Natalie Pembleton, 13, of Yorba Linda, has epilepsy and takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. Natalie sits in Parque De Los Vaqueros in Placentia on Thursday, August 16, 2018.(Photo by Mark Rightmire, Orange County Register/SCNG)
Natalie Pembleton, 13, of Yorba Linda, sits in Parque De Los Vaqueros in Placentia with her mother, Catherine, on Thursday, August 16, 2018. Natalie has epilepsy and takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. (Photo by Mark Rightmire, Orange County Register/SCNG)
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Natalie Pembleton, 13, of Yorba Linda, has epilepsy and takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. Natalie sits in Parque De Los Vaqueros in Placentia on Thursday, August 16, 2018.(Photo by Mark Rightmire, Orange County Register/SCNG)
Catherine Pembleton, of Yorba Linda, talks about her daughter Natalie, 13, having epilepsy on Thursday, August 16, 2018 in Placentia. Natalie takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. (Photo by Mark Rightmire, Orange County Register/SCNG)
Natalie Pembleton, 13, of Yorba Linda, who has epilepsy, swings in Parque De Los Vaqueros in Placentia on Thursday, August 16, 2018. Natalie takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. (Photo by Mark Rightmire, Orange County Register/SCNG)
Natalie Pembleton, 13, right, of Yorba Linda, sits in Parque De Los Vaqueros in Placentia with her mother, Catherine, left, on Thursday, August 16, 2018, as she talks about having epilepsy. She takes the drug, Epidiolex, to control her seizures. It is the first FDA-approved drug that contains a purified drug substance derived from marijuana to treat rare, severe forms of epilepsy. (Photo by Mark Rightmire, Orange County Register/SCNG)
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Two years ago, Natalie Pembleton was having so many epileptic seizures each day that her family had to pull her out of sixth grade.
The 12-year-old Yorba Linda resident was eventually placed in a drug-induced coma to give her fragile young body a rest while her medical team at UCLA scrambled to find a solution. Shed been hospitalized for months, and doctors were getting ready to risk brain surgery that could have left her partially paralyzed and unable to speak.
Today, Natalie is 13 and getting ready to start eighth grade. And both her parents and doctors say its thanks to Epidiolex, the first government-approved drug derived from marijuana.
“We think its a blessing from God that He sent here for Natalie,” the teens mom, Catherine Pembleton, said of the drug.
Epidiolex wont be widely available until this fall, but some seriously ill patients have been getting an early chance to try it out. And so far — as Natalies case and mounting evidence from researchers shows — the results are promising.
“Im feeling good,” Natalie said as she got ready to go for a swing in her favorite park. “I feel like I can finally live my life like normal kid.”
Some patients experience minor side effects with Epidiolex, such as tiredness and appetite changes. And doctors say the drug doesnt seem to work for every epilepsy patient, though its too early to have solid answers.
There also arent any head-to-head studies to show whether the pricey pharmaceutical drug calms seizures better than cannabis products people can buy now at dispensaries and health food stores, according to Dr. Shaun Hussain, a pediatric neurologist at UCLA whos treated Natalie for years. Thats because the federal status of marijuana, and the resulting stigma surrounding the drug, continue to severely hamper research and treatment options.
So, as promising as Epidiolex and similar drugs in the works seem to be, Hussain said this is far from the pinnacle of medical cannabis research.
“Its an important step forward,” he said, “but its really just the beginning.”
Novel drug from ancient source[hhmc]
Epidiolex is a syrup made by using a proprietary strain of marijuana to produce purified cannabidiol, or CBD.
CBD is one of more than 100 compounds found in cannabis. The chemical doesnt make consumers high. But, though Hussain said we dont fully know how it works in the brain, CBD has been proven to ease seizures in some patients with epilepsy.
The Food and Drug Administration approved Epidiolex on June 25. Before then, the government had only signed off on three drugs made from synthetic marijuana, all to treat nausea and appetite issues caused by diseases such as cancer and AIDS.
Epidiolex is cleared to treat patients 2 years and older who suffer from Lennox-Gestaut or Dravet syndromes, rare forms of epilepsy that begin in childhood. But GW Pharmaceuticals — the British company behind the drug, with U.S. headquarters in Carlsbad — has said they expect it will also be used to treat other types of epilepsy.
The concentrated CBD syrup reduced both the severity and frequency of seizures in a majority of adult and child patients with a variety of forms of epilepsy that had been resistant to other treatments, according to a study by University of Alabama researchers, published in the journal “Epilepsy & Behavior” earlier this month.
But before Epidiolex can become widely available, the Drug Enforcement Administration must remove the medicine from its Schedule I category on the Controlled Substances list. Marijuana and all of its compounds are currently in that category, which is supposed to be reserved for drugs that have a high potential for abuse and no proven medical use. The DEA is expected to carve out a special exception for Epidiolex by late September.
In the meantime, many epilepsy sufferers have been relying either on CBD oil made from hemp, which is widely available in health food stores and online, or on CBD oil made from marijuana, which can be purchased at licensed dispensaries.
Many families have reported positive, even seemingly miraculous results with these oils. The most famous of these cases is Charlotte Figi, a Colorado girl who was profiled on CNN in 2013 after CBD oil helped rescue her from a nearly catatonic state.
Despite their promise, there are drawbacks to store-bought CBD oils.
Not all hemp-based versions are lab tested for safety or properly labeled. Theres no regulatory agency currently overseeing production of these products, and even cannabis attorneys cant agree on whether CBD made from hemp is fully legal.
Marijuana-based versions are only legally available with a doctors recommendation in states that have approved medical cannabis for epilepsy. In California, Dr. Bonni Goldstein of Lawndale said products many parents have come to rely on have disappeared from the market overnight as the marijuana industry is forced to comply with new regulation and licensing requirements.
With both types of store-bought CBD oil, doctors often wont discuss treatment regimens because cannabis remains federally illegal. For that reason, parents may run the risk of legal or custody issues if they chose to medicate their children with standard CBD oil. Also, the products can cost families from perhaps $1,000 to tens of thousands of dollars a year, with no insurance coverage for any cannabis-based treatments.
Epidiolex is expected to cost about $32,500 for a patient for one year. Thats significantly less than Hussain feared it might cost, and its on par with many other epilepsy medications. And since Epidiolex is government sanctioned and will be shipped by pharmacies directly to patients, its expected to be covered by many insurance policies — at least for patients with Lennox-Gestaut or Dravet syndromes.
How insurance companies will handle patients with other forms of epilepsy — like the type that plagues Natalie — wont be clear until the drug hits the market this fall.
Hope for Natalie[hhmc]
Natalie is one of about 1,200 patients who have so far been able to access the drug for free through the generosity of GW Pharmaceuticals and the governments “compassionate use” program. The program allows physicians to request permission to use a drug that hasnt yet been approved when they have patients with life-threatening conditions where no standard treatment is available.
Natalie was first hospitalized with severe epilepsy when she was 5 years old.
“I just remember laying in bed and shaking,” Natalie said. “And I kind of felt scared to death because I didnt know what was happening.”
A combination of pharmaceutical drugs helped get things under control, and Natalie had only about one major seizure each year for seven years. But when she turned 12 in fall 2016, as those puberty hormones surged, her seizures came back with a vengeance.
Natalie checked into UCLA that October and didnt leave for 150 days. Nurses made time pass by tacking up posters of her favorite singer, Shawn Mendes, playing his music on repeat, and even getting him to record two video messages that made her swoon.
For two weeks around that Christmas, Natalie was in a coma while doctors tried to use an experimental drug to stop her seizures. Shed taken pharmaceuticals that said they could make her blind and ones that made her depressed to the point of feeling suicidal. But nothing calmed the electrical charges in her brain.
Natalies parents had read about CBD helping other children with epilepsy but had so far stuck with the doctors recommendations. When they heard the next option on the table was to remove a portion of Natalies left frontal lobe where the seizure activity was triggering, they asked Dr. Hussain about giving CBD a try.
Five years ago, before news of Charlotte Figi broke, Hussain said he would have considered the option of giving a child a cannabis-based medicine to treat seizures “lunacy.” And even when they were agonizing over Natalies treatment, he said there still wasnt any good data to indicate Epidiolex would help with the particular form of epilepsy she faced.
“We were getting pretty desperate,” Hussain said. “We got to the point where we didnt have other medical options.”
He and his colleagues plowed through the red tape needed to get approval for the trial drug. And they started giving Natalie her first round of Epidiolex in January 2017.
“We felt like she was a pioneer when we were giving her that first dose,” Catherine said.
Natalie recalls thinking: “This is either going to be helpful or Im going to have to go through surgery.”
By day 12 of taking Epidiolex, a study by Hussain and his colleagues at UCLA says Natalie had gone from as many as 20 seizures each day to no noticeable episodes. On day 21, she got to go home. And on day 64 of taking Epidiolex, in March 2017, she was diagnosed as seizure-free.
Her mom now drives once a month to UCLA to pick up the bottle of Epidiolex, cradling it the entire way for safe-keeping.
“I look at it like liquid gold,” Catherine said.
This summer, Natalie went ziplining at summer camp and on a family cruise to Alaska, where the teen begged her mom to stop quoting the “Titanic” line: “Iceberg, right ahead!” Shes looking forward to joining the drama club at school in the coming year and maybe choir, so she can get even better at singing Shawn Mendes songs.
Hussain said he hates to think about what would have happened to Natalie had Epidiolex not been available.
Going forward, he said Epidiolex and other CBD drugs that may come to market will be near the top of the list of therapies hell consider when he encounters patients with stubborn seizures.
“I dont think it should be viewed as a last resort,” he said.
Because of the lingering stigma, Catherine initially only told her closest friends that they were giving Natalie a drug derived from marijuana. She hopes that sharing Natalies story now will encourage more people to consider trying CBD treatments.
Natalie has only one complaint about her medicine. Despite company claims that the syrup is strawberry flavored, she grimaced as she shook her head of brown curls.
“It tastes bitter. Not good.”
Her moms only complaint?
“I wish it would have been around sooner.”
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