Medical marijuana continues to make the news these days, and not just over the ethical and legal issues involved in destigmatising and legalising of this heretofore illicit substance; this week the news was abuzz about its potential use as a new candidate therapeutic for severe epilepsy. There have been other proposed benefits in adults of the plant-derived substance that maybe seemed more obvious (pain, for example) but it is very intriguing that researchers may have stumbled upon something that will benefit children in particular.
The UK's GW Pharmaceuticals has been a proponent of the more pharmaceutically-minded sector of the medical marijuana movement for the past several years, and has hinted at promise for the green stuff in the treatment of severe, treatment-resistant epilepsy. This is an extremely debilitating condition and regulatory authorities have granted GW entry to an expanded access program which allows such patients to receive unapproved products in the face of little other option or hope.
What got the attention of the big news networks this week was the announcement by GW at the American Academy of Neurology that their experimental drug Epidiolex reduced seizure rates by as much as 50% (or more) in an open-label study. Now, no one wants to overreach on this and claim that the data obtained replaces the need for a full placebo-controlled clinical trial, but the idea was to get an early read on things and generate some excitement on GW's aligned Phase III trial for the drug candidate.
Notwithstanding the use of the word "high" in the title above, the active ingredient in GW's treatment is cannabidiol, a component of marijuana that is not involved in inducing the high associated with the plant's use. This aspect should help to somewhat legitimise the use of the drug (because we can't have terminally sick people experiencing a high, can we?!) and even separate it from the term "medical marijuana" given that no smoking is involved - which is a very good thing given that children are going to be primary recipients of the therapy.
GW has developed an oral droplet form of the cannabis-derived molecule which does retain the beneficial medical effects of the compound, including its recognised anti-seizure and anti-inflammatory properties. Like marijuana itself, the legality of cannabidiol is somewhat hazy (you have to peer through the smoke!) in that it varies from territory to territory. But in many cases (including in the USA), cannabidiol remains classified as a Schedule I controlled substance, making even possession of it illegal.
The clinical study being run by NYU's Dr. Orrin Devinsky has achieved a remarkable 54% reduction of seizures observed in 137 patients who were on the therapy for 12 weeks, including reaching similar numbers in those afflicted with either Dravet or Lennox-Gastaut syndrome. GW has or is preparing to enrol patients in Phase III trials in the latter two categories (electrically distinct forms of seizures) this year, and such data are extremely encouraging, especially as Epidiolex will be given fast track review by the FDA as an orphan-designated drug candidate.
As esoteric as the venture may seem alongside more classical pharma medicines, GW is building a solid development pipeline from its cannabinoid platform, and there are ongoing trials in cancer pain, diabetes, ulceritive colitis, schizophrenia and epilepsy. If they are on the right track, as they appear to be based on some of the recent data shared with the media, then they could be in for a very substantial payday via their relatively diverse pipeline. They previously partnered their oral cabbaninoid Sativex (for multiple sclerosis) with Bayer, have also partnered it for cancer pain, and this drug has since been launched in 15 countries with regulatory approval in another dozen - so they are going places.
GW's cause is garnering attention from various epilepsy advocate organizations such as the Epilepsy Foundation in the USA, who are lobbying federal agencies to build on the promise achieved to date by loosening regulations and increasing access to cannabis-derived medication when it is warranted. Their current mandate includes -
- Calling on the Drug Enforcement Administration to implement a lesser schedule for marijuana so that it can be more easily accessible for medical research
- Supporting appropriate changes to state laws to increase access to medical marijuana as a treatment option for epilepsy, including pediatric use as supported by a treating physician
- Supporting the inclusion of epilepsy as a condition that uses medical marijuana as a treatment option where it is currently available
- Supporting research on multiple forms of cannabis and seizures
There is definitely some movement in the right direction for advocates, patients and drug developers alike, and if GW can achieve the data hoped for in their placebo-controlled Phase III trials then they may well be riding high while keeping seizures in epileptics low. Their current high of $114 on Nasdaq is surely a reflection of that optimism and the future for all concerned might be looking very green indeed when the smoke begins to clear!
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