AdultStemCell.com

In a boost for the field of regenerative medicine, a small biotechnology company has received regulatory approval in Canada for what it says is the first manufactured drug based on stem cells.

The company, Osiris Therapeutics of Columbia, Md., said Thursday that Canadian regulators had approved its drug Prochymal, to treat children suffering from graft-versus-host disease, a potentially deadly complication of bone marrow transplantation.

“It’s really a good day for the concept and the hope behind stem cell therapies becoming a reality,” C. Randal Mills, the chief executive of Osiris, said in an interview.

Prochymal is a preparation of mesenchymal stem cells, which are obtained from the bone marrow of healthy young adult donors. The stem cells are separated out from the marrow and expanded in culture, so that one donation is enough to make as many as 10,000 doses.

Because these are adult stem cells, they do not raise the ethical concerns of embryonic stem cells, whose creation usually involves the destruction of human embryos.

Graft-versus-host disease occurs when the immune cells in a bone-marrow transplant see the recipient’s organs as foreign and attack them, causing potentially severe damage to the skin, liver and digestive tract. This happens most often when the donor is not an exact match for the recipient.

Doctors try using steroids or other drugs to damp the immune attack, but in many cases those don’t work, and the patient may die.

Prochymal is approved in Canada for children whose condition is not controlled by steroids. In a small trial, about 60 percent of such children had a clinically meaningful response to the drug, Osiris said.

“Any drug or a cell that has activity in the patients with severe disease is exciting and important,” said Dr. Joanne Kurtzberg, director of the pediatric blood and marrow transplant program at Duke University Medical Center.

Dr. Kurtzberg, who helped Osiris present its case to Canadian regulators, said the drug has saved some children’s lives from graft-versus-host disease and could lead to more successful bone marrow transplants.

Osiris is not expected to gain much revenue from patients with a rare disease in Canada. But it is a welcome success for a 20-year-old company that has had its share of failures.

In 2009, Prochymal failed in two late-stage clinical trials, showing little to no advantage over placebo in treating graft-versus-host disease. The company is also trying to develop Prochymal as a treatment for Crohn’s disease, diabetes, heart attacks and other illnesses, but has had some failures there as well.

Sanofi, the big French company that had the rights to sell Prochymal outside North America, said in February that it had discontinued its work on the drug.

Dr. Mills, Osiris’s chief executive, said the company realized the drug was most effective in the most severe cases of graft-versus-host-disease that did not respond to steroids, leading it to do the small trial in children.

Dr. Mills said that the Food and Drug Administration indicated that it would require more data before approval, prompting Osiris to seek approval in Canada first. He said the company would apply to the F.D.A. later this year.

Stem cells are already used in medicine. Bone marrow or stem cell transplants are used to treat various cancers and genetic diseases. But those transplants are medical procedures, not products sold by a drug company.

There are cell therapies that have been approved by regulators, such as Carticel, a Genzyme product that uses a patient’s own cells to repair cartilage in injuries. Last year the F.D.A. approved a cord blood product for use in transplantation. Those products are not manufactured for off-the-shelf use like Prochymal is, Dr. Mills said.

Regenerative Sciences, Inc., a company dedicated to advancing orthopedic care through non-surgical adult stem cell procedures, today announced that it has secured a $2M investment from philanthropist, visionary and businessman John C. Malone, PhD, chairman of Liberty Media Corporation. In addition to advancing Regenerative Sciences’ clinical and lab-based stem cell research, the investment will help support the national expansion of their Regenexx™ Physician Network.

Regenerative Sciences’ Regenexx procedures utilize a patient’s own stem cells to help repair a broad range of common injuries and degenerative conditions, including cartilage lesions, torn ligaments and tendons, osteoarthritis and bulging spinal discs. For many, the procedures offer a viable alternative to arthroscopic surgery, open-joint surgery, or joint replacement surgery. Regenexx patients experience little or no downtime from the procedures and avoid the lengthy rehabilitation period associated with most surgical procedures.

“We are proud of our accomplishments in the field of regenerative interventional orthopedics and it’s exciting that our work has drawn the attention of such a noted entrepreneur and philanthropist,” said Christopher J. Centeno, M.D., Chief Executive Officer of Regenerative Sciences. “Dr. Malone shares our vision for forging the next generation of minimally invasive regenerative treatments. This investment will not only bolster our existing stem cell research programs and make our procedures available in all regions of the U.S., but it will help us maintain a leadership role in clarifying the regulatory space for physician stem cell use.”

Regenerative Sciences is at the forefront of regenerative orthopedic medicine within the United States and the company is bringing the future of orthopedic treatments to patient care today.

About Regenerative Sciences

Regenerative Sciences is an outgrowth of the Centeno-Schultz clinic, where we are reinventing orthopedic care for the 21st century using key biologics such as stem cells, next generation tools and devices, and unique therapeutic approaches. Our signature initiative, Interventional Orthopedics, allows doctors to treat orthopedic conditions through injection, rather than traditional invasive surgery. The Regenexx Physician Network brings together like-minded physicians from around the country to offer more patients access to our innovative procedures. For more information on Regenerative Sciences and Regenexx procedures, visit: www.regenexx.com

About John C. Malone, PhD

Dr. John C. Malone holds a bachelor’s degree in electrical engineering and economics from Yale University, where he was a Phi Beta Kappa and merit scholar. He also holds a master’s degree in industrial management and a Ph.D. in operations research from Johns Hopkins University.

Dr. Malone is Chairman of Liberty Media Corporation, a position he has held since 1990. Dr. Malone is also the Chairman of the Board of Liberty Global, Inc. (LGI), a position he has held since June, 2005. From 1996 to March 1999 when Tele-Communications, Inc. (TCI) merged with AT&T Corp., he was also Chairman and Chief Executive Officer of TCI. Previous to that, from 1973 to 1996, Dr. Malone served as President and CEO of TCI. He currently serves on the Board of Directors for CATO Institute, Expedia, Inc., Discovery Communications, Inc., and SiriusXM.

Neuralstem, Inc. (NYSE Amex: CUR) updated the progress of its ongoing Phase I human clinical trial of the company’s spinal cord stem cells in the treatment of ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig’s disease) at Emory University in Atlanta, Georgia. The company announced that, after reviewing the safety data from the first six non-ambulatory patients, the trial’s Safety Monitoring Board has unanimously approved moving to the next group of ALS patients, all of whom will be ambulatory.

“We are pleased with the progress of the trial to date, and look forward to moving directly into more recently-diagnosed patients,” commented Dr. Eva Feldman, PhD, MD,  Principal Investigator of the trial and a consultant to Neuralstem. Dr. Feldman is Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System.

“We are encouraged by the Board’s approval to advance the trial to patients who have an earlier stage of the disease,” said Neuralstem’s CEO and president, Richard Garr. “While the primary endpoint of the trial is safety, we also hope to see some secondary endpoints showing efficacy. We are grateful to the patients, and their families, for participating in this trial.”

The first six patients treated in the trial were non-ambulatory. Of these, the first three received five injections each, unilaterally, in the lumbar region of the spinal cord. The next three received ten injections each, bilaterally in the lumbar region. All of the remaining patients in the trial will be ambulatory, and therefore represent earlier stages of disease progression. Of the ambulatory group, the first three patients will receive five injections each, unilaterally, in the lumbar region. The next three will receive ten injections each, bilaterally, in the lumbar region. After the required FDA approval, the final six patients in the trial will receive injections in the cervical region, representing both a progression of treatment up the spinal cord, as well as into more recently-diagnosed patients.

About The Trial

The Phase I trial to evaluate the safety of Neuralstem’s spinal cord stem cells in the treatment of ALS, the first FDA-approved ALS stem cell trial, has been underway since January, 2010. The trial will ultimately consist of up to 18 ALS patients, who will be examined at regular intervals post-surgery, with final review of the data to come six months after the last patient is treated. While the trial is primarily evaluating the safety of the cells and procedure, it will also seek some secondary efficacy endpoints including  attenuation of motor function loss, maintenance of respiratory capacity, and stabilization of patients along the ALS functional rating scale.

Cord Blood America, Inc. (http://www.cordblood-america.com) (OTC Bulletin Board: CBAI), the umbilical cord blood stem cell preservation company focused on bringing the life saving potential of stem cells, a biological insurance policy, to families nationwide and internationally, today said that clinical trials ongoing in the U.S. using umbilical cord blood in the treatment of cerebral palsy, high-risk hematologic cancer, injured spinal cords and Type 1 diabetes provide significant further evidence of the importance of storing umbilical cord blood at the time of birth.

“We are asked with some frequency why store these stem cells available only at birth.  On our web site at www.corcell.com is a list of more than 70 diseases that are already treated by these stem cells, including acute and chronic leukemia’s, severe aplastic anemia, Hodgkin’s Disease, a number of inherited metabolic diseases and immune system disorders and malignancies including Ewing Sarcoma and Sickle Cell Disease,” said Matthew Schissler, co-founder and CEO.  ”Once in a while, it is my duty and obligation to step away from the day-to-day and reflect on the larger scope of work in the umbilical cord blood stem cell arena.  In doing so, I recognize that our loyal shareholders may not be privy to all of the data we receive, involving the outstanding studies with these cells.  It is my hope that a simple statement such as this, can direct our shareholders to the resources publicly available.”

“What is truly exciting is that the National Institutes of Health at www.clinicaltrials.gov now lists clinical trials ongoing at such prestigious research institutions as Duke University and Memorial Sloan-Kettering Cancer Center to understand the possible usage of stem cell infusions for the treatment of intractable diseases, including Type I diabetes in children, cerebral palsy in children, chronic spinal cord injuries, lymphoma and other blood borne cancers,” Mr. Schissler said.

“Also, there is research ongoing elsewhere in the world to use umbilical cord blood stem cells to combat Alzheimer’s, cardiac disease, lupus, multiple sclerosis, muscular dystrophy, Parkinson’s Disease, rheumatoid arthritis and stroke.  This truly is an excellent time to be involved in this sector as we work toward our goal of becoming the most significant stem cell company in the world,” said Mr. Schissler.

Cleveland, Ohio leveraged buyout firm Riverside Co. has acquired private cord blood bank Celvitae Biomedica S.A. in Madrid, Spain for undisclosed terms.

Celvitae is one of two cord blood banks licensed in Spain and authorized to work in the public health system, Riverside said in a release. It also has major commercial agreements with some of the leading insurance companies in the Spanish market.

Last year, Celvitae struck a strategic agreement with MD Anderson Internacional Espana, a cancer treatment center in Europe that ensures all cord blood stem cell samples collected in Spain are processed within 24 hours at its Madrid facilities.

). On * average, the noscript tag is called from less than 1% of internet * users. */–>Riverside will add Celvitae to its Crioestaminal platform, the Cantanhede, Portugal company that is a pioneer and market leader in the isolation and storage of cord blood stem cells. More than 40,000 parents have trusted the company to store their children’s stem cells.

Riverside acquired Crioestaminal last year. The Celvitae acquisition was made from Riverside Europe Fund IV. Riverside is co-headquartered in New York City.

New stem cell studies at the University of Maryland Dental School demonstrate that surgeons could one day routinely use strong, mold-able, and inject-able pastes to regenerate needed bone tissue to repair broken bones, fractures, genetic defects, even combat bone wounds.

The Dental School’s Huakun Xu, PhD, MS; Michael Weir, PhD, MS; and Ryan Zhao, MD, PhD, presented their findings today at the World Stem Cell Summit at the Baltimore Convention Center before hundreds of stem cell experts from 25 countries.

The Dental School presentation showed that human stem cells seeded in a tissue engineering scaffolding exhibited “excellent attachment and osteogenic differentiation,” which is the process of laying down new bone material.

The researchers said the new findings buoy hopes that an inject-able paste of stem cells will be available one day to fill any shape of cavity from bone defects, breaks or wounds by regenerating needed bone tissue.

In test tube studies, stem cells from bone marrow, when placed into an inject-able scaffold of calcium phosphate and chitosan, started growing and forming minerals needed for new bone tissue.

Xu, an associate professor, is the principal investigator of a $230,000 grant from the Maryland Stem Cell Research Fund for “Stem Cell Delivery via Inject-able, Nano-apatite Scaffolds for Bone Engineering,” and a $1.84 million grant from the National Institute of Dental and Craniofacial Research.

The Dental School researchers have so far tested four scaffolding materials for gripping and holding the stem cells. “Which of the materials will be used in a commercial product really depends on where you want to place the material, whether in the jaw bone, the cranium or other bones,” said Weir, a research assistant professor.

Weir said, “Ultimately we want this to be an inject-able paste so we can put it into voids that are not square, rectangular or circular, that they are irregular shapes that need to be filled. The paste will include the cells.”

Xu added that such a product could also be used in periodontal bone repair, mandibular and maxillary ridge augmentation, reconstruction of frontal sinus and craniofacial skeletal defects, and other stress-bearing orthopedic applications. After a tumor removal or traffic accident, there may be a need to repair the damage or void left. It will beneficial, he said, to have a paste that can be shaped easily to achieve a high degree of aesthetics. After shaping, the paste hardens to form a solid scaffold full of pores and channels and still containing stem cells throughout, still living and growing to form new bone. Eventually the scaffold material degrades and is replaced entirely by new bone tissue grown from the stem cells.

The researchers found that a significant number of the cells were alive after a few weeks in the scaffolding material. They then discovered that the cells were differentiating into osteoblasts, essentially turning into bone cells. (From Greek words for bone, an osteoblast cell is responsible for bone formation.)

After staining the scaffold, the researchers found the osteoblasts forming “a lot” of the mineral, which then forms the bone after only 21 days, said Weir. In a subsequent experiment, the cells survived even better when mixed in a gel of the scaffolding material.

The researchers have recorded similar success with umbilical cord-derived stem cells, which “appear to be more potent in terms of growth and transforming into osteoblasts on the scaffold than the cells from bone marrow,” said Xu. It is likely that the umbilical cord cells are more vital because they are younger than stem cells obtained from the adult bone marrow and in theory will act more quickly to repair wounds or bone defects.

Xu explained: “When a 16-year-old breaks a bone, it usually takes a few weeks to heal. In a 60-year-old, it likely takes a few months. Umbilical cord stem cells are only 9 months old and hence are fast in healing.” Xu said human umbilical cord stem cells have the promise to be a superior alternative to bone marrow-derived stem cells, the latter requiring an invasive procedure to harvest. For combat medics, the umbilical cord derived stem cells could potentially be on the shelf and used in the field without causing immunuorejection, said Xu.

Xu said that after a literature search, he believes his laboratory is the first to investigate the seeding of umbilical cord-derived stem cells in injectable and load-bearing scaffolding for bone tissue engineering.

“We are excited about the promise of encapsulating umbilical cord stem cells in an injectable scaffold for stem cell delivery and bone regeneration.” Xu said. “Our research is still in an early stage. We will perform more systematic investigations and animal studies. If indeed human umbilical cord stem cells delivered using injectable scaffolds are more effective in bone regeneration than the commonly studied bone marrow stem cells, it will broadly impact the field of stem cell-based regenerative medicine.”