Anti-CD117 conditioning for islet and hematopoietic cell transplantation in type 1 diabetes

Bhagchandani P, Ramos SA, Rodriguez B, et al. Curing Autoimmune Diabetes in Mice With Islet and Hematopoietic Cell Transplantation After CD117 Antibody-Based Conditioning. Journal of Clinical Investigation. 2025; (doi: 10.1172/JCI190034).

Scientists developed a conditioning protocol that, followed by allogeneic bone marrow transplantation and islet transplantation, produces a favorable mix of host and donor hematopoietic stem cells with the potential to cure autoimmune type 1 diabetes. The regimen involves antibody-based targeting of CD117 combined with baricitinib-mediated inhibition of JAK-STAT signaling. In murine models, the resulting mixed chimerism improved tolerance of allografted organs, prevented graft-versus-host disease (GVHD), supported a healthy chimera, and reversed established type 1 diabetes in non-obese mice. The non-toxic, non-myeloablative regimen, which does not rely on chemotherapy, also prevented onset of full-blown disease in prediabetic non-obese mice. Importantly, the novel conditioning regimen succeeded without chronic immune suppression or GVHD.

Read More