Frank Accurso, MD, with the University of Colorado School of Medicine and Children’s Hospital Colorado, has been honored by the Clinical Research Forum for his work in pioneering a genetically based treatment for cystic fibrosis that is benefiting Colorado kids and young adults.
The clinical trial Accurso led resulted in federal approval of the new treatment just last January. Already, dozens of children with cystic fibrosis (CF) are being treated with the drug Kalydeco in Colorado.
A CU pediatrics professor who practices at Children’s Hospital Colorado, Accurso is one of 10 recipients nationally of the Forum’s Clinical Research Achievement Awards.
In the past, doctors only could treat complications caused by CF, a fatal disease. Two decades ago, scientists had figured out that a defect in the protein CFTR causes cystic fibrosis. That pointed the way for researchers. Kalydeco, developed through efforts of the Cystic Fibrosis Foundation and Vertex Pharmaceuticals, Inc., showed promise.
Accurso led the clinical trial that showed the new treatment, using the drug Kalydeco, helps about 4 percent of CF patients by targeting the mutation and improving lung function. The New England Journal of Medicine published the results. Kalydeco was approved for use in late January, 2012.
He hopes as many as 90 percent of CF patients eventually will benefit. (Accurso and current patients are available for interviews).
Along with Accurso’s CF work, the awards recognize new therapies developed for leukemia and a rare lung condition, as well as ways to identify immune system targets of anti-HIV therapies and to find risk factors for cardiovascular disease.
“Clinical research is key to our efforts to turn discoveries into health, “said National Institutes of Health Director Francis S. Collins, MD, PhD. “NIH is a major supporter of clinical research and I am delighted to see this important field get the recognition it so richly deserves.”
Winning researchers were honored today during the Clinical Research Forum annual meeting and awards dinner in Washington, DC, where they also presented their work.
Listen to the Colorado Public Radio report on the breakthrough >>