New Drug Cuts Tourette Tics in Half Without Side Effects

Children struggling with Tourette syndrome may soon have access to the first medication designed specifically for their condition, one that significantly reduces tics without causing weight gain or other debilitating side effects.

A Phase III clinical trial led by Cincinnati Children's Hospital has shown that ecopipam cuts the risk of tic relapse in half for children with Tourette syndrome. The study, published in JAMA Neurology on May 26, 2026, tracked 216 participants across 77 sites.

Tourette syndrome affects about 1% of school-aged children, causing uncontrollable motor and vocal tics like blinking, shrugging, or grunting. The condition typically appears before age 10 and is more common in boys.

Until now, families faced a difficult choice. The only FDA-approved medications for tics were originally designed to treat schizophrenia, and they come with serious side effects including weight gain, shakiness, stiffness, and even Parkinson-like symptoms. In rare cases, long-term use can trigger another movement disorder that causes people to repeatedly protrude their tongues.

Dr. Donald Gilbert, the principal investigator and movement disorders expert at Cincinnati Children's, has been studying ecopipam for years. His team discovered that the drug works differently in the brain, targeting D1 dopamine receptors instead of D2 receptors.

This seemingly small change makes a huge difference. In the trial, 104 participants received either ecopipam or a placebo for 12 weeks after an initial treatment period. Those taking ecopipam maintained their improvement and showed 50% less risk of relapse.

The side effects were generally mild to moderate. Most importantly, patients didn't gain weight or develop other movement disorders that plagued earlier treatments.

Why This Inspires

This breakthrough means pediatricians may feel more comfortable prescribing treatment, and families may be more willing to try medication. For years, many parents avoided drug treatment because the side effects seemed worse than the condition itself.

The trial is complete, but participants can continue taking the drug while drugmaker Emalex Biosciences seeks FDA approval. Some patients outside the study are already receiving ecopipam through an Expanded Access Program for those who haven't found relief with other treatments.

The medication has received Orphan Drug and Fast Track designation from the FDA, which could speed up the approval process. If approved, it would mark the first time in history that children with Tourette syndrome have a medication designed specifically for them.

For families who have watched their children struggle with uncontrollable tics while fearing the harsh side effects of available treatments, this drug represents genuine hope wrapped in solid science.