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.