mTOR inhibitors are a type of drug which interrupt the chemical reactions needed for tumours to grow. When researchers realised reducing the activity of mTOR proteins could treat TSC, many pharmaceutical companies were already trying to develop mTOR inhibitors to treat cancers.
TSC patient groups funded small pre-clinical investigations into mTOR inhibitors with successful results, so the idea was taken to small clinical trials. In 2006, a small clinical trial in the US tested the impact of mTOR inhibitors on four TSC patients with SEGA (a type of brain tumour). The results showed significant reduction in tumour size and improvement in patient outcomes over a 2.5 to 20 month period.
Another phase II trial investigated the use of sirolimus, an mTOR inhibitor, on TSC patients with angiomyolipoma (a type of kidney tumour). This research was funded by the LAM Foundation and the TSA and it had successful outcomes.
The TSA used the results of these successful research projects to approach pharmaceutical companies, asking them to include TSC in their investigations into the mTOR inhibitors they were working on.