Summary
Nicotine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. ClinicalTrials.gov lists 1 registered trial linking Nicotine to Epilepsy: 1 has completed. None of the registered studies carries a drug-development phase label, which is typical for behavioural, device and supplement protocols. It plans or enrolled 33 participants. Registered activity dates to 2024. The literature layer holds 9 publications for this pair: 2 Cochrane reviews, 1 meta-analysis, 3 systematic reviews and 3 clinical trial publications. Publication years run from 2003 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Epilepsy ranks 187 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: 60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by syndrome and duration of freedom).
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 51.5 (trials 2.5, literature 29.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 0 recruiting, 0 active / not yet recruiting, 1 completed, 0 other |
| Linked publications | 9 (3 systematic reviews, 3 clinical trial publications, 2 Cochrane reviews, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | ChEMBL |
| Linked via biomarker / target | AARS1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT06427967 | A Novel Social Emotional Learning Curriculum for Youth With Epilepsy 2024 | completed | Not applicable | 33 |
Published literature
- Systematic review Nicotine as target-therapy in Sleep-Related Hypermotor Epilepsy due to nAChR genes mutation: case report and systematic review of literatureTiscornia M, Bosisio L, Torta F et al. · Sleep medicine · 2026 · PMID 41192108
- Meta-analysis Exploring the Smoking-Epilepsy Nexus: a systematic review and meta-analysis of observational studies : Smoking and epilepsyKang Y, Kim S, Jung Y et al. · BMC medicine · 2024 · PMID 38433201
- Cochrane review How do smoking, vaping, and nicotine affect people with epilepsy and seizures? A scoping review protocolNarrett JA, Khan W, Funaro MC et al. · PloS one · 2023 · PMID 37418386
- Systematic review Sudden Death in Adults Caused by Intracranial Pathology: A Systematic ReviewBarbosa M, Junior J, Esmeraldo A et al. · Turkish neurosurgery · 2023 · PMID 36799274
- Cochrane review Use of Topiramate in the Spectrum of Addictive and Eating Disorders: A Systematic Review Comparing Treatment Schemes, Efficacy, and Safety FeaturesNourredine M, Jurek L, Angerville B et al. · CNS drugs · 2021 · PMID 33591567
- Systematic review Nicotine: A Targeted Therapy for Epilepsy Due to nAChR Gene VariantsFox J, Thodeson DM, Dolce AM · Journal of child neurology · 2021 · PMID 33284031
- Clinical trial publication Nicotine increases sleep spindle activityO'Reilly C, Chapotot F, Pittau F et al. · Journal of sleep research · 2019 · PMID 30565327
- Clinical trial publication Impact of an electronic cigarette on smoking reduction and cessation in schizophrenic smokers: a prospective 12-month pilot studyCaponnetto P, Auditore R, Russo C et al. · International journal of environmental research and public health · 2013 · PMID 23358230
- Clinical trial publication Nicotine as an antiepileptic agent in ADNFLE: an N-of-one studyWilloughby JO, Pope KJ, Eaton V · Epilepsia · 2003 · PMID 12919397
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 ChEMBL, in this case via the biomarker or target AARS1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Nicotine approved for Epilepsy?
Nicotine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Nicotine in clinical trials for Epilepsy?
ClinicalTrials.gov lists 1 registered trial linking Nicotine to Epilepsy: 1 has completed. None of the registered studies carries a drug-development phase label, which is typical for behavioural, device and supplement protocols. It plans or enrolled 33 participants. Registered activity dates to 2024.
What does the evidence show for Nicotine in Epilepsy?
Nicotine has both completed registered trials and synthesis-level publications linked to Epilepsy. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in ChEMBL, in this case via the biomarker or target AARS1. 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 2026-07-05. Page built 2026-10-07.