Training in the Design and Development of Infectious Disease Therapeutics

项目摘要/摘要 传染病仍然是全世界儿童死亡的主要原因。新疗法迫在眉睫 需要取代目前因抗药性和新出现的威胁而受到影响的治疗方法。 当前的新冠肺炎疫情完美地说明了这一紧迫性。圣犹大儿童研究医院 SJCRH是培养这方面博士后人才的理想儿科研究和治疗中心。这个 医院的翻译和协作研究环境与其专业知识和基础设施相结合 在成功的治疗开发方面有经过验证的记录。我们的儿科病人，特别是那些 治疗癌症，通常是免疫功能受损的人，面临着各种感染源的风险 以及在没有免疫清除的情况下进化更快的病原体。SJCRH已经开发出一种国际化的 公认的传染病计划，可追溯到50多年前的建立。本节目 包括细菌、病毒和真菌的治疗和预防策略方面的主要研究工作 感染剂。该培训计划利用了这些优势和开发新技术的强大历史 为博士后研究员提供独特和强化的研究培训。书中的16位导师 这些项目来自传染病系、结构生物学系、化学生物学系和 治疗学、免疫学和药学。他们为此带来了互补的专业知识 企业不仅寻求了解感染的生物学，而且还努力开发治疗方法 与他们战斗。这些专业知识包括药物发现、高通量筛选、药物化学、 对感染和治疗的免疫反应，以及疫苗平台和佐剂。这些努力与以下方面有关 在GMP生产和临床试验方面拥有一流的现场能力。该计划的总体目标 是表征致病机制，确定治疗干预的目标，并开发 通过最先进的治疗方法取得进展的疫苗和先导药物化合物 将基础设施发展为安全有效的药物。教主之间的合作 为培训计划提供跨学科的方法。此外，4名临床合作者将提供 患者护理、临床试验和国际医学方面的培训，5名培训协作者将提供 专门技术方面的培训。将指导所有受训人员撰写赠款、严谨和可重复性、道德规范。 和辅导。与制药公司的互动和对国际网站的访问 SJCRH全球课程是该课程不可或缺的组成部分。为了促进独立，实习生将 在导师“团队”的指导下开发自己的研究项目，并申请独立资助。我们 请求支持三名博士后实习生，该计划将继续努力招募URM实习生 为多一名市建局学员提供机构支援。最后，由一名 已经成立了外部咨询委员会，以跟踪成功情况并确定需要改进的领域。

Project Summary/Abstract Infectious diseases remain the leading cause of death of children worldwide. New therapeutics are urgently needed to replace current treatments that are being compromised by drug resistance and emerging threats. The current COVID-19 pandemic perfectly illustrates the urgency. St. Jude Children’s Research Hospital (SJCRH) is the ideal pediatric research and treatment center to train postdoctoral fellows in this effort. The hospital’s translational and collaborative research environment combined with its expertise and infrastructure have a proven track record of successful therapeutic development. Our pediatric patients, particularly those treated for cancer, are typically immunocompromised and are at risk from a wide variety of infectious agents and pathogens that evolve more rapidly without immune clearance. SJCRH has developed an internationally recognized infectious disease program that dates from its establishment over 50 years ago. This program includes major research efforts in therapeutic and prophylactic strategies for bacterial, viral and fungal infectious agents. The training program leverages these strengths and the strong history of developing new therapeutics to provide unique and enhanced research training to postdoctoral fellows. The 16 preceptors in the program are from the departments of Infectious Diseases, Structural Biology, Chemical Biology & Therapeutics, Immunology and Pharmaceutical Sciences. They bring complementary expertise to this enterprise that not only seeks to understand the biology of infection but also strives to develop therapies to combat them. This expertise includes drug discovery, high-throughput screening, medicinal chemistry, immune responses to infection and therapies, and vaccine platforms and adjuvants. These efforts are linked to on-site best in class capabilities in GMP manufacturing and clinical trials. The broad goals of the program are to characterize pathogenic mechanisms, identify targets for therapeutic intervention, and develop vaccines and lead drug compounds that progress through the state-of-the-art therapeutics development infrastructure into safe and effective medicines. Collaborations between the preceptors provide a cross-disciplinary approach to the training program. In addition, 4 clinical collaborators will provide training in patient care, clinical trials and international medicine, and 5 training collaborators will provide training in specialized techniques. All trainees will be instructed in grant writing, rigor and reproducibility, ethics and mentoring. Interactions with pharmaceutical companies and visits to international sites in conjunction with the SJCRH Global Program are integral parts of the curriculum. To promote independence, trainees will develop their own research projects guided by a ‘team’ of mentors and apply for independent funding. We request support for three postdoctoral trainees, and the program will continue its efforts to recruit URM trainees by providing institutional support for an additional URM trainee. Finally, a formal evaluation process led by an External Advisory Committee has been established to track success and identify areas for improvement.