Last week, the Food and Drug Administration issued an announcement with a title only a federal bureaucrat could write: “FDA approves Kalydeco to treat rare form of cystic fibrosis.’’ In fact, it was a blockbuster development, the first drug that would treat the cause, rather than the symptoms, of cystic fibrosis, a medication that showed such breathtaking results in clinical trials that it was sanctioned by the FDA in about half the typical time.
Behind that announcement sits a human narrative, a quarter century in the making, of loss, hope, and triumph.
Joey O’Donnell was, by every account, an extraordinary boy who faced a ferocious enemy, a disease that attacks the lungs and airways and leaves no survivors. At school, he served as class president and rode the backs of his many friends to get up stairs. On the Little League field, he was a natural hitter who smiled as others ran the bases for him. At home, he choked down 50 pills a day and endured intense physical therapy without ever uttering a syllable of complaint. When he died in 1986, Joey was 12.
“Just a smart, beautiful kid with rotten lungs,’’ his father said.
His death wasn’t only a tragic end, but an unusual beginning to a collaboration involving his father, Boston businessman Joe O’Donnell; a Cambridge-based bioscience company, Vertex Pharmaceuticals; and a national nonprofit, the Cystic Fibrosis Foundation. They joined forces on a project that was at turns backbreaking and groundbreaking, and now find themselves within grasp of a cure for a disease that, just a dozen years ago, seemed incurable.
This first product of the collaboration, Kalydeco, is a pill that will vastly improve, almost certainly prolong, and perhaps even save the lives of some 1,200 cystic fibrosis patients who have the specific mutation it is designed to treat. It is widely believed to be a precursor to drugs that will treat a vast majority of the 30,000 children and young adults in the United States with cystic fibrosis, a disease that leads to the hardening of mucus in the airwaves and lungs and thus diminished ability to breathe and eat.
