Reprogrammed Cells in Pediatric HSCT



The handling and reprogramming of cells and the following development of CART-cell therapies lead the area of pediatric stem cell transplantation to another level. This area is constantly being changed to include bioengineering. Bioengineering has been solving problems such as the absence of a readily available HLA-matched donor. Nowadays, there is a potential new standard of treatment for pediatric patients with high-risk hematologic malignancies and nonmalignant disorders who need hematopoietic stem cell transplantation but do not have access to a HLA-matched donor, and the modern alternative is to use bioengineering to improve the current alternative for these patients, which is HLA-haplo-HSCT (human leukocyte antigen-haploidentical HSCT). The major challenge of HLA-haplo-HSCT is the intense bidirectional alloreactivity that leads to high incidences of graft rejection and GvHD, and the modern techniques using adjunct bioengineered T- or B-cells have been reducing that undesired reactions [63–66].


  1. 63.
    Ruggeri A, Merli P, Kapoor N, et al. Administration of BPX-501 cells following Aβ T and B-cell-depleted HLA haploidentical HSCT (haplo-HSCT) in children with acute leukemias (AL). Biol Blood Marrow Transplant. 2019;25:S7–S75.Google Scholar
  2. 64.
    National Institutes of Health. Inxight: drugs. Rimiducid. (Nov 5, 2019).
  3. 65.
    Kingwell K. CAR T therapies drive into new terrain. Nat Rev Drug Discov. 2017;16(5):301–4. Scholar
  4. 66.
    Fuchs EJ, Luznik L. HLA-haploidentical hematopoietic cell transplantation. UpToDate. 2019. (Nov 5, 2019).
  5. 67.
    Bellicum Pharmaceuticals. Investor presentation. (Nov 5, 2019).

Copyright information

© Springer Nature Switzerland AG 2020

Authors and Affiliations

  1. 1.CuritibaBrazil

Personalised recommendations