Development of Remission-Inducing Therapy for TSC Tumors

Tuberous sclerosis complex (TSC) is a rare genetic disease that causes benign tumors to grow in many different organs of the body. Although TSC tumors are not metastatic, these tumors can still cause problems, some critical, when the growth is located in vital organs such as the brain, kidney, and lung. Currently, there is no cure for TSC and treatment is largely devoted to surgical removal of tumors. Sometimes drugs such as rapamycin or related drugs are used to keep tumors from growing. Unfortunately, rapamycin only stops the tumor growth, does not make the tumors go away, and tumors enlarge further as soon as rapamycin is discontinued. In addition, long-term treatment can lead to side effects, such as diabetes, and resistance. In other words, TSC is an incurable lifelong disease, and the current treatments available only stop disease progression. They are not a cure. It is imperative to develop a new therapeutic approach that can only eliminate tumor rather just block growth but also do so without major side effects.