[HTML][HTML] Antibody-drug conjugates plus Janus kinase inhibitors enable MHC-mismatched allogeneic hematopoietic stem cell transplantation

SP Persaud, JK Ritchey, S Kim, S Lim… - The Journal of …, 2021 - Am Soc Clin Investig
SP Persaud, JK Ritchey, S Kim, S Lim, PG Ruminski, ML Cooper, MP Rettig, J Choi
The Journal of Clinical Investigation, 2021Am Soc Clin Investig
Despite the curative potential of hematopoietic stem cell transplantation (HSCT),
conditioning-associated toxicities preclude broader clinical application. Antibody-drug
conjugates (ADCs) provide an attractive approach to HSCT conditioning that minimizes
toxicity while retaining efficacy. Initial studies of ADC conditioning have largely focused on
syngeneic HSCT. However, to treat acute leukemias or induce tolerance for solid organ
transplantation, this approach must be expanded to allogeneic HSCT (allo-HSCT). Using …
Despite the curative potential of hematopoietic stem cell transplantation (HSCT), conditioning-associated toxicities preclude broader clinical application. Antibody-drug conjugates (ADCs) provide an attractive approach to HSCT conditioning that minimizes toxicity while retaining efficacy. Initial studies of ADC conditioning have largely focused on syngeneic HSCT. However, to treat acute leukemias or induce tolerance for solid organ transplantation, this approach must be expanded to allogeneic HSCT (allo-HSCT). Using murine allo-HSCT models, we show that pharmacologic Janus kinase 1/2 (JAK1/2) inhibition combined with CD45- or cKit-targeted ADCs enables robust multilineage alloengraftment. Strikingly, myeloid lineage donor chimerism exceeding 99% was achievable in fully MHC-mismatched HSCT using this approach. Mechanistic studies using the JAK1/2 inhibitor baricitinib revealed marked impairment of T and NK cell survival, proliferation, and effector function. NK cells were exquisitely sensitive to JAK1/2 inhibition due to interference with IL-15 signaling. Unlike irradiated mice, ADC-conditioned mice did not develop pathogenic graft-versus-host alloreactivity when challenged with mismatched T cells. Finally, the combination of ADCs and baricitinib balanced graft-versus-host disease and graft-versus-leukemia responses in delayed donor lymphocyte infusion models. Our allo-HSCT conditioning strategy exemplifies the promise of immunotherapy to improve the safety of HSCT for treating hematologic diseases.
The Journal of Clinical Investigation