Research that began more than 10 years ago in the lab of Michael Dyer, PhD, Developmental Neurobiology, has advanced treatment of the eye cancer retinoblastoma.
Between 250 and 300 cases of retinoblastoma are diagnosed in the U.S. every year. Most patients are infants or toddlers when the tumor is found. Long-term survival in the U.S. is excellent, but treatment side effects may be severe. Sometimes, a cure requires radiation or surgery to remove an eye.
The need for new drugs and drug combos prompted Dyer’s research. Working in the lab, his team found the drug topotecan was effective in laboratory models of retinoblastoma. Working closely with Carlos Rodriguez-Galindo, MD, Global Pediatric Medicine, and Matthew Wilson, MD, Surgery, the team asked whether topotecan could be substituted for etoposide in three-drug chemotherapy. This treatment might be more effective and safer, since etoposide leaves patients at risk for leukemia.
Now, St. Jude researchers have shown that topotecan should be part of up-front chemo for patients with advanced retinoblastoma. With 10 years of follow-up, a study of 26 patients found that the three-drug chemo with topotecan cured their cancer. The treatment also reduced the need for surgery or radiation therapy. And more than 80 percent of the survivors had measurable vision. These results are better than previously reported results.
“These findings are the culmination of a true team effort,” said Rachel Brennan, MD, Oncology. “This is a shining example of how St. Jude is paving the way from the lab to the clinic for new approaches to therapy that focus on the whole patient, not just the eye.”
The research appeared in the Journal of Clinical Oncology.