Top-line results were reported today for a new study in Tourette syndrome of the dopamine D1 receptor antagonist ecopipam.
This was a study designed to test whether, if someone’s tics improve with 3 months of ecopipam, they continue to have more benefit if you continue it compared to if you switch it for a placebo (a pill with no active ingredient). The study was double-blind, meaning that neither the patients nor the study physicians or local staff knew whether the patients were kept on active drug. Blinding is essential to know whether the drug is truly useful. The study enrolled 167 children or teens and 49 adults at a number of centers in the US, Canada, or the EU (including WashU).
The main pre-planned analysis compared how long it took after the possible switch date for someone’s tics to relapse (“relapse” was defined carefully in advance). About two thirds of patients switched to placebo over the next 3 months, compared to only about two fifths of patients maintained on ecopipam. These results were very unlikely to have occurred by chance (less than one chance in 100). The risk of relapse was twice as great in those switched to placebo. The numbers were roughly the same in the children as in the overall group. As in previously reported studies, side effects were mild and consisted mostly of sleep changes in one sixth of participants, with anxiety, fatigue or headache each occurring in less than one in ten participants.
Overall, this is great news for patients with tics bothersome enough to require more than behavior therapy. Most patients improve substantially with currently available treatments, but many patients have insufficient benefit or problematic side effects. Ecopipam is not currently available, but the company is expected to apply soon to the FDA for the drug to be approved for sale in the U.S.
NOTE to Americans: as you may be aware, this research (at many participating sites) used resources paid for in part by facilities and administrative payments related to NIH grants, and many sites included researchers including young physicians interested in clinical research. Careful scrutiny of new medications, and the speed at which they can be assessed for safety and efficacy by the FDA, require skilled and experienced FDA staff including physicians, pharmacologists, pharmacists, statisticians, and others. All of these resources—F&A costs, support for new researchers, and the FDA—have recently been slashed by the current administration in the U.S. Hundreds of millions of dollars to Missouri research centers, and about 10% of FDA staff, were cut in the past few weeks. In my opinion, these changes hack away at muscle, not fat, in our medical research system. If you have an opinion about these actions, please express that opinion now to your senators and representative.
Opinions are those of the author, not necessarily those of WashU, Emalex Biosciences, the FDA, or the NIH.