New topoisomerase I drug combination for ovarian cancer
Project 2: Next Generation TOP1 Inhibition for the Treatment of Ovarian Cancer
This work tests whether a long-acting topoisomerase I drug given with the PARP blocker rucaparib can overcome resistance in ovarian cancer patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project ID | NIH-11178540 on NIH RePORTER |
What this research studies
From a patient perspective, researchers are combining a next-generation topoisomerase I inhibitor (including a long-acting nanoparticle form called PLX038) with the PARP inhibitor rucaparib to try to treat ovarian cancers that have become resistant to PARP drugs. They use patient-derived tumor models and laboratory tests that detect drug-induced DNA–protein complexes to see if the combination kills tumors that no longer respond to PARP inhibitors. The team is particularly focused on tumors with BRCA1/2 or other defects in DNA repair that initially respond to PARP blockers but later relapse. The approach aims to find combinations that work better than single drugs while reducing bone marrow side effects seen with older topoisomerase I drugs.
Who could benefit from this research
Good fit: Ideal candidates would be people with recurrent ovarian cancer, especially those whose tumors are resistant to PARP inhibitors or who have BRCA1/2-related DNA repair defects.
Not a fit: People without ovarian cancer or whose tumors are not driven by DNA-repair defects or PARP-inhibitor resistance are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could offer a new treatment option for ovarian cancers that stop responding to PARP inhibitors and extend responses with fewer marrow side effects.
How similar studies have performed: Preclinical studies and patient-derived tumor models have shown promise for long-acting TOP1 inhibitors and PARP inhibitors separately and in combination, but clinical data on this specific combo are limited.
Where this research is happening
Rochester, United States
- Mayo Clinic Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Kaufmann, Scott H — Mayo Clinic Rochester
- Study coordinator: Kaufmann, Scott H
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.