Pubdate: Tue, 15 Mar 2016
Source: Seattle Times (WA)
Copyright: 2016 The Seattle Times Company
Contact:  http://seattletimes.nwsource.com/
Details: http://www.mapinc.org/media/409
Author: Andrew Pollack, The New York Times

POT-BASED DRUG CUTS SEIZURES, FIRM SAYS

Medical Marijuana

Epidiolex Tested on Kids With Rare Form of Epilepsy

An experimental drug derived from marijuana has succeeded in reducing 
epileptic seizures in its first major clinical trial, the product's 
developer announced Monday, a finding that could lend credence to the 
medicalmarijuana movement.

The developer, GW Pharmaceuticals, said the drug, Epidiolex, achieved 
the main goal of the trial, reducing convulsive seizures when 
compared with a placebo in patients with Dravet syndrome, a rare form 
of epilepsy.

If Epidiolex wins regulatory approval, it would be the first 
prescription drug in the United States that is extracted from marijuana.

Parents whose children with epilepsy are not being helped by 
conventional drugs have been flocking to try marijuana extracts, 
prepared by medical-marijuana dispensers.

A number of states, in response to pressure from parents of epileptic 
children, have passed or considered legislation to make it easier to 
obtain marijuana-based products. And some families have become 
"marijuana refugees," moving to Colorado where it has been easier to 
obtain a particular extract, known as Charlotte's Web after the girl 
who first used it to control seizures.

While many parents, though far from all, have reported significant 
reductions in seizures, experts have been cautious about anecdotal 
reports, saying that such treatments needed to be compared with a 
placebo to make sure they work.

"The results of this Epidiolex pivotal trial are important and 
exciting, as they represent the first placebo-controlled evidence to 
support the safety and efficacy of pharmaceutical cannabidiol in 
children with Dravet syndrome," Dr. Orrin Devinsky of the 
Comprehensive Epilepsy Center at New York University Langone Medical 
Center, said in a statement.

Devinsky was the trial's principal investigator and said Dravet 
syndrome was one of the most severe and difficult-to-treat types of epilepsy.

Epidiolex contains almost pure cannabidiol, a component of marijuana 
that does not make people high.

While Epidiolex could be the first prescription drug in the United 
States extracted from marijuana, two drugs already on the market, 
dronabinol and nabilone, are synthetic versions of delta-9 THC, the 
principal component of marijuana that causes the highs. Those drugs 
are approved to treat nausea and vomiting caused by cancer 
chemotherapy when other anti-nausea drugs fail to work. Dronabinol is 
also approved to treat weight and appetite loss in patients with AIDS.

The Epidiolex study involved 120 patients with an average age of 10 
and an average frequency of 13 convulsive seizures a month at the 
start of the study, despite taking an average of three other drugs. 
Half of the children were given the drug and half given a placebo, in 
addition to the epilepsy medicines they were already taking.

The company said that for the patients who received Epidiolex, the 
frequency of convulsive seizures fell by 39 percent during the 
14-week treatment period, compared with a four-week period just 
before the treatment started. For those getting placebo, the 
reduction was 13 percent. The difference between the two groups was 
statistically significant.

Eight patients getting Epidiolex withdrew from the trial because of 
side effects; one patient getting the placebo left. Major side 
effects included drowsiness, diarrhea, decreased appetite, fatigue, 
fever, vomiting and upper respiratory infection. But GW said that, 
overall, the drug was well tolerated and that the dropout rate from 
the trial was lower than in many studies of other epilepsy drugs.

More than 300 U.S. children have been taking Epidiolex under an 
expanded-access program, in which an experimental drug can be given 
to desperate patients before the product is approved.

The company said Monday that it would meet with the Food and Drug 
Administration to see if Epidiolex could be specifically approved for 
Dravet syndrome based on this single study. There are no drugs 
approved specifically for Dravet syndrome, which typically starts in 
infancy and affects about 5,000 children in the United States, 
according to the company.

The company, which specializes in cannabis-based pharmaceuticals, 
already sells Sativex to treat spasticity associated with multiple 
sclerosis. It is approved in many countries, though not the United States.
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MAP posted-by: Jay Bergstrom