An experimental drug shows promise in reducing tics in young people with Tourette syndrome.
Ecopipam, which failed as a weight loss medication, may reduce tics by 30% in kids and teens with Tourette without the unpleasant side effects of current treatments, researchers say.
“This drug significantly reduced tics, compared to placebo, and did not have side effects associated with other approved medications that cause weight gain and neuropsychiatric side effects like mood changes and unwanted movements,” said lead researcher Dr. Donald Gilbert, a pediatric movement disorders and Tourette syndrome specialist at Cincinnati Children’s Hospital.
Tourette syndrome is a neurological disorder that causes motor and verbal tics. These are repetitive movements and vocalizations such as blinking, shrugging, clearing the throat or yelling out a word or phrase. The urge to produce these movements or sounds is difficult to control.
Other studies suggest that problems with dopamine, a neurotransmitter in the brain, is linked to Tourette, and that D1 dopamine receptors are key.
“There are no drugs on the market for anything that bind to and block that receptor,” Gilbert said. “[Ecopipam is] a medication that, if approved, would be a completely different way of treating tics and other brain problems.”
Current drug treatments for Tourette include Haldol, Risperdal and Xenazine, which are also used to treat mental disorders, such as schizophrenia. They can cause significant weight gain and depression.
Ecopipam will probably never go head-to-head with these other drugs, Gilbert said. “But if I have a choice, having over 20 years of clinical experience with the drugs that are on the market, I would prefer to use this one [ecopipam],” he added.
Gilbert noted that because of the side effects from current Tourette drugs, parents are often reluctant to have their children take them.
The new study was funded by Emalex Biosciences, LLC, maker of ecopipam.
Gilbert’s team randomly selected 149 youths, ages 6 to 17, with Tourette to receive ecopipam or a placebo for three months.
The researchers used two scales to gauge progress — on both, higher scores represented more severe symptoms and negative effects on everyday life.
After three months, those taking ecopipam had fewer and less severe tics than the placebo group, the researchers found. On average, ecopipam lowered motor and vocal tic severity from 35 to 24 on a 50-point scale, a decrease of 30%.
Participants taking placebos improved from an average tic severity score of 35 to 28, a 19% decrease, according to the study.
On a separate 100-point scale of tic symptoms and severity, there were similar improvements.
Ecopipam reduced severity scores from an average of 68 to 46, a decrease of 32%. The placebo group saw their average score drop from 66 to 54, a decrease of 20%.
In terms of side effects, 34% of the ecopipam group had headaches and fatigue, as did 21% of those taking placebos, Gilbert noted.
He hopes that the drug will eventually be approved by the U.S. Food and Drug Administration. Ecopipam is a pill taken once a day. The dose, as with most childhood medications, is based on a patient’s weight.
Gilbert explained that for many people with Tourette, the symptoms lessen over time, and most patients won’t need medication as adults.
“There may be a medicine coming that has a different way of acting in the brain that helps tics without causing weight gain and neuropsychiatric side effects,” Gilbert said.
Dr. Ivan Pavkovic, a pediatric neurologist at Cohen Children’s Medical Center in New Hyde Park, N.Y., said that the preferred treatment for Tourette now is cognitive behavioral therapy, which teaches kids about what triggers their tics and how to control them.
This therapy is as effective as medication, but it’s not available to most children, he added.
“It’s very difficult to access that therapy,” said Pavkovic, who was not part of the study. “When I give people a list of providers, they’re booked out for months and months and months and many of them don’t accept insurance.”
Cognitive behavioral therapy also requires a big commitment on the part of both the child and parents, he said. “In many ways, it’s more difficult than taking a pill,” Pavkovic said.
“The exciting thing about this study, as somebody who treats tics, is that people are working on this and coming up with new medications that have fewer side effects,” he said. “That that’s a good thing, any way you look at it.”
The findings are slated for presentation next Tuesday at a meeting of the American Academy of Neurology, in Seattle. Findings presented at medical meetings are considered preliminary until published in a peer-reviewed journal.
The U.S. Centers for Disease Control and Prevention has more about Tourette syndrome.
SOURCES: Donald Gilbert, MD, MS, pediatric movement disorders and Tourette Syndrome specialist, Cincinnati Children’s Hospital; Ivan Pavkovic, MD, pediatric neurologist, Cohen Children’s Medical Center, New Hyde Park, N.Y.; presentation, April 5, 2022, American Academy of Neurology meeting, Seattle