Advanced Therapy and Clinical Trials to Treat Patients with Optic Nerve Diseases

Abstract

Optic nerve diseases include a wide variety of pathogenic conditions triggering injury or dysfunction of the optic nerves that lead to visual impairment or blindness in one or both eyes. Despite their pathogenic variety, most of them proceed through common mechanisms that allow them to investigate together. Nevertheless, roles of the cells, tissues, genes, growth factors, and proteins, and all underlying pathophysiological mechanisms need to be studied fully for better management of each optic nerve disease. This review presents a collection of information regarding ongoing and completed clinical trials (CT) of advanced therapies that deliver stem cell and gene therapy treatments as drugs to patients with optic nerve diseases as well as successes and failures achieved in treating these patients in the last few years. These drugs seem safe from creating neurotoxicity. It describes outcomes of a bibliographic search for stem cell therapy, gene therapy, and neuroprotection-based CT registered in the International ClinicalTrials.gov, the European EudraCT, and the Spanish REEC database, and related papers published in the PUBMED database by applying different search terminologies. This review overall informs the patients of optic neurodiseases that advanced therapies are progressing successfully in search of effective and safe treatments for them.

Keywords: Clinical trial; Gene therapy; Neuroprotection; Optic nerve disease; Stem cell therapy.