Novel molecular targeted therapies for refractory thyroid cancer
Related Experiment Video
Updated: Jun 2, 2026

Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Cesar A Perez1, Edgardo S Santos, Belisario A Arango
1Cleveland Clinic Florida, Weston, Florida, USA.
Abstract:
The incidence of thyroid cancer continues to increase and this neoplasia remains the most common endocrine malignancy. No effective systemic treatment currently exists for iodine-refractory differentiated or medullary thyroid carcinoma, but recent advances in the pathogenesis of these diseases have revealed key targets that are now being evaluated in the clinical setting. RET (rearranged during transfection)/PTC (papillary thyroid carcinoma) gene rearrangements, B-Raf gene mutations, and vascular endothelial growth factor receptor 2 (VEGFR-2) angiogenesis pathways are some of the known genetic alterations playing a crucial role in the development of thyroid cancer. Several novel agents have demonstrated promising responses. Of the treatments studied, multi-kinase inhibitors such as axitinib, sorafenib, motesanib, and XL-184 have shown to be the most effective by inducing clinical responses and stabilizing the disease process. Randomized clinical trials are currently evaluating these agents, results that may soon change the management of thyroid cancer.
Insights
Thyroid cancer is increasing, with no cure for advanced types. Novel multi-kinase inhibitors targeting genetic pathways show promise in clinical trials for improved thyroid cancer treatment.
Area of Science:
- Oncology
- Endocrinology
- Molecular Biology
Background:
- Thyroid cancer incidence is rising, representing the most frequent endocrine malignancy.
- Current systemic treatments are ineffective for iodine-refractory differentiated or medullary thyroid carcinoma.
- Understanding disease pathogenesis has identified key molecular targets for new therapies.
Purpose of the Study:
- To review recent advances in understanding thyroid cancer pathogenesis.
- To evaluate novel therapeutic agents targeting identified genetic alterations.
- To discuss the potential impact of these agents on thyroid cancer management.
Main Methods:
- Review of scientific literature on thyroid cancer genetics and targeted therapies.
- Analysis of clinical trial data for novel agents targeting RET/PTC, B-Raf, and VEGFR-2 pathways.
- Focus on multi-kinase inhibitors including axitinib, sorafenib, motesanib, and XL-184.
Main Results:
- Key genetic alterations like RET/PTC rearrangements, B-Raf mutations, and VEGFR-2 pathways are crucial in thyroid cancer development.
- Novel agents, particularly multi-kinase inhibitors, have demonstrated promising clinical responses and disease stabilization.
- Axitinib, sorafenib, motesanib, and XL-184 show significant efficacy in preliminary studies.
Conclusions:
- Targeted therapies are emerging as a promising strategy for managing advanced thyroid cancer.
- Multi-kinase inhibitors represent a potential breakthrough in treating iodine-refractory differentiated and medullary thyroid carcinoma.
- Ongoing randomized clinical trials are expected to redefine the treatment landscape for thyroid cancer.
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...
Treatment Resistant Cancers
Treatment Resistent Cancers
Tumor Immunotherapy

