Harvard Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA, USA.
Stem Cell Program and Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, USA.
Cell Cycle. 2021 Aug;20(15):1455-1467. doi: 10.1080/15384101.2021.1947567. Epub 2021 Jul 19.
Melanoma is the deadliest form of skin cancer. While clinical developments have significantly improved patient prognosis, effective treatment is often obstructed by limited response rates, intrinsic or acquired resistance to therapy, and adverse events. Melanoma initiation and progression are associated with transcriptional reprogramming of melanocytes to a cell state that resembles the lineage from which the cells are specified during development, that is the neural crest. Convergence to a neural crest cell (NCC)-like state revealed the therapeutic potential of targeting developmental pathways for the treatment of melanoma. Neural crest cells have a unique sensitivity to metabolic dysregulation, especially nucleotide depletion. Mutations in the pyrimidine biosynthesis enzyme dihydroorotate dehydrogenase (DHODH) particularly affect neural crest-derived tissues and cause Miller syndrome, a genetic disorder characterized by craniofacial malformations in patients. The developmental susceptibility of the neural crest to nucleotide deficiency is conserved in melanoma and provides a metabolic vulnerability that can be exploited for therapeutic purposes. We review the current knowledge on nucleotide stress responses in neural crest and melanoma and discuss how the recent scientific advances that have improved our understanding of transcriptional regulation during nucleotide depletion can impact melanoma treatment.
黑色素瘤是最致命的皮肤癌形式。虽然临床的发展显著改善了患者的预后,但有效的治疗往往受到有限的反应率、对治疗的内在或获得性耐药性以及不良反应的阻碍。黑色素瘤的发生和进展与黑素细胞的转录重编程有关,使其细胞状态类似于发育过程中细胞来源的谱系,即神经嵴。向神经嵴细胞(NCC)样状态的趋同揭示了靶向发育途径治疗黑色素瘤的治疗潜力。神经嵴细胞对代谢失调,特别是核苷酸耗竭具有独特的敏感性。嘧啶生物合成酶二氢乳清酸脱氢酶(DHODH)的突变特别影响神经嵴衍生的组织,并导致米勒综合征,这是一种遗传疾病,其特征是患者的颅面畸形。神经嵴对核苷酸缺乏的发育易感性在黑色素瘤中是保守的,为治疗目的提供了一种可利用的代谢脆弱性。我们回顾了神经嵴和黑色素瘤中核苷酸应激反应的现有知识,并讨论了最近改善我们对核苷酸耗竭期间转录调控理解的科学进展如何影响黑色素瘤的治疗。