Identification of hnRNP-A1 as a pharmacodynamic biomarker of type I PRMT inhibition in blood and tumor tissues

Sci Rep. 2020 Dec 17;10(1):22155. doi: 10.1038/s41598-020-78800-6.

Abstract

Arginine methylation has been recognized as a post-translational modification with pleiotropic effects that span from regulation of transcription to metabolic processes that contribute to aberrant cell proliferation and tumorigenesis. This has brought significant attention to the development of therapeutic strategies aimed at blocking the activity of protein arginine methyltransferases (PRMTs), which catalyze the formation of various methylated arginine products on a wide variety of cellular substrates. GSK3368715 is a small molecule inhibitor of type I PRMTs currently in clinical development. Here, we evaluate the effect of type I PRMT inhibition on arginine methylation in normal human peripheral blood mononuclear cells and utilize a broad proteomic approach to identify type I PRMT substrates. This work identified heterogenous nuclear ribonucleoprotein A1 (hnRNP-A1) as a pharmacodynamic biomarker of type I PRMT inhibition. Utilizing targeted mass spectrometry (MS), methods were developed to detect and quantitate changes in methylation of specific arginine residues on hnRNP-A1. This resulted in the development and validation of novel MS and immune assays useful for the assessment of GSK3368715 induced pharmacodynamic effects in blood and tumors that can be applied to GSK3368715 clinical trials.

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacokinetics*
  • Antineoplastic Agents / therapeutic use
  • Antineoplastic Agents, Immunological / chemistry
  • Antineoplastic Agents, Immunological / pharmacokinetics
  • Antineoplastic Agents, Immunological / pharmacology
  • Arginine / metabolism
  • Biomarkers*
  • Cells, Cultured
  • Chromatography, Liquid
  • Drug Monitoring
  • Enzyme Activation
  • Enzyme Inhibitors / pharmacokinetics
  • Enzyme Inhibitors / therapeutic use
  • Gene Expression Regulation, Neoplastic / drug effects
  • Heterogeneous Nuclear Ribonucleoprotein A1 / blood
  • Heterogeneous Nuclear Ribonucleoprotein A1 / metabolism*
  • Humans
  • Leukocytes, Mononuclear / drug effects
  • Leukocytes, Mononuclear / metabolism
  • Mass Spectrometry
  • Methylation
  • Mice
  • Molecular Targeted Therapy
  • Neoplasms / blood
  • Neoplasms / drug therapy
  • Neoplasms / metabolism
  • Protein-Arginine N-Methyltransferases / antagonists & inhibitors*
  • Protein-Arginine N-Methyltransferases / genetics
  • Repressor Proteins / antagonists & inhibitors*
  • Repressor Proteins / genetics
  • Substrate Specificity

Substances

  • Antineoplastic Agents
  • Antineoplastic Agents, Immunological
  • Biomarkers
  • Enzyme Inhibitors
  • Heterogeneous Nuclear Ribonucleoprotein A1
  • Repressor Proteins
  • hnRNPA1 protein, human
  • Arginine
  • PRMT1 protein, human
  • Protein-Arginine N-Methyltransferases