UCL Cancer Institute, University College London, 72 Huntley Street, London WC1E 6BT, UK.
Centre for Cardiovascular Sciences, Queens Medical Research Institute, University of Edinburgh, 47 Little France Crescent, Edinburgh EH16 4TJ, UK.
Biomolecules. 2019 Aug 1;9(8):331. doi: 10.3390/biom9080331.
Genetic activation of the class I PI3K pathway is very common in cancer. This mostly results from oncogenic mutations in PIK3CA, the gene encoding the ubiquitously expressed PI3Kα catalytic subunit, or from inactivation of the PTEN tumour suppressor, a lipid phosphatase that opposes class I PI3K signalling. The clinical impact of PI3K inhibitors in solid tumours, aimed at dampening cancer-cell-intrinsic PI3K activity, has thus far been limited. Challenges include poor drug tolerance, incomplete pathway inhibition and pre-existing or inhibitor-induced resistance. The principle of pharmacologically targeting cancer-cell-intrinsic PI3K activity also assumes that all cancer-promoting effects of PI3K activation are reversible, which might not be the case. Emerging evidence suggests that genetic PI3K pathway activation can induce and/or allow cells to tolerate chromosomal instability, which-even if occurring in a low fraction of the cell population-might help to facilitate and/or drive tumour evolution. While it is clear that such genomic events cannot be reverted pharmacologically, a role for PI3K in the regulation of chromosomal instability could be exploited by using PI3K pathway inhibitors to prevent those genomic events from happening and/or reduce the pace at which they are occurring, thereby dampening cancer development or progression. Such an impact might be most effective in tumours with clonal PI3K activation and achievable at lower drug doses than the maximum-tolerated doses of PI3K inhibitors currently used in the clinic.
PI3K 通路的基因激活在癌症中非常常见。这主要是由于 PIK3CA 基因突变所致,PIK3CA 基因编码普遍表达的 PI3Kα 催化亚基,或由于抑癌基因 PTEN 失活所致,PTEN 是一种脂质磷酸酶,可拮抗 I 类 PI3K 信号。目前,旨在抑制肿瘤细胞内在 PI3K 活性的针对实体瘤的 PI3K 抑制剂的临床影响有限。挑战包括药物耐受性差、不完全的通路抑制以及预先存在的或抑制剂诱导的耐药性。针对肿瘤细胞内在 PI3K 活性的药理学靶向治疗原则还假设 PI3K 激活的所有促进癌症的作用都是可逆的,但事实可能并非如此。新出现的证据表明,遗传 PI3K 通路激活可诱导和/或允许细胞耐受染色体不稳定性,即使在细胞群体中的一小部分中发生,也可能有助于促进和/或驱动肿瘤演变。虽然很明显,这种基因组事件不能通过药理学逆转,但可以通过使用 PI3K 通路抑制剂来防止这些基因组事件的发生和/或降低它们发生的速度,从而抑制癌症的发展或进展,从而利用 PI3K 在调节染色体不稳定性方面的作用。这种影响在具有克隆性 PI3K 激活的肿瘤中最为有效,并且可以在比目前临床上用于治疗的最大耐受剂量的 PI3K 抑制剂更低的药物剂量下实现。