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Expand human umbilical cord blood hematopoietic stem cells with PPAR-a agonists

Award Number: R21HL175169
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/15/2024
PERIOD OF PERFORMANCE END DATE: 07/31/2026

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Expand human umbilical cord blood hematopoietic stem cells with PPAR-a agonists - Expand human umbilical cord blood hematopoietic stem cells with PPAR-a agonists Abstract Hematopoietic stem cells (HSCs) are defined by their self-renewal potential and ability to differentiate into multiple blood lineages. Hematopoietic stem cell transplant (HSCT) is a mainstay of life-saving therapy for hematopoietic malignancies and hypoproliferative disorders. The use of umbilical cord blood (UCB)-derived HSCs provides many advantages over adult HSCs, including enhanced long-term immune recovery, decreased graft versus host disease, and availability of donors from a broad population, expanding the availability of HSCT for groups currently underrepresented in bone marrow registries. The use of haploidentical transplants addresses many of these issues, but UCB would still be a highly useful resource if UCB units contained sufficient numbers of HSCs. However, the low cell dose in most UCB units limits their use, as insufficient HSCs leads to delayed engraftment, graft failure, and severe infectious complications. Even a modest expansion of HSCs from UCB can solve many of these problems, and thus HSC expansion from UCB has remained an important goal for the field. Our goal is to use cryopreserved UCB from the NHLBI Biologic Biospecimen Repository to develop new methods to expand functional HSCs for therapeutic applications. Recent studies have achieved ex vivo expansion of HSCs using cytokine cocktails combined with small molecules, but these approaches require exposure to high concentrations of cytokines. Cytokines induce differentiation and impair the self-renewal function of primitive HSCs. HSC expansion with minimal cytokine exposure would therefore be ideal for clinical applications. Our previous studies demonstrated a combination of two inhibitors (CHIR99021 and rapamycin) maintains human and mouse long-term HSCs ex vivo in the absence of cytokines or serum. Based on this finding, we performed a high throughput screen and identified several PPAR-a agonists, which are used clinically to treat hypertriglyceridemia, that significantly expand long-term functional UCB HSC ex vivo while minimizing exposure to cytokines. In this project, we will carry out studies to optimize and implement our PPAR-a agonists-based expansion method using cryopreserved UCB stored in the NHLBI Biologic Biospecimen Repository.


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 = $127,501 )
2025	2025	CORIELL INSTITUTE FOR MEDICAL RESEARCH, INC.	403 HADDON AVE	CAMDEN	NJ	08103	CAMDEN	USA	Blood Diseases and Resources Research	000	2	8/13/2025	NON-COMPETING CONTINUATION	$127,501
														Subtotal = $127,501

Issue Date FY: 2024 ( Subtotal = $146,876 )
2024	2024	CORIELL INSTITUTE FOR MEDICAL RESEARCH, INC.	403 HADDON AVE	CAMDEN	NJ	08103	CAMDEN	USA	Blood Diseases and Resources Research	000	1	9/6/2024	NEW	$146,876
														Subtotal = $146,876

Grand Total All Awards = $274,377

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Expand human umbilical cord blood hematopoietic stem cells with PPAR-a agonists

Award Number: R21HL175169

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/15/2024

PERIOD OF PERFORMANCE END DATE: 07/31/2026

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