By Staff Reports
(DGIwire)–Tourette syndrome (TS) 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 of TS and as many as one in 100 exhibit milder and less complex symptoms. For anyone who suspects that a family member might have TS, here are three questions to ask:
- What are the symptoms of TS? Tics are classified as simple or complex. Simple tics can include eye blinking and other eye movements, facial grimacing, shoulder shrugging and head or shoulder jerking, reports NINDS; simple vocalizations can include throat-clearing, sniffing or grunting. Complex tics can combine facial or body movements and sometimes involve inappropriate behavior or self-harm.
- How does TS progress over time? Early symptoms manifest themselves on average between the ages of three and nine, according to NINDS. Initial symptoms usually occur in the head and neck area and may progress to the trunk and extremities. Motor tics generally precede the development of vocal tics and simple tics often precede complex tics.
- What research is being conducted in TS? One approach, adopted by Therapix Biosciences, involves studying the potential impact on TS from a proprietary combination of two substances called cannibinoids. 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 TS symptoms.
“We have nearly completed our Phase IIa clinical study at Yale University to assess the potential for our investigatory compound THX-TS01, which combines THC and PEA,” says Ascher Shmulewitz, MD, PhD, Chairman of the Board of Directors of Therapix. “The goal is to create a therapy that offers benefits currently lacking in available treatments.”
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 of THX-TS01 at Yale University are expected in early 2018; Therapix will proceed accordingly from there.
“Our goal is to one day provide a therapeutic treatment that may give TS patients and their doctors an optimal treatment paradigm for the debilitating, unmet medical need,” Dr. Shmulewitz adds.