Catastrophic neurological disorders in children are among the greatest medical challenges of our time. Many of these have a genetic contribution, often driven by a single gene mutation, particularly in pediatric cases. While tremendous advances in our understanding of the molecular basis of many of these diseases have been made, and symptom management and palliative therapies have improved survival, the underlying causes and clinical trajectory for many of these disorders remain to be addressed. A huge unmet need exists today for the majority of these disorders where no effective treatments are currently available, especially in those that are progressive and disabling in childhood. There is a unique opportunity to accelerate the development of novel therapies for seriously ill children living with these neurological disorders. The Pediatric Translational Neuroscience Initiative (PTNI) leverages the scientific strength and expertise in experimental therapeutics of St. Jude to rise to the challenge of altering the landscape of pediatric neurological disease.
PTNI in action
The Genomic Medicine Initiative (GEMINI), a St. Jude Blue Sky research program, develops gene-targeted therapies for children with rare genetic diseases, optimizing and accelerating precision medicine through focused research.
GEMINI demonstrates PTNI's integrated model in concrete terms: identifying genetic disorders, developing therapies in-house and moving to clinical investigation with control over design, safety, synthesis and timeline. More importantly, GEMINI proves St. Jude's willingness to pursue diseases that lack commercial partners — diseases where the conventional therapeutic development model simply does not work. GEMINI serves as both a signature program and a compelling narrative that illustrates PTNI's distinctive value and mission-driven commitment.
The Center for Pediatric Neurological Disease Research (CPNDR) is tasked with advancing basic research investigating complex, pressing questions relating to pediatric neurological diseases.
The Center for Experimental Neurotherapeutics (CENT) is designed to facilitate the rapid clinical development of promising treatments for catastrophic pediatric neurological diseases.
The Office of Strategy & Alliances (OSA) drives engagement and collaboration with external stakeholders to accelerate and maximize the impact of research activities conducted across PTNI
