Drug intelligence / Profile preview

autologous hematopoietic stem cell transplant

Development stage
Phase 2
Lead developer
University Health Network
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

This experimental cell therapy approach, evaluated in the ASCOTT (Autologous Hematopoietic Stem Cell Transplantation for Allogeneic Organ Transplant Tolerance) trial, aims to induce immune tolerance in liver transplant recipients. Led by Dr. Gary Levy at the University Health Network and Dr. Harold Atkins at The Ottawa Hospital, the procedure involves the collection of a patient's own CD34+ hematopoietic stem cells (HSCs) followed by a conditioning regimen of immune ablation using busulfan, cyclophosphamide, and rabbit anti-thymocyte globulin (ATG). The cryopreserved autologous stem cells are then reinfused to rebuild and "re-educate" the recipient's immune system. The primary therapeutic goal is to delete alloreactive T cell clones and promote the expansion of regulatory T cells (Tregs), thereby establishing operational tolerance to the transplanted liver and eliminating the need for lifelong immunosuppressive medications.

Other names
Autologous HSCTASCOTT trial therapyCD34+ hematopoietic stem cell transplant

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