Title:Progress in Small Molecule Therapeutics for the Treatment of Retinoblastoma
Volume: 16
Issue: 6
Author(s): Eleanor M. Pritchard, Michael A. Dyer and R. Kiplin Guy
Affiliation:
Keywords:
Chemoreduction, Ocular drug delivery, Retinoblastoma.
Abstract: While mortality is low for intraocular retinoblastoma patients in the developed world who receive aggressive
multimodal therapy, partial or full loss of vision occurs in approximately 50% of patients with advanced bilateral
retinoblastoma. Therapies that preserve vision and reduce late effects are needed. Because clinical trials for
retinoblastoma are difficult due to the young age of the patient population and relative rarity of the disease, robust
preclinical testing of new therapies is critical. The last decade has seen advances towards identifying new therapies
including the development of animal models of retinoblastoma for preclinical testing, progress in local drug delivery to
reach intraocular targets, and improved understanding of the underlying biological mechanisms that give rise to
retinoblastoma. This review discusses advances in these areas, with a focus on discovery and development of small
molecules for the treatment of retinoblastoma, including novel targeted therapeutics such as inhibitors of the MDMX-p53
interaction (nutlin-3a), histone deacetylase (HDAC) inhibitors, and spleen tyrosine kinase (SYK) inhibitors.
