Steve Chung, MD: Cannabidiol in Tuberous Sclerosis Complex


The neurologist at Banner-University Medicine Neuroscience Institute offered his perspective on data from an open-label safety assessment of cannabidiol (Epidiolex) in the treatment of tuberous sclerosis complex.

“The good news, again, is there weren’t any surprising side effects that we did not know about…in that sense, it is a very positive study showing sustained safety of this medication.”

Findings of an interim analysis of the phase 3 GWPCARE6 open-label extension trial (NCT02544750) of GW Pharmaceuticals’ cannabidiol (CBD; Epidiolex) were accepted for presentation at the American Academy of Neurology (AAN) 2020 Annual Meeting that demonstrated the therapy’s well-tolerated profile in patients with tuberous sclerosis complex (TSC) across a long-term treatment period.

Adverse events (AEs) were documented in 93% of patients, with 15% experiencing a serious AE, and 6% discontinuing due to AEs. The most common AEs, occurring in >20% of patients, were diarrhea, seizure, and decreased appetite, which were observed in 42%, 22%, and 20%, respectively. There was 1 death in the study deemed unrelated to treatment. The authors also noted that elevated alanine aminotransferase (ALT)/aspartate aminotransferase (AST) more than 3 times the upper limit of normal were reported in 8.5% (n = 17), with 12 of those patients on concomitant valproate.

To gain some outside perspective on how these data were interpreted in the clinical community, NeurologyLive spoke with Steve Chung, MD, neurologist, Banner-University Medicine Neuroscience Institute, who was not affiliated with the study, who shared his thoughts on the data and discussed what promise CBD might hold in the treatment of TSC.

For more coverage of AAN 2020, click here.


Thiele E, Bebin EM, Filloux F, et al. Long-term safety and efficacy of cannabidiol (CBD) for the treatment of seizures in patients with tuberous sclerosis complex (TSC) in an open-label extension (OLE) trial (GWPCARE6). Neurology. 2020;94(15 Suppl): 0677.

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