A young patient holds her experimental cystic fibrosis medication, Vertex’s Kalydeco. The WSJ reports today on the two-decade quest to develop drugs for cystic fibrosis following the discovery of the gene responsible for the respiratory disease. In addition to the scientific hurdles, a big challenge facing those seeking treatments was finding a company willing to make the investment. “Very few returned my call,” recalls Robert Beall, chief executive of the Cystic Fibrosis Foundation . The foundation ended up taking a role normally reserved for companies or venture funds, and became one of the pioneers of patient advocacy groups’ serious influence in drug R&D. Foundations are spending more than $100 million a year to fund work on diseases including type 1 diabetes, Parkinson’s and multiple sclerosis, according to clinical research-information firm CenterWatch . The Cystic Fibrosis Foundation’s venture philanthropy began in the 1990’s, after scientists had come up with an idea of what the gene did and how mutations resulted in the disease. Beall said he didn’t even bother asking the largest drug makers to investigate cystic fibrosis,

because of their focus at the time on so-called blockbuster drugs offering billions of dollars in returns from sales to large numbers of patients. Cystic fibrosis, which afflicts 30,000 people in the U.S., wouldn’t be appealing. Instead, Beall targeted smaller companies with the technology to look for compounds that could correct for the cellular problems caused by a mutation. To further entice these small firms, the CF Foundation offered millions of dollars to do the drug discovery work. “We took the risk” so the companies would start and projects would develop the momentum, Beall says. The foundation has now given or committed to give $315 million to companies for drug research. Among the first to receive foundation funding was Aurora Biosciences in San Diego. It was later bought by Vertex Pharmaceuticals. Yesterday Vertex filed for FDA approval of Kalydeco , which aims to correct for a disease-causing mutation found in 4% of cystic fibrosis patients. Image: Brett Deering for the WSJ

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Not Just a Cheerleader: Foundation Helped Drive Cystic-Fibrosis Research