Several phase 1/2 clinical trials have focused on investigating the safety and feasibility of retinal cell therapy in diseases such as age-related macular degeneration, retinitis pigmentosa, and Stargardt disease. Technical improvements are anticipated in areas such as surgical delivery methods, composite cellular patches, and synthetic basement substrates for optimal retinal protection and regeneration. Immunosuppression protocols are being optimized in order to avoid the side effects of long-term systemic immunosuppression. The development of appropriate clinical trial endpoints will facilitate assessment of the efficacy of retinal stem cell therapy in phase 3 clinical trials. Greater regulatory oversight and public education are needed to eliminate the dangers associated with direct-to-consumer cell therapy, so that the progress of properly designed and supervised clinical trials is not impeded.
KeywordsCell therapy Clinical trials Age-related macular degeneration Retinitis pigmentosa Stargardt disease Immunosuppression Surgical delivery Regulatory oversight
- 24.The Food and Drug Administration. FDA warns about US stem cell therapies. 2017. https://www.fda.gov/ForConsumers/ConsumerUpdates/ucm286155.htm2018.
- 25.The Food and Drug Administration. FDA seeks permanent injunctions against two stem cell clinics. 2018. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm607257.htm. Accessed 15 May 2018.