Clinical Trial of GWP42006/Cannabinoid for Epilepsy & Tuberous Sclerosis

One of the reasons we travel to Boston is in hopes of being part of a clinical trial, like the one Oliver will get to participate in next year. As you can imagine, any FDA approved Cannabis clinical trail requires many levels of approval. Dr.Theile tells us she has two people working on this clinical trial almost round the clock. We're thankful there is a physician willing to work so tirelessly for our sweet son, who has already endured 2 brain surgeries, the implantation and explantation of a VNS device, a therapeutic diet, and the failed use of 17 anti-epileptic medications (one of which can cause permanent peripheral blindness). We literally thank God someone is willing to carry our banner before our government and regulatory agencies to relieve the suffering of our only child. We are honored and feel blessed to be part of a landmark study on the efficacy of cannabis for intractable pediatric epilepsy. As you can tell, we've literally tried everything modern medicine has to offer...

As best we understand it here is how it works. There are 3-4 sites in the US for this clinical trial for pediatric epilepsy patients. The trial is sponsored by GW Pharmaceuticals out of the UK and is a Phase I trial. This company has been working on Cannibas for medical treatment for years, and is a world-wide leader. We think we might buy some stock (it is a publicly traded company) NASDAQ: GWPH. At Mass General there were 25 kids selected, and they'll all get the real med, no placebos. Around 4 or 5 of the kids have TSC, others have epilepsy for other reasons (Dravet, Syndrome, Angelman Syndrome, etc.). The trial at Mass General has to go through their Internal Review Board (IRB), which is notorious for being the most difficult anywhere on the face of the earth. NYU's trial of GWP42006 just got through their IRB, so Dr.Thiele is hopeful Mass General won't be too far behind them. Assuming the endless emails from the regulatory powers (and their are many) get resolved, the trials should begin in February and last a year. One of the challenges is that despite this being totally legal and approved for this trial, cannabis is still a Schedule I drug in the US.

In many clinical trials of this kind, the drug manufacturer will give travel considerations to families. Not so in this case. We'll need to fly to Boston to start the trial twice in two weeks after it begins, and every month thereafter (or at least that is our understanding at the moment). As you can imagine, this is going to be seriously expensive! We're totally willing to do whatever it takes to make this happen for our sweet Ollie.

If the drug works for Oliver and we'd like to continue in year #2, we can. This is great news; however, we have to pay out of pocket and it could cost 10,000-15,000 a year. You read correctly, 10-15K a year. Apparently, another one of the TSC drugs (Affinitor) costs 10-15K a month, so I suppose we shouldn't complain?

We are so, so grateful that we get to do this in a clinical trial and not some other way. We're thankful we can still have comprehensive care from Dr.Thiele, and that we're clear on the source of the cannabis that Oliver will receive. We know many other parents who have not had the luxury of being selected for a clinical trial for Cannabis (only 75-100 kids on the entire United States are going to have this opportunity!)

To be 100% clear, what makes people "high" from cannabis is the THC. The form of Cannabis that Oliver will get will be in a liquid form and have no THC. He won't get "high" and he won't be smoking anything (yes, we have been asked this more than once). They extract the helpful ingredients from the plant and leave the rest out.  No brownies, no smoke, just plain-old medicine bottles.

As with most things brand-new, there are many unknowns. Will we have to take Oliver with us every month or can we just go to get the medication? What will all the plane tickets and other expenses cost over the next year?

Obviously,  most important quest of all:  Will GWP42006 work for Oliver? Will it give him some relief from the endless barrage of seizures? Will it un-cloud his mind and help him speak more freely?

If the trial doesn't work, then we will be looking into a second surgery for Oliver. Amazingly, on the plane ride to Boston I happened to sit by a man who works with a very new form of neurosurgery called Six Pillars. I have lots of research to do on this, but it sounds like it has some promise, and I'm forwarding it to Dr.Duhaime (Oliver's neurosurgeon). You never know who God will put in your path...