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Hope in sight for retinoblastoma

Nature Medicine volume 13, pages 30–31 (2007)Cite this article

The retinoblastoma gene (RB) was the first tumor suppressor gene cloned. But, until recently, little clinical progress had been made to target human tumors deficient in RB function. A new approach emerges from studying retinoblastoma tumors.

Most, if not all, human tumor cells have mutations in the RB pathway, which acts as a checkpoint at the G1/S transition of the cell cycle. Thus, therapeutic approaches against cells with altered function of the pRB protein would benefit a large number of cancer patients1. However, limited progress has been made in identifying drugs that specifically recognize tumor cells with loss of pRB function. For instance, retinoblastoma, the pediatric retinal tumor that is the archetypal RB-mutant tumor, is still often treated by enucleation, radiation and systemic chemotherapy, resulting in blindness and increased risk of secondary tumors2.

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Figure 1: Possible points of therapeutic interventions against human cancers with alterations in the RB pathway.

Katie Ris

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Authors and Affiliations

  1. Departments of Pediatrics and Genetics, Stanford University, Stanford, 94305, California, USA

    Julien Sage

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  1. Julien Sage
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Sage, J. Hope in sight for retinoblastoma. Nat Med 13, 30–31 (2007). https://doi.org/10.1038/nm0107-30

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  • DOI: https://doi.org/10.1038/nm0107-30

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