The US national Tourette Syndrome Association (TSA) has entered into its first-ever clinical research collaboration with a pharmaceutical company
The US national Tourette Syndrome Association (TSA) has entered into its first-ever clinical research collaboration with a pharmaceutical company. The 39-year-old not-for-profit organisation is partnering with Maryland-based Psyadon Pharmaceuticals on a clinical trial of Psyadon's drug ecopipam for managing the symptoms of tourette syndrome (TS), an inherited neurological disorder characterised by involuntary movements and sounds.
The phase IIa open-label trial is testing the ability of ecopipam to reduce tic severity as well as its safety in people 18 to 65 years of age with TS. The study is being conducted at four US sites. Psyadon and TSA expect to release data from the trial by mid-2012.
In addition to providing funding and guidance for the study, TSA will help with subject recruitment for the trial, making use of the 33 chapters and more than 150 support groups in its national network.
The joint effort "marks the beginning of a new phase in our efforts to facilitate the development of effective and safe medications that are urgently needed for people with TS," said Judit Ungar, president of the NY-based group. "The TSA-Psyadon collaboration sets the precedent for our future partnerships with the pharmaceutical industry."
The antipsychotics Orap (pimozide) and Haldol (haloperidol) are already approved by the US Food and Drug Administration (FDA) for TS, and a number of drugs are also used off-label to treat the condition. However, the currently available therapies often provide only mild to moderate benefit and have challenging side effect profiles, according to TSA.
Ecopipam, which was granted a US orphan-drug designation for the treatment of TS last year, is a synthetic benzazepine derivative that acts as a dopamine D1 antagonist and has been shown in animal studies to interact with nerve cells and systems in the brain thought to be linked to TS. "Neither ecopipam nor any other drug with a similar mechanism has been tested in TS. Therefore, the results of this study will be immensely valuable," said Dr Kevin McNaught, vice president for TSA medical and scientific programmes.
Psyadon Pharmaceuticals was founded in 2008 to focus on developing drugs for the treatment of central nervous system diseases.