Since our humble beginnings, the TS Alliance has funded nearly $21.2 million in support of basic, translational and clinical research. The funding has supported research on the genetics of TSC, basic mechanisms of both tumor growth and central nervous system manifestations of TSC, translational research, and clinical trials, as well as the development of research tools such as biomarkers, animal models, and a database of TSC1 and TSC2 genetic variants. TS Alliance-funded research continues to play a vital role by encouraging and fostering collaborations between basic and clinical researchers.
As part of our advocacy for research and promoting young investigators, we funded and cultivated a new generation of senior scientists. By supporting small, short-term research projects we have enabled researchers to produce data on which to build applications for funding of much larger projects from other sources. This seed funding, combined with a dedicated Government Advocacy Team pressing for Federal research funding, has leveraged an additional $347 million in research grants in the past 17 years from the National Institutes of Health (NIH) and the Department of Defense’s Tuberous Sclerosis Complex Research Program (TSCRP).
In our quest for a cure, funds provided by the TS Alliance for more than a decade have contributed to a number of significant scientific breakthroughs. TS Alliance-sponsored research helped support the identification of the two genes that cause TSC, develop a genetic test and initiate the first clinical trials in TSC. Today, because of this very early research and basic scientific understanding of the disease, there are currently two FDA-approved indications for TSC – one for the treatment of brain tumors and the other for the treatment of kidney tumors common in TSC. As a critical driver of TSC research around the world, the TS Alliance has been an active collaborator in amazing advances, including the discovery of a predictive biomarker of which infants with TSC would develop epilepsy leading to the first ever preventative clinical trial for epilepsy in the United States. This same study design may be able to be replicated in many other genetic causes of epilepsy and for other manifestations of TSC.