HIV Drug Shows Potential To Combat Genetic Form of Dystonia in Preclinical Study (UF Health Newsroom)

HIV Drug Shows Potential To Combat Genetic Form of Dystonia in Preclinical Study

A new study found that an FDA-approved medication to treat HIV restored multiple brain abnormalities in a mouse model of a genetic form of dystonia.

Dystonia is a neurological movement disorder that causes muscles to contract involuntarily, leading to repetitive or twisting movements that limit independence and might also cause pain. The preclinical findings were featured on the cover of the Aug. 18, 2021 edition of the journal Science Translational Medicine.

Using drug screening, drug testing and imaging methods, researchers reported that ritonavir corrected abnormal protein features in the brain and had therapeutic effects in a mouse model of DYT1, restoring multiple brain abnormalities. DYT1 dystonia is an early-onset genetic form of the disorder. The mice received higher concentrations of the medication than humans are given for HIV treatment, so human clinical trials are needed to test dosage levels and safety, the researchers said, and to determine whether the same results are found in people.  

“We hope that this study will provide a promising therapeutic target in DYT1 dystonia, which is desperately needed for patients suffering from this disorder and their families,” said co-author David Vaillancourt, Ph.D., chair of the department of applied physiology and kinesiology in the University of Florida College of Health & Human Performance and director of the neuroimaging laboratory at the Norman Fixel Institute for Neurological Diseases at UF Health.

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