North Shore-LIJ Doctors Testing New Tourette Syndrome Drug

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 (Hdc) 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 Cathy Budman, MD, 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 (NSCH), 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 (ADHD) or obsessive-compulsive disorder (OCD). Anxiety, depression and anger control problems can also complicate the disease.

The brain chemistry changes that have been identified in Tourette open the door to the development of effective treatments. AZD5213 may be able to counteract these changes, with the potential to provide symptomatic relief with less of the negative side effects associated with existing treatments, said Dr. Budman. 
For more information on the clinical trial, call study coordinator Arif Hafeez at 516-562-3224.

About North Shore-LIJ
One of the nation's largest health systems, North Shore-LIJ delivers world-class clinical care throughout the New York metropolitan area, pioneering research at The Feinstein Institute for Medical Research and a visionary approach to medical education highlighted by the Hofstra North Shore-LIJ School of Medicine. North Shore-LIJ cares for people at every stage of life at 16 hospitals and nearly 400 outpatient physician practices throughout the region. North Shore-LIJ’s owned hospitals and long-term care facilities house more than 6,000 beds, employ more than 10,000 nurses and have affiliations with more than 9,400 physicians. With a workforce of more than 47,000, North Shore-LIJ is the largest  private employer in New York State.  For more information, go to

About The Feinstein Institute for Medical Research
Headquartered in Manhasset, NY, The Feinstein Institute for Medical Research is home to international scientific leaders in many areas including Parkinson’s disease, Alzheimer’s disease, psychiatric disorders, rheumatoid arthritis, lupus, sepsis, human genetics, pulmonary hypertension, leukemia, neuroimmunology, and medicinal chemistry. The Feinstein Institute, part of the North Shore-LIJ Health System, ranks in the top 6th percentile of all National Institutes of Health grants awarded to research centers. For more information, visit


Topics: News

Focus onHealth TV

Watch Focus onHealth, Northwell Health's TV show. It's the healthy way to stay informed!