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Tracey Rose, MD, MPH, and William Kim, MD

A new study demonstrates patients with advanced bladder cancers whose tumors have a mutated FGFR3 gene respond to immunotherapy treatment in a manner that is similar to patients without that mutation, a discovery that runs counter to previous assumptions. Led by scientists in the Department of Medicine and members of the Lineberger Comprehensive Cancer Center, the study has important implications for patients who have not been offered immunotherapy because of their genetic profiles.

“Clinical trials have shown that bladder cancers with FGFR3 mutations have fewer immune cells, primarily T cells, than cancers without the mutation, said Tracy Rose, MD, MPH, assistant professor in the division of oncology, and the paper’s co-first author, in Oncology News. “Because tumors with low levels of immune cells tend to respond poorly to immune checkpoint blockades, it has been hypothesized that those patients would have low response rates to immunotherapy.”

Rose and William Y. Kim, MD, the Rush S. Dickson Distinguished Professor of Medicine and professor of Genetics, designed the study to compare tumor tissue samples and clinical trials data from 17 patients with FGFR3-mutated bladder cancer to 86 patients whose tumors did not have the mutation. The investigators found that patients with FGFR3 mutations responded to immunotherapy equally as well as those without the mutations.

The paper was published in the British Journal of Cancer.