How strong is the evidence for stem-cell therapy in ALS (Motor Neurone Disease)?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is relatively mature, with multiple Phase 3/4 registrations: the registry holds 65 records, 25 completed, 5 at Phase 3 or 4, and 8 stopped early (12%). MSC-specific work accounts for 29 of those records. These counts describe research activity and maturity, not benefit, safety or certainty. Snapshot 2026-08-29.
What counts as a real result in ALS (Motor Neurone Disease)
The endpoint that matters is rate of change on the ALSFRS-R functional scale, plus survival and respiratory function, against a control arm. ALS has documented plateaus and rare reversals, so any single-arm report of 'stabilisation' is uninterpretable. Ask which arm the comparison came from before reading anything into it.
What we can say from registrations alone
- Volume: 65 registered studies since 2007.
- Maturity: 5 confirmatory-phase records.
- Delivery: 25 completed; 8 terminated, withdrawn or suspended.
- Scale: median planned enrolment 20 participants, largest 1,049.
The completed studies
Completed does not mean published, and published does not mean positive. These are the finished records — the starting point for checking whether results ever appeared.
| 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 |
| 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 |
| NCT01363401 — Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis | Phase 1, Phase 2 | Completed | 72 | Corestemchemon, Inc. | 2011-02 |
| NCT01254539 — Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA) | Phase 1, Phase 2 | Completed | 63 | Fundacion para la Formacion e Investigacion Sanitarias de la | 2010-10 |
| NCT02290886 — A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral | Phase 1, Phase 2 | Completed | 52 | Andalusian Initiative for Advanced Therapies - Fundación Púb | 2014-07 |
What registrations cannot tell you
Whether results were positive. Whether they were published at all — a large share of registered studies never report outcomes, which biases what the literature appears to show. And whether the product a commercial clinic sells resembles the product studied: cell source, dose, processing and delivery route vary enormously between a registered protocol and an off-protocol injection.
How to use this when someone is selling you a treatment
Ask which specific registered study supports the offer, then compare its intervention description with your quote. If the answer is a general gesture at "ALS (Motor Neurone Disease) research", you are being sold the field's activity rather than evidence about the product. The Provider Question Builder turns that gap into questions.
Frequently asked
How strong is the evidence for stem-cell therapy in ALS (Motor Neurone Disease)?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is relatively mature, with multiple Phase 3/4 registrations: 65 registered records, 5 at Phase 3/4 and 8 stopped early (2026-08-29).
Has any stem-cell product been approved for this condition?
Check the regulator's own register for the exact product name. Marketing frequently blurs approval for one narrow indication into approval in general.
Why do clinics cite hundreds of studies?
It converts activity into the appearance of proof. A hundred early-phase registrations do not add up to one adequately controlled result.
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