Tyler’s Hope Funded Breakthrough Discovery

​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. 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. 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 analyzed MRI data for the research team, which was led by Nicole Calakos, M.D., Ph.D., a professor of neurobiology and neurology at Duke University. Calakos said she is encouraged that the MRI findings showed lasting correction in the mice after a brief treatment with ritonavir and hopes that the drug holds potential not just to address symptoms of dystonia but possibly treat the underlying cause.