Monday, November 2, 2009

CIRM Awards Grants Supporting ALS Research

There has been another big investment to fuel ALS stem cell therapies. The California Institute for Regenerative Medicine (CIRM) has awarded support to 14 pre-clinical projects this year. This comes in the context of other recent support for such projects: Neuraltem was recently approved for a Phase 1 ALS clinical trial using a stem cell approach, and BrainStorm Cell Therapeutics, of Petach Tikva, Israel, a competitor for Prize4Life's Avi Kremer ALS Treatment Prize, also recently secured funding to complete pre-clinical stem cell trials for ALS, and expects to commence in early 2010.

The InVivo blog reports that the CIRM’s international partners contributed funding, bringing the total awards to an unprecedented $250 million.

Particularly interesting for our purposes is the $15.6 million grant given to the Salk Institute for translational research. The support is meant to focus on developing a novel ALS therapy using a stem cell approach. Notably, this award is the first CIRM funding explicitly expected to result in FDA approval for clinical trials.

Dr. Sam Pfaff, investigator at the Howard Hughes Medical Institute and professor at the Salk's Gene Expression Laboratory, has been named Principal Investigator (co-PI's are Dr. Larry Goldstein and Dr. Don Cleveland, both from UCSD). Dr. Pfaff will lead research on the four-year project.

The grant will support research focusing on the role of astrocytes in treating ALS. Astrocytes are star-shaped cells that provide nutrients for nearby motor neurons, jointly acting as a support system. Pfaff, Goldstein, and Cleveland will cultivate astrocyte ‘precursor’ cells—astrocytic ‘seeds’—and identify those best suited to be implanted in animal models. They hope that, by implanting astrocyte ‘precursors’ into animals, the precursors will fully mature into astrocytes and provide support for sick motor neurons. Because astrocytes are also able to ‘clean up’ the toxic areas around diseased motor neurons, they could potentially slow or stop the progression of the disease.

When the astrocyte 'precursors' have been tested for efficacy and safety, and have been approved by the FDA, the team will move into clinical trials—that is, they will test this approach in humans.

Additionally, one San Diego-area biotechnology company was selected to receive a CIRM grant. Academic researchers have historically received the majority of such funding, and industry executives have been displeased with the lack of opportunities available to companies. Though CIRM is one of the largest sources of funding for human embryonic stem cell research in the world, only a handful of its hundreds of grants have ever been awarded to companies.

by Meghan Kallman

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