ALS Association

Last updated
ALS Association
AbbreviationThe ALS Association
Founded1985;39 years ago (1985)
Type Non-profit
Focusadvocacy and patient services
Location
Area served
United States
President and CEO
Calaneet Balas
Chairman
 Scott Kauffman
Revenue
$37 million (2019) [1]
Website als.org

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. [2]

Contents

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]

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]

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. [21] [22]

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. [23] 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. [24] 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. [25]

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 in cities across the United States. Since its inception in 2000, this event has raised more than $265 million. [26]

Related Research Articles

Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. Several ASOs have been approved in the United States, the European Union, and elsewhere.

Primary lateral sclerosis (PLS) is a very rare neuromuscular disease characterized by progressive muscle weakness in the voluntary muscles. PLS belongs to a group of disorders known as motor neuron diseases. Motor neuron diseases develop when the nerve cells that control voluntary muscle movement degenerate and die, causing weakness in the muscles they control.

<span class="mw-page-title-main">Neurodegenerative disease</span> Central nervous system disease

A neurodegenerative disease is caused by the progressive loss of neurons, in the process known as neurodegeneration. Neuronal damage may also ultimately result in their death. Neurodegenerative diseases include amyotrophic lateral sclerosis, multiple sclerosis, Parkinson's disease, Alzheimer's disease, Huntington's disease, multiple system atrophy, tauopathies, and prion diseases. Neurodegeneration can be found in the brain at many different levels of neuronal circuitry, ranging from molecular to systemic. Because there is no known way to reverse the progressive degeneration of neurons, these diseases are considered to be incurable; however research has shown that the two major contributing factors to neurodegeneration are oxidative stress and inflammation. Biomedical research has revealed many similarities between these diseases at the subcellular level, including atypical protein assemblies and induced cell death. These similarities suggest that therapeutic advances against one neurodegenerative disease might ameliorate other diseases as well.

Progressive bulbar palsy (PBP) is a medical condition. It belongs to a group of disorders known as motor neuron diseases. PBP is a disease that attacks the nerves supplying the bulbar muscles. These disorders are characterized by the degeneration of motor neurons in the cerebral cortex, spinal cord, brain stem, and pyramidal tracts. This specifically involves the glossopharyngeal nerve (IX), vagus nerve (X), and hypoglossal nerve (XII).

Mecasermin rinfabate, also known as rhIGF-1/rhIGFBP-3, is a drug consisting of recombinant human insulin-like growth factor 1 (IGF-1) and recombinant human insulin-like growth factor binding protein-3 (IGFBP-3) which is used for the treatment of amyotrophic lateral sclerosis.

<span class="mw-page-title-main">ALS Therapy Development Institute</span>

The ALS Therapy Development Institute is a non-profit biotechnology research organization focused on finding treatments for amyotrophic lateral sclerosis (ALS). With a staff including more than 30 scientists, it operates a research and development program centered on ALS.

<span class="mw-page-title-main">SOD1</span> Protein-coding gene in the species Homo sapiens

Superoxide dismutase [Cu-Zn] also known as superoxide dismutase 1 or hSod1 is an enzyme that in humans is encoded by the SOD1 gene, located on chromosome 21. SOD1 is one of three human superoxide dismutases. It is implicated in apoptosis, familial amyotrophic lateral sclerosis and Parkinson's disease.

<span class="mw-page-title-main">KIF5A</span> Protein-coding gene in humans

Kinesin family member 5A is a protein that in humans is encoded by the KIF5A gene. It is part of the kinesin family of motor proteins.

<span class="mw-page-title-main">ALS</span> Rare neurodegenerative disease

Amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND) or Lou Gehrig's disease in the United States, is a rare, terminal neurodegenerative disorder that results in the progressive loss of both upper and lower motor neurons that normally control voluntary muscle contraction. ALS is the most common form of the motor neuron diseases. ALS often presents in its early stages with gradual muscle stiffness, twitches, weakness, and wasting. Motor neuron loss typically continues until the abilities to eat, speak, move, and, lastly, breathe are all lost. While only 15% of people with ALS also fully develop frontotemporal dementia, an estimated 50% face at least some minor difficulties with thinking and behavior. Depending on which of the aforementioned symptoms develops first, ALS is classified as limb-onset or bulbar-onset.

Pridopidine is an orally administrated small molecule investigational drug. Pridopidine is a selective and potent Sigma-1 Receptor agonist. It is being developed by Prilenia Therapeutics and is currently in late-stage clinical development for Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS).

Ozanezumab is a monoclonal antibody designed for the treatment of ALS and multiple sclerosis.

<span class="mw-page-title-main">C9orf72</span> Protein-coding gene in humans

C9orf72 is a protein which in humans is encoded by the gene C9orf72.

Project MinE is an independent large scale whole genome research project that was initiated by 2 patients with amyotrophic lateral sclerosis and started on World ALS Day, June 21, 2013.

<span class="mw-page-title-main">Les Turner ALS Foundation</span>

The Les Turner ALS Foundation is a non-profit organization based in Chicago that provides amyotrophic lateral sclerosis (ALS) patient services; supports events, education and awareness about ALS; and funds ALS research. Since it was founded, it has raised over $64 million.

There are more than 25 genes known to be associated with amyotrophic lateral sclerosis (ALS) as of June 2018, which collectively account for about 70% of cases of familial ALS (fALS) and 10% of cases of sporadic ALS (sALS). About 5–10% of cases of ALS are directly inherited. Overall, first-degree relatives of an individual with ALS have a 1% risk of developing ALS. ALS has an oligogenic mode of inheritance, meaning that mutations in two or more genes are required to cause disease.

Research on amyotrophic lateral sclerosis (ALS) has focused on animal models of the disease, its mechanisms, ways to diagnose and track it, and treatments.

Bryan J. Traynor is a neurologist and a senior investigator at the National Institute on Aging, and an adjunct professor at Johns Hopkins University. Dr. Traynor studies the genetics of human neurological conditions such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). He led the international consortium that identified pathogenic repeat expansions in the C9orf72 gene as a common cause of ALS and FTD. Dr. Traynor also led efforts that identified other Mendelian genes responsible for familial ALS and dementia, including VCP, MATR3, KIF5A, HTT, and SPTLC1.

Merit Cudkowicz is an American neurologist and neuroscientist who studies amyotrophic lateral sclerosis (ALS). Cudkowicz is Julieanne Dorn Professor of Neurology at Harvard Medical School, director of the ALS clinic and the Neurological Clinical Research Institute at Massachusetts General Hospital (MGH), and chair of the Department of Neurology at MGH. Cudkowicz has led several large-scale collaborations and clinical trials to test novel treatments for ALS and as of 2020, researching ways to detect early biomarkers of ALS to improve diagnosis.

Amylyx Pharmaceuticals, Inc is a biopharmaceutical company headquartered in Cambridge, Massachusetts. Amylyx is best known for AMX0035, an experimental therapy for amyotrophic lateral sclerosis. AMX0035 was approved for medical use in Canada as Albrioza, in June 2022, and in the United States, as Relyvrio, in September 2022. It was withdrawn in April, 2024.

<span class="mw-page-title-main">Tofersen</span> Medication

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with ALS. It is administered as an intrathecal injection.

References

  1. ↑ "ALSA Form 990" (PDF). Retrieved 11 October 2019.
  2. ↑ "5 Important Things the ALS Association Does". January 16, 2018.
  3. ↑ "ALS Association Invests $3M in Platform Trial to Accelerate Development of Treatments". 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. ↑ "Our Partners". Target ALS. Archived from the original on 2018-02-17. Retrieved 2018-02-16.
  6. 1 2 3 4 5 "5 years later, man with ALS who popularized Ice Bucket Challenge still inspires". Today. 19 July 2019. Retrieved 2019-11-06.
  7. 1 2 3 4 5 Strub, Chris. "Ice Bucket Challenge Boosted ALS Association Annual Funding By 187%: Report". Forbes. Retrieved 2019-11-06.
  8. 1 2 3 4 "Cold Cash: What to Do When Unexpected Money Pours In". The Chronicle of Philanthropy. 2019-07-17. Retrieved 2019-11-12.
  9. 1 2 "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. 1 2 "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. 1 2 3 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. 1 2 3 "Ice Bucket Challenge Greatly Aided ALS Research, Study Reports". 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 . PMC   9134321 . 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. ↑ "The Ice Bucket Challenge and the promise — and the pitfalls — of viral charity". Vox. 20 July 2019.
  22. ↑ "Ice bucket challenge: Where did the money go?". NBC News. 31 August 2019.
  23. ↑ RTI International (May 2019). Evaluation of the ALS Association Grant Programs (PDF) (Report).
  24. ↑ Ohlheiser, Abby (August 30, 2014). "ALS Association withdraws controversial applications to trademark 'ice bucket challenge'".
  25. ↑ Court, Emma. "ALS patient group unhappy with how $115 million raised by the Ice Bucket Challenge is being spent". MarketWatch. Retrieved 2018-02-16.
  26. ↑ "5 Important Things the ALS Association Does". ALS News Today. 16 January 2018. Archived from the original on 3 February 2020. Retrieved 3 February 2020.
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