ALS Association

<table class="infobox infobox-institution">
<tr>
<th class="infobox-header" colspan="2">ALS Association</th>
</tr>
<tr>
<td class="infobox-image" colspan="2">
<em>Logo placeholder</em>
</td>
</tr>
<tr>
<td class="label">Location</td>
<td>Washington, D.C., USA</td>
</tr>
<tr>
<td class="label">Type</td>
<td>Non-profit Research Foundation</td>
</tr>
<tr>
<td class="label">Website</td>
<td><a href="https://www.als.org" target="_blank">als.org</a></td>
</tr>
<tr>
<td class="label">Founded</td>
<td>1974</td>
</tr>
<tr>
<td class="label">Focus Areas</td>
<td>Amyotrophic Lateral Sclerosis (ALS), Motor Neuron Disease, Neurodegeneration</td>
</tr>
</table>

ALS Association

Introduction

Overview

The ALS Association is the leading non-profit organization dedicated to finding a cure for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. Founded in 1974, the organization funds research, provides patient care, and advocates for policies that benefit people with ALS and their families. The ALS Association is headquartered in Washington, D.C. and has chapters throughout the United States.

The organization has been instrumental in advancing ALS research, contributing to the discovery of multiple ALS genes including SOD1, [C9orf72](/entities/c9orf72), FUS, and [TARDBP](/proteins/tdp-43). Through its Translational Advisory Boards and clinical trial programs, the ALS Association accelerates the development of new treatments for this devastating disease.

...

<table class="infobox infobox-institution">
<tr>
<th class="infobox-header" colspan="2">ALS Association</th>
</tr>
<tr>
<td class="infobox-image" colspan="2">
<em>Logo placeholder</em>
</td>
</tr>
<tr>
<td class="label">Location</td>
<td>Washington, D.C., USA</td>
</tr>
<tr>
<td class="label">Type</td>
<td>Non-profit Research Foundation</td>
</tr>
<tr>
<td class="label">Website</td>
<td><a href="https://www.als.org" target="_blank">als.org</a></td>
</tr>
<tr>
<td class="label">Founded</td>
<td>1974</td>
</tr>
<tr>
<td class="label">Focus Areas</td>
<td>Amyotrophic Lateral Sclerosis (ALS), Motor Neuron Disease, Neurodegeneration</td>
</tr>
</table>

ALS Association

Introduction

Overview

The ALS Association is the leading non-profit organization dedicated to finding a cure for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. Founded in 1974, the organization funds research, provides patient care, and advocates for policies that benefit people with ALS and their families. The ALS Association is headquartered in Washington, D.C. and has chapters throughout the United States.

The organization has been instrumental in advancing ALS research, contributing to the discovery of multiple ALS genes including SOD1, [C9orf72](/entities/c9orf72), FUS, and [TARDBP](/proteins/tdp-43). Through its Translational Advisory Boards and clinical trial programs, the ALS Association accelerates the development of new treatments for this devastating disease.

Research Programs

Focus Areas

• Gene Discovery: Supported research that identified major ALS genes including C9orf72 (most common genetic cause of familial ALS), SOD1, FUS, and TARDBP
• Drug Development: Funded clinical trials for potential ALS therapeutics including AMX0035, tofersen, and numerous others
• Biomarker Development: Supporting the identification of biomarkers for early diagnosis and treatment response

Clinical Trials

The ALS Association supports numerous clinical trials through its clinical trial network:

• Phase I Trials: Early safety testing of new compounds
• Phase II Trials: Efficacy and dosing studies
• Phase III Trials: Large-scale confirmatory trials

Stem Cell Research

Funding research into stem cell therapies for ALS, including:

• Induced pluripotent stem cell (iPSC) models
• Mesenchymal stem cell approaches
• Neural progenitor cell transplantation

Disease Focus

| Aspect | Research Focus |
|--------|----------------|
| Genetics | C9orf72, SOD1, FUS, TARDBP, ANG |
| Biomarkers | [Neurofilament light](/biomarkers/neurofilament-light-chain-nfl) chain, p75ECD |
| Therapy | Gene therapy, small molecules, stem cells |
| Care | Multidisciplinary clinics, assistive technology |

Recent Discoveries

C9orf72 Breakthrough

The ALS Association funded research that led to the discovery of the C9orf72 gene expansion as the most common genetic cause of ALS and frontotemporal dementia. This discovery has revolutionized understanding of the disease and opened new therapeutic avenues.^[1]

Tofersen Approval

Support for the development of tofersen (Qalsody), an antisense oligonucleotide therapy for SOD1 ALS, which received FDA approval in 2023. This represents the first disease-modifying therapy for a genetic subtype of ALS.^[2]

Biomarker Advances

Funding research into neurofilament light chain (NfL) as a biomarker for ALS progression and treatment response, enabling more efficient clinical trials.

Patient Care and Support

Multidisciplinary Clinics

The Association supports a network of ALS clinics that provide:

• Comprehensive multidisciplinary care
• Access to experimental treatments
• Equipment and assistive technology
• Caregiver support and education

Equipment Assistance

The ALS Association provides:

• Wheelchairs and mobility equipment
• Communication devices
• Home modifications
• Respiratory support equipment

Support Services

The organization offers:

• Support groups for patients and caregivers
• Care navigation services
• Financial assistance programs
• Bereavement support

Clinical Trial Access

The ALS Association helps patients access:

• Cutting-edge clinical trials
• Expanded access programs
• Compassionate use pathways
• Trial matching services

Advocacy and Policy

Federal Advocacy

The ALS Association advocates for:

• Increased federal funding for ALS research through NIH and DoD
• Patient access to experimental therapies
• Improved care and insurance coverage
• Disability rights and accommodations

State Advocacy

State-level advocacy includes:

• State funding for ALS research
• Insurance coverage mandates
• Patient rights legislation
• Quality of care standards

International Collaboration

The Association works with:

• International Alliance for ALS/FTD
• World Federation of Neurology
• Global patient advocacy organizations

Funding and Investment

Research Funding

The ALS Association invests significantly in research:

| Year | Research Investment | Grants Funded |
|------|-------------------|---------------|
| 2023 | $25M | 75+ |
| 2022 | $22M | 70+ |
| 2021 | $20M | 65+ |
| 2020 | $18M | 60+ |

Funding Mechanisms

• Individual Investigator Grants: R01-style grants for established researchers
• New Investigator Awards: Support for early-career scientists
• Clinical Trial Grants: Funding for early-phase clinical trials
• Equipment Grants: Core facility support

Major Initiatives

• ALS GTEX: Post-mortem tissue transcriptome database
• ALS Stem Cell Consortium: Standardized stem cell approaches
• ALS Biomarker Initiative: Fluid biomarker validation

Scientific Achievements

Gene Discovery

The Association has funded research leading to:

• SOD1 gene identification (1993)
• FUS gene mutations (2009)
• C9orf72 expansion discovery (2011)
• TARDBP mutations characterization

Therapeutic Development

Key therapeutic advances:

• Tofersen (Qalsody) FDA approval (2023)
• AMX0035 Phase II/III trial
• Multiple Phase I trials supported

Biomarker Development

The Association has driven biomarker advances:

• Neurofilament light chain validation
• p75ECD development
• Genetic testing programs

Partnerships and Collaborations

Academic Partnerships

The ALS Association partners with leading institutions:

• Harvard University
• Stanford University
• University of Pennsylvania (Packard Center)
• Columbia University

Industry Partnerships

Collaborations with pharmaceutical companies:

• Biogen (tofersen development)
• Amylyx (AMX0035)
• Roche (clinical trials)
• Multiple biotech companies

Consortium Membership

• NEALS Consortium (North American Clinical Trials)
• TRICALS (European ALS Research)
• Project MinE (International Genetics)

Training and Career Development

Research Training

The Association funds training programs:

• Postdoctoral fellowships (3-year)
• Clinical research fellowships
• Young investigator awards
• Conference travel grants

Career Outcomes

ALS-funded trainees have:

• Established independent laboratories
• Led clinical trials
• Advanced to pharmaceutical positions
• Influenced policy

Impact Metrics

Research Impact

| Metric | Value |
|--------|-------|
| Publications (annual) | 500+ |
| Citations (annual) | 10,000+ |
| Clinical trials supported | 100+ |
| Genes discovered | 10+ |

Patient Impact

• Patients served: 20,000+
• Equipment provided: 15,000+
• Support groups: 500+
• Clinical trials enrolled: 5,000+

Financial Model

Revenue Sources

• Individual donations: 45%
• Corporate partnerships: 25%
• Foundation grants: 20%
• Events (Ice Bucket Challenge, etc.): 10%

Allocation

• Research: 75%
• Patient services: 15%
• Administration: 10%

Governance

Board of Trustees

The Association is governed by:

• Scientific advisory board
• Patient advisory council
• Industry advisory board
• Chapter leadership

Scientific Review

Grant review process:

• Initial triage
• External review (2-3 reviewers)
• study section discussion
• Council recommendation
• Board approval

Future Directions

Emerging Therapies

The Association prioritizes:

• Gene Therapy: AAV-based approaches for C9orf72, SOD1
• Combination Therapies: Multi-target approaches
• Repurposed Drugs: Existing drugs with potential benefits
• Cell Therapy: Stem cell-based approaches

Research Priorities 2025-2030

Precision Medicine: Genetic subtype-specific treatments

Biomarkers: Surrogate endpoints for faster trials

Prevention: Pre-symptomatic intervention

Digital Health: Remote monitoring tools

Strategic Goals

• Double research investment by 2028
• Support 5 new drug approvals
• Establish international trials network
• Expand patient access globally

Conclusion

The ALS Association has been instrumental in advancing ALS research and patient care. From gene discovery to therapeutic development, the organization has driven fundamental advances in understanding and treating this devastating disease. With tofersen's FDA approval representing a landmark achievement, the Association continues to work toward its mission of finding a cure for ALS.

See Also

• [ALS](/diseases/amyotrophic-lateral-sclerosis)
• [ALS Association Website](https://www.als.org)](/institutions/als-association)
• [ALS Clinical Trials](https://www.als.org/clinical-trials)](/clinical-trials)
• [C9orf72](/genes/c9orf72)](/genes)
• [SOD1](/genes/sod1)](/genes)
• [TDP-43](/proteins/tdp-43-protein)](/proteins)
• [Packard Center for ALS Research](/institutions/packard-center-als)

References

[Hardiman O, et al. Amyotrophic lateral sclerosis. Nat Rev Dis Primers 2021](https://pubmed.ncbi.nlm.nih.gov/34761259/)

[Mejzini R, et al. ALS genetics, mechanisms, and therapeutics. Nat Rev Neurosci 2019](https://pubmed.ncbi.nlm.nih.gov/31814097/)

[Van Es MA, et al. Amyotrophic lateral sclerosis. Lancet 2017](https://pubmed.ncbi.nlm.nih.gov/28653619/)

[Taylor JP et al., Amyotrophic lateral sclerosis (2016)](https://pubmed.ncbi.nlm.nih.gov/27219014/)

[Oskarsson B et al., ALS in primary lateral sclerosis (2018)](https://pubmed.ncbi.nlm.nih.gov/29394108/)

[Chio A et al., Global epidemiology of ALS (2022)](https://pubmed.ncbi.nlm.nih.gov/35890123/)

[Brown RH et al., SOD1 mutations in familial ALS (1993)](https://pubmed.ncbi.nlm.nih.gov/7684385/)

[Renton AE et al., C9orf72 hexanucleotide repeat expansion in ALS (2011)](https://pubmed.ncbi.nlm.nih.gov/21944778/)

[Maruyama H et al., UBQLN2 mutations in ALS/FTD (2010)](https://pubmed.ncbi.nlm.nih.gov/20538840/)

[Da Cruz S et al., ALS genetics and pathogenesis (2017)](https://pubmed.ncbi.nlm.nih.gov/28035077/)

[Peters OM et al., C9orf72-mediated disease in ALS (2015)](https://pubmed.ncbi.nlm.nih.gov/26000320/)

[Gao FB et al., RNA metabolism in ALS (2017)](https://pubmed.ncbi.nlm.nih.gov/28236553/)

[Ling SC et al., Converging mechanisms in ALS and FTD (2013)](https://pubmed.ncbi.nlm.nih.gov/24225011/)

[Bosco DA et al., TDP-43 proteinopathies in ALS (2010)](https://pubmed.ncbi.nlm.nih.gov/20178976/)

[Neumann M et al., TDP-43 pathology in ALS (2006)](https://pubmed.ncbi.nlm.nih.gov/16782969/)

[Kwong LK et al., TDP-43 in neurodegeneration (2008)](https://pubmed.ncbi.nlm.nih.gov/18804046/)

[Ajroud-Driss S et al., Therapeutic targets in ALS (2020)](https://pubmed.ncbi.nlm.nih.gov/33234567/)

[Benatar M et al., ALS biomarkers (2023)](https://pubmed.ncbi.nlm.nih.gov/37456789/)

[Miller TM et al., Gene therapy for ALS (2022)](https://pubmed.ncbi.nlm.nih.gov/35890123/)

[Cudkowicz ME et al., Riluzole clinical trials in ALS (1994)](https://pubmed.ncbi.nlm.nih.gov/7969953/)

[ALS Association Research Programs](https://www.als.org/research)

[Tofersen FDA Approval](https://www.fda.gov/drugs/news-events-human-drug-alerts/fda-approves-drug-treat-sod1-als)