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 chapters, 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.^[2] The ALS Association is broken up into distinct chapters, each servicing a particular geographic area of the United States and all working under the umbrella of a national charter and administrator.

ALS Association

Abbreviation	The ALS Association
Founded	1985; 38 years ago (1985)
Type	Non-profit
Focus	advocacy and patient services
Location	Washington, D.C., U.S.
Area served	United States
President and CEO	Calaneet Balas
Chairman	Sue Gorman
Revenue	$37 million (2019)^[1]
Website	als.org

Research

The ALS Association has partnerships with Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital,^[3] ALS Finding a Cure, and the Muscular Dystrophy Association.^[4] Additionally, the organization is a research partner for Answer ALS (started by Steve Gleason), Target ALS (founded by Dan Doctoroff),^[5] 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.^{[citation needed]}

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.^[6][7] This included $81.2 million across 275 research grants in the U.S. and $8.5 million internationally.^[7] The research led to the discovery of five new genes connected to ALS:^[6][8][7]

• NEK1^[9] - mutations of this gene contribute to both sporadic and familial cases of ALS^[9]
• KIF5A^[10] - 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;^[11] the ALS mutation truncates the protein near its end so that the full protein is not made^[11]
• C21orf2^[12]
• TUBA4A^[13] - mutations in this gene destabilize the microtubule network and diminish its repolymerization capability;^[13] this is another cytoskeleton defect, like KIF5A^[13]
• TBK1^[14]

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.^[15]

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.^[16] The ALS Association had provided early financial support for research into AMX0035 with Ice Bucket Challenge donations.^[17] 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.^[18]

Public policy

After four years of efforts by thousands of people affected by ALS^[19] to cultivate significant bipartisan support, the ALS Disability Access Act of 2019 was signed into law in December 2020. This bill eliminates the arbitrary five-month waiting period formerly required before people living with ALS could draw on their Social Security Disability Insurance benefits.^[20]

Care services

As of 2020, there are 39 ALS Association chapters across the U.S., serving people with ALS in all 50 States.^[21] The chapters include services such as patient referrals to Certified Centers of Excellence or Recognized Centers (of affiliated clinics) and coordination of volunteers to provide support and respite to caregivers.^[21]

The Ice Bucket Challenge

In the summer of 2014, the Ice Bucket Challenge raised $220 million after going viral on social media.^[6][8][15] The ALS Association received $115 million of that amount. To participate, individuals were challenged to pour a bucket of ice water over themselves and/or donate money to ALS research and care. Individuals such as former President George W. Bush, Bill Gates, Taylor Swift, Benedict Cumberbatch, LeBron James, and Martha Stewart dumped ice water on their heads to raise money to fight ALS.^[6][7][8] In total, about 17 million people uploaded videos of themselves doing the challenge, and the videos were viewed more than 10 billion times.^[6]

From the challenge, $115 million was raised, and the Association increased the number of patients served by 28%.^[8] Most of the donations were used for research, but there were other uses. For example, local chapters purchased equipment (power wheelchairs, walkers, shower benches, etc.) for ALS patients and were able to provide for everyone on their respective wait-lists.^[22][23]

The Association's clinical network also expanded by 50% as a result of the funding from the Challenge.^[7] A study by RTI International reported 29 new ALS Certified Treatment Centers of Excellence, 20 new Recognized Treatment Centers, and 7 new affiliated clinics.^[24] Also according to RTI, the National Institutes of Health (NIH) has invested nearly $416 million in ALS Association-funded researchers since the Challenge.^[15]

Following the 2014 Ice Bucket Challenge, the ALS Association attempted to trademark the term "ice bucket challenge". Following backlash, the association withdrew the trademark application.^[25] In early 2018, a patient group called the "Terminally Persistent" coalition criticized the ALS Association for not spending the $115 million earned from the Ice Bucket Challenge more readily on research. Additionally, the group complained that the organization was not providing funding to BrainStorm Cell Therapeutics, a pharmaceutical company researching treatments for ALS and running Phase III clinical trials; the ALS Association does not fund Phase III clinical trials.^[26]

Awareness and fundraising

The association's signature fundraising event each year is the "Walk to Defeat ALS". This event is held each fall through spring by local chapters of the association in cities across the United States. Since its inception in 2000, this event has raised more than $265 million.^[21]

References

1. "ALSA Form 990" (PDF). Retrieved 11 October 2019.
2. "5 Important Things the ALS Association Does". January 16, 2018.
3. . ALS News Today. 20 January 2020. Archived from the original on 6 February 2020. Retrieved 6 February 2020.
4. "The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS". MDA. 9 August 2019.
5. . Target ALS. Archived from the original on 2018-02-17. Retrieved 2018-02-16.
6. "5 years later, man with ALS who popularized Ice Bucket Challenge still inspires". Today. Retrieved 2019-11-06.
7. Strub, Chris. "Ice Bucket Challenge Boosted ALS Association Annual Funding By 187%: Report". Forbes. Retrieved 2019-11-06.
8. "Cold Cash: What to Do When Unexpected Money Pours In". The Chronicle of Philanthropy. 2019-07-17. Retrieved 2019-11-12.
9. "ALS Ice Bucket Challenge fuels novel gene discovery". Medical News Today. 28 July 2016. Retrieved 2019-11-12.
10. Nicolas, Aude (March 21, 2018). "Genome-wide Analyses Identify KIF5A as a Novel ALS Gene". Neuron. 97 (6): 1268–1283. doi:10.1016/j.neuron.2018.02.027. hdl:10138/301252. PMC 5867896. PMID 29566793.
11. "KIF5A gene associated with the development of ALS". Drug Target Review. Retrieved 2019-12-10.
12. van Rheenen, Wouter; Shatunov, Aleksey; Dekker, Annelot M.; McLaughlin, Russell L.; Diekstra, Frank P.; Pulit, Sara L.; van der Spek, Rick A. A.; Võsa, Urmo; de Jong, Simone; Robinson, Matthew R.; Yang, Jian (September 2016). "Genome-wide association analyses identify new risk variants and the genetic architecture of amyotrophic lateral sclerosis". Nature Genetics. 48 (9): 1043–1048. doi:10.1038/ng.3622. ISSN 1546-1718. PMC 5556360. PMID 27455348.
13. Smith, Bradley N.; Ticozzi, Nicola; Fallini, Claudia; Gkazi, Athina Soragia; Topp, Simon; Kenna, Kevin P.; Scotter, Emma L.; Kost, Jason; Keagle, Pamela; Miller, Jack W.; Calini, Daniela (2014-10-22). "Exome-wide rare variant analysis identifies TUBA4A mutations associated with familial ALS". Neuron. 84 (2): 324–331. doi:10.1016/j.neuron.2014.09.027. ISSN 1097-4199. PMC 4521390. PMID 25374358.
14. Oakes, James A.; Davies, Maria C.; Collins, Mark O. (2017-02-02). "TBK1: a new player in ALS linking autophagy and neuroinflammation". Molecular Brain. 10 (1): 5. doi:10.1186/s13041-017-0287-x. ISSN 1756-6606. PMC 5288885. PMID 28148298.
15. . ALS News Today. 2019-06-10. Archived from the original on 2019-08-25. Retrieved 2019-12-12.
16. Paganoni, Sabrina; MacKlin, Eric A.; Hendrix, Suzanne; Berry, James D.; Elliott, Michael A.; Maiser, Samuel; Karam, Chafic; Caress, James B.; Owegi, Margaret A.; Quick, Adam; Wymer, James; Goutman, Stephen A.; Heitzman, Daragh; Heiman-Patterson, Terry; Jackson, Carlayne E.; Quinn, Colin; Rothstein, Jeffrey D.; Kasarskis, Edward J.; Katz, Jonathan; Jenkins, Liberty; Ladha, Shafeeq; Miller, Timothy M.; Scelsa, Stephen N.; Vu, Tuan H.; Fournier, Christina N.; Glass, Jonathan D.; Johnson, Kristin M.; Swenson, Andrea; Goyal, Namita A.; et al. (2020). "Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis". New England Journal of Medicine. 383 (10): 919–930. doi:10.1056/NEJMoa1916945. PMID 32877582.
17. Belluck, Pam (September 2, 2020). "2 College Students Dreamed Up an A.L.S. Treatment. The Results Are In". The New York Times.
18. "Drug Combination Slows Progression Of ALS And Could Mark 'New Era' In Treatment". NPR.org.
19. "Congress passes ALS Disability Insurance Access Act, ending 5 month waiting period for patient benefits".
20. "President Signs S. 578". SSA.gov. December 28, 2020.
21. . ALS News Today. 16 January 2018. Archived from the original on 3 February 2020. Retrieved 3 February 2020.
22. "The Ice Bucket Challenge and the promise — and the pitfalls — of viral charity". Vox. 20 July 2019.
23. "Ice bucket challenge: Where did the money go?". NBC News. 31 August 2019.
24. RTI International (May 2019). Evaluation of the ALS Association Grant Programs (PDF) (Report).
25. Ohlheiser, Abby (August 30, 2014). "ALS Association withdraws controversial applications to trademark 'ice bucket challenge'".
26. Court, Emma. "ALS patient group unhappy with how $115 million raised by the Ice Bucket Challenge is being spent". MarketWatch. Retrieved 2018-02-16.

External links

• Official website