Dr. Elizabeth Thiele is well known for her work, and has co-authored several studies on the use of cannabidiol in children with Dravet syndrome, Lennox-Gastaut syndrome, and now tuberous sclerosis complex.
Her accolades and achievements are renowned, but her post as professor of neurology at Harvard Medical School is what really distinguishes her as an expert in her field. Neurology Advisor interviewed her at the 2016 Child Neurology Society Annual Meeting to discuss the state of cannabidiol research for the treatment of refractory epilepsy.
Dr. Thiele has been working with children with epilepsy for 20 years, and says it has been very exciting to see the number of new medications developed over the last 10 to 15 years. There are many more treatment options than when she started studying this field of medicine.
Many children’s seizures remain refractory
The frustration for families living with epileptic children, the people taking care of patients, and doctors are that there are still large numbers of children whose seizures are not controlled despite the new medications. These children remain refractory, and there continues to be a big unmet need for effective treatments in epilepsy.
Over the last years, there have been increasing numbers of anecdotal reports that it can be effective in treating epilepsy in children after medical marijuana became available in California, and then in Colorado.
A selection of our products
GW Pharma spent many years
Several years ago, GW Pharma from the UK was identified as a company very interested in cannabinoids for the treatment of epilepsy for many, many years. They had been doing pre-clinical work, and a group of doctors got together with GW to discuss if cannabidiol would be a safe and effective treatment.
Expanded access program
The expanded access program was formed, with five academic centers putting in an identification investigation into new drug application for cannabidiol treatments with the FDA and DEA, asking to test this medication on children.
Each centre was granted permission to put 25 children on the program. Each centre chose their 25 most highly refractory epilepsy patients. Most of them has been on 10, 12, 14, or even 16 different medications. Many had epilepsy surgery, many were on diets, many had been on the Vagus nerve stimulator and all of them were still having uncontrolled daily seizures.
The expanded access program results were very encouraging. There was a significant reduction in seizures, and cannabidiol CBD was well tolerated. There were side effects, but doctors learned they were due to medication interactions, which they could control.
Application to the FDA
Based on the expanded access program data, which consisted of open labeled trials, not randomized controlled trials, GW was encouraged to proceed with applications to the FDA to further investigate cannabinoids as treatment in epilepsy.
Randomized trials were sponsored by GW, and met with positive results. Again, this was very encouraging as GW plans on submitting for approval on studies into Lennox-Gastaut syndrome and Dravet syndrome. The epilepsy community is hopeful that the medication will be approved.
Dr. Thiele is very hopeful that it will be given the green light, because she is not happy with the concept of patients who still have to use marijuana preparations that are not consistent from batch to batch, causing complications and difficulties. Another complication pointed out by studies is that some products labeled high in CBD have been found to contain no CBD when analyzed. A big concern is cannabis not being tested for pesticides or heavy metals, and other contaminants, which could have significant negative impact on the health of a child.
Dr. Thiele’s ideal world
In Dr. Thiele’s ideal world, the FDA will recognize these problems, and acknowledge that there are many people who can benefit from CBD. She wants to see doctors having access to cannabidiol to treat epilepsy, and she hopes that approval will be given sooner rather than later.