Di Santo Andrea, Tarchi Livio, Villa Gianluca, Castellini Giovanni, Ricca Valdo, Squecco Roberta, Papini Anna Maria, Real-Fernandez Feliciana, Rovero Paolo
Department of Neuroscience, Psychology, Pharmacology and Infant Health, Interdepartmental Research Unit of Peptide and Protein Chemistry and Biology, University of, Florence, Via Ugo Schiff, 6, Sesto Fiorentino, FI, 50019, Italy.
Department of Health Science, Psychiatry Unit, University of Florence, Largo Brambilla 3, Florence, FI, 50134, Italy.
ChemMedChem. 2025 May 5;20(9):e202400961. doi: 10.1002/cmdc.202400961. Epub 2025 Feb 17.
Growth differentiation factor 15 (GDF15) is a TGF-β superfamily member involved in diverse physiological and pathological processes. It is expressed in various tissues and its circulating levels rise during exercise, aging, pregnancy, and conditions such as cancer, cardiovascular disease, and infections. The biological activities of GDF15, including anorexia and cachexia, are primarily mediated through the GFRAL receptor, localized in the brainstem and functioning via RET co-receptor recruitment. This signaling is crucial for energy homeostasis and nausea induction. Recent studies suggest a broader GFRAL distribution, potentially explaining GDF15's distinct roles. These findings sparked interest in leveraging GDF15-GFRAL pathways for therapeutic development. Two primary strategies include GDF15 analogues as GFRAL agonists for obesity treatment and GDF15-derived peptides as antagonists to counteract cancer-induced cachexia and related disorders. This review highlights advancements in understanding GDF15-GFRAL signaling and its implications, summarizing bioactive GDF15-derived molecules, their pharmacological applications, and offering insights into novel treatment avenues for GDF15-associated conditions.
生长分化因子15(GDF15)是转化生长因子-β(TGF-β)超家族成员,参与多种生理和病理过程。它在各种组织中表达,并且在运动、衰老、怀孕以及癌症、心血管疾病和感染等情况下,其循环水平会升高。GDF15的生物学活性,包括厌食和恶病质,主要通过定位于脑干并通过招募RET共受体发挥作用的GFRAL受体介导。这种信号传导对于能量稳态和恶心诱导至关重要。最近的研究表明GFRAL分布更广泛,这可能解释了GDF15的不同作用。这些发现引发了人们对利用GDF15 - GFRAL途径进行治疗开发的兴趣。两种主要策略包括将GDF15类似物作为GFRAL激动剂用于肥胖症治疗,以及将GDF15衍生肽作为拮抗剂来对抗癌症引起的恶病质和相关疾病。这篇综述重点介绍了在理解GDF15 - GFRAL信号传导及其影响方面的进展,总结了具有生物活性的GDF15衍生分子、它们的药理学应用,并为GDF15相关病症的新型治疗途径提供了见解。