Wednesday, November 26, 2025 11/26/2025

Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets

Award Number: R01CA272872
ORGANIZATION: NATIONAL CANCER INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/23/2022
PERIOD OF PERFORMANCE END DATE: 08/31/2027

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Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets - PROJECT SUMMARY/ABSTRACT Infantile rhabdomyosarcoma (RMS) is a newly identified and poorly understood aggressive cancer with immature skeletal muscle properties that affects young children. Recent clinical sequencing efforts have identified a spectrum of likely biologically related gene fusions that genetically define infantile RMS: the most common being a fusion between two transcriptional co-activators with roles in normal muscle development, termed VGLL2- NCOA2. Although we know the defining oncogenic event, there are no therapies targeting the genetics of the disease. Thus, patients are treated with general chemotherapy, surgery, and radiation, often ineffectively or with harsh long-term side effects. There is a pressing need to understand the biology of infantile RMS, contextualize infantile RMS biology with other RMS sub-types, and leverage this information to delineate therapeutic targets. Progress is limited by a lack of animal models, cell lines, and PDXs to study the disease dynamics. My long-term goal is to integrate vertebrate zebrafish, mouse, cell culture models and patient samples to identify conserved genetic programs for RMS tumorigenesis and novel therapeutic strategies. Toward that end, this project’s main objective is to elucidate the underlying biology and therapeutic targets in fusion-oncogene driven infantile RMS. Our central hypothesis is that VGLL2-NCOA2 leverages normal developmental programs, including ARF6, to mediate infantile RMS tumorigenesis, and that targeting these pathways is a therapeutic opportunity. Our specific aims will address this hypothesis by: (Aim 1) Delineating VGLL2-NCOA2 structure-function requirements for tumorigenesis, (Aim 2) Establishing mechanisms of VGLL2-NCOA2 and ARF6 cooperation in rhabdomyosarcoma, and (Aim 3) Determining VGLL2-NCOA2 muscle lineage context and temporal prerequisites for tumorigenesis. Completing these studies at the interface of developmental and cancer biology will generate significant new knowledge regarding fusion-oncogene leveraging of developmental programs and will identify potential therapeutic targets. The proposed research is conceptually innovative because it leverages a cross-species comparative oncology approach to pinpoint the most important molecular drivers in a new disease, and it is experimentally innovative because it utilizes multiple new vertebrate (zebrafish and mouse) infantile RMS models developed by our group. Successful execution of this project will generate exciting data focused on delineating the basic biology of infantile rhabdomyosarcoma to directly impact and guide clinical care.


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 = $454,644 )
2025	2025	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	002	4	9/10/2025	SUPPLEMENT FOR EXPANSION	$71,772
2025	2025	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	001	4	9/8/2025	NON-COMPETING CONTINUATION	$382,872
2025	2024	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	000	3	6/6/2025	NON-COMPETING CONTINUATION	$0
2025	2024	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	003	3	9/11/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $454,644

Issue Date FY: 2024 ( Subtotal = $460,376 )
2024	2024	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	002	3	7/29/2024	SUPPLEMENT FOR EXPANSION	$68,183
2024	2024	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	001	3	7/25/2024	NON-COMPETING CONTINUATION	$363,728
2024	2024	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	000	2	5/8/2024	SUPPLEMENT FOR EXPANSION	$28,465
														Subtotal = $460,376

Issue Date FY: 2023 ( Subtotal = $375,214 )
2023	2023	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	001	2	9/11/2023	NON-COMPETING CONTINUATION	$375,214
2023	2022	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	000	1	10/19/2022	NEW	$0
														Subtotal = $375,214

Issue Date FY: 2022 ( Subtotal = $382,872 )
2022	2022	RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL	700 CHILDRENS DR	COLUMBUS	OH	43205	FRANKLIN	USA	Cancer Cause and Prevention Research	000	1	9/23/2022	NEW	$382,872
														Subtotal = $382,872

Grand Total All Awards = $1,673,106

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Understanding Infantile Rhabdomyosarcoma Biology and Therapeutic Targets

Award Number: R01CA272872

ORGANIZATION: NATIONAL CANCER INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/23/2022

PERIOD OF PERFORMANCE END DATE: 08/31/2027

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