Elizabeth Thiele, MD, PhD: CBD’s Low-Dose Efficacy and Safety in TSC

Recently presented data from the GWPCARE6 trial of cannabidiol, or CBD (Epidiolex; GW Pharmaceuticals) at the 73rd annual meeting of the American Epilepsy Society (AES), December 6-10, 2019, in Baltimore, Maryland, suggested that the treatment may be effective and safe in patients with tuberous sclerosis complex. The agent was approved in June 2018 for the treatment of Lennox-Gastaut and Dravet syndromes, so its efficacy in an additional condition is welcome for the epilepsy community.

To find out more about the findings of the trial, NeurologyLive sat with investigator 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, at AES 2019. She offered insight into the safety outcomes from the study and discussed how they compare to prior observations.

Notably, she mentioned that the low dose of 25 mg/kg showed a similar level of efficacy to the higher 50 mg/kg dose, suggesting that many patients may be able to get benefit from the therapy while avoiding a higher likelihood of experiencing the adverse events which can accompany higher dose treatments. Thiele also shared some insight into her clinical experience with the therapy, both on and off label.