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

To begin identifying which of these 200+ molecular targets might prove to be the most critical to developing new therapies for specific childhood cancers, a systematic review of existing data around targets is needed. In collaboration with NCI and FDA, the FNIH is convening key stakeholders and knowledge leaders in the pediatric oncology field in quarterly Convening Experts in Oncology to Address Children’s Health (COACH) meetings.

At each meeting, participants discuss 3-5 targets of significant preclinical and clinical need. A summary report of each discussion and recommendations for each target is released to the broader community after the meeting.

We hope, the findings of these discussions will help decide which targets to focus testing efforts. In addition, the meeting outputs from COACH could ultimately inform pediatric treatment development as therapies that address the identified high priority targets emerge within the field.

These are invitation-only meetings. If your organization is interested in partnering with us on this effort, please reach out to the FNIH project contacts.

Meeting Summaries and Recommendations

July 22, 2022

Targets Reviewed: EZH2, MDM2, CD47.

View Report Infographic
September 19, 2022

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

View Report Infographic
December 15, 2022

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

View Report Infographic
March 15, 2023

Targets Reviewed: ATR, HDAC, PIK3 alpha.

View Report Infographic
June 13, 2023

Targets Reviewed: BCL-2, MCL-1, BCL-xL. Report to be posted soon.

View Report
September 18, 2023

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

December 1, 2023

Targets Reviewed: CCR8, TEAD1, HER2

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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