使用复制选择性溶瘤腺病毒针对肿瘤抑制基因的基因组改变进行胰腺癌的基因治疗。
Gene therapy for pancreatic cancer targeting the genomic alterations of tumor suppressor genes using replication-selective oncolytic adenovirus.
作者信息
Sunamura Makoto, Oonuma Masaru, Motoi Fuyuhiko, Abe Hisashi, Saitoh Yukoh, Hoshida Toru, Ottomo Shigeru, Horii Akira, Matsuno Seiki
机构信息
Division of Gastroenterological Surgery, Tohoku University, Graduate School of Medicine, Sendai, Japan.
出版信息
Hum Cell. 2002 Sep;15(3):138-50. doi: 10.1111/j.1749-0774.2002.tb00108.x.
In order to develop an effective therapeutic intervention for patients with pancreatic cancer, we examined the genetic alternations of pancreatic cancer. Based on these results, we are developing a new gene therapy targeting the genetic character of pancreatic cancer using mutant adenoviruses selectively replication-competent in tumor cells. Loss of heterozygosity (LOH) of 30% or more were observed on chromosome arms 17p (47%), 9p (45%), 18q (43%), 12q (34%), and 6q (30%). LOH of 12q, 17p, and 18q showed the significant association with poor prognosis. These data strongly suggest that mutation of the putative suppressor genes, TP53 and SMAD4 play significant roles in the disease progression. Based on this rationale, we are developing a new gene therapy targeting tumors without normal TP53 function. E1B-55kDa-deleted adenovirus (AxE1AdB) can selectively replicate in TP53-deficient human tumor cells but not cells with functional TP53. We evaluated the therapeutic effect of this AxE1AdB on pancreatic cancer without normal TP53 function. The growth of human pancreatic tumor in SCID mice model was markedly inhibited by the consecutive injection of AxE1AdB. Furthermore, AxE1AdB is not only the strong weapon but also useful carrier of genes possessing anti-tumor activities as a virus vector specific to tumors without normal TP53 function. It was reported that uracil phosphoribosyl transferase (UPRT) overcomes 5FU resistance. UPRT catalyzes the synthesis of 5-fluorouridine monophosphate (FUMP) from Uracil and phosphoribosylpyrophosphate (PRPP). The antitumor effect of 5FU is enhanced by augmenting 5-fluorodeoxyuridine monophosphate (FdUMP) converted from FUMP, which inhibits Thymidylate Synthetase (TS). The therapeutic advantage of restricted replication competent adenovirus that expresses UPRT (AxE1AdB-UPRT) was evaluatedin an intra-peritoneal disseminated tumor model. To study the anti-tumor effect of AxE1AdB-UPRT/5FU, mice with disseminated AsPC-1 tumors were administered the adenovirus, followed by the 5FU treatment. It was shown that the treatment with AxE1AdB-UPRT/5FU caused a dramatic reduction of the disseminated tumor burden without toxicity in normal tissues. These results revealed thatthe AxE1AdB-UPRT/5FU system is a promising tool for intraperitoneal disseminated pancreatic cancer.
为了开发针对胰腺癌患者的有效治疗干预措施,我们研究了胰腺癌的基因改变。基于这些结果,我们正在开发一种新的基因疗法,该疗法利用在肿瘤细胞中具有选择性复制能力的突变腺病毒,针对胰腺癌的基因特征进行治疗。在17号染色体短臂(47%)、9号染色体短臂(45%)、18号染色体长臂(43%)、12号染色体长臂(34%)和6号染色体长臂(30%)上观察到30%或更高的杂合性缺失(LOH)。12号染色体长臂、17号染色体短臂和18号染色体长臂的杂合性缺失与预后不良显著相关。这些数据强烈表明,假定的抑癌基因TP53和SMAD4的突变在疾病进展中起重要作用。基于这一原理,我们正在开发一种针对无正常TP53功能肿瘤的新基因疗法。E1B-55kDa缺失腺病毒(AxE1AdB)可在TP53缺陷的人肿瘤细胞中选择性复制,但不能在具有功能性TP53的细胞中复制。我们评估了这种AxE1AdB对无正常TP53功能的胰腺癌的治疗效果。在SCID小鼠模型中,连续注射AxE1AdB可显著抑制人胰腺肿瘤的生长。此外,AxE1AdB不仅是一种强大的武器,而且作为一种针对无正常TP53功能肿瘤的病毒载体,还是具有抗肿瘤活性基因的有用载体。据报道,尿嘧啶磷酸核糖转移酶(UPRT)可克服5-氟尿嘧啶(5FU)耐药性。UPRT催化由尿嘧啶和磷酸核糖焦磷酸(PRPP)合成5-氟尿苷单磷酸(FUMP)。通过增加由FUMP转化而来的5-氟脱氧尿苷单磷酸(FdUMP)来增强5FU的抗肿瘤作用,FdUMP可抑制胸苷酸合成酶(TS)。在腹腔播散性肿瘤模型中评估了表达UPRT的限制复制能力腺病毒(AxE1AdB-UPRT)的治疗优势。为了研究AxE1AdB-UPRT/5FU的抗肿瘤作用,给患有播散性AsPC-1肿瘤的小鼠注射腺病毒,随后进行5FU治疗。结果表明,AxE1AdB-UPRT/5FU治疗可显著降低播散性肿瘤负荷,且对正常组织无毒性。这些结果表明,AxE1AdB-UPRT/5FU系统是治疗腹腔播散性胰腺癌的一种有前景的工具。