Therapeutic Action of 5-alpha Reductase Inhibitors in Tourette Syndrome

Grant Type
Grant Year
Institution Location
Institution Organization Name
University of Cagliari Italy
Investigators Name
Devoto, Paola, PhD

Androgens and other neurosteroids are involved in the pathophysiology of several neuropsychiatric disorders. Cogent evidence indicates that Tourette Syndrome (TS) is significantly more prevalent in males than females. In addition, drugs that inhibit androgen activity have been shown to reduce tic intensity. Capitalizing on these premises, we have focused our research on drugs that inhibit 5-alpha reductase, one of the major proteins involved in the synthesis of neuroactive androgens. These drugs are currently used for the treatment of benign prostate cancers and male-pattern baldness, and have limited side effects. After encouraging findings on animal models of Tourette Syndrome, we discovered that treating adult male TS patients with Finasteride, a drug that inhibits 5-alpha reductase, led to a reduction in tic severity and compulsive symptoms, with few side effects. Unfortunately, drugs that inhibit androgen activity cannot be used in children because they interfere with sexual development. Our longterm goal is to harness some of the critical neurobiological targets addressed by these drugs to develop novel, effective pharmacological strategies that could be used to treat children and adolescents with TS. This TSA funding will enable us to determine how 5-alpha reductase inhibitors function in the brain. Well-validated animal models of Tourette Syndrome will be used to identify the brain regions and neurotransmitter systems directly affected by these drugs. The outcomes of this work may have a significant impact on the identification of critical targets that can be addressed by novel therapies for Tourette Syndrome. Paola Devoto, Ph.D., Marco Bortolato, M.D. University of Cagliari, Monserrato, Italy Award: $68,000 Commentary: Men and woman have different hormones that are primarily responsible for determining sexual characteristics, but which can also influence brain function. It is well known that TS affects more boys than girls. Using this information and working in animals and adults, Drs. Devoto and Bortolato were able to show that medications that inhibit the function of a male hormone can be used to treat Tourette Syndrome. Unfortunately, these medicines cannot be used to treat boys with TS because they would inhibit the normal sexual development of the child. To overcome this problem, Drs. Devoto and Bortolato are looking to see how these medicines work in the brain, in the hope that they will be able to identify other chemical compounds that will act on the brain cells and stop the tics, but will not affect the normal sexual development of the child. Tourette Association of America Inc. – Research Grant Award 2009-2010