Precision Medicine in Pediatric Cancer

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More research and funding are needed to bring the benefits of precision medicine to pediatric patients.
More research and funding are needed to bring the benefits of precision medicine to pediatric patients.

Precision medicine based on genomic sequencing and targeted therapy has yielded potent tangible benefits for adult patients with cancer. Despite evidence of similar promise for pediatric patients, its study and use for children with cancer lags far behind.

As the authors of a recently published review of major developments and current limitations in the field said:

“While molecular targets in adult tumors have been the focus of most pharmaceutical efforts, pediatric patients have largely not yet benefited from these due to limited overlap with molecular events driving adult tumors, small number of patients, and safety concerns in young children.”1

They noted, however, that “this is beginning to change.”

The review included an examination of the early findings from 4 ongoing studies, including the University of Michigan Pediatric Michigan Oncology Sequencing (PEDS-MIONCOSEQ) study, the Baylor College of Medicine Advancing Sequencing in Childhood Cancer Care (BASIC3) study, the multi-institutional Individualized Therapy for Relapsed Malignancies in Childhood (INFORM) study coordinated through the German Cancer Research Center, and the Individualized Cancer Therapy (iCat) study, a multi-institutional effort through the Dana-Farber/Boston Children's Hospital Cancer and Blood Disorders Center.2

Significantly, the review stated, all 4 studies demonstrated that “clinical genomic analysis has the potential to identify potentially clinically relevant alterations in a substantial fraction of patients with pediatric cancer.”

That raises the possibility for clinicians to identify specific therapeutic strategies.

Precision medicine approaches have produced a number of effective targeted agents and combined therapies for the treatment of adult cancers. Inhibitor drugs aimed at tyrosine kinase alterations have increased the life expectancy of patients with chronic myeloid leukemia. And, as the authors of the recently published review stated, targeted therapies benefit patients with lung cancer with epidermal growth factor receptor (EGFR) alterations, as well as those with anaplastic lymphoma kinase (ALK) alterations.

Some show positive results in children.

“Extending the utility of drugs initially developed for adult cancers and repurposing them for pediatric tumors sharing the same target have become a major source of new clinical studies for pediatrics, and there are several particularly notable examples,” the authors wrote.

But multiple challenges impede rapid advances in providing precision medicine therapies for children. Among the most significant is the nature of pediatric cancers and the relative rarity of cases, compared to the number of adult patients. Pediatric cancers mutate differently from adult cancers; they are also the subject of fewer clinical trials and, consequently, of fewer targeted therapies.

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“The relative paucity of targetable mutations in pediatrics is compounded by limited access to newer targeted therapeutic agents due to the availability of fewer pediatric clinical studies and smaller number of eligible patients for each study,” the authors wrote. “Despite these challenges, initial pilot studies of genomic medicine in pediatric oncology have been both fruitful and encouraging.”

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