Pediatric matched stem cell and organ transplant therapy

About the Project

More than 1500 children per year in the US receive organ transplants. While short-term outcomes have improved, long-term outcomes remain dismal. With the rare exception of monozygotic twins as donors, patients need lifelong immunosuppression. Even with this treatment, up to half of patients require a second transplant. 

Dr. Bertaina’s group has pioneered translational approaches that prevent both rejection and the need for post-transplant immunosuppression. Specifically, patients were given abT-cell/CD19 B-cell depleted hematopoietic stem cells (HSC) prior to kidney transplantation, both from the same donor, thus establishing a donor-derived immune system incapable of graft rejection and removing any need for chronic immunosuppression. 

With funding and clinical research coordination from the IMA, Dr. Bertaina initiated a phase1b/2a clinical trial to study the safety and efficacy of performing TCRab+ T-cell/CD19+ B-cell depleted (abdepleted) HSC transplant prior to kidney transplant using the same donor. As a result of the IMA’s support, Dr. Bertaina received a $12 million grant from the California Institute for Regenerative Medicine to enroll more patients and expand the diseases that are treated with her approach. The study will determine the ability of abdepleted-HSC transplantation to permit the withdrawal of immunosuppression following organ transplantation.

Recent News

Stanford Medicine teams awarded $18 million to improve kidney transplant and gene-editing techniques (Stanford Medicine News)