Elizabeth Thiele, MD, PhD, director, pediatric epilepsy and the Herscot Center for Tuberous Sclerosis Complex, Massachusetts General Hospital, and professor of neurology, Harvard Medical School, recently presented data from the GWPCARE6 trial of cannabidiol (CBD, Epidiolex; GW Pharmaceuticals) at the 73rd annual meeting of the American Epilepsy Society (AES), December 6-10, 2019, in Baltimore, Maryland.

The data suggest that CBD can significantly reduce seizures associated with tuberous sclerosis complex (TSC), with similar efficacy observed between lower and higher doses of 25 mg/kg and 50 mg/kg. All told, those treated with CBD experienced reductions in TSC-associated seizure frequency of 49% with the 25 mg/kg dose and 48% for the 50 mg/kg dose, nearly double that of the placebo group, which experienced reductions of 27% (25 mg: P = .0009; 50 mg: P = .0018).

To find out more about the findings and the study itself, as well as the state of care in TSC and why there remains a clear unmet need for effective therapy in these patients, NeurologyLive sat with Thiele at AES 2019.

Rujuta Bhatt, MD of the ULCA Medical Center describes the pathophysiology of tuberous sclerosis complex as well as some of the more common symptoms that present with this rare disease.

An overview of data released at the AAN 2020 Annual Meeting on the safety and efficacy of cannabidiol for the treatment of seizures in patients with tuberous sclerosis complex.

Ryan Long with mother Jody Long and neurologist Mustafa Sahin in the Clinical and Translational Study Unit at Boston Children's Hospital. Ryan is participating in a Tuberous Sclerosis Complex (TSC) research study pioneered by Sahin. It is hoped that the drug being tested in this study, RAD001, will improve cognitive outcomes in patients with TSC, a genetically-based developmental disorder.