Director: J. Paul Taylor, MD, PhD
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.
The Center Without Walls (CWW) leverages resources and capabilities within the broader PTNI and is available to St. Jude faculty from all departments that are engaged on relevant neuroscience research. The CWW encourages institution-wide collaborations that advance the understanding of pediatric neurological diseases. Membership is open to clinical and basic science faculty working on neuroscience-themed research and allows access to PTNI core facilities and resources. By providing technical and intellectual support, the CWW will create new capabilities and foster greater productivity.
Investigators across the institution will have opportunities to secure internal grant funding for developmental and small-scale projects that align with the goals and mission of the PTNI. These multi-year institutional grants are designed to bolster investigators as they generate preliminary data and formulate hypotheses to secure extramural support to further expand research efforts.
The strategic alliances built through PTNI will operate in two directions. PTNI scientists and clinicians will evaluate industry developments and identify promising therapeutic candidates for exploration in the Center for Experimental Neurotherapeutics (CENT). The research emerging from the Center for Pediatric Neurological Disease Research (CPNDR) will animate and incentivize industry partners to develop therapies for diseases on which we can make meaningful impact. As such, PTNI not only seeds gene targets and therapeutic candidates, but also provides access to relevant, often rare, patient populations for clinical trials.