UCSD, Salk, Burnham researchers get nearly $55 million in CIRM grants

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Three local research teams have been award more than $54.8 million in grants from the California Institute of Regenerative Medicine for projects to develop drugs for leukemia, Lou Gherig’s disease and brain tumors.

In addition, Torrey Pines Mesa-based Novocell Inc, will receive a total of $20 million in the form of a loan.

The institute, known as CIRM, was established in 2004 after voters approved Proposition 71 to fund stem cell research at research institutions and companies throughout California. On Wednesday, the governing board approved more than $250 million in funding for 14 multidisciplinary teams of California researchers, including some from UCSD, the Salk Institute and the Burnham Institute for Medical Research.

CIRM President Alan Trounson said the pace of the Disease Team projects stands in contrast to the decade or more that’s usually required to reach clinical trials.

“Scientists have talked for years about the need to find ways to speed the pace of discovery,” he said. “By encouraging applicants to form teams composed of the best researchers from around the world we think CIRM will set a new standard for how translational research should be funded.”

The four-year grants mark the first CIRM funding explicitly expected to result in FDA approval for a clinical trial, according to a press release. The researchers — basic and industry scientists and clinicians working in collaboration with international partners, the Medical Research Council, UK, and the Cancer Stem Cell Consortium, Canada— will receive incremental funding upon achieving set milestones.

The local grants include:

— $20 million to a team led by Dennis A. Carson, M.D., director of the Moores UCSD Cancer Center and professor of medicine, and Catriona Jamieson, M.D., Ph.D., assistant professor of medicine and director of the Cancer Stem Cell Research Program at the Moores center.

Their team will work to develop novel drugs against leukemia stem cells, collaborating with a Canadian research team led by pioneering leukemia stem cell scientist John Dick, Ph.D., at the University of Toronto.

The research team will develop six existing candidate molecules targeting leukemia stem cells — but not normal, blood-forming or hematopoietic stem cells — and these will be tested against both chronic and acute forms of leukemia.

— $15.6 million to a Salk Institute team focusing on developing a novel stem-cell based therapy for Amyotrophic Lateral Sclerosis (ALS) — or Lou Gehrig’s Disease.

Sam Pfaff, Ph.D., a professor in the Salk’s Gene Expression Laboratory and an investigator for the Howard Hughes Medical Institute, will lead the group that includes co-principal investigators Drs. Larry Goldstein and Don Cleveland, both of UCSD.

They hope to develop an ALS therapy based on using human embryonic stem cells to create astrocyte precursor cells for transplant into patients, where the hope is the astrocytes would mature into new and healthy glial cells.

— $19.2 million to a team led by Mitchel Berger of UC San Francisco with co-principal investigator Webster Cavenee, a professor at UCSD School of Medicine and the Ludwig Institute for Cancer Research at UCSD. It also includes Evan Snyder, M.D., Ph.D., director and professor of the Burnham Institute’s Stem Cells and Regenerative Biology program.

They will work to find a treatment for brain tumors using neural stem cells that have been genetically modified to carry a tumor-killing drug.

— $20 million to Novcell, which will work with a team from UCSF headed by Dr. Jeff Bluestone for work on a cellular therapy for the treatment of diabetes.

Other diseases being targeted by the teams include HIV/AIDS, type1 diabetes, macular degeneration, heart attack and stroke, sickle cell anemia and skin disease.

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