How EP300 gene changes affect bladder and upper tract urothelial cancer treatment
The Role of EP300 Mutations in Bladder Cancer Pathogenesis and Drug Response
['FUNDING_R01'] · SLOAN-KETTERING INST CAN RESEARCH · NIH-11247499
The team wants to find out if EP300 gene changes make some bladder cancers resistant to the FGFR drug erdafitinib and whether blocking the IL-6–JAK1–STAT3 pathway can help those patients respond better.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SLOAN-KETTERING INST CAN RESEARCH (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11247499 on ClinicalTrials.gov |
What this research studies
If you have bladder or upper tract urothelial cancer, researchers are studying tumors with loss-of-function changes in the EP300 gene to see how those changes drive resistance to the FGFR inhibitor erdafitinib. They are using tumor samples and a large genomically characterized patient cohort together with lab models where EP300 is knocked out or mutated to map the signaling changes. The team will test whether drugs that block IL-6–JAK1–STAT3 signaling can reverse resistance in lab models and look for signals in patient data that support moving this approach into clinical testing. Findings may guide future combination treatments or biomarker-driven trials for patients with EP300-mutant tumors.
Who could benefit from this research
Good fit: Ideal candidates are people with metastatic bladder or upper tract urothelial cancer whose tumors have EP300 loss-of-function mutations or who are being treated with or considered for FGFR inhibitors like erdafitinib.
Not a fit: Patients without EP300 mutations or those not eligible for FGFR-targeted therapy are unlikely to directly benefit from the interventions tested in this project.
Why it matters
Potential benefit: If successful, this work could identify a way to overcome erdafitinib resistance and lead to new combination therapies for patients with EP300‑mutant urothelial cancer.
How similar studies have performed: FGFR inhibitors like erdafitinib have shown benefit for some patients, but using IL-6–JAK1–STAT3 blockade to overcome EP300-related resistance is a novel strategy supported so far mainly by preclinical data.
Where this research is happening
NEW YORK, UNITED STATES
- SLOAN-KETTERING INST CAN RESEARCH — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SOLIT, DAVID B. — SLOAN-KETTERING INST CAN RESEARCH
- Study coordinator: SOLIT, DAVID B.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.