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Targeted Therapies in Pediatric Cancers

September 9–12, 2021

Chaired By

 Steven G. Dubois, MD of Dana-Farber Cancer Institute
 Martine F. Roussel, PhD of St. Jude Children’s Research Hospital

Meeting Description

Several novel targeted therapies based on rational scientific data hold promise to offer new and effective treatment not only for adult but also pediatric cancers. The new FDA pediatric legislative initiatives has galvanized pharmaceutical companies to extrapolating efficacy from adult data or other data to the pediatric population to streamline pediatric drug development and help to increase the number of approvals for pediatric use. Several clinical trials are ongoing for primary and recurrent and difficult to treat pediatric cancers that hopefully will advance children’s care.

Meeting Summary

The Foundation hosted its first forum since the start of the COVID-19 pandemic. For those who were able to gather in person, the meeting took place in Rancho Santa Fe, California. A virtual option was available for those who were unable to travel. The focus of the meeting was Targeted Therapies in Pediatric Cancers. The meeting co-chairs were Steven DuBois (Dana-Farber) and Martine Roussel (St. Jude). Fourteen scientists joined in person and four scientists participated virtually, allowing us to see how a Forbeck Forum might work using a hybrid format.

Several key themes emerged from this forum. First, it is clear that there is an unacceptable delay in getting novel agents into the pediatric clinic. While the RACE Act will help, there remain regulatory issues related to timeline for fulfilling these trials, role of trials in adolescents only to fulfill RACE requirements, and the pressing need for combination studies. Academic and consortium trials need to consider regulatory requirements and provide data that will potentially be “fit for filing” to meet regulatory requirements. Moreover, increasing requirements for companion diagnostics are not in line with clinical practice for broader (rather than more narrow) testing for specific genomic changes. Second, multiple novel agents targeting epigenetic modifiers are in the pediatric clinic and represent opportunities for innovation. Third, there is tremendous interest in cell states as mediators of drug sensitivity and the potential to shift cells into states that are more sensitive. Fourth, a major theme was resistance to targeted therapies, strategies for mitigating resistance including combination approaches, and developing new preclinical models and screens to evaluate novel approaches to overcoming resistance.

Forum Participants

Jessie Brown, PhD
Columbia University
 Forbeck Scholar

Michael Cox, PharmD, MHSc, BCOP
Loxo Oncology

Timothy Cripe, MD, PhD, FAAP, CPI
Nationwide Children's Hospital, Ohio

Steven G. Dubois, MD
Dana-Farber Cancer Institute

Adam Durbin, MD, PhD
Dana Farber Cancer Institute
 Forbeck Scholar

Maryam Fouladi, MD, MSc, FRCPC
Cincinnati Children's Hospital

Birgit Geoerger, MD
Gustave Roussy, Paris, France

E. Anders Kolb, MD
Nemours Center for Cancer and Blood Disorders

Elizabeth Lawlor, MD, PhD
Seattle Children's Hospital

Loretta Li, MD
Lurie Children's Hospital of Chicago
 Forbeck Scholar

Charles Mullighan, MD
St. Jude Children's Hospital

Martine Roussel, PhD
St. Jude Children's Hospital

Ari Shilatifard, PhD
Northwestern University

Kevan Shokat, PhD

Kim Stegmaier, MD
Dana-Farber Cancer Institute

Sarah Tasian, MD
Children's Hospital of Philadelphia