As the issue of medical marijuana heats up in Minnesota, we must keep in mind that at the heart of the debate are families seeking remedies for their children who live with severe forms of epilepsy like Dravet syndrome. Our hearts are with these families and their children.
Yet, as physicians and researchers specializing in the treatment of this challenging spectrum of disorders, we must guard against the professional and lay communities making treatment decisions that are not based in sound research and science.
While there are a number of anecdotal reports of positive outcomes from a particular strain of marijuana known as Charlotte’s Web, robust scientific evidence for the use of marijuana for treatment of epilepsy is lacking. The lack of information does not mean that marijuana is ineffective. It merely means that we do not know if marijuana is a safe and efficacious treatment for epilepsy.
Charlotte’s Web is thought to contain relatively little tetrahydrocannabinol, or THC, the primary component that produces a high. Instead, the strain has high amounts of another compound — cannabidiol, or CBD. This is not smoked but is used in an oil form.
In addition, little is known about the long-term effects of using marijuana in infants and children on memory, learning and behavior. This is of particular concern, because both clinical data in adolescents and adults and laboratory data in animals demonstrate potential negative effects on critical neurological functions. Such safety concerns, coupled with a lack of evidence of efficacy in controlled studies, result in a risk-benefit ratio that does not yet support use of marijuana for treatment of seizures.
Several members of the American Epilepsy Society are now conducting clinical trials of CBD. There are several steps in a clinical trial, and we need to wait to draw conclusions until there has been a trial with a control group or a placebo-controlled trial. The preliminary steps underway will not have a placebo group and will be used for dose finding and tolerability and to establish an understanding of how human bodies absorb and process the drug. If these initial safety studies are encouraging, then further controlled studies will be needed to determine if CBD is effective in the treatment of seizures and in which patient populations (what ages and what types of epilepsy).
These studies are critical, as the pathway to finding new drugs and treatments is full of treatments once thought to be the “miracle cure” that were rejected after the rigors of a clinical trial. These studies are especially important in a condition like epilepsy that has a variable course. Sometimes significant improvement can actually be a result of the unpredictable ebb and flow of the disease.
Treatments cannot advance without clinical trials. The American Epilepsy Society is supportive of well-designed clinical research to determine the safety and efficacy of marijuana in the treatment of epilepsy. We urge the entire community of medical professionals, patients, families and regulators to focus their efforts on getting accurate information and allowing proper research to be done.
The recent discussions surrounding medical marijuana highlight the fact that the epilepsy community desperately needs funding to find new therapies and approaches for patients with resistant or refractory seizures and someday find targets for cures. Yet none of these giant steps forward will be possible without funding and robust research that safeguards the health of study participants while uncovering important new findings.
States around the country are having the same debate as Minnesota. We hope the needs of people living with epilepsy and their families will be a strong voice in this debate. However, we also urge that the eagerness to find treatments will not overshadow the need to conduct rigorous research and testing. Together as an epilepsy community we must take this step to find the answers for people living with these severe forms of epilepsy.
Elson So is president of the American Epilepsy Society.