Tourette syndrome in children: a promising new molecule

Gilles de la Tourette syndrome, characterized by motor, sound and vocal tics, affects approximately one in 2000 people in France. However, no current treatment can completely cure it. Faced with this therapeutic lack, the discovery of researchers from the Cincinnati Children’s Hospital, published in the journal Pediatrics, looks promising.

A drug with a novel mode of action

In this randomized study conducted at 68 sites (United States, Canada, Germany, France, Poland), 153 patients aged 6 to 18 with severe tics were divided into two groups. The first group had to take a placebo drug, while the other had to consume an “ecopipam” molecule (a selective dopamine type D1 blocker).

Previous research suggests that problems with dopamine, a neurotransmitter in the brain, may be linked to symptoms of Tourette syndrome, and that dopamine D1 receptors play a key role.

Dopamine receptors are found in the central nervous system. When they receive dopamine, they create signals for various mental and physical functions like movement. Different receivers help control different functions. Ecopipam is the first drug to target the D1 receptor instead of the D2 receptor, which is the one targeted by drugs currently on the market.

Fewer severe tics were recorded

The primary endpoint was the 12-week mean change in Yale Global Tic Severity Score total tics score. Global clinical impression of Tourette’s syndrome severity was the secondary endpoint.

Result ?

• According to the researchers, the average reduction in severity of the score of tics proved to be better in the ecopipam group (-10 points against -6 points) compared to that of the other group.
• The improvement in the overall clinical impression of the severity of Tourette’s syndrome was also greater in the ecopipam group.
• Study participants had no excessive weight gain (on the contrary, the study reports less weight gain in treated patients), adverse movements, or metabolic side effects, which are commonly reported with agents antipsychotics commonly used to treat patients with Tourette’s syndrome.

Some side effects to note

On the other hand, inconveniences (headaches, insomnia, fatigue) were more often observed in the treated group.

A limitation of the study is its three-month duration, although this is standard for this type of study. It will be important to know if the improvement in symptoms persists longer and if other side effects (especially cardiovascular) do not occur.

Nevertheless, this new molecule could constitute an interesting therapeutic option in children and adolescents suffering from Tourette’s syndrome, given the few options available until now.