Driven by loss, father inspires tireless pursuit of a cure

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.