A ranzco position statement on ocular stem cell therapy

Abstract

Purpose: A greater understanding of the function and location of ocular stem cells (SCs) has led to rapid advances in possible therapeutic applications. However, ocular SC therapy is associated with significant technical challenges and potential long‐term complications. Whilst other international organisations have developed guidelines regarding the use of ocular SC therapy, a RANZCO position statement does not yet exist. Our purpose was to develop a position statement on the role of ocular SC therapy from an Australian perspective in the context of international advances.

Methods: A systematic literature review of ocular SC therapy was conducted, registered clinical trials reviewed and expert opinions sought. Collated data was used to develop a position statement based on current guidelines whilst also considering activities occurring outside the expected pathways to clinical translation.

Results: This position statement supports advancements in knowledge, whilst endeavouring to minimise patient harm. The literature suggests that limbal epithelial progenitor cells for ocular surface transplantation are a viable therapy based on Phase I/II clinical trials, and that the development of more robust clinical trials is reasonable. Further pre‐clinical evaluation and/or clinical trials of all other cellular‐based interventions that rely on induced pluripotent SCs, human embryonic SCs, mesenchymal SCs and other potential SC sources are required.

Conclusion: Expert opinion and systematic review of the safety and efficacy of ocular SCs were used to develop a position statement on ocular SC therapy. This has recently been approved by the RANZCO Board as the college’s first position statement on the topic.