Unlock the Cure

In 2011, the TS Alliance initiated the Unlock the Cure campaign, a vital step toward implementing the next generation research program needed to move potential treatments along the path from the lab (pre-clinical) to the tuberous sclerosis complex (TSC) community (clinical trials) more rapidly and efficiently.

The TS Alliance’s research strategy focused on key points along the drug discovery path:

  • Increasing funding of TS Alliance-Supported Research to ensure we have a pipeline for basic and translational studies to feed our Drug Discovery Program.
  • Advancing our Drug Screening Program to identify FDA-approved drugs that can be “repurposed” for treatment in TSC and to uncover new compounds that could lead to new treatments.
  • Developing a Clinical Research Network to expedite implementation of future clinical trials and recruitment of individuals for clinical studies.
  • Identifying Biomarkers needed for future clinical trials, which will be a requirement for measuring progression of disease and effectiveness of new treatments.
  • Expanding the TSC Natural History Database to help identify lifelong trends in the physical traits of TSC to improve quality of life and long-term outcomes as well as to develop a method of quickly identifying individuals for future clinical trials.

The first phase of Unlock the Cure made tremendous strides across each of the five focus areas:

  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


Since its inception, Unlock the Cure has raised nearly $5.35 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.

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).

As part of this effort the TS Alliance will invest in two new research initiatives, in addition to those detailed previously, to further advance research beyond the first phase of Unlock the Cure:

  1. To build upon findings from the Drug Screening program, the TS Alliance is creating a Preclinical Consortium to test identified candidate drugs for new treatments in a collaborative and systematic manner.
  2. To facilitate research on key questions relevant to developing personalized therapies, the TS Alliance is coordinating the collection of blood and tissue samples at a Biosample Repository that will work in tandem with the TSC Natural History Database.