Abstract

Neuroprotection in glaucoma is any medical treatment by which decline in visual function can be slowed or prevented by supporting the health and survival of neural cells, independent of lowering of intraocular pressure (IOP). This is achieved by targeting mechanisms to inhibit or delay retinal ganglion cell death and promote cell survival pathways. Despite demonstrating promising results in preclinical trials, many neuroprotective strategies have failed to show success in subsequent clinical trials. Of the clinical trials performed, many have been hampered by slow disease progression and questions surrounding biomarker sensitivity. Adaptive clinical trial design, enriched populations and the use of state-of-the-art clinical endpoints are required to improve assessment of therapeutic efficacy. We review the neuroprotective strategies in glaucoma that have been investigated in clinical trials, and appraise experimental designs, the strength of the original hypotheses and preceding work to examine why so few candidates have successfully translated into clinical research.

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