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Johns Hopkins Pediatric

Tackling a Rare, Aggressive Form of Cancer


Small cell neuroendocrine bladder carcinoma (SCBC) is an aggressive, lethal variant of urothelial carcinoma, and much about it, until recently, has been a mystery. Because it’s so rare – affecting less than 1 percent of all people with bladder cancer – “its molecular characteristics remain elusive, and no standard treatment options are available,” says scientist Woonyoung Choi, Ph.D.

It turns out that small cell neuroendocrine bladder carcinoma has a lot in common with small cell neuroendocrine lung cancer, and this may lead to new strategies for treatment.

Choi, in collaboration with medical oncologist Jean Hoffman-Censits, M.D., is working to shed some light on SCBC. Recent genomics studies have shown the overall similarity of small cell neuroendocrine tumors in the lung and other sites, Choi says. Based on these observations, she discovered subtypes of SCBC that are driven by three key transcriptional regulators – which are similar to those found in small cell lung cancer. “The next step is to comprehensively characterize the biological characteristics of each subtype, in order to identify novel therapeutic targets for patients with deadly SCBC.”

With Hoffman-Censits, Choi is investigating how well these novel subtypes respond to therapy in a Phase 2 clinical trial of neoadjuvant chemotherapy plus atezolizumab in patients with SCBC.

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