# Novel Cord Blood-Derived Cellular Therapies

> **NIH NIH P01** · UNIVERSITY OF TX MD ANDERSON CAN CTR · 2021 · $2,569,848

## Abstract

OVERALL PROGRAM SUMMARY
Umbilical cord blood (CB) is a valuable source of hematopoietic progenitor cells (HPCs) for the
treatment of a broad range of malignant and nonmalignant disorders in individuals who otherwise
would lack a suitable HLA-matched donor. Ultimately, the future of CB transplant will rest on how well
certain limitations of this procedure are addressed, including the relatively low numbers of HPCs in CB
collections; the slower times to engraftment and immune recovery; and the apparent reduced ability
of CB cells to home to bone marrow. Hence, the initial long-term goal of this P01 grant-supported
research was to enhance the efficacy of CB transplant for hematologic malignancies. Although still
focused on several of the program's original objectives, this competitive renewal application now
encompasses CB-based therapies with the potential to extend the benefits of our research to
nontransplant settings. This emerging emphasis has led to a change in the title of the proposal, from
“Improving Cord Blood Transplantation” to “Novel Cord Blood Cellular Therapies,” and to a new overall
central hypothesis: that CB-derived HPCs can be modified in diverse ways ex vivo to improve the
outcomes not only of CB transplant but also of cellular therapies in general. The investigators chosen
for this renewal effort represent four research projects and three Core services, all with stellar records
of collaborative investigation in transplant medicine, cell therapy, immunology, and adult and pediatric
hematology – predicting synergistic interactions in pursuit of the specific aims outlined here. Project
1 (E. Shpall, R. Sackstein) seeks to further enhance CB engraftment by combining, in MSC-mediated
expansion with exofucosylation of CB cells to boost HPC numbers and homing capacity, and to lessen
or abrogate GvHD using CB tissue-derived MSCs. In Project 2 (C. Bollard, D. Nixon), the safety,
feasibility and efficacy of infusing ex vivo-expanded CB-derived T cells targeting four viruses will be
tested in patients undergoing CB transplant for resistant hematologic malignancies, The investigators
will also determine whether the highly effective virus-specific T-cell (VST) strategy can be extended to
the prevention of HIV post-transplant. Project 3 (K. Rezvani, J. Orange) plans to prospectively
assess an intriguing hypothesis that the persistence and activity of CB-natural killer cells against
lymphoid malignancies can be enhanced, without undue toxicity, by genetically modifying these cells
to express a CD19-specific CAR and IL-15. Finally, Project 4 (J. Molldrem, G. Al-Atrash, Q. Ma) will
continue to develop a novel strategy to lower relapse rates in the myeloid leukemias by redirecting T-
cell specificity to the HLA-A2-restricted PR1 epitope, using both animal models and human trials.

## Key facts

- **NIH application ID:** 10247034
- **Project number:** 5P01CA148600-10
- **Recipient organization:** UNIVERSITY OF TX MD ANDERSON CAN CTR
- **Principal Investigator:** Catherine M. Bollard
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $2,569,848
- **Award type:** 5
- **Project period:** 2011-09-22 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10247034

## Citation

> US National Institutes of Health, RePORTER application 10247034, Novel Cord Blood-Derived Cellular Therapies (5P01CA148600-10). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10247034. Licensed CC0.

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