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Everolimus for the treatment of neuropsychological deficits in tuberous sclerosis complex: findings from the TRON multicentre randomised controlled trial

Saxena A, Drew CJ, Cannings-John R, Pickles T, Mills LJ, Sampson JR et al. · 2026 · Journal of medical genetics · Atlas ID SAX2026

What this study shows

In a multicentre RCT of TSC patients (n=38 randomised, 2:1 everolimus:placebo), 24 weeks of everolimus produced similarly large 'responder' rates for neuropsychological improvement as placebo (87% vs 75%), suggesting large practice/placebo effects rather than a clear drug benefit on cognition -- despite everolimus's established efficacy for TSC tumours and epilepsy. Adverse events were more frequent with everolimus (88% vs 61.5%).

Abstract

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Mammalian target of rapamycin (mTOR) inhibitors are effective treatments for tumours and epilepsy in tuberous sclerosis complex (TSC). This study aimed to determine the effects of the mTOR inhibitor everolimus and a placebo on neuropsychological functioning in TSC. Individuals with TSC aged 16-60 years and IQ>60 who scored <=5th percentile in one or more of 10 memory or executive function variables were randomised 2:1 to 24 weeks everolimus or placebo and retested at baseline and 4, 12, 24 and 36 weeks.

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At a glance

Evidence type H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type2 - Human Clinical Trial
Model systemHuman (TSC patients, aged 16-60, RCT)
JournalJournal of medical genetics
Year2026
Peer reviewedYes
Record last updated2026-08-22
SourceDOI 10.1136/jmg-2026-111529 · PMID 42562625

Extracted findings

InterventionEverolimus (mTOR inhibitor) vs placebo, 24 weeks, randomised 2:1
TargetmTOR (mTORC1)
ModelHuman (TSC patients, aged 16-60 years)
EffectNo clear benefit of everolimus over placebo on neuropsychological function; both arms showed large responder rates (87% vs 75%), likely reflecting practice/placebo effects rather than a true drug effect

In the Atlas

Related topics

EverolimusTuberous sclerosis complex

Open questions that cite this study

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Cite this paper

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Saxena, A., et al. (2026). Everolimus for the treatment of neuropsychological deficits in tuberous sclerosis complex: findings from the TRON multicentre randomised controlled trial. Journal of medical genetics. https://doi.org/10.1136/jmg-2026-111529

@article{SAX2026,
  author       = {Saxena, A. and Drew, C. J. and Cannings-John, R. and Pickles, T. and Mills, L. J. and Sampson, J. R. and others},
  title        = {{Everolimus for the treatment of neuropsychological deficits in tuberous sclerosis complex: findings from the TRON multicentre randomised controlled trial}},
  journal      = {Journal of medical genetics},
  year         = {2026},
  doi          = {10.1136/jmg-2026-111529},
  note         = {PMID: 42562625},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper. 

Barton, O. (2026). Oliver's mTOR Atlas (record SAX2026) [Data set]. https://mtor-atlas.org/study/SAX2026/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_SAX2026,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record SAX2026},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/SAX2026/},
  doi          = {10.5281/zenodo.22059963}
}