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Published on: December 18, 2016
The mTOR pathway in treatment of epilepsy: a clinical update
Jennifer L Griffith1, Michael Wong1
1Department of Neurology, Washington University School of Medicine, St. Louis, MO 63110, USA.
Abstract:
Nearly a third of patients with epilepsy have seizures refractory to current medical therapies. In the search for novel drug targets, the mTOR pathway has emerged as key in the regulation of neuronal function, growth and survival, and other cellular processes related to epileptogenesis. Hyperactivation of the mTOR pathway has been implicated in tuberous sclerosis complex and other 'mTORopathies', clinical syndromes associated with cortical developmental malformations and drug-resistant epilepsy. Recently published clinical trials of mTOR inhibitors in tuberous sclerosis complex have shown that these drugs are effective at decreasing seizure frequency. Future studies may establish whether mTOR inhibitors can provide effective treatment for patients with diverse genetic and acquired epilepsies, including preventative, disease-modifying therapies.
Insights
Drug targets for epilepsy are being explored via the mTOR pathway. mTOR inhibitors show promise in reducing seizures for specific conditions and may offer future epilepsy treatments.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Epilepsy affects many patients with drug-resistant seizures.
- The mTOR pathway is crucial for neuronal function and implicated in epileptogenesis.
- Hyperactivation of the mTOR pathway is linked to tuberous sclerosis complex and related epilepsies.
Purpose of the Study:
- To explore the role of the mTOR pathway as a potential drug target for epilepsy.
- To review the efficacy of mTOR inhibitors in treating drug-resistant epilepsy.
Main Methods:
- Review of recent clinical trials and scientific literature on mTOR pathway and epilepsy.
- Analysis of mTOR pathway's role in cellular processes related to epileptogenesis.
Main Results:
- Clinical trials of mTOR inhibitors in tuberous sclerosis complex demonstrate reduced seizure frequency.
- The mTOR pathway is a validated target in specific epilepsy syndromes.
Conclusions:
- mTOR inhibitors are effective in managing seizures in certain mTORpathies.
- Further research is warranted to assess mTOR inhibitors for diverse epilepsies, potentially as preventative or disease-modifying therapies.
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