ALS destroys the nerve cells that control muscles, leading to paralysis; about 30,000 Americans live with it and roughly 5,000 to 6,000 are diagnosed each year. Riluzole and edaravone modestly slow it. The real progress is in genetic forms: drugs targeting SOD1 and now FUS work, and the field is testing whether treating gene carriers before symptoms can prevent ALS entirely.
- 286studies open or running
- 18phase 3 trials
- 2,460papers published this year
- 40NIH-funded projects
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Now
Nov 2026
A gene drug slows SOD1-ALSHappened 108 people followed
Starting tofersen early slowed decline more than starting 6 months later.
108: people with SOD1-ALS followed long term.
A second genetic ALS drug worksHappened
Ulefnersen met its primary endpoint for FUS-ALS in phase 3; details pending.
Broad-acting drugs keep failingHappened
Arimoclomol did not slow ALS, and a C9orf72 antisense drug showed no benefit.
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6 months
by May 2027
Full FUSION data and a filingLikely
Effect size and safety for ulefnersen should be presented, with a likely FDA filing.
-
12 months
by Nov 2027
Can ALS be prevented in gene carriers?Possible
ATLAS gives tofersen to SOD1 carriers when a blood marker rises, before symptoms; due August 2027.
Platform trial resultsPossible
The HEALEY platform (1,500 people) tests several drugs at once; due July 2027.
-
3 years
by late 2029
Pridopidine in a dedicated phase 3Long shot
PREVAiLS (500 people) follows a signal in earlier ALS; due March 2028.
Masitinib confirmatory trialLong shot
AB Science tests masitinib with riluzole (495 people); due December 2027.
-
5 years
by late 2031
Faster drug testing through platformsPossible
The UK's MND-SMART (1,150 people) keeps adding and dropping drugs; runs to 2030.
The research paths
Gene-targeted drugs
108 people followedDetails and 1 trialsHide
Antisense drugs that lower toxic proteins from specific genes.
WatchFUSION full data; ATLAS prevention.
Platform trials
Details and 2 trialsHide
Testing many drugs at once to find winners faster.
WatchHEALEY and MND-SMART results.
Broad-acting drugs
Details and 3 trialsHide
Drugs for all ALS, with a long record of failures.
WatchPridopidine, masitinib, COYA 302.
Borrowed from other conditions
From Spinal muscular atrophy
Antisense drugs
Approved (SOD1)
The same technology as nusinersen, which transformed SMA.
From Huntington's disease
Pridopidine
Phase 3
A sigma-1 receptor drug first developed for Huntington's.
Open to join now
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ALS
Pridopidine
Locations and eligibility → -
ALS
Masitinib with riluzole
Locations and eligibility → -
ALS, UK
MND-SMART platform
Locations and eligibility → -
ALS, Italy
Acetyl-L-carnitine
Locations and eligibility →
See every recruiting ALS trial →
When the trials finish
Completion dates the trials registered. Results usually follow 3 to 12 months later.
- Heating up
- Maturing
- Early
- Cooling
- Fully enrolled, waiting on results
- Still enrolling
What just happened
Ulefnersen succeeds for FUS-ALS
FUSION topline.
Early tofersen slows SOD1-ALS
JAMA Neurology.
Standard treatment today
- All patientsRiluzole; edaravone for some; multidisciplinary clinic care.
- SOD1 gene variantTofersen (Qalsody).
- FUS gene variantEarly access to ulefnersen; trials.
- Symptom careBreathing support (non-invasive ventilation), feeding tube, speech devices, physical therapy.
- Family membersGenetic counseling and testing; prevention trials for SOD1 carriers.
How to read this page, and sourcesHow to read this page, and sources
- About 10% of ALS is inherited; most new drugs so far work only for specific gene variants. Genetic testing is recommended for everyone with ALS.
- Forecasts are editorial judgments by Find a Trial, not predictions from sponsors or regulators.
See ALS trials recruiting now →