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Targeting RET in medullary thyroid cancer.
Endocrine-Related Cancer
|July 31, 2025
Summary
Medullary thyroid cancer (MTC) treatment has advanced with tyrosine kinase inhibitors targeting RET mutations. Newer, selective RET inhibitors offer improved efficacy and safety, though overcoming resistance remains crucial.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Medullary thyroid cancer (MTC) is a rare endocrine malignancy.
- RET proto-oncogene mutations are found in 25-65% of MTC cases, leading to aberrant kinase activity.
- Targeted therapies have revolutionized MTC treatment, particularly for RET-mutated tumors.
Purpose of the Study:
- To review the advancements in MTC treatment.
- To highlight the role of tyrosine kinase inhibitors in managing MTC.
- To discuss the evolution from multikinase inhibitors to selective RET inhibitors.
Main Methods:
- Review of clinical studies and literature on MTC therapeutics.
- Analysis of the efficacy and side-effect profiles of various tyrosine kinase inhibitors.
- Examination of the mechanism of action for RET-targeted therapies.
Main Results:
- Multikinase inhibitors (vandetanib, cabozantinib) demonstrated efficacy in advanced MTC.
- Selective RET inhibitors (selpercatinib, pralsetinib) show high efficacy with improved safety profiles.
- These newer agents reduce off-target effects, enhancing patient outcomes.
Conclusions:
- Tyrosine kinase inhibitors, especially selective RET inhibitors, represent significant progress in MTC therapy.
- Current treatments have improved disease control and patient well-being.
- Further research is needed to address and overcome treatment resistance in MTC.

