ALS Awareness Month: How Research Has Had an Impact

There is significant progress – and a new reason to be hopeful for those with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. It’s a rare disease that shows up first as muscle twitches and cramping since it affects the neurological functioning of voluntary muscles. There is no cure for ALS, and over time, the symptoms get progressively worse. But a couple of factors have accelerated findings, and the outlook is not nearly so dire now for people with ALS and their families.

The Ice Bucket Challenge of 2014 played a big part. The social media phenomenon raised more than $100 million for the ALS Foundation, which enabled the organization to nearly triple what it was spending on research. By May of 2023, three new drugs were approved, the latest by accelerated approval because of its potential. It’s a gene-based therapy that was developed to treat a specific strain of ALS, but is now being studied as a possible way to prevent the disease from even showing up.

While some gene mutations have been identified as associated with ALS, the vast majority of cases are considered sporadic, or non-familial. What’s exciting to many researchers now is the possibility that these new therapies get closer to the basic cause of the disease, meaning they could be used to treat the non-genetic cases as well.

Advocates say they work toward a future without ALS and these new developments illustrate that they’re clearly headed in that direction. And the money raised for research has made a huge contribution. But as with so many neurodegenerative disorders, there’s one thing nearly everyone can do to help advance the science, that doesn’t cost the giver a dime. Plan now to donate your brain when the time comes. Researchers say there is no substitute for post-mortem human brain tissue—it’s the most precious gift one can give.