ALS Association

The ALS Association is an American nonprofit organization that funds global amyotrophic lateral sclerosis (ALS) research, provides care services and programs to people affected by ALS through its nationwide network of clinical care centers, and works with ALS advocates around the country for state and federal policies that serve people living with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

Research

The ALS Association has partnerships with Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, ALS Finding a Cure, and the Muscular Dystrophy Association. Additionally, the organization is a research partner for Answer ALS (started by Steve Gleason), Target ALS (founded by Dan Doctoroff), and ALS ONE. The organization also provides funding for the ALS Research Forum, a project of Prize4Life, which has since merged with the ALS Association.

Because of the awareness and funding from the Ice Bucket Challenge, the Association committed nearly $90 million in research funding between 2014 and 2018, a 187% increase in its annual research funding. This included $81.2 million across 275 research grants in the U.S. and $8.5 million internationally. The research led to the discovery of five new genes connected to ALS:

NEK1 - mutations of this gene contribute to both sporadic and familial cases of ALS

KIF5A - the KIF5A protein functions within neurons to transport materials up and down the axon, a nerve fibre that carries information to muscle cells and is also called the cytoskeleton; the ALS mutation truncates the protein near its end so that the full protein is not made

C21orf2

TUBA4A - mutations in this gene destabilize the microtubule network and diminish its repolymerization capability; this is another cytoskeleton defect, like KIF5A

TBK1

According to Hemali Phatnani, director of the Center for Genomics of Neurodegenerative Disease at the New York Genome Center, funds raised from the Challenge led to the creation of one of the largest resources of ALS whole genome-sequencing data, which has been shared with partners around the world.

In September 2020, the New England Journal of Medicine reported that a new drug combination, AMX0035, was safe and effective at slowing the progression of ALS in a clinically meaningful way. The ALS Association had provided early financial support for research into AMX0035 with Ice Bucket Challenge donations. After the clinical trial outcomes were published, The ALS Association launched a petition asking Amylyx Pharmaceuticals and the Food and Drug Administration to work together to make the drug available to people with ALS as quickly as possible.

Public policy

After four years of efforts by thousands of people affected by ALS to cultivate significant bipartisan support, the ALS Disability Access Act of 2019 was signed into law in December 2020.