Development of Novel Therapies for Pediatric Cancer

描述（由申请人提供）： 儿科癌症是发生在历史上被制药和生物技术行业忽视的患者中的孤儿恶性肿瘤;因此，儿科癌症可以被认为是“双孤儿”-孤儿患者中发生的孤儿疾病。虽然在过去的40年里，在儿童癌症的管理方面取得了巨大的进步，但各种类型的儿童恶性肿瘤的现有治疗方法在疗效和毒性方面仍然存在许多缺点。该应用程序的主要目标是通过利用位于St. Jude儿童研究医院的AP 4中心提供的激励措施，促进儿科癌症治疗，预防和诊断新技术的开发。这个圣裘德AP 4中心的第二个目标-一个对中心的成功创建和发展至关重要的目标，以及它最终成为自我维持的能力-将把中心的活动纳入更广泛的区域努力的范围内，非营利和营利组织主要（但不限于）位于美国南部，致力于发展强大的区域医疗保健和生物技术产业。为了确保拟议的圣裘德AP 4中心的可行性和成功，我们将利用这些组织中的许多个人的投入，他们在临床转化和基础生物医学科学方面具有专业知识，从慈善机构，风险投资，政府和其他来源筹集资金，能够与政治人物，地方和联邦政府机构有效互动，在知识产权的保护和许可方面，在新的治疗、预防和诊断技术的临床和临床开发方面，以及在营销和商业化战略方面。这些区域利益集团的重要参与也在一定程度上是因为认识到，即使有AP 4赞助的激励措施，也很难从现有的制药和生物技术公司中为AP 4中心开发的选定的“小市场”技术找到合作伙伴。因此，我们需要一个完善的基础设施，以促进发展新的森林-将这些新方法商业化，以更好地管理儿童癌症。

DESCRIPTION (provided by applicant): Pediatric cancers are orphan malignancies that occur in patients who have historically been overlooked for the most part by the pharmaceutical and biotechnology industries; thus, the pediatric cancers can be considered double orphans" - orphan diseases that occur in orphan patients. Although dramatic strides have been achieved over the past 40 years in the management of the pediatric cancers, there remain many shortcomings in the efficacies and toxicities of available therapies for the various types of childhood malignancy. The major goal of this application is to facilitate the development of new technologies for the treatment, prevention, and diagnosis of pediatric cancer by leveraging the incentives provided through an AP4 Center to be based at St. Jude Children's Research Hospital. The secondary goal of this St. Jude AP4 Center - a goal critical to the successful creation and development of the Center, as well as its ability to ultimately become self-sustaining - will be to incorporate the activities of the Center within the context of a broader, regional effort involving academic, not-for-profit and for-profit organizations located primarily (but not exclusively) in the Southern U.S. that are committed to the development of a robust regional health care and biotechnology industry. To ensure the viability and success of the proposed St. Jude AP4 Center, we will utilize the input of numerous individuals from these organizations who have expertise in the clinical-translational and basic biomedical sciences, in fund-raising efforts from charitable, venture capital, governmental and other sources, in the ability to interact effectively with political figures, local and federal government agencies, in the protection and licensing of intellectual property, in the pie-clinical and clinical development of new therapeutic, preventative, and diagnostic technologies, and in marketing and commercialization strategies. The crucial involvement of these regional interests also grows in part out of the realization that it will likely remain difficult to identify partners from existing pharma and biotech for selected 'small market" technologies developed within this AP4 Center, even with AP4-sponsored incentives, necessitating the need for a well-developed infrastructure to foster the development of new for-profits to commercialize these novel methodologies for the better management of childhood cancer.