Emerging drugs for Cushing's disease

Area of Science:

• Endocrinology
• Pharmacology

Background:

• Uncontrolled hypercortisolism in Cushing's disease (CD) significantly increases morbidity and mortality.
• There is an ongoing need for effective medical therapies to manage CD.
• Research into novel and existing therapeutic agents for CD is actively progressing.

Purpose of the Study:

• To review the current landscape of medical therapies for Cushing's disease.
• To discuss the efficacy, advantages, and disadvantages of various drugs used in CD treatment.
• To explore the potential for combination therapies in managing hypercortisolism.

Main Methods:

• Discussion of clinical trial designs for validating existing drugs and testing new compounds for CD.
• Reporting of results from ongoing clinical trials for drugs including ketoconazole, LCI699, mifepristone, etomidate, and pasireotide.
• Emphasis on the comparative analysis of drug properties and potential synergistic effects.

Main Results:

• Metyrapone is recommended for rapid control of hypercortisolaemia.
• Ketoconazole serves as a second-line treatment, with LCI699 as a potential future alternative.
• Mifepristone, etomidate, cabergoline, and pasireotide offer options for specific clinical scenarios.

Conclusions:

• Cushing's disease management requires careful risk-benefit assessment of therapeutic options.
• A range of pharmacological agents exist, each with specific indications and limitations.
• The choice of therapy depends on the urgency of hypercortisolism control and patient-specific factors.