Drug Discovery and Repurposing for Retinoblastoma

Part of the Essentials in Ophthalmology book series (ESSENTIALS)


Retinoblastoma, a rare cancer of the eye, presents an urgent and unmet clinical need for an improved and effective treatment. Discovery of novel drugs and therapy would require an emphasis and refocus on adopting approaches that are fast, efficacious, and innovative, so as to bridge the growing gap between research labs and clinic. To this end, three main approaches with a potential of high return on investment and better value proposition have been described. First, use of high-throughput phenotypic screens to test a focused set of FDA-approved drugs presents an excellent opportunity to repurpose existing drugs and foster translational research, with minimal time commitment. This is also complemented by the advent of alternate routes of localized drug delivery, which opens doors to revisit shelved drugs previously ignored due to their high systemic toxicity. In addition, testing natural products offer the capability to broaden the limited structural diversity within the commercially available chemical collections and can fuel identification of new chemical entities. Second, non-toxic nutraceuticals can be evaluated for their applicability as companion effectors to enhance ongoing chemotherapy, improving the health of young patients. Third, use of three-dimensional models to test chemotherapeutic agents for retinoblastoma is proposed; such models are true prototypes of an in vivo tumor, and perhaps more realistic indicators of a drug’s efficacy and higher potential of clinical success. Taken together, these three approaches can be viewed as the rapid routes towards drug discovery with hopes to establish a quick and potent therapy for retinoblastoma.


Chemical Drug discovery High-throughput Multi-cellular tumor spheroids Natural products Phenotypic Polypharmacology Repurpose Retinoblastoma Screening Target-based Three-dimension culture Translational research 


Compliance with Ethical Requirements

All authors (Bhavneet Bhinder, Jeni P. Mahida, Glorymar Ibáñez, Kathryn Champ, Christophe Antczak, and Hakim Djaballah) declare that they have no conflict of interest.

No human or animal studies were carried out by the authors for this article.


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Copyright information

© Springer International Publishing Switzerland 2015

Authors and Affiliations

  1. 1.HTS Core Facility, Memorial Sloan Kettering Cancer CenterNew YorkUSA
  2. 2.Thermo Fisher ScientificBangaloreIndia
  3. 3.Novartis Institutes for Biomedical Research, Center for Proteomic ChemistryCambridgeUSA
  4. 4.Institut Pasteur KoreaGyeonggi-doKorea

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