Helping mutated p53 fold correctly to lower cancer risk in Li‑Fraumeni syndrome
Official title: Refolding Mutant p53: A Strategy for Cancer Prevention in Li-Fraumeni Syndrome
Funded at Research Inst of Fox Chase Can Ctr · NIH-11168825 · Data updated 2026-11-12
This project tests drugs that help mutated p53 protein fold properly to lower cancer risk for people with Li‑Fraumeni syndrome.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst of Fox Chase Can Ctr NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 1, 2022 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $612,511 |
| NIH project number | 5U54CA272686-04 |
| Project ID | NIH-11168825 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be people diagnosed with Li‑Fraumeni syndrome who carry germline TP53 missense mutations.
✕Less likely to help
People without TP53 mutations, those whose cancers are driven by other causes, or individuals with advanced established tumors may not benefit from this prevention-focused approach.
What this research studies
From your perspective, researchers are developing small molecules that bind and stabilize the p53 protein so it folds into its normal shape. They focus on Li‑Fraumeni syndrome, where inherited TP53 mutations often make p53 unstable and dysfunctional. In the lab the team tests these “refolder” compounds across many cancer cell lines and in animal models to see if restored p53 activity can block early lesion formation. If preclinical results are promising, the researchers plan to advance toward testing in at‑risk people.
Why it matters
Potential benefit: If successful, these drugs could lower cancer risk in people with Li‑Fraumeni syndrome by restoring normal p53 function before tumors form.
How similar studies have gone: Related refolding approaches (for example APR‑246/eprenetapopt) showed preclinical promise and some early clinical activity in p53‑mutant cancers, but prevention in Li‑Fraumeni individuals is not yet proven.
Where this research is happening
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Research Inst of Fox Chase Can Ctr
Philadelphia, United States Active
Researchers
- Principal investigator: Golemis, Erica a. — Research Inst of Fox Chase Can Ctr
- Study coordinator: Golemis, Erica a.
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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.