Overview

While there are over 1,000 drugs in development for adult cancers, only a handful of these drugs are currently being tested in pediatric cancers. One promising avenue for more effective pediatric cancer drug development is to focus on earlier and improved assessment of the potential relevance of drugs developed for adult cancers. To prioritize these emerging adult therapies for pediatric development, a better understanding of a therapy’s ability to target relevant pathways in a range of pediatric cancers needs to be conducted. This often means intensive preclinical testing.

To aide in the identification of which pathways are relevant to pediatric cancer, the FDA generated the Relevant Molecular Target List, which consists of 200+ distinct molecular targets of interest in pediatric cancers and is part of the FDA Reauthorization Act of 2017 (FDARA). The list is based on a detailed examination of peer-reviewed publications and publicly available databases looking for any data that a target could be playing a role in one or more pediatric cancers.

Goals
  • Convene Quarterly Collaboration Meetings in Preclinical Pediatric Oncology to foster discussion and collaboration across the pediatric oncology field.

  • Identify pediatric oncology targets that should be accelerated through preclinical testing.

  • Publish summary reports to share progress on target analysis.

  • Assist in guiding a streamlined way forward to more effective pediatric cancer drug development

From 2022 through 2024, in collaboration with the National Cancer Institute and the FDA, the FNIH convened key stakeholders and knowledge leaders in the pediatric oncology field in quarterly Convening Experts in Oncology to Address Children’s Health (COACH) meetings to systematically review existing data on targets. At each meeting, participants discussed three to five targets of significant preclinical and clinical need. A summary report of the discussions and recommendations for each target was released to the broader community after the meetings.

We hope these reports can help inform decisions on target selection for testing efforts. In addition, the outputs from COACH could ultimately support the development of pediatric treatments as therapies emerge to address high-priority targets.

Meeting Summaries and Recommendations

July 22, 2022
Targets Reviewed: EZH2, MDM2, CD47.

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September 19, 2022

Targets Reviewed: MEN-1, IL-2, PARP.

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December 15, 2022

Targets Reviewed: PD(L)1+CD73, PD(l)1+LAG3, PD(L)1+TIGIT, B7H3.

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March 15, 2023
Targets Reviewed: ATR, HDAC, PIK3 alpha.

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June 13, 2023

Targets Reviewed: BCL-2, MCL-1, BCL-xL.

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September 18, 2023

Targets Reviewed: ADCs: CD22, CD33, PTK7, B7H3

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December 1, 2023

Targets Reviewed: CCR8, TEAD1, HER2

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March 11, 2024
Targets: GPC2/3, SHP2, CDK9

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Partners

Public-Sector Partners
  • National Cancer Institute (NCI)
  • U.S. Food and Drug Administration (FDA)
  • European Medicines Agency (EMA)
  • NCI-supported Pediatric Preclinical in Vivo Testing (PIVOT) Program Principal Investigators
  • Innovative Therapies for Children with Cancer Pediatric Preclinical Proof of Concept Platform (ITCCP-4) Academic Partners
Private-Sector Partners
  • AbbVie
  • BMS/Celgene Corporation
  • Day One Biopharmaceuticals
  • Genentech/Roche
  • Gilead
  • Johnson and Johnson
  • Oncoheroes Biosciences
  • Pfizer
  • PhRMA
  • Sanofi
Advocacy Partners
  • Children’s Cancer Cause
  • Coalition Against Childhood Cancer (CAC2)
  • CureSearch for Children’s Cancer
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