'Game-changer': FDA approves drug for rare form of ALS
PITTSBURGH (KDKA) — The FDA has approved a new medication to treat amyotrophic lateral sclerosis.
The treatment targets ALS like no other medication on the market.
According to Dr. Sandeep Rana from Allegheny Health Network, at least 30,000 people are dealing with ALS in the United States, including an estimated 300 to 500 people in the Pittsburgh area.
"ALS is a devastating illness," Rana said. "It's a neurological disease where patients start to get weak. They lose muscle strength. They lose muscle mass."
It can impact people's ability to swallow, speak and breathe. It can eventually become fatal.
There are three medications on the market that help protect the cells being attacked by toxins ALS creates, but this week the FDA approved a new medication, Qalsody, that acts more offensively in cases where ALS is genetic.
"The medicine that has been approved is for a very subset of this generic form of ALS," Rana said. "It silences that gene so it does not produce that toxic protein and potentially it could be a game-changer for this small subset of patients."
Rana said in studies, the drug slowed the progression of ALS symptoms in patients but did not stop them. The hope is the medication can prevent the disease entirely for people who have the gene but are presymptomatic.
"Once you have identified a family with this gene disorder and you screen the other family members and identify other family members who have the gene and are still not manifesting the disease, that would be the very potentially optimal candidate for this drug," Rana said.
