Customized Nanotechnology Based Treatment of Oral Cancer

 描述（由申请人提供）：在本提案中，Privo描述了其研发计划，以获得美国食品药品监督管理局（FDA）的研究新药（IND）地位，其局部纳米颗粒化疗晶片用于安全治疗口腔癌。根据口腔癌基金会的数据，今年将有超过43,250名美国人被诊断出患有口腔癌和咽癌，造成8,000多人死亡，大约每小时一人。2011年，全世界诊断出超过64万例新的口腔癌病例，只有一半的人在初次诊断后能活5年以上。到2020年，全球每年的发病率预计将增加到超过840，000，增长30%，每年的死亡率将增加到近480，000，增长约37%。目前，顺铂处于用于治疗口腔癌的基于铂的化疗药物的最前沿。然而，由于传统的推注全身静脉内给药，其显著的全身毒性经常阻碍其成功给药 (IV)局Privo Technologies是麻省理工学院Langer实验室的一家公司，该实验室开发了一种纳米工程ChemoThin Wafer（CTW（c）），用于局部和局部递送化疗药物顺铂（CIS）至口腔癌肿瘤。该技术已获得超过300万美元的非稀释性资金，其中包括最近批准的国家癌症研究所（NCI）为其IND临床前研究提供的230万美元资金。Privo的治疗已被证明比金标准静脉注射（IV）顺铂更安全，更有效。CTW（c）易于管理，只需1小时即可完成申请。在6次治疗后，它在动物中引起了完全的肿瘤消退。在该提案中，Privo要求NIDCR提供资金，当与NCI资金相结合时，将为Privo提供足够的资源，以获得FDA研究新药（IND）地位。该提案包括在临床试验前执行所需的化学生产和控制（CMC）工艺，开发所需的试验，包括活性药物成分（顺铂）的鉴别试验、效价试验和纯度试验，建立GMP生产，以及采购药用级原材料。Privo认为，其CTW（c）与NIDCR的目标密切相关，即为口腔癌提供更安全、更有效、成本更低的治疗方法。

 DESCRIPTION (provided by applicant): In this proposal, Privo describes its research and development plans in order to obtain Food and Drug Administration (FDA) Investigational New Drug (IND) status for its localized nanoparticle based chemotherapy wafer for safely treating oral cancer. According to the Oral Cancer Foundation, over 43,250 Americans will be diagnosed with oral and pharyngeal cancer this year, causing over 8,000 deaths, roughly one person every hour. In 2011, over 640,000 new oral cancer cases were diagnosed worldwide, and only half will live more than 5 years after initial diagnosis. By 2020, the annual worldwide incidence is predicted to increase to over 840,000, a 30% rise, and the annual mortality to increase to nearly 480,000, approximately a 37% increase. Currently, cisplatin is at the forefront of platinum based chemotherapeutics for treating oral cancer. However, its successful administration is frequently hindered by its significant systemic toxicity as a result of traditional bolus systemic intravenous (IV) administration. Privo Technologies is a company with roots in MIT's Langer Lab which has developed a nanoengineered ChemoThin Wafer (CTW(c)) for topical and local delivery of chemotherapy agent cisplatin (CIS) to oral cancer tumors. This technology has been awarded more than $3 million in non- dilutive funding, including a recent approval of $2.3 million funding from the National Cancer Institute (NCI) for its IND enabling preclinical studies. Privo's treatmen has been shown to be safer and more effective than the gold standard intravenous (IV) cisplatin. CTW(c) is easy to administer, and takes only 1 hour to complete the application. It has caused complete tumor regression in animals after 6 treatments. In this proposal, Privo requests funding from NIDCR which, when combined with the NCI funding, will provide Privo the adequate resources to obtain its FDA Investigational New Drug (IND) status. This proposal includes performing the Chemistry Manufacturing and Control (CMC) process required prior to clinical trials, developing required assays including the identity assay, potency assay of the active pharmaceutical ingredient (cisplatin), and purity assay, the establishment of GMP manufacturing, and sourcing pharmaceutical grade raw materials. Privo believes that its CTW(c) aligns closely with the aims of NIDCR in providing a safer, more effective, and less costly treatment for oral cancer.