Targeting SAMHD1 with the Vpx protein to improve cytarabine therapy for hematological malignancies

Nat Med. 2017 Feb;23(2):256-263. doi: 10.1038/nm.4265. Epub 2017 Jan 9.

Abstract

The cytostatic deoxycytidine analog cytarabine (ara-C) is the most active agent available against acute myelogenous leukemia (AML). Together with anthracyclines, ara-C forms the backbone of AML treatment for children and adults. In AML, both the cytotoxicity of ara-C in vitro and the clinical response to ara-C therapy are correlated with the ability of AML blasts to accumulate the active metabolite ara-C triphosphate (ara-CTP), which causes DNA damage through perturbation of DNA synthesis. Differences in expression levels of known transporters or metabolic enzymes relevant to ara-C only partially account for patient-specific differential ara-CTP accumulation in AML blasts and response to ara-C treatment. Here we demonstrate that the deoxynucleoside triphosphate (dNTP) triphosphohydrolase SAM domain and HD domain 1 (SAMHD1) promotes the detoxification of intracellular ara-CTP pools. Recombinant SAMHD1 exhibited ara-CTPase activity in vitro, and cells in which SAMHD1 expression was transiently reduced by treatment with the simian immunodeficiency virus (SIV) protein Vpx were dramatically more sensitive to ara-C-induced cytotoxicity. CRISPR-Cas9-mediated disruption of the gene encoding SAMHD1 sensitized cells to ara-C, and this sensitivity could be abrogated by ectopic expression of wild-type (WT), but not dNTPase-deficient, SAMHD1. Mouse models of AML lacking SAMHD1 were hypersensitive to ara-C, and treatment ex vivo with Vpx sensitized primary patient-derived AML blasts to ara-C. Finally, we identified SAMHD1 as a risk factor in cohorts of both pediatric and adult patients with de novo AML who received ara-C treatment. Thus, SAMHD1 expression levels dictate patient sensitivity to ara-C, providing proof-of-concept that the targeting of SAMHD1 by Vpx could be an attractive therapeutic strategy for potentiating ara-C efficacy in hematological malignancies.

Publication types

  • Video-Audio Media

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Animals
  • Antimetabolites, Antineoplastic / pharmacology*
  • Antimetabolites, Antineoplastic / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / pharmacology*
  • Apoptosis / drug effects*
  • Arabinofuranosylcytosine Triphosphate / metabolism
  • Child
  • Child, Preschool
  • Cytarabine / pharmacology*
  • Cytarabine / therapeutic use
  • Disease Models, Animal
  • Female
  • Humans
  • In Vitro Techniques
  • Infant
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / metabolism
  • Male
  • Mice
  • Molecular Targeted Therapy
  • Monomeric GTP-Binding Proteins / drug effects*
  • Monomeric GTP-Binding Proteins / metabolism
  • Prognosis
  • SAM Domain and HD Domain-Containing Protein 1
  • Viral Regulatory and Accessory Proteins / pharmacology*

Substances

  • Antimetabolites, Antineoplastic
  • VPX protein, Simian immunodeficiency virus
  • Viral Regulatory and Accessory Proteins
  • Cytarabine
  • Arabinofuranosylcytosine Triphosphate
  • SAM Domain and HD Domain-Containing Protein 1
  • SAMHD1 protein, human
  • Monomeric GTP-Binding Proteins