HIV drug shows potential to combat form of dystonia in preclinical study

composite photo with the magazine cover displaying a brain scan on the left and photo of doctor david vaillancourt on the right
Dr. David Vaillancourt co-authored a study featured on the cover of August’s edition of the journal Science Translational Medicine.

By Todd Taylor

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

The preclinical findings, featured on the cover of August’s edition of the journal Science Translational Medicine, identified ritonavir as a drug candidate with disease-modifying potential for DYT1. DYT1 is an early-onset genetic form of dystonia, a neurological disorder that causes muscles to contract involuntarily, leading to repetitive or twisting movements that limit independence.

Using in vitro drug screening, in vivo testing and ex vivo diffusion imaging methods, researchers reported that ritonavir corrected protein features and had therapeutic effects in a mouse model of DYT1, restoring multiple brain abnormalities when administered during an early developmental period.

“We hope that this study will provide a proof of principle for a promising therapeutic target in DYT1 dystonia, which is desperately needed for patients and 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.

The study was led by the laboratory of Nicole Calakos, M.D., Ph.D., a professor of neurobiology at Duke University, and it was supported by a grant from Tyler’s Hope for a Dystonia Cure, a Gainesville-based foundation dedicated to advancing research and promoting awareness and education of DYT1 dystonia.

“This work is a great example of researchers collaborating and breaking down some of the barriers that have historically impeded progress by being siloed,” said Rick Staab, co-founder of Tyler’s Hope for a Dystonia Cure. “This is an exciting breakthrough and although there are more steps to go, I believe these findings will prove to have a huge impact on our quest to provide better treatments and ultimately find a cure for this devastating disorder.”

Read the full press release.

Read the paper in Science Translational Medicine.