Tourette Syndrome Trial Will not Proceed due to Efficacy Issues

A clinical trial of Lu AG06466 for the treatment of Tourette syndrome ended in failure recently, as it did not meet its primary endpoints in comparison to a placebo. While researchers are disappointed in this outcome, they are hopeful that it will be successful as a therapy for other neurological and psychiatric disorders.

About Tourette Syndrome

Tourette syndrome is a nervous system disorder that is characterized by tics, which are involuntary movements or sounds. About 100,000 to 300,000 people are affected by this disorder in the United States.

Symptoms of Tourette syndrome vary greatly between individuals, as everybody experiences different tics. These tics can be brought on by a variety of triggers, such as stress, fatigue, illness, or excitement. Symptoms of this syndrome include:

  • Jerking of the arms and head
  • Repeated eye blinking
  • Nose twitching
  • Bending or twisting
  • Shrugging
  • Obscene gestures or words
  • Grunting or barking
  • Coughing and clearing the throat
  • Repeating words
  • Attention deficit disorder
  • Obsessive compulsive disorder

The exact cause of this condition is unknown, although it is suspected to be a combination of inherited and environmental factors. Others believe that chemical imbalances within the brain result in Tourette syndrome.

No cure exists for Tourette syndrome. Treatment consists of medications that block dopamine, Botox injections, seizure medications, anti-depressants, and behavioral therapy. If one is affected by an extremely severe case, doctors may use a device that sends electrical impulses to the brain.

The Study

In the placebo controlled, double blind, and randomized trial 48 participants took either a placebo or Lu AG06466. Results were taken at 28 and 56 days. Unfortunately, this treatment did not meet the primary endpoint on the Yale Global Tic Severity Scale (YGTSS-TTS); there was no significant difference between the placcebo and Lu AG06466. While these results were disappointing, the drug showed minimal adverse effects in less than 10% of participants, making it suitable to be indicated in the treatment of other disorders.

Lu AG06466 and its Future

Lu AG06466 inhibits the monoacylglycerol lipase (MAGL), and also selectively modulates the endocannabinoid system. Its purpose is to lower the neuroinflammation and neurotransmission that are major characteristics of a myriad of psychiatric and neurological disorders.

Researchers are going to continue to investigate the use of this drug for the treatment of other brain disorders. They will evaluate Lu AG06466 at the Lundbeck La Jolla Research Center as a single compound and in combination with other compounds. The hope is that it will be a successful treatment for a different condition.

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