Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

Nat Commun. 2019 Feb 6;10(1):617. doi: 10.1038/s41467-018-08201-x.

Abstract

Hematopoietic stem cell transplantation (HSCT) is a curative therapy for blood and immune diseases with potential for many settings beyond current standard-of-care. Broad HSCT application is currently precluded largely due to morbidity and mortality associated with genotoxic irradiation or chemotherapy conditioning. Here we show that a single dose of a CD117-antibody-drug-conjugate (CD117-ADC) to saporin leads to > 99% depletion of host HSCs, enabling rapid and efficient donor hematopoietic cell engraftment. Importantly, CD117-ADC selectively targets hematopoietic stem cells yet does not cause clinically significant side-effects. Blood counts and immune cell function are preserved following CD117-ADC treatment, with effective responses by recipients to both viral and fungal challenges. These results suggest that CD117-ADC-mediated HSCT pre-treatment could serve as a non-myeloablative conditioning strategy for the treatment of a wide range of non-malignant and malignant diseases, and might be especially suited to gene therapy and gene editing settings in which preservation of immunity is desired.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bone Marrow / drug effects
  • Bone Marrow Transplantation
  • Candida albicans / pathogenicity
  • Cell Death
  • Cell Line
  • Female
  • Genetic Therapy
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cells / drug effects*
  • Humans
  • Immunoconjugates / administration & dosage
  • Immunoconjugates / pharmacology*
  • Mice
  • Mice, Inbred C57BL
  • Neoplasms
  • Proto-Oncogene Proteins c-kit / immunology*
  • Tissue Donors

Substances

  • Immunoconjugates
  • Proto-Oncogene Proteins c-kit