The North Shore-LIJ Health System’s Movement Disorders Center will be one of nine sites in the United States taking part in a clinical trial of a new drug for Tourette syndrome, the health system announced today.
The drug, AZD5213, targets the histamine H3 receptor. Histamine is commonly associated with allergies and the immune system, but it also plays a role in regulating dopamine and the neurotransmitter is intimately tied to the symptoms of Tourette.
The trial was sparked by geneticists identifying a rare mutation in a gene for histidine decarboxylase in a family with Tourette syndrome – a father and his eight sons.
The mutation, which blocks histamine production, has only been found in that family, but researchers have created transgenic mice with the same mutation and those animals develop Tourette and compulsive-like behaviors. The animal studies showed that the mutations disrupted dopamine modulation and that histamine infusion reduced the dopamine levels.
“That scientists have replicated it in an animal model validates the hypothesis that the pathophysiology may be similar in human patients,” said Dr. Cathy Budman, director of the Movement Disorders Program in Psychiatry, investigator at the Feinstein Institute for Medical Research and expert on Tourette syndrome who will oversee the study site for North Shore-LIJ.
The study will test safety, tolerability, pharmacokinetics and efficacy in adolescent patients, between 12 and 17 years old. In addition to safety, the Yale Global Tic Severity Scale will be used to see whether tics were reduced while on the study drug.
“We are hoping that this new investigational drug will prove to be effective for patients,” said Dr. Budman.
According to the 2007 National Survey of Children’s Health, an estimated three in 1,000 children will develop Tourette syndrome although up to 1/200 may show milder tic symptoms. It is three times as likely in boys as in girls. Most patients experience their worst symptoms during adolescence, but symptoms may persist throughout life. Many patients also suffer from other conditions, including attention deficit-hyperactivity disorder or obsessive-compulsive disorder. Anxiety, depression and anger control problems can also complicate the disease.For more information on the clinical trial, call study coordinator Arif Hafeez at (516) 562-3224.
