The Tuberous Sclerosis Alliance selects PsychoGenics to perform drug screening for the TSC Preclinical Consortium
The Tuberous Sclerosis Alliance (TS Alliance) is facilitating the creation of a Preclinical Consortium with academia and industry partners to test drugs in validated models of tuberous sclerosis complex (TSC). The Consortium’s initial focus is on epilepsy and tumors associated with TSC. The TS Alliance has recently signed an agreement with PsychoGenics, Inc., to establish the necessary drug screening platforms on behalf of the Consortium. Other partnerships will also be established in the coming months to cover other preclinical needs. Meet TS Alliance at the 2016 BIO International Convention to discuss shared interests and partnership opportunities or contact us.
About TSC: TSC is a genetic disorder that can be inherited from one parent with TSC or can result from a spontaneous genetic mutation. TSC causes tumors in many different organs such as brain, eyes, heart, kidney, skin and lungs, but aspects of TSC that most strongly impact quality of life are associated with the brain: seizures, developmental delay, intellectual disability and autism. At least two children born each day will have TSC. Current estimates place tuberous sclerosis complex-affected births at one in 6,000. Nearly 1 million people worldwide are estimated to have TSC, with approximately 50,000 in the United States.
Two genes have been identified that can cause tuberous sclerosis complex: the TSC1 hamartin gene, and the TSC2 tuberin gene. Both the TSC1 and TSC2 genes suppress tumor growth in the body by regulation of the mTOR pathway. In the fall of 2010, the FDA approved the first drug for TSC to treat subependymal giant cell astrocytomas (a type of brain tumor). In 2012, the same drug was approved to treat angiomyolipomas (a type of kidney tumor). Major advancements in treatments such as these require preclinical studies to test the effectiveness of experimental drugs in animal models of TSC. With the TSC Preclinical Consortium, prioritization of candidate treatments will be achieved based on comparing head-to-head data using consistent animal models and testing procedures. The TS Alliance is ideally positioned to drive this collaboration—with input from academic, regulatory, and industry stakeholders—by maintaining constant focus on the needs of people with TSC.
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