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Investigators at St. Jude Children’s Research Hospital have demonstrated in laboratory studies a potential new treatment for the pediatric eye cancer retinoblastoma that appears to be more effective than the current standard therapy. A report on their work appears in the October 15 issue of Clinical Cancer Research.
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; and that the new combination 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.
A major roadblock 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 Michael Dyer, PhD, Developmental Neurobiology, senior author of the Clinical Cancer Research report.
Therefore, it is important that any new treatments being considered for clinical trials have already demonstrated in the laboratory a high likelihood of success in clinical trials. Until last year when St. Jude researchers 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.
Barrett Haik, MD, St. Jude Ophthalmology; Carlos Rodriguez-Galindo, MD, St. Jude Hematology-Oncology; and Matthew Wilson, MD, St. Jude Ocular Oncology, are pioneers in the development of clinical trials to improve treatment for retinoblastoma, according to Dyer. Rodriguez-Galindo and Wilson recently demonstrated that etoposide can be eliminated from the triple drug therapy; and in January 2005 they opened a clinical trial (RET-5) at St. Jude that is focused on topotecan chemotherapy.
Nikia Laurie, PhD, Developmental Neurobiology, is first author of the current paper and did much of the work on this project. Other St. Jude authors include Jonathan Gray and Jiakun Zhang, MD, PhD, Developmental Neurobiology; Mark Leggas, Mary Relling, PharmD, and Clinton Stewart, PharmD, Pharmaceutical Sciences.
Last update: November 2005