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How Patients' Families Pushed Doctors To Study A Marijuana Extract For Treatment Of Seizures

An incredible story of patient advocacy.

04/05/2017 8:50 AM IST | Updated 04/05/2017 8:50 AM IST
Nir Elias / Reuters

There's a dictum that medical students around the world get to hear often: Listen to your patients and their families, and ignore their words and pleas at your own peril. Good clinicians remind themselves of this every single day in order to treat their patients well. However, this dictum takes on a new meaning if one looks at the story of the use of cannabidiol to treat severe seizures.

The initial data from the studies have been promising with a reduction in enrolled participants' seizures by about a third.

Marijuana has been used for thousands of years to treat a variety of medical conditions. Its first definitively documented use to treat seizures was in the 19thcentury. There was a paucity of research data, in part due to the fact that it is considered a drug for recreational use and carries a resultant negative image particularly due to its addictive potential. However, in 2013, it received worldwide publicity because of the case of Charlotte Figi who suffered from a severe form of epilepsy called Dravet syndrome, and benefited from a marijuana extract that was high in cannabidiol (CBD—medicinal, non-psychoactive component) and low in tetrahydrocannabinol (THC—psychoactive ingredient). Charlotte lived in Colorado, which was one of the first states in the US to legalise the use of medical marijuana back in 2012. She suffered from scores of seizures every day, with none of the traditional treatments helping her. She then improved with a mixture high in CBD and low in THC. Subsequently, the high-profile CNN reporter Dr. Sanjay Gupta broke the then 6-year-old Charlotte's story to the world, and what followed is quite unparalleled in the history of health sciences. Desperate families of patients with severe, difficult-to-treat seizures flocked to Colorado. Stanley Brothers, based in Colorado and in the business of medical marijuana for a while before Charlotte's case became public, made her even more famous by naming their product Charlotte's Web and promising to deliver a product high in CBD and low in THC. Neurologists in the US started getting requests from families to prescribe CBD, only to get careful negative responses that tried to highlight the lack of scientific data. To make matters worse, there was much confusion about the medicinal and psychoactive components. Many outlets allegedly even indulged in taking advantage of distraught families.

Subsequently, a pharmaceutical company out of the UK started randomised, double-blind, placebo-controlled trials to study the effect of pure CBD on seizures associated with severe epilepsies including Dravet syndrome. The US Food and Drug Administration (FDA) has not yet approved CBD to treat seizures and epilepsy. However, the initial data from the studies have been promising with a reduction in enrolled participants' seizures by about a third. Since then, families of epilepsy patients have successfully campaigned in several states to help pass legislation to allow CBD use. There are methodological concerns raised by sceptical experts regarding the clinical trials of CBD. There's also the concern that CBD has helped patients not because of its anti-seizure properties, but because it interacted with other drugs that the patients were already on, raising their blood levels, and thus leading to better seizure control. It remains to be seen if the drug will be approved, and reconfirmed to be effective and safe in the post-marketing phase.

[P]atients and their families are equal partners when it comes to not just the treatment but also research in order to push the science forward.

Irrespective of the ultimate outcome of various trials, the medical fraternity's experience with CBD has been quite unprecedented. Typically, drug development goes through quite a mundane initial phase of discovery, then preclinical research in animals to assess safety. This is followed by clinical research on humans to determine safety and efficacy. Public interest typically ensues if the drug is safe and effective. In case of CBD for seizures, the process has been anything but mundane or smooth. However, it has highlighted the power of patient advocacy, both by the families and by the communities. It has reminded doctors and researchers that patients and their families are equal partners when it comes to not just the treatment but also research in order to push the science forward.

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