Everolimus drug study with TSC patients to measure impact on brain
Tuberous Sclerosis patients are planned to benefit from a $15,000 check presented by to Dr. Orrin Devinksy, Director of the Comprehensive Epilepsy Center at NYU Langone Medical Center. The donation was given by Bcureful ® board members Lori Nerheim of Chicago and Laurie Marshall of New York, in support of the upcoming everolimus research project at NYU Langone Medical Center.
“Now is one of the most exciting times in TSC research,” said Dr. Devinsky. “Among all disorders, I think TSC is one in which medical research has made some of the greatest advances in the past decade. We have identified the two TSC genes, we know where they are, we know what proteins they code for… and hope for a cure for TSC is realistic.”
This study will look at the effects of the everolimus chemotherapy drug and its impact on the brain before TSC patients undergo brain surgery. Approval by the FDA in August 2012 was based on a greater than 50% reduction in tumor volume in trial patients. The goal of this study is to understand the effects of the drug on the basic problem in TSC which is the “brakes” are removed on a critical pathway that regulates cell growth. Understanding how everolimus “puts the brakes back” on this pathway can help us to improve existing therapies, identify new targets, and hopefully understand how this drug can help control seizures.
Julie Scroggins, Founder and Chair of Bcureful, shared “Bcureful’s mission is to accelerate research towards a cure for TSC and we are proud to make this award to NYU Langone Medical Center for this very promising study.”
“We are committed to raising the additional $85,000.00 needed to fully fund this research project and improve the future for so many affected by TSC and related conditions,” added Scroggins.
Bcureful is a 501(c)(3) non-profit organization founded to focus resources on Tuberous Sclerosis Complex, or TSC. TSC is a genetic disorder that affects 1 in 6,000 newborns causing non- malignant tumors to develop in the brain and other vital organs. Although the growths are almost always benign, they can cause devastating effects on multiple aspects of development. It is estimated that 1 to 2 million people worldwide have TSC. Research for TSC has led to breakthroughs in autism, epilepsy and cancer.
To learn more about Bcureful and make a donation, visit: www.Bcureful.org