Rare Disease Report

Drug Derived From Cannabis to Begin Trials in Children with Dravet and Lennox-Gastaut Syndromes

APRIL 08, 2014
RDR Staff

Cannabis rids children of seizures!  Makes for a great headline, doesn’t it?  It also makes for a good TED talk and a CNN special.  But is there any peer reviewed, randomized, placebo-controlled clinical evidence supporting the use of cannabis to treat children with seizures, such as children with Davet syndrome or Lennox-Gastaut syndrome?

To date, the data is limited and anecdotal. But that will soon change.  And it will change using a drug in which the psychoactive part of cannabis – THC – has been removed.  GW Pharmaceuticals recently obtained orphan drug designation for Epidiolex®, an oral liquid formulation that contains plant-derived cannabidiol for the treatment of children with Dravet syndrome or Lennox-Gastaut syndrome – two rare conditions in which children are besieged with epileptic seizures at a very young age. 

Cannabidiol is a non-psychoactive molecule from the cannabis plant.

Although there are anecdotal accounts of cannabidoil helping children with seizures, the only way to get the drug approved is through examination of its safety and efficacy in well designed clinical trials approved by the FDA. And that is just what GW Pharmaceuticals is doing.  At an investor meeting in March, GW Pharmaceuticals noted their phase 2 trial will begin the second half of 2014 with the intention of starting two pivotal phase 3 trials in 2015 for both Dravet and Lennox-Gastaut syndromes.

In addition, the FDA has given permission for Epidiolex to be given to 125 patients with pediatric epilepsy syndromes who have failed to respond to more conventional anticonvulsant therapy. The company hopes to present some of that data in the near future.

We look forward to reporting on that data when in becomes available.

Dravet Syndrome

Dravet syndrome or severe myoclonic epilepsy of infancy (SMEI), is a severe form of epilepsy.  It appears during the first year of life with frequent febrile seizures.  Later, other types of seizures typically arise, including myoclonus (involuntary muscle spasms).  Status epilepticus – a state of continuous seizure requiring emergency medical care – also may occur.  Children with Dravet syndrome typically experience poor development of language and motor skills, hyperactivity, and difficulty relating to others. 

Anticonvulsants may reduce seizures in Dravet syndrome. A ketogenic diet (high fat, low carbohydrates) may be beneficial in some patients. At present, no orphan drugs are approved for Dravet syndrome.

For more information about Dravet syndrome, visit http://www.dravetfoundation.org/

Lennox-Gastaut syndrome

Lennox-Gastaut syndrome is a severe form of epilepsy. Seizures usually begin before 4 years of age. Seizure typesinclude tonic (stiffening of the body, upward deviation of the eyes, dilation of the pupils, and altered respiratory patterns), atonic (brief loss of muscle tone and consciousness, causing abrupt falls), atypical absence (staring spells), and myoclonic (sudden muscle jerks).

Treatment for Lennox-Gastaut syndrome usually involves a plethora of anticonvulsant medications.  Medications with orphan drug approval for Lennox-Gastaut syndrome include clobazam, lamotrigine, felbamate, rufinamide, and topiramate.

For more information about Lennox-Gastaut syndrome, visit http://www.lgsfoundation.org/

image of marijuana plant courtesy of wikimedia commons.

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