Researchers at the University of Basel and the Institute of Molecular and Clinical Ophthalmology Basel (IOB), who are currently developing gene therapy for Stargardt disease, have affirmed the value of microperimetry (MP) testing in monitoring disease progression.
“To develop a treatment for Stargardt disease, we first of all need sensitive examination procedures for measuring disease progression. Without these we will not be able to assess the efficacy of novel therapeutic approaches,” explained University of Basel Chair Prof Hendrik Scholl, who is also Clinical Director of IOB and Principal Investigator for the international multicentre Progression of Atrophy Secondary to Stargardt disease (ProgStar) study.
“In the ProgStar study, we have examined structural abnormalities in the retina over time in Stargardt disease using various techniques. Structural changes are important outcome measures in interventional studies, but they must be associated with functional loss to be meaningful.”
Study authors said data presented in the ProgStar study provides evidence that MP is a sensitive test for detecting progression within a short study period, i.e. the yearly rate of change of macular function in patients with Stargardt disease. MP measurements therefore may serve as a useful functional outcome parameter for clinical trials investigating the efficacy of emerging treatments that aim to slow or stop disease progression.
Prof Scholl said the findings would be helpful in his team’s development of a gene therapy for Stargardt disease that uses base editing to change the mutated genetic code directly by reverting disease-causing mutations back to normal.
References
Etienne M. Schönbach, MD; Rupert W. Strauss, MD; Beatriz Muñoz, MS; et al. Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months ProgStar Report No. 13 JAMA Ophthalmol. Published online May 28, 2020. doi:10.1001/jamaophthalmol.2020.1735
