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| Location |
Washington, D.C., USA |
| Type |
Non-profit Research Foundation |
| Website |
als.org |
| Founded |
1974 |
| Focus Areas |
Amyotrophic Lateral Sclerosis (ALS), Motor Neuron Disease, Neurodegeneration |
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, FUS, and TARDBP. Through its Translational Advisory Boards and clinical trial programs, the ALS Association accelerates the development of new treatments for this devastating disease.
- 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
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
Funding research into stem cell therapies for ALS, including:
- Induced pluripotent stem cell (iPSC) models
- Mesenchymal stem cell approaches
- Neural progenitor cell transplantation
| Aspect |
Research Focus |
| Genetics |
C9orf72, SOD1, FUS, TARDBP, ANG |
| Biomarkers |
Neurofilament light chain, p75ECD |
| Therapy |
Gene therapy, small molecules, stem cells |
| Care |
Multidisciplinary clinics, assistive technology |
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]
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]
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
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
The ALS Association provides:
- Wheelchairs and mobility equipment
- Communication devices
- Home modifications
- Respiratory support equipment
The organization offers:
- Support groups for patients and caregivers
- Care navigation services
- Financial assistance programs
- Bereavement support
The ALS Association helps patients access:
- Cutting-edge clinical trials
- Expanded access programs
- Compassionate use pathways
- Trial matching services
¶ Advocacy and Policy
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-level advocacy includes:
- State funding for ALS research
- Insurance coverage mandates
- Patient rights legislation
- Quality of care standards
The Association works with:
- International Alliance for ALS/FTD
- World Federation of Neurology
- Global patient advocacy organizations
¶ Funding and Investment
The ALS Association invests significantly in research:
| Year |
Research Investment |
Grants Funded |
| 2023 |
$25M |
75+ |
| 2022 |
$22M |
70+ |
| 2021 |
$20M |
65+ |
| 2020 |
$18M |
60+ |
- 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
- ALS GTEX: Post-mortem tissue transcriptome database
- ALS Stem Cell Consortium: Standardized stem cell approaches
- ALS Biomarker Initiative: Fluid biomarker validation
The Association has funded research leading to:
- SOD1 gene identification (1993)
- FUS gene mutations (2009)
- C9orf72 expansion discovery (2011)
- TARDBP mutations characterization
Key therapeutic advances:
- Tofersen (Qalsody) FDA approval (2023)
- AMX0035 Phase II/III trial
- Multiple Phase I trials supported
The Association has driven biomarker advances:
- Neurofilament light chain validation
- p75ECD development
- Genetic testing programs
¶ Partnerships and Collaborations
The ALS Association partners with leading institutions:
- Harvard University
- Stanford University
- University of Pennsylvania (Packard Center)
- Columbia University
Collaborations with pharmaceutical companies:
- Biogen (tofersen development)
- Amylyx (AMX0035)
- Roche (clinical trials)
- Multiple biotech companies
- NEALS Consortium (North American Clinical Trials)
- TRICALS (European ALS Research)
- Project MinE (International Genetics)
¶ Training and Career Development
The Association funds training programs:
- Postdoctoral fellowships (3-year)
- Clinical research fellowships
- Young investigator awards
- Conference travel grants
ALS-funded trainees have:
- Established independent laboratories
- Led clinical trials
- Advanced to pharmaceutical positions
- Influenced policy
| Metric |
Value |
| Publications (annual) |
500+ |
| Citations (annual) |
10,000+ |
| Clinical trials supported |
100+ |
| Genes discovered |
10+ |
- Patients served: 20,000+
- Equipment provided: 15,000+
- Support groups: 500+
- Clinical trials enrolled: 5,000+
- Individual donations: 45%
- Corporate partnerships: 25%
- Foundation grants: 20%
- Events (Ice Bucket Challenge, etc.): 10%
- Research: 75%
- Patient services: 15%
- Administration: 10%
The Association is governed by:
- Scientific advisory board
- Patient advisory council
- Industry advisory board
- Chapter leadership
Grant review process:
- Initial triage
- External review (2-3 reviewers)
- study section discussion
- Council recommendation
- Board approval
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
- Precision Medicine: Genetic subtype-specific treatments
- Biomarkers: Surrogate endpoints for faster trials
- Prevention: Pre-symptomatic intervention
- Digital Health: Remote monitoring tools
- Double research investment by 2028
- Support 5 new drug approvals
- Establish international trials network
- Expand patient access globally
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.