Research

Research & Clinical Trials

ALS research includes treatment trials, observational studies, biomarker studies, natural-history studies, genetic research, presymptomatic research, device studies, and expanded-access programs. This section explains how ALS research works and provides a structured clinical-trial finder designed specifically for people affected by ALS.

Research foundation

What ALS Research Needs to Advance

ALS research needs two things at the same time: sustained funding and people willing and eligible to participate. Funding can build research capacity, but funding alone cannot answer questions about what happens in people.

Where ALS Gene-Specific Research Stands

Compare the current verified treatment and research status of selected ALS-associated genes. Statuses describe research records, not individual treatment or trial eligibility.

Verified comparison of selected ALS-associated genes
Gene	FDA-approved gene-specific treatment	Treatment	Current treatment / research status	Penetrance / what is known	Source / last verified
SOD1	Yes	tofersen (Qalsody)	FDA-approved gene-specific treatment; clinical trials underway	Penetrance varies by variant	FDA approves treatment of ALS associated with a mutation in the SOD1 gene ALN-SOD in SOD1-associated ALS — ClinicalTrials.gov ATLAS / tofersen in presymptomatic SOD1 carriers — ClinicalTrials.gov From Mutation to Manifestation: Penetrance in Amyotrophic Lateral Sclerosis Last verified: 10-03-2026 Verification history Previous: Existing source-linked gene-page narrative: QALSODY is approved for SOD1-associated ALS; penetrance varies by variant. New: Structured table row: FDA-approved gene-specific treatment = Yes; treatment = tofersen (Qalsody); research status includes active investigational and presymptomatic SOD1 studies. Status: Verified
C9orf72	No	No FDA-approved gene-specific treatment	Clinical trials underway	Incomplete / variable penetrance; risk differs between families	Metformin in C9orf72 ALS/FTD — ClinicalTrials.gov ACALS / acamprosate in C9orf72 ALS — ClinicalTrials.gov Assessment of risk of ALS conferred by the GGGGCC hexanucleotide repeat expansion in C9orf72 among first-degree relatives of patients with ALS carrying the repeat expansion Personalised penetrance estimation for C9orf72-related amyotrophic lateral sclerosis and frontotemporal dementia Last verified: 10-03-2026 Verification history Previous: Existing source-linked gene-page narrative: no FDA-approved C9orf72-targeting treatment; targeted therapeutic research is ongoing; penetrance is incomplete and variable. New: Structured table row: no FDA-approved gene-specific treatment; active C9orf72-associated investigational study records identified; family-level risk remains variable. Status: Verified
FUS	No	No FDA-approved gene-specific treatment	Clinical trials underway	Incomplete / variable penetrance	FUSION / ION363 in FUS-ALS — ClinicalTrials.gov RAG-21 in FUS-associated ALS — ClinicalTrials.gov Reduced penetrance in genetic ALS/FTD spectrum disorders: implications for genetic counseling, predictive testing and treatment Last verified: 10-03-2026 Verification history Previous: Existing source-linked gene-page narrative: no FDA-approved FUS-targeting treatment; gene-targeted research is ongoing; penetrance varies by variant. New: Structured table row: no FDA-approved gene-specific treatment; FUS-associated investigational records include an active Phase 3 record and an early-phase record. Status: Verified
TARDBP	No	No FDA-approved gene-specific treatment	Early clinical development	Incomplete / variable penetrance	Personalized antisense oligonucleotide therapy for TARDBP ALS — ClinicalTrials.gov Reduced penetrance in genetic ALS/FTD spectrum disorders: implications for genetic counseling, predictive testing and treatment From Mutation to Manifestation: Penetrance in Amyotrophic Lateral Sclerosis Last verified: 10-03-2026 Verification history Previous: Existing source-linked gene-page narrative: no FDA-approved TARDBP-targeting therapy; variant-specific research is ongoing; penetrance is incompletely characterized. New: Structured table row: no FDA-approved gene-specific treatment; early-phase personalized TARDBP antisense study record identified. Status: Verified
TBK1	No	No FDA-approved gene-specific treatment	Observational research only; no active gene-specific therapeutic trial identified	Incomplete / variable penetrance	ALLFTD longitudinal study — ClinicalTrials.gov Reduced penetrance in genetic ALS/FTD spectrum disorders: implications for genetic counseling, predictive testing and treatment From Mutation to Manifestation: Penetrance in Amyotrophic Lateral Sclerosis Last verified: 10-03-2026 Verification history Previous: Existing source-linked gene-page narrative: no FDA-approved TBK1-targeting treatment; ALS/FTD mechanism and variant research is ongoing; penetrance is reduced and age-dependent. New: Structured table row: no FDA-approved gene-specific treatment; the current verified TBK1 record is observational ALS/FTD research rather than a therapeutic trial. Status: Verified

Last verified: 10-03-2026

The table links to authoritative treatment, clinical-trial, and specialist literature sources. A gene without an approved treatment may still have active clinical, observational, biomarker, or natural-history research.

Sustained research funding

Funding helps support laboratory research, staff and investigators, sequencing and genetic analysis, biomarker development, trial infrastructure, participant recruitment, long-term follow-up, data collection, and the manufacturing and testing of experimental therapies.

Sources: Research Projects

People willing and eligible to participate

Human research depends on volunteers. Researchers may need participants for clinical trials, natural-history studies, observational studies, biomarker studies, genetic studies, longitudinal studies, presymptomatic studies, gene-specific research, and prevention or interception research.

Funding alone cannot test an intervention in people, validate a biomarker against clinical outcomes, or show how disease changes over time. Eligibility is study-specific, and participation is voluntary; joining a study does not guarantee personal benefit or faster treatment development.

Clinical trials
Natural-history studies
Observational studies
Biomarker studies
Genetic studies
Longitudinal studies
Presymptomatic studies
Gene-specific research
Prevention or interception research

Sources: NIH Clinical Research Trials You: The Basics · Learn About Studies · Why Awareness of Clinical Research Matters · Amyotrophic Lateral Sclerosis (ALS)

Build your research literacy

Learn About ALS Research

Understand study types, trial phases, placebo, randomization, endpoints, biomarkers, results, expanded access, and Right to Try.

Learn the basics

Search the multi-source registry

Find ALS Research Opportunities

Browse ClinicalTrials.gov records alongside verified academic-center studies that may not have an NCT number.

Find a study

Explore first-party sources

Research Centers

See verified ALS-center research pages, local study information, and center-specific contacts.

View centers

1240Unique opportunities

1235ClinicalTrials.gov records

5Institution-only studies

221Recruiting

4Needs confirmation

42Presymptomatic studies

200Genetic studies

49C9orf72 studies

76Georgia opportunities

Sources & Further Reading

These source cards link directly to the original material. External websites open in a new tab.

ClinicalTrials.gov / National Library of Medicine

Learn About Studies

Official plain-language information about clinical studies, participation, and study records.