Cell Replacement Therapy for Retinal and Optic Nerve Diseases Report Summary

 

Key Findings

The report highlights that RPCs, PSCs, and MSCs have already entered early-stage clinical trials. These cell types have shown the ability to differentiate into retinal pigment epithelium (RPE), photoreceptors, or ganglion cells and have demonstrated safety and some indicators of efficacy. However, there are challenges to overcome, such as the inhibition of cell proliferation and/or differentiation in vitro, and the limited long-term survival and functioning of grafts in vivo.

Challenges and Future Directions

• The ability to enrich for specific retinal subtypes.
• Ensuring cell survival post-transplantation.
• Cell delivery methods, which may require a scaffold to induce correct cell polarization.
• The need to induce localized retinal detachment for subretinal placement of the transplanted cell.
• Evaluating the risk of tumor formation caused by undifferentiated stem cells and prolific progenitor cells.

Despite these challenges, the report concludes that stem/progenitor cells represent the most promising strategy for treating retinal and optic nerve diseases in the near future. The report also suggests that therapeutics assisted by gene techniques, neuroprotective compounds, and artificial devices could be applied to meet clinical needs.