Marijuana clinical trial a victory for epileptic kids, not legalization

Here's the key points:



1. FDA continues to believe that the drug approval process represents the best way to ensure that safe and effective new medicines from marijuana are available as soon as possible for the largest numbers of patients, and it is important and appropriate to apply these same scientific standards to the development and assessment of any therapeutic uses of marijuana.





less The drug approval process vs. marijuana as detailed in testimony by Douglas Throckmorton, a deputy director in the FDA, to the Committee on Oversight and Government Reform in the U.S. House of Representatives in 2014.FDA continues to believe that the drug approval process represents the best way to ensure that safe and effective new medicines from marijuana are available as soon as possible for the largest numbers of patients, and it is important and appropriate to apply these same scientific standards to the development and assessment of any therapeutic uses of marijuana. The drug approval process vs. marijuana as detailed in testimony by Douglas Throckmorton, a deputy director in the FDA, to the Committee on Oversight and Government Reform in the U.S. House of Representatives ... more Photo: Gilles Mingasson, Getty Images Photo: Gilles Mingasson, Getty Images Image 1 of / 18 Caption Close Marijuana clinical trial a victory for epileptic kids, not legalization 1 / 18 Back to Gallery

In what could be a landmark clinical trial on the benefits of a marijuana-derived drug for epilepsy, a UK-based pharmaceutical company says it's drug has significantly reduced seizures in the hardest hit kids.

GW Pharmaceuticals reported Monday:

"The primary efficacy endpoint was a comparison between Epidiolex and placebo measuring the percentage change in the monthly frequency of convulsive seizures during the 14-week treatment period compared with the 4-week baseline observation period. In this study, patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent compared with a reduction on placebo of 13 percent, which was highly statistically significant (p=0.01). "A series of sensitivity analyses of the primary endpoint confirmed the robustness of this result. The difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period."

The company said it's heading to the offices of the FDA to get approval for their drug based on these results. The company already has one drug ("Sativex") on the market in more than 20 countries (not the US, trials are ongoing) and hopes "Epidiolex (cannabidiol)" will be its first US approved cannabinoid medicine.

Again, that's great news ...

"The results of this Epidiolex pivotal trial are important and exciting as they represent the first placebo-controlled evidence to support the safety and efficacy of pharmaceutical cannabidiol in children with Dravet syndrome, one of the most severe and difficult-to-treat types of epilepsy," said Dr. Orrin Devinsky of New York University Langone Medical Center's Comprehensive Epilepsy Center, in the news release.

"These data demonstrate that Epidiolex delivers clinically important reductions in seizure frequency together with an acceptable safety and tolerability profile, providing the epilepsy community with the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future," he added.

And, it could be big money for the pharma: GW's stocks jumped and increased the value of the company by double, according to Reuters.

But, that won't mean the whole plant will be taken off the Schedule 1 list or made legal through federal-level action. In fact, it could mean just the opposite. The FDA, DEA and Department of Justice want to control drugs mainly for safety, which is a good thing. (... if they are actually making people safer and not just sticking a bunch of people in jail to no good effect as they have for decades in the war on marijuana.)

"In addition to its role in scheduling drugs, FDA sometimes works with the Department of Justice (DOJ), including DEA, and other state and Federal agencies on criminal investigations involving the illegal sale, use, and diversion of controlled substances," stated Douglas Throckmorton, a deputy director in the FDA, in testimony in the U.S. House in 2014.

He explained the hardline FDA role as ...

"In addition to establishing the safety and efficacy of the investigational drug, manufacturers also must demonstrate that they are able to consistently manufacture a high-quality drug product. This is an essential part of drug development and presents special challenges when the drug is derived from a botanical source, such as marijuana. "Botanicals include herbal products made from leaves, roots, stems, seeds, pollen or any other part of a plant. Botanical products pose challenges that are unique to this class of product, including lot-to-lot consistency. These unpurified products, which may be either from a single plant source or from a combination of different plant substances, can have effects through mechanisms that are either unknown or undefined, making it difficult to determine if the product is causing the change in a patient's condition, or the change is related to some other factor."

So, the backbone of legalization will be social justice, prison-sentencing reform, political commonsense, economic reality, etc. And, medical marijuana will eventually be something aficionados call the cannabis they buy from a recreational source. Just a guess.

Jake Ellison can be reached at 206-448-8334 or jakeellison@seattlepi.com. Follow Jake on Twitter at twitter.com/Jake_News. Also, swing by and *LIKE* his page on Facebook. If Google Plus is your thing, check out our marijuana coverage here.