Background  

Upper tract urothelial cancer (UTUC) is a cancer of the inner lining of the kidney and the tube that connects it to the bladder (also called the the ureter). It often behaves like cancers that start in the bladder, and UTUC is usually treated with surgery and chemotherapy. When patients have large or advanced tumors, they usually undergoradical nephroureterectomy, or removal of one kidney and ureter. While this surgery can be effective, it has serious risks. Losing a kidney  decreases kidney function, increases the chance of heart disease, and sometimes even requires lifelong dialysis (kidney replacement therapy). 

Interestingly, about 15% of patients who have chemotherapy before this surgery are later found to have no cancer left when the kidney and ureter are removed. This means that some patients may have been cured by chemotherapy alone and may not have needed major surgery. The challenge is that doctors do not currently have reliable tools to know who responded to chemotherapy before surgery. 

What This Research Proposes to Address 

The researcher has developed a new urine test, called a urinary biomarker. In early studies, this test was able to better tell the difference between low-risk and high-risk UTUC. Even more importantly, it showed which patients had a complete response to chemotherapy, meaning the cancer may already have been eliminated before surgery. 

The next step was to study this test in more patients who are having surgery, with or without chemotherapy before the surgery. By doing so, researchers will see how well the test will work in real-world settings. 

Why This Research Is Important 

Right now, doctors use very small biopsies and scans to figure out how advanced the cancer is. But these methods are often inaccurate. A tiny sample of a large tumor may not show the whole picture, and imaging tests don’t always give dependable results. Without better information, patients may go through surgery they might not actually need. 

If this test proves successful, it could change how UTUC is treated. Doctors would be able to more accurately measure the stage and grade of a patient’s cancer before any major decisions are made. They could also tell whether chemotherapy alone has worked, helping some patients avoid organ removal altogether. 

This would give doctors much better information to guide treatment. Most importantly, it could improve outcomes for patients by reducing unnecessary surgeries, protecting kidney function, and offering more personalized care. 

Final Report Summary

Researchers supported by BCAN discovered that tumors in the upper urinary tract release pieces of genetic material into the urine. By studying this material, doctors can get information about a patient’s cancer without needing surgery or invasive procedures. This “urine biopsy” can reflect the unique genetic makeup of each person’s tumor and may even provide more details than the small tissue samples doctors usually take. 

Currently, tissue biopsies from the upper urinary tract are difficult because doctors can only collect tiny samples. These small pieces often do not represent the entire tumor, which can lead to mistakes or missed diagnoses in as many as half of cases. Because urine naturally carries tumor material, it may offer a clearer, more accurate picture of the cancer. 

This new urine-based approach could help doctors better understand the stage and grade of upper tract tumors. With this information, they can make smarter decisions about whether surgery, chemotherapy, or other treatments are the best option. Thanks to BCAN’s funding, the Abbosh lab has been developed and established this technology, moving it closer to being a tool that doctors can use in everyday patient care.

Satyal, U., Srivastava, A., & Abbosh, P. H. (2019). Urine Biopsy-Liquid Gold for Molecular Detection and Surveillance of Bladder Cancer. Frontiers in oncology, 9, 1266. https://doi.org/10.3389/fonc.2019.01266

None Reported as of August 2025

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