Related Experiment Video

Updated: Jul 1, 2025

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
10:27

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

Published on: July 25, 2020

7.2K

Pediatric Cancer Drug Development: Leveraging Insights in Cancer Biology and the Evolving Regulatory Landscape to

Rosane Charlab1, Ruby Leong2, Stacy S Shord2

  • 1Office of Clinical Pharmacology, Office of Translational Sciences, U.S. Food and Drug Administration, Silver Spring, Maryland 20993, USA Rosane.CharlabOrbach@fda.hhs.gov.

Cold Spring Harbor Perspectives in Medicine
|March 11, 2024
PubMed

Insights

Pediatric anticancer drug development lags adult therapies due to multifactorial reasons. New strategies are needed to address unique pediatric cancer biology and optimize drug development for children.

Area of Science:

  • Oncology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Anticancer drug development for pediatric patients has historically lagged behind adult cancer therapeutics, including targeted and immune-oncology agents.
  • This disparity is multifactorial, influenced by regulatory, biological, and developmental considerations unique to childhood cancers.

Purpose of the Study:

  • To highlight the challenges and opportunities in pediatric anticancer drug development.
  • To propose strategies for efficient development and dosage optimization of anticancer drugs for pediatric patients.

Main Methods:

  • Review of current landscape of pediatric cancer drug development.
  • Analysis of regulatory factors and biological underpinnings of pediatric cancers.
  • Consideration of innovative clinical trial designs and model-informed drug development.

Main Results:

  • Recent regulatory changes and shared genetic drivers with adult cancers present new opportunities.
  • Unique biological drivers in pediatric cancers necessitate distinct therapeutic strategies.
  • Effective development requires a multi-pronged approach including biological research, innovative trials, and pediatric-specific formulations.

Conclusions:

  • Addressing the historical lag in pediatric anticancer drug development requires dedicated pediatric-specific strategies.
  • Integrating biological insights, innovative trial designs, and pediatric considerations is crucial for optimizing drug development and dosage for children.
  • A concerted effort is needed to accelerate the availability of effective anticancer therapies for pediatric patients.

More Related Videos

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms08:46

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms

Published on: December 9, 2015

10.6K
Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies09:29

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies

Published on: September 30, 2016

13.7K

Related Concept Videos

Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
4.9K
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...
4.4K
Drug Discovery: Overview01:26

Drug Discovery: Overview

Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
7.9K
Drug Regulation01:25

Drug Regulation

Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
1.4K
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...
2.9K
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...
345