Biomarker & Intervention Discovery

Amyotrophic Lateral Sclerosis (ALS)

This page lists 10 druggable gene targets, 43 approved or trial-tested drug candidates and 15 recent clinical trials for Amyotrophic Lateral Sclerosis (ALS), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

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 →
Research Tracker Chronic Disease Interventions Amyotrophic Lateral Sclerosis (ALS)

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.

ATXN2 398C9orf72 0CSF1R 258FUS 3KIF5A 1KIT 278SLC1A2 118SOD1 32TARDBP 119UNC13A 0

ATXN2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
N-OLEOYLDOPAMINE ? unclear Mechanistic DGIdb
THIRAM ? unclear Mechanistic DGIdb
SORAFENIB TOSYLATE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL603952 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL299853 ? unclear Mechanistic DGIdb
FANGCHINOLINE ? unclear Mechanistic DGIdb
PARAROSANILINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL604321 ? unclear Mechanistic DGIdb
FLUPENTHIXOL ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL576362 ? unclear Mechanistic DGIdb
VER-3323 ? unclear Mechanistic DGIdb
ALEXIDINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL461193 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL536375 ? unclear Mechanistic DGIdb
FLAVOKAWAIN B ? unclear Mechanistic DGIdb
PIPERLONGUMINE ? unclear Mechanistic DGIdb
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL585502 ? unclear Mechanistic DGIdb
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL523200 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL546493 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL429095 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL579318 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL493863 ? unclear Mechanistic DGIdb
OXYPHENBUTAZONE ? unclear Mechanistic DGIdb
PROTOAPIGENONE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1889303 ? unclear Mechanistic DGIdb
CALCIMYCIN ? unclear Mechanistic DGIdb
PSEUDOCHELERYTHRINE ? unclear Mechanistic DGIdb
BAY 11-7082 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL482050 ? unclear Mechanistic DGIdb
CP55940 ? unclear Mechanistic DGIdb
MITOXANTRONE HYDROCHLORIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL546597 ? unclear Mechanistic DGIdb
TPCK ? unclear Mechanistic DGIdb
KEPONE ? unclear Mechanistic DGIdb
OSAJIN ? unclear Mechanistic DGIdb
SPHAEROPSIDIN A ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL599098 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL602946 ? unclear Mechanistic DGIdb

Showing top 40 of 398 agents ranked by evidence tier.

C9orf72

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

CSF1R

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
VATALANIB ↓ inhibits Mechanistic DGIdb
PAZOPANIB ↓ inhibits Mechanistic DGIdb
GTP-14564 ↓ inhibits Mechanistic DGIdb
PLX5622 ↓ inhibits Mechanistic DGIdb
ONATASERTIB ↓ inhibits Mechanistic DGIdb
ARRY-382 ↓ inhibits Mechanistic DGIdb
ILORASERTIB ↓ inhibits Mechanistic DGIdb
EMACTUZUMAB ? unclear Mechanistic DGIdb
CABIRALIZUMAB ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL546797 ? unclear Mechanistic DGIdb
ALSTERPAULLONE ? unclear Mechanistic DGIdb
CT-1578 ↓ inhibits Mechanistic DGIdb
EDICOTINIB ↓ inhibits Mechanistic DGIdb
ENRUPATINIB ↓ inhibits Mechanistic DGIdb
LINIFANIB ↓ inhibits Mechanistic 3.0 nM (IC50) DGIdb
IL-34 ↑ activates Mechanistic DGIdb
SEGIGRATINIB ↓ inhibits Mechanistic DGIdb
CYC-116 ? unclear Mechanistic DGIdb
PEXIDARTINIB ↓ inhibits Mechanistic DGIdb
BLZ-945 ? unclear Mechanistic DGIdb
ENMD-2076 ↓ inhibits Mechanistic DGIdb
AXATILIMAB ? unclear Mechanistic DGIdb
AC710 ↓ inhibits Mechanistic DGIdb
HESPERADIN ↓ inhibits Mechanistic DGIdb
DACTOLISIB ? unclear Mechanistic DGIdb
IACS-9439 ↓ inhibits Mechanistic DGIdb
IBCASERTIB ↓ inhibits Mechanistic DGIdb
M-CSF ? unclear Mechanistic DGIdb
ELZOVANTINIB ↓ inhibits Mechanistic DGIdb
SORAFENIB ? unclear Mechanistic DGIdb
TANDUTINIB ? unclear Mechanistic 3430.0 nM (IC50) DGIdb
LENOGRASTIM ? unclear Mechanistic DGIdb
QUIZARTINIB ↓ inhibits Mechanistic DGIdb
MCS-110 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL365617 ? unclear Mechanistic DGIdb
GR-389988 ↓ inhibits Mechanistic DGIdb
PROGESTERONE ? unclear Mechanistic DGIdb
VIMSELTINIB ↓ inhibits Mechanistic DGIdb
SUNITINIB MALATE ↓ inhibits Mechanistic DGIdb
PLX-7486 ↓ inhibits Mechanistic DGIdb

Showing top 40 of 258 agents ranked by evidence tier.

FUS

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TRETINOIN ? unclear Mechanistic DGIdb
CHEMBL5653589 ↓ inhibits Mechanistic 36.62 nM (Kd) ChEMBL
CHEMBL3752910 ↓ inhibits Mechanistic 17.47 nM (Kd) ChEMBL

KIF5A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL2031572 ↓ inhibits Mechanistic 50000.0 nM (IC50) ChEMBL

KIT

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
OSI-930 ? unclear Mechanistic DGIdb
CHMFL-KIT-8140 ↓ inhibits Mechanistic DGIdb
IMATINIB ↓ inhibits Mechanistic 260.0 nM (IC50) DGIdb
PEXIDARTINIB ↓ inhibits Mechanistic DGIdb
REGORAFENIB ↓ inhibits Mechanistic DGIdb
SORAFENIB ↓ inhibits Mechanistic 740.0 nM (Kd) DGIdb
TANDUTINIB ↓ inhibits Mechanistic 170.0 nM (IC50) DGIdb
RIPRETINIB ↓ inhibits Mechanistic DGIdb
XL820 ↓ inhibits Mechanistic DGIdb
VATALANIB ↓ inhibits Mechanistic 730.0 nM (IC50) DGIdb
PAZOPANIB HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
ENMD-2076 ↓ inhibits Mechanistic DGIdb
TRAMETINIB DIMETHYL SULFOXIDE ? unclear Mechanistic DGIdb
PEMBROLIZUMAB ? unclear Mechanistic DGIdb
VELZATINIB ↓ inhibits Mechanistic DGIdb
PONATINIB ? unclear Mechanistic DGIdb
CABOZANTINIB S-MALATE ↓ inhibits Mechanistic DGIdb
CRIZOTINIB ? unclear Mechanistic DGIdb
LABUXTINIB ↓ inhibits Mechanistic DGIdb
BAY 61-3606 ? unclear Mechanistic DGIdb
CATEQUENTINIB ↓ inhibits Mechanistic DGIdb
PLX5622 ↓ inhibits Mechanistic DGIdb
RISVODETINIB ↓ inhibits Mechanistic DGIdb
VENETOCLAX ? unclear Mechanistic DGIdb
XL999 ? unclear Mechanistic DGIdb
EDICOTINIB ↓ inhibits Mechanistic DGIdb
AMUVATINIB ↓ inhibits Mechanistic DGIdb
ILORASERTIB ↓ inhibits Mechanistic DGIdb
AC710 ↓ inhibits Mechanistic DGIdb
LENVATINIB ? unclear Mechanistic DGIdb
AVAPRITINIB ↓ inhibits Mechanistic DGIdb
MOTESANIB ↓ inhibits Mechanistic DGIdb
SU-11652 ↓ inhibits Mechanistic DGIdb
JNJ-28312141 ↓ inhibits Mechanistic DGIdb
RG-1530 ↓ inhibits Mechanistic DGIdb
MASITINIB ↓ inhibits Mechanistic DGIdb
SERALUTINIB ↓ inhibits Mechanistic DGIdb
FAMITINIB ↓ inhibits Mechanistic DGIdb
MIDOSTAURIN ↓ inhibits Mechanistic DGIdb
SORAFENIB TOSYLATE ↓ inhibits Mechanistic DGIdb

Showing top 40 of 278 agents ranked by evidence tier.

SLC1A2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
[3H]D-ASPARTIC ACID ? unclear Mechanistic DGIdb
THREO-3-METHYLGLUTAMATE ↓ inhibits Mechanistic DGIdb
SYM2081 ↓ inhibits Mechanistic DGIdb
ASPARTIC ACID ? unclear Mechanistic 32000.0 nM (IC50) DGIdb
WAY-213613 ↓ inhibits Mechanistic DGIdb
(R)-AS-1 ~ modulates Mechanistic DGIdb
[3H]SYM2081 ? unclear Mechanistic DGIdb
DL-TBOA ↓ inhibits Mechanistic DGIdb
DIHYDROKAINATE ↓ inhibits Mechanistic 89000.0 nM (IC50) DGIdb
CHEMBL1790042 ↓ inhibits Mechanistic 1300.0 nM (IC50) ChEMBL
CHEMBL1790043 ↓ inhibits Mechanistic 2200.0 nM (IC50) ChEMBL
CHEMBL475341 ↓ inhibits Mechanistic 7000.0 nM (IC50) ChEMBL
CHEMBL79140 ↓ inhibits Mechanistic 64000.0 nM (IC50) ChEMBL
CHEMBL1790044 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL2113110 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL424838 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL2113111 ↓ inhibits Mechanistic 600.0 nM (IC50) ChEMBL
CHEMBL2113112 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL2113113 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL373283 ↓ inhibits Mechanistic 500.0 nM (IC50) ChEMBL
CHEMBL2113114 ↓ inhibits Mechanistic 700.0 nM (IC50) ChEMBL
CHEMBL2113115 ↓ inhibits Mechanistic 80.0 nM (IC50) ChEMBL
CHEMBL2113116 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL2113117 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL2113118 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL2113119 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL2113120 ↓ inhibits Mechanistic 500.0 nM (IC50) ChEMBL
CHEMBL425569 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL2113121 ↓ inhibits Mechanistic 130.0 nM (IC50) ChEMBL
CHEMBL1628669 ↓ inhibits Mechanistic 80.0 nM (IC50) ChEMBL
CHEMBL198315 ↓ inhibits Mechanistic 700.0 nM (IC50) ChEMBL
CHEMBL2113122 ↓ inhibits Mechanistic 60.0 nM (IC50) ChEMBL
CHEMBL2113123 ↓ inhibits Mechanistic 400.0 nM (IC50) ChEMBL
CHEMBL2113124 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL2113125 ↓ inhibits Mechanistic 200.0 nM (IC50) ChEMBL
CHEMBL2113126 ↓ inhibits Mechanistic 100.0 nM (IC50) ChEMBL
CHEMBL2113127 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL2113128 ↓ inhibits Mechanistic 300.0 nM (IC50) ChEMBL
CHEMBL2113129 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL1628570 ↓ inhibits Mechanistic 700.0 nM (IC50) ChEMBL

Showing top 40 of 118 agents ranked by evidence tier.

SOD1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
DOXYCYCLINE ANHYDROUS ? unclear Mechanistic DGIdb
TOFERSEN ? unclear Mechanistic DGIdb
TETRACYCLINE ? unclear Mechanistic DGIdb
OXYTETRACYCLINE ANHYDROUS ? unclear Mechanistic DGIdb
CHEMBL272641 ↓ inhibits Mechanistic 67900.0 nM (IC50) ChEMBL
1,4-BIS(3'-PYRAZOLYLAMINO)BENZO[G]PHTHALAZINE ↓ inhibits Mechanistic 96600.0 nM (IC50) ChEMBL
CHEMBL2165614 ↑ activates Mechanistic 3700.0 nM (EC50) ChEMBL
CHEMBL2165613 ↑ activates Mechanistic 2840.0 nM (EC50) ChEMBL
CHEMBL2165612 ↑ activates Mechanistic 1020.0 nM (EC50) ChEMBL
CHEMBL2165611 ↑ activates Mechanistic 510.0 nM (EC50) ChEMBL
CHEMBL2165610 ↑ activates Mechanistic 1070.0 nM (EC50) ChEMBL
CHEMBL2165609 ↑ activates Mechanistic 580.0 nM (EC50) ChEMBL
CHEMBL1939222 ↑ activates Mechanistic 67.0 nM (EC50) ChEMBL
CHEMBL2165608 ↑ activates Mechanistic 870.0 nM (EC50) ChEMBL
CHEMBL2165607 ↑ activates Mechanistic 720.0 nM (EC50) ChEMBL
CHEMBL2165606 ↑ activates Mechanistic 1960.0 nM (EC50) ChEMBL
CHEMBL2165605 ↑ activates Mechanistic 790.0 nM (EC50) ChEMBL
CHEMBL2165604 ↑ activates Mechanistic 1470.0 nM (EC50) ChEMBL
CHEMBL2165603 ↑ activates Mechanistic 1410.0 nM (EC50) ChEMBL
CHEMBL2165602 ↑ activates Mechanistic 4700.0 nM (EC50) ChEMBL
CHEMBL2165601 ↑ activates Mechanistic 2880.0 nM (EC50) ChEMBL
CHEMBL1643557 ↑ activates Mechanistic 170.0 nM (EC50) ChEMBL
CHEMBL1643556 ↑ activates Mechanistic 710.0 nM (EC50) ChEMBL
CHEMBL1643541 ↑ activates Mechanistic 1930.0 nM (EC50) ChEMBL
CHEMBL2179266 ↓ inhibits Mechanistic 83120.0 nM (IC50) ChEMBL
CHEMBL2179265 ↓ inhibits Mechanistic 65980.0 nM (IC50) ChEMBL
CHEMBL2179268 ↓ inhibits Mechanistic 71460.0 nM (IC50) ChEMBL
CHEMBL3355357 ↓ inhibits Mechanistic 56600.0 nM (IC50) ChEMBL
CHEMBL3355358 ↓ inhibits Mechanistic 58900.0 nM (IC50) ChEMBL
CHEMBL4127450 ↓ inhibits Mechanistic 100000.0 nM (IC50) ChEMBL
CHEMBL1782791 ↓ inhibits Mechanistic 46600.0 nM (IC50) ChEMBL
CHEMBL5653589 ↓ inhibits Mechanistic 16280.26 nM (Kd) ChEMBL

TARDBP

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TPCK ? unclear Mechanistic DGIdb
ESFLURBIPROFEN ? unclear Mechanistic DGIdb
DEXIBUPROFEN ? unclear Mechanistic DGIdb
MORUSINOL ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL64239 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL591178 ? unclear Mechanistic DGIdb
IBUPROFEN, SODIUM SALT ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL592611 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1479114 ? unclear Mechanistic DGIdb
PHENYLETHENYLSEMICARBAZONE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL591876 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL524407 ? unclear Mechanistic DGIdb
CHRYSIN DIMETHYL ETHER ? unclear Mechanistic DGIdb
NAPROXEN SODIUM ? unclear Mechanistic DGIdb
FORMESTANE ? unclear Mechanistic DGIdb
ONONETIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL153505 ? unclear Mechanistic DGIdb
EQUILIN ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL579318 ? unclear Mechanistic DGIdb
RILUZOLE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1545737 ? unclear Mechanistic DGIdb
ADENINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1889303 ? unclear Mechanistic DGIdb
DIFENPIRAMIDE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL74852 ? unclear Mechanistic DGIdb
BENZOPHENONE-THIOSEMICARBAZONE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL600107 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1553566 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL599924 ? unclear Mechanistic DGIdb
PAXILLINE ? unclear Mechanistic DGIdb
PIPERLONGUMINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL597449 ? unclear Mechanistic DGIdb
JNJ1661010 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1523215 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1902074 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1522984 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1415851 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL66279 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL47875 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL1445650 ? unclear Mechanistic DGIdb

Showing top 40 of 119 agents ranked by evidence tier.

UNC13A

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Amyotrophic Lateral Sclerosis (ALS): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments Completed
NCT06429059 Phase: 2 Started: 2024-06-12

GenieUs developed an analysis platform that will be tested to separate study participants with ALS into four categories based on blood work. These general categories are neuroinflammation, oxidative stress, impaired autophagy \& axonal transport, and mitochondrial dysfunction. Once a disease categor...

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS Recruiting
NCT07322003 Phase: 3 Started: 2026-02-01

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is: Does pridopidine slow disease progression of ALS? Researchers will compare p...

A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS) Recruiting
NCT07855211 Phase: 1/2 Started: 2026-10-07

In this study, researchers will learn more about the effects of BIIB147, in participants with amyotrophic lateral sclerosis, also known as ALS. ALS is a rare disease that damages the motor neurons in the brain and spinal cord that help control the movement of muscles in the body. It is a progressive...

Regulatory T Cells for Amyotrophic Lateral Sclerosis Active Not Recruiting
NCT05695521 Phase: 1 Started: 2023-04-03

Phase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients with Amyotrophic Lateral Sclerosis.

Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms Active Not Recruiting
NCT04856982 Phase: 3 Started: 2021-05-17

In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. One of the causes of ALS is a change (or mutation) in the SOD1 gene, ...

Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS Recruiting
NCT07287397 Phase: 1/2 Started: 2025-12-19

PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Tw...

FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS Recruiting
NCT07674667 Phase: 1/2 Started: 2026-08-20

The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS. TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the...

Study of COYA 302 for the Treatment of ALS Active Not Recruiting
NCT07161999 Phase: 2/3 Started: 2025-10-01

The ALSTARS trial will be conducted across 20-25 sites in the US and Canada, and will evaluate the safety and efficacy of an investigational treatment called COYA 302 for adults with Amyotrophic Lateral Sclerosis (ALS). COYA 302 is an investigational and proprietary biologic combination therapy with...

A Study to Assess the Safety, Tolerability, and Pharmacology of Darifenacin in Patients With ALS Recruiting
NCT06249867 Phase: 2 Started: 2024-11-08

Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of t...

Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS Recruiting
NCT06126315 Phase: 2/3 Started: 2025-03-26

Phase II/III multicenter, randomized, double-blind, placebo-controlled trial on acetyl-L-carnitine (ALCAR) in subjects living with amyotrophic lateral sclerosis (ALS). Primary study aim: The clinical objective consists of assessing the efficacy of ALCAR (two different dosages will be tested: 1.5g/da...

The NO-ALS Study: A Trial of Nicotinamide/Pterostilbene Supplement in ALS. Active Not Recruiting
NCT04562831 Phase: N/A Started: 2020-10-07

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is 3 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK per year Research i.a. from t...

TP04HN106 in the Treatment of Patients With Amyotrophic Lateral Sclerosis Recruiting
NCT06726577 Phase: 1/2 Started: 2026-06-04

This trial adopts a multicenter, randomized, double-blind, placebo-controlled parallel design. This experiment is divided into two groups: the experimental drug group and the placebo group. Successful participants will be randomly assigned to the two groups, with an expected enrollment of 60 partici...

Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS) Recruiting
NCT07204977 Phase: 1 Started: 2026-02-02

Background: Amyotrophic lateral sclerosis (ALS) is a disorder that damages nerve cells in the brain and spinal cord. It can cause muscle weakness, paralysis, and loss of movement. The symptoms grow worse over time. Half of all people with ALS live only 3 to 5 years after diagnosis. Current drug trea...

A Study to Evaluate, Safety, Tolerability, and PK of AP-101 in Participants With ALS Completed
NCT05039099 Phase: 2 Started: 2021-09-02

The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

RAPA-501 Therapy for ALS Recruiting
NCT04220190 Phase: 2/3 Started: 2025-01-02

RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).

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 10-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).

43 agents shown (approved or with trial evidence) — 41 not found by the gene-target search above.

Riluzole

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT02588677 NCT06676423 NCT00868166

RanolazineNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06527222 NCT06527222

creatineNOT in gene-target list

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01257581 NCT00355576

MexiletineNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01811355 NCT01849770

100% of individual calory requirementNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06877143

120% of individual calory requirementNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06877143

DexpramipexoleNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01536249

Cimetidine plus DexpramipexoleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01536249

EzogabineNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02450552

Tofersen

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02623699

COYA 302NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07161999

Mind Extender (MindEx)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05936619

Reference capsule GDC-0134NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03237741

Prototype capsule GDC-0134NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03237741

rabeprazoleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03237741

RasagilineNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01879241

leuprolideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00004771

testosteroneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00004771

dextromethorphan HBr and quinidine sulfateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03883581

probioticNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03324399

Masitinib (4.5)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02588677

Masitinib (3.0)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02588677

LevosimendanNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03505021

NX210cNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06365216

Beta Hydroxybutyrate Ester (KetoneAid KE4)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04820478

ZilucoplanNOT in gene-target list

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04436497

lithiumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00925847

MT-1186NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05568615

sodium phenylbutyrateNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00107770

AAV9 Gene therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07401121

Respecting Choices First StepsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03516994

Five Wishes FormNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03516994

LevetiracetamNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00324454

TCH346NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00230074

nL-CHCHD-001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06392126

50 mg UsnoflastNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07023835

75 mg UsnoflastNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07023835

RT001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04762589

R(+) pramipexole dihydrochloride monohydrateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00140218

NSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00397423

DeferiproneNOT in gene-target list

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03293069

ECAS-NNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03579017

MoCANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03579017

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.