Elizabeth Thiele, MD, PhD

“The higher dose wasn’t way more effective than the lower dose, and that suggests that, for the majority of patients, the lower dose should be effective. I think that’s good because a lower dose of medication usually means better tolerability.”

, or CBD (Epidiolex; GW Pharmaceuticals) at the 

 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 inquire more about the findings of the trial, 

 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.

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.

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The director of pediatric epilepsy and the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, and professor of neurology at Harvard Medical School spoke to the safety outcomes from GWPCARE6 and CBD’s low-dose efficacy.

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

“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 

, December 6-10, 2019, in Baltimore, Maryland.

 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: 

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, 

The director of pediatric epilepsy and the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, and professor of neurology at Harvard Medical School discussed the findings of the GWPCARE6 trial of cannabidiol.

Author | Elizabeth Thiele, MD, PhD

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

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