Electroporation Based Cytokine Gene Therapy for Melanoma

描述（由申请人提供）： 尽管该技术已显示出前景，但细胞因子疗法在很大程度上 由于低反应率和高相关毒性，令人失望。基因 已经提出了基于治疗的细胞因子递送策略作为一种手段 改善这些免疫调节剂的治疗和毒理学作用， 分子。 然而，这种方法的临床实施受到以下因素的阻碍： 缺乏临床上可行的基因递送载体。 最近的研究表明 体内电穿孔可以显著改善 治疗剂进入肿瘤组织。 Ichor开发了电穿孔技术 技术，使安全，有效，可重复的临床 治疗的应用。该公司目前正在进行第一阶段 电穿孔介导药物博来霉素靶向的临床研究 胰腺肿瘤 最初的临床前工作表明，这些相同的 技术还可以显着改善肿瘤内递送 核苷酸构建体。 Ichor建议将其专有的 细胞因子基因导入肿瘤的电穿孔技术 组织. 拟议研究的范围包括啮齿动物研究， 建立有效的治疗方案和大型动物毒理学， 生物分布研究 FLAIR第一阶段的成功完成将导致 转移性黑色素瘤患者治疗的临床研究， 在FLAIR第二阶段进行。 拟定商业应用： 不可用

DESCRIPTION (provided by applicant): Although the technique has shown promise, cytokine therapy has largely disappointed due to low response rates and high associated toxicity. Gene therapy based strategies for cytokine delivery have been proposed as a means improve the therapeutic and toxicologic effects of these immunomodulatory molecules. However, clinical implementation of this approach is hampered by the lack of a clinically viable gene delivery vector. Recent studies suggest that in vivo electroporation can dramatically improve the delivery of therapeutic agents into tumor tissue. Ichor has developed electroporation technology to enable the safe, effective, and reproducible clinical application of the therapy. The company is currently conducting a Phase I clinical study of electroporation mediated delivery of the drug bleomycin into pancreatic tumors. Initial preclinical work suggests that these same techniques can also dramatically improve the intratumoral delivery of nucleotide constructs. Ichor proposes to adapt its proprietary electroporation technology for the delivery of cytokine genes into tumor tissue. The scope of the proposed research includes rodent studies designed to establish effective treatment protocols and large animal toxicology and biodistribution studies. Successful completion of FLAIR Phase I will lead to clinical investigation of the therapy in patients with metastatic melanoma, to be conducted under FLAIR Phase II. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE