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Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
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Development of Molecularly Targeted Therapies to Treat Pediatric Malignancies

Clinton F Stewart1, Giles W Robinson2

  • 1Department of Pharmaceutical Sciences, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.

Clinical Pharmacology and Therapeutics
|August 25, 2017
PubMed

Insights

Future cancer drug development for children should focus on pediatric-specific targets. This approach aims to improve treatment effectiveness and reduce side effects for young cancer patients, moving closer to a cure.

Area of Science:

  • Pediatric oncology
  • Cancer drug development
  • Molecularly targeted therapies

Background:

  • Historically, pediatric cancer treatments were adapted from adult indications.
  • This approach has yielded useful drugs but may not be optimal for children.
  • Pediatric cancer requires specific therapeutic strategies.

Purpose of the Study:

  • To advocate for the specific development of molecularly targeted therapies (MTTs) for pediatric tumors.
  • To emphasize the need to prioritize pediatric-specific cancer research.
  • To improve outcomes for children diagnosed with cancer.

Main Methods:

  • This study is a conceptual analysis and recommendation based on current trends in pediatric oncology.
  • It reviews the history of drug development in pediatric cancer.
  • It highlights the potential of molecularly targeted therapies.

Main Results:

  • Current drug development for pediatric cancer often originates from adult studies.
  • There is a need for targeted therapies designed specifically for pediatric tumors.
  • Optimizing MTTs for pediatric cancers can maximize efficacy and minimize toxicity.

Conclusions:

  • Future development of molecularly targeted therapies should be pediatric-focused.
  • Specific targeting of pediatric tumors will enhance antitumor efficacy.
  • This strategy is crucial for minimizing morbidity and long-term disability in pediatric cancer survivors.

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