Marijuana-Based Epilepsy Drug is Close to FDA Approval

The FDA has taken a significant step toward approving a marijuana-based drug to treat severe epilepsy in children, a treatment that sufferers say could be life-changing.

A 13-member panel of federal advisers voted  unanimously last week to back a drug known as Epidiolex. The drug has shown some potential risks, such as  liver damage, but it has also displayed a marked ability to control seizures in children with rarer and more severe forms of epilepsy.

Approval by the wider FDA is still pending, and the agency will make that decision by late June. The unanimous backing of federal advisers has earned praise, and the prospect of the first plant-derived prescription cannabidiol medicine in the US market is an exciting one.

“Epidiolex represents hope for the many individuals living with intractable seizures and rare epilepsies, who every day face incredible challenges and disabling seizures, and live with the continual risk of serious injury and death,” Philip Gattone, president and CEO of the Epilepsy Foundation, told NBC News.

Epidolex could be a breakthrough for epilepsy treatment among children with early childhood epilepsy that comes with conditions like Lennox-Gastaut syndrome  or the rare genetic anomaly that causes Dravet syndrome.

The drug is what’s known as cannabidiol, or CBD, one of  more than 113 cannabinoids that can be derived from cannabis. Unlike some of its counterparts, it does not contain the properties that allow people to get high.

Several states have approved marijuana for medical purposes, but the FDA has not yet approved it.

CBD oil, of course, is popular on the internet with claims that it can cure or treat a whole range of health problems, even cancers. Unlike Epidiolex and other treatments currently making their way through testing, these assertions are not backed by scientific studies.

Health watchdogs are keen to point out that when buying these oils over the internet, you have no guarantee that what you are getting is even what it claims to be, let alone that it is safe or that it works.

Nevertheless, some people feel they have been forced into trying cannabis oil as a treatment because of the way in which the federal government has stood in the way of research despite clear indications that marijuana-derived treatments are medically valuable.

The FDA has indicated that it will approve the drug, but that approval will be limited to treating Lennox-Gastaut syndrome and Dravet syndrome. Still, the drug would be open to so called “off-label” prescriptions, where doctors could prescribe it to patients if they felt there were a reasonable health benefit.

To give a clue of just how potentially life-changing this kind of treatment might be for young epilepsy sufferers, it’s worth looking at statements from patients who are involved in the research trials.

“I had seizures for 10 years,” 16-year-old Sam Vogelstein, who was started taking Epidiolex five years ago during the first clinical trials, said at the FDA public hearing. ”My parents tell me there were times I had seizures 100 times a day.”

He said that the drug changed his life and that he hasn’t experienced a seizure in over two years. Vogelstein said he was not paid by the company to speak Thursday.

Not everyone will have such a positive experience with Epidolex. For example there is concern that the side effects may worsen due to patients being on a combination of therapies, something that is not unusual with epilepsy treatments.

The drug will not be right for everyone, but the makers say that this is the first of its kind treatment and that it can improve some sufferers’ lives, an option that was not there before.

Speaking in broader terms, this illustrates just how crucial research surrounding marijuana and its derivatives is. We don’t yet know why, exactly, the drug works to help patients with severe epilepsy, only that it does.

Marijuana-derived products have shown some promise–though, perhaps, not as much as some have claimed–to treat other conditions and may be particularly helpful in the mental health sphere to treat problems like PTSD.

However, federal restrictions on marijuana make research into its properties and applications difficult. The federal government tightly controls the supply of medical grade plants. This has to change if we are to unlock marijuana’s potential for medical treatments.

The War on Drugs has always been wrong-headed, but it is stories like this that demonstrate the often hidden ways in which that war is also harming the general public.

Related at Care2

Photo credit: Thinkstock.

35 comments

Marie W
Marie W5 months ago

Thank you

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Ruth S
Ruth S8 months ago

Medical marijuana is OK, recreational is NOT!

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bob Petermann
bob Petermann8 months ago

Get this done as soon as possible. Thanks

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Chrissie R
Chrissie R9 months ago

Thank you for posting

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Cindy S
Cindy Smith9 months ago

thanks

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Tania N
Tania N10 months ago

Thanks for sharing.

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Tania N
Tania N10 months ago

Thanks for sharing.

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Tania N
Tania N10 months ago

Thanks for sharing.

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Winn A
Winn Adams10 months ago

It can't come soon enough

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Lenore K
Lenore K10 months ago

good, speed it up

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