Use of Preclinical Models to Improve Treatment of Retinoblastoma

October 2005 | Volume 2 | Issue 10 | e332 Retinoblastoma is a rare childhood cancer of the retina. Approximately one in 20,000 children are affected worldwide; only 250 to 300 new cases are reported in the United States each year. Enucleation (see Glossary) is usually the treatment of choice for children with unilateral disease. Treatment of children with bilateral retinoblastoma is more challenging, as eye and vision preservation become priorities. Historically, bilateral retinoblastoma was treated with a combination of laser therapy, cryotherapy, and radiotherapy. Today, patients with bilateral retinoblastoma fi rst receive upfront chemotherapy to reduce the tumor burden, and then undergo aggressive focal therapies. This approach has increased the rate of eye salvage and decreases or delays the use of radiation therapy. Unfortunately, recent advances in targeted chemotherapy have not benefi ted patients with retinoblastoma because our knowledge is limited regarding the signaling pathways affected following the inactivation of the retinoblastoma gene RB1. Also, retinoblastoma clinical trials take years to complete because so few patients