Discovery of epi-enprioline as a novel drug for the treatment of vincristine resistant neuroblastoma

Wondossen Sime, Mohamed Jemaà, Yasmin Abassi, Vito Alessandro Lasorsa, Julie Bonne Køhler, Karin Hansson, Daniel Bexell, Martin Michaelis, Jindrich Cinatl, Daniel Strand, Mario Capasso, Ramin Massoumi

Research output: Contribution to journalArticlepeer-review


Neuroblastoma is a childhood solid tumour originating from undifferentiated neural progenitor cells of the sympathetic nervous system. Drug resistance of childhood cancer neuroblastoma is a serious clinical problem. In the present study, we aimed to identify novel drugs that can inhibit the growth and survival of chemoresistant neuroblastoma. High-throughput screening identified a small molecule, epi-enprioline that was able to induce apoptosis of vincristine-resistant neuroblastoma cells via the mitochondrial apoptotic pathway. Epi-enprioline reduced tumour growth in multiple preclinical models, including an orthotopic neuroblastoma patient-derived xenograft model in vivo. In summary, our data suggest that epi-enprioline can be considered as a lead compound for the treatment of vincristine-resistant neuroblastoma uncovering a novel strategy, which can be further explored as a treatment for drug-resistant neuroblastoma.

Original languageEnglish
Article number6577
Number of pages15
JournalInternational Journal of Molecular Sciences
Issue number18
Publication statusPublished - 2020

Subject classification (UKÄ)

  • Cancer and Oncology

Free keywords

  • Apoptosis
  • Chemoresistance
  • Epi-enprioline
  • Neuroblastoma
  • Vincristine


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