Ruxolitinib binding to human serum albumin: bioinformatics, biochemical and functional characterization in JAK2V617F+ cell models

Sci Rep. 2019 Nov 8;9(1):16379. doi: 10.1038/s41598-019-52852-9.

Abstract

Ruxolitinib is a type I JAK inhibitor approved by FDA for targeted therapy of Philadelphia-negative myeloproliferative neoplasms (MPNs), all characterized by mutations activating the JAK2/STAT signaling pathway. Treatment with ruxolitinib improves constitutional symptoms and splenomegaly. However, patients can become resistant to treatment and chronic therapy has only a mild effect on molecular/pathologic remissions. Drugs interaction with plasma proteins, i.e. human serum albumin (HSA), is an important factor affecting the intensity and duration of their pharmacological actions. Here, the ruxolitinib recognition by the fatty acid binding sites (FAs) 1, 6, 7, and 9 of HSA has been investigated from the bioinformatics, biochemical and/or biological viewpoints. Docking simulations indicate that ruxolitinib binds to multiple sites of HSA. Ruxolitinib binds to the FA1 and FA7 sites of HSA with high affinity (Kr = 3.1 μM and 4.6 μM, respectively, at pH 7.3 and 37.0 °C). Moreover, HSA selectively blocks, in a dose dependent manner, the cytotoxic activity of ruxolitinib in JAK2V617F+ cellular models for MPN, in vitro. Furthermore this event is accompanied by changes in the cell cycle, p27Kip1 and cyclin D3 levels, and JAK/STAT signaling. Given the high plasma concentration of HSA, ruxolitinib trapping may be relevant in vivo.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Substitution
  • Binding Sites
  • Cell Line
  • Computational Biology
  • Enzyme Inhibitors / metabolism*
  • Enzyme Inhibitors / pharmacology
  • Fatty Acids / chemistry
  • Fatty Acids / metabolism
  • Humans
  • In Vitro Techniques
  • Janus Kinase 2 / antagonists & inhibitors
  • Janus Kinase 2 / genetics*
  • Janus Kinase 2 / metabolism*
  • K562 Cells
  • Kinetics
  • Molecular Docking Simulation
  • Mutant Proteins / antagonists & inhibitors
  • Mutant Proteins / genetics*
  • Mutant Proteins / metabolism*
  • Mutation, Missense
  • Myeloproliferative Disorders / blood
  • Myeloproliferative Disorders / drug therapy
  • Myeloproliferative Disorders / genetics
  • Nitriles
  • Pyrazoles / metabolism*
  • Pyrazoles / pharmacology
  • Pyrimidines
  • Serum Albumin, Human / chemistry*
  • Serum Albumin, Human / metabolism*
  • Signal Transduction
  • Thermodynamics

Substances

  • Enzyme Inhibitors
  • Fatty Acids
  • Mutant Proteins
  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib
  • JAK2 protein, human
  • Janus Kinase 2
  • Serum Albumin, Human