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Cincinnati tested the marijuana drug that the FDA has just approved

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For the past three years, a big, black safe in the Medical Arts Building at the University of Cincinnati stored Dr. Michael Privitera’s supply of medical marijuana.

A lifelong researcher on the mystery of brain seizures, Privitera was running a clinical study on a medical-marijuana drug and epilepsy. Under unusual government scrutiny, the study turned in such astonishing results that on Monday, the U.S. Food and Drug Administration approved the drug, Epidiolex, the first time the government has signed off on a disease treatment made from the marijuana plant.

“It’s historic,” said Privitera. “We did the right kind of science. We took the anecdotal evidence, extracted the substance in marijuana that helps, tested it, and we followed the testing guidelines used for every other drug. This study really proves it, and we can make sure it’s safe.”

One study participant is William Plott, 27, of Westwood, who has suffered from seizures nearly all his life. Glenn and Cassandra Plott said Epidiolex has so eased the raging electrical storm in their son’s brain that he now can do simple things for the first time, such as making eye contact. “We feel fortunate to be part of the study,” Cass Plott said.

The FDA action could be meaningful for marijuana itself. Since 1970, the federal government has listed marijuana on its Schedule I of the most dangerous substances with no therapeutic value. With marijuana performing impressively under rigorous controls, pressure likely will grow to pull the drug from the federal all-bad list, which would broaden horizons for research and development.

How a little girl led the way

Among the nation’s loudest advocates for medical marijuana are families of children with epilepsy. In 2013, their network lit up with news of a Colorado girl, Charlotte Figi, who had suffered hundreds of seizures a day. No pharmaceutical controlled her disease, so her desperate parents turned to marijuana.

Humans have used marijuana as medicine for thousands of years. Only recently did science learn that marijuana – Latin name cannabis – contains dozens of intriguing compounds. One, cannabidiol or CBD, showed early promise in controlling seizures without marijuana’s mind-altering effect, caused by its best-known compound, delta-9-tetrahydrocannabinol or THC.

The Figis worked with Colorado developers to create an oil high in CBD, and it worked on Charlotte. Word of “Charlotte’s Web” oil flew around the internet, and a wave of families moved to Colorado to get a doctor’s recommendation to treat their sick children with CBD patches, oils and lotions. The Plotts debated moving, too.

‘The most complicated study’

Privitera founded and directs the Epilepsy Center at the UC Health Gardner Neuroscience Institute. He read about Charlotte’s Web oil and decided he was willing to study anything to fight the debilitating disease that affects a million Americans.

Privitera’s friend Dr. Orrin Devinsky, of New York University Langone Comprehensive Epilepsy Center, was launching a rigorous study on a pure-CBD oil manufactured by the British firm GW Pharmaceuticals.

One group of study subjects would get a low dose of Epidiolex. One group got a high dose. A third got a placebo. Neither doctors nor patients would know who was getting what. The hope was to put the brake on Lennox-Gastaut syndrome, which triggers terrifying “drop seizures” that mash the halt button in the brain. The body locks up, and gravity takes over, often resulting in disabling head injuries.

GW Pharmaceuticals was paying for the study. Privitera was determined to get UC included. Devinsky cautioned: Not only would the FDA be watching, so would the U.S. Drug Enforcement Administration.

Privitera said no problem, but, “I didn’t realize what I was signing up for. I’ve been doing this work for 30 years, and this was far and away the most complicated and difficult study we’ve ever had to do.”

The DEA, a crime-fighting and interdiction agency, rarely gets involved with medical research. “The first time the DEA person came by, he sat down and said, ‘Tell me what epilepsy is,’ ” Privitera said. The doctor explained that epilepsy is an umbrella term for seizure disorders brought on by short circuits in the brain’s wiring

The easy part: Recruiting patients

The DEA required unusually heightened controls for the drug, including the big, black safe in the Medical Arts Building for storage. To participate in the study, Privitera first needed a Schedule I license, which required a nine-month review including fingerprinting.

The drug could only be dispensed from the address on Privitera’s Schedule I license, the Medical Arts Building.

The DEA would only examine records there, too, so at every audit, nurse coordinator Donna Schwieterman rolled a full file cart from Privitera’s office in the Stetson Building to the Medical Arts Building across busy Martin Luther King Boulevard.

Privitera had to account for every drop of Epidiolex. Every bottle that left the building had to be returned, empty or not. Privitera had to hire a disposal company to collect and destroy the bottles and any leftover oil.

The study got underway as the landscape for marijuana in Ohio shifted. A well-financed 2015 campaign for full legalization failed 2-1, but polls showed that Ohioans wanted access to marijuana as medicine. The Ohio General Assembly created a medical program by June 2016. Though not a fan, Republican Gov. John Kasich signed the bill.

By far the easiest part of the Epidiolex study was recruiting subjects. “The patients involved, I must say, were just really courageous,” Privitera said. “Even the people who didn’t get a benefit all said, ‘I would do it again, to help other people with epilepsy.’ ”

William’s story

The Plotts read about Charlotte’s Web oil and took hope.

Glenn Plott, 61, for about 20 years has been director of production for the Cincinnati Opera. Cass Plott, 62, is a professional quilter who cares for William full time. They have a younger son, Jeremy.

The Plotts say the seizures began in 1991 when William was 6 months old, and he “fell off the developmental chart,” Cass Plott said, beginning the odyssey of pharmaceuticals. Nothing worked well, or for long. William’s seizures were lengthy, frequent and severe, requiring him to wear a helmet. No genetic, organic or structural cause has been found.

Along with a heavy drug combination, William got a vagus nerve stimulator, an implant that sends electrical signals to the brain. He does not speak. He cannot care for himself. The Plotts keep scrupulous notes about William in large three-ring binders.

William’s neurologist, Dr. David Ficker, is a colleague of Privitera in the Neuroscience Institute. When the Plotts considered a Colorado move, Ficker suggested getting William into the Epidiolex study. First came paperwork and examinations to establish baselines, but then one day in late 2015, Cass filled an oral syringe with Epidiolex. It smelled like strawberries. She squirted it into William’s mouth. He swallowed it.

Confronting the ‘drop seizure’

The 14-week study launched at sites in England, Spain and the United States, including UC and Nationwide Children’s Hospital in Columbus. The 225 patients ranged from 2 and 55 and were having two or more drop seizures a week.

The big, black safe on the third floor of the Medical Arts Building soon filled with GW Pharmaceuticals’ white boxes. The DEA permitted only Privitera, Ficker and the other UC medical doctors in the study to open the combination safe, which was behind a double-locked door with the keys in a locked box in another room. The DEA also conducted surprise inspections of the study site.

The eye-popping results were published in March in the prestigious New England Journal of Medicine. The average reduction in drop seizures was 37 percent at a 10mg dose and 41 percent at a 20mg dose. Even the placebo patients had a 17 percent reduction. In some patients, Privitera said, the decrease was as high as 90 percent.

Drug could be in stores by year end

The drug is safe. The most serious side effects were sleepiness, reduced appetite and diarrhea – that last not surprising, Privitera said, since patients consumed a lot of oil through the study. In April, an FDA review committee voted 13-0 in April to recommend approval. The final OK June 25 means the drug could be in pharmacies by year’s end.

For all the work to test the drug, the study didn’t reveal anything except that CBD oil provably reduces seizures. “We don’t know how it works. We don’t know why it works. But it does work,” Privitera said. “Now it’s time for more research.”

Government restraints on studying a Schedule I drug make research prohibitively expensive. FDA approval for Epidiolex could show that researchers can manage studies of medical marijuana as they do any other experimental drug, without the involvement of law enforcement, or the necessity of a big safe.

Ohio is the 25th state to enact a medical-marijuana program, which officially begins Sept. 8, although drug production is behind schedule by several months. Doctors certified by the Ohio Medical Board can write recommendations for 21 qualifying conditions, including epilepsy.

What CBD oil revealed in William

“We are not of the reefer-madness mindset,” said Cass Plott. “We’re not let’s-get-high people.”  But during a Father’s Day conversation at their Westwood house, the Plotts say they are believers in CBD. They need only look at their William notebooks to track the significant improvement over more than two years.

The change was so dramatic that “we knew almost immediately that he wasn’t on the placebo,” Cass said. For a while early on, William’s appetite exploded. Glenn said he and Cass wondered, “Does he have the munchies?”

William used to have 1,000 seizures a year. Now he has about 200 that are less severe, although an early June fall broke his collarbone. He takes 15.2 milligrams of Epidiolex daily. He goes to a day program at the Center for Practical Living in Mount Healthy.

The biggest improvement, the Plotts said, is that William can express himself. Slowly, in small ways, their grown son is revealing himself.

“He knows to stay away from my mail, but sometimes, we’ll watch him just walk over to it, do you see me, do you see me? It’s one of his favorite things,” Glenn said. “It’s fun. Like having a 5-foot-6 toddler in the house.”

On Father’s Day, William orbited the rooms of his house then beelined for the kitchen. His mother steered William to his bedroom, where he fell into bed and beamed when she tucked him in. The nap was brief, though, and William rose again. He reached to grab his mother’s hand and turned to walk for the front door. He wanted to go outside.

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