SILVER SPRING, Md., Nov. 29, 2018 /PRNewswire/ -- Today, the Tuberous Sclerosis Alliance (TS Alliance) announced a new staff member to drive its tuberous sclerosis complex (TSC) Preclinical Consortium. Dean Aguiar, PhD, has joined the TS Alliance as Director of Preclinical Research to strengthen the consortium's capabilities. 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.
Affecting 50,000 in United States and 1 million worldwide, TSC is a rare genetic disorder that causes tumors to form in vital organs, including the heart, brain, eyes, kidneys, skin, liver and lungs. It's also a leading genetic cause of both epilepsy and autism.
Dr. Aguiar brings more than 17 years of research and development in biopharmaceutical and medical device industries experience to the TS Alliance. In his prior role as Program Director at The Hartwell Foundation, he gained significant experience in pediatric disease from oncology and inflammation to neurodevelopmental disorders including autism, ADHD and epilepsy. Dr. Aguiar provided guidance to academic investigators regarding the scientific evidence and regulatory path required to successfully translate technologies toward commercial viability. He also established partnerships with industry to gain access to proprietary drugs and a possible path for licensing. Dr. Aguiar earned his PhD in Biochemistry from Rush University at Rush Presbyterian St. Luke's Medical Center in 1996 and completed post-doctoral training at the University of Minnesota.
"We are truly excited to have Dean on board to take the Preclinical Consortium to the next level," said TS Alliance President & CEO Kari Luther Rosbeck. "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."
The TS Alliance's Preclinical Consortium, established in 2015, evaluates ideas for new treatments in animal models relevant to TSC. Academic and pharmaceutical industry researchers use data generated by the consortium to make decisions on which new ideas are worthy of advancing toward testing in humans. The consortium is coordinated and wholly funded by the TS Alliance thanks to generous financial support by the Gerry and Bill Cowlin Foundation, the Engles Family Foundation on behalf of the Engles Collaborative Research Fund, the consortium's industry members and other donors.
"In partnership with academic members of the consortium, the TS Alliance selected the most robust, reproducible and translatable animal models since no single model fully recapitulates all features of TSC in humans," explained Steven L. Roberds, PhD, TS Alliance Chief Scientific Officer. "The TS Alliance holds licenses to use these models for testing compounds on behalf of academic investigators and for-profit companies and oversees execution of candidate drug testing at contract research labs including PsychoGenics and Porsolt."
The TS Alliance finances the cost of testing compounds nominated by academic members, with data shared among all consortium members. Company members may also finance testing of proprietary compounds and retain exclusive use of those data. Since running its first experiment in August 2016, the consortium has tested 21 compounds in mouse models of epilepsy or tumor growth, and many more are in the queue.
The Tuberous Sclerosis Alliance is the only US-based nonprofit dedicated to finding a cure for tuberous sclerosis complex while improving the lives of those effected. Founded in 1974, it works to improve quality of life for individuals and families affected by TSC by stimulating and sponsoring research; creating programs, support services and resource information; and developing and implementing public and professional programs designed to heighten awareness of the disease. For more information, visit www.tsalliance.org.