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We Need Proof on Marijuana

]by  ORRIN DEVINSKY and DANIEL FRIEDMAN

Many people have heard the story of Charlotte Figi, a young girl from Colorado with severe epilepsy. After her parents began giving her a marijuana strain rich in cannabidiol (CBD), the major nonpsychoactive ingredient in marijuana, Charlotte reportedly went from having hundreds of seizures per week to only two or three per month. Previously, her illness, Dravet Syndrome, was a daily torture despite multiple high doses of powerful anti-seizure drugs.

As news of Charlotte’s story moved from the Internet to a CNN story by Dr. Sanjay Gupta to Facebook pages, some families of children with similar disorders moved to Colorado, which recently legalized marijuana, to reap what they believe are the benefits of the drug.

Dozens of other anecdotes of miraculous responses to marijuana treatments in children with severe epilepsy are rife on Facebook and other social media, and these reports have aroused outsize hopes and urgent demands. Based on such reports, patients and parents are finding official and backdoor ways to give marijuana to their children.

But scientific studies have yet to bear out the hopes of these desperate families. The truth is we lack evidence not only for the efficacy of marijuana, but also for its safety. This concern is especially relevant in children, for whom there is good evidence that marijuana use can increase the risk of serious psychiatric disorders and long-term cognitive problems.

The recent wave of state legislatures considering and often approving medical marijuana raises significant concerns. By allowing marijuana therapy for patients with diseases such as difficult-to-control epilepsy, are state legislatures endorsing the medical benefits and safety of a broad range of marijuana species and strains before they have been carefully tested and vetted? Marijuana contains around 80 cannabinoids (THC is the major psychoactive cannabinoid, largely responsible for the high) and more than 400 other compounds. The chemical composition of two genetically identical plants can vary based on growing conditions, soil content, parasites and many other factors.

While the language of the legislation may be cautious, there is an implied endorsement of medical benefit for marijuana when a legislature passes a bill and a governor signs it into law, and the tremendous gaps in our knowledge are not effectively conveyed to the public.

Where is the data showing that marijuana is effective for epilepsy? Although parents may report improvements in their children, it is important to remember that the placebo response is powerful, and the placebo response is greater in pediatric than adult studies.

Before more children are exposed to potential risks, before more desperate families uproot themselves and spend their life savings on unproven miracle marijuana cures, we need objective data from randomized placebo-controlled trials.

Based on studies showing that CBD can prevent seizures in animals and safety data from patients treated with a drug containing CBD and THC in Europe for multiple sclerosis spasms, we and other academic epilepsy centers are planning a controlled trial with pure CBD. As an initial step, we have approval from the Food and Drug Administration, the Drug Enforcement Administration and the Bureau of Narcotic Enforcement to treat children with CBD derived from marijuana plants in order to understand its safety and tolerability and potential drug interactions. This information will help us plan the placebo-controlled trials that we hope will begin in 2014 and will be completed within two years. There is no reason such studies cannot be done with other products derived from marijuana, such as the oil with high CBD and low THC sold in Colorado that was used by Charlotte Figi.

Paradoxically, however, as state governments increasingly make “medical” marijuana available to parents to give to their children, the federal government continues to label the nonpsychoactive CBD — as well as THC — as Schedule 1 drugs. Such drugs are said to have “no currently accepted medical use in the United States, a lack of accepted safety for use under medical supervision, and a high potential for abuse.” This designation hamstrings doctors from performing controlled studies. While it is possible to study Schedule 1 drugs in a controlled laboratory setting, it is extremely difficult to study these substances in patients. For our study, we keep the CBD in a 1,200-pound safe in a locked room, in a building with an alarm system.

To foster research, we need to change compounds derived from marijuana from Schedule 1 to a less restrictive category. It is troubling that while few barriers exist for parents to give their children marijuana in Colorado, there are significant federal roadblocks preventing doctors from studying it in a rigorous scientific manner.

When patients have not been able to get successful medical treatment, and they live in a state where the law allows medical marijuana for children — we are not suggesting they smoke the drug — compassionate use is reasonable.

But for the long-term health of Charlotte and other patients like her, we urgently need valid data.