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Personalized neuroblastoma vaccines

Award Number: U01CA281881
ORGANIZATION: NATIONAL CANCER INSTITUTE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/01/2023
PERIOD OF PERFORMANCE END DATE: 07/31/2028

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Personalized neuroblastoma vaccines - PROJECT SUMMARY This Multiple Principal Investigator (MPI) Project proposal for the Pediatric Immunotherapy Network is focused on high-risk neuroblastoma, a diverse and enigmatic malignancy arising from the developing sympathetic nervous system that remains lethal in 50% of patients despite intensive multi-modal therapy. There is an urgent unmet need for developing novel therapeutic interventions to decrease the incidence of relapse, increase overall survival, and reduce devastating toxicities associated with standard therapy. The primary goal of this Project is to achieve improved outcomes for patients with high-risk neuroblastoma through the development of a personalized vaccination strategy targeting individualized neoantigens. The central hypothesis is that high-risk neuroblastomas, despite a low tumor mutation burden (TMB), harbor a sufficient number of neoepitopes through canonical and non-canonical mutations to identify, predict, and validate optimal neoantigen peptides to engineer effective multivalent personalized neuroblastoma vaccines. The motivation for the proposed research is the urgent need to improve survival and to decrease treatment-related morbidities for patients with high-risk neuroblastoma. Indeed, the majority of high-risk neuroblastoma patients achieve a remission with standard therapy, and here we seek to engage the adaptive immune system to eradicate residual disease and prevent relapse. We will test our hypothesis through the two Specific Aims: 1) define the neoantigen landscape of high- risk neuroblastoma patient and genetically engineered mouse model (GEMM) tumors; 2) develop and test a readily translatable personalized vaccination strategy. In Aim 1 we will both provide the proof-of-concept that a multivalent vaccine consisting of both CD4+ and CD8+ epitopes is feasible for each high-risk patient and also credential our GEMM system for preclinical vaccination trials in Aim 2. We will use an integrative proteogenomic approach to identify up to eight immunogenic peptides for each personalized vaccine. In Aim 2, we will test both preventative and therapeutic efficacy of self-assembling nanoparticle multivalent peptide vaccines using our GEMM system on the CB57BL/6 background, and then compare this vaccine platform to a new lipid-peptide polymer vaccine system optimized to deliver peptides to dendritic cells. This Project proposes an innovative experimental strategy to identify, prioritize, and validate neoantigens in high-risk neuroblastoma, and a clinically relevant neuroblastoma GEMM system for preclinical evaluation of neuroblastoma vaccines. The significance of the proposed Project is the creation and validation of a novel immunotherapeutic approach that has the potential to revolutionize high-risk neuroblastoma standard of care by providing durable cures and decreased therapy- related morbidity. The expected outcome of this collaborative MPI Project is to establish the preclinical proof-of- concept for a personalized neuroblastoma clinical trial that we would seek to launch in the out years of this grant given the infrastructure we have in place. The successful completion of this Project will also enable personalized neoantigen-based vaccine development for other pediatric malignancies.


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 = $809,608 )
2025	2025	THE CHILDREN'S HOSPITAL OF PHILADELPHIA	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	21st Century Cures Act - Beau Biden Cancer Moonshot	000	3	8/11/2025	NON-COMPETING CONTINUATION	$809,608
														Subtotal = $809,608

Issue Date FY: 2024 ( Subtotal = $955,310 )
2024	2024	THE CHILDREN'S HOSPITAL OF PHILADELPHIA	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	21st Century Cures Act - Beau Biden Cancer Moonshot	002	2	9/5/2024	SUPPLEMENT FOR EXPANSION	$0
2024	2024	THE CHILDREN'S HOSPITAL OF PHILADELPHIA	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	21st Century Cures Act - Beau Biden Cancer Moonshot	001	2	8/5/2024	SUPPLEMENT FOR EXPANSION	$178,000
2024	2024	THE CHILDREN'S HOSPITAL OF PHILADELPHIA	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	21st Century Cures Act - Beau Biden Cancer Moonshot	000	2	7/30/2024	NON-COMPETING CONTINUATION	$777,310
														Subtotal = $955,310

Issue Date FY: 2023 ( Subtotal = $839,128 )
2023	2023	CHILDREN'S HOSPITAL OF PHILADELPHIA, THE	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	21st Century Cures Act - Beau Biden Cancer Moonshot	000	1	7/18/2023	NEW	$839,128
														Subtotal = $839,128

Grand Total All Awards = $2,604,046

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Personalized neuroblastoma vaccines

Award Number: U01CA281881

ORGANIZATION: NATIONAL CANCER INSTITUTE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

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

PERIOD OF PERFORMANCE START DATE: 08/01/2023

PERIOD OF PERFORMANCE END DATE: 07/31/2028

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