# Bone Marrow Transplantation in Human Disease

> **NIH NIH P01** · JOHNS HOPKINS UNIVERSITY · 2022 · $2,221,695

## Abstract

Overall
Project Summary
The development 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) blood or
marrow (BMT) in patients up to at least age 75, but the outcomes of haplo BMT with PTCy, confirmed by now
many international as well as registry studies, appear similar to those seen with matched donors. Our group
has also shown that PTCy also allows the application of safe and effective mismatched unrelated donor BMT.
The reduction in GVHD complications associated with PTCy has lowered the non-relapse mortality (NRM) to
less than 10% even in the mismatched setting, removing one of the major hurdles to successful allogeneic
(allo) BMT, donor availability. PTCy has helped open up alloBMT to virtually anyone in need. With the reduction
in NRM, relapse has become by far the major complication of alloBMT. Emerging data suggest that a new non-
tolerant and non-exhausted transplanted immune system has the ability to augment the activity of most
anticancer agents, small molecule as well as immunologic. Thus, the major objective of this proposal is to
study maintenance therapy after alloBMT as a means to reduce relapse. In fact, our data, as well as from other
groups, already appear promising with such an approach, perhaps best exemplified by the impressive results
seen in FLT3/ITD AML with alloBMT and post-transplant FLT3 tyrosine kinase inhibitors despite many of these
agents showing limited activity in the non-transplant setting. There are also emerging data suggesting that
other agents generally lacking curative activity as single agents, such as rituximab in lymphomas, TKIs in Ph+
leukemias, and azaciditine for acute myeloid leukemia/myelodysplastic syndrom may demonstrate curative
activity in the post-alloBMT setting. The minimal residual disease state post-alloBMT should also optimize
novel antitumor approaches, in that they will be utilized at lowest tumor burden as well as tumor heterogeneity.
This new proposal will build on our work demonstrating that the post alloBMT setting provides a unique
environment for decreasing relapse after alloBMT. In addition, the now near universal availability of the
procedure, enabled by the favorable safety and cost profile associated with PTCy, opens up examining BMT
for unique applications. The overall goals/specific aims of this proposal are to: 1) investigate the ability of post-
transplant maintenance therapy to decrease relapse after BMT, 2) study alloBMT as a platform for enhancing
novel therapies for solid tumors, and 3) study whether alloBMT with PTCy can eliminate HIV.

## Key facts

- **NIH application ID:** 10485949
- **Project number:** 5P01CA225618-04
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** RICHARD J JONES
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $2,221,695
- **Award type:** 5
- **Project period:** 2019-05-21 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10485949

## Citation

> US National Institutes of Health, RePORTER application 10485949, Bone Marrow Transplantation in Human Disease (5P01CA225618-04). Retrieved via AI Analytics 2026-06-11 from https://api.ai-analytics.org/grant/nih/10485949. Licensed CC0.

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