Rare Disease Report

New Drugs Needed to Target Pediatric Cancers

JULY 18, 2018
Rare Disease Report® Editorial Staff
At the American Society of Clinical Oncology (ASCO) 2018 annual meeting in Chicago, Illinois, E. Anders Kolb, MD, Director of Nemours Center for Cancer and Blood Disorders at Nemours/Alfred I. duPont Hospital for Children, emphasizes the difference between pediatric and adult cancers and the need to develop new treatments developed specifically for children.



Interview Transcript (modified slightly for readability):

Kolb: “I’m a pediatric oncologist, and for a long time, we have been operating under the assumption that we can take drugs that have been developed in adults and apply them to children. I think what we’re learning more and more as we get more genomic data to describe pediatric cancers is that pediatric cancers are very, very different from adults, and the therapies that are out there right now are most likely not going to work for a majority of patients.

We need to think about developing novel therapies that are designed specifically for kids and not assume that drugs that were developed for adults will work in children.

There are some obvious differences between pediatric and adult cancers. Children don’t get colon cancer, prostate cancer, or breast cancer. They get cancers in tissues that are not necessarily exposed to a lifetime of use and abuse. They get cancers in tissues that are protected from the environment—the brain, soft tissues, bone marrow—and the malignancies form shortly after birth. They form at a time when exposures aren’t at the highest risk for disease, and I think that’s different from a lot of adult malignancies. We’ve known this for a long time.

But even where children and adults share the same type of cancer, such as acute myeloid leukemia, for example, or sarcoma, we’re starting to understand now that the genetic basis, the genetic driver of events in pediatric and adult tumors, are vastly different.

Children with acute myeloid leukemia tend to get large structural events, mutations, that define their malignancies, whereas adults will get an acquisition of a series of point mutations that define their acute myeloid leukemia.  

At any rate, the treatment has to be different. We have a toolbox that’s full of a bunch of sledgehammers, and that’s all we have for many of these treatments. As we get more and more information about the driver events, the mutations that are causing pediatric cancers, we can start to get more precise therapies—therapies that are designed to treat the cancer and have fewer side effects.”  
 

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