Development of Novel Therapies for Pediatric Cancer

描述(由申请人提供)： 儿科癌症是发生在制药和生物技术行业历史上大部分被忽视的患者身上的孤儿恶性肿瘤；因此，儿科癌症可以被视为“双重孤儿”--发生在孤儿患者中的孤儿疾病。尽管在过去的40年里，儿童癌症的治疗已经取得了巨大的进步，但在各种类型的儿童恶性肿瘤的现有治疗方法的有效性和毒性方面，仍然存在许多不足。该应用程序的主要目标是通过利用总部设在圣裘德儿童研究医院的AP4中心提供的激励措施，促进治疗、预防和诊断儿科癌症的新技术的开发。圣裘德AP4中心的次要目标是将中心的活动纳入更广泛的地区性努力范围内，这些组织主要(但不完全)位于美国南部，致力于发展强大的地区性医疗保健和生物技术产业。为了确保拟议的圣裘德AP4中心的可行性和成功，我们将利用来自这些组织的众多个人的投入，他们拥有临床翻译和基础生物医学科学方面的专业知识，来自慈善机构、风险资本、政府和其他来源的筹款努力，与政治人物、地方和联邦政府机构有效互动的能力，知识产权的保护和许可，PIE临床和临床开发新的治疗、预防和诊断技术，以及营销和商业化战略。这些地区利益集团的关键参与部分也是因为认识到，即使有AP4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在AP4中心内开发的选定的“小市场”技术寻找合作伙伴，这就需要有一个完善的基础设施来促进新的营利性开发，以便将这些用于更好地管理儿童癌症的新方法商业化。

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.