Related Experiment Video
Updated: May 23, 2026

An Orthotopic Mouse Model of Anaplastic Thyroid Carcinoma
Published on: April 17, 2013
In search of a real "targeted" therapy for thyroid cancer
1Departments of Medicine and Otorhinolaryngology: Head and Neck Surgery, Abramson Cancer Center, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19010, USA. brosem@mail.med.upenn.edu
Abstract:
Over the past 5 years, patients with progressive radioactive iodine-refractory thyroid cancer have responded to "targeted" multikinase inhibitors, which inhibit angiogenesis and not the tumor cell. Here, selumetinib targets the mitogen-activated protein kinase pathway in papillary thyroid carcinoma and shows limited single-agent activity in the patients with tumors that harbor the (V600E)BRAF mutation.
Insights
Selumetinib targets the mitogen-activated protein kinase pathway in advanced radioactive iodine-refractory thyroid cancer. This targeted therapy showed limited effectiveness in patients with BRAF V600E mutations.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Radioactive iodine-refractory thyroid cancer is a progressive disease.
- Multikinase inhibitors targeting angiogenesis have shown promise.
- The mitogen-activated protein kinase (MAPK) pathway is implicated in cancer progression.
Purpose of the Study:
- To evaluate the efficacy of selumetinib, a MAPK pathway inhibitor.
- To assess selumetinib's activity in papillary thyroid carcinoma.
- To investigate the role of BRAF V600E mutations in treatment response.
Main Methods:
- Selumetinib was administered as a single agent.
- The study population included patients with progressive radioactive iodine-refractory thyroid cancer.
- Tumor genetic analysis identified BRAF V600E mutations.
Main Results:
- Selumetinib demonstrated limited single-agent activity.
- Patients with tumors harboring the BRAF V600E mutation showed a restricted response.
- The drug targets the MAPK pathway, not directly the tumor cell.
Conclusions:
- Selumetinib has limited efficacy as a single agent in this patient population.
- BRAF V600E mutation status may influence treatment outcomes.
- Further research into targeted therapies for refractory thyroid cancer is warranted.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Combination Therapies and Personalized Medicine
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...
Tumor Immunotherapy
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
Modified-Release Drug Delivery Systems: Site-Targeted

