Targeting a DNA repair weakness in Ewing sarcoma
Exploiting a Novel DNA Repair Defect in Ewing Sarcoma
['FUNDING_R01'] · DANA-FARBER CANCER INST · NIH-11240282
This project aims to exploit a DNA repair weakness to develop new treatments for children and young adults with Ewing sarcoma.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | DANA-FARBER CANCER INST (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11240282 on ClinicalTrials.gov |
What this research studies
Researchers found that Ewing sarcoma cells have a specific fault in a DNA repair pathway called microhomology-mediated end joining (MMEJ) caused by changes in the EWSR1 gene and loss of POLQ protein. The team uses laboratory models, CRISPR gene editing, and cancer cell lines to study how that repair fault makes tumors unusually sensitive to certain chemotherapy drugs and radiation. By understanding this weakness, they plan to test drug combinations in preclinical experiments that could selectively kill Ewing sarcoma cells. The work aims to translate those findings into treatment strategies that could be offered at clinical centers.
Who could benefit from this research
Good fit: Ideal candidates would be children, adolescents, and young adults diagnosed with Ewing sarcoma, particularly those with relapsed or metastatic disease or whose tumors show the specific DNA repair defect.
Not a fit: Patients without Ewing sarcoma or whose tumors lack the specific DNA repair defect are unlikely to benefit from these targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to targeted therapies that make chemotherapy or radiation more effective for patients with Ewing sarcoma, especially those with relapsed or metastatic disease.
How similar studies have performed: Approaches that target DNA repair defects (for example, PARP inhibitors in BRCA-mutant cancers) have worked in other tumors, but exploiting MMEJ/POLQ defects in Ewing sarcoma is a newer and less-tested idea.
Where this research is happening
BOSTON, UNITED STATES
- DANA-FARBER CANCER INST — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: D'ANDREA, ALAN D. — DANA-FARBER CANCER INST
- Study coordinator: D'ANDREA, ALAN D.
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.
Conditions: Cancers