Summary
Cyclosporine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Amyotrophic Lateral Sclerosis (ALS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Amyotrophic Lateral Sclerosis (ALS). ClinicalTrials.gov lists 1 registered trial linking Cyclosporine to Amyotrophic Lateral Sclerosis (ALS): 1 is currently recruiting. None of the registered studies carries a drug-development phase label, which is typical for behavioural, device and supplement protocols. It plans or enrolled 8 participants. Registered activity dates to 2025. The literature layer holds 2 publications for this pair: 2 randomised controlled trial publications. Publication years run from 1988 to 1989. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
The RepurpOS disease-intelligence file for Amyotrophic Lateral Sclerosis ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Essentially zero — uniformly progressive, fatal within 2-5 years median; <10% survive >10 years; spontaneous prolonged stabilisation in <1% (SOD1 heterozygous late-onset).
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 29.0 (trials 3.0, literature 6.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 1 recruiting, 0 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 2 (2 randomised controlled trial publications) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | ATXN2 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT07539662 | Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS) 2025 | enrolling by invitation | Not applicable | 8 |
Published literature
- Randomized controlled trial Effects of cyclosporine therapy on plasma lipoprotein levelsBallantyne CM, Podet EJ, Patsch WP et al. · JAMA · 1989 · PMID 2733125
- Randomized controlled trial A double-blind study of the effectiveness of cyclosporine in amyotrophic lateral sclerosisAppel SH, Stewart SS, Appel V et al. · Archives of neurology · 1988 · PMID 3281637
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ATXN2. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Cyclosporine approved for Amyotrophic Lateral Sclerosis (ALS)?
Cyclosporine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Amyotrophic Lateral Sclerosis (ALS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Amyotrophic Lateral Sclerosis (ALS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Cyclosporine in clinical trials for Amyotrophic Lateral Sclerosis (ALS)?
ClinicalTrials.gov lists 1 registered trial linking Cyclosporine to Amyotrophic Lateral Sclerosis (ALS): 1 is currently recruiting. None of the registered studies carries a drug-development phase label, which is typical for behavioural, device and supplement protocols. It plans or enrolled 8 participants. Registered activity dates to 2025.
What does the evidence show for Cyclosporine in Amyotrophic Lateral Sclerosis (ALS)?
Trials of Cyclosporine in Amyotrophic Lateral Sclerosis (ALS) are registered but none has completed, so there is no outcome evidence from those studies yet; the record is a signal of research interest. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target ATXN2. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.