Training in the Design and Development of Infectious Disease Therapeutics

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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.