TSC Alert

Welcome to Our New Director of Preclinical Research

Please help us welcome Dean Aguiar, PhD, to the TS Alliance team! Dean is our new Director of Preclinical Research. He will lead the Preclinical Consortium’s future efforts, including the goal to partner with additional companies and academic researchers to help find new ways to treat the multiple, severe and sometimes life-threatening manifestations of TSC. He will also oversee other consortium objectives, including increasing the diversity of models available as well as adding models for TSC-associated neuropsychiatric disorders, an additional epilepsy model and human cell-based models.

What is the TSC Preclinical Consortium?

The Preclinical Consortium engages academic and pharmaceutical industry researchers to help accelerate development of new therapies for TSC. A Steering Committee sets the long-term goals and priorities. Working Groups decide the best models and experimental designs for drug testing and provide oversight for rigorous quality control and interpretation of results. By using robust and reproducible models, protocols, and assays, the Preclinical Consortium ensures data are comparable from experiment to experiment. Compounds are handled and tested by experienced providers, and data are shared with all members of the consortium.

Members belong to industry, academia, or nonprofit organizations. All members can propose compounds for the screen and benefit from the shared data. Additionally, Company Members may submit compounds for testing and retain confidentiality and ownership of data by paying for experiments as defined in the Consortium Operating Agreement.

Since its inception, nine companies have joined the consortium to test candidate drugs, among them Aeonian Pharmaceuticals, Cavion, Greenwich Biosciences, Ovid, PIQUR Therapeutics and UCB.

Submit Compounds to Test

The TSC Preclinical Consortium is soliciting nominations for compounds to test in one or more of three robust mouse models of TSC manifestations. Though nominations are accepted year-round, submissions received on or before December 15 will be evaluated by February 15, 2019. Nominations received after December 15 will be accumulated and evaluated after June 15.

To submit a nomination, please read through the instructions and fill out the nomination form.

Compounds nominated may include:

  • Drugs with a rationale for re-purposing to treat TSC and which are in clinical development or marketed for other indications.
  • Analogs of existing drugs which may have improved efficacy or safety.
  • Tool compounds to investigate novel mechanisms of action.