Elizabeth Thiele, MD, PhD: GWPCARE6 Trial of CBD in Tuberous Sclerosis Complex

“There’s a significant unmet need for effective treatments. Over the past 10 to 15 years, we’ve had many new treatments come out, but we all know that those treatments, unfortunately, haven’t really decreased the incidence of refractory epilepsy.”

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 73^rd 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.

For more coverage of AES 2019, click here.

REFERENCE

Thiele E, Wong M. Cannabidiol (CBD) treatment in patients with seizures associated with tuberous sclerosis complex: a randomized, double-blind, placebo-controlled phase 3 trial (GWPCARE6). Presented at: AES 2019; December 6—10; Baltimore, Maryland. Abstract 1.293.