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Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Modified-Release Drug Delivery Systems: Site-Targeted01:24

Modified-Release Drug Delivery Systems: Site-Targeted

Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase01:11

Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase

Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...
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...

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Related Experiment Video

Updated: Jun 23, 2026

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms
08:46

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Published on: December 9, 2015

Targeting targeted agents: open issues for clinical trial design.

Emilio Bria1, Massimo Di Maio, Paolo Carlini

  • 1Department of Medical Oncology, Regina Elena National Cancer Institute, Rome, Italy. emiliobria@yahoo.it

Journal of Experimental & Clinical Cancer Research : CR
|May 26, 2009
PubMed
Summary

New molecularly targeted agents show limited clinical benefit in solid tumors despite promising early data. Current trial designs may be inadequate for assessing these novel therapies, necessitating a shift towards biomarker-driven approaches.

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Last Updated: Jun 23, 2026

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms
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Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
09:19

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo

Published on: February 6, 2015

Area of Science:

  • Oncology
  • Clinical Pharmacology
  • Translational Medicine

Background:

  • Molecularly targeted agents have recently entered the market for solid tumor treatment, generating significant scientific and societal interest.
  • Despite promising preclinical and Phase II data, many Phase III trials of targeted agents have yielded negative or marginally significant results.
  • The clinical impact of targeted therapies has been limited, failing to widely displace traditional chemotherapy.

Purpose of the Study:

  • To evaluate the efficacy and clinical relevance of molecularly targeted agents in solid tumor treatment.
  • To identify reasons for the discrepancy between trial outcomes and expectations for targeted therapies.
  • To propose methodological improvements for clinical trial design in the era of targeted cancer therapy.

Main Methods:

  • Review of recent randomized Phase III clinical trials involving molecularly targeted agents for solid tumors.
  • Analysis of the statistical significance versus clinical relevance of trial endpoints.
  • Assessment of current clinical trial methodologies in the context of targeted drug development.

Main Results:

  • Many targeted agent trials have failed to meet primary endpoints or shown only small, clinically negligible improvements.
  • The mechanisms of action for many targeted drugs remain incompletely understood, complicating efficacy assessment.
  • Existing trial designs, traditionally used for chemotherapeutics, are often inadequate for evaluating biomarker-driven targeted therapies.

Conclusions:

  • The translation of molecularly targeted agents into significant clinical practice improvements has been challenging.
  • A critical re-evaluation of clinical trial design is needed, emphasizing stronger hypotheses and biomarker-based patient selection.
  • Optimized trial designs focusing on early-phase identification of targets and robust response evidence are crucial for efficient drug development.