January 25, 2024 | Office of the President & Chief Research Officer

Stanley Manne Children's Research Institute President's Message

Manne Research Institute's Commitment to

Advancing Pediatric Early-Phase Clinical Trials

Dear Friends and Colleagues,


As a premier pediatric medical center, our dedication to caring for children with severe and often rare diseases drives us forward and leaves us looking for new healthcare solutions. We strive to continually enhance our understanding of these diseases to provide the best treatment options. This commitment manifests in our emphasis on first-in-child early-phase clinical trials, where we explore advanced therapeutics for children with rare or difficult-to-treat diseases.


First-in-child early-phase clinical trials, while potentially high-risk and costly, are crucial in our pursuit of new treatments. We take pride in being among the few children’s medical centers in the country with the specialized capabilities to conduct these studies. For example, Drs. Ashley Plant-Fox and Angela Waanders spearhead early-phase clinical trials for rare brain tumors. Similarly, Drs. Nancy Kuntz and Vamshi Rao lead trials investigating treatments for neuromuscular disorders.

Our efforts have yielded promising results, with some of our early-phase clinical trials leading to later-stage research and FDA-approved breakthroughs. A notable example is the development of a drug to treat pediatric patients with spinal muscular atrophy. However, we need to do more and expand our efforts to provide research access to advanced therapeutics to more children. To this end, we have strategic plans to increase the number of early-phase studies we conduct, backed by our commitment to invest in our investigators, staff, and resources to strengthen our clinical research infrastructure. We do this with our commitment to equitable and diverse access to our clinical trials. We believe that diversity in our trials enhances the generalizability of our findings, promotes health equity, and reduces health disparities. Our goal is to make our trials more accessible and welcoming to diverse populations, thereby working toward reducing healthcare inequities and improving health outcomes for all children.


Guiding this evolution are Ashley Hoambrecker, Senior Director of the Office of Clinical and Community Trials, and Bill Muller, MD, PhD, Scientific Director of the Clinical and Community Trials Pillar. They believe that fostering a research culture across Lurie Children’s is crucial in translating the ideas of our basic scientists into clinical interventions. Their approach includes strategies like creating clinical research support units within our Clinical Research Services line.


Their vision is to create a structure that encourages all our clinicians, even those with limited experience in clinical research, to explore cutting-edge therapies. This approach, coupled with institutional systems that allow expertise to expand across disciplines, positions Manne Research Institute as a desirable location for early-phase trials in partnership with industry, particularly for smaller companies with limited resources.


Our commitment to providing advanced therapeutics to critically ill children has never been more crucial. Building out a world-class first-in-child early-phase clinical trials unit will provide patients with limited options access to cutting-edge therapies. To be a pediatric hospital and research institute enterprise that consistently provides new options to our most critically ill patients with complex diagnoses is to give hope to the families that there might be something better for their child and children in the future.


With kindness and respect,

Pat

Patrick C. Seed, MD, PhD, FAAP, FIDSA
President & Chief Research Officer
Stanley Manne Children’s Research Institute
Children's Research Fund Chair in Basic Science
Director, Host - Microbial Interactions, Inflammation, and Immunity (HMI3) Program
Professor of Pediatrics, Microbiology & Immunology
Northwestern University Feinberg School of Medicine
Ann & Robert H. Lurie Children’s Hospital of Chicago
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