Antiepileptic effect and safety profile of Rapamycin in children with TSC

About this study

Treatment with mTOR pathway inhibitors is important in drug resistant epilepsy (DRE). This study sought to evaluate the safety and efficacy of 12 months of Rapamycin treatment in 32 paediatric patients aged from 11 months to 14 years, all of whom had drug-resistant TSC-associated epilepsy.

The data was collected in clinical practice, with subjective data reporting by the children and their families.

Outcomes of the study

After 6 months of treatment with Rapamycin the researchers found that there was at least a 50% reduction in the number of seizures per week in 18 of the children, no change occurred in another 12, and seizure frequency had worsened in 2 children.

After 12 months, two-thirds of the children had at least a 50% reduction of seizure frequency.  28% of the children achieved complete remission from seizures.  The authors noted a slightly better effect in the group with the TSC1 gene mutation.

The team found that using both Vigabatrin and Rapamycin at the same time was favourable, as it led to better treatment outcomes.


The study showed that there is value in staying Rapamycin for at least 6-12 months as the therapeutic benefit was linear and seemed to increase the longer that patients were on the drug.

While the therapeutic effect and safety profile of Rapamycin was satisfactory, treatment did not precipitate a spectacular or quick improvement by any means and the authors suggest more studies, especially randomised controlled trials are needed.

It is also important to note that this study was conducted on a small cohort with a wide age range.

Antiepileptic Effect and Safety Profile of Rapamycin in Pediatric Patients With Tuberous Sclerosis Complex.Sadowski K, Sijko K, Domańska-Pakieła D, Borkowska J, Chmielewski D, Ulatowska A, Józwiak S, Kotulska K.Front Neurol. 2022 Apr 29;13:704978. doi: 10.3389/fneur.2022.704978. eCollection 2022.PMID: 35572924 Free PMC article.

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This information is intended to provide some insights into recent TSC-related research.  It is not intended to, and it should not, constitute medical or other advice.  Readers are warned not to take any action without first seeking medical advice.