In 2021, we launched RUSH1F, the natural history study for Usher syndrome type 1F, in partnership with Foundation Fighting Blindness. Conducted at 11 clinical sites worldwide, the study tracks the natural progression of vision loss in the absence of treatment and provides the critical data needed to prepare for future clinical trials.
Natural history studies are especially important for rare diseases because they establish the benchmark against which new therapies are measured. Without understanding how vision changes over time without treatment, researchers cannot determine whether an experimental therapy is slowing, stopping, or reversing vision loss. In addition, the data help researchers design clinical trials, identify meaningful outcome measures, and support regulatory discussions with the FDA. Completing a natural history study is therefore a critical milestone on the path to a clinical trial.
The Marjorie C. Adams Trust provided additional funding for the project. We are deeply grateful to Foundation Fighting Blindness and the Trust for their partnership in achieving this critical milestone for the Usher syndrome type 1F community.
RUSH1F enrolled 44 individuals with Usher syndrome type 1F, ages 8 and older, who have been followed over four years to assess changes in vision and retinal structure. Coordinated by the Jaeb Center for Health Research in Tampa, Florida, and led by an international team of investigators chaired by Dr. Katarina Stingl of the University of Tübingen, the study is defining the outcome measures needed to evaluate future therapies and helping pave the way for the first clinical trials for Usher syndrome type 1F.
The successful completion of the RUSH1F natural history in 2027 study will represent a landmark achievement for the Usher 1F community. This more than $2 million international effort was made possible through a true partnership, with Usher 1F Collaborative providing $1.1 million in support, the Marjorie C. Adams Trust contributing $115,000, and Foundation Fighting Blindness providing $1 million to fund the remaining costs. Together, these investments have established the clinical foundation needed to advance promising therapies into human clinical trials, bringing hope to families affected by Usher syndrome type 1F.
