Rapamycin，亦称sirolimus，成了后来肾脏移植的标准排异抑制药物。在过去5 年，科学家揭示了rapamycin的机理，即它用mTO束缚和转动蛋白质R并随后停止细胞生长和蛋白质合成因此目标细胞异常。去年，从事Tuberous Sclerosis研究的科学家表明引起基因TSC的二个蛋白质为，tuberin和hamartin，并在检测中直接或间接的抑制mTOR活性。因此，Tuberous Sclerosis患者的tuberin或hamartin异常，mTOR活动会增加以致细胞生长失控最终导致hamartomas。一个治疗Tuberous Sclerosis患者的想法是恢复细胞生长控制并消除hamartomas。Rapamycin，因为它能控制mTOR，是Tuberous Sclerosis患者的一种mTOR调控方案。一个临床试验测试将会进行如果成功，将非常迅速，使这种药物用于大部分Tuberous Sclerosis患者。

Rapamycin, also known as sirolimus, has become a standard immunosuppressive drug following kidney transplant. In the past 5 years, scientists have uncovered the mechanism of action for rapamycin, namely that it binds and turns off a protein called mTOR and subsequently shuts down cell growth and protein synthesis so target cells stop working properly. Last year, scientists working on Tuberous Sclerosis have shown that the two causative genes for TSC, tuberin and hamartin also serve to, directly or indirectly, keep mTOR activity in check. Consequently, in patients with Tuberous Sclerosis where either tuberin or hamartin is broken, mTOR activity is increased and cells grow in a somewhat unconstrained fashion with resulting hamartomas. One treatment goal for patients with Tuberous Sclerosis would be to regain control of cell growth and eliminate the hamartomas. Rapamycin, because it can control mTOR, may be a useful solution for Tuberous Sclerosis patients where mTOR is dysregulated. A clinical trial to test this hypothesis will be shortly underway and a successful outcome can, very expediously, make this drug available to all patients with Tuberous Sclerosis.