Targeting myostatin in cancer cachexia: Exploring the potential of MSTN gene therapy

Cancer cachexia is a debilitating syndrome associated with advanced-stage cancer and marked by involuntary weight and muscle loss. One of the most promising targets for intervention is myostatin, a protein known for suppressing muscle growth. This paper investigates the role of myostatin in cancer-induced muscle wasting and explores MSTN gene therapy as a potential treatment. A systematic review of 20 peer-reviewed articles published between 2018 and 2025 was conducted using PubMed, ScienceDirect, and Google Scholar. Results from animal models showed that myostatin inhibition improved muscle mass, preserved strength, and even reduced tumor progression. In addition, gene therapy targeting the MSTN gene demonstrated potential in halting muscle atrophy. However, human clinical trials testing agents such as LY2495655 and STM 434 reported mixed efficacy and adverse effects, including fatigue, anorexia, and bleeding complications. These inconsistencies between preclinical and clinical outcomes highlight the need for continued research. With further clinical testing and refinement, MSTN-targeted therapies may provide a new pathway to improve quality of life and prognosis in cancer patients suffering from cachexia.