Wednesday, July 2, 2025 7/2/2025

Identifying Novel Therapeutics for High Risk Medulloblastoma Patients

Award Number: K01NS119351
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP
PERIOD OF PERFORMANCE START DATE: 01/01/2021
PERIOD OF PERFORMANCE END DATE: 12/31/2025

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Identifying Novel Therapeutics for High Risk Medulloblastoma Patients - ABSTRACT Among pediatric brain tumors, medulloblastoma is the most common form. While most children with medulloblastoma can be cured, ~25% of these kids will die, and those that survive will live with severe long-term side effects as a result of the devastating effects of current therapies on the developing brain. In patients with medulloblastoma, prognosis depends heavily on the molecular makeup of the tumor. New genomic approaches have enabled to classify medulloblastoma into molecular subgroups, allowing physicians to better predict patient outcome and design treatment adequately. Among these subgroups, patients having alterations in the developmental pathway SONIC HEDGEHOG (SHH), along with mutations in the tumor suppressor gene P53 have a poor outcome. These tumors are resistant to therapy what results in rapid tumor relapse, which almost uniformly leads to patient death. Loss of P53 activity results in significant genomic instability and, consequently, large scale alterations in the signaling networks that drive cellular proliferation, survival, differentiation and/or stemness are created. Subsequently, a smaller number of these networks are selected for during the tumorigenic process and behave as tumor drivers. Here, I hypothesize that the growth of TRP53 mutant medulloblastoma will be abrogated by targeting this P53-specific set of signaling pathways. My previous work allowed me to identify two distinct drivers of these tumors, controlling independently tumor growth and propagation. The goal of this proposal is to understand how these identified tumor drivers control overall medulloblastoma viability, and to further validate them in murine and human derived TRP53 mutant SHH medulloblastoma models.


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 = $216,345 )
2025	2025	THE MEDICAL UNIVERSITY OF SOUTH CAROLINA	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	5	12/24/2024	NON-COMPETING CONTINUATION	$216,345
														Subtotal = $216,345

Issue Date FY: 2024 ( Subtotal = $216,345 )
2024	2024	THE MEDICAL UNIVERSITY OF SOUTH CAROLINA	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	4	1/26/2024	NON-COMPETING CONTINUATION	$216,345
2024	2023	THE MEDICAL UNIVERSITY OF SOUTH CAROLINA	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	12/5/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $216,345

Issue Date FY: 2023 ( Subtotal = $216,345 )
2023	2023	MEDICAL UNIVERSITY OF SOUTH CAROLINA THE	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	3/7/2023	NON-COMPETING CONTINUATION	$216,345
														Subtotal = $216,345

Issue Date FY: 2022 ( Subtotal = $216,345 )
2022	2022	MEDICAL UNIVERSITY OF SOUTH CAROLINA THE	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	12/23/2021	NON-COMPETING CONTINUATION	$216,345
														Subtotal = $216,345

Issue Date FY: 2021 ( Subtotal = $216,345 )
2021	2021	MEDICAL UNIVERSITY OF SOUTH CAROLINA THE	179 ASHLEY AVE	CHARLESTON	SC	29425	CHARLESTON	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	12/25/2020	NEW	$216,345
														Subtotal = $216,345

Grand Total All Awards = $1,081,725

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Identifying Novel Therapeutics for High Risk Medulloblastoma Patients

Award Number: K01NS119351

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: TRAINING/TRAINEESHIP

PERIOD OF PERFORMANCE START DATE: 01/01/2021

PERIOD OF PERFORMANCE END DATE: 12/31/2025

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