Pharmacodynamic measures within tumors expose differential activity of PD(L)-1 antibody therapeutics

Proc Natl Acad Sci U S A. 2021 Sep 14;118(37):e2107982118. doi: 10.1073/pnas.2107982118.

Abstract

Macromolecules such as monoclonal antibodies (mAbs) are likely to experience poor tumor penetration because of their large size, and thus low drug exposure of target cells within a tumor could contribute to suboptimal responses. Given the challenge of inadequate quantitative tools to assess mAb activity within tumors, we hypothesized that measurement of accessible target levels in tumors could elucidate the pharmacologic activity of a mAb and could be used to compare the activity of different mAbs. Using positron emission tomography (PET), we measured the pharmacodynamics of immune checkpoint protein programmed-death ligand 1 (PD-L1) to evaluate pharmacologic effects of mAbs targeting PD-L1 and its receptor programmed cell death protein 1 (PD-1). For PD-L1 quantification, we first developed a small peptide-based fluorine-18-labeled PET imaging agent, [18F]DK222, which provided high-contrast images in preclinical models. We then quantified accessible PD-L1 levels in the tumor bed during treatment with anti-PD-1 and anti-PD-L1 mAbs. Applying mixed-effects models to these data, we found subtle differences in the pharmacodynamic effects of two anti-PD-1 mAbs (nivolumab and pembrolizumab). In contrast, we observed starkly divergent target engagement with anti-PD-L1 mAbs (atezolizumab, avelumab, and durvalumab) that were administered at equivalent doses, correlating with differential effects on tumor growth. Thus, we show that measuring PD-L1 pharmacodynamics informs mechanistic understanding of therapeutic mAbs targeting PD-L1 and PD-1. These findings demonstrate the value of quantifying target pharmacodynamics to elucidate the pharmacologic activity of mAbs, independent of mAb biophysical properties and inclusive of all physiological variables, which are highly heterogeneous within and across tumors and patients.

Keywords: PET; drug disposition; immune checkpoint therapy; pharmacodynamics; pharmacokinetics.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Antineoplastic Agents, Immunological / pharmacology*
  • Apoptosis
  • B7-H1 Antigen / antagonists & inhibitors*
  • Breast Neoplasms / diagnostic imaging
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / metabolism
  • Breast Neoplasms / pathology
  • Cell Proliferation
  • Female
  • Fluorine Radioisotopes / pharmacokinetics*
  • Humans
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Peptide Fragments / pharmacokinetics*
  • Positron-Emission Tomography / methods*
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors*
  • Radiopharmaceuticals / pharmacokinetics
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents, Immunological
  • B7-H1 Antigen
  • CD274 protein, human
  • Fluorine Radioisotopes
  • PDCD1 protein, human
  • Peptide Fragments
  • Programmed Cell Death 1 Receptor
  • Radiopharmaceuticals
  • Fluorine-18