Ocular melanomas are rare, with an incidence of approximately 5/1,000,000/year. Though local therapies are curative in many patients, approximately 50% will eventually develop metastases.

Historically, prediction of prognosis relied on clinico-pathological features, but advances in molecular testing has refined our ability to identify risk more accurately. Cytogenetic and gene expression profiling is being increasingly used. Currently follow up and surveillance programmes vary widely, though national consensus guidelines are being developed in various countries.

Treatment with systemic therapies has been generally disappointing, but recent developments in immunotherapies and in our understanding of molecular basis for ocular melanoma has provided new optimism. Unfortunately, early case reports of activity of tyrosine kinase inhibitors targeting c-Kit, were not borne out in a randomised phase II study in metastatic ocular melanoma. The identification of driver mutations in GNAQ and GNA11 and the ability to inhibit the MAPK and PI3K pathways has led to development of new treatment strategies. In an open label phase II study the MEK inhibitor selumetenib has shown promise and results of a randomised placebo-controlled trial are awaited. Combination of MEK inhibition with chemotherapy and dual pathway inhibition approaches are being actively pursued.

These and other approaches have given hope for new treatments in the future. International initiatives such as the International Rare Cancer Initiatives (IRCI) is facilitating developments in the treatment of rare cancers such as ocular melanoma.