Career Enhancement Core

Boston Children'S Hospital, Boston MA

Investigators

Abstract

ABSTRACT/SUMMARY: The Spastic Paraplegia Centers of Excellence – Research Network (SP-CERN) - RDCRC Career Enhancement Core (CEC) is designed to address a significant gap in hereditary spastic paraplegia (HSP) research and clinical practice: The shortage of trained clinician-scientists and trial physicians with specialized expertise in HSP. This shortage is a major barrier to developing effective treatments and conducting clinical trials, which are essential for integrating new therapies into routine clinical practice. With the advent of promising therapies for HSP, it is crucial to cultivate a new generation of leaders who can advance this field. The long-term objective of this project is to develop a robust training program that equips clinician-scientists with the skills and knowledge necessary to lead future HSP research and clinical trials. This initiative will enhance the capacity to translate genetic discoveries into clinical applications, ultimately improving patient outcomes and quality of life for individuals affected by HSP. The CEC has three specific aims: (1) Prepare investigators to become Principal Investigators in HSP-focused clinical trials through the Advanced Clinical Trial Training Program, providing at least three one-year fellowships annually, including rigorous training in clinical trial design, patient recruitment, regulatory requirements, and data analysis; (2) Offer comprehensive training in clinical research through the Clinical Scientist Training Program, encompassing both clinical and laboratory-based research, by offering at least two two-year fellowships annually that cover all aspects of clinical research and provide opportunities to conduct clinically relevant laboratory projects; (3) Extend the benefits of SP-CERN’s research projects and teaching resources to a wider audience of trainees by granting access to SP-CERN’s research projects, seminars, and workshops to predoctoral, postdoctoral, and clinical trainees not directly supported by SP-CERN funds. This project aligns with the NIH’s mission to advance scientific knowledge and improve public health by addressing a critical need in HSP research. By developing the next generation of clinician-scientists, the CEC will accelerate the development and clinical integration of new therapies, enhancing the quality of life for individuals affected by HSP.