SMA Biomarkers Subproject for Institution # SP0014530

Project: Research project

Project Details


DESCRIPTION (provided by applicant): There is a critical need for rapid translation of novel preclinical data into new treatments for patients with a variety of neurological diseases. Despite exponential growth of basic science knowledge, there has been only limited translation of new findings into therapeutics. There are multiple reasons that fewer than 1 in 250 molecules that undergo preclinical testing are ultimately approved for clinical use;one important factor is the difficulty in recruiting subjects who have diseases of low prevalence. This application has been developed in response to the NINDS RFA to participate as a Clinical Site in the Network for Excellence in Neuroscience Clinical Trials (NEXT). The Neurology Clinical Trials Center (NCTC) at Northwestern University's Feinberg School of Medicine (NUFSM) is a collaborative effort that coordinates faculty and resources of three major Northwestern-University-affiliated clinical facilities: Northwestern Memorial Hospital (NMH), Children's Memorial Hospital (CMH) and the Rehabilitation Institute of Chicago (RIC). The NCTC faculty is comprised of highly skilled clinician investigators with expertise across all neurological subspecialties and access to a largepatient base of diverse and well-characterized adult and pediatric populations with a variety of neurological diseases. The NCTC is uniquely qualified to participate in NEXT and to contribute to the development and successful completion of the network clinical trials and biomarker validation studies. The key personnel have extensive experience in clinical and translational research in their respective subspecialties;leadership roles in major research programs (including NINDS-funded projects);track records of cutting-edge collaborative projects;access to large and diverse populations of patients with neurological conditions;and success in enrolling patients and control subjects in research studies and clinical trials sponsored by NIH, private foundations and industry sources. The established infrastructures of our Northwestern University Clinical and Translational Sciences Institute (NUCATS) supported by an NIH-funded Clinical and Translational Science Award (CTSA) and our departmental NCTC will assure timely subject recruitment by our Center. The NCTC also has a critical mass of investigators with expertise in spinal muscular atrophy (SMA) research and access to SMA pediatric and adult populations. The NCTC has all of the required resources and equipment necessary to support NEXT projects. In addition, there is substantial expertise at Northwestern University (NU) in the design and execution of translational studies for future NEXT projects. Participation in NEXT will enable the NCTC to contribute expertise and research subjects to neurological clinical trials across multiple subspecialties in adult and pediatric populations, consolidate efforts across disciplines, and provide training for junior investigators to enable them to develop independent research careers.
Effective start/end date9/30/116/30/16


  • National Institute of Neurological Disorders and Stroke (1U10NS077271-01)


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