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Updated: Jul 21, 2025

Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Personalized Medicine in Medullary Thyroid Carcinoma: A Broad Review of Emerging Treatments
Rui Sousa Martins1,2,3, Tito Teles Jesus1,2, Luís Cardoso1,2,4
1Instituto de Investigação e Inovação em Saúde (i3S), Universidade do Porto, 4200-135 Porto, Portugal.
Abstract:
Medullary thyroid carcinoma (MTC) arises from parafollicular cells in the thyroid gland, and although rare, it represents an aggressive type of thyroid cancer. MTC is recognized for its low mutational burden, with point mutations in RET or RAS genes being the most common oncogenic events. MTC can be resistant to cytotoxic chemotherapy, and multitarget kinase inhibitors (MKIs) have been considered a treatment option. They act by inhibiting the activities of specific tyrosine kinase receptors involved in tumor growth and angiogenesis. Several tyrosine kinase inhibitors are approved in the treatment of advanced MTC, including vandetanib and cabozantinib. However, due to the significant number of adverse events, debatable efficiency and resistance, there is a need for novel RET-specific TKIs. Newer RET-specific TKIs are expected to overcome previous limitations and improve patient outcomes. Herein, we aim to review MTC signaling pathways, the most recent options for treatment and the applications for personalized medicine.
Insights
Medullary thyroid carcinoma (MTC) is aggressive but has few mutations. Novel RET-specific tyrosine kinase inhibitors (TKIs) are needed to improve MTC treatment outcomes beyond current options.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Medullary thyroid carcinoma (MTC) is a rare, aggressive thyroid cancer originating from parafollicular cells.
- MTC typically exhibits a low mutational burden, with common oncogenic events involving RET or RAS gene mutations.
- Current treatments like multitarget kinase inhibitors (MKIs) face challenges including adverse events, variable efficacy, and resistance.
Purpose of the Study:
- To review MTC signaling pathways.
- To discuss current and emerging treatment options for advanced MTC.
- To explore the potential of personalized medicine in MTC management.
Main Methods:
- Literature review of MTC signaling pathways.
- Analysis of approved and investigational therapies for MTC.
- Discussion of personalized medicine approaches.
Main Results:
- MTC is characterized by specific genetic alterations, primarily in RET and RAS pathways.
- Approved MKIs like vandetanib and cabozantinib offer treatment but have limitations.
- There is a clear need for more effective and specific therapies, such as novel RET-specific TKIs.
Conclusions:
- Understanding MTC signaling is crucial for targeted therapy development.
- Newer RET-specific TKIs hold promise for improved efficacy and reduced toxicity in MTC.
- Personalized medicine strategies are essential for optimizing MTC treatment outcomes.
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