Robert McCoppin
A marijuana extract significantly reduced seizures in severely
epileptic children, according to a landmark study conducted in part at
Lurie Children's Hospital of Chicago.
Supporters said the results greatly improve the chances for the drug, called Epidiolex, to win eventual approval by federal regulators for prescription use to treat Dravet syndrome, a debilitating type of epilepsy that strikes in early childhood. The drug would be the first derived from the marijuana plant to win such approval.
More broadly, the findings also support claims made by advocates of medical marijuana, but represent a much more controlled and limited use of the plant.
Children with Dravet syndrome typically have frequent seizures that cause problems with language, motor skills and relating to others. In the case of one patient in the trial at Lurie, 5-year-old Caleb Yunk, the disease has not been as severe but caused chronic seizures that led to difficulties with skills such as running, jumping and balancing.
In the trial, Caleb swallowed about 3 milligrams of a strawberry-flavored syrup twice a day. Once he started treatment, his parents said, the Milwaukee-area boy went 100 days without a seizure, and those he did have lasted less than half as long. Because Caleb's seizures can be triggered by overheating, his parents had kept him indoors on hot days but on the drug, he showed enough progress to go swimming with his friends.
"His eyes sparkle more," said his mother, Kelley Yunk. "He's more engaged. I think he's more cheerful. For our family, it was such a huge blessing."
Much of the improvement came from being able to take Caleb off another drug and reduce his use of two others — medication that has serious side effects and made him "dopey," his parents said. Side effects seen during the Epidiolex trial included sleepiness, diarrhea and decreased appetite but were mostly mild or moderate, according to the manufacturer's reported results.
Epidiolex is almost pure cannabidiol, or CBD, a non-psychoactive compound found in marijuana, without the THC component that gets users high.
The study at Lurie was part of a nationwide clinical trial conducted by the drug's maker, GW Pharmaceuticals. While a preliminary study also produced encouraging results, the company said this was the first trial of plant-based CBD involving a randomized, double-blind placebo control, known as the gold standard of disease research.
Double-blind means neither the patient nor the researchers know whether the patient is getting the drug or a placebo, in this case a similar liquid that doesn't contain any medicine, to avoid results being biased by optimistic interpretations.
The study involved about 120 patients, aged 2 to 18, who have Dravet syndrome and whose seizures were resistant to existing medications. Among patients who received the drug, convulsive seizures were reduced by an average of 39 percent, compared to 19 percent for the placebo, over 14 weeks last summer, according to results announced in March.
Officials are hopeful that this will speed up approval by the U.S. Food and Drug Administration to make Epidiolex available by prescription.
The study is one of four trials of Epidiolex to treat Dravet and a similarly serious form of epilepsy known as Lennox-Gastaut syndrome. Both conditions are rare, with an estimated combined patient population of roughly 30,000, and Dravet has no FDA-approved medication specifically for its treatment.
The promising results mean the company will seek a review by the U.S. Food and Drug Administration by the end of this year and hopes to win approval to prescribe Epidiolex by the end of 2017, GW Pharmaceuticals CEO Justin Gover said. The drug has Fast Track designation from the FDA.
Initially, Epidiolex would be only for children who have not responded sufficiently to other medications.
It's too early to predict the cost of the drug, Gover said, but the company hopes to convince insurance companies to cover it.
"We're obviously delighted" with the results, he said, calling it a "significant milestone."
UK-based GW Pharmaceuticals already offers Sativex, a drug derived from marijuana that contains equal parts THC and CBD and treats muscle spasms and stiffness in multiple sclerosis patients in Europe and Canada, though it is not available in the U.S. except in trials.
In a major disappointment for advocates, trials last year failed to show that Sativex worked better than a placebo for cancer pain overall. But some less-frail patients who had pain despite using opioids did benefit, so researchers said it merited further investigation. GW Pharmaceuticals also plans to test treatments for various other conditions, including glioma, an aggressive form of brain cancer.
"We believe the lawsuit is completely baseless and without merit," he said.
Company officials emphasized that Epidiolex is very different than marijuana, because it is formulated as a concentrate of just one component of the plant, with pharmaceutical controls to guarantee the same product in every dose.
But medical marijuana advocates consider the Epidiolex results as verification of what they have been saying for years based on patients' stories, that marijuana can help those with epilepsy, which affects about half a million children in the U.S.
Ross Morreale, chairman of the Medical Cannabis Alliance of Illinois and co-founder of marijuana grower Ataraxia, said state law requires lab tests to guarantee potency and purity of medical marijuana, and it is already helping patients with a variety of medical conditions, without waiting for federal approval.
Illinois children with epilepsy may qualify to access medical marijuana under state law, but it remains illegal under federal law, and many doctors here are reluctant or prohibited by their employers from recommending it.
The principal investigator for the study at Lurie hospital was Dr. Linda Laux, a pediatric neurologist who specializes in helping children with treatment-resistant epilepsy.
Epidiolex did bring some positive changes in the patients' behavior, attention and focus, as well as motor skills such as walking, she said. Patients have been able to continue taking the drug after the trial.
"These results are very exciting and significant," Laux said. "This is absolutely a ray of sunshine and hope for these families."
Morgan Murphy, spokeswoman for the Epilepsy Foundation of Greater Chicago, welcomed the study results, which made her optimistic that the drug will get FDA approval.
"We've been waiting so long for something
to come around," she said. "We're getting such a positive reaction from
patients. It's a great step forward."
Supporters said the results greatly improve the chances for the drug, called Epidiolex, to win eventual approval by federal regulators for prescription use to treat Dravet syndrome, a debilitating type of epilepsy that strikes in early childhood. The drug would be the first derived from the marijuana plant to win such approval.
More broadly, the findings also support claims made by advocates of medical marijuana, but represent a much more controlled and limited use of the plant.
Children with Dravet syndrome typically have frequent seizures that cause problems with language, motor skills and relating to others. In the case of one patient in the trial at Lurie, 5-year-old Caleb Yunk, the disease has not been as severe but caused chronic seizures that led to difficulties with skills such as running, jumping and balancing.
In the trial, Caleb swallowed about 3 milligrams of a strawberry-flavored syrup twice a day. Once he started treatment, his parents said, the Milwaukee-area boy went 100 days without a seizure, and those he did have lasted less than half as long. Because Caleb's seizures can be triggered by overheating, his parents had kept him indoors on hot days but on the drug, he showed enough progress to go swimming with his friends.
"His eyes sparkle more," said his mother, Kelley Yunk. "He's more engaged. I think he's more cheerful. For our family, it was such a huge blessing."
Much of the improvement came from being able to take Caleb off another drug and reduce his use of two others — medication that has serious side effects and made him "dopey," his parents said. Side effects seen during the Epidiolex trial included sleepiness, diarrhea and decreased appetite but were mostly mild or moderate, according to the manufacturer's reported results.
Epidiolex is almost pure cannabidiol, or CBD, a non-psychoactive compound found in marijuana, without the THC component that gets users high.
The study at Lurie was part of a nationwide clinical trial conducted by the drug's maker, GW Pharmaceuticals. While a preliminary study also produced encouraging results, the company said this was the first trial of plant-based CBD involving a randomized, double-blind placebo control, known as the gold standard of disease research.
Double-blind means neither the patient nor the researchers know whether the patient is getting the drug or a placebo, in this case a similar liquid that doesn't contain any medicine, to avoid results being biased by optimistic interpretations.
The study involved about 120 patients, aged 2 to 18, who have Dravet syndrome and whose seizures were resistant to existing medications. Among patients who received the drug, convulsive seizures were reduced by an average of 39 percent, compared to 19 percent for the placebo, over 14 weeks last summer, according to results announced in March.
Officials are hopeful that this will speed up approval by the U.S. Food and Drug Administration to make Epidiolex available by prescription.
The study is one of four trials of Epidiolex to treat Dravet and a similarly serious form of epilepsy known as Lennox-Gastaut syndrome. Both conditions are rare, with an estimated combined patient population of roughly 30,000, and Dravet has no FDA-approved medication specifically for its treatment.
The promising results mean the company will seek a review by the U.S. Food and Drug Administration by the end of this year and hopes to win approval to prescribe Epidiolex by the end of 2017, GW Pharmaceuticals CEO Justin Gover said. The drug has Fast Track designation from the FDA.
Initially, Epidiolex would be only for children who have not responded sufficiently to other medications.
It's too early to predict the cost of the drug, Gover said, but the company hopes to convince insurance companies to cover it.
"We're obviously delighted" with the results, he said, calling it a "significant milestone."
UK-based GW Pharmaceuticals already offers Sativex, a drug derived from marijuana that contains equal parts THC and CBD and treats muscle spasms and stiffness in multiple sclerosis patients in Europe and Canada, though it is not available in the U.S. except in trials.
In a major disappointment for advocates, trials last year failed to show that Sativex worked better than a placebo for cancer pain overall. But some less-frail patients who had pain despite using opioids did benefit, so researchers said it merited further investigation. GW Pharmaceuticals also plans to test treatments for various other conditions, including glioma, an aggressive form of brain cancer.
"We believe the lawsuit is completely baseless and without merit," he said.
Company officials emphasized that Epidiolex is very different than marijuana, because it is formulated as a concentrate of just one component of the plant, with pharmaceutical controls to guarantee the same product in every dose.
But medical marijuana advocates consider the Epidiolex results as verification of what they have been saying for years based on patients' stories, that marijuana can help those with epilepsy, which affects about half a million children in the U.S.
Ross Morreale, chairman of the Medical Cannabis Alliance of Illinois and co-founder of marijuana grower Ataraxia, said state law requires lab tests to guarantee potency and purity of medical marijuana, and it is already helping patients with a variety of medical conditions, without waiting for federal approval.
Illinois children with epilepsy may qualify to access medical marijuana under state law, but it remains illegal under federal law, and many doctors here are reluctant or prohibited by their employers from recommending it.
The principal investigator for the study at Lurie hospital was Dr. Linda Laux, a pediatric neurologist who specializes in helping children with treatment-resistant epilepsy.
Epidiolex did bring some positive changes in the patients' behavior, attention and focus, as well as motor skills such as walking, she said. Patients have been able to continue taking the drug after the trial.
"These results are very exciting and significant," Laux said. "This is absolutely a ray of sunshine and hope for these families."
Morgan Murphy, spokeswoman for the Epilepsy Foundation of Greater Chicago, welcomed the study results, which made her optimistic that the drug will get FDA approval.
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