GENE THERAPY FOR HUMAN MALIGNANT MELANOMA

描述：（申请人的描述） 黑色素瘤是最恶性的皮肤癌。 在美国， 黑色素瘤的发病率比其他任何癌症都要高。 黑素瘤是一 最常发生在25-30岁的女性中的癌症， 最近取代白血病，成为世界上损失工作时间最多的人。 美国的 目前还没有批准的治疗方法， 20%的回复率 因此，恶性黑色素瘤的更好疗法是 迫切需要。 Introgen Therapeutics正在开发一种新的抗肿瘤基因 治疗，Ad-mda 7，并已证明它的抗肿瘤潜力， 乳腺癌和肺癌细胞系。 在这里，我们建议执行 原理验证实验，以评估Ad-mda 7在 黑素瘤 将使用人黑素瘤进行生长抑制研究 细胞系，如果成功，将继续评估Ad-mda 7在 体内使用人黑素瘤异种移植物。 如果这些目标能够实现，我们 将测试单独的Ad-mda 7和与常规化疗药物的组合 使用转移性肿瘤模型的药物。 在第一阶段STTR结束时，我们 计划收集必要的有效性数据以进行安全性研究 并根据Ad-mda 7的临床试验测试，在II期STTR中提交IND。 拟定商业应用： Ad-mda 7是一种基因治疗药物，作为一种新的治疗药物进行测试，用于治疗 恶性黑素瘤 恶性黑色素瘤是一种可怕的皮肤癌，几乎没有可用的治疗方法 选项. 黑色素瘤和恶性黑色素瘤的发病率呈上升趋势。 约 预计今年将有41，000人被诊断患有黑色素瘤，其中超过7，000人 死于这种疾病。 本文概述的研究将指导临床前 开发用于黑素瘤的Ad-mda 7。 随后的II期研究将评估 Ad-mda 7在以提交BLA为目标的临床试验中的治疗潜力 以及随后的市场推广。

DESCRIPTION: (Applicant's Description) Melanoma is the most malignant of skin cancers. In the USA, the incidence of melanoma is increasing more rapidly than any other cancer. Melanoma is the most frequently occurring cancer in women from the ages of 25-30, and has recently replaced leukemia as responsible for the most lost work hours in the United States. Currently there is no approved therapy that achieves more that a 20% response rate. Therefore, better therapies for malignant melanoma are urgently needed. Introgen Therapeutics is developing a novel anti-tumor gene therapeutic, Ad-mda7, and has demonstrated it's anti-tumor potential against breast and lung cancer cell lines. Here, we propose to perform proof-of-principle experiments to evaluate the efficacy of Ad-mda7 in melanoma. Growth inhibition studies will be performed using human melanoma cell lines in vitro and, if successful, will progress to evaluate Ad-mda7 in vivo using human melanoma xenografts. If these first aims are successful, we will test Ad-mda7 alone and in combination with conventional chemotherapeutic agents using metastatic tumor models. At the end of this Phase I STTR, we plan to have gathered the necessary efficacy data to proceed to safety studies and IND filing in a Phase II STTR, based on clinical trial testing of Ad-mda7. PROPOSED COMMERCIAL APPLICATION: Ad-mda7 is a gene therapy drug to be tested as a novel therapeutic for the treatment of malignant melanoma. Malignant melanoma is a dire skin cancer with few available treatment options. The incidence of melanoma and malignant melanoma is increasing. Approximately 41,000 people are predicted to diagnosed with melanoma this year, with more than 7,000 deaths resulting from this disease. The studies outlined here will guide the preclinical development of Ad-mda7 for melanoma. Subsequent Phase II studies will evaluate the therapeutic potential of Ad-mda7 in clinical trials with the goal of submission of a BLA to the FDA and subsequent marketing.