First drug derived from marijuana is about to be approved by the FDA | Colorado Springs News

At the age of 3 months, Marissa Parsons began having frequent seizures, some of which lasted half an hour. For nearly two decades, her desperate parents tried drugs after drugs to treat her rare type of infantile epilepsy, but the drugs produced mostly side effects – pancreatitis, hives and extreme drowsiness – and she was frequently hospitalized. Family outings have become almost impossible. Finally, said her mother, Marissa stopped smiling.

Three years ago, Marissa was enrolled in a clinical trial at Nationwide Children's Hospital in Columbus, Ohio, testing a liquid anti-epileptic drug made from a marijuana component. Marissa, who can not speak and uses a wheelchair, quickly went from eight seizures a day to half that number; these days, the 21-year-old has a few weeks during which she has no crisis. She regularly goes to the baseball games and group concerts of her sisters. In 2016, she and her family visited Mount Rushmore and the Grand Canyon. Plus, she smiles again.

"We can function as a typical family," said his mother, Ronda Parsons. "That, and his smile, mean the world to me."

The drug that helped Marissa, called Epidiolex, is about to become the first drug approved by the FDA and derived from marijuana. In April, an advisory committee unanimously recommended the approval of the drug for two of the most serious types of infantile epilepsy, and the agency should make its decision here. the end of June. The drug is an oral solution containing highly purified cannabidiol, or CBD, which is one of the many chemicals in the cannabis plant. It contains only traces of the psychoactive THC element, and does not induce euphoria.

For those who have long argued that cannabis offers medical benefits, an FDA approval would be an important step, "a recognition that the plant is a rich source of compounds that have potential therapeutic activity," said Justin Gover, director General of GW Pharmaceuticals. London-based company that developed the drug. "We are only scratching the surface of what could be a range of cannabis-based drugs."

The FDA has already licensed drugs containing synthetic versions of THC for nausea in patients receiving chemotherapy and for other uses, but it has not approved any drug derived from the plant itself. Marijuana and its components, including CBD, are controlled substances in Schedule 1 – which means that they are prohibited because they are considered to have high potential for use. abuse, no medical value and serious implications for safety. If approved by the FDA, cannabidiol would likely be reclbadified by the Drug Enforcement Administration within 90 days.

The company is seeking permission to use the drug to treat Lennox-Gastaut and Dravet syndromes, which cause uncontrolled daily seizures and expose patients to a high risk of other physical and mental impairments. intellectuals, injuries and premature deaths. The disorders affect fewer than 45,000 people in the United States, but experts expect Epidiolex to also be prescribed for other types of epilepsy.

The drug, which would continue to be manufactured in Britain, would be marketed by Greenwich Biosciences, the US subsidiary of GW Pharmaceuticals. The drug manufacturer is testing other CBD treatments for glioblastoma and schizophrenia.

Shlomo Shinnar, president of the American Epilepsy Society and neurologist at Montefiore Medical Center in New York City, said the drug would be "a very valuable addition" to limited options for the treatment of severe epilepsy of the disease. 39; childhood. Shinnar said he would welcome the approval of a drug that had undergone a rigorous FDA review for safety and effectiveness.

Many parents already give children unregulated CBD formulations that are available at medical marijuana clinics in many states where such operations are legal.

Heather Jackson, who lives in Colorado Springs, began giving her son Zaki a compound called Charlotte's Web, a type of unregulated CBD, six years ago. His son, who was 12 years old at the time, had uncontrollable seizures since the age of 6 months. Shortly after starting the CBD, she said, the seizures stopped and did not return for four years. Now he has occasional minor seizures, Jackson said. She co-founded Realm of Caring Foundation to provide support services to people using CBD therapy and does not plan to change her son to Epidiolex.

Igor Grant, a psychiatrist at the University of California at San Diego and director of the Center for Medicinal Cannabis Research, said that an FDA approval for Epidiolex could entice companies to investigate CBD at home. Other purposes. There are "clues" that could be useful for treating anxiety, post-traumatic stress disorder and inflammatory diseases such as Crohn's, but "all is that speculation at this point" , he said. Early next year, his center plans to launch a four-year study on CBD and autism.

In many ways, the story of Epidiolex began with Sam Vogelstein, a young man from Berkeley, California, who had made up to a hundred seizures a day. In late 2012, Sam, who was 11 years old at the time, flew with his mother to London to try the GW Pharmaceuticals complex; he was the first person in the United States to take drugs. His seizures have declined sharply, and his remarkable response, along with calls from other patients and doctors, has prompted the company to begin making the unapproved drug available in the US as part of the expanded program. "access" from the FDA. The company then launched clinical trials in several medical centers. These tests showed significant reductions in seizures.

In April, Sam Vogelstein testified at the FDA Advisory Committee meeting in support of the drug. The audience applauded. Now 17, he is taking Epidiolex and an older medicine and has not had seizures for two and a half years. This fall, he will be a junior in high school.

"He wants to be a neurologist," said his father, Fred Vogelstein, a contributor to Wired magazine. "He wants to help others."