Retinoblastoma: what is new in 2007-2008.

PURPOSE OF REVIEW: This review highlights many of the most important articles published from June 2007 to May 2008 on retinoblastoma. Significant advances in molecular biology, translational research, and clinical reports are detailed.

RECENT FINDINGS: The most significant recent findings in the molecular biology of retinoblastoma include the evidence for aneuploidy and genomic instability as cancer causes rather than the long-held Knudson's 'two-hit' hypothesis; the evidence that retinoma may represent a precursor lesion for retinoblastoma prior to the acquisition of genomic instability; and the evidence that a horizontal interneuron may be the cell of origin in murine knockout retinoblastoma and may be capable of clonal expansion after differentiation. Translational studies also demonstrate promise for the use of topotecan and 2-deoxy-D-glucose in children. Finally, the introduction of intraarterial chemotherapy for human intraocular retinoblastoma appears to be safe and effective and may eliminate the need for enucleation in many patients.

SUMMARY: Exciting new advances in both the basic science and clinical applications of new therapies continue to emerge for this rare disease. We expect that local control rates even for advanced intraocular retinoblastoma will soon reach 100% in the developed world as a result of new findings in the clinic and in the laboratory.