Pubdate: Fri, 06 Dec 2013
Source: Hunterdon County Democrat (Flemington, NJ)
Copyright: 2013 Hunterdon County Democrat
Contact: http://drugsense.org/url/3yHVqfQj
Website: http://www.nj.com/hunterdon-county-democrat/
Details: http://www.mapinc.org/media/5109
Author: Susan K. Livio

FDA-APPROVED MEDICAL MARIJUANA CLINICAL TRIAL GETS UNDERWAY NEXT MONTH
FOR KIDS WITH EPILEPSY

A nationally renowned pediatric neurologist at Saint Barnabas has
gotten FDA approval to study whether a cannabis-based drug could
prevent seizures in children diagnosed with severe forms of epilepsy.

The development is sure to be watched closely in New Jersey, where a
number of families whose children have Dravet syndrome, a potentially
deadly form of epilepsy, say they cannot obtain yet a useful form of
medicinal marijuana through the state Department of Health.

The U.S. Food and Drug Administration gave Orrin Devinsky, director of
the New York University and Saint Barnabas Comprehensive Epilepsy
Center, Roberta Cilio of the University of California - San
Francisco's Neurology Department, and GW Pharmaceuticals of the United
Kingdom permission to use the experimental drug, Epidiolex, to treat
125 children with seizure disorders for whom traditional medicines
have failed.

Enrollment in the trial will begin next month, Devinsky
said.

"I think this is a big step forward in the science of Cannabidiods,"
said Devinsky, referring to the spectrum of active ingredients in
marijuana. "We will finally get some some data on epilepsy, and this
should provide the basis to plan a randomized double-blind study."

The drug is a liquid form of pure cannabidiol, a non-psychoactive
component of the cannabis plant that according to anecdotal reports
has shown to reduce the frequency and intensity of seizures in
children. The FDA's Nov. 15th approval also gives Epidiolex "orphan
drug" status, providing GW full marketing rights to be the lone
producer of the product for seven years, the company said in a statement.

Orphan diseases are rare, occurring in less than 200,000 of people in
the nation. There are 5,440 documented cases of Dravet syndrome in the
United States and 6,710 in Europe, although the actual number may be
higher because the condition is under-diagnosed, according to a
statement from the pharmaceutical company. The seizures can damage the
brain, delay development and cause lifelong intellectual
disabilities.

Meghan and Brian Wilson of Scotch Plains, who have fought a very
public battle to obtain medicinal marijuana for their 2-1/2-year-old
daughter Vivian, said they are eager to see the drug trial get
underway. But Vivian will not be enrolled next month out of concern
for her fragile health.

Based on the experiences of other families whose children have Dravet,
the children who were weaned off the anti-seizure drug Benzodiazepine
- -- a tranquilizer that prevents or stops seizures by slowing down the
central nervous system -- saw the most benefits from cannabidiol.
Cutting back on the "Benzo," even very slowly, has triggered many
severe seizures, Vivian's mother said.

"I think it's a great step in the direction of valid scientific
research with cannabis," she said. "I'd like to see the data before we
add on another pharmaceutical, because at the end of the day it is a
another pharmaceutical.

These are the first trials in human for this particular
compound.

Brian Wilson said getting Vivian into the trial would take a "hard
core withdrawal" from the Benzo. "I'd hate to expose Vivian to that
until we know it will work."

Parents whose children have Dravet, Lennox-Gastaut syndrome and other
serious seizure disorders, say they are eager to try cannabidiol
produced by growers in Colorado that has helped reduce seizure
activity in more than 100 children. But none of the three medical
marijuana dispensaries in New Jersey -- including one that opened
Wednesday and another Oct. 28 - carry the strains of pot that have
shown any promise. No one has submitted a proposal approved by the
state to produce a pot-infused edible product, which under state law
are available for children.

The Wilsons purchased marijuana from Compassionate Care Foundation,
Inc. in Egg Harbor, cooked it down into an oil, but cannot find a lab
in the state willing to test its potency.

GW already has a track record producing marijuana-based medicine.
Savitex, a cannabis-based spray used to treat muscle spasticity for
patients with multiple sclerosis, is available in 22 countries. It is
under FDA review.

"Through its efforts, GW aims to provide the necessary evidence to
confirm the promise of CBD in epilepsy and ultimately enabling
children to have access to an FDA-approved prescription," according to
a statement from Stephen Wright, GW's research and development director.

Photo: Meghan Wilson holds the marijuana they just obtained as her 
husband Brian gets their 2 year old daughter Vivian ready to go outside 
the Compassionate Care Foundation, New Jersey's 2nd medical marijuana 
dispensary on opening day, Oct. 28. 2 year old Vivian suffers from a 
rare and severe form of epilepsy called Dravet syndrome which 
conventional medicine cannot control. (Tony Kurdzuk/The Star-Ledger)
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MAP posted-by: Matt