What research stage is stem-cell therapy for ALS (Motor Neurone Disease) at?
Confirmatory-phase work accounts for 5 of the 65 records here; the remainder is dose-finding, safety or observational (2026-08-29).
Phase distribution
| Phase label | Records | Share |
|---|---|---|
| Phase 1 | 38 | 48% |
| Phase 2 | 24 | 30% |
| Not applicable | 12 | 15% |
| Phase 3 | 5 | 6% |
| Early Phase 1 | 1 | 1% |
What the phase mix means for ALS (Motor Neurone Disease)
Because ALS progresses fast, sponsors can run relatively short trials, so the field reached later-phase designs earlier than in slower neurodegenerative conditions. A Phase 3 label here means a powered attempt at slowing decline on a functional scale — not a claim of reversal.
The later-phase studies themselves
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT06973629 — Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY) | Phase 3 | Not Yet Recruiting | 200 | Brainstorm-Cell Therapeutics | 2025-06-30 |
| NCT03280056 — Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients | Phase 3 | Completed | 196 | Brainstorm-Cell Therapeutics | 2017-08-28 |
| NCT04745299 — Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS | Phase 3 | Completed | 123 | Corestemchemon, Inc. | 2021-02-24 |
| NCT04220190 — RAPA-501 Therapy for ALS | Phase 2, Phase 3 | Recruiting | 41 | Rapa Therapeutics LLC | 2025-01-02 |
| NCT01933321 — Effect of Intrathecal Administration of Hematopoietic Stem Cells in Patients With Amyotrophic Lateral Sclerosis (ALS) | Phase 2, Phase 3 | Completed | 14 | Hospital Universitario Dr. Jose E. Gonzalez | 2012-09 |
How to read phase labels
Phase labels describe what a study was designed to answer. A Phase 1 result showing improvement answers a question nobody asked of it, because the design has no control arm and too few participants to distinguish an effect from noise.
Median planned enrolment across sampled studies here is 20 participants. Small single-arm studies can flag safety problems; they cannot separate a treatment effect from natural fluctuation, placebo response or regression to the mean.
The largest studies by planned enrolment
Size is a crude proxy for what a study can detect, but it separates a serious attempt from a case series presented as evidence.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT02574390 — Answer ALS: Individualized Initiative for ALS Discovery | n/a | Completed | 1,049 | Johns Hopkins University | 2015-12 |
| NCT02795052 — Neurologic Stem Cell Treatment Study | n/a | Recruiting | 500 | MD Stem Cells | 2016-06 |
| NCT06973629 — Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY) | Phase 3 | Not Yet Recruiting | 200 | Brainstorm-Cell Therapeutics | 2025-06-30 |
| NCT03280056 — Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients | Phase 3 | Completed | 196 | Brainstorm-Cell Therapeutics | 2017-08-28 |
| NCT04745299 — Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS | Phase 3 | Completed | 123 | Corestemchemon, Inc. | 2021-02-24 |
Frequently asked
What research stage is stem-cell therapy for ALS (Motor Neurone Disease) at?
5 of 65 records are labelled Phase 3 or 4 (2026-08-29).
Is a Phase 3 trial proof that it works?
No — it is a design capable of testing that question. Proof depends on the reported result, replication, and whether the outcome measured matters to patients.
Why do so many records have no phase?
Observational studies and some cell-therapy protocols are registered without a phase label. It is a registry field, not a quality rating.
Registry and literature counts retrieved 2026-08-29 (ClinicalTrials.gov API v2; PubMed E-utilities). Registration or publication volume measures research activity, not effectiveness or approval. Educational information — not medical advice; verify product, indication, legal pathway and evidence with an independent qualified physician. Page generated 2026-08-29. See our editorial standards.
More on this condition
- ALS (Motor Neurone Disease): cell-therapy evidence and registered trials
- How much does stem cell therapy for ALS (Motor Neurone Disease) cost? (2026)
- ALS (Motor Neurone Disease) stem cell therapy — your questions answered (2026)
- ALS (Motor Neurone Disease): cell-therapy evidence by therapy class
- MSC vs HSCT for ALS (Motor Neurone Disease): what the registries show