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Researchers find possible pediatric eye cancer treatment

Investigators at St Jude Children's Research Hospital have found a potential new treatment for the pediatric eye cancer retinoblastoma that appears to be more effective than the current standard therapy.

The study showed in laboratory models that combination therapy with topotecan and carboplatin is superior to the standard triple-drug therapy using vincristine, carboplatin and etoposide.

The new combination developed by the scientists eliminates the use of etoposide, a drug known to increase the risk of developing acute myeloblastic leukemia, and the study suggested that vincristine contributes little to the treatment of retinoblastoma, and therefore can be eliminated from therapy.

An obstacle in efforts to design better treatments is that there are not enough patients for researchers to enroll in large clinical trials designed to investigate new treatments, according to Dr Michael Dyer, an assistant member of the department of Developmental Neurobiology and senior author of the report that was published in Clinical Cancer Care.

Therefore, it is critically important that any new treatment being considered for clinical trials have already demonstrated in the laboratory a high likelihood of success in clinical trials. Until last year when researchers at St Jude developed the first knockout model of retinoblastoma and the two other models presented in this work, there were few options for preclinical studies on retinoblastoma.