Researchers from the University of Maryland School of Medicine (UMSOM), the National Institutes of Health’s National Eye Institute (NEI), and the National Institute on Deafness and Other Communication Disorders (NIDCD) have recently gathered data from patients and mice with the genes causing Usher syndrome. This data was used to map out the natural history of vision impairment and help find the cell process that led to progressive vision loss. The team then tested a retinoid therapy that was able to improve the vision in mice with Usher syndrome type 1F. A similar approach is being evaluated for people with Usher syndrome. The FDA has approved retinoid drugs similar to the one used in this study for other conditions and there is enough safety and tolerability data to expedite the approval for its testing in patients with Usher syndrome. These researchers hope to work with companies that created these drugs to start a clinical trial for patients with Usher syndrome type 1F to see if it can also help prevent continuing vision loss.
What this means for Usher syndrome: There is some success in using retinoid therapy in mice with Usher syndrome to improve vision. Researchers might be able to start working with drug companies that produce retinoid therapies to launch clinical trials for individuals with Usher syndrome type 1F to see if these therapies can prevent continuing vision loss.