Sequential combinations of chemotherapeutic agents with BH3 mimetics to treat rhabdomyosarcoma and avoid resistance

Cell Death Dis. 2020 Aug 15;11(8):634. doi: 10.1038/s41419-020-02887-y.

Abstract

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in childhood and adolescence. Refractory/relapsed RMS patients present a bad prognosis that combined with the lack of specific biomarkers impairs the development of new therapies. Here, we utilize dynamic BH3 profiling (DBP), a functional predictive biomarker that measures net changes in mitochondrial apoptotic signaling, to identify anti-apoptotic adaptations upon treatment. We employ this information to guide the use of BH3 mimetics to specifically inhibit BCL-2 pro-survival proteins, defeat resistance and avoid relapse. Indeed, we found that BH3 mimetics that selectively target anti-apoptotic BCL-xL and MCL-1, synergistically enhance the effect of clinically used chemotherapeutic agents vincristine and doxorubicin in RMS cells. We validated this strategy in vivo using a RMS patient-derived xenograft model and observed a reduction in tumor growth with a tendency to stabilization with the sequential combination of vincristine and the MCL-1 inhibitor S63845. We identified the molecular mechanism by which RMS cells acquire resistance to vincristine: an enhanced binding of BID and BAK to MCL-1 after drug exposure, which is suppressed by subsequently adding S63845. Our findings validate the use of DBP as a functional assay to predict treatment effectiveness in RMS and provide a rationale for combining BH3 mimetics with chemotherapeutic agents to avoid tumor resistance, improve treatment efficiency, and decrease undesired secondary effects.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Apoptosis / drug effects
  • Apoptosis / physiology
  • Biomarkers, Pharmacological / analysis
  • Biomarkers, Pharmacological / blood
  • Cell Line, Tumor
  • Doxorubicin / pharmacology
  • Drug Resistance, Neoplasm / drug effects*
  • Humans
  • Male
  • Mice, Nude
  • Mitochondria / drug effects
  • Mitochondria / metabolism
  • Myeloid Cell Leukemia Sequence 1 Protein / drug effects
  • Myeloid Cell Leukemia Sequence 1 Protein / metabolism
  • Neoplasm Recurrence, Local / drug therapy
  • Peptide Fragments / pharmacology*
  • Proto-Oncogene Proteins / pharmacology*
  • Proto-Oncogene Proteins c-bcl-2 / metabolism
  • Pyrimidines / pharmacology
  • Rhabdomyosarcoma / drug therapy*
  • Thiophenes / pharmacology
  • Vincristine / pharmacology
  • Xenograft Model Antitumor Assays
  • bcl-X Protein / drug effects
  • bcl-X Protein / metabolism

Substances

  • Antineoplastic Agents
  • Bax protein (53-86)
  • Biomarkers, Pharmacological
  • MCL1 protein, human
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Peptide Fragments
  • Proto-Oncogene Proteins
  • Proto-Oncogene Proteins c-bcl-2
  • Pyrimidines
  • S63845
  • Thiophenes
  • bcl-X Protein
  • Vincristine
  • Doxorubicin