For the first time in Nebraska, legal testing of a marijuana-based drug treatment will begin this summer.
By- Bill Kelly
Researchers at the University of Nebraska Medical Center (UNMC) this week began recruiting 25 test subjects suffering from epileptic seizures since childhood.
“There are individuals in Nebraska who suffer from treatment-resistant seizures for which currently available treatment options have been ineffective,” said Dr. Deepak Madhavan, principal investigator for the UNMC study, in a statement released by the hospital. “This study will broaden our knowledge of the safety and effectiveness of cannabidiol in treating these patients.”
A change in state law (LB390) allowed the narrowly focused research to begin, even while state policy makers proved reluctant to authorize widespread availability of medical cannabis.
During the news conference announcing the project, State Senator Sue Crawford, who introduced the bill, called the process “a long journey.”
She took on the cause in 2013 after hearing “heart-wrenching stories” of families unable to find effective drug treatments for their children’s seizures. Around the same time some individuals found success in untested treatments drawn from the cannabis extract known as CBDs.
“It took a while to build support for the CBD research because it was linked to marijuana,” Crawford said. “Getting the support to pass the bill required pulling together support from a broad coalition, including many folks who were skeptical at first” including law enforcement and anti-drug forces.
All were given assurances that the drugs being sought were not the type acquired for recreational use. ““Our aim with the bill was to provide access to low THC cannabis that was federally compliant and allowed these patients to work closely with their physicians,” Crawford said in her prepared statement.
The state also provided $500,000 in funding for the study.
Dr. Chris Kratochvil, vice president of research at Nebraska Medicine, told reporters taking on the research is “the right thing to do.”
“If there is a treatment that would be useful to patients, especially patients (who) are not responding to other treatments, than it really was our obligation to study this,” Kratochvil added.
The drug, Epidiolex, is formulated from pure cannabidiol extracted from the Sativa strain of marijuana. Its maker, British drug company GWPharma, has started clinical trials. The company plans to submit a new drug application to Food and Drug Administration (FDA) early in 2017.
Recently the drug company announced clinical trials in the late stages show encouraging results. More than 40 percent of patients given the drug show a significant reduction in seizures. Few people are reporting harmful or unpleasant side effects.
The research in Nebraska hopes to compliment other studies by the manufacturer and other research hospitals.
“These together with the kind of study we can do here will continue to develop a database so we can understand how safe and how effective this kind of treatment will be.” Kratochvil said.
Letters will be sent to neurologists and pediatricians in Nebraska with information about the study. Patients interested in volunteering are required to get a referral from their doctor, followed by an evaluation to assure they are suitable subjects for the study.
A group of clinicians and scientists will determine who is selected to participate in the study. Qualified patients ranging in age from infants to 60-years-old, must have treatment-resistant seizures that aren’t controlled with available treatments.
In exchange for getting the experimental drug at no charge, patients must have up to 15 in-person visits and a series of follow-up discussions through phone or email.
In information prepared for patients they are told “depending on clinical outcomes the study may be extended beyond two years.”
Among the families hoping to participate are the study are Mike and Lisa Bartenhagen of Gretna. Their 14-year-old son Broc began having small seizures shortly after birth. They grew in intensity until he was diagnosed with Lennox-Gastaut Syndrome, a severe form of epilepsy.
“We are optimistically hopeful if he gets on the trial it will help, Bartenhagen said. “If he is able to get on the trial and it does not help him, I think it’s an opportunity to further (understanding) of the medication and see if it will help some other children.
Broc has “a lot of seizures every day,” Mike Bartenhagen told reporters. “They are difficult to control. Most kids (with Lennox-Gastaut) don’t get control so it’s more about managing the seizures.”
The condition means the teenager is developmentally delayed, is unable to speak and finds movement possible but difficult. Since the onset of the epilepsy, the Bartenhagens have attempted 23 different procedures and treatments, including a variety of drugs, four brain surgeries and a special diet.
Broc’s doctor will refer his name to the research team who will review his medical history to verify whether he meets the criteria to participate.
Those who are selected as test subjects won’t get the drug immediately.
“The patients will then be monitored for two months to look for the frequency of seizures, to see if it makes sense to try this treatment,” said Kratochvil, adding it is “also to establish a baseline so we know is there a change once the treatment is initiated.”
The liquid formula will be given patients twice a day. There will be up to 15 visits to the clinic and regular updates by phone and email.
The testing currently being done by the manufacturer uses two groups of test subjects for comparison. One gets the drug and a second gets a placebo that contains no active ingredients. The technique allows for a blind comparison of results.
The UNMC study will provide all of the 25 participants with the actual drug.
“The idea is this is giving access to a potentially therapeutic treatment before all the safety and efficacy studies are done” said Kratochvil.
The study could stretch over the next two years, and regardless of the outcome, there’s hope the results, combined with related cannabidiol research will help provide some insight into controlling epileptic seizures.