Accurso’s pioneering treatment for cystic fibrosis earns honor

By Staff


Frank Accurso, M.D., a pediatric professor at the University of Colorado School of Medicine who practices at Children’s Hospital Colorado, recently was 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 led by Accurso resulted in federal approval of the new treatment in January. Dozens of children with cystic fibrosis (CF) now are being treated with the drug Kalydeco in Colorado.

In the past, doctors only could treat complications caused by CF, a fatal disease. Two decades ago, scientists figured out that a defect in the protein CFTR causes the disease and 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. He hopes as many as 90 percent of CF patients eventually will benefit.

Accurso is one of 10 recipients nationally of the Forum’s Clinical Research Achievement Awards. He, along with the other winning researchers were honored during the Clinical Research Forum annual meeting and awards dinner in Washington, D.C., earlier this month, where they also presented their work.