Childhood Cancer

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Overcoming Obstacles to Drug Development in Pediatric Cancer

Institution: 
Stanford University School of Medicine
Researcher(s): 
Crystal L. Mackall, MD
Grant Type: 
Crazy 8 Pilots
Year Awarded: 
2019
Type of Childhood Cancer: 
General Pediatric Cancer
Project Description: 

Co-Principal Investigator

Mark Kieran, MD/PhD, Bristol-Myers Squibb

Co-Investigators

Peter C. Adamson, MD, Children’s Hospital of Philadelphia
Lia Gore, MD, Children’s Hospital of Colorado
Melinda Merchant, MD/PhD, AstraZeneca
Martha Donoghue, MD, FDA
Wendy Long, PhD, Dana-Farber Cancer Institute
Maureen M. O’Brien, MD, Cincinnati Children’s Hospital

Project Summary

Although much effort has been put toward increasing the accessibility of novel cancer therapies for children, the global system of pediatric oncology drug development continues to be suboptimal. The reasons for its current state are manifold and involve a complex interplay of incentives and barriers arising across a spectrum comprising the pharmaceutical industry, regulatory agencies, and the academic medical community. This proposal seeks funding to support a meeting of global stakeholders spanning these groups, including the advocacy community, to identify and address obstacles to timely pediatric cancer drug development and to promote a globally unified approach. The approach will leverage early progress driven by the ACCELERATE initiative, first launched in Europe, but now including colleagues from other continents, including North America. A summary report from the meeting will be drafted to serve as a blueprint for future work.

Project Update - January 2021: ALSF supported the 5th multi-stakeholder Pediatric Strategy Forum organized by the ACCELERATE group, who aim to improve the global development of drugs for children with cancer. The meeting brought together 72 participants from North America and Europe including academic experts, patient advocate groups, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) as well as 11 biopharmaceutical companies to discuss moving a new class of drugs called epigenetic modifiers toward the clinic for pediatric cancer patients. The meeting resulted in a prioritized list of eight classes of drugs to be moved into pediatric development. To learn more about the forum and its outcomes, read the meeting publication here.