Neurotrophic Factors in the Treatment of Inherited Retinal Diseases.

Abstract

Inherited retinal diseases (IRDs) are the leading cause of blindness in working-age individuals worldwide. Their genetic etiology is especially heterogenous, so the development of gene-specific therapies is unlikely to meet the medical needs of the entire patient community. Considering these challenges, a complementary strategy could be to develop therapies independent of the underlying gene variant causing retinal degeneration. As the retina is a neural tissue, it is in theory amenable to neuroprotective therapies that could help prolong cell survival or promote retinal function. Many neurotrophic factors have shown favorable results in preclinical animal models of neurodegenerative diseases, but unfortunately these findings have not yet translated into successful human clinical trials. The clinical development of these new therapies is mostly impeded by selection of pertinent clinical end points and time-to-readout, as the majority of IRDs show a relatively slow disease progression rate. Despite these challenges, several strategies have moved forward into clinical development.