Targeting the protein SIRT5 with new drugs for Ewing sarcoma
Official title: SIRT5 inhibitors and degraders as novel treatments for Ewing sarcoma
Funded at University of Miami School of Medicine · NIH-11330232 · Data updated 2026-11-12
New drugs that block or break down a protein called SIRT5 are being developed to treat children and young adults with Ewing sarcoma.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | January 1, 2023 |
| Project end | December 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $514,887 |
| NIH project number | 5R01CA253986-05 |
| Project ID | NIH-11330232 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young adults diagnosed with Ewing sarcoma—especially those with metastatic or relapsed disease or tumors shown to depend on SIRT5—would be the likely candidates for future trials.
✕Less likely to help
Patients with other cancer types, tumors that do not rely on SIRT5, or individuals outside the pediatric/young-adult age range are unlikely to benefit from this specific approach.
What this research studies
This research is creating drugs that either inhibit SIRT5 or cause cancer cells to degrade SIRT5 to stop Ewing sarcoma from growing. The team will test these compounds in laboratory-grown tumor cells and animal models, measuring tumor cell death and changes in gene activity. They will study how SIRT5 affects histone modifications and the abnormal gene program driven by the EWS-FLI1 fusion protein. Promising laboratory results could lead to early human trials in the future.
Why it matters
Potential benefit: If successful, these treatments could provide a more targeted way to kill Ewing sarcoma cells and might reduce the long-term side effects seen with standard chemotherapy and radiation.
How similar studies have gone: Laboratory work has shown some cancers, including Ewing sarcoma, depend on SIRT5 and die when it is removed, but SIRT5-targeting drugs have not yet been proven effective in patients.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Lombard, David Benner — University of Miami School of Medicine
- Study coordinator: Lombard, David Benner
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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.