Abstract Details

Title Epidiolex (Cannabidiol) in Treatment Resistant Epilepsy

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) (-)

Poster/Presentation
Number 005

Objective

Background Cannabidiol (CBD) is a component of Cannabis sativa with anticonvulsant activity in pre-clinical models of epilepsy, independent of activity at known endogenous cannabinoid receptors.

Design/Methods Data has been collected on demographics, seizure counts, and safety through case report forms and tabulated in this series of open-label trials. Eligibility was determined and documented in protocols specific to each site after FDA and IRB review. Seizures were recorded as convulsive (countable) or non-convulsive. Atonic seizures were also specifically recorded. CBD (supplied by GW Pharmaceuticals) was given as a liquid and daily dose titrated up to 25mg/kg.

Results Data were collected on 213 patients with treatment-resistant epilepsies for safety evaluation. 137 patients had at least 12 weeks continuous exposure and were included in efficacy calculations. Etiologies included Dravet and Lennox-Gastaut (LGS) syndromes as well as over 10 other conditions. Total convulsive and non-convulsive seizures showed a median percent reduction from baseline of -53.7% at week 12. Convulsive seizure frequency among Dravet patients treated for at least 12 weeks (N=23) was reduced by 52.7% at week 12. In patients with LGS treated for 12 weeks (N=11), atonic seizure frequency was reduced by a median 54.5% at week12. Adverse events >10% were somnolence (21%), diarrhea (17%), fatigue (17%), and decreased appetite (16%). Nine patients (4%) discontinued for AEs.

Conclusions CBD showed reductions in seizure frequency across multiple drug-resistant epilepsy syndromes and seizure types and was generally well-tolerated in this open-label cohort. Controlled trials are indicated to characterize efficacy and safety.

Authors/Disclosures
Orrin Devinsky, MD, FAAN (NYU Epilepsy Center)	Dr. Devinsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for California Cannabis Enterprises. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Privateer Holdings. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Receptor Holdings. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rettco. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Silver Spike. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Tevard. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Tilray. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Tevard Biosciences. Dr. Devinsky has stock in Engage. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in California Cannabis Enterprises. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in Silver Spike. Dr. Devinsky has stock in Docklight Brands. Dr. Devinsky has stock in Egg Rock/Papa Barkley. Dr. Devinsky has stock in Leafly. Dr. Devinsky has stock in Left Coast Ventures. Dr. Devinsky has stock in Privateer Holdings. Dr. Devinsky has stock in Receptor Holdings. Dr. Devinsky has stock in Qstate. The institution of Dr. Devinsky has received research support from GW Pharma. The institution of Dr. Devinsky has received research support from Zogenix. The institution of Dr. Devinsky has received research support from PTC Pharma. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from DOD. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from Epilepsy Foundation. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NSF. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received publishing royalties from a publication relating to health care.
	No disclosure on file
Daniel Friedman, MD (NYU Langone Medical Center)	The institution of Dr. Friedman has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Epilepsy Study Consortium (non-profit). Dr. Friedman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Meili Technology. Dr. Friedman has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurelis. Dr. Friedman has stock in Neuroview Technology. The institution of Dr. Friedman has received research support from NIH. The institution of Dr. Friedman has received research support from CDC. The institution of Dr. Friedman has received research support from Epitel. The institution of Dr. Friedman has received research support from Neuropace. Dr. Friedman has received intellectual property interests from a discovery or technology relating to health care. Dr. Friedman has received intellectual property interests from a discovery or technology relating to health care. Dr. Friedman has received publishing royalties from a publication relating to health care. Dr. Friedman has received personal compensation in the range of $500-$4,999 for serving as a Speaker Honorarium with SK Life Sciences. Dr. Friedman has received personal compensation in the range of $500-$4,999 for serving as a Speaker Honorarium with AAN. Dr. Friedman has received personal compensation in the range of $500-$4,999 for serving as a Travel Reimbursement with Epilepsy Foundation of America.
Elizabeth Thiele, MD (Massachusetts General Hospital)	Dr. Thiele has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for GW Pharma/Jazz. Dr. Thiele has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for zogenix/UCB. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for nobelpharma. An immediate family member of Dr. Thiele has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Thor labs. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for takeda. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stoke Therapeutics. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for livanova. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Azurity. Dr. Thiele has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Marinus Pharmaceuticals. The institution of Dr. Thiele has received research support from GW Pharma/Jazz. The institution of Dr. Thiele has received research support from Zogenix/UCB. The institution of Dr. Thiele has received research support from Stoke Therapeutics.
Eric D. Marsh, MD, PhD (Children's Hospital of Philadlephia)	Dr. Marsh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acadia Pharmacuticals. Dr. Marsh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stoke Therapeutics. Dr. Marsh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acadia Pharmaceuticals. The institution of Dr. Marsh has received research support from NIH. The institution of Dr. Marsh has received research support from Rett Syndrome Research Trust. The institution of Dr. Marsh has received research support from International Rett Syndrome Foundation. The institution of Dr. Marsh has received research support from Eagles Autism Challenge. The institution of Dr. Marsh has received research support from LouLou Foundation. The institution of Dr. Marsh has received research support from International CDKL5 Resarch Foundation. The institution of Dr. Marsh has received research support from Acadia Pharmaceuticals. The institution of Dr. Marsh has received research support from Marinus. The institution of Dr. Marsh has received research support from Stoke Therapeutics. The institution of Dr. Marsh has received research support from Takeda Pharmaceuticals. Dr. Marsh has received personal compensation in the range of $500-$4,999 for serving as a Grant Review with NIH. Dr. Marsh has received personal compensation in the range of $5,000-$9,999 for serving as a Expert Witness with Department of Human Services. Dr. Marsh has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Medscape.
Linda Laux, MD (Childrens Hospital)	Dr. Laux has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stoke Therapeutics. Dr. Laux has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex.
Ian Miller, MD	Dr. Miller has received personal compensation for serving as an employee of Marinus Pharmaceuticals. Dr. Miller has stock in Marinus Pharmaceuticals.
Robert Flamini, MD	Dr. Flamini has received personal compensation in the range of $0-$499 for serving as a Consultant for Biocodex. Dr. Flamini has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Eisai. Dr. Flamini has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz Pharmaceuticals.
Angus Wilfong, MD (Pediatric Neurology)	No disclosure on file
Francis M. Filloux, MD (Pediatric Neurology)	Dr. Filloux has nothing to disclose.
Matthew H. Wong, MD (Matthew Wong)	No disclosure on file
	No disclosure on file
	No disclosure on file
Judith S. Bluvstein, MD	Dr. Bluvstein has nothing to disclose.
Anup D. Patel, MD, FAAN (Nationwide Children's Hospital and the Ohio State University College of Medicine)	The institution of Dr. Patel has received research support from PCORI. Dr. Patel has received personal compensation in the range of $5,000-$9,999 for serving as a Webinar Development with Medscape. Dr. Patel has a non-compensated relationship as a Chair Quality Committee with AAN that is relevant to AAN interests or activities. Dr. Patel has a non-compensated relationship as a Committee Member with Child Neurology Society that is relevant to AAN interests or activities. Dr. Patel has a non-compensated relationship as a Board of Directors - member with AAN Institute that is relevant to AAN interests or activities.
Maria Roberta Cilio, MD	No disclosure on file

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