Assessing single-stranded oligonucleotide drug-induced effects in vitro reveals key risk factors for thrombocytopenia

PLoS One. 2017 Nov 6;12(11):e0187574. doi: 10.1371/journal.pone.0187574. eCollection 2017.

Abstract

Single-stranded oligonucleotides (ON) comprise a promising therapeutic platform that enables selective modulation of currently undruggable targets. The development of novel ON drug candidates has demonstrated excellent efficacy, but in certain cases also some safety liabilities were reported. Among them are events of thrombocytopenia, which have recently been evident in late stage trials with ON drugs. The underlying mechanisms are poorly understood and the risk for ON candidates causing such events cannot be sufficiently assessed pre-clinically. We investigated potential thrombocytopenia risk factors of ONs and implemented a set of in vitro assays to assess these risks. Our findings support previous observations that phosphorothioate (PS)-ONs can bind to platelet proteins such as platelet collagen receptor glycoprotein VI (GPVI) and activate human platelets in vitro to various extents. We also show that these PS-ONs can bind to platelet factor 4 (PF4). Binding to platelet proteins and subsequent activation correlates with ON length and connected to this, the number of PS in the backbone of the molecule. Moreover, we demonstrate that locked nucleic acid (LNA) ribosyl modifications in the wings of the PS-ONs strongly suppress binding to GPVI and PF4, paralleled by markedly reduced platelet activation. In addition, we provide evidence that PS-ONs do not directly affect hematopoietic cell differentiation in culture but at higher concentrations show a pro-inflammatory potential, which might contribute to platelet activation. Overall, our data confirm that certain molecular attributes of ONs are associated with a higher risk for thrombocytopenia. We propose that applying the in vitro assays discussed here during the lead optimization phase may aid in deprioritizing ONs with a potential to induce thrombocytopenia.

MeSH terms

  • Bone Marrow / drug effects
  • Enzyme-Linked Immunosorbent Assay
  • Flow Cytometry
  • Humans
  • Oligonucleotides / adverse effects*
  • Oligonucleotides / chemistry
  • Oligonucleotides / metabolism
  • Platelet Activation / drug effects
  • Platelet Membrane Glycoproteins / metabolism
  • Protein Binding
  • Risk Factors
  • Surface Plasmon Resonance
  • Thrombocytopenia / chemically induced*

Substances

  • Oligonucleotides
  • Platelet Membrane Glycoproteins
  • locked nucleic acid
  • platelet membrane glycoprotein VI

Grants and funding

All authors are employees of F. Hoffmann-La Roche Ltd. at the Roche Innovation Center Basel, Switzerland, or Roche Innovation Center Copenhagen A/S, Denmark. The funder provided support in the form of salaries for authors (SS, ABR, MW, AD, CBL, YT, CMG, SH, EK, CP, JCR, TS, AR), but did not have any additional role in the study design, data collection and analysis, decision to publish, or preparation of the manuscript. The specific roles of these authors are articulated in the ‘author contributions’ section.