Patients thrill to reports of a promising antisense drug against Huntington disease, but no one is sure yet whether it works. Dardengo's father, shown holding her son Joel in a 1989 photo, died of Huntington disease. Michelle Dardengo walks her dog near her home in Coquitlam, Canada. The dark shadow of Huntington disease fell squarely over Michelle Dardengo's life on the day in 1986 that her 52-year-old father was found floating in the river in Tahsis, the remote Vancouver Island mill town where she grew up. Richard Varney had left his wedding ring, watch, and wallet on the bathroom counter; ridden his bike to a bridge that spans the rocky river; and jumped. The 4.5-meter drop broke his pelvis. The town doctor happened to be fishing below and pulled Varney out as he floated downstream, saving his life. The once funny man who read the Encyclopedia Britannica for pleasure; the good dancer who loved ABBA, the Three Tenors, and AC/DC; the affable volunteer firefighter--that man was disappearing. He was being replaced by an erratic, raging misanthrope wedded to 40-ounce bottles of Bacardi whose legs would not stay still when he reclined in his La-Z-Boy.
Huntington's disease is brutal in its simplicity. The disorder, which slowly bulldozes your ability to control your body, starts with just a single mutation, in the gene for huntingtin protein. Huntington's simplicity is exciting, because theoretically, it means you could treat it with a single drug targeted at that errant protein. But in the 24 years since scientists discovered it the gene for huntingtin, the search for suitable drugs has come up empty. This century's riches of genetic and chemical data seem like it should have sped up research, but so far, the drug pipeline is more faucet than fire hydrant.