Progress in small molecule therapeutics for the treatment of retinoblastoma

Eleanor M. Pritchard, Eleanor M. Pritchard, Michael A. Dyer, Michael A. Dyer, Michael A. Dyer, R. Kiplin Guy

Research output: Contribution to journalArticlepeer-review

44 Scopus citations


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.

Original languageEnglish
Pages (from-to)430-454
Number of pages25
JournalMini-Reviews in Medicinal Chemistry
Issue number6
StatePublished - Apr 1 2016

Bibliographical note

Publisher Copyright:
© 2016 Bentham Science Publishers.


  • Chemoreduction
  • Ocular drug delivery
  • Retinoblastoma

ASJC Scopus subject areas

  • Drug Discovery
  • Pharmacology


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