On June 25th 2018 the U.S Food and Drug Administration approved the first drug derived from marijuana. The drug Epidiolex will be used for the treatment of two rare but severe forms of seizures. The drug will be used for those who suffer from Lennox-Gastaut syndrome and Dravet syndrome. The medicine has been approved for use of patients 2 years of age and older.
Although there are a few side effects of Epidiolex such as sleepiness, decreased appetite and weakness to name a few the benefits seem to outweigh the small amount of side effects.
The breakthrough to Epidiolex becoming FDA approved started with one family’s fight. Sam Vogelstein started suffering from seizures at the age of 4 years old. Within a short period of time Sam was suffering 100 seizures a day. After reading an article about a seizure study on rats using CBD oil, Sam’s mother knew he needed access to the medicine.
CBD was not easy to get however back in 2011. Then she learned of a British pharmaceutical company called GW Pharmaceutical. They were making medicine derived from CBD for Multiple Sclerosis patients.
Sam’s parents sent emails and called the company for months before the company decided that with a doctor’s supervision they would allow Sam to try the drug for two weeks. With Sam suffering no side effects the medicine showed promise almost immediately. With Sam only having 30 seizures in a day, down from nearly 100 a day. After just 3 days Sam only suffered one seizure per day.
After seeing the results this medicine had on Sam, his parents knew they needed to be able to keep using it outside of London and back at home. They talked with a neurologist at the University of California named Roberta Cilio who along with Sam’s parents petitioned the FDA to allow Sam to continue the drugs usage under the compassionate use program.
After four months the FDA allowed Sam to enroll in his own one-person trial at UCSF. Sam was the first patient in the world to receive Epidiolex as a treatment.
CBD is still classified as a Schedule 1 substance. Now that the FDA has approved Epidiolex the DEA will need to reclassify CBD and is expected to do so in the fall of 2018.
Although Epidiolex was technically limited to patients with two types of seizures, doctors will be able to prescribe it for other uses. The DEA must formally reclassify CBD into a different category before sales can begin of Epidiolex.
Other studies have shown promise with one study showing 33% of patients participating with Dravet syndrome being seizure free after a 12-week trial.
Another study showed that after treatment 15% of patients were seizure free. 50% of the group experienced on average reduction in seizure frequency. The patients tested had seizures that were highly resistant to normal treatment.
In another case, prior to the study with Epidiolex Patient A’s daughter usually had 50-100 seizures a day. After starting use with Epidiolex her daughter is down to 2-5 seizures a week. In addition to improved seizure control her daughter can concentrate on her surroundings and she is more alert.
In September of 2018 the DEA reclassified Epidiolex to a schedule 5 status. Which is the lowest restriction classification for controlled substances. The DEA decided to limit the rescheduling of CBD to a specific formulation of an FDA approved drug product.
CBD remains classified as a schedule 1 substance with exception of approved drugs. Schedule 5 drugs are considered to have the lowest potential for abuse.
The average cost a year for Epidiolex for treatment is $32,500. However, insurance will cover most of the cost for most patients. GW Pharmaceuticals also has set up a program to help patients further.
Epidiolex is made of cannabidiol and is a non-psychoactive drug and it was approved by the FDA because of the results of three clinical trials that involved 516 patients. The success of those patients have paved the way for this drug to help so many other patients who will now be able to live a life that would not have been possible without this drugs approval.
Other drugs with cannabis compounds are on the market already. Such as a drug for nausea and vomiting called Dronabinol (Marinol) however it is made with a synthetic form of THC. Epidiolex is the first drug made directly from the cannabis plant, rather than being created synthetically it is grown in the U.K. at a facility. With help from the U.S Department of Health and Human services he DEA was able to make a final scheduling decision for Epidiolex.
The breakthrough of Epidiolex being approved is a reminder that when drugs are evaluated properly that ingredients including marijuana can lead to important medical therapies.
At the same time the FDA said it is prepared to take action on illegal marketing of products containing CBD that have unproven medical claims. Products with uncertain dosages and formulations can keep patients from getting proper care and recognized therapies that could be helping to treat them of serious illness and fatal diseases that need proper dosages.
With all the new breakthroughs coming about with cannabis being used in the medical industry, patients have new hope that new breakthroughs for many different conditions may be on the way soon leading the way to a new way of life for people who suffer serious illnesses daily.
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Published on Apr 19, 2018
Courtesy of NBC News