A tailormade drug slowed a man's ALS. Is it the future of treatment?

Dr. Rakesh Parekh was in his mid-40s when he first noticed something was wrong: During his workouts, his left arm was weaker and struggled to keep up.

Dr. Rakesh Parekh was in his mid-40s when he first noticed something was wrong: During his workouts, his left arm was weaker and struggled to keep up. Initially, Parekh, an emergency medicine doctor and father of three, assumed he was overdoing it in the gym.

“There was no part of me that wanted to believe that was in jeopardy,” said Parekh, 52, of Orlando, Florida.

Parekh had watched his father die of amyotrophic lateral sclerosis, the progressive neurological disease commonly known as ALS or Lou Gehrig’s disease. In 2021, a neurologist confirmed what Parekh feared: he had the same incurable diagnosis.

“I really didn’t think that there was something out there that could prolong my life or improve my life,” Parekh said.

That wasn’t the case. A genetic test told Parekh’s doctors that his disease was caused by a mutation on a gene called CHCHD10, information that his father wouldn’t have had just a decade prior. Though exceptionally rare, CHCHD10 is one of the roughly 40 genes that scientists have identified as the cause of about 20% of ALS cases.