Growth factor applications and clinical translation: advances and challenges

Background Growth factors (GFs), as key molecules regulating cellular processes, demonstrate therapeutic potential in fields such as regenerative medicine, oncology, and metabolic regulation. However, inherent pharmacokinetic limitations, off-target effects, and safety concerns significantly hinder clinical translation. Although in-depth studies on mechanisms of action, smart delivery systems, and regulatory networks have improved GFs’ stability, targeting, and safety – leading to clinical approvals of some related drugs – high costs, complex large-scale production processes, and the intricacy of the in vivo microenvironment continue to restrict the scope and breadth of GFs’ clinical translation.Discussion This review systematically examines the classification and fundamental research progress of GFs, emphasizing their current applications in neurodegenerative diseases, bone regeneration, and metabolic disorders. It thoroughly analyzes major challenges in clinical translation, including suboptimal pharmacokinetic properties, difficulties in precise delivery, potential side effects, and high industrial production costs. To address these bottlenecks, the paper summarizes corresponding solutions, such as structural optimization through protein engineering and chemical modification, development of smart responsive delivery systems (e.g. extracellular vesicle-based platforms), and utilization of multi-factor sequential release technologies to mimic physiological repair processes.Conclusion By synthesizing current advances and existing constraints, this study aims to provide a comprehensive roadmap and technical reference for accelerating the development of next-generation GF therapies. Future research should focus on developing scalable delivery platforms, reducing production costs through synthetic biology, and strengthening biomarker-based translational studies. Multidisciplinary integration holds promise for advancing GFs therapies towards greater precision, safety, and efficacy, positioning them as core therapeutic tools in the era of precision medicine for addressing degenerative diseases, tissue damage, and metabolic disorders.