Overview of management and therapeutic advances in medullary thyroid cancer

Medullary thyroid carcinoma (MTC) is a rare cancer of the thyroid’s calcitonin-producing C cells. This review covers recent advances in MTC treatment, emphasizing surgical and systemic therapies. For localized MTC, surgery remains the primary and most effective treatment, with total thyroidectomy an...

Full description

Saved in:
Bibliographic Details
Main Authors: Mark A Jara, Luciana Audi Castroneves
Format: Article
Language:English
Published: Bioscientifica 2025-03-01
Series:Endocrine Oncology
Subjects:
Online Access:https://eo.bioscientifica.com/view/journals/eo/5/1/EO-24-0077.xml
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850036310575677440
author Mark A Jara
Luciana Audi Castroneves
author_facet Mark A Jara
Luciana Audi Castroneves
author_sort Mark A Jara
collection DOAJ
description Medullary thyroid carcinoma (MTC) is a rare cancer of the thyroid’s calcitonin-producing C cells. This review covers recent advances in MTC treatment, emphasizing surgical and systemic therapies. For localized MTC, surgery remains the primary and most effective treatment, with total thyroidectomy and lymph node dissection providing the highest potential for cure. However, prognosis worsens significantly with local and distant metastases, underscoring the importance of early diagnosis and intervention. MTC can be sporadic or hereditary, with the latter associated with germline RET proto-oncogene mutations linked to multiple endocrine neoplasia types 2A and 2B. Genetic discoveries have enabled preventive measures such as prophylactic thyroidectomy, increasing the cure rate of hereditary cases. Since 2011, systemic treatment options have expanded with multikinase inhibitors (MKIs), such as vandetanib and cabozantinib, and selective RET inhibitors such as selpercatinib and pralsetinib. MKIs extend progression-free survival in advanced cases by targeting tumor growth and angiogenesis but can cause off-target effects. RET inhibitors offer precision treatment for RET-mutated tumors, showing high efficacy and fewer side effects, though resistance to these inhibitors has emerged, and current research focuses on developing next-generation inhibitors to overcome these barriers. Effective MTC management, particularly given its rarity, benefits from specialized high-volume centers. Precision medicine, standardized therapy selection and ongoing research are essential for improving outcomes in both RET-positive and RET-negative MTC patients.
format Article
id doaj-art-6ba6086e774f435885e4bfc0f5e2f89b
institution DOAJ
issn 2634-4793
language English
publishDate 2025-03-01
publisher Bioscientifica
record_format Article
series Endocrine Oncology
spelling doaj-art-6ba6086e774f435885e4bfc0f5e2f89b2025-08-20T02:57:13ZengBioscientificaEndocrine Oncology2634-47932025-03-015110.1530/EO-24-00771Overview of management and therapeutic advances in medullary thyroid cancerMark A Jara0Luciana Audi Castroneves1Department of Endocrinology and Metabolism. University of Miami, Miami, Florida, USADepartment of Endocrinology. Instituto do Câncer do Estado de São Paulo, Faculdade de Medicina da Universidade de São Paulo, São Paulo, BrazilMedullary thyroid carcinoma (MTC) is a rare cancer of the thyroid’s calcitonin-producing C cells. This review covers recent advances in MTC treatment, emphasizing surgical and systemic therapies. For localized MTC, surgery remains the primary and most effective treatment, with total thyroidectomy and lymph node dissection providing the highest potential for cure. However, prognosis worsens significantly with local and distant metastases, underscoring the importance of early diagnosis and intervention. MTC can be sporadic or hereditary, with the latter associated with germline RET proto-oncogene mutations linked to multiple endocrine neoplasia types 2A and 2B. Genetic discoveries have enabled preventive measures such as prophylactic thyroidectomy, increasing the cure rate of hereditary cases. Since 2011, systemic treatment options have expanded with multikinase inhibitors (MKIs), such as vandetanib and cabozantinib, and selective RET inhibitors such as selpercatinib and pralsetinib. MKIs extend progression-free survival in advanced cases by targeting tumor growth and angiogenesis but can cause off-target effects. RET inhibitors offer precision treatment for RET-mutated tumors, showing high efficacy and fewer side effects, though resistance to these inhibitors has emerged, and current research focuses on developing next-generation inhibitors to overcome these barriers. Effective MTC management, particularly given its rarity, benefits from specialized high-volume centers. Precision medicine, standardized therapy selection and ongoing research are essential for improving outcomes in both RET-positive and RET-negative MTC patients.https://eo.bioscientifica.com/view/journals/eo/5/1/EO-24-0077.xmlmedullary thyroid carcinomaret
spellingShingle Mark A Jara
Luciana Audi Castroneves
Overview of management and therapeutic advances in medullary thyroid cancer
Endocrine Oncology
medullary thyroid carcinoma
ret
title Overview of management and therapeutic advances in medullary thyroid cancer
title_full Overview of management and therapeutic advances in medullary thyroid cancer
title_fullStr Overview of management and therapeutic advances in medullary thyroid cancer
title_full_unstemmed Overview of management and therapeutic advances in medullary thyroid cancer
title_short Overview of management and therapeutic advances in medullary thyroid cancer
title_sort overview of management and therapeutic advances in medullary thyroid cancer
topic medullary thyroid carcinoma
ret
url https://eo.bioscientifica.com/view/journals/eo/5/1/EO-24-0077.xml
work_keys_str_mv AT markajara overviewofmanagementandtherapeuticadvancesinmedullarythyroidcancer
AT lucianaaudicastroneves overviewofmanagementandtherapeuticadvancesinmedullarythyroidcancer