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Two heads are better than one
SAN FRANCISCO—In 2004, California voters, through the passage of the statewide ballot issue Proposition 71, overwhelmingly called for the establishment of an entity to make grants and loans available for stem cell research. Seven years later, this entity—the California Institute for Regenerative Medicine (CIRM)—has been called the world's largest backer of stem cell research, and with a recently signed agreement, the CIRM is now collaborating with perhaps the largest sponsor of research funding in the world: the U.S. National Institutes of Health (NIH).
A memorandum of understanding announced at the end of October and signed by CIRM President Alan Trounson and NIH Deputy Director for Intramural Research Michael Gottesman has established a framework to advance the parties' "complementary and synergistic goals," and to "help NIH and CIRM researchers work together to bring their special talents in stem cell and regenerative medicine research to bear upon prevention, diagnosis and treatment of various diseases."
"The goal is to use this agreement as a springboard to create synergy between our funding streams and expand the capacity of each agency to move advances in basic research more rapidly into the clinic," said Dr. Mahendra Rao, director of the NIH Center for Regenerative Medicine, in a statement.
It was the appointment of Rao that "allowed us to get this agreement done efficiently," says Trounson.
"We're very pleased to be able to work with what is clearly the strongest research agency in the world, and broadly across many areas of medicine," Trounson says. "This partnership joins our strengths in stem cells and regenerative medicine with their broad medicine capabilities."
Since its inception, the CIRM has used bond proceeds to fund basic and applied biomedical research focused on developing diagnostics and therapies, and on other vital research opportunities that it believes will lead to life-saving medical treatments. All proposals are peer-reviewed to support the most promising scientific research. Research grants are made only to California-based research institutions.
"In the beginning, we established the infrastructure that is creating the training grounds and scientists other areas to join in stem cell research being conducted in California—scientists who already had track records in stem cell research," Trounson explains. "We then added a facility, and later built 12 new research institutes in California. We have allocated more than $1 billion to these institutes, and all but two are open."
Funding research involving all types of stem cells, the CIRM's projects focus on specific disease therapies with the end goal of generating proof-of-concept for their therapeutic approach, finding a candidate drug or cell type to be developed into a therapy or filing an investigation new drug (IND) application with the U.S. Food and Drug Administration to begin clinical trials. In future years, the agency will fund increasing amounts of new therapy development in different diseases. The CIRM spreads its funding around to a wide variety of disease areas, including blood/immune disorders, bone/cartilage disorders, cancer, endocrine disorders/diabetes, cardiovascular disease, gastrointestinal/liver disease, muscular disorders, nervous system disorders/injuries, sensory organ disorders and reproductive disorders.
"There is a sort of very sharp front edge to the work. Some of the studies are progressing very quickly, while others are still making their way through the pipeline," Trounson says.
But the CIRM "has always been interested in linking up with federal agencies—and the NIH funds a lot of work in the area of stem cell research," Trounson notes.
Specifically, the agency will work with the NIH through its trans-NIH Center for Regenerative Medicine (NIH CRM), the newly established NIH Center for Translational Therapeutics (NCTT) and the NIH Clinical Center. The latter, the world's largest hospital dedicated entirely to clinical research, is what the CIRM found most attractive, says Trounson. The center houses basic, translational and clinical research efforts of the NIH intramural research community.
"They have the largest facility focused on clinical trials in the United States, maybe the world," he says. "They have a fantastic program there, and it's something I wouldn't really try to replicate in California, but thought that getting access to the clinical center would be great. We thought it would be useful to utilize resources that are already available, not duplicate them."
There are several opportunities for collaboration between the two parties. The first
relates to the current round of applications for CIRM's Disease Team Therapy Development Research awards. During the planning phase of these awards, California teams could develop collaborations with researchers at the NIH Clinical Center on various aspects of a preclinical, Phase I, Phase I/II or Phase II clinical trial.
Another option is to give California researchers the opportunity to take part in NIH Clinical Center visiting fellowship or clinical investigator training programs. Finally, CIRM institutions may be given access to unique resources that may not be available at their home institution.
In addition to translational and clinical projects, the two groups are exploring ways for the CIRM and NIH scientists to collaborate in earlier translational or basic biology projects.
"It takes time to get through the clinical trials process, so you can demonstrate in humans how cell products or regenerative medicine is going to work," Trounson concludes. "On the other hand, I remain very optimistic that this is going to be a big game-changer in modern medicine."