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Development and deployment of computational methods to create novel immunotherapies for children with cancer

Award Number: R50CA293895
ORGANIZATION: NATIONAL CANCER INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2026
PERIOD OF PERFORMANCE END DATE: 08/31/2031

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Development and deployment of computational methods to create novel immunotherapies for children with cancer - Cancer is the leading cause of childhood death from disease in the United States and other developed countries. Neuroblastoma (NBL), an extracranial pediatric malignancy that arises from aberrant development of the sympathetic nervous system, accounts for 12% of all childhood cancer deaths. NBL is the most common cancer diagnosed during infancy and survival rates for children with high-risk disease are poor despite intensive multimodal therapy. Moreover, survivors typically have lifelong therapy-induced morbidities, further defining a dire need for new rational therapeutic approaches. Our research program is rooted in cancer genomics and has made several important contributions to our understanding of the genetic basis of NBL under the leadership of Dr. John Maris. We have uniquely assembled perhaps the largest cohort of patient samples, preclinical models, and proteogenomic data. This allows us to lead various multi-institutional collaborations within a translational ecosystem where Dr. Alvin Farrel, a collaborative computational scientist embedded within the Maris Lab, can develop bioinformatics pipelines and analyze petabytes of data leading to the discovery of new therapeutic targets to improve outcomes for children with cancer. Dr. Farrel developed a computational pipeline to characterize the immunopeptidome of NBL which was used to discover a peptide derived from PHOX2B that is recurrently presented on common HLA alleles in NBL. PHOX2B is normally silenced epigenetically before birth and is not expressed in any normal postnatal tissues making it a perfect immunotherapeutic target. Targeting the PHOX2B peptide in the major histocompatibility complex groove with peptide-centric chimeric antigen receptor (PC-CAR) T cells killed NBL tumors in multiple pre-clinical studies which paved the way for a first-in- human first-in-child Phase 1/1b clinical trial (NCT07007117). We have treated 3 patients to date with no off-target toxicity and early signs of efficacy. Now, Dr. Farrel plans to apply these peptide discovery pipelines to other solid tumors. We are confident that this approach can be successful in other cancers as the efficiency of PC-CARs depend greatly on tumor HLA expression, yet PC-CARS killed NBL tumors despite its very low HLA expression. In addition to PC-CARs, we plan to achieve improved survival and decreased treatment-related morbidities for patients with high-risk NBL through the development of a personalized vaccination strategy targeting individualized neoantigens. We hypothesize that NBL generates sufficient tumor specific (neo)epitopes that can be discovered and developed into safe and effective immunotherapies. In a collaborative effort, we will define the neoantigen landscape of NBL and develop a personalized proteogenomic vaccination strategy. Here, Dr. Farrel has three major objectives: 1) Continue to support the collaborative efforts within our research program, 2) build on our immunopeptidomic bioinformatic pipelines to discover PC-CAR targets in other solid tumors, and 3) develop bioinformatic pipelines to support our immunotherapeutic and personalized vaccine strategies to reduce relapse in NBL. Together, these objectives will help improve outcomes for children with cancer.


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: 2026 ( Subtotal = $181,610 )
2026	2026	THE CHILDREN'S HOSPITAL OF PHILADELPHIA	3401 CIVIC CENTER BLVD	PHILADELPHIA	PA	19104	PHILADELPHIA	USA	Cancer Treatment Research	000	1	8/27/2026	NEW	$181,610
														Subtotal = $181,610

Grand Total All Awards = $181,610

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Development and deployment of computational methods to create novel immunotherapies for children with cancer

Award Number: R50CA293895

ORGANIZATION: NATIONAL CANCER INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/01/2026

PERIOD OF PERFORMANCE END DATE: 08/31/2031

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