Pubdate: Thu, 30 Oct 2014
Source: Houston Chronicle (TX)
Copyright: 2014 Houston Chronicle Publishing Company Division, Hearst Newspaper
Contact:  http://www.chron.com/
Details: http://www.mapinc.org/media/198
Author: Todd Ackerman
Page: A1

POT-BASED EPILEPSY DRUG OFFERS HOPE FOR SUFFERERS

Izaiah Ruiz's epilepsy was so severe, his daily life so miserable, 
that his grandmother says she would have sold her Montgomery County 
home and moved to Colorado for what many say is a new, miracle 
treatment: marijuana.

But Lori Fountain couldn't make the finances work. As her 6-year-old 
grandson suffered, all she could do was follow online the progress of 
patients who sought treatment in Colorado, where marijuana is legal. 
Their families reported patients suddenly were seizure-free, verbal, 
able to dress and feed themselves thanks to treatment with a strain 
of marijuana, a preparation known as Charlotte's Web, which has 
become something of a national phenomenon.

Seemingly without hope, Fountain a few weeks ago got unbelievable 
news from Texas Children's Hospital: Izaiah would be one of the first 
patients in an international trial of an experimental drug derived 
from marijuana that has shown dramatic benefits in a select few 
patients who have already received it. The drug, Epidiolex, is a 
highly purified extract of cannabis that does not contain THC, the 
ingredient that alters mood.

Fountain said she cannot wait for Friday, when she brings Izaiah to 
Texas Children's for testing.

"If this drug could work and give Izaiah any semblance of a normal 
life, I can't tell you how ecstatic I'll be," said Fountain, who 
described the boy's constant seizures, inability to talk and failure 
to comprehend Christmas or other holidays. "I can't explain what it'd 
mean to watch him playing outside, going down a slide, taking part in 
an Easter egg hunt."

Texas Childrlen's is expected to enroll the most patients in the 
10-site trial, which is being conducted on patients with a rare and 
severe form of epilepsy known as Dravet syndrome.

The syndrome, which occurs in about one in every 30,000 births, is 
characterized by dozens or even hundreds of seizures a day, some 
life-threatening. Patients typically struggle with developmental 
delays, a variety of health problems and an increased risk of what is 
known as Sudden Unexplained Death in Epilepsy.

Difficult to control

Dravet syndrome seizures are difficult to control and frequently 
don't respond to medications available now. Some drugs that work in 
many epilepsy patients make Dravet syndrome worse.

Dr. Angus Wilfong, the study's principal investigator at Texas 
Children's, says he hopes the trial shows Epidiolex is the safe and 
effective treatment desperately needed - that it lives up to the 
expectations generated by the anecdotes.

"Charlotte's Web has received a lot of attention, but there's no 
proof it works," said Wilfong, a pediatric neurologist at Texas 
Children's and Baylor College of Medicine. "This will be a 
scientifically rigorous study of a standardized product that 
shouldn't cause any issues of addiction."

Two Dravet syndrome patients who previously received Epidiolex at 
Texas Children's through a compassionate-use program approved by the 
Food and Drug Administration showed dramatic benefits, Wilfong said.

Though he said he thinks a drug synthesized from marijuana would be 
more potent than Charlotte's Web, Wilfong cautioned he didn't expect 
the drug would make any Dravet syndrome patient seizure-free.

Dr. Elson So, a neurologist and president of the American Epilepsy 
Society, said the Epidiolex trial is important "not to disprove 
Charlotte's Web but to make sure we don't miss out on a compound that 
could be very good for epilepsy." He noted that without such a study, 
the politics of marijuana could blind people to a useful compound.

Two phases of study

The study is being conducted in two stages. The first part will 
enroll 30 children 4 to 10 years old and focus on determining safe 
and effective doses. Patients, tested the first month for the 
severity of their Dravet syndrome, thereafter will receive any of 
number of potencies of the drug or a placebo.

The second part, expected to begin in early 2015, will enroll 80 
children 2 to 18 years old.

Epidiolex, a liquid medicine taken by mouth or through a feeding 
tube, is made by GW Pharmaceuticals. It has been studied in a 
previous "precursor" trial, in which researchers reported on results 
with patients, not all with Dravet syndrome, not randomized to either 
a treatment or placebo arm and taking other medications. The 
researchers showed some benefit.

The new trial is only for pediatric patients with confirmed Dravet 
syndrome. The first patient began testing in Atlanta last week. 
Izaiah will be the second patient.

Fountain said she hopes Izaiah gets Epidiolex from the start, rather 
than a placebo, but she takes solace that if he doesn't, he'll get 
the drug soon thereafter.

"I've been dealing with this since Izaiah was 2 months old, when he 
had his first seizure," said Fountain, who stays with the boy around 
the clock, including when he's sleeping. "We've almost lost him a 
couple times and know that without new treatment, he may not reach 
his teenage life. This drug is our hope."
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MAP posted-by: Jay Bergstrom