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Experimental Drug Shown to Reduce Tourette’s Symptoms

Brain illustration


17 January 2017. Results from a clinical trial show an experimental drug is effective in reducing the severity of tics in children with Tourette’s syndrome, a neurological disorder. Findings from the trial were released today by Psyadon Pharmaceuticals in Germantown, Maryland, developer of ecopipam, the drug tested.

Tourette’s syndrome is a neurological disorder characterized by motor tics, involuntary movements in the face, head, or other parts of the body as well as vocal tics expressed as sniffs or grunts to loud hoots or recognizable, but often inappropriate words. Tourette Association of America says these spoken-word vocal tics, commonly associated with Tourette syndrome, occur in 10 to 15 percent of cases. The association estimates 1 in 160 children between the ages of 5 and 17 in the U.S. has Tourette’s syndrome.

Ecopipam addresses receptors of dopamine, a neurotransmitter or signaling chemical in the brain that affects a range of motor and cognitive functions, including movements. The company cites evidence showing individuals with Tourette’s syndrome have overactive dopamine systems that contribute to the disorder, with a family of receptors for dopamine, known as D1 receptors playing a key role in the symptoms.

“Ecopipam works,” says Psyadon CEO Richard Chipkin in a company statement, “by blocking the dopamine D1 receptor, which differentiates it from any other drug either on the market or currently in development.”

The intermediate-stage clinical trial tested ecopipam with 40 children, age 7 to 17 having Tourette’s syndrome, exhibiting both motor and vocal tics. The study randomly assigned participants to take ecopipam or a placebo daily, before bedtime for 4 weeks. The main efficacy measure was the Yale Global Tic Severity Scale. a standard rating scale completed by clinicians. The study team also looked for a number of safety measures, including reports of adverse events and psychological effects, such as depression, ADHD, and feelings of suicide.

The results show recipients of ecopipam reported lower scores on the Yale rating scale than their counterparts taking the placebo, both 2 weeks and 4 weeks after the treatments, with the differences large enough to be statistically reliable. The company also says rates of side effects were comparable between the ecopipam and placebo groups, with no statistically reliable changes in clinical lab tests, electrocardiograms, or body weight increases from taking the drug.

Donald Gilbert, director of the Tourette’s Syndrome Clinic at Cincinnati Children’s Hospital Medical Center, who led the research team conducting the trial says, “These results demonstrate the efficacy of D1 antagonism, an entirely new approach that doctors and scientists have speculated about for years. As importantly, the subjects in the study tolerated ecopipam very well, with no weight gain, sedation, or heart problems.”

Psyadon says ecopipam can also treat other movement disorders associated with D1 dopamine receptors, and is recruiting participants for an intermediate- and late-stage clinical trial testing the drug among adults with childhood-onset stuttering.

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