Immune Boost from a Tiny Fraction of a Cord Blood Transplant Graft

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

Related Publication(s)

Vander Lugt, M. T., Chen, X., Escolar, M. L., Carella, B. A., Barnum, J. L., Windreich, R. M., Hill, M. J., Poe, M., Marsh, R. A., Stanczak, H., Stenger, E. O., & Szabolcs, P. (2020). Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Advances, 4(13), 3041–3052. https://doi.org/10.1182/bloodadvances.2020001940