Stem Cell-Based Therapeutics in Ophthalmology: Application Toward the Design of Clinical Trials

Stem cell-based therapeutics have been proposed as a technology for restoration of anatomic structure and visual function for retinal degenerative diseases. Success in animal studies and preliminary trials may offer hope for patients afflicted by a variety of retinal degenerations. However, as clinical trials expand and advance to latter phases, it is important to address key study design issues. This chapter discusses the parameters for research into stem cell-based therapeutics. Efficacy endpoints for studies can be defined along objective physiologic, psychofunctional, anatomic, and functional living axes. Pupillometry, electroretinography, and radiologic tools are discussed as objective tools for the assessment of treatment outcome. Additionally, optical coherence tomography(OCT), fundus autofluorescence (FAF), and other imaging tools may be used. Psychofunctional tests may be less reliable among a pediatric population. Finally, improvements in functional living may be reported by patients and assessed by various measures.