2009 Annual Report: Moving Toward the Clinic

Researcher working with stem cells in a low oxygen environment

Sanford-Burnham
Medical Research Institute

	Disease Team Projects
	HIV/AIDS Amyotrophic lateral sclerosis Type 1 diabetes Epidermolysis Bullosa Malignant glioma Heart failure Leukemia Macular degeneration Sickle cell disease Solid tumor Stroke

News at CIRM:

Moving Toward the Clinic

In naming Proposition 71 The California Stem Cell Research and Cures Act its authors emphasized the ultimate goal of delivering life-saving regenerative medical treatments and cures to the people of California and the world.

Each incremental breakthrough made by a CIRM-funded researcher brings us closer to achieving these therapies for the more than 70 currently incurable diseases and injuries that could
be addressed by stem cell science.

Of the more than $1 billion committed by CIRM for research funding, the largest portion is for research that bridges basic discoveries to clinical applications. The Early Translation and Disease Team awards granted in 2009 together provide $300 million for such projects.

Early Translational Awards
The Early Translational grants provided more than $70 million to fund 16 grants focused on moving basic research toward human clinical trials and are an important part of CIRM’s strategy to advance the best basic research into clinical application. These grants were given to either advance molecules or cell types with high potential for use in regenerative medicine or to address a bottleneck in the development of new therapies.

In 2009, the Early Translational grants funded research that could help advance therapies for diseases like type 1 diabetes, Parkinson’s disease, arthritis and blood diseases in infants. Underscoring the importance of this leg of CIRM’s grant strategy, the Early Translational grants are expected to be awarded annually.

Disease Team Awards

CIRM’s Disease Team Awards launched an innovative model for organizing interdisciplinary research teams within California and around the world. These awards encourage the integration of basic, translational and clinical research in an innovative team approach that has the potential to advance therapies into the clinic more rapidly.

Recipients of Disease Team Awards are expected to file a request to begin clinic trials with the U.S. Food and Drug Administration in just 48 months—or less. 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. “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,” he said. Notably, eight teams have significant participation from pharmaceutical or biotech industry scientists who are well versed in the clinical and regulatory path of drug development.

In October, CIRM’s Governing Board awarded 14 Disease Team Awards that have the potential to lead to breakthroughs for up to 11 different diseases including several types of cancer, HIV/AIDS, type 1 diabetes, stroke, ALS, sickle cell anemia and a rare genetic skin disorder, epidermolysis bullosa. Advancing a therapy or cure for any one of these diseases would be an unprecedented medical breakthrough.

The grants were awarded to research spanning three approaches: embryonic stem cells, adult stem cells and applications of stem cells to develop or deliver traditional small molecule therapies.

Researchers in California had the choice of building their teams with scientists from within the state, or augmenting the teams with additional expertise from CIRM’s international Collaborative Funding Partner program. Four of the succesful teams had partnerships with Canadian or U.K.  scientists, with the two countries committing approximately $35 million and $8 million, respectively. (Read more about CIRM's international partnerships.)

One award went to a for-profit company—Novocell, which is developing a therapy for type 1 diabetes—and will take the form of a loan. Robert Klein, chairman of the CIRM Governing Board said, “In providing stem cell funding in the form of loans, CIRM is able to fund more science and make a more significant impact on the speed of bringing new stem cell-based therapies to the people of California and the world.”