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Pediatric High-Risk Cancer Preclinical Model Resource

External Institution: The Children’s Hospital of Philadelphia (CHOP)

External Collaborators: Kathrin Bernt, MD, Yael Mosse, MD, Rebecca Linn, MD

FDA Collaborators: Martha Donoghue, MD

Project Start Date: September 2021

Regulatory Science Challenge

Cancer is a major reason for childhood deaths, ranking third behind suicides and accidents. Most cancers in children are treated with surgery, radiation or chemotherapy approaches that were developed decades ago. While there are many new exciting therapeutic approaches for adults with cancer, children lag behind. There are many reasons for this discrepancy: 

  • Children are not just small adults. Their cancers are different. New drugs developed for specific adult cancers may not work in the kind of cancers children get.
  • Overall, childhood cancer is thankfully rare. However, this means that there is not much of a financial incentive for large pharmaceutical companies to design and develop new drugs.
  • There is a shortage of model systems of childhood tumors that researchers or drug companies could use to see whether some drugs developed for adults might work for children, or to develop new drugs specifically for children.

In an effort to boost drug development for pediatric cancers, the FDA has decided to address this third point – a lack of model systems. The program instituted by the FDA will seek to develop a resource of pediatric tumor models that are made available to researchers and companies to increase drug development for childhood cancers.

Project Description and Goals

CHOP is a major children’s’ hospital with many active clinical trials, where children with rare or difficult to treat cancers come from all over the world. CHOP also has a cutting-edge research program for both leukemias and solid tumors in children. We will capitalize on this infrastructure to develop models for pediatric cancers in the following three sub-projects:

1. Build a database of tumor models that we will share with the scientific community that is easily searchable and has a streamlined process for distribution.
2. Import 150 tumor models already existing at CHOP into the database.
3. Develop 60 additional tumor models specifically from patients with rare or highly aggressive tumors that have the greatest need for new treatments. 

Models will be made available to requesting investigators free of charge to facilitate the rapid preclinical testing of promising new therapies for cancer in children.

Further Information

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