DEC 20, 2019 6:52 PM PST

Drug Repurposing May Provide Hope for Deadly Childhood Seizure Disorder

WRITTEN BY: Nouran Amin

The life-threatening and treatment-resistant seizure disorder among children known as the ‘Dravet Syndrome’ may soon have new safe and effective therapeutic. The treatment is actually an old drug previously used for obesity in adults and claimed ‘risky’ due to heart failure side-effects. The drug is called fenfluramine and was shown to reduce the occurrence of seizures by more than half among Dravet syndrome patients.

Dravet syndrome develops during infancy and is described as prolonged and frequent seizure episodes that cause developmental delays and speech impairments. About 10 to 15 percent of Dravet patients pass away by age 25 due to seizure related heart problems and suffocation.

"If children with Dravet syndrome can be diagnosed and effectively treated when they are young, they may be spared further neurodevelopmental delays caused by repeated seizures," said co-first author Joseph Sullivan, MD, professor of neurology and pediatrics at UCSF Benioff Children's Hospitals, and director of the Pediatric Epilepsy Center of Exellence. "The results of this study may have significant quality-of-life implications for patients with Dravet syndrome and their families."

Learn more about what it’s like to live with Dravet syndrome:

 

In the study, participants who averaged 40 convulsive seizures in one month before the clinical trial were randomly assigned one of the three groups: 0.7mg/kg/day of fenfluramine, with a maximum daily dose of 26 mg per day; a lower dose of 0.2 mg/kg/day; or a placebo. Results showed that study participants that received the higher of the two study doses had 62 percent fewer seizure episodes per month than those on the placebo, and those patients who were on the lower dose had 32 percent less convulsive seizures per month than the placebo participants.

Findings of the study were published in The Lancet.

Despite great results, researchers acknowledge why fenfluramine had been taken down from the market by the U.S. Food and Drug Administration in 1997 and that is because of its risk of causing damage to the heart valves.

"This was a concern, but we knew the drug used in this study had undergone tests to enable pediatric use and that patients would be followed closely as part of the study," said Sullivan, who was the principal investigator in the North American study sites. "At the start of the study, when I asked parents what they would do if their child developed heart valve abnormalities, yet had significantly fewer seizures, they said they were willing to accept some risk. They knew that every seizures comes with the risk of sudden unexpected death."

In addition, children were excluded from the trial if they had any heart problems. Although no cases of heart valve damage or pulmonary hypertension was noted, there were some unpleasant side-effects among those that took high doses such as diarrhea, lethargy, and sleepiness. Overall, researchers found out that cases of Dravet patients in Belgium who were clinically on fenfluramine were successful. "Some of these patients have been taking fenfluramine for up to 30 years, with sustained, clinically meaningful reductions in seizure activity without evidence of cardiopulmonary disease," says Sullivan.

Source: University of California-San Francisco

About the Author
  • Nouran earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
AUG 25, 2020
Cannabis Sciences
FDA Approves Cannabis Drug to Treat Rare Genetic Disease
AUG 25, 2020
FDA Approves Cannabis Drug to Treat Rare Genetic Disease
The US Food and Drug Administration has approved Epidiolex, a cannabidiol (CBD) oral solution, to treat seizures linked ...
SEP 11, 2020
Immunology
Could a Drug for Cats Treat COVID?
SEP 11, 2020
Could a Drug for Cats Treat COVID?
Canadian researchers have discovered that a drug used to treat coronavirus infections in cats shows promise in helping C ...
SEP 13, 2020
Drug Discovery & Development
Scientists Find Way to Reduce Inflammation from Vaccines
SEP 13, 2020
Scientists Find Way to Reduce Inflammation from Vaccines
Researchers from the University of Chicago have found a new way to reduce vaccine-related inflammation from adjuvants, a ...
SEP 19, 2020
Drug Discovery & Development
Novel Drug Delivery System for Neurological Diseases
SEP 19, 2020
Novel Drug Delivery System for Neurological Diseases
Scientists at Rutgers have developed a smart drug delivery system that targets inflammation in damaged nerves and helps ...
OCT 01, 2020
Cancer
Understanding in vivo Metabolomics: C13 Isotope Studies
OCT 01, 2020
Understanding in vivo Metabolomics: C13 Isotope Studies
One key to understanding cancer metabolomics lies in the ability to accurately replicate the natural environment of the ...
OCT 15, 2020
Neuroscience
Neurometabolism: A bold, new frontier
OCT 15, 2020
Neurometabolism: A bold, new frontier
Billions of neurons communicate with each other through trillions of synapses to create a functioning, unique system we ...
Loading Comments...