CF Foundation Drug Development Model is “Proof of Principle” for Other Diseases, says NIH Director in U.S. Senate Briefing
May 24, 2010
Francis Collins, M.D., Ph.D., director of the National Institutes of Health (NIH), highlighted the Cystic Fibrosis Foundation’s success at bridging the gap between basic science discoveries and creating new patient therapies at a May 20 U.S. Senate briefing.
The briefing was co-hosted by FasterCures/The Center for Accelerating Medical Solutions, Sens. Richard Shelby (R-Ala.) and Richard Durbin (D-Ill.), and the CF Foundation.
“There is great progress being made in cystic fibrosis research,” said Collins, who noted how advancements in treatments and care have transformed CF from a pediatric illness to one where nearly half of people with the disease are age 18 or older. The Foundation’s model, he said, is “proof of principle of what we ought to be able to try to do for many other diseases.”
Collins said his goal is to take the hope and promise of recent research developments in life-threatening diseases such as cystic fibrosis “and turn that into real results.”
“We have more than 30 drugs in various stages of clinical trials, many of which are directed to treating the basic defect of cystic fibrosis,” said Robert Beall, Ph.D., president and CEO of the CF Foundation, who also spoke at the briefing.
Beall added: “These efforts have changed this disease from one of hopelessness and despair into one of hope, optimism and excitement among the entire CF community. We have created a roadmap of how to take promising therapies for a disease from the test tube to the bed side.”
The briefing highlighted how federal funding for medical research at the NIH and other agencies can be leveraged to more quickly solve pressing medical challenges, create new therapies for patients and save lives.
Currently, the ability to translate advancements into treatments severely lags behind the pace of innovation. It takes 10-15 years, on average, for an experimental drug to travel from a lab to U.S. patients, according to the Tufts Center for the Study of Drug Development.
Dr. Collins, a distinguished geneticist who led the international effort to map the human genome, is a co-discoverer of the cystic fibrosis gene. The NIH is the primary federal agency for conducting and supporting medical research.
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