Nemunaitis Jackie M, Nemunaitis John
Mary Crowley Cancer Research Centers, 1700 Pacific Avenue, Ste 110, Dallas, TX 75201, USA.
Future Oncol. 2008 Dec;4(6):759-68. doi: 10.2217/14796694.4.6.759.
Li-Fraumeni syndrome is a rare autosomal dominant cancer predisposition syndrome. The majority of families fulfilling definition of Li-Fraumeni syndrome demonstrate inherited abnormalities involving the p53 gene. Cells with dysfunctional p53 are predisposed to the development of cancer phenotype. Advexin (Introgen Therapeutics Inc., TX, USA) is an adenoviral-based experimental therapeutic that provides delivery of wild-type p53 to cancer cells and demonstrates anticancer activity following adequate expression of p53. Theoretically, correction of p53 function in cancer developing in patients with Li-Fraumeni syndrome through treatment with Advexin will provide anti-tumor activity. One patient with Li-Fraumeni syndrome has been reported to have responded to Advexin. This review will summarize background knowledge of Li-Fraumeni syndrome, mechanisms of Advexin and clinical response of cancer to Advexin with a focus on Li-Fraumeni syndrome.
李-弗劳梅尼综合征是一种罕见的常染色体显性癌症易感综合征。大多数符合李-弗劳梅尼综合征定义的家族表现出涉及p53基因的遗传性异常。p53功能失调的细胞易发生癌症表型。Advexin（美国得克萨斯州Introgen Therapeutics公司）是一种基于腺病毒的实验性治疗药物，可将野生型p53递送至癌细胞，并在p53充分表达后显示出抗癌活性。理论上，通过Advexin治疗纠正李-弗劳梅尼综合征患者发生癌症时的p53功能将提供抗肿瘤活性。据报道，一名李-弗劳梅尼综合征患者对Advexin有反应。本综述将总结李-弗劳梅尼综合征的背景知识、Advexin的作用机制以及癌症对Advexin的临床反应，重点关注李-弗劳梅尼综合征。
