DALLAS, July 28, 2022 /PRNewswire/ -- Tonight during the opening of the 2022 World TSC Conference in Dallas, the Ramesh and Kalpana Bhatia Family Foundation announced a new collaboration with the TSC Alliance, working together to uncover biomarkers, test new treatments and connect researchers with families to better address TSC-associated neuropsychiatric disorders (TAND), which impact nearly everyone living with tuberous sclerosis complex (TSC). The $2.5 million foundational gift is the largest single donation in the TSC Alliance's history.
TSC is a rare genetic disorder that causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin and lungs. Changes in the brain caused by TSC have the biggest impact on quality of life, from seizures and developmental delays to intellectual disabilities, behavioral challenges and autism.
This new funding will create "Anya's Accelerator," which will focus on furthering TAND translational research. TAND includes a wide range of cognitive, behavioral and psychiatric challenges linked to the disease's effect on brain function. Autism spectrum disorder, intellectual disabilities, neuropsychological deficits, aggressive behaviors and more can severely affect people with TSC and their families. However, much is still unknown about TAND issues and how to effectively treat them, hence the need for more targeted research.
"The TSC Alliance is incredibly grateful to the Ramesh and Kalpana Bhatia Family Foundation for this historic donation," said Kari Luther Rosbeck, President & CEO of the TSC Alliance. "We know TSC-associated neuropsychiatric disorders are incredibly challenging for people with TSC and their loved ones, and this funding will give us the ability to start finding answers and, hopefully, identify effective interventions."
"Clinical trials for treating TAND are challenging for many reasons, particularly because TAND affects different people in different ways," explained Steven L. Roberds, PhD, Chief Scientific Officer at the TSC Alliance. "This program will work to overcome these challenges by developing ways to measure the most impactful aspects of TAND for each individual and identifying biomarkers to enable targeting of specific treatments for care of specific individuals."
The three-year "Anya's Accelerator" research program will focus on three major goals:
- Developing quantifiable patient-reported outcomes for the most impactful aspects of TAND utilizing the combined expertise of individuals and families living with TSC and clinical researchers;
- Identifying biomarkers and predictors of specific aspects of TAND through collaborative and inclusive analysis of existing samples and data; and
- Improving the translatability of and testing candidate drugs in TAND-relevant animal models by incorporating biomarkers or predictors relevant to those identified in humans.
"We are so grateful for the work and progress the TSC Alliance has made over the past 20 years. Anya's Accelerator will provide an opportunity to advance TAND research and innovation and offer hope for improved treatments, cures and prevention methods for those diagnosed with TSC and other neurological conditions," said Anita Bhatia, Executive Director, Ramesh and Kalpana Bhatia Family Foundation. "This is our foundation's most meaningful and personal partnership to date, as my daughter, Anya, was diagnosed with TSC at eight weeks old. Without the incredible work of the TSC community and the development of new treatments that were studied and approved, her livelihood would have been gravely different."
The TSC Alliance is an internationally recognized nonprofit that does everything it takes to improve the lives of people with TSC. It drives research, improves quality care, works to increase access and advocates for all affected by the disease. For more information, visit tscalliance.org.
The Ramesh and Kalpana Bhatia Family Foundation, founded in 2006, is a private family foundation established by Ramesh and Kalpana Bhatia. The Foundation is dedicated to making an impactful change around the world by funding innovative solutions and programs that enrich communities and serve the common good. The Foundation's grant making focuses on local, national and international organizations that support medical and healthcare research, education, mental health, intellectual and physical diversities, human rights and social justice, women and girls' empowerment and emergency responses. rkbhatiafoundation.org.
Senior Vice President, Strategic Communications
SOURCE TSC Alliance