Pharmacodynamics and proteomic analysis of acalabrutinib therapy: similarity of on-target effects to ibrutinib and rationale for combination therapy

Leukemia. 2018 Apr;32(4):920-930. doi: 10.1038/leu.2017.321. Epub 2017 Nov 3.

Abstract

Acalabrutinib, a highly selective Bruton's tyrosine kinase inhibitor, is associated with high overall response rates and durable remission in previously treated chronic lymphocytic leukemia (CLL); however, complete remissions were limited. To elucidate on-target and pharmacodynamic effects of acalabrutinib, we evaluated several laboratory endpoints, including proteomic changes, chemokine modulation and impact on cell migration. Pharmacological profiling of samples from acalabrutinib-treated CLL patients was used to identify strategies for achieving deeper responses, and to identify additive/synergistic combination regimens. Peripheral blood samples from 21 patients with relapsed/refractory CLL in acalabrutinib phase I (100-400 mg/day) and II (100 mg BID) clinical trials were collected prior to and on days 8 and 28 after treatment initiation and evaluated for plasma chemokines, reverse phase protein array, immunoblotting and pseudoemperipolesis. The on-target pharmacodynamic profile of acalabrutinib in CLL lymphocytes was comparable to ibrutinib in measures of acalabrutinib-mediated changes in CCL3/CCL4 chemokine production, migration assays and changes in B-cell receptor signaling pathway proteins and other downstream survival proteins. Among several CLL-targeted agents, venetoclax, when combined with acalabrutinib, showed optimal complementary activity in vitro, ex vivo and in vivo in TCL-1 adoptive transfer mouse model system of CLL. These findings support selective targeting and combinatorial potential of acalabrutinib.

Publication types

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

MeSH terms

  • Adenine / analogs & derivatives
  • Adoptive Transfer / methods
  • Animals
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • B-Lymphocytes / drug effects
  • B-Lymphocytes / metabolism
  • Benzamides / administration & dosage
  • Bridged Bicyclo Compounds, Heterocyclic / administration & dosage
  • Cell Movement / drug effects
  • Chemokine CCL3 / metabolism
  • Chemokine CCL4 / metabolism
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Combined Modality Therapy / methods
  • Drug Resistance, Neoplasm / drug effects
  • Humans
  • Leukemia, Lymphocytic, Chronic, B-Cell / drug therapy*
  • Leukemia, Lymphocytic, Chronic, B-Cell / metabolism*
  • Mice
  • Piperidines
  • Protein Kinase Inhibitors / administration & dosage
  • Protein-Tyrosine Kinases / metabolism
  • Proteomics
  • Pyrazines / administration & dosage
  • Pyrazoles / administration & dosage
  • Pyrimidines / administration & dosage
  • Signal Transduction / drug effects
  • Sulfonamides / administration & dosage

Substances

  • Benzamides
  • Bridged Bicyclo Compounds, Heterocyclic
  • Chemokine CCL3
  • Chemokine CCL4
  • Piperidines
  • Protein Kinase Inhibitors
  • Pyrazines
  • Pyrazoles
  • Pyrimidines
  • Sulfonamides
  • ibrutinib
  • Protein-Tyrosine Kinases
  • acalabrutinib
  • Adenine
  • venetoclax