# Translational Research in Blood and Marrow Transplantation

> **NIH NIH UG1** · JOHNS HOPKINS UNIVERSITY · 2020 · $149,251

## Abstract

Project Summary
Our program has focused on the translation of transplantation biology from the laboratory to the clinic to
improve the outcome of blood and marrow transplantation (BMT). The development high-dose post-transplant
cyclophosphamide (PTCy) to modulate GVHD is a prime example of our group’s successful translational
research. Not only does PTCy allow safe haploidentical (haplo) BMT in patients up to at least age 75, but
results are now similar to those seen with matched donors. Accordingly, the ability to successfully utilize
mismatched donors now permits virtually anyone in need of BMT to undergo the procedure. In addition to
moving allogeneic BMT into non-malignant indications, our group has been concentrating on reducing relapses
after allogeneic BMT for malignancy. With the reduction in non-relapse mortality, relapse has become by far
the major complication of allogeneic BMT. Emerging data suggest that a new non-tolerant and non-exhausted
transplanted immune system may enhance the activity of most anticancer agents. Post-allogeneic BMT
maintenance therapy is already generating encouraging results; this is perhaps best exemplified by the
impressive results seen in FLT3/ITD AML patients who received allogeneic BMT and post-transplant FLT3
tyrosine kinase inhibitors, despite these agents showing limited activity in the non-transplant setting. The
minimal residual disease (MRD) state post-allogeneic BMT may also optimize antitumor approaches, in that
they will be utilized at lowest tumor burden as well as tumor heterogeneity. Thus, we hypothesize that post-
transplant maintenance may be the best strategy for decreasing relapse rates after allogeneic BMT. However,
although our group and others have had successes in piloting novel clinical post-transplant maintenance
strategies, it is difficult for a single program to carry out larger trials with these promising approaches. Thus,
with the rapid development of promising new therapies, a formal clinical trials network that can rapidly and
efficiently conduct multi-center trials in BMT is critically important. Our specific objectives as a Core Clinical
Center are to: 1) Participate in multicenter trials through the BMT CTN, and 2) Propose a phase 2, multicenter,
adaptively randomized trial to screen multiple novel regimens for treating relapse after allogeneic BMT for
AML/MDS. An important goal will be to match individual regimens to specific disease subtypes. The ultimate
goal will be to move promising approaches into post-transplant maintenance to prevent relapse.
Despite remarkable progress in hematologic malignancies with most patients now responding to initial therapy,
the majority eventually relapse and die of the disease. Allogeneic blood or marrow transplantation remains the
only curative option for many of these patients, but relapse remains the most common cause for failure even in
this setting. Novel approaches that reduce relapse without increasing toxicity are needed.

## Key facts

- **NIH application ID:** 9999020
- **Project number:** 5UG1HL069310-20
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** RICHARD J JONES
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $149,251
- **Award type:** 5
- **Project period:** 2001-09-30 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9999020

## Citation

> US National Institutes of Health, RePORTER application 9999020, Translational Research in Blood and Marrow Transplantation (5UG1HL069310-20). Retrieved via AI Analytics 2026-06-10 from https://api.ai-analytics.org/grant/nih/9999020. Licensed CC0.

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