By Paul M. Barrett October 30, 2014 – Published via Bloomberg Business
The 2014 World Cup elevated soccer to the top of Aidan Leffler’s roster of obsessions, rivaled only by endangered big cats—especially jaguars, “the coolest”—and Star Wars spaceships. In recognition of his new interest, he’s set up a miniature soccer field with 4-foot-wide goals in his backyard in suburban Bellevue, Wash. “Watch this!” he shouts, preparing to fire a penalty kick.
Small for his age, Aidan, 11, moves awkwardly, shoulders high and hunched. He uses a lightweight plastic beach ball, not a regulation leather soccer ball. He begins his approach, pulls back his right foot, and … collapses to the grass.
Mitch Leffler, the sole spectator, moves toward his son. “I’m OK,” Aidan says. “I can do it.” He struggles onto his hands and knees, raises his butt, places his hands one at a time on his thighs, and slowly pushes himself into an upright position. “My leg just wasn’t there,” he says matter-of-factly. His father nods, and the game resumes.
Aidan has Duchenne, the deadliest strain of muscular dystrophy. It’s inherited maternally on the X chromosome and mostly afflicts boys. Parents typically sense something is wrong when their sons at 3 or 4 don’t run around or they start falling for no obvious reason. Beginning in the legs, Duchenne destroys muscle, which is replaced by fat and scar tissue. Victims lose the ability to walk by adolescence. Eventually the disease causes cardiac and/or respiratory complications that lead to death by the mid-20s. One in 3,500 newborns has Duchenne, which translates to around 15,000 cases in the U.S. There’s no cure.
“Aidan doesn’t really understand yet,” his mother, Mindy, says, “but it’s basically a slow-motion death sentence.”
There’s reason to hope—not for a miracle, but for a reprieve. Three small biotech companies are competing to develop drugs designed to address the cellular defects that cause some cases of Duchenne. If proven safe and effective, the drugs would turn Duchenne into a less devastating form of muscular dystrophy. Clinical trials, however, have yielded uneven results, and the U.S. Food and Drug Administration has made equivocal pronouncements about which of the drugs, if any, have a shot at approval. Even a marginally effective drug would likely command an astronomical price, making the winning company a billion-dollar sensation.