Many inherited non-malignant disorders can be cured or alleviated in children by hematopoietic stem cell transplant (HSCT). Umbilical cord blood (UCB) units are a rapidly available stem cell source and offer considerable flexibility in human leukocyte antigen (HLA) matching, making UCB transplantation an option for more than 95% of pediatric patients regardless of their ethnic background. Although reduced-intensity conditioning (RIC) regimens promise decreased treatment-related morbidity and mortality, graft failure and infections have limited their use in chemotherapy-naïve patients undergoing unrelated UCB transplant compared to patients who have received prior chemotherapy.
Description
University of Pittsburgh researchers have designed a UCB transplant regimen that is safe and suitable for every patient suffering from nonmalignant disorders—excluding chromosomal breakage syndromes or SCID— who may benefit from less-intensive conditioning regimens. This regimen consists of a delayed infusion of a small fraction (3-5%) of the original cord blood unit, previously set aside and re-cryopreserved without other manipulation. This “immune boost” supports T cell reconstitution and associated immune function, improving recipient response to HSCT.
Applications
• Primary immunodeficiency diseases
• Hemoglobinopathies
• Bone marrow failure syndromes
• Inborn errors of metabolism
• Oher non-malignant disorders
Advantages
• Prevents graft failure and aids immune reconstitution
• Obviates the need for pharmacokinetic dose adjustments, rendering it applicable even in resource limited centers and countries.
Invention Readiness
In vivo data
IP Status
https://patents.google.com/patent/US20230210911A1
