Wednesday, October 1, 2025 10/1/2025

BMT Core - MSK

Award Number: UG1HL069315
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/30/2001
PERIOD OF PERFORMANCE END DATE: 06/30/2031

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BMT Core - MSK - PROJECT SUMMARY/ABSTRACT The mission of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) is to “evaluate promising therapeutic approaches in multi-institutional clinical trials to improve the outcomes of blood and bone marrow transplantation and other cellular therapies for patients facing life-threatening blood disorders.” Memorial Sloan Kettering Cancer Center (MSK) is an NCl-designated Comprehensive Cancer Center with one of the largest BMT programs in the country. The BMT program at MSK, founded in 1973 and divided into pediatric and adult services, is a FACT-accredited program for both hematopoietic cell transplant (HCT) and immune effector cell (IEC) therapy. MSK has been a Core Clinical Center (CCC) of the BMT CTN since its inception. Dr. Perales (Service Chief, Adult BMT; PI, MSK BMT CTN), Dr. Giralt (Deputy Head, Division of Hematologic Oncology; Co- I), Dr. Boelens (Service Chief, Pediatric BMT and Cell Therapy; Co-I), and all members of the Adult and Pediatric BMT and Cellular Therapy Services consider participation in the BMT CTN an integral part of their mission. In this proposal, we detail our ongoing commitment to the success of the Network through an oversight structure that includes a BMT CTN MSK Executive Committee, a BMT CTN Protocol Management Team, and a BMT CTN Internal Advisory Board, which leverage senior leadership of the Division of Hematologic Oncology and Pediatric BMT Service as well as outstanding scientists and investigators at MSK. As part of our continued commitment to Network leadership, we propose a multicenter phase II clinical trial in adults and children with severe sickle cell disease (SCD), in which we will evaluate a pharmacologic immunosuppressive strategy prior to reduced toxicity myeloablative mismatched unrelated (MMUD) HCT, using post-transplant cyclophosphamide (PTCY)- based graft-versus-host disease (GvHD) prophylaxis. The trial will employ a novel platform to mitigate the risk of graft failure in a patient population at high risk for allograft rejection and treatment-related complications, as well as pharmacokinetic-based models to guide individualized dosing of fludarabine, busulfan, and rabbit ATG for optimal drug exposure. We propose 3 specific aims: (1) Demonstrate MSK has the clinical and translational expertise, novel cell and gene therapy capabilities, and early- and late-phase trial experience to fulfill the obligations of a BMT CTN CCC and advance the Network’s mission and goals; (2) Complete a phase II study in which patients with severe SCD will receive an HCT from an MMUD donor with PTCY-based GvHD prophylaxis (the primary endpoint will be to estimate primary graft failure at 1-year post-HCT); and (3) Describe how MSK’s operational and scientific approaches to conducting HCT trials will support the mission and activities of the BMT CTN. The proposed study is highly relevant to the BMT CTN, as it addresses an unmet need for a well-tolerated alternative donor platform with stable engraftment in patients with SCD, and is in line with the goal of the BMT CTN to prioritize clinical studies that advance HCT approaches for non-malignant blood diseases.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $264,000 )
2025	2025	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	001	25	7/16/2025	NON-COMPETING CONTINUATION	$264,000
2025	2023	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	23	2/18/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $264,000

Issue Date FY: 2024 ( Subtotal = $264,000 )
2024	2024	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	004	24	9/11/2024	COMPETING CONTINUATION	-$1,500
2024	2024	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	003	24	8/15/2024	COMPETING CONTINUATION	$265,500
2024	2023	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	002	23	8/8/2024	NON-COMPETING CONTINUATION	$0
2024	2023	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVENUE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	23	2/10/2024	NON-COMPETING CONTINUATION	$0
2024	2023	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVENUE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	001	23	3/5/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $264,000

Issue Date FY: 2023 ( Subtotal = $194,309 )
2023	2023	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	23	7/7/2023	NON-COMPETING CONTINUATION	$194,309
														Subtotal = $194,309

Issue Date FY: 2022 ( Subtotal = $197,052 )
2022	2022	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	22	7/1/2022	NON-COMPETING CONTINUATION	$197,052
														Subtotal = $197,052

Issue Date FY: 2021 ( Subtotal = $200,089 )
2021	2021	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	21	6/18/2021	NON-COMPETING CONTINUATION	$200,089
														Subtotal = $200,089

Issue Date FY: 2020 ( Subtotal = $204,275 )
2020	2020	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	20	6/9/2020	NON-COMPETING CONTINUATION	$204,275
														Subtotal = $204,275

Issue Date FY: 2019 ( Subtotal = $208,408 )
2019	2019	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	19	5/17/2019	NON-COMPETING CONTINUATION	$208,408
														Subtotal = $208,408

Issue Date FY: 2018 ( Subtotal = $198,989 )
2018	2018	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	002	18	6/19/2018	NON-COMPETING CONTINUATION	$192,604
2018	2017	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	003	17	6/25/2018	COMPETING CONTINUATION	$6,385
2018	2017	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	001	17	5/1/2018	COMPETING CONTINUATION	-$590
2018	2017	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	17	1/3/2018	COMPETING CONTINUATION	$590
														Subtotal = $198,989

Issue Date FY: 2017 ( Subtotal = $193,800 )
2017	2017	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	001	17	8/22/2017	COMPETING CONTINUATION	$0
2017	2017	SLOAN-KETTERING INSTITUTE FOR CANCER RESEARCH	1275 YORK AVE	NEW YORK	NY	10065	NEW YORK	USA	Blood Diseases and Resources Research	000	17	7/27/2017	COMPETING CONTINUATION	$193,800
														Subtotal = $193,800

Grand Total All Awards = $1,924,922

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BMT Core - MSK

Award Number: UG1HL069315

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/30/2001

PERIOD OF PERFORMANCE END DATE: 06/30/2031

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