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t m c ยป p u l s e | d e c e m b e r 2 0 1 4 13 in it?" said Angus Wilfong, M.D., medical director of the Comprehensive Epilepsy Program at Texas Children's Hospital and associate professor of pediatrics and neurology at Baylor College of Medicine. Wilfong has worked with epilepsy patients for years. He developed a revolutionary MRI-guided laser brain surgery now in use throughout the country. The stories of Charlotte's Web caught his attention, and when GW Pharmaceuticals developed the phar- maceutical-grade Epidiolex, they were eager to try it. "My research team became very interested in getting access to it for some of our patients who are suffering desperately from epilepsy, because we knew that it was going to be pure CBD," said Wilfong. "We know that if we give a child this formulation, it has exactly 100 milligrams of CBD in every millili- ter and doesn't have anything else in it." Becoming one of the ten sites to test Epidiolex worldwide, however, was not easy, particularly given that medical marijuana is not legal at the federal level or at the state level in Texas. Representatives of the United States Drug Enforcement Administration interviewed Wilfong and toured his pharmacy. The Epidiolex itself must be kept inside a locked vault. "It's a very rigorous process because marijuana and any derivative of mari- juana is considered a Schedule 1 drug by the DEA and can't be prescribed legally according to the federal govern- ment," said Wilfong. "Only researchers can use it under very strict regulation and guidelines from the government." This trial is the very first worldwide research study on a medical marijuana product. The first stage, in which Izaiah is enrolled, is largely intended to deter- mine dosage. Thirty patients between the ages of four and ten will be ran- domly assigned a low dose, a medium dose, a high dose or a placebo. Once that round is complete, at the begin- ning of 2015 a second round will begin covering a broader age range of two to 18 and a greater number of patients. "We're hopeful that after the studies are done, we'll show it works for child- hood epilepsy," said Wilfong. "That it's safe and doesn't have abuse potential, and hopefully it will be taken off the Schedule 1 list." As Wilfong and his team fulfilled their obligations to the DEA and top: angus wilfong, m.d., evaluates patient scarlett for the epidiolex trial (credit: allen Kramer/texas children's hospital) lower left: izaiah with his grandmother, lori fountain lower right: epidiolex (credit: gw pharmaceuticals) prepared to seek out patients, Izaiah's grandmother, Lori Fountain, had been doing her own research. "I belong to a Dravet support group, so I have read and seen everything about Charlotte's Web and how it has helped some of these children," said Fountain. "I've seen these children be able to function better, to speak where they didn't have so many words before." Fountain and her family contem- plated moving to Colorado until they heard of the trial at Texas Children's. She was instantly on board. "I jumped. I said, 'I want to try this. Anything to help him. Anything to have a normal life,'" said Fountain. "I will stop the world and rearrange it to make sure we can get in the study." Wilfong said her excitement is not unusual when it comes to CBD. People are "clamoring for it." "We're really, really desperate for new treatments, so there's been this groundswell of interest related to med- ical marijuana and derivatives of the marijuana plant," he said. Dravet syndrome is rare, but seizures and epilepsy are not. One in 10 people will have a seizure, and one in 100 have epilepsy. Despite how common it is, there is still no fail-safe treatment. "Our current treatments for epilepsy only work about 60 percent of the time, so that means there's 40 percent of children and adults living with epilepsy who have uncontrollable seizures," said Wilfong. "Some of those people living with uncontrollable seizures can benefit from brain surgery. Lots of people that have uncontrolled seizures can't." Izaiah's seizures are relatively under control, thanks to medication. That con- trol, however, comes at a price. Fountain says side effects make him drowsy and glassy-eyed. "I don't see the brightness in his eyes that some children have," she said. "He always looks like he's just tired and I would like to see that look go away." The prevalence of seizure disorders combined with a frustrating lack of options has made Colorado popular in the last few years, but the results are mixed. Given the lack of regulation there, however, a scientifically sound clinical trial using the pharmaceuti- cal-grade Epidiolex is vital for deter- mining the efficacy of CBD. Wilfong's team has already seen some success. The first patient began taking Epidiolex six months ago and has significantly improved. "He was having over 200 seizures a day," said Wilfong. "I just saw him in the clinic recently and he's having one to seven seizures a day now." Though promising, Wilfong cautions the full trial must be complete before drawing credible conclusions. "We believe it was due to the CBD, but it's not proof," he said. "The only way you can prove something works is to study it in a scientific, controlled study." Still, for Fountain, any news of success brings hope. Fountain spends nearly every waking moment with her grandson. She is by his side at school and she sleeps in his room every night. She is fiercely protective of Izaiah and has an answer for anyone who ques- tions using a medicine derived from marijuana. "There are some people that have not been so nice about it and I've asked them to spend a day in my shoes," she said. "Please come spend one day in my shoes and then tell me that you wouldn't do everything you could possi- bly for your child." Stigma against marijuana has been an obstacle to researching drugs derived from the plant. Fountain and Wilfong are both quick to point out Epidiolex does not contain any tetrahy- drocannabinol, or THC, the compound that gives users a high. "The THC is what gets you high. The CBD does not," Fountain said. "I don't want to get my grandson high. That's not the point." The point of taking part in this study, Fountain's greatest wish, is simple: to give her grandson a normal childhood. Ask her to talk about him and her voice immediately softens; her devotion to Izaiah is clear. "Every day that we wake up is a good day, because that means he's in my life one more day. He could have that one seizure that could kill him, and that's on a daily basis," she said. "I just want to give my little boy a normal life. He may not make it to be a teenager. He may not make it to be an adult. But to give him a quality life, to be a normal little child as best as he can be with Dravet syndrome would be priceless."