The children enrolled in the study all suffer from epilepsy so severe that traditional drugs have little or no effect. Some suffer up to 90 seizures a day, which if left uncontrolled can lead to brain damage.
Last month, they began receiving doses of a liquid-based drug derived from the cannabis plant called Epidiolex. Manufactured by U.K.-based drug company GW Pharmaceuticals, the drug contains no THC, or tetrahydrocannabinol, the main psychoactive compound in marijuana.
Instead, the drug is pure CBD — cannabidiol — a nonpsychoactive compound found in marijuana that has had medical efficacy in adults for conditions such as multiple sclerosis and chronic pain.
Researchers hope to find a “desperately needed” treatment for children with uncontrolled seizures and to see if CBD, “well-tolerated and safe in adults,” has the same palliative potential for kids.
In April, UCSF became the first research hospital in the country to give Epidiolex to children.
Results were positive enough to prompt the current study, led by Dr. Maria Roberta Cilio and Dr. Joseph Sullivan of the hospital’s Pediatric Epilepsy Center.
A total of 150 patients may eventually be enrolled in the study. Research began at UCSF Benioff Children’s Hospital and at New York University’s Langone Medical Center, with patients at four more medical centers to follow pending FDA approval.
Children have become a recent focal point in the country’s debate over medical marijuana. Small amounts of the plant are available to adults with a doctor’s recommendation in 20 states, including California.
But anecdotal accounts of cannabis’s ability to calm seizures have led some parents to bypass the research phase and relocate to places like Colorado, where adults can legally purchase and cultivate the drug, in order to give marijuana to their children.
Marijuana remains banned under federal law.
GW Pharmaceuticals is the first such large pharmaceutical firm to embrace the palliative power of cannabis.
The company already markets a cannabinoid-based spray called Sativex for MS spasticity in the U.K., Germany, Canada, Spain and Denmark.
Sativex is in clinical trials in the U.S. for MS and cancer-related pain.