(DGIwire) – What is it like to live with Tourette syndrome? A 21-year-old woman with the disorder recently shared her reflections on MTV’s UK website. She described her first encounter with the physical challenges it brings: an incident at age six when she was blinking rapidly and was told by her grandmother that she was too beautiful to be screwing her face up like that. Yet she was trying to offset a bout of tics, something she said she still did years later—whether screwing up her face to stop herself blinking quickly, seizing her limbs up to stop them from flailing, or swallowing hard to inhibit clearing her throat incessantly. All of which, she wrote, usually proves futile.
She described being asked if she was making funny noises to get in character during a drama lesson, and noted how her eyes and throat began to hurt from exhausting attempts to repress her tics. Given the fact there is currently no cure for the disorder, she urged greater inclusion of Tourette syndrome in any discussion of mental health.
Tourette syndrome is a neurological disorder, usually first noticed in childhood, characterized by repetitive, involuntary movements and vocalizations called tics. The National Institute of Neurological Disorders and Stroke (NINDS) estimates that 200,000 Americans have the most severe form and as many as one in 100 exhibit milder and less complex symptoms.
“Those living with Tourette syndrome face a distinct set of challenges that require understanding from friends, family members and coworkers,” says Ascher Shmulewitz, MD, PhD, Chairman of the Board of Directors of Therapix Biosciences. “What all stakeholders should know is that research is ongoing to find new therapies with greater effectiveness than any currently available options.”
One approach to novel therapy, adopted by Therapix Biosciences, involves studying the potential impact on Tourette syndrome from a proprietary combination of two substances called cannabinoids. The first, dronabinol, is a synthetic form of THC, which has been approved as a medical treatment for decades; the second is palmitoylethanolamide (PEA), which naturally occurs in various foods and is listed as a medicinal food ingredient. A series of studies published in the Journal of Clinical Psychiatry and elsewhere have suggested that THC might address Tourette symptoms.
The company has nearly completed its Phase IIa clinical study at Yale University to assess the potential for its investigatory compound THX-TS01, which combines THC and PEA. The company is pursuing the development of THX-TS01 by utilizing a 505(b)(2) regulatory strategy based on prior US Food and Drug Administration (FDA) approval of dronabinol; the company is also seeking Orphan Drug Designation from the FDA. Top-line results from the Phase IIa study are expected in early 2018; Therapix will proceed accordingly from there.
“Hopefully there will one day be an effective treatment to address the symptoms of Tourette syndrome in a swift and significant way,” Dr. Shmulewitz adds.