Imagine being trapped in a paralyzed body.
That's how the ALS Association describes the progression of a neurological disease called ALS, which 31,000 people in the United States are living with.
Thankfully, there is hope for a better quality of life thanks to a drug that the Food and Drug Administration just approved.
In 2014, people all over the world joined in on the ALS ice bucket challenge. It raised $115 million in donations, according to the ALS Association.
CEO Calaneet Balas says that money is what funded the research for a new FDA-approved ALS treatment.
"It's a big day," Balas said. "It's a really big victory for this community."
ALS, also known as Amyotrophic Lateral Sclerosis or Lou Gehrig's disease, is a terminal, progressive neurodegenerative disease. Mark Weston was diagnosed in 2019.
"People lose the ability to move their muscles," Balas said. "So that might be you can't walk. Then eventually, maybe you can't use your arms, you can't use your fingers, you can't speak, you can't swallow. Eventually, you can't breathe on your own, and then you pass away."
However, a new treatment called RELYVRIO was shown in clinical trials to slow the progression of the disease and extend life.
"This might buy some people some time," Weston said. "So that if you extend your life by five months or six months, or maybe a year, maybe another drug will come along that might be a more effective treatment."
Weston says RELYVRIO is one of three FDA-approved treatments now on the market. He's currently taking pills for a different treatment that's also intended to prolong his life. Whether or not he starts RELYVRIO, he said, will depend on his Medicare coverage.
"The company has priced it at, I believe, $13,600 a month or annually, $158,000," Weston said. "And it's all going to depend on what my insurance company decides to do."
According to pharmaceutical company Amylyx, physicians will be able to prescribe RELYVRIO immediately, and they anticipate specialty pharmacies to start to fill prescriptions and ship RELYVRIO to people living with ALS in the next four to six weeks. The company says it has been actively engaging in discussions with insurance companies to help ensure that this important therapy will be accessible to as many people living with ALS as quickly as possible.
Weston says it's not a cure but a positive step in the right direction.
"Well, I am thrilled," Weston said. "It's really exciting for people with ALS and their family members to have a third choice now."
A written statement from pharmaceutical company Amylyx said the following:
"Last week’s FDA approval of RELYVRIO is an exciting milestone for the ALS community and is a major step toward achieving Amylyx’s mission to one day end the suffering caused by neurodegenerative diseases. RELYVRIO is the first approved drug for ALS to demonstrate a statistically significant benefit in function in a clinical trial as well as an observed benefit on survival in a longer-term post hoc analysis. These robust data were published in the peer-reviewed medical journals New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry. Amylyx’s goal is that every person who is eligible for RELYVRIO will have access as quickly and efficiently as possible, as we know people with ALS and their families have no time to wait. Our teams are in place and ready to help people living with ALS gain access to this important new therapeutic option."