NEW YORK CITY — Jeff Vierstra began receiving experimental spinal infusions targeting a mutated FUS gene at Columbia University's Eleanor and Lou Gehrig ALS Center in New York City in what is described as the first known attempt to prevent amyotrophic lateral sclerosis. Vierstra, a scientist and skier who carries the same FUS gene mutation that killed multiple members of his family, has received the infusions every few months for the past three years.

The experimental approach, pioneered at the Gehrig ALS Center, targets a rare genetic form of ALS. Vierstra, along with his sisters Erin and Leigh, tested positive for a mutation in the FUS gene. Both sisters developed symptoms of ALS and participated in a clinical trial at Columbia for an experimental treatment targeting the mutated gene. Both Erin and Leigh Vierstra died of complications from the disease.

The family's history with ALS extends further. Vierstra's mother died of complications from the disease when he was two years old. All of his mother's siblings died of ALS in their late thirties and early forties.

Dr. Neil Shneider, a neurologist at the center, examined Vierstra and found abnormalities on an electromyography test of his muscles. "We thought this meant that this was an early sign of disease onset and that he was at risk for developing full blown disease," Shneider said. He then offered Vierstra the same experimental FUS gene-targeting treatment that had been given to his sisters in an attempt to prevent the onset of ALS.

"I jumped at the opportunity to do that," Vierstra said. After one year of treatment, the abnormalities detected in his muscle tests had normalized. He has not developed ALS and has outlived multiple family members who died of the disease.

ALS causes progressive degeneration of motor neurons that control muscle movement, leading to gradual loss of the ability to walk, talk, and eventually breathe. There is currently no cure for the disease. An estimated 35,000 people in the United States are living with ALS, according to the Centers for Disease Control and Prevention.

"I think there's real hope and opportunity to make this a liveable disease, one that isn't fatal," Shneider said. He and the ALS Center at Columbia are working to extend their research beyond FUS-linked forms of the disease.