Biomarker & Intervention Discovery
Amyotrophic Lateral Sclerosis (ALS)
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
NIH funding$252MFY2025 · RCDC estimate
Spontaneous remissionEssentially zero — uniformly progressive, fatal within 2-5 years median; <10% survive >10 years; spontaneous prolonged…
Best-intervention remissionRiluzole: 3-month median survival extension (modest); edaravone (Radicava): slows functional decline by 33% at 24…
Gap sizeVery large — ALS has no effective treatment; existing drugs provide modest or biomarker-based benefit; curative…
Primary barrierbiological
Full remission profile →
Remission & chronicity
Spontaneous remission
Essentially zero — uniformly progressive, fatal within 2-5 years median; <10% survive >10 years; spontaneous prolonged stabilisation in <1% (SOD1 heterozygous late-onset)
Best-intervention remission
Riluzole: 3-month median survival extension (modest); edaravone (Radicava): slows functional decline by 33% at 24 weeks in a specific ALS subgroup (ALSFRS-R); AMX0035 (sodium phenylbutyrate + taurursodiol): 6.5-month OS extension (CENTAUR trial — modest); tofersen (SOD1-ALS): 38% reduction in neurofilament light (biomarker); 2-year mortality benefit for SOD1-ALS
Gap size
Very large — ALS has no effective treatment; existing drugs provide modest or biomarker-based benefit; curative approaches are distant
Primary barrier
biological
ALS is arguably the most therapeutically challenging neurodegenerative disease; 25+ Phase 3 trials have failed; tofersen (SOD1-targeting antisense oligonucleotide) represents the first mechanism-targeted approach and the most promising signal yet; multigenicity and sporadic forms make uniform treatment impossible
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Amyotrophic Lateral Sclerosis (ALS)” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."