Unlock the Cure

Since its inception in 2011, Unlock the Cure has raised nearly $9 million for TSC research. The impact this campaign has had on our ability to build the framework for the next great medical breakthrough has been truly outstanding. You can find a full summary of the first phase of Unlock the Cure as well as a report on current progress here.

  Before campaign By end of campaign (December 31, 2014)
Research Grants Broad portfolio Focus on new TSC investigators and translational research
Drug Screening Two projects funded Six projects funded; 14 hits or combinations with interesting activity
Clinical Research Network Idea Running three clinical studies and part of Rare Diseases Clinical Research Network with SHANK3 and PTEN disorders
Biomarkers Idea Launched TSC Biosample Repository project (running two clinical studies), and funded four research projects
TSC Natural History Database 1,279 enrolled 1,504 enrolled and utilizing an updated research-friendly platform and five scientific articles published


In 2015, the TS Alliance entered phase two of this campaign, Unlock the Cure ACT (Accelerate, Collaborate, Target) Now! This phase will focus on driving collaborative science and building connections among the programs developed during the initial Unlock the Cure campaign. The result will be an inter-dependent research program where all components work together seamlessly to efficiently deliver new treatment options and continue to expand our understanding of TSC.


The next four-year campaign is focused on three distinct calls to action:

  • ACCELERATE research and the development of new treatments by seamlessly integrating all elements of our research platform.
  • COLLABORATE with the TSC community to shape research meaningful to their quality of life, to actively participate and to mobilize to raise awareness and funds.
  • TARGET specific research initiatives to advance toward preventative treatments and personalized medicine.

Our goal is to raise $25 million by the end of 2018, of which $5 million will be raised from private sources and $20 million leveraged from government sources directed to clinical research consortium projects (such as preventative clinical trials).

The Tuberous Sclerosis Alliance has been changing the lives of individuals and families in the TSC community by driving TSC research since 1984. The Unlock the Cure campaign created a research platform on key points along the drug discovery path:

  • Increasing funding of Research Grants to cultivate a new generation of scientists;
  • Creating a Preclinical Consortium to systematically test candidate drugs and move them toward clinical trials;
  • Expanding the Natural History Database to understand the progression of TSC throughout a lifetime;
  • Establishing a Biosample Repository as a resource to help researchers design personalized treatments in the future;
  • Developing a Clinical Research Consortium to rapidly conduct clinical studies, including the first preventative clinical trial for epilepsy in United States

Together, these programs build on the successful Unlock the Cure campaign by driving collaborative science and ultimately deliver new treatment options while expanding our understanding of TSC.