifestations include joint pain and impairment to movement, and surrounding tissues are often affected with local inflammation. The etiology of degenerative arthritis is not completely understood; however, injury, age, and genetics have been considered among the risk factors.

Degenerative arthritis is a progressively debilitating disease that affects mostly cartilage, with associated changes in bone. Cartilage has limited intrinsic healing and regenerative capacities.

Through this trial

Due to the increasing incidence of degenerative arthritis and the aging population coupled with inefficient therapeutic choices, novel cartilage repair strategies are in need. The market for a therapeutically efficacious product for this indication is estimated to be very large.

About an IND

An Investigational New Drug (IND) is a procedure for the authorization to perform a clinical trial in the United States. An IND is required whenever the performance of a clinical trial in the United States is intended. The IND includes information related to the quality, manufacture and control of the Investigational Medicinal Product, data from pre-clinical studies and clinical intent-to-use. An overall risk-benefit assessment, critical analyses of the pre-clinical data in relation to the potential risks and benefits of the proposed study are required to be part of the IND.

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RNL BIO Co. Ltd. Files IND To Commence Phase II Clinical Trial Of RNL-JointStem For Osteoarthritis In The United States

Stem cell discovery could aid research into new treatments

May 2, 2013 Scientists have made a fundamental discovery about how the properties of embryonic stem cells are controlled.

The study, which focuses on the process by which these cells renew and increase in number, could help research to find new treatments.

Researchers have found that a protein, which switches on genes to allow embryonic stem cells to self-renew, works better when the natural occurring level of the protein is reduced.

It was previously thought that once levels of this protein -- called Oct 4 -- were reduced the numbers of new stem cells being produced would also fall.

The finding will inform stem cell research, which is looking to find treatments for conditions including Parkinson's, motor neuron, liver and heart disease.

During embryonic development, cells that have the capacity to become any cell type in the body -- called pluripotent stem cells -- can either renew themselves by multiplying in number or differentiate to become cells found in different parts of the body, for instance skin or liver.

This need for pluripotent cells to increase in number is important so that there is a sufficient supply of them to be differentiated into other cell types.

Scientists at the Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh found that when there were lower levels of Oct 4, the protein bound much more tightly to key parts of DNA in cells.

The strong attraction of Oct 4 to these sections of DNA enabled the efficient switching on of key genes that caused pluripotent stem cells to renew.

The findings could help to improve the way in which stem cells are cultured in the laboratory, providing a better understanding of the processes needed for cells to divide and multiply or to generate different cell types.

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Stem cell discovery could aid research into new treatments

Researchers outline concerns about unproven stem cell therapies

May 3, 2013 An international group of leading stem cell researchers has issued a statement that specifies concerns about the development and use of unproven stem cell therapies. The commentary is published online today in The EMBO Journal ahead of a debate in the Italian parliament on whether to change a recent law that allows certain untested stem cell therapies to be used by the public health system. The authors of the commentary argue that rigorous clinical testing and regulation of stem cell therapies are essential to introduce safe and effective medical interventions for patients.

"Stem cells may offer unprecedented opportunities to develop treatments for many diseases with unmet medical needs. This will take time. However, only rigorous science and responsible regulation can ensure the safe and effective translation of science into effective therapies," remarked Paolo Bianco, Pathologist, Stem Cell Biologist, Professor of Pathology at the University of Roma "La Sapienza" and one of the 13 authors of the commentary who come from Italy, Germany, the United Kingdom, The Netherlands and the United States.

The concerns of the scientific community have been heightened by pending legislative action that may allow routine administration of unproven stem cell therapies to patients in Italy. Despite a lack of rigorous clinical trials to test safety or efficacy, a ban of the treatment by health authorities, and a lack of peer review by the scientific community, the privately funded Stamina Foundation has been using cultured mesenchymal stem cells that have been exposed to putative conditions that favour neuronal differentiation to treat different diseases in severely or terminally ill patients.

The Italian Chamber decides shortly whether they will proceed with controversial legislation passed in the Senate on 21 March that allows the unproven stem cell treatment developed by the Stamina Foundation to be used for severely or terminally ill new patients for 18 months. "The adoption of this law may set a dangerous precedent for patients looking to be treated with other unproven stem cell therapies in Europe and other countries," remarked Hans Clevers, Professor of Molecular Genetics and President of the Royal Netherlands Academy of Arts and Sciences.

"Irrational and unverified stem cell treatments based on methods that are not validated or scientifically documented should not reach patients. Preventing this from happening is a specific responsibility of health authorities and governments worldwide to make sure that the hope and trust of patients are not misused," remarked Elena Cattaneo, Director of the Centre for Stem Cell Research at the University of Milan, Italy, and one of the scientists who contributed to the commentary. "Patients can be harmed and killed by medicines that have not been proven to be safe and effective via rigorously controlled clinical trials. The use of medicines that have not been manufactured to the highest possible standards is irresponsible."

Preclinical and clinical tests have been used successfully in the past for the introduction of therapies for bone marrow transplantation and the regeneration of skin and cornea in patients. The authors of the commentary emphasize that cell therapies must be approved by international and national regulatory agencies and remain under the strict vigilance of health authorities. Regulations already in place in the European Union insist that stem cell therapies follow the same safety and efficacy rules as pharmaceuticals. They need to be prepared and manufactured in highly controlled environments with precise protocols, traceability and accountability.

"It is disconcerting that the Italian Senate has passed amendments that permit the use of unproven stem cell therapies without proper vigilance or proper experiment, reclassifying them as transplants," commented Bianco. "Infusions of mesenchymal stem cells are not transplants in any way. In Europe and the United States, all kinds of cell preparations that are administered to patients following ex vivo culturing are classified as medicines, and monitored by drug agencies such as the US Food and Drug Administration, the European Medicines Agency and, in Italy, the Agenzia Italiana del Farmaco or AIFA. A host of proper and improper commercial interests might benefit from these new rules that abrogate both safety and proper ways of experimentation. Patients may be harmed," concluded Bianco.

Sean Morrison, Professor and Director of the Children's Medical Center Research Institute at UT Southwestern Medical Center in the United States, who is not an author on the paper, added: "Patients are ultimately not helped by therapies that are not based on sound science and that are not tested in systematic clinical trials. Efforts to water down regulation in this area may create opportunities for some individuals to prey on the hopes of desperate patients."

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Girl gets stem cell windpipe in historic operation

A two-year-old girl born without a windpipe now has a new one grown from her own stem cells, making her the youngest patient in the world to benefit from the experimental treatment.

Hannah Warren has been unable to breathe, eat, drink or swallow on her own since she was born in South Korea in 2010. Until the operation at a U.S. hospital, she had spent her entire life in a hospital in Seoul. Doctors there told her parents there was no hope and they expected her to die.

The stem cells came from Hannah's bone marrow, extracted with a special needle inserted into her hip bone. They were seeded in a lab onto a plastic scaffold, where it took less than a week for them to multiply and create a new windpipe.

The windpipe was implanted April 9 in a nine-hour procedure.

Early signs indicate the windpipe is working, Hannah's doctors announced Tuesday, although she is still on a ventilator. They believe she will eventually be able to live at home and lead a normal life.

"We feel like she's reborn," said Hannah's father, Darryl Warren.

"They hope that she can do everything that a normal child can do but it's going to take time. This is a brand new road that all of us are on," he said in a telephone interview. "This is her only chance but she's got a fantastic one and an unbelievable one."

Warren choked up and his wife, Lee Young-mi, was teary-eyed at a hospital news conference Tuesday. Hannah did not attend because she is still recovering from the surgery. She developed an infection after the operation but now is acting like a healthy 2-year-old, her doctors said.

Warren said he hopes the family can bring Hannah home for the first time in a month or so. Hannah turns 3 in August.

"It's going to be amazing for us to finally be together as a family of four," he said. The couple has an older daughter.

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Girl gets stem cell windpipe in historic operation