EFCO Report to the Community
Epilepsy Foundation of Colorado
Cannabidiol (CBD) as a Medication for Children with Epilepsy
The use of marijuana for children with epilepsy has been in the national spotlight since CNN’s Sanjay Gupta featured in August 2013 the story of Charlotte Figi, a Colorado child with Dravet Syndrome. Charlotte’s seizures declined dramatically after she began taking a medication called “Charlotte’s Web” made from marijuana plants. The medication differs from standard marijuana in that it has high levels of cannabidiol (CBD), the component believed to be effective in controlling seizure activity, and very low levels of THC, the psychoactive component that makes marijuana users high.
Colorado has been at the epicenter of this development because the Stanley brothers, the producers of this medication, are located here. In fact, families from around the United States have moved to Colorado in order to access Charlotte’s Web for their children, since federal law prohibits transporting marijuana across state lines, even to other states that permit use of marijuana for medical purposes.
Many of the issues surrounding use of CBD by children with epilepsy have received extensive coverage from major media sources (click here for links to relevant articles). This summary provides information about the medications and their use, the status of related research, and resources for families, medical professionals, and others contemplating treatment decisions. It also suggests regulatory changes that could encourage needed medical research and expand treatment options for patients with severe forms of epilepsy.
The medications being taken by children with epilepsy are far different from the marijuana widely used for recreational purposes.
- These medications are taken in liquid form, not smoked.
- These medications are high in cannabidiol (CBD), a component in marijuana that has appeared to be successful in reducing seizures in some animal studies, and very low in THC, the psychoactive component of marijuana (that is, the substance that makes users “high”).
Two medications containing CBD are being used to treat children with severe epilepsy; currently one has a waiting list (Charlotte's Web, also known as "Realm Oil") and the other is not available in Colorado (Epidiolex).
- Charlotte’s Web, extracted from marijuana plants by the Stanley brothers and dispensed in Colorado Springs, can be used legally in Colorado.
- As of late February 2014, according to the Stanleys’ Realm of Caring Foundation, 187 pediatric and 27 adult patients with epilepsy are taking Charlotte’s Web. There is an extensive waiting list. As of December 2013, a licensee of the Stanley brothers is providing Charlotte’s Web in California.
- Epidiolex, a medication containing CBD and developed by the British company GW Pharmaceuticals, has received Investigational New Drug (IND) approval by the Food and Drug Administration and is available only by physician application to the FDA. Currently about 10 physicians in the United States have been approved to treat patients using Epidiolex; none of them are in Colorado.
Early results are promising, but more research is needed (and it is happening).
- The Realm of Caring Foundation reports that, among the initial cohort of 27 children with epilepsy who received Charlotte’s Web, 23 (85%) experienced a reduction of 50% or more in seizure frequency. Margaret Gedde, MD, who has seen in her clinical practice many of the children taking Charlotte’s Web, estimates that overall one-quarter of children with epilepsy have had dramatic improvement after taking Charlotte’s Web and about another 50 percent have had some improvement.
- Doctors in Colorado and researchers at Stanford University have collected summaries of patient histories suggesting positive impact of CBD in children with epilepsy.
- However, these reports are anecdotal; no rigorous clinical trials of either medication have been completed yet.
- GW Pharmaceuticals is completing design arrangements with the FDA for clinical trials of Epidiolex and expects to initiate these studies in 2014.
- The Stanleys are pursuing efforts to develop a clinical trial of Charlotte’s Web using a study design similar to that of the Epidiolex studies.
In the meantime, medications containing CBD should be available to children with severe epilepsy, but should be considered a last resort until further research is completed.
- Administration of Charlotte’s Web and Epidiolex to children who have epilepsy has been limited to severe, intractable cases where other established medication regimens have proved unsuccessful. These children and their families have had few if any treatment options left.
- Until clinical trials provide more information about the efficacy of these medications, they should not be prescribed unless other medications with more established track records have been tried without success.
- The possible long-term effects of CBD use by young children are unknown.
Parents and professionals contemplating the use of CBD for children with epilepsy should be cautious in arriving at informed treatment decisions.
- The FDA plays no role in ensuring the quality and consistency of medical marijuana products, and marijuana dispensaries in Colorado are not required to submit their products to any independent testing.
- The Stanleys have submitted Charlotte’s Web for third-party testing in an attempt to ensure that the medication has consistent chemical content. This testing is not legally required. In late 2013 the Stanleys hired an in-house chemist with pharmaceutical industry experience to conduct testing; they indicate that they continue to send about one-fifth of their samples for third-party verification.
- Other dispensaries or growers in Colorado may claim to offer products that are high in CBD and/or low in THC. Given the lack of regulatory oversight or testing requirements, the only reliable way to verify the CBD or THC content of these medications (if it has not been verified already) is to have the product independently tested by a qualified laboratory.
- There have been reports of psychoactive reactions in children with severe epilepsy who took cannabis obtained from other sources as an experimental treatment.
- Due to the newness of these medications, little is known about how they operate in the body or about possible interactions with other medications that patients may also be taking.
- The unusual legal status of medical marijuana in the United States today presents additional complications. For example, medications containing CBD cannot legally be transported across state lines or administered by school nurses.
- Charlotte’s Web and Epidiolex are not covered by health insurance.
- The cost of Charlotte’s Web ranges from $100 to $600 per month, depending on dosage level and patient weight. Some families with financial need receive discounts through the Stanleys’ dispensary.
- GW Pharmaceuticals is currently providing Epidiolex at no cost for patients in INDs and forthcoming clinical trials. If the FDA approves Epidiolex for medical use following clinical trials, it could then become eligible for insurance coverage.
Government actions could improve the current situation regarding medications containing CBD, with regard to both treatment and research.
- We support the proposal by Colorado state health director Larry Wolk, MD, MSPH to earmark funds for research on the medical impact of marijuana-related treatments.
- Marijuana is currently classified as a Schedule I substance, with no medicinal value, on the federal schedules of controlled substances. A change in this classification could make it easier to conduct research on CBD-based medications, or for physicians to obtain and prescribe them. We join the national Epilepsy Foundation in calling on the Drug Enforcement Administration to implement a lesser schedule for medications containing CBD so that they can be more accessible for medical research.
- The federal government should provide assurance to healthcare organizations that their eligibility for federal funding will not be endangered if members of their organizations participate in research on medical marijuana.
- Federal and state governments should consider permitting children with severe illnesses to carry legitimate medications containing CBD across state lines.
- Expanded regulatory activity may be warranted to protect against the marketing of “high-CBD” medications for children that could also be high in THC or pose other risks.