Well-Being Medical Advances

New drug shows promise for autism-linked syndromes

Phase 2 clinical trial results suggest it can reduce frequency of seizures in some child patients.
illustration of pediatrician with a parent and child

Story at a glance

  •  Some autism-linked syndromes are associated with seizures.

  • Researchers are testing a new drug that may help reduce the frequency of convulsive and drop seizures.

  • The experimental drug will need to go through larger clinical trials to test for efficacy.

A new drug candidate shows promise in reducing seizures in children with epilepsy linked to autism, according to a study published in Epilepsia.  

The drug, called soticlestat, has completed phase 2 clinical trials. It must complete phase 3 trials before being approved for use. 

The drug was tested in children who have Dravet syndrome (DS) or Lennox–Gastaut syndrome (LGS), which are considered developmental and epileptic encephalopathies or brain disease. The phase 2 clinical trial was conducted as a randomized controlled trial, with half of the participants receiving a placebo.  

The children in the study were aged 2 to 17 and had three or more convulsive seizures per month with DS and four or more drop seizures per month with LGS. A convulsive seizure is defined as “rhythmic convulsions that last less than 5 minutes,” and a drop seizure, also called an atonic seizure, is defined as a seizure that results in sudden loss of muscle strength. 

Among the 129 participants who completed the trials, median reduction in seizures was about 30 percent. For patients with DS, the median reduction was about 50 percent. And for patients with LGS, the median reduction was about 17 percent. 

It’s still early days for this drug. It will need to go through larger trials with more participants to further show its effectiveness and safety. Experts are hopeful though, since the participants seemed to benefit from it and the drug was well tolerated.  

“This is important because in these patients, new treatments often impose a large burden of adverse effects that can worsen quality of life in spite of a reduction in seizure frequency,” Emilio Perucca, a clinical professor of medicine at Austin Health at the University of Melbourne in Australia who did not participate in the study, told Spectrum

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