Targeting mutations in the TERT gene to treat bladder cancer
Official title: Targeting the mutant promoter of Telomerase Reverse Transcriptase (TERT)
Funded at Cedars-Sinai Medical Center · NIH-10930836 · Data updated 2026-11-12
This study is looking for new treatments for bladder cancer by finding ways to target a specific gene mutation that many bladder cancer patients have, with the goal of creating medicines that can kill cancer cells while leaving healthy cells alone.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cedars-Sinai Medical Center NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | September 18, 2023 |
| Project end | July 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $563,904 |
| NIH project number | 5R01CA275078-02 |
| Project ID | NIH-10930836 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with bladder cancer, particularly those with TERT promoter mutations.
✕Less likely to help
Patients with bladder cancer who do not have TERT promoter mutations may not benefit from this research.
What this research studies
This research focuses on bladder cancer, specifically targeting mutations in the TERT gene that are found in approximately 70% of bladder cancer cases. The team is developing innovative assays to screen for small molecules that can selectively reduce the expression of the mutated TERT without affecting the normal version of the gene. By using advanced techniques like CRISPR and high-throughput screening, they aim to identify compounds that can specifically kill bladder cancer cells while sparing healthy cells. This approach could lead to new, targeted therapies for patients with bladder cancer.
Why it matters
Potential benefit: If successful, this research could lead to the development of a targeted treatment for bladder cancer that minimizes harm to healthy cells.
How similar studies have gone: Other research has shown promise in targeting genetic mutations in cancer, suggesting that this approach could be effective.
Where this research is happening
-
Cedars-Sinai Medical Center
Los Angeles, United States Active
Researchers
- Principal investigator: Theodorescu, Dan — Cedars-Sinai Medical Center
- Study coordinator: Theodorescu, Dan
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.