Pubdate: Mon, 17 Nov 2014
Source: Boston Globe (MA)
Copyright: 2014 Globe Newspaper Company
Contact: http://services.bostonglobe.com/news/opeds/letter.aspx?id=6340
Website: http://bostonglobe.com/
Details: http://www.mapinc.org/media/52
Author: Kay Lazar

HOPE THAT A PRESCRIPTION CAN MIMIC MARIJUANA'S BENEFITS

Haley Osborn lives at a crossroads. Down one path, her parents are 
searching for marijuana they hope might ease their daughter's 
relentless seizures. And down the other, researchers are hunting for 
ways to mine marijuana's potential medicinal properties for patients 
like Haley to create consistent, reliable prescription drugs.

The 7-year-old Georgetown youngster is racked by seizures 15 to 20 
times a day despite taking an experimental drug made from an active 
ingredient in marijuana, cannabidiol, or CBD, that has shown early 
promise for some children whose seizures were not quelled by 
traditional medicine.

Haley's mother, Jill Osborn, is grateful her daughter is taking part 
in a CBD study in Boston. But Osborn wishes she also had access to 
marijuana rich in CBD to give her daughter. She worries that the CBD 
extracted from marijuana for the experimental drug is missing other 
active ingredients in the plant that, some scientists believe, work 
best as an ensemble.

"I'm not a scientist or an expert," Osborn said. "Just a mom trying 
to save her daughter."

Two years after Massachusetts voters approved legalizing marijuana 
for medical use, Osborn and many other patients are still waiting for 
dispensaries to open, even as other states legalize the drug for 
medical and recreational use. Meanwhile, researchers nationwide are 
striving to unlock marijuana's potential as a prescription drug to 
treat many conditions, from psychiatric disorders and glaucoma to 
seizures, inflammation, and chronic pain.

At least two Massachusetts researchers recently received federal 
grants to further that work, one to develop a process for 
manufacturing pharmaceutical-quality CBD, and the other to develop a 
synthetic compound that mimics some of marijuana's benefits without 
getting patients high. In addition, a pharmaceutical company has just 
expanded its support of a national trial, which includes Haley and 
other patients at MassGeneral Hospital for Children, to test CBD 
extract on children with seizures.

While marijuana is made up of hundreds of chemicals, CBD holds a 
special lure for scientists. Unlike THC, the ingredient that causes 
marijuana's "high," CBD is nonpsychoactive, and patient stories 
abound about CBD-rich marijuana strains calming seizures. But few 
rigorous studies have been done to prove that it works for seizures 
or other maladies, in part because federal drug policy has made it 
difficult for researchers to access the only government-sanctioned 
supply of marijuana, some scientists say. Twenty-three states allow 
marijuana for medical use, but federal regulators still consider it 
an illicit drug.

Trevor Castor, chief executive and lead researcher at Aphios 
Corporation, a Woburn biotechnology company, will be gaining access 
to the government's research-grade marijuana for a $225,000 grant he 
received in September from the National Institute on Drug Abuse. 
Castor aims to develop a process for extracting and manufacturing 
high-quality CBD for researchers to use in studies of patients with 
multiple sclerosis, epilepsy, and other diseases of the central 
nervous system. The goal is to produce it in a capsule form.

"There is a lot of interest from the medical marijuana marketplace, 
and that is pushing institutions to try and investigate why these 
things are working and how well are they working," Castor said.

If Castor accomplishes his early goals in the research during the 
next several months, he will receive about $1.5 million more to 
continue his work, said Dr. Wilson Compton, the national institute's 
deputy director.

"We are encouraging research in this area," Compton said.

At Northeastern University, assistant professor of pharmacy Ganesh 
Thakur received a $2 million federal grant in September from the 
National Eye Institute to create a compound to treat glaucoma, an eye 
disease that blinds 60 million people worldwide.

Thakur's goal is to design a drug that mimics marijuana's ability to 
reduce the fluid pressure inside the eye, a hallmark of glaucoma, but 
will not get patients high.

Scientists have found that when patients use marijuana, the plant's 
psychoactive ingredient, tetrahydrocannabinol, or THC, interacts with 
a protein in the eye, known as CB1, to lower eye pressure. 
Researchers have also found that the brain naturally produces very 
small amounts of THC-like compounds.

Thakur's work will not use marijuana, but will create a drug that, he 
hopes, behaves like marijuana without the side effects, enhancing the 
interaction between the THC-like compound in a patient's brain with 
the CB1 proteins in the eye.

"Chronic smoking of cannabis leads to impaired memory," Thakur said. 
"Maybe we can make something better and eliminate those side effects."

Dr. Hemin Chin, director of glaucoma and optic neuropathies at the 
National Eye Institute, said the government is funding Thakur's work 
because patients need better treatments.

"If you are using a compound for a long time, a patient develops 
super sensitivity, or the compound is not as effective as it was in 
the beginning," Chin said.

At MassGeneral Hospital for Children, some of the children with 
seizures who are taking a CBD extract as part of an ongoing national 
study are already reaping benefits. Data released last month by GW 
Pharmaceuticals, a British company supplying the experimental 
medication, showed that for the 58 patients nationwide who had been 
on the drug at least 12 weeks, roughly 40 percent had the frequency 
of their seizures reduced by at least half.

The data also indicated that the most common side effects were 
sleepiness and fatigue.

"It is going to be definitely effective for a population of kids with 
epilepsy, but I know it is not the silver bullet," said Dr. Elizabeth 
Thiele, director of Mass. General's pediatric epilepsy program.

"These are kids who have been on 10 previous treatments without 
seizure control," said Thiele, who has enrolled 38 children, and 
expects a total of 50, in the Boston section of the study.

"If even one of them becomes seizure free, that's impressive," Thiele said.

The CBD extract, so far, has not reduced the frequency of Haley 
Osborn's seizures, and she has been on the drug since April. But the 
drug, dissolved in sesame oil and strawberry flavored, has 
significantly improved Haley's reading ability, which had been 
delayed by seizures, and she is much more alert, able to process 
thoughts more quickly, and displaying a sense of humor - a quality 
her parents hadn't seen before.

"That part of it has been great, and that's why we haven't given up 
on the study," Jill Osborn said.

Rules of the study forbid patients to use any marijuana, so that 
researchers are certain the effects of the experimental drug, called 
Epidiolex, are not tainted. Haley is scheduled to be enrolled in the 
study until April. But her parents, who agonize over the accounts 
they hear from other states about children's seizures melting away 
with marijuana use, will face a dilemma early next year. That's when 
Massachusetts's first marijuana dispensaries are scheduled to open.
- ---
MAP posted-by: Jay Bergstrom