Spinal Muscular Atrophy: cell-therapy evidence by therapy class
Across every stem-cell intervention, ClinicalTrials.gov holds 5 registered records for Spinal Muscular Atrophy — 1 recruiting, 0 at Phase 3 or 4, in 4 countries. Snapshot 2026-08-29.
The condition, and why cells are proposed
Spinal muscular atrophy is caused by SMN1 loss, and it is the clearest example in this field of gene-targeted therapy transforming a disease while cell therapy remained peripheral. Approved gene therapy and splicing modifiers changed the natural history; cell approaches did not.
Therapy classes side by side
| Therapy class | Registered | Recruiting | Phase 3/4 | Countries |
|---|---|---|---|---|
| MSC | 2 | 0 | 0 | 2 |
| HSCT | 0 | 0 | 0 | 0 |
| exosomes | 1 | 0 | 0 | 1 |
| PRP | 0 | 0 | 0 | 0 |
Counts come from separate registry queries per class; a study testing two interventions can appear in more than one row, so rows do not sum to the all-interventions total.
Geography of the research
United States accounts for about 25% of country mentions among sampled studies; 4 countries host at least one site.
| Country | Studies with a site there |
|---|---|
| United States | 1 |
| Antigua and Barbuda | 1 |
| Israel | 1 |
| Iran | 1 |
The largest registered studies
Sorted by planned enrolment — size is not quality, but a 300-participant trial can answer questions a 12-participant series cannot.
| 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 |
| NCT00874783 — Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases | n/a | Recruiting | 120 | Hadassah Medical Organization | 2009-04 |
| NCT03067857 — Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease | Phase 1, Phase 2 | Unknown | 40 | Stem Cells Arabia | 2016-09 |
| NCT07105371 — Patients With ALS and Other Motor Disorders Will be Treated With Mesenchymal Cell Exosome Solution | Phase 1 | Completed | 18 | The Foundation for Orthopaedics and Regenerative Medicine | 2024-07-14 |
| NCT02855112 — Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients | Phase 1, Phase 2 | Unknown | 10 | Tehran University of Medical Sciences | 2015-06 |
Most recently registered
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT07105371 — Patients With ALS and Other Motor Disorders Will be Treated With Mesenchymal Cell Exosome Solution | n/a | Completed | n/a | The Foundation for Orthopaedics and Regenerative Medicine | 2024-07-14 |
| NCT03067857 — Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease | n/a | Unknown | n/a | Stem Cells Arabia | 2016-09 |
| NCT02574390 — Answer ALS: Individualized Initiative for ALS Discovery | n/a | Completed | n/a | Johns Hopkins University | 2015-12 |
| NCT02855112 — Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients | n/a | Unknown | n/a | Tehran University of Medical Sciences | 2015-06 |
| NCT00874783 — Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases | n/a | Recruiting | n/a | Hadassah Medical Organization | 2009-04 |
Reading this honestly
None of these numbers say the therapy works. They say how much organised research exists and how far along it is; effect sizes live in published results, not registry entries. For Spinal Muscular Atrophy specifically, the practical question is which registered study — if any — matches the product a provider is offering, and what its outcome measures actually are.
Frequently asked
How many stem-cell trials exist for Spinal Muscular Atrophy?
5 registered records mention a stem-cell intervention for this condition (2026-08-29 snapshot), of which 1 were recruiting.
Which therapy class is furthest along for Spinal Muscular Atrophy?
Ranked by Phase 3/4 records: MSC (0), HSCT (0), exosomes (0), PRP (0).
Does a recruiting trial mean I can be treated?
No. Trials have eligibility criteria, and joining one is not the same as buying a treatment. Paying a clinic for an unproven intervention outside a registered study gives you neither the protections nor the data collection of a trial.
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.
Download the query data
Query totals are full API matches; status, phase and country counts describe the first up to 400 returned records per query. Query classes overlap and must not be summed as unique trials. Counts are not treatment outcomes. Snapshot: 2026-08-29. Methods and field definitions.