Optimizing theranostic radiopharmaceutical therapy to combat resistance to PARP inhibition in advanced ovarian cancer

NIH RePORTER · NIH · R01 · $636,515 · view on reporter.nih.gov ↗

Abstract

Project Summary (limited to 30 lines) Ovarian cancer is a devastating gynecologic malignancy with 13,770 estimated US deaths in 2021 with a 49.1% relative 5 year survival rate (SEER). Leveraging the considerable fraction of women with ovarian cancer who have germline or somatic homologous recombination deficiencies (HRD), poly (ADP-ribose) polymerase 1 inhibitors (PARPi) have become standard of care for ovarian cancer. Unfortunately, most patients treated with PARPi develop resistance. Interestingly, most PARPi resistant patients continue to express activated PARP-1 (the target of PARPi) in the nucleus. This finding has guided our alpha theranostic radiopharmaceutical approach to treating PARPi-resistant ovarian cancer to deliver cytotoxic alpha particles directly to the nucleus of cancer cells to create lethal, double stranded DNA breaks. Our platform is based on a small molecule similar to the approved PARPi, rucapararib, labeled with 18F for PET ([18F]fluorthanatrace, [18F]FTT) and 211At for alpha therapy ([211At]parthanatrace, [211At]PTT). Our overarching hypothesis is that [211At]PTT can overcome PARPi resistance with an acceptable therapeutic ratio and that the theranostic pair of [211At]PTT and [18F]FTT will allow accurate pre-treatment dosimetry to guide both efficacy and tolerability of therapy. Using mouse models of ovarian cancer, we plan to (1) measure the comparative dynamic biodistribution of the proposed theranostic pair, [18F]FTT and [211At]PTT to verify preliminary data supporting similar tumor uptake and tissue kinetics, (2) understand the dose-response relationship in tumor and organs at risk (both those expressing considerable PARP-1 and those with off target agent biodistribution); (3) carry out pre-clinical studies to assess toxicity and efficacy of the [211At]PTT /[18F]FTT theranostic approach with optimized dose fractionation. Our specific aims are as follows: SA1—Biodistribution and Pharmacokinetics: Test the comparative biodistribution of [18F]FTT and [211At]PTT in normal tissue and ovarian cancer. SA2—Dose-response relationship: Develop and validate on- and off-target normal organ dose limits and tumor dose-response relationship utilizing [18F]FTT PET/CT to predict dosimetry from [211At]PTT. SA3—Pre-clinical trials: Determine the optimal dosing scheme and estimate efficacy in patient derived ovarian cancer murine models. Successful completion of these aims will provide data needed for a first in human clinical trial of [18F]FTT -guided [211At]PTT therapy in women with advanced PARPi-resistant ovarian cancer and will provide insight into tumor factors mediating effective alpha particle therapy to guide patient selection in clinical trials and clinical practice.

Key facts

NIH application ID
10851717
Project number
5R01CA278882-02
Recipient
UNIVERSITY OF PENNSYLVANIA
Principal Investigator
Michael David Farwell
Activity code
R01
Funding institute
NIH
Fiscal year
2024
Award amount
$636,515
Award type
5
Project period
2023-05-31 → 2028-04-30