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🗺️ Disease Atlas · Nervous system disorder

amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord.

🔔 Follow weekly
🧬6329genes / proteins with evidence
💊185drugs and clinical candidates
🧪12+recruiting trials worldwide
✅5approved
📈25phase 3
🧠

Understand amyotrophic lateral sclerosis

Brain and nerves · Nerve cells that slowly stop working
🎓 Learn more

What it is

Trouble walking or running

Trouble writing

Speech problems

From MedlinePlus: Amyotrophic Lateral Sclerosis (US National Library of Medicine) ↗

🧠 Brain and nerves: In neurodegenerative diseases, nerve cells in the brain or spinal cord stop working and die over time - affecting memory (Alzheimer's disease), movement (Parkinson's disease) or muscles (ALS).

❓ Why it happens

  • Proteins fold the wrong way and clump together inside or between nerve cells - amyloid and tau in Alzheimer's disease, alpha-synuclein in Parkinson's disease.
  • Inflammation, some genes (such as APOE4) and, above all, age raise the risk.

🫀 What it does to the body

  • As connections between nerve cells are lost, memory, thinking, movement or speech fade.
  • The changes start years - often decades - before the first symptoms.

🧱 Why it is hard to cure

  • Most medicines cannot easily pass the blood-brain barrier that protects the brain.
  • By the time symptoms appear, many nerve cells are already lost, and they do not grow back.
  • Many drug trials have failed, partly because the diseases are found late.

🚀 What research is trying now

  • Antibodies that clear amyloid and modestly slow early Alzheimer's disease (approved in some countries).
  • Blood tests that detect Alzheimer's changes years earlier.
  • Gene-targeted medicines, such as one approved in the US for an inherited form of ALS.

General information about brain and nerves, reviewed by people; it does not describe any one person. Below: the evidence for amyotrophic lateral sclerosis itself.

🤖

AI briefing

Where the science on amyotrophic lateral sclerosis stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens amyotrophic lateral sclerosis.

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🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
SOD1 superoxide dismutase 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.89
TARDBP TAR DNA binding proteinPROTAC🧊 AlphaFold structure ↗ 0.85
FUS FUS RNA binding proteinPROTACsmall molecule🧊 AlphaFold structure ↗ 0.84
UBQLN2 ubiquilin 2PROTACantibody🧊 AlphaFold structure ↗ 0.83
TBK1 TANK binding kinase 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.83
SQSTM1 sequestosome 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.82
ANG angiogeninPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.82
TUBA4A tubulin alpha 4aPROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.81
DCTN1 dynactin subunit 1PROTAC🧊 AlphaFold structure ↗ 0.81
VCP valosin containing proteinPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.80
VAPB VAMP associated protein B and CPROTACantibody🧊 AlphaFold structure ↗ 0.80
FIG4 FIG4 phosphoinositide 5-phosphatasePROTACantibody🧊 AlphaFold structure ↗ 0.79
SETX senataxinPROTAC🧊 AlphaFold structure ↗ 0.79
OPTN optineurinPROTACsmall molecule🧊 AlphaFold structure ↗ 0.78
ALS2 alsin Rho guanine nucleotide exchange factor ALS2PROTAC🧊 AlphaFold structure ↗ 0.78

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

💊

Drugs and candidates

Every medicine linked to amyotrophic lateral sclerosis, by how far it has got
Approved · 5
EdaravoneApproved
MecobalaminApproved
RiluzoleSodium channel alpha subunit blockerApproved
Sodium PhenylbutyrateApproved
TofersenSOD1 mRNA antisense inhibitorApproved
In phase 2 / 3 · 20
AbacavirHuman immunodeficiency virus type 1 reverse transcriptase inhibitorPhase 3
ArimoclomolPhase 3
CeftriaxoneBacterial penicillin-binding protein inhibitorPhase 3
CreatinePhase 3
CyanocobalaminPhase 3
DebamestrocelPhase 3
Show all 20
DexpramipexolePhase 3
DextromethorphanSigma opioid receptor agonist; Glutamate [NMDA] receptor subunit epsilon 1 antagonistPhase 3
DolutegravirHuman immunodeficiency virus type 1 integrase inhibitorPhase 3
LamivudineHuman immunodeficiency virus type 1 reverse transcriptase inhibitor; DNA polymerase/rever…Phase 3
LevosimendanPhosphodiesterase 3 inhibitor; Troponin, cardiac muscle positive modulatorPhase 3
Lithium CarbonateInositol-1(or 4)-monophosphatase 1 inhibitor; Glycogen synthase kinase-3 inhibitorPhase 3
MasitinibFibroblast growth factor receptor 3 inhibitor; Stem cell growth factor receptor inhibitorPhase 3
MecaserminInsulin-like growth factor I receptor agonistPhase 3
MinocyclineBacterial 70S ribosome inhibitorPhase 3
NabiximolsPhase 3
OlesoximePhase 3
PridopidineDopamine D2 receptor modulatorPhase 3
QuinidineSodium channel alpha subunit blockerPhase 3
RavulizumabComplement C5 inhibitorPhase 3
Early trials · 20
AcetylcysteinePhase 1/2
Alendronate SodiumFarnesyl diphosphate synthase inhibitorPhase 1/2
Alendronic AcidFarnesyl diphosphate synthase inhibitorPhase 1/2
BaricitinibTyrosine-protein kinase JAK1 inhibitor; Tyrosine-protein kinase JAK2 inhibitorPhase 1/2
BosutinibTyrosine-protein kinase ABL inhibitor; Tyrosine-protein kinase SRC inhibitorPhase 1/2
CopperPhase 1/2
Show all 20
Darbepoetin AlfaErythropoietin receptor agonistPhase 1/2
Dextromethorphan HydrobromideSigma opioid receptor agonist; Glutamate [NMDA] receptor subunit epsilon 1 antagonistPhase 1/2
EnoxacinTopoisomerase IV inhibitor; DNA gyrase inhibitorPhase 1/2
Epoetin AlfaErythropoietin receptor agonistPhase 1/2
Epoetin BetaErythropoietin receptor agonistPhase 1/2
IstradefyllineAdenosine A2a receptor antagonistPhase 1/2
LacosamideSodium channel alpha subunit blockerPhase 1/2
PramipexoleD2-like dopamine receptor agonistPhase 1/2
PterostilbenePhase 1/2
PyrimethamineDihydrofolate reductase inhibitorPhase 1/2
Quinidine SulfateSodium channel alpha subunit blockerPhase 1/2
TamuzimodPhase 1/2
TelberminVascular endothelial growth factor receptor 1 and 2 (Flt-1 and KDR) agonistPhase 1/2
TrametinibDual specificity mitogen-activated protein kinase kinase 2 inhibitor; Dual specificity mi…Phase 1/2
🧪

Recruiting trials

Worldwide, open to new patients now
Controlling Amyotrophic Lateral Sclerosis Motor Neuron Excitability StudyNCT06649955 · NA · 📍 BostonHypercaloric PEG Nutrition in ALS to Sustain Energy HomeostasisNCT06877143 · NA · 📍 UlmInvestigation on the Cortical Communication SystemNCT06207591 · NA · 📍 UtrechtNeurodegenerative Diseases Progression Markers (MARKERS-NDD)NCT06596746 · n/a · 📍 CassinoSpinal Cord Associative Plasticity for ALSNCT06172621 · NA · 📍 The BronxA Study of LTX-002 in Adult Participants With Amyotrophic Lateral SclerosisNCT07660614 · Phase 1, Phase 2 · 📍 LübeckA Registered Cohort Study on Amyotrophic Lateral SclerosisNCT04008329 · n/a · 📍 FuzhouClinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS PatientsNCT05923905 · Phase 4 · 📍 BeijingSafety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy VolunteersNCT05395624 · Phase 1, Phase 2 · 📍 San FranciscoExpression Profile Study of Macrophages From Patients Affected by ALS or Other Related Motor ImpairmentsNCT04816227 · n/a · 📍 ParisBiospecimen Biorepository for the Study of ALS, ALS-FTD and Similar Neurodegenerative DisordersNCT05116943 · n/a · 📍 JacksonvilleIntermuscular Coherence as a Biomarker for ALSNCT05104710 · n/a · 📍 Irvine Search more trials →
📚

Newest research

Latest papers on PubMed
When the Brain Signals Danger: Roles of Calreticulin and HMGB1 in Cell Death Mechanisms in Neurooncology and Neurodegeneration.Biochemistry (Mosc) 2026 · PMID 42927744A CNS-targeted atovaquone nanotherapy mitigates complement activation and neuromuscular degeneration in ALS.Mater Today Bio 2026 · PMID 42926777CCT2 expression marks a translation-activated state in sporadic ALS motor neurons and is associated with functional decline.bioRxiv 2026 · PMID 42926625Constructing functional neural circuits to model amyotrophic lateral sclerosis with induced pluripotent stem cell-derived brain o…Neural Regen Res 2026 · PMID 42925563Unlocking therapeutic potential of Wnt signaling pathway in neurodegenerative diseases.Metab Brain Dis 2026 · PMID 42925434Feasibility and apparent safety of functional electrical stimulation-assisted cycling in amyotrophic lateral sclerosis and primar…Eur J Transl Myol 2026 · PMID 42924853Selected traditional Chinese medicines for treating amyotrophic lateral sclerosis: a Bayesian network meta-analysis based on rand…Front Pharmacol 2026 · PMID 42923269Riluzole-Derived Benzo[d]thiazoles as CK1δ Inhibitors for Amyotrophic Lateral Sclerosis-Related Neurodegeneration.ChemMedChem 2026 · PMID 42922081 Ask a research question in the Hypothesis Lab →
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