California Institute of Regenerative Medicine awards multi‑million grants to stem‑cell biotech firms

Through its Common Fund the NIH has established the Center for Regenerative Medicine (NIH CRM), to support this field, with the goal of accelerating the translation of stem cell-based clinical therapies. State funding filling the void With stem cell research in general not attracting a significant amount of venture funding, the California Institute of Regenerative Medicine (CIRM), which was established in 2004 with $3 billion for stem cell research at California universities and research institutions, has begun to fill the void left by traditional venture capital firms. To date CIRM has allocated $150 million in funding to help move promising stem cell-based therapies from the bench into clinical trials. "We are a lot closer to having promising therapies ready for clinical trials, so it makes sense that we step up our engagement with industry to help fund those trials and move those therapies closer to approval by the FDA," said Duane Roth, vice chair of the governing board of CIRM. CIRM’s funding for translational research is good news for biotech companies, providing them with a source of funds in a field where it remains challenging to raise private capital. Three biotech companies have been funded so far under CIRM’s Strategic Partnership Awards initiative. A grant of $10.1 million was awarded to ViaCyte Inc. to continue preclinical research and initiate clinical testing of an embryonic stem cell-based therapy for patients with insulin-dependent diabetes. Meanwhile, Bluebird Bio Inc. will use a $9.3 million grant to support a Phase I/II study to evaluate the safety and efficacy of LentiGlobin, the company’s programme for the treatment of the inherited blood disorder beta-thalassemia, which will be initiated in the US in 2013. StemCells Inc. has been awarded up to $20 million to fund preclinical development of its product consisting of purified human neural stem cells for treating Alzheimer's disease, with the goal of filing for permission to carry out a clinical trial in that time. In July, CIRM approved a separate award to the company for up to $20 million to fund preclinical development of a cell therapy for spinal cord injury. Public Markets It has been a good year for stem cell companies on the public markets, with the thirteen publicly-listed stem cell companies showing an average increase in share price of 11.6 percent in the year-to-date. This boost came from a regulatory approval and positive clinical trials results. It was a big breakthrough for the field as a whole when the Canadian regulator Health Canada approved Prochymal, Osiris Therapeutics Inc.'s allogeneic stem cell treatment for graft-vs.-host disease (GvHD) in children. The decision marked the world's first regulatory approval of a manufactured stem cell product and the first therapy approved for GVHD — a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis. The company’s stock value has almost doubled in the course of this year. Meanwhile, Newark, California-based StemCells, Inc. has seen its shares rise 148 per cent in the year to date. In addition to its CIRM grants, the company recently reported clinical and preclinical data demonstrating the therapeutic potential of a cell therapy for treating myelination disorders. Pluristem Therapeutics Inc.’s stock value also has jumped 44 per cent, on the strength of reporting a single case study in which a patient with aplastic bone marrow who received an intramuscular injection of its PLacental expanded cells under compassionate use saw an improvement. The company was also able to successfully complete a public offering which netted about US$30 million. Positive clinical trials will begin to encourage investment While still in their early stages of development - and with clinical trials having only involved a limited number of patients - reports to date have been very promising, and provide further validation for encouraging investment in stem cell therapeutics. For example, data from a human embryonic stem cell trial conducted by Advanced Cell Technology and published in medical journal The Lancet, showed that two patients with Stargardt’s disease, a degenerative eye condition, had regained some vision. In addition, positive early data from a spinal cord injury trial involving StemCells’ neural stem cells indicated that two patients with no feeling below the site of injury were able to regain sensation, while in another study from the company, patients with a rare myelination disorder were able to create myelin, an advance that holds promise for treating multiple sclerosis and cerebral palsy. This scientific progress has helped breath a new sense of optimism into the US stem cell sector. The Fiscal Cliff apart, it seems likely this momentum will continue now Obama has secured a second term. Peter Winter is a writer, editor and analyst on the global biotechnology industry. He is currently editor of BioWorld Insight. Steven Victor MD IntelliCell BioSciences Chairman/CEO 460 Park Avenue 17th Floor New York, New York 10022 www. IntelliCellBioSciences.com HOUSE_OVERSIGHT_013483