# BMT Core- University of Michigan Core Clinical Consortium for the BMT Clinical Trials Network (BMT CTN)

> **NIH NIH UG1** · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2024 · $255,457

## Abstract

ABSTRACT
The University of Michigan Consortium brings together physician scientists from the two major cancer centers in
the State of Michigan: the University of Michigan in Ann Arbor and the Karmanos Cancer Institute in Detroit. In
addition, both the University of Michigan (U-M) and the Karmanos Cancer Institute (KCI) are affiliated with
pediatric hospitals—U-M with Mott Children’s Hospital and KCI with the Children’s Hospital of Michigan (CHM).
The U-M Consortium has been a strong supporter of BMT CTN Network activities throughout the current grant
cycle, with more than 300 subjects (and counting) enrolled into CTN trials since 2017. The U-M Consortium will
have an emphasis on non-malignant blood disorders, with more than 2,000 patients with hemoglobinopathies
and immunologic disorders currently being followed. The organizational structure pairs hematologists with
transplant physicians who have expertise in these conditions. The Consortium has deep experience in translating
early-phase clinical trials to multicenter studies, has comprehensive biorepositories as foundations for its
programs, and has a team of physician-scientists (Dr. Greg Yanik, Dr. Sung Won Choi, Dr. Joseph Uberti, and
Dr. John Magenau) with an established track record of academic success. This research proposal examines a
new paradigm in bone marrow transplantation, focusing on inhibition of epigenetic pathways as a potential
mechanism both to prevent transplant-related organ toxicity and to mitigate graft versus host disease (GVHD).
The role of histone deacetylase inhibitors has been an area of intense research at U-M for the past two decades,
with pre-clinical studies at both cancer centers having led to five multicenter clinical trials over this period. NIH-
funded clinical trials using a histone deacetylase inhibitor (Vorinostat) have been performed in recipients of
reduced and myeloablative conditioning, in a variety of donor cell sources (sibling, unrelated, haplo-identical)
and in both adults and pediatric patients. The proposed project will study the role of epigenetic modifiers in
patients undergoing transplant for non-malignant blood disorders, including those with hemoglobinopathies or
immune-hematologic disorders. The study will use a novel composite endpoint: one-year GVHD-free, event-free
survival, with events defined as death due to any cause, graft rejection/failure, or second HCT, whichever occurs
first. GVHD will be defined as grade III-IV acute GVHD or chronic GVHD requiring systemic immune suppression.
Secondary endpoints include overall survival, primary and secondary graft failure, chimerism and infectious
events. This trial will not only mitigate the severity of GVHD in patients with non-malignant blood disorders, but
also advance the understanding of the biology of epigenetic modifiers in HCT. The U-M Consortium is committed
to the scientific agenda of the BMT CTN and fully anticipates that this research strategy will provide new insights
into reducing...

## Key facts

- **NIH application ID:** 10936878
- **Project number:** 2UG1HL069330-24
- **Recipient organization:** UNIVERSITY OF MICHIGAN AT ANN ARBOR
- **Principal Investigator:** SUNG WON CHOI
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $255,457
- **Award type:** 2
- **Project period:** 2001-09-30 → 2031-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10936878

## Citation

> US National Institutes of Health, RePORTER application 10936878, BMT Core- University of Michigan Core Clinical Consortium for the BMT Clinical Trials Network (BMT CTN) (2UG1HL069330-24). Retrieved via AI Analytics 2026-06-11 from https://api.ai-analytics.org/grant/nih/10936878. Licensed CC0.

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