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Updated: Aug 15, 2025

Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Targeted Therapy for Anaplastic Thyroid Carcinoma: Advances and Management
1The First Clinical Medical College, Zhejiang Chinese Medical University, Hangzhou 310053, China.
Abstract:
Anaplastic thyroid carcinoma (ATC) is a rare and highly fatal cancer with the worst prognosis of all thyroid carcinoma (TC) histological subtypes and no standard treatment. In recent years, the explosion of investigations on ATC-targeted agents has provided a new treatment strategy for this malignant condition, and a review of these studies is warranted. We conducted a comprehensive literature search for ATC-targeted drug studies and compiled a summary of their efficacy and adverse effects (AEs) to provide new insights. Multiple clinical trials have demonstrated the efficacy and safety of dabrafenib in combination with trametinib for the treatment of ATC, but vemurafenib and NTRK inhibitors showed limited clinical responses. We found that the previously valued therapeutic effect of lenvatinib may be unsatisfactory; combining tyrosine kinase (TK) inhibitors (TKIs) with other agents results in a higher rate of clinical benefit. In addition, specific medications, including RET inhibitors, mTOR inhibitors, CDK4/6 inhibitors, and Combretastatin A4-phosphate (CA4P), offer tremendous therapeutic potential. The AEs reported for all agents are relatively numerous but largely manageable clinically. More clinical trials are expected to further confirm the effectiveness and safety of these targeted drugs for ATC.
Insights
Targeted therapies show promise for anaplastic thyroid carcinoma (ATC), a rare and aggressive cancer. Dabrafenib plus trametinib is effective, while other agents like RET and mTOR inhibitors offer future potential.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Anaplastic thyroid carcinoma (ATC) is a rare, aggressive thyroid cancer subtype with a poor prognosis and no established standard treatment.
- Recent advancements in targeted therapies offer new treatment strategies for ATC.
Purpose of the Study:
- To review the efficacy and adverse effects of targeted agents investigated for anaplastic thyroid carcinoma.
- To provide insights into emerging therapeutic options for ATC.
Main Methods:
- A comprehensive literature search was conducted to identify studies on ATC-targeted drug therapies.
- Efficacy and adverse event data from clinical trials were compiled and summarized.
Main Results:
- Dabrafenib in combination with trametinib demonstrated efficacy and safety in clinical trials for ATC.
- Vemurafenib and NTRK inhibitors showed limited clinical responses.
- Combining tyrosine kinase inhibitors (TKIs) with other agents improved clinical benefit rates.
- RET inhibitors, mTOR inhibitors, CDK4/6 inhibitors, and Combretastatin A4-phosphate (CA4P) show significant therapeutic potential.
Conclusions:
- Targeted therapies, particularly combinations involving TKIs, represent a promising strategy for anaplastic thyroid carcinoma treatment.
- While adverse effects are noted, they are generally manageable.
- Further clinical trials are necessary to confirm the long-term effectiveness and safety of these targeted agents in ATC.
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