Related Experiment Video

Updated: Jan 27, 2026

Culture of Bladder Cancer Organoids as Precision Medicine Tools
08:39

Culture of Bladder Cancer Organoids as Precision Medicine Tools

Published on: December 28, 2021

5.4K

Ushering in the next generation of precision trials for pediatric cancer

Steven G DuBois1,2,3, Laura B Corson1,2, Kimberly Stegmaier1,2,3

  • 1Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.

Science (New York, N.Y.)
|March 16, 2019
PubMed

Insights

Precision oncology is improving outcomes for children with cancer. This review covers factors, regulations, and challenges in developing targeted pediatric cancer drugs, including those targeting difficult proteins.

Area of Science:

  • Oncology
  • Genomics
  • Pediatric Medicine

Background:

  • Precision oncology, guided by tumor genomic profiles, is transforming cancer treatment.
  • Evidence supports precision oncology's effectiveness in pediatric cancer patients.
  • Pediatric drug development faces unique challenges in the era of targeted therapies.

Purpose of the Study:

  • To review key factors influencing pediatric drug development for precision oncology.
  • To discuss the evolving regulatory landscape for pediatric cancer clinical trials.
  • To explore novel approaches for targeting difficult-to-treat pediatric cancers.

Main Methods:

  • Review of current literature on precision oncology in pediatric cancer.
  • Analysis of regulatory frameworks impacting pediatric drug trials.
  • Discussion of challenges and innovations in pediatric cancer drug development.

Main Results:

  • Precision oncology demonstrates efficacy in specific pediatric cancer cases.
  • An emerging regulatory framework is expediting pediatric clinical trials.
  • Specific design challenges exist for pediatric cancer clinical trials.
  • New strategies are needed for targeting challenging proteins like transcription factors.

Conclusions:

  • Precision oncology offers significant promise for treating childhood cancers.
  • Regulatory adaptations are crucial for advancing pediatric targeted therapies.
  • Addressing unique pediatric trial designs and therapeutic targets is essential for future progress.

More Related Videos

Detection of Targetable Alterations in Non-small Cell Lung Cancer using Next-generation Sequencing05:17

Detection of Targetable Alterations in Non-small Cell Lung Cancer using Next-generation Sequencing

Published on: October 10, 2025

379
The Generation of Higher-order Laguerre-Gauss Optical Beams for High-precision Interferometry12:14

The Generation of Higher-order Laguerre-Gauss Optical Beams for High-precision Interferometry

Published on: August 12, 2013

22.4K

Related Concept Videos

Uncertainty in Measurement: Accuracy and Precision03:37

Uncertainty in Measurement: Accuracy and Precision

Scientists typically make repeated measurements of a quantity to ensure the quality of their findings and to evaluate both the precision and the accuracy of their results. Measurements are said to be precise if they yield very similar results when repeated in the same manner. A measurement is considered accurate if it yields a result that is very close to the true or the accepted value. Precise values agree with each other; accurate values agree with a true value. 
100.8K
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...
4.9K
Trial and Error and Algorithm01:12

Trial and Error and Algorithm

A problem-solving strategy is a plan of action used to find a solution. Different strategies have distinct action plans. Trial and error involves trying different solutions until one works. For instance, to fix a broken printer, you might check ink levels, ensure the paper tray isn't jammed, and verify the printer's connection to your laptop. This method can be time-consuming but is commonly used. Thomas Edison, for example, used trial and error to find a suitable filament for the light...
401
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...
10.3K
Accuracy and Precision01:52

Accuracy and Precision

Scientists typically make repeated measurements of a quantity to ensure the quality of their findings and to evaluate both the precision and the accuracy of their results. Measurements are said to be precise if they yield very similar results when repeated in the same manner. A measurement is considered accurate if it yields a result that is very close to the true or the accepted value. Precise values agree with each other; accurate values agree with a true value.  Highly accurate...
15.1K
Pharmacokinetics in Pediatric Patients: Drug Excretion01:26

Pharmacokinetics in Pediatric Patients: Drug Excretion

In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
225