Potential therapeutic effect of curcumin, a natural mTOR inhibitor, in tuberous sclerosis complex.

Background: Curcumin is a polyphenol natural product of the plant Curcuma longa. Recent studies suggest that curcumin inhibit mTOR activity in vitro, which prompts us to investigate curcumin function as a new class of mTOR inhibitor suitable for tuberous sclerosis complex (TSC) treatment.

Purpose: We aim to investigate the efficacy of curcumin in the treatment of TSC related manifestations in animal model.

Study Design: Solid lipid curcumin particle (SLCP), a novel curcumin formulation, was used to treat TSC related manifestations in Tsc2 knockout mice.

Methods: The novel object recognition test was used to analyze the recognition memory function. The long-term potentiation was studied using electrophysiological analysis. Western blotting was used to assess the protein expression and activation status.

Results: Recognition memory deficit began as early as 4 weeks of age in both male and female Tsc2 mice. Oral administration with SLCP activates AMPK activity and inhibits mTOR activity in the brain tissue of Tsc2 mice, and can rescue the electrophysiological abnormality and object recognition memory loss in the mice.

Conclusions: Our results suggest that SLCP could be an effective treatment for TSC patients.