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Published on: January 29, 2021
Tumor-Directed Therapeutic Targets in Cushing Disease
Marily Theodoropoulou1, Martin Reincke1
1Medizinische Klinik und Poliklinik IV, Ludwig Maximilian University Munich, Munich, Germany.
Effective tumor-targeted therapies for Cushing disease (CD), a rare cause of hypercortisolism, are limited. Further research is crucial to establish novel therapeutic options for this severe condition.
Area of Science:
- Endocrinology
- Oncology
- Pharmacology
Background:
- Cushing disease (CD) is the most common cause of endogenous hypercortisolism.
- CD leads to severe comorbidities and high mortality.
- Current tumor-targeted therapies for CD are limited.
Purpose of the Study:
- To review current and potential tumor-targeted pharmacological therapies for Cushing disease.
- To highlight the unmet need for effective treatments in CD.
Main Methods:
- Literature search of PubMed using keywords: "corticotroph", "Cushing's disease", and therapeutic agents.
- Review of associated references from retrieved papers.
- Exploration of ClinicalTrials.gov for "Cushing disease" to identify potential therapeutics.
Main Results:
- Current therapies include dopamine agonists (cabergoline) and somatostatin analogs (pasireotide), with variable efficacy, response escape, and side effects.
- Preclinical studies suggest potential agents like retinoic acid, silibinin, and roscovitine for corticotroph pathophysiology.
- The efficacy and safety of these potential drugs require further determination.
Conclusions:
- There is a significant lack of effective tumor-targeted pharmacological therapy for many Cushing disease patients.
- Coordinated research efforts are essential to establish the efficacy and safety of novel therapeutics for CD.
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