March 17, 2014 at 9:05 AM ET
After medications failed to quiet 2-year-old Grace Burriesci’s seizures, her family packed up and moved from New York to Colorado, hoping that a special strain of medical marijuana grown there could help their daughter.
Diagnosed with Dravet Syndrome when she was a year old, the little girl had been suffering as many as 400 seizures a day. Her family feared that if the seizures couldn’t be brought under control, they’d lose Grace.
“The FDA-approved pharmaceuticals weren’t working for her and unfortunately, kids that have her condition are actually passing away from it, so it’s a matter of life and death,” Grace’s dad, Biagio Burriesci, told TODAY.
After researching her condition, the Burriesci family learned about a strain of marijuana called Charlotte’s Web that has been helping other kids with intractable seizures.
This particular type of marijuana has been selectively bred to have low levels of a cannabinoid called THC, which is the plant’s psychoactive ingredient, and higher levels of cannabidiol, or CBD.
While both cannabinoids impact pain, nausea and seizures, CBD isn’t psychoactive, experts say. So that means that kids taking this form don’t get high.
Cannabinoids work by hijacking normal brain circuitry. In other words, they are very similar to substances our own brains naturally make called endocannabinoids, which serve to quiet excessive activity, whether it’s in the immune system, the gut or the nervous system.
When cells become overactive, a switch in the brain is thrown and endocannabinoids are released, calming things down. The hope is that marijuana will tune up that system.
In Grace’s case, marijuana hasn’t completely banished seizures, but it’s cut them back from 400 per day, to about 20, her parents said.
Results like that are what have brought nearly 200 families to Colorado seeking an option that has little scientific research to back it up, but plenty of anecdotal stories.
Not long ago, another family moved down the street from Grace and her parents. Just like the Burriescis, the Botkers were hoping to help a child with uncontrollable seizures. Their 7-year-old daughter Greta had tried medications and even surgery to quiet the storms in her brain, but to no avail.
That all changed when she started taking Charlotte’s Web. Her parents say Greta’s seizures have dropped from 15 per day to three.
Though it means that her parents must split their time between their home in Colorado and their farm in Minnesota, they say it’s worth it.
“There’s nothing we wouldn’t do to try to help her, and that’s why we’re here in Colorado,” Mark Botker, Greta’s dad, said.
While success stories like these are promising, the American Epilepsy Society cautions parents that there isn’t solid evidence that marijuana works — or is even safe for kids.
“The recent anecdotal reports of positive effects of the marijuana derivative cannabidiol for some individuals with treatment-resistant epilepsy give reason for hope,” the American Epilepsy Society said in a statement. “However, we must remember that these are only anecdotal reports, and robust scientific evidence for the use of marijuana is lacking. The lack of information does not mean that marijuana is ineffective for epilepsy. It merely means that we do not know if marijuana is a safe and effective treatment for epilepsy.” You can read the full AES position on medical marijuana here.
That lack of research may only be temporary. In October of last year the FDA approved two investigational trials of cannabidiol for the treatment of pediatric epilepsy, one at the New York University school of Medicine, the other at the University of California San Francisco.
The pediatric neurology group at the David Geffen School of Medicine at Mattel Children's Hospital UCLA is awaiting OK from the FDA and the university to begin a trial. “At the moment we’re anticipating 25 patients per group at each center,” said Dr. Raman Sankar, a professor and chief of the division of pediatric neurology. “Our conservative hope is that this will begin sometime this year, and maybe within the next few months.”
Sankar’s group will be using a formulation developed by GW Pharmaceuticals, a British company, that has gotten regulatory approval in Europe for another cannabinoid formulation, named Sativex, for the treatment of spasticity caused by multiple sclerosis.
Once scientists realized that Dravet Syndrome was caused by a genetic mutation, it put the illness in a different light. It also suggested that this might be a kind of epilepsy that could be helped by therapeutics like cannabidiol.
One issue that has held up research for many years is the difficulty for researchers to get supplies of marijuana because it is listed as a Schedule I drug by the DEA, along with heroin and LSD, Sankar said.
“You can’t very easily do even a mouse study with it,” he said.