Related Experiment Video

Updated: Oct 20, 2025

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids
08:52

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids

Published on: November 22, 2021

4.3K

The Landscape of Pediatric Precision Oncology: Program Design, Actionable Alterations, and Clinical Trial Development

Karin P S Langenberg1, Eleonora J Looze1, Jan J Molenaar1,2

  • 1Princess Máxima Center for Pediatric Oncology, Heidelberglaan 25, 3584 CS Utrecht, The Netherlands.

Cancers
|September 10, 2021
PubMed

Insights

Precision medicine in pediatric cancer utilizes molecular profiling to guide targeted treatments for high-risk cases. While feasible, standardized definitions and broader outcome data are needed for future advancements.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Precision medicine programs are increasingly used for pediatric patients with high-risk, relapsed, or refractory malignancies.
  • Comprehensive molecular profiling is key to selecting patients for targeted therapies.

Purpose of the Study:

  • To review the characteristics of precision medicine initiatives in pediatric oncology.
  • To demonstrate the feasibility and potential of molecular-driven precision medicine.
  • To identify challenges and future perspectives in the field.

Main Methods:

  • Review of existing precision medicine programs and initiatives for pediatric cancer.
  • Analysis of molecular profiling strategies and identification of actionable events.
  • Examination of early biomarker-driven clinical trial data.

Main Results:

  • Actionable molecular events are identified in a significant subset of pediatric cancer patients.
  • Comparing results across studies is challenging due to non-standardized definitions and profiling methods.
  • Early biomarker-driven trials show clinical benefit in some patients, but data is limited.

Conclusions:

  • Molecular-driven precision medicine is feasible and holds significant potential for pediatric oncology.
  • Standardization of definitions and methods is crucial for comparing outcomes.
  • Future directions include liquid biopsies, immune monitoring, collaborative trials, and novel targeted agents.
Keywords:
next-generation sequencingprecision medicinetargeted therapy

More Related Videos

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts10:27

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts

Published on: July 25, 2020

7.5K
Establishment of Orthotopic Patient-derived Xenograft Models for Brain Tumors using a Stereotaxic Device07:44

Establishment of Orthotopic Patient-derived Xenograft Models for Brain Tumors using a Stereotaxic Device

Published on: May 2, 2025

424

Related Concept Videos

Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.9K
Preclinical Development: Overview01:28

Preclinical Development: Overview

Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
5.3K
Pharmacokinetics in Pediatric Patients: Drug Metabolism01:24

Pharmacokinetics in Pediatric Patients: Drug Metabolism

In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
40
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.4K
Clinical Trials01:16

Clinical Trials

Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
9.8K
Cancer Survival Analysis01:21

Cancer Survival Analysis

Cancer survival analysis focuses on quantifying and interpreting the time from a key starting point, such as diagnosis or the initiation of treatment, to a specific endpoint, such as remission or death. This analysis provides critical insights into treatment effectiveness and factors that influence patient outcomes, helping to shape clinical decisions and guide prognostic evaluations. A cornerstone of oncology research, survival analysis tackles the challenges of skewed, non-normally...
481