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New Approach Being Tested for People Who Suffer from Tourette’s Disorder

January 27, 2014

Summit, NJ – Medical researchers are hopeful that a new investigational drug being tested in clinical trials will prove to be an effective treatment for Tourette’s disorder (also known as Tourette syndrome), a neurological disorder that arises in childhood and causes involuntary motor and vocal tics.  The new drug, AZD5213, targets the human histamine H3 receptor.  In the brain, this receptor regulates neurotransmitters associated with Tourette’s disorder, including dopamine and histamine.  A mutation that affects histamine synthesis was recently confirmed to be the cause of Tourette’s in a father and all eight of his children, a finding supported by research in mice. If approved for treatment of Tourette's disorder, AZD5213 may represent an alternative to antipsychotics, which don’t work well in all patients and can cause serious negative side effects.  Discovered by AstraZeneca, AZD5213 has been shown to have a favorable safety profile, with no serious drug-related side effects reported in Phase 1 and Phase 2 clinical trials completed at the time of this report.

”AZD5213 represents an exciting new approach to the treatment of Tourette’s disorder, and we are currently recruiting teenagers suffering from the disorder to participate in the clinical trial,” said Roger Kurlan, MD, Director, Movement Disorders Program at Atlantic Neuroscience Institute in Summit, NJ.  Dr. Kurlan is a leading expert in the treatment of Tourette’s disorder and is one of approximately six medical researchers involved in the clinical trial.

According to the 2007 National Survey of Children’s Health (NSCH), an estimated 3 in 1000 American children can be expected to develop Tourette's. The disorder is three times as likely in boys as in girls, and twice as likely for whites as for blacks.  Most patients experience their worst symptoms in their early teen years. Symptoms may persist throughout life, although most patients see improvement as they approach and enter adulthood.

Exaggerated portrayals of Tourette’s disorder have been used for comic relief in films and TV shows, usually featuring characters who shout obscenities involuntarily.  In reality, this type of verbal tic is present in only 10 to 15 percent of Tourette’s sufferers.  “In real life, Tourette’s disorder is no laughing matter,” said Dr. Kurlan.  “These patients often struggle with self-esteem and socialization issues, which generally arise at a crucial time in their social development.  Sadly, many are victims of bullying and the ridicule of their peers.”Tourette’s is most often present in combination with other disorders, such as attention deficit-hyperactivity disorder (ADHD) or obsessive-compulsive disorder (OCD), and Tourette’s patients are commonly troubled by depression, anxiety, and developmental delays.  However, many patients with Tourette’s are able to overcome their handicaps and create successful and rewarding lives.  Comic Dan Aykroyd recently let it be known that he suffered from Tourette’s syndrome and Asperger’s, and film producer Howard Hughes was known to suffer from Tourette’s and OCD.  Soccer star Tim Howard, goalie for Manchester United, NASCAR driver Steve Wallace, TV personality Howie Mandel, and basketball player Mahmoud Abdul-Rauf have also struggled with Tourette’s disorder, as did such well-known historical figures as Dr. Samuel Johnson and Wolfgang Amadeus Mozart.

About AZD5213 and the Clinical Trial

AZD5213 is a brain-permeable, selective inverse agonist of the human histamine H3 receptor that has already been studied in clinical trials in other indications, including Alzheimer’s disease.  Tourette’s disorder is associated with changes in brain chemistry that appear to cause the characteristic symptoms of the disorder.  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.  More than 200 human subjects have already received single or multiple doses of AZD5213, with no serious drug-related adverse side effects.

The clinical trial is a 6-month, multicenter, randomized, safety, tolerability, pharmacokinetic, and preliminary efficacy study of AZD5213 in adolescents 12 to 17 years of age with Tourette’s disorder.  The trial includes an up to 21-day screening period, 6 months of once-daily treatment with placebo or AZD5213, and a 3-week follow-up period.  Because of the crossover design of the trial, all enrolled patients will receive AZD5213 for at least part of the trial.  Approximately 24 subjects will be treated in this study.  Safety will be carefully ensured by a battery of safety tests administered at visits throughout the study, and efficacy will be determined by means of questionnaires designed to assess the severity of symptoms associated with Tourette’s disorder.

About Tourette’s Disorder

The symptoms of Tourette’s disorder are typically noticed first in childhood, with the average onset between the ages of 3 and 9 years.  The disorder occurs in people from all ethnic and racial groups, although it is twice as common in whites as in blacks, and males are affected about three times as often as females.  It is estimated that 200,000 Americans have the most severe form of Tourette’s, and as many as one in 100 exhibit milder and less complex symptoms, such as chronic motor or vocal tics.  Although Tourette’s disorder can be a chronic condition, with symptoms lasting a lifetime, most people with the condition experience their worst tic symptoms in their early teens, with improvement occurring in the late teens and continuing into adulthood.

To find out about patient services and support, contact the National Tourette's Syndrome Association.

Read more about the disorder on the National Institute of Neurological Disorders and Stroke (NINDS) Tourette Syndrome Information Page.

For more information on participating in this trial at Atlantic Neuroscience Institute at Overlook Medical Center, please call 908-522-5901 or email.