Down Syndrome (Ds) is characterized by trisomy of the 21st chromosome. The additional chromosomal DNA results in a reduced risk of bladder cancer in humans. We hypothesized that this could be explored to identify novel therapeutic targets to improve treatment of this disease. Our preliminary findings confirmed a reduction in tumor formation in a mouse model of bladder cancer using Ds mice. Additionally, we identified a potential therapeutic target. Funding through the BCAN Innovation Award has allowed us to further explore these findings.

Using two separate genetic mouse models of Ds, we have found some Ds genotypes reduce tumor formation in a carcinogen driven model, whereas others do not. We are currently determining why this difference between the mouse strains occurs, and how this relates to bladder cancer. Additionally, we have found that an inhibitor of a pathway identified in our earlier studies paradoxically results in increased tumors when used in a mouse with an intact immune system. We believe this may be a result of changes in the immune system due to the drug. The project remains ongoing with plans to use these data to find new drug targets and further understand the possible role of the immune system and how Ds protection from bladder cancer can be used to improve patient outcomes.

None Reported as of August 2025

Department of Defense Peer Reviewed Cancer Research Program, “Targeting Dendritic Cells in Bladder Cancer Immunotherapy”, 2024, $653,928

Ben Woolbright, PhD; University of Kansas Medical Center
Katie Dennis, MD; University of Kansas Medical Center
Mihaela Sardiu, PhD; University of Kansas Medical Center