Mapping the Matrix

The progress against childhood cancer is a shining success of modern medicine. Today, the five-year survival rate (the medical definition of a cure) has climbed to more than 80 percent.1 But the speed of this progress has slowed, and without significant changes in how new cancer medicines are developed for children, their survival rates may plateau.

Biologically, kids are not little adults, and childhood cancers are inherently different than cancer in adults. The challenge in getting new medicines to children comes down to two areas: numbers and lack of collaboration.

Before cancer medicines are approved for use, they must undergo rigorous testing through clinical trials in hundreds of volunteers. But the number of children with cancer is much, much smaller than the number of adults with cancer. That means it’s difficult to enroll enough patients in a trial to understand the medicine’s potential safety and efficacy.

When several companies work independently to develop treatments with similar mechanisms of action for the same cancer, they inadvertently compound the problem by competing to recruit from an already small patient population. Currently, some trials can’t recruit enough patients to generate meaningful data.

This situation has created a painful irony: in adults, more competition fosters innovation and more options; for kids, more competition creates barriers and leaves fewer or no options. Children with terminal diseases have no time to wait indefinitely for these treatments to be approved. We need a better system to bring new medicines to children more efficiently.

This year, Vice President Joseph Biden’s Cancer Moonshot and other pediatric-focused initiatives began, planning to bring potential immunotherapies and other treatments to kids with cancer. Researchers are looking at the underlying cause of the disease, and this approach of studying the molecular drivers of cancer also holds promise for children. Increasing evidence shows that although adult and pediatric cancers are often very different, they may share similar molecular underpinnings.

These initiatives that prioritize children’s cancers are welcome, but also highlight some of the problems of bringing medicines developed for adults to children.

Today’s drug development guidelines require companies to test a drug in children only for the cancer type that it has been developed for in adults. These guidelines do not reflect the latest science. For many pediatric cancers, there is no adult counterpart. But promising cancer medicines developed to target a specific mutation should be explored for pediatric uses, even if it’s in a different cancer type or if it has not been shown to work in adults.

On September 23, my colleagues joined leaders in the field at the Pediatric Master Protocols FDA workshop to push for improvements in bringing new cancer drugs to children. One area they discussed is how to better structure pediatric clinical trials based on the latest science.

We’re exploring this concept with a protocol called iMATRIX, which pairs medicines with their known molecular targets, regardless of the cancer type. Matching approved and investigational medicines by their mechanism of action expands the number of eligible pediatric trial participants. Similar approaches have yielded positive results in adults. Now is the time to apply this same “outside the box” thinking for children with cancer.

iMATRIX also invites collaboration within the industry for trial patients and opens up previously untested avenues for currently existing as well as new investigational therapies. The hope is that this approach will ultimately generate more robust clinical data and efficiency. By running smaller studies that produce equally rigorous results, researchers may accelerate the development of promising medicines for children. As of today, the iMATRIX study is currently enrolling children and young adults with certain types of cancer, and we are having discussions with regulatory authorities and other companies researching pediatric cancers about how to accelerate the addition and removal of potential medicines to optimize the iMATRIX platform

Doing the best for our children is not an individual effort. Collaborative public initiatives like the Cancer Moonshot are paving the way to revitalizing progress in pediatric cancer research. That same spirit is needed in the private sector, where companies must not continue to work in isolation to develop medicines for children with cancer. iMATRIX was designed to prioritize the use of investigational or approved medicines developed by different companies in an unbiased manner. This early stage framework will use input from health authorities, pediatric oncology experts and patient advocacy groups to move research forward.

Our society’s greatest feats in cancer treatment have not been achieved in a vacuum. Nowhere is that work more important than in pediatric cancer. Children with cancer don’t need the industry to spin their wheels - they need everyone to come together to help them.


1 Last accessed 8/25/16.

2 Last accessed 8/25/16.

3 Last accessed 8/25/16.

4 Pica & Bourgeois (2016). Discontinuation and Nonpublication of Randomized Clinical Trials Conducted in Children. Pediatrics. doi: 10.1542/peds.2016-0223.