Experts urge caution when seeking stem cell treatments

ORLANDO, Fla. Boca Raton, Fla., parents Gary and Judy Susser say they know the hope and promise of stem-cell therapy. Nine years ago they traveled to Mexico for stem cell injections for their son Adam, who has cerebral palsy.

"Maybe it will do some good," Gary Susser said he and his wife thought at the time. They spent $25,000.

But the Sussers stopped stem cell injections in 2005, after spending about $25,000 and seeing no improvement. Today, armed with more information, the Sussers are grateful the treatments didn't harm Adam, now 12. While they are advocates of "responsible" stem cell research, they warn other parents against making trips to Costa Rica, Mexico, Russia or other offshore clinics for experimental treatments.

With promising breakthroughs making the news, as well as Internet hype, desperate parents and seriously ill patients may look to stem cell therapy as the modern miracle that could cure them. And one day, stem cells may be routinely used to repair damaged cells, improve the treatment of diseases and even cure paralysis.

But there are hidden dangers to today's stem cell treatments, both in the U.S. and offshore, scientists said at the recent World Stem Cell Summit in West Palm Beach, Fla. They pointed to reports of deaths, tumors, lumbar punctures and other potential harm, as well as vulnerable people being conned out of thousands of dollars.

Patients are "buying hope," said University of Miami scientist James Guest, working on The

Scientists urge consumers look for regulated clinical trials at universities and research institutions, saying that even those are not without risks.

"Clinics are operating out of loopholes, a gray area disguised as the practice of medicine," said George Q. Daley, director of the stem cell transplantation program at MI/Children's Hospital in Boston.

Industry researcher Douglas Sipp has kept records of more than 400 companies advertising stem cell products or procedures on websites since 2007. When he rechecked this summer, Sipp said, 80 of the sites were no longer online, though they could have simply changed web addresses.

Some of the offshore clinics have been closed by individual countries after patients died, according to Sipp, who leads the research unit for Science Policy and Ethics Studies at the RIKEN Center in Japan.

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Experts urge caution when seeking stem cell treatments

Study shows potential of differentiated iPS cells in cell therapy without immune rejection

A new study from Boston University School of Medicine (BUSM) shows that tissues derived from induced pluripotent stem (iPS) cells in an experimental model were not rejected when transplanted back into genetically identical recipients. The study, published online in Cell Stem Cell, demonstrates the potential of utilizing iPS cells to develop cell types that could offer treatment for a wide range of conditions, including diabetes, liver and lung diseases, without the barrier of immune rejection.

Ashleigh Boyd, DPhil, and Neil Rodrigues, DPhil, the study's senior authors, are assistant professors of dermatology at BUSM and researchers at the Center for Regenerative Medicine (CReM) at Boston University and Boston Medical Center (BMC). They also are lead investigators at the National Institutes of Health's Center of Biomedical Research Excellence (COBRE) at Roger Williams Medical Center, a clinical and research affiliate of BUSM.

iPS cells can be developed from adult cell types, such as skin or blood, by returning them to a stem cell state using genetic manipulation. iPS cells are capable of maturing (differentiating) into all the specific cell types in the body, making them a powerful tool for biological research and a source of tissues for transplantation based therapies. Given that iPS cells can be made in a patient-specific manner, there should be great potential for them to be transplanted back into the same patient without rejection. Yet a study published in Nature in 2011 demonstrated that iPS cells transplanted in the stem cell state were rejected in genetically identical recipients.

"The Nature study provocatively suggested that tissues derived from patient-specific iPS cells may be immunogenic after transplantation. However, it never directly assessed the immunogenicity of the therapeutically relevant cell types that could be utilized in regenerative medicine and transplantation," said Rodrigues.

The BUSM researchers evaluated this matter by taking adult cells from an experimental model and deriving iPS cells from them. They then differentiated the iPS cells into three cell types: neuronal (nerve); hepatocytes (liver); and endothelial (blood vessel lining) cells. These three cell types represent each of the three germ layers present during embryonic development mesoderm, ectoderm and endoderm. Cells from these layers differentiate and ultimately develop into the body's tissue and organ systems. Using experiments to mirror the potential clinical use of patient-specific iPS cells in cell therapy, the team transplanted each of the differentiated cells into a genetically identical experimental model and found no signs of an elevated immune response or indications of rejection.

The study results suggest that using patient-specific iPS cells should overcome issues of immune rejection in transplantation, which will be a significant problem for potential embryonic stem cell-derived therapies. Immune rejection in transplantation is treated clinically by immunosuppressive drugs but they can have serious side-effects, including the risk of developing cancer.

"If the use of immunosuppressive drugs can be avoided, as may be the case for patient-specific iPS cell based therapies, it would be preferable. Our results are very promising and future work should be directed at assessing whether tissues derived from human iPS cells will similarly lack immunogenicity," said Boyd.

Journal reference: Cell Stem Cell

Provided by Boston University Medical Center

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Study shows potential of differentiated iPS cells in cell therapy without immune rejection

Rush researchers studying stem cell therapy to repair damaged knee cartilage

Public release date: 24-Jan-2013 [ | E-mail | Share ]

Contact: Deb Song deb_song@rush.edu 312-942-0588 Rush University Medical Center

(CHICAGO) Rush University Medical Center is conducting the nation's first clinical study of an innovative stem cell drug, Cartistem, to repair knee cartilage damaged by aging, trauma or degenerative diseases such as osteoarthritis.

Cartistem is manufactured from mesenchymal stem cells derived from allogeneic (donor) umbilical cord blood. Umbilical cord blood is a readily accessible source of high-quality stem cells, is associated with minimal health risks and carries relatively few ethical concerns.

The stem cells are mixed with hyaluronan, a natural polymer that plays a major role in wound healing and is a building block of joint cartilage. Cartistem is surgically administered into the area of cartilage damage following an arthroscopic surgery as an adjunct to microfracture, a commonly used technique used to repair cartilage damage.

The principal investigator on the study is Dr. Brian Cole, a professor in the department of orthopedics and anatomy and cell biology at Rush University Medical Center. Dr. Cole is the head of Rush's Cartilage Restoration Center and is also the head team physician for the Chicago Bulls. Cole and his co-researchers will assess the drug's safety as well as its ability to regenerate cartilage repair tissue and reduce pain in patients with localized cartilage loss in the knee.

Treating cartilage damage can be problematic because the tissue does not contain blood vessels or nerves and therefore has a limited ability to re-grow. Various treatments for cartilage degeneration, such as drug therapy, arthroscopy and joint replacement, yield mixed results and are unable to regenerate damaged tissue.

"Finding a biological solution for cartilage regeneration in orthopedics is one of the fastest growing areas of research and development in our specialty, said Cole. "Rush is spearheading this field of research with the ultimate goal of safely improving outcomes and sparing patients from having more complicated surgery at a relatively young age."

The two-year, phase I/IIa study will enroll a total of 12 participants aged 18 years and older, with a body mass index of less than 35. Initially, six individuals with lesions sized 2 to 5 centimeters will be recruited into the study; an additional six volunteers with lesions larger than 5 centimeters will be enrolled sequentially. Each participant will undergo eligibility screening followed by a 12-month observation period to determine the safety and efficacy of the drug with an additional long-term follow-up evaluation at 24 months.

"With a burgeoning aging, yet active population, our patients are looking for effective non-joint replacement solutions to treat their damaged knee cartilage," said Cole. "This research is significant in that it utilizes a commonly performed operation (microfracture) in an effort to improve upon variable outcomes."

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Rush researchers studying stem cell therapy to repair damaged knee cartilage

Stem cell therapy to repair damaged knee cartilage

Jan. 24, 2013 Rush University Medical Center is conducting the nation's first clinical study of an innovative stem cell drug, Cartistem, to repair knee cartilage damaged by aging, trauma or degenerative diseases such as osteoarthritis.

Cartistem is manufactured from mesenchymal stem cells derived from allogeneic (donor) umbilical cord blood. Umbilical cord blood is a readily accessible source of high-quality stem cells, is associated with minimal health risks and carries relatively few ethical concerns.

The stem cells are mixed with hyaluronan, a natural polymer that plays a major role in wound healing and is a building block of joint cartilage. Cartistem is surgically administered into the area of cartilage damage following an arthroscopic surgery as an adjunct to microfracture, a commonly used technique used to repair cartilage damage.

The principal investigator on the study is Dr. Brian Cole, a professor in the department of orthopedics and anatomy and cell biology at Rush University Medical Center. Dr. Cole is the head of Rush's Cartilage Restoration Center and is also the head team physician for the Chicago Bulls. Cole and his co-researchers will assess the drug's safety as well as its ability to regenerate cartilage repair tissue and reduce pain in patients with localized cartilage loss in the knee.

Treating cartilage damage can be problematic because the tissue does not contain blood vessels or nerves and therefore has a limited ability to re-grow. Various treatments for cartilage degeneration, such as drug therapy, arthroscopy and joint replacement, yield mixed results and are unable to regenerate damaged tissue.

"Finding a biological solution for cartilage regeneration in orthopedics is one of the fastest growing areas of research and development in our specialty, said Cole. "Rush is spearheading this field of research with the ultimate goal of safely improving outcomes and sparing patients from having more complicated surgery at a relatively young age."

The two-year, phase I/IIa study will enroll a total of 12 participants aged 18 years and older, with a body mass index of less than 35. Initially, six individuals with lesions sized 2 to 5 centimeters will be recruited into the study; an additional six volunteers with lesions larger than 5 centimeters will be enrolled sequentially. Each participant will undergo eligibility screening followed by a 12-month observation period to determine the safety and efficacy of the drug with an additional long-term follow-up evaluation at 24 months.

"With a burgeoning aging, yet active population, our patients are looking for effective non-joint replacement solutions to treat their damaged knee cartilage," said Cole. "This research is significant in that it utilizes a commonly performed operation (microfracture) in an effort to improve upon variable outcomes."

"Notably, this is a treatment for patients with localized cartilage damage and not for patients who are diagnosed with diffuse or bone on bone arthritis who have otherwise been told they require a knee replacement." said Cole.

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Stem cell therapy to repair damaged knee cartilage

Keynote Message of Secretary Enrique T. Ona: Stem Cell Medicine 1st National Convention

Keynote Message of Secretary Enrique T. Ona

Philippine Society for Stem Cell Medicine

1st National Convention The Truth and Fallacies about Stem Cell Therapy

January 16, 2013, Pandanggo Hall, Manila Hotel

The establishment of the Philippine Society for Stem Cell Medicine composed of physicians with interest in stem cell therapy is opportune, with the increasing demand for the use of stem cells as therapy in oncology, end organ diseases and regenerative medicine, here now in the Philippines and worldwide. I congratulate the founding members, led by Dr. Jose Sabili, your Chairman and Dr Rey Melchor Santos, your President for recognizing the need to organize and professionalize the practice of stem cell therapy in this country.

This two-day national convention, with the theme The Truth and Fallacies about Stem Cell Therapy is very timely as we in the Department of Health and the medical profession try to clear the air of misinformation and half-truths regarding this popular mode of treatment. We owe it to our patients and the general public to ensure that proper information and guidance regarding this novel medical approach is available. To protect themselves and their loved ones, the public must know the most current and accurate information about stem cells and its various applications, including some of which are purely experimental. We must ensure that only safe and ethical uses of stem cells are being used in the Philippines.

Today, we see the proliferation of centers offering stem cell treatments for medical and aesthetic purposes. Some stem cell programs here have expert personnel and clinical facilities and advanced laboratory equipment and technologies, reputed to be more advanced than other institutions abroad. We are concerned, however, that other facilities might not have the minimum capabilities especially trained personnel staff and equipment needed to perform stem cell therapies safely and effectively.

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Keynote Message of Secretary Enrique T. Ona: Stem Cell Medicine 1st National Convention

BrainStorm’s CEO Issues Annual Letter to Shareholders

NEW YORK & PETACH TIKVAH, Israel--(BUSINESS WIRE)--

BrainStorm Cell Therapeutics (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today that its CEO, Dr. Adrian Harel, has issued an Annual Letter to Shareholders summarizing the companys extremely significant developments of 2012:

Dear BrainStorm Shareholders,

2012 has been a pivotal year for BrainStorm, as the company has achieved the most significant milestones since its inception. Weve made dramatic progress in our clinical trials, establishing the safety of NurOwn cells in a first group of ALS patients and demonstrating promising indications of clinical improvement in some patients. As a result weve been fast-tracked to a Phase IIa preliminary efficacy trial after only one year of testing. Weve also made key advances in product development, and secured the largest amount of financing in any single year of operations. I am proud to share with you the major highlights of the past twelve months:

I. Clinical Trials Progress

In early June, we performed the 12th ALS patient transplantation in our Phase I/II clinical trial with NurOwn at Hadassah Medical Center in Jerusalem. In July, we submitted a positive interim safety report to the Israeli Ministry of Health (MOH) on the first 12 ALS patients in the study, demonstrating that treatment was well-tolerated and that there were promising indications of clinical improvement in some of the patients.

Just as we welcomed in 2013, the MOH approved acceleration to a Phase IIa dose-escalating trial to further evaluate the safety and preliminary efficacy of NurOwn treatment. In the Phase IIa trial, which we are launching this month, a second group of 12 patients will receive combined intramuscular and intrathecal administration of NurOwn cells in three cohorts, with increasing doses. The study participants, who have already been recruited, will be monitored for three to six months after transplantation.

In addition, preparations are well underway for a Phase II clinical trial in the USA, scheduled for 2013, pending FDA approval.

II. Advances in Product Development

In December 2012, we signed a strategic agreement with Octane Biotech of Kingston, Ontario, to jointly develop a proprietary bioreactor for production scale-up of NurOwn. The customized bioreactor will enable us to significantly increase our production capabilities by using a single clean room for multiple patients, reducing costs and time. We are very excited to be working with Octane, given their expertise in developing automated production processes for mesenchymal cell therapy technologies. The project is currently being launched.

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BrainStorm’s CEO Issues Annual Letter to Shareholders

Vitro Biopharma Launches New Stem Cell Products to Accelerate Bone Growth

GOLDEN, Colo., Jan. 22, 2013 (GLOBE NEWSWIRE) -- Vitro Diagnostics, Inc. (VODG), dba Vitro Biopharma, announced the launch of a series of products with application to accelerated bone growth, fracture healing and development of new osteoporosis drugs. The new products are osteoblasts derived from human adult stem cells known as mesenchymal stem cells (MSCs). These cells lead to bone growth and function to maintain bone density and in the recovery from bone fractures. Osteoporosis, a disease characterized by reduction in bone density is thought to result from decreased osteoblast production by MSCs; bone growth necessary for repair of bone fractures also depends on osteoblasts. The new products include native and labeled MSC-derived human osteoblasts in various formats including fluorescent and magnetic cells for use in different imaging methods. For example, magnetic osteoblast cells labeled with super paramagnetic iron oxide (SPIO) may be used for in-vivo imaging of osteoblasts using magnetic resonance imaging (MRI) a common clinical imaging procedure. The availability of native and multiply-labeled osteoblasts provides numerous options to conduct in-vitro high throughput screening cell assays and to coordinate these studies with in-vivo studies.

Dr. Jim Musick, Vitro Biopharma's President & CEO, said, "We are pleased to announce commercial availability of our MSC-derived human osteoblasts, the latest in our line of MSC-derived differentiated cells intended for applications in drug discovery and development as well as regenerative medicine. Diseases and injury to the skeletal system pose significant health risk and impairment of function. The incidence of osteoporosis continues to rise with the aging population. Bone fractures are common injuries requiring long healing times and significant loss of production. Improved treatment of osteoporosis and accelerated recovery from bone fractures both provide significant health benefits. We are pleased to offer our new products to assist in the achievement of these objectives. We have additional related products in our pipeline and plan to engage in animal studies in the near future to determine the application of our new products to accelerated healing and regenerative medicine."

About Vitro Biopharma

Vitro Diagnostics, Inc. dba Vitro Biopharma (OTCQB:VODG; http://www.vitrobiopharma.com), owns US patents for production of FSH, immortalization of pituitary cells, and a cell line that produces beta islets for use in treatment of diabetes. In 2011, Vitro Biopharma out-licensed its intellectual property related to treatment of infertility to Dr. James Posillico, a renowned expert in Assisted Reproductive Technologies. Vitro Biopharma also owns a pending US patent for generation of pluripotent stem cells and an additional pending patent for methods of mesenchymal stem cell (MSC) generation and related materials. Vitro Biopharma's mission is "Harnessing the Power of Cells(TM)" for the advancement of regenerative medicine to its full potential. Vitro Biopharma operates within a modern biotechnology manufacturing, R&D and corporate facility in Golden, Colorado. Vitro Biopharma manufactures and sells "Tools for Stem Cell and Drug Development(TM)", including human mesenchymal stem cells and derivatives, the MSC-Gro(TM) Brand of optimized media for MSC self-renewal and lineage-specific differentiation. In addition to our FSH patent licensee, Vitro Biopharma maintains several strategic partnerships including an alliance with Neuromics, Inc. (www.neuromics.com). Neuromics, Inc. is a primary distributor of Vitro Biopharma products and a well established manufacturer and distributor of a large variety of life science research products especially focused on cell-based assay systems We jointly manufacture stem cell assay systems with HemoGenix(R), Inc. (http://www.hemogenix.com/), known as the LUMENESC(TM) quantitative assay for determination of MSC quality, potency and response to toxic agents. Stemgenesis, Inc. (http://www.stemgenesisinc.com ) is a Chinese-based firm with operations in Qingdao, Shandong Province, China and with US operations in Sacramento, CA. Vitro Biopharma has an agreement with Stemgenesis, Inc. for distribution of its stem cell products into select Chinese provinces. Also, Vitro Biopharma's CEO is a consultant on an NSF grant at the City College of New York to advise Dr. Lane Gilcrest, Professor of Materials Science and Engineering, and his colleagues regarding the development of novel extracellular materials for use in self-renewal and differentiation of mesenchymal stem cells.

Safe Harbor Statement

Certain statements contained herein and subsequent statements made by and on behalf of the Company, whether oral or written may contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward looking statements are identified by words such as "intends," "anticipates," "believes," "expects" and "hopes" and include, without limitation, statements regarding the Company's plan of business operations, product research and development activities, potential contractual arrangements, receipt of working capital, anticipated revenues and related expenditures. Factors that could cause actual results to differ materially include, among others, acceptability of the Company's products in the market place, general economic conditions, receipt of additional working capital, the overall state of the biotechnology industry and other factors set forth in the Company's filings with the Securities and Exchange Commission. Most of these factors are outside the control of the Company. Investors are cautioned not to put undue reliance on forward-looking statements. Except as otherwise required by applicable securities statutes or regulations, the Company disclaims any intent or obligation to update publicly these forward looking statements, whether as a result of new information, future events or otherwise.

CONTACT:

Dr. James Musick

Chief Executive Officer

Vitro Biopharma

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Vistagen Therapeutics Successfully Completes Final Phase 1 Safety Study of AV-101

SOUTH SAN FRANCISCO, CA--(Marketwire - Jan 23, 2013) - VistaGen Therapeutics, Inc. ( OTCQB : VSTA ), a biotechnology company applying stem cell technology for drug rescue, predictive toxicology and drug metabolism screening, today announced the successful completion of its final Phase 1 safety study of AV-101, a novel orally available prodrug candidate being developed for treatment of multiple conditions involving chronic neuropathic pain.The study results indicate that AV-101 is safe and well tolerated, with favorable bioavailability and pharmacokinetics.

"This important confirmation of AV-101's safety is the final step in our Phase 1 program for AV-101," said Shawn K. Singh, JD, VistaGen's Chief Executive Officer. "With $8.8 million of funding from the National Institutes of Health (NIH) and outstanding strategic development and regulatory support from Cato Research Ltd., we have successfully completed the required studies enabling Phase 2 clinical development of AV-101 for multiple large market neurological diseases and conditions.In addition, recent data from the NIH suggest that the same neural pathway modified by AV-101 may be useful for treating depression.Launching a broad strategic collaboration to advance development and commercialization of AV-101 is among our key goals in 2013."

About the Final AV-101 Phase 1 Safety Study

VistaGen's final AV-101 Phase 1 safety study was a randomized, double-blind, placebo-controlled, dose-escalation clinical trial conducted at the University of California, San Diego (UCSD).The study involved three cohorts of healthy volunteers, each receiving multiple daily treatments of one of three dose levels of orally administered AV-101 over a 14-day period. The primary objectives of the study were to evaluate the safety, tolerability and pharmacokinetics (PK) of three different daily doses of AV-101 compared to placebo controls.A total of 46 healthy volunteers completed the study.The oral administration of AV-101 was safe and well tolerated by all subjects at all three dose levels tested.In addition, the PK of AV-101 was fully characterized across the range of three dose levels in the study.The data indicate that AV-101 had good bioavailability and a favorable PK profile.

"The primary safety and tolerability endpoints of the Phase 1 program were met.This is a very safe compound with no observed side effects," commented Mark S. Wallace, MD, Chair of the Division of Pain Medicine, Department of Anesthesiology at UCSD and the principal investigator of the study."AV-101 is an exciting prodrug compound that acts through a promising mechanism to treat pain.I am excited to move this compound into Phase 2 studies for the treatment of pain."

About AV-101

Aimed at multi-billion dollar neurological disease and disorders and depression markets, AV-101, also known as "L-4-chlorokynurenine" (4-Cl-KYN), is a novel, orally available prodrug that is converted in the brain into an active metabolite, 7-chlorokynurenic acid (7-Cl-KYNA), which regulates an important neurotransmitter in the brain called the N-methyl-D-aspartate (or NMDA) receptor.A synthetic analogue of kynurenic acid, a naturally occurring neural regulatory compound, 7-Cl-KYNA is one of the most potent and selective blockers of the regulatory GlyB-site of the NMDA receptor.

VistaGen's AV-101 IND application covers clinical development for neuropathic pain. In addition to neuropathic pain, VistaGen expects the results of its Phase 1 clinical program to be useful for supporting the development of AV-101 for other neurological disorders including depression and epilepsy.

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.

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Vistagen Therapeutics Successfully Completes Final Phase 1 Safety Study of AV-101