Disease overview

Alterations

168

Molecular (A)

100

Clinical (B–E)

Direct drugs

190

Via biomarker

118

Natural agents

Natural products

Merged ranked

305

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, GreenMedInfo · Pipeline phase 7 · Updated 2026-07-06

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

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Very large — ALS has no effective treatment; existing drugs provide modest or biomarker-based benefit; curative approaches are distant

Primary barrier

biological

Barrier detail: 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

Last SoC change: 2023 — tofersen (Qalsody) FDA approved for SOD1-ALS based on neurofilament biomarker; AMX0035 (Relyvrio) approved 2022 — subsequently voluntary market withdrawal 2024 after Phase 3 PHOENIX trial failure; edaravone 2017; riluzole 1995

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0004976 · ORPHA: ORPHA:300605

Alterations

Showing 80 of 168 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal lower motor neuron morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal pyramidal sign	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal pyramidal tract morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal upper motor neuron morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Amyotrophic lateral sclerosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ankle clonus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Apathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Areflexia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Aspiration	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Babinski sign	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bowing of the vocal cords	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bradykinesia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bulbar palsy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bulbar signs	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Centrally nucleated skeletal muscle fibers	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cerebral cortical atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cognitive impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased nerve conduction velocity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Degeneration of anterior horn cells	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Degeneration of the lateral corticospinal tracts	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dementia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Disinhibition	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Distal amyotrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Distal lower limb muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Distal muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Dysarthria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dysphagia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating creatine kinase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Fasciculations	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Frontotemporal dementia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gait disturbance	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hand muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Hoarse voice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyperreflexia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyporeflexia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Increased variability in muscle fiber diameter	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Language impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Limb muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Loss of ambulation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Muscle spasm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Mutism	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Neuronal loss in central nervous system	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Personality changes	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Postural tremor	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Progressive muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Proximal amyotrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Proximal lower limb muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Proximal muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Proximal upper limb muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Pseudobulbar paralysis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Respiratory insufficiency due to muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Rimmed vacuoles	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Shoulder girdle muscle weakness	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Skeletal muscle atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sleep apnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Spasticity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Tetraparesis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
AGTPBP1 ATP/GTP binding carboxypeptidase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AKAP12 A-kinase anchoring protein 12	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALDH1A3 aldehyde dehydrogenase 1 family member A3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALS Functional Rating Scale	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
ALS2 alsin Rho guanine nucleotide exchange factor ALS2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ANG angiogenin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ANXA11 annexin A11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ATXN2 ataxin 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Absence of adverse events	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
C9orf72 C9orf72-SMCR8 complex subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCNF cyclin F	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CCSAP centriole, cilia and spindle associated protein	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CFAP410 cilia and flagella associated protein 410	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CHCHD10 coiled-coil-helix-coiled-coil-helix domain containing 10	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CHMP2B charged multivesicular body protein 2B	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CHRNA3 cholinergic receptor nicotinic alpha 3 subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DAO D-amino acid oxidase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DCTN1 dynactin subunit 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DNAJC7 DnaJ heat shock protein family (Hsp40) member C7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DRD2 dopamine receptor D2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERBB4 erb-b2 receptor tyrosine kinase 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERLIN1 ER lipid raft associated 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
CIPROFLOXACIN	Small molecule	Approved	Strong	100	3 trials 2 Trial pubs 2 RCT View evidence Registry trials NCT04090684 — Ciprofloxacin/Celecoxib Combination in Patients With ALS (COMPLETED) PHASE1 NCT05357950 — A Phase IIb, Multi-Center, Multinational, Double-Blind, Placebo-Controlled Study, With an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in Subjects With ALS (COMPLETED) PHASE2 NCT04165850 — Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
SIROLIMUS	Small molecule	Approved	Strong	100	1 trials View evidence Registry trials NCT03359538 — Rapamycin Treatment for ALS (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS	Small molecule	Approved	Strong	100	2 trials View evidence Registry trials NCT04302870 — Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial (RECRUITING) PHASE2, PHASE3 NCT01884571 — Immunosuppression in Amyotrophic Lateral Sclerosis (ALS) (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMSACRINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APOMORPHINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENZOIC ACID Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BEVACIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CHLORHEXIDINE GLUCONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CHLORPROMAZINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CLEMASTINE FUMARATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
COCAINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	1 trials 2 RCT View evidence Registry trials NCT07539662 — Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS) (ENROLLING_BY_INVITATION) NA Published literature Randomized controlled trial Effects of cyclosporine therapy on plasma lipoprotein levels (JAMA, 1989) Randomized controlled trial A double-blind study of the effectiveness of cyclosporine in amyotrophic lateral sclerosis (Archives of neurology, 1988) CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ECONAZOLE NITRATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ECULIZUMAB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EFGARTIGIMOD ALFA Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPIRUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FENRETINIDE Repurposing	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOXETINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
IDARUBICIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IDARUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
IMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAPROTILINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
MASOPROCOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
MECAMYLAMINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
MECAMYLAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
MEFLOQUINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
METHAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHIXENE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
METHOXSALEN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MITOXANTRONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Sources	Links
ABACAVIR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ARIMOCLOMOL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CEFTRIAXONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CREATINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CYANOCOBALAMIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DEBAMESTROCEL	Cell therapy	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DEXPRAMIPEXOLE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DEXTROMETHORPHAN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DOLUTEGRAVIR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LAMIVUDINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LEVOSIMENDAN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LITHIUM CARBONATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MASITINIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MECASERMIN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MINOCYCLINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
NABIXIMOLS	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
OLESOXIME	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PRIDOPIDINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
QUINIDINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RAVULIZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RELDESEMTIV	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TAUROLITHOCHOLIC ACID	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TIRASEMTIV	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ULEFNERSEN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
VALPROIC ACID	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ABATACEPT	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ACETYLCARNITINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALBUMIN HUMAN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALBUTEROL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALDESLEUKIN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMANTADINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMBROXOL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ANAKINRA	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ANX-005	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
APILIMOD MESYLATE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ARGX-119	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ARUNDIC ACID	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ASTAXANTHIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ATROPINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ATSM COPPER (II)	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMPHETAMINE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMSACRINE Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	—
APOMORPHINE HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENZOIC ACID Repurposing via DAO	Small molecule	Approved	Strong	95	—	DGIdb	—
BEVACIZUMAB Repurposing via ANXA11	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CHLORHEXIDINE GLUCONATE Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	—
CHLORPROMAZINE HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CIPROFLOXACIN Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	3 trials 2 Trial pubs 2 RCT View evidence Registry trials NCT04090684 — Ciprofloxacin/Celecoxib Combination in Patients With ALS (COMPLETED) PHASE1 NCT05357950 — A Phase IIb, Multi-Center, Multinational, Double-Blind, Placebo-Controlled Study, With an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in Subjects With ALS (COMPLETED) PHASE2 NCT04165850 — Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
CLEMASTINE FUMARATE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
CLOMIPRAMINE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing via ATXN2	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
COCAINE Repurposing via CHRNA3	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing via ATXN2	Small molecule	Approved	Strong	95	1 trials 2 RCT View evidence Registry trials NCT07539662 — Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS) (ENROLLING_BY_INVITATION) NA Published literature Randomized controlled trial Effects of cyclosporine therapy on plasma lipoprotein levels (JAMA, 1989) Randomized controlled trial A double-blind study of the effectiveness of cyclosporine in amyotrophic lateral sclerosis (Archives of neurology, 1988) CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOXORUBICIN HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ECONAZOLE NITRATE Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ECULIZUMAB Repurposing via CHRNA3	Small molecule	Approved	Strong	95	—	DGIdb	—
EFGARTIGIMOD ALFA Repurposing via CHRNA3	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPIRUBICIN HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FENRETINIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOXETINE HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
IDARUBICIN Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IDARUBICIN HYDROCHLORIDE Repurposing via ATXN2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
IMIPRAMINE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAPROTILINE Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	—
MASOPROCOL Repurposing via ATXN2	Small molecule	Approved	Strong	95	—	DGIdb	—
MECAMYLAMINE Repurposing via CHRNA3	Small molecule	Approved	Strong	95	—	DGIdb	—
MECAMYLAMINE Repurposing via AGTPBP1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets

Natural Products

79 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked)

Reference lookups: GreenMedInfo · Examine.com

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Creatine Natural product Cochrane review Serum creatinine levels in patients with amyotrophic lateral sclerosis:… Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease Cochrane review Treatment for cramps in amyotrophic lateral sclerosis/motor neuron dise…	Amino acid	Phase III	Moderate	17	8 trials 4 Cochrane 6 Meta-analysis 22 Trial pubs 9 RCT View evidence Registry trials NCT00023075 — Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis (COMPLETED) NCT02709330 — ALS Reversals - Lunasin Regimen (COMPLETED) PHASE2 NCT00070993 — Creatine for the Treatment of Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00005766 — Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00005674 — Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis [ALS] (COMPLETED) PHASE2 NCT01257581 — Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS) (COMPLETED) PHASE2 NCT00355576 — Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00069186 — Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis (UNKNOWN) PHASE3 Published literature Cochrane review Serum creatinine levels in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis (Amyotrophic lateral sclerosis & frontotemporal degeneration, 2020) Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2012) Cochrane review Treatment for cramps in amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2012) Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2010) Randomized controlled trial A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results (Journal of neurology, 2012) Randomized controlled trial Effect of nutritional supplementation with milk whey proteins in amyotrophic lateral sclerosis patients (Arquivos de neuro-psiquiatria, 2010) CT.gov search · Cochrane · PubMed	Examine.com, GreenMedInfo	PubChem GreenMedInfo Examine.com
Creatine Natural product Cochrane review Serum creatinine levels in patients with amyotrophic lateral sclerosis:… Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease Cochrane review Treatment for cramps in amyotrophic lateral sclerosis/motor neuron dise…	Amino acid	Phase III	Emerging	16	8 trials 4 Cochrane 6 Meta-analysis 22 Trial pubs 9 RCT View evidence Registry trials NCT00023075 — Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis (COMPLETED) NCT02709330 — ALS Reversals - Lunasin Regimen (COMPLETED) PHASE2 NCT00070993 — Creatine for the Treatment of Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00005766 — Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00005674 — Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis [ALS] (COMPLETED) PHASE2 NCT01257581 — Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS) (COMPLETED) PHASE2 NCT00355576 — Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 NCT00069186 — Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis (UNKNOWN) PHASE3 Published literature Cochrane review Serum creatinine levels in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis (Amyotrophic lateral sclerosis & frontotemporal degeneration, 2020) Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2012) Cochrane review Treatment for cramps in amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2012) Cochrane review Creatine for amyotrophic lateral sclerosis/motor neuron disease (The Cochrane database of systematic reviews, 2010) Randomized controlled trial A randomized controlled clinical trial of growth hormone in amyotrophic lateral sclerosis: clinical, neuroimaging, and hormonal results (Journal of neurology, 2012) Randomized controlled trial Effect of nutritional supplementation with milk whey proteins in amyotrophic lateral sclerosis patients (Arquivos de neuro-psiquiatria, 2010) CT.gov search · Cochrane · PubMed	Examine.com	PubChem Examine.com
Vitamin E Natural product Clinical trial publication Vitamin E serum levels and controlled supplementation and risk of amyot… Clinical trial publication Vitamin E intake and risk of amyotrophic lateral sclerosis Clinical trial publication High dose vitamin E therapy in amyotrophic lateral sclerosis as add-on…	Nutraceutical	Phase III	Emerging	10	3 trials 9 Trial pubs View evidence Registry trials NCT00372879 — Clinical Trial of Vitamin E to Treat Muscular Cramps in Patients With ALS (COMPLETED) PHASE3 NCT04140136 — The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS) (UNKNOWN) PHASE2 NCT04244630 — Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial (RECRUITING) PHASE2 Published literature Clinical trial publication Vitamin E serum levels and controlled supplementation and risk of amyotrophic lateral sclerosis (Amyotrophic lateral sclerosis & frontotemporal degeneration, 2013) Clinical trial publication Vitamin E intake and risk of amyotrophic lateral sclerosis (Annals of neurology, 2005) Clinical trial publication High dose vitamin E therapy in amyotrophic lateral sclerosis as add-on therapy to riluzole: results of a placebo-controlled double-blind study (Journal of neural transmission (Vienna, Austria : 1996), 2005) Clinical trial publication [The effect of selegiline and vitamin E in the treatment of ALS: an open randomized clinical trials] (Neurologia i neurochirurgia polska, 2001) Clinical trial publication A double-blind, placebo-controlled randomized clinical trial of alpha-tocopherol (vitamin E) in the treatment of amyotrophic lateral sclerosis. ALS riluzole-tocopherol Study Group (Amyotrophic lateral sclerosis and other motor neuron disorders : official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases, 2001) CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Vitamin D3 Natural product Clinical trial publication Network pharmacology analysis and clinical efficacy of the traditional… Human Study In ALS patients, we report that a severe vitamin D deficiency accelerat… Human Study Suboptimal vitamin D levels are frequently found in ALS patients (81%).	Nutraceutical	Preclinical	Emerging	10	2 trials 1 Trial pubs View evidence Registry trials NCT02893605 — Study of Gene Polymorphisms Involved in the Metabolism and Action of Vitamin D in Amyotrophic Lateral Sclerosis (COMPLETED) NCT01823380 — Influence of the Vitamin D Blood Levels on the Amyotrophic Lateral Sclerosis Phenotype (COMPLETED) NA Published literature Clinical trial publication Network pharmacology analysis and clinical efficacy of the traditional Chinese medicine Bu-Shen-Jian-Pi. Part 3: Alleviation of hypoxia, muscle-wasting, and modulation of redox functions in amyotrophic lateral sclerosis (International journal of clinical pharmacology and therapeutics, 2024) CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Vitamin B12 Natural product Human Study Methylcobalamin may prevent muscle wasting in amyotrophic lateral scler… Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Human Study In ALS patients, we report that a severe vitamin D deficiency accelerat…	Nutraceutical	Phase III	Emerging	10	2 trials View evidence Registry trials NCT04557410 — Open Label Study: Treatment of ALS Fatigue With PolyMVA (COMPLETED) PHASE1 NCT03548311 — Clinical Trial of Ultra-high Dose Methylcobalamin for ALS (COMPLETED) PHASE3 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Omega-3 Fatty Acids Natural product Human Study "Consumption of foods high inω-3 PUFAs may help prevent or delay t… Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Animal Study Branched chain amino acids may have a therapeutic role in ALS.	Nutraceutical	Phase II	Emerging	10	3 trials View evidence Registry trials NCT03659422 — Dietary Approach to Improving Quality of Life in Amyotrophic Lateral Sclerosis (WITHDRAWN) NA NCT06280079 — Ultra-high-caloric, Fatty Diet in ALS (RECRUITING) NA NCT00983983 — High Fat/High Calorie Trial in Amyotrophic Lateral Sclerosis (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Zinc Natural product Clinical trial publication Apilimod dimesylate in C9orf72 amyotrophic lateral sclerosis: a randomi… Clinical trial publication Blood superoxide dismutase, catalase and glutathione peroxidase activit… Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th…	Nutraceutical	Phase II	Emerging	10	1 trials 2 Trial pubs View evidence Registry trials NCT01259050 — Safety Study of High Doses of Zinc in ALS Patients (COMPLETED) PHASE1, PHASE2 Published literature Clinical trial publication Apilimod dimesylate in C9orf72 amyotrophic lateral sclerosis: a randomized phase 2a clinical trial (Brain : a journal of neurology, 2024) Clinical trial publication Blood superoxide dismutase, catalase and glutathione peroxidase activities in familial and sporadic amyotrophic lateral sclerosis (Neurodegeneration : a journal for neurodegenerative disorders, neuroprotection, and neuroregeneration, 1996) CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Curcumin Natural product Human Study Efficacy of mecasin for treatment of amyotrophic lateral sclerosis. Human Study Enhanced acute muscle activation in ALS patients following liposomal cu… Animal Study Curcumin has anti-apoptotic properties in a transgenic amyotrophic late…	Food compound	Phase II	Emerging	7	2 trials 2 Trial pubs View evidence Registry trials NCT04654689 — Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients (COMPLETED) PHASE2 NCT04499963 — Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS) (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Resveratrol Natural product Human Study Enhanced acute muscle activation in ALS patients following liposomal cu… Human In Vitro Resveratrol's neural protective effects for the injured embryoid b… Transgenic Animal Study Coenzyme Q10 demonstrated significant therapeutic effects in transgenic…	Food compound	Phase II	Emerging	7	1 trials View evidence Registry trials NCT04654689 — Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Melatonin Natural product Human Study Free testosterone is significantly decreased in ALS, indicating that su… Human Study Vitamin E supplementation may contribute to preventing amyotrophic late… Animal Study A ketogenic diet in combination with nutritrional supplementation has r…	Nutraceutical	Phase II	Emerging	7	1 trials View evidence Registry trials NCT06429059 — ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Coenzyme Q10 Natural product Meta-analysis Aberrations in Oxidative Stress Markers in Amyotrophic Lateral Sclerosi… Human Study Coenzyme Q10 levels as high as 3,000 mg a day are safe and well-tolerat… Human Study There is increased mitochondrial oxidative damage in patients with spor…	Nutraceutical	Phase II	Emerging	7	2 trials 1 Meta-analysis 3 RCT View evidence Registry trials NCT00243932 — Clinical Trial of High Dose CoQ10 in ALS (COMPLETED) PHASE2 NCT06429059 — ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments (COMPLETED) PHASE2 Published literature Meta-analysis Aberrations in Oxidative Stress Markers in Amyotrophic Lateral Sclerosis: A Systematic Review and Meta-Analysis (Oxidative medicine and cellular longevity, 2019) CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Alpha-Lipoic Acid Natural product Human: Case Report Alpha-lipoic acid, selenium, vitamins, micronutrients and a vegetarian… Transgenic Animal Study Lipoic acid significantly improves survival time in a transgenic mouse… Insect Study α-Lipoic acid attenuates oxidative stress and neurotoxicity in a Drosop…	Nutraceutical	Phase I	Emerging	7	2 trials View evidence Registry trials NCT04518540 — Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis (UNKNOWN) NA NCT04557410 — Open Label Study: Treatment of ALS Fatigue With PolyMVA (COMPLETED) PHASE1 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Ashwagandha Natural product Transgenic Animal Study Ashwagandha treatment reversed the cytoplasmic mislocalization of hTDP-… Animal Study Protective effects of Withania somnifera extract in a model of amyotrop…	Ayurvedic	Phase II	Emerging	7	2 trials View evidence Registry trials NCT06429059 — ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments (COMPLETED) PHASE2 NCT05031351 — NF-κB Inhibition in Amyotrophic Lateral Sclerosis (UNKNOWN) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Selenium Natural product Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th… Human: Case Report Alpha-lipoic acid, selenium, vitamins, micronutrients and a vegetarian…	Nutraceutical	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Ginseng Natural product Animal Study Anti-inflammatory effects of a novel herbal extract in the muscle and s…	Botanical	Preclinical	Emerging	7	16 pubs View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo
Probiotics Natural product Animal Study "Galactooligosaccharide improves the animal survival and alleviate…	Probiotic	Preclinical	Emerging	7	2 trials View evidence Registry trials NCT03324399 — A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With ALS (COMPLETED) NA NCT06051123 — Effects of Probiotics in Amyotrophic Lateral Sclerosis-Frontotemporal Dementia Spectrum Disorder (ALS-FTDSD) Patients (RECRUITING) NA CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo
Astragalus Natural product Animal Study Anti-inflammatory effects of a novel herbal extract in the muscle and s…	TCM herb	Preclinical	Emerging	7	1 Cochrane View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo
L-Arginine Natural product Animal Study A ketogenic diet in combination with nutritrional supplementation has r… Animal Study L-arginine has potent in vitro and in vivo neuroprotective properties a…	Amino acid	Preclinical	Emerging	7	2 Trial pubs View evidence CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Niacin Natural product Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Human Study In ALS patients, we report that a severe vitamin D deficiency accelerat… Human Study Serum vitamin E levels have a protective effect on ALS risk.	Nutraceutical	Phase II	Emerging	7	5 trials View evidence Registry trials NCT03489200 — EH301 for the Treatment of ALS (COMPLETED) NA NCT07414212 — Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Amyotrophic Lateral Sclerosis (ALS) (RECRUITING) PHASE1, PHASE2 NCT05095571 — A Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Extension Study (RECRUITING) NA NCT04562831 — The NO-ALS Study: A Trial of Nicotinamide/Pterostilbene Supplement in ALS. (ACTIVE_NOT_RECRUITING) NA NCT04244630 — Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial (RECRUITING) PHASE2 CT.gov search · Cochrane · PubMed	GreenMedInfo	PubChem GreenMedInfo
Vitamin B12: Methylcobalamin Natural product Human Study Methylcobalamin may prevent muscle wasting in amyotrophic lateral scler… Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Human Study In ALS patients, we report that a severe vitamin D deficiency accelerat…	Nutraceutical	Phase III	Emerging	6	2 trials View evidence Registry trials NCT04557410 — Open Label Study: Treatment of ALS Fatigue With PolyMVA (COMPLETED) PHASE1 NCT03548311 — Clinical Trial of Ultra-high Dose Methylcobalamin for ALS (COMPLETED) PHASE3 CT.gov search · Cochrane · PubMed	GreenMedInfo	GreenMedInfo
Insulin-Like Growth Factor (Igf-1) Natural product Human Study Lead exposure is a risk factor for amyotrophic lateral sclerosis. Human Study Statin drug use may be linked to neuromuscular diseases and an amyotrop… Human Study Statin drugs may cause ALS-like neurodegeneration in susceptible indivi…	Nutraceutical	Phase I	Emerging	3	—	GreenMedInfo	GreenMedInfo
Polyunsaturated Fatty Acids (PUFAs) Natural product Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Human Study "Consumption of foods high inω-3 PUFAs may help prevent or delay t… Animal Study Branched chain amino acids may have a therapeutic role in ALS.	Nutraceutical	Phase I	Emerging	3	—	GreenMedInfo	GreenMedInfo
Ginkgo Biloba Natural product Transgenic Animal Study Gingko biloba has a gender-specific neuroprotective effect in a transge…	Botanical	Preclinical	Emerging	2	—	GreenMedInfo	GreenMedInfo
Cannabidiol Natural product Persistent sodium currents in neurons: potential mechanisms and pharmac… A Mapping Literature Review of Medical Cannabis Clinical Outcomes and Q… Safety and efficacy of nabiximols on spasticity symptoms in patients wi…	Food compound	Preclinical	Emerging	2	—	GreenMedInfo	PubChem GreenMedInfo
Cannabinoids Natural product Meta Analysis, Review A systematic review and meta-analysis off the effects of cannabinoids i… Animal Study A cannabinoid CB2 receptor selective compound, delays disease progressi… Animal Study Cannabinol may have therapeutic value in ameliorating symptoms in ALS.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Estradiol (E(2)) Natural product Animal Study Estradiol slows neurodegeneration in a female mouse experimental model… Animal Study Female sex hormones such as estradiol and exercise have a possible neur… Animal Study Steroid hormones may have neuroprotective properties in ALS.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Cannabis Natural product Human: Case Report Cannabis use is not uncommon in ALS patients who reported significant b… Animal Study Cannabinol may have therapeutic value in ameliorating symptoms in ALS. Animal Study Cannabis contains a number of compounds which may have therapeutic valu…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Progesterone Natural product Human Study Progesterone levels correlate positively with survival and prognosis in… Animal Study Progesterone has neuroprotective properties in a mouse model of spinal… Animal Study Steroid hormones may have neuroprotective properties in ALS.	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Bee Venom Natural product Animal Study Bee venom attenuates neuroinflammatory events and extends survival in a… Transgenic Animal Study Bee venom combined with acupuncture was more effective at reducing infl… Review Bee venom may have therapeutic value in ALS.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Glycoaminoglycans Natural product Animal Study Co-administration of IGF-I and glycosaminoglycans greatly delays motor… Animal Study Glycoaminoglycans have neurotrophic and neuroregenerative effects in a… Animal Study Glycosaminoglycans boost insulin-like growth factor-I-promoted neuropro…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Polyphenols Natural product Dietary polyphenols represent a phytotherapeutic alternative for gut dy… The pivotal role of JAK/STAT and IRS/PI3K signaling pathways in neurode… Targeting Ubiquitin-Proteasome Pathway by Natural Products: Novel Thera…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Testosterone Natural product Human Study Free testosterone is significantly decreased in ALS, indicating that su… Animal Study Steroid hormones may have neuroprotective properties in ALS. Animal Study Testosterone may have neuroprotective properties.	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Antioxidant Formulas Natural product Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th… Human Study A higher intake of food rich in antioxidants such as fruit and vegetabl… Human Study Antioxidants, carotenes, fruits, and vegetables were associated with hi…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
EGCG (Epigallocatechin Gallate) Natural product Human In Vitro Neurorescue activity, APP regulation and amyloid-beta peptide reduction… Animal Study EGCG has multifunctional therapeutic effects in the mouse model of ALS Animal Study EGCG may provide a potential therapeutic candidate for ALS as a disease…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Stilbenes Natural product Animal Study Resveratrol upregulated heat shock proteins and extended the survival o…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Fruit: All Natural product Human Study A higher intake of food rich in antioxidants such as fruit and vegetabl… Human Study Antioxidants, carotenes, fruits, and vegetables were associated with hi…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Vegetables: All Natural product Human Study A higher intake of food rich in antioxidants such as fruit and vegetabl… Human Study Antioxidants, carotenes, fruits, and vegetables were associated with hi…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Glutathione Natural product Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th… Commentary A dietary reduction in glutathione precursors, e.g. glutamate, cysteine…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Anthocyanins Natural product Animal Study Anthocyanins have significant potential as therapeutic agents in a prec… Review Anthocyanins and their metabolites as therapeutic agents for neurodegen…	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Genistein Natural product Transgenic Animal Study Genistein could be a promising treatment for human amyotrophic lateral… Animal Study Genistein is neuroprotective in murine models of familial amyotrophic l…	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Rosmarinic Acid Natural product Animal Study, In Vitro Study Neuroprotection comparison of rosmarinic acid and carnosic acid in prim… Animal Study Rosmarinic acid has therapeutic value in motor dysfunction and life spa…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Coconut Oil Natural product Animal Study Non-invasive assessment of disease progression and neuroprotective effe…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Hesperidin Natural product Review Antioxidant and anti-inflammatory effects of citrus flavonoid hespereti…	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Red Wine Extract Natural product	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Flavonoids Natural product	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Capsaicin Natural product Human Study Capsaicin may improve swallowing impairment in patients with amyotrophi…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Peony Natural product Human Study Efficacy of mecasin for treatment of amyotrophic lateral sclerosis.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Salvia Miltiorrhiza Natural product Human Study Efficacy of mecasin for treatment of amyotrophic lateral sclerosis.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
B-Complex Natural product Human Study High homocysteine levels are associated with neurodegenerative conditio… Human Study High homocysteine levels may be associated with the pathogenesis of neu…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Traditional Chinese Medicine: Jwsjz Decoction Natural product Human Study JWSJZ decoction may be a safe treatment for ALS, and may have delayed t… Animal Study The Chinese Herbal Formula: Wen-Pi-Tang delays the onset and progressio…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Copper Natural product Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Superoxide Dismutase Natural product Human Study Selenium, glutathione, copper/zinc and superoxide dismutase may have th…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Whey Natural product Human Study Whey protein improves the condition of patients with amyotrophic latera…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Catechin Natural product Human In Vitro Neurorescue activity, APP regulation and amyloid-beta peptide reduction…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Honokiol Natural product Animal Study, In Vitro Study Honokiol showed promising preclinical potential as a multiple target dr…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Palmitoylethanolamide Natural product Human: Case Report Palmitoylethanolamide treatment led to an improved clinical picture.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Galactooligosaccharides Natural product Animal Study "Galactooligosaccharide improves the animal survival and alleviate…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Arginine-Alpha-Ketoglutarate (Aakg) Natural product Human: Case Report Alpha-lipoic acid, selenium, vitamins, micronutrients and a vegetarian… Animal Study A ketogenic diet in combination with nutritrional supplementation has r… Animal Study L-arginine has potent in vitro and in vivo neuroprotective properties a…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
GABA (Gamma-Aminobutyric Acid) Natural product Animal Study A ketogenic diet in combination with nutritrional supplementation has r…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Medium Chain Triglycerides Natural product Animal Study A ketogenic diet in combination with nutritrional supplementation has r… Animal Study Branched chain amino acids may have a therapeutic role in ALS. Animal Study Linking respiratory chain uncoupling to amyotrophic lateral sclerosis i…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Strawberry Natural product Animal Study Anthocyanins have significant potential as therapeutic agents in a prec…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Glycyrrhiza Uralensis Natural product Animal Study Anti-inflammatory effects of a novel herbal extract in the muscle and s…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Baicalein Natural product Animal Study Baicalein benefits amyotrophic lateral sclerosis via reduction of Intra…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Amino Acids: Branched Chain Natural product Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Animal Study Branched chain amino acids may have a therapeutic role in ALS. Animal Study Co-administration of IGF-I and glycosaminoglycans greatly delays motor…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Caprylic Acid Natural product Animal Study Caprylic triglyceride as a novel therapeutic approach to effectively im…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Fisetin Natural product In Vitro Study, Transgenic Animal Study Fisetin exerts antioxidant and neuroprotective effects in multiple muta…	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Phytoestrogens Natural product Animal Study Genistein is neuroprotective in murine models of familial amyotrophic l…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Riboflavin (Vitamin B-2) Natural product Human Study A high intake of poluunsaturated fatty acids and vitamin E is associate… Human Study In ALS patients, we report that a severe vitamin D deficiency accelerat… Human Study Serum vitamin E levels have a protective effect on ALS risk.	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Carnosic Acid Natural product Animal Study, In Vitro Study Neuroprotection comparison of rosmarinic acid and carnosic acid in prim…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Pterostilbene Natural product Animal Study Nicotinamide riboside and pterostilbene cooperatively delay motor neuro…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Oxaloacetate Natural product Animal Study Oxaloacetate treatment preserves motor function in SOD1G93A mice and no…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Sophora Flavescens Natural product Animal Study Oxymatrine extends survival by attenuating neuroinflammation in a mouse…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Burdock Natural product Animal Study Pathology reduction and motor behavior improvement associated with ultr…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Pyruvate Natural product Transgenic Animal Study Pyruvate slows disease progression in a transgenic mouse model of ALS.	Food compound	Preclinical	Emerging	0	—	GreenMedInfo	PubChem GreenMedInfo
Rosemaric Acid Natural product Animal Study Rosmarinic acid has therapeutic value in motor dysfunction and life spa…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Rosemary Natural product Animal Study Rosmarinic acid has therapeutic value in motor dysfunction and life spa…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Scolopendra Subspinipes Mutilans Natural product Animal Study Scolopendra subspinipes mutilans attenuates neuroinflammation in a mous…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
Wen Pi Tang Natural product Animal Study The Chinese Herbal Formula: Wen-Pi-Tang delays the onset and progressio…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo
5-HTP (5-Hydroxytryptophan) Natural product Animal Study The serotonin precurser 5-HTP improves locomotor function and survival…	Nutraceutical	Preclinical	Emerging	0	—	GreenMedInfo	GreenMedInfo

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

Disease overview

Remission & chronicity

Alterations

Therapeutics

Natural Products

Part of the Open Source Medicine Foundation Network