Jasani Neel, Xu Xiaonan, Posorske Benjamin, Kim Yumi, Wang Kaizhen, Vera Olga, Tsai Kenneth Y, DeNicola Gina M, Karreth Florian A
Department of Molecular Oncology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida.
Cancer Biology PhD Program, University of South Florida, Tampa, Florida.
Cancer Res. 2025 Jan 15;85(2):314-328. doi: 10.1158/0008-5472.CAN-24-2471.
Overexpression of phosphoglycerate dehydrogenase (PHGDH), the rate-limiting enzyme in the serine synthesis pathway, promotes melanomagenesis, melanoma cell proliferation, and survival of metastases in serine-low environments such as the brain. Here, we found that PHGDH is universally increased in melanoma cells and required for melanomagenesis. Although PHGDH amplification explained PHGDH overexpression in a subset of melanomas, oncogenic BRAFV600E also promoted PHGDH transcription through mTORC1-mediated translation of ATF4. Importantly, depletion of PHGDH in genetic mouse melanoma models blocked tumor formation. In addition to BRAFV600E-mediated upregulation, PHGDH was further induced by exogenous serine restriction. Surprisingly, BRAFV600E inhibition diminished serine restriction-mediated PHGDH expression by preventing ATF4 induction. Consequently, melanoma cells could be specifically starved of serine by combining BRAFV600E inhibition with exogenous serine restriction, which promoted cell death in vitro and attenuated melanoma growth in vivo. In summary, this study identified that PHGDH is essential for melanomagenesis and regulated by BRAFV600E, revealing a targetable vulnerability in BRAFV600E-mutant melanoma. Significance: BRAFV600E promotes the expression of the serine synthesis enzyme PHGDH, which is required for melanoma formation, and can be targeted to sensitize melanoma to dietary serine restriction, providing a melanoma cell-specific treatment strategy.
磷酸甘油酸脱氢酶(PHGDH)是丝氨酸合成途径中的限速酶,其过表达会促进黑色素瘤的发生、黑色素瘤细胞增殖以及在丝氨酸水平低的环境(如脑)中转移灶的存活。在此,我们发现PHGDH在黑色素瘤细胞中普遍上调,且是黑色素瘤发生所必需的。虽然PHGDH扩增解释了一部分黑色素瘤中PHGDH的过表达,但致癌性BRAFV600E也通过mTORC1介导的ATF4翻译促进了PHGDH转录。重要的是,在基因小鼠黑色素瘤模型中敲除PHGDH可阻断肿瘤形成。除了BRAFV600E介导的上调外,外源性丝氨酸限制可进一步诱导PHGDH。令人惊讶的是,BRAFV600E抑制通过阻止ATF4的诱导而减弱了丝氨酸限制介导的PHGDH表达。因此,通过将BRAFV600E抑制与外源性丝氨酸限制相结合,可使黑色素瘤细胞特异性缺乏丝氨酸,这在体外促进细胞死亡,在体内减弱黑色素瘤生长。总之,本研究确定PHGDH对黑色素瘤发生至关重要且受BRAFV600E调控,揭示了BRAFV600E突变型黑色素瘤中一个可靶向的脆弱点。意义:BRAFV600E促进丝氨酸合成酶PHGDH的表达,这是黑色素瘤形成所必需的,并且可作为靶点使黑色素瘤对饮食中丝氨酸限制敏感,从而提供一种黑色素瘤细胞特异性治疗策略。