Enhanced Funding Shapes Shift Toward Innovative Therapies for Infrequent Brain Ailments
The University of Rochester Medical Center (URMC) has secured new funding for its Department of Neurology's NINDS T32 Postdoctoral Training Program in Experimental Therapeutics, marking a significant milestone in its ongoing commitment to advancing neurological research. This latest award brings the medical center's total federal funding for the program to $5.7 million.
The focus of the new funding is on projects to accelerate gene therapy studies and approvals, increase diversity in clinical trial participation, and train clinical researchers. The URMC team, led by Principal Investigator Robert Holloway, MD, MPH, will leverage this funding to continue its groundbreaking work in gene therapies for Batten disease, neuromuscular diseases such as spinal muscular atrophy, Duchenne muscular dystrophy, and myotonic dystrophy, among others.
Christine Annis serves as the program coordinator for UR-NEXT, the Rochester site named under the T32 Program. She will oversee the implementation of the new funding, ensuring its effective allocation towards the program's objectives. Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White, a community engagement specialist, will also contribute as UR-NEXT co-investigators.
The field of neurology is undergoing a transformation with the development of new gene therapies addressing the genetic mechanisms of diseases. URMC's Centre for Health + Technology (CHeT) and Clinical Trials Coordination Center has managed over 100 multi-center clinical research studies and participated in the development of nine FDA-approved compounds. CHeT researchers are leaders in harnessing digital tools to allow volunteers to participate in clinical trials remotely and the development of patient-reported outcome measures, particularly for rare diseases.
The new funding will enable the URMC team to continue its outreach efforts in the Rochester and rare disease communities to build trust and empowerment. The Medical Center also hosts several patient registries for rare diseases, providing a platform for patients to connect, share experiences, and participate in clinical trials.
In 2011, the University of Rochester Medical Center was selected to participate in the National Institute of Neurological Disorders and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT). NeuroNEXT is a network of institutions supported by NINDS to conduct clinical trials in neurological disorders. It aims to improve the efficiency and effectiveness of clinical trials by providing a centralized infrastructure for study design, trial management, and data analysis. Participating sites, like URMC, are chosen for their expertise and resources in conducting clinical trials.
For specific information about URMC's involvement and plans for gene therapy trials, it would be best to directly contact the institution or check their official website for updates on their participation in NeuroNEXT and any ongoing or planned gene therapy trials. The Medical Center is also home to three NINDS-funded Clinical Trial Readiness projects for facioscapulohumeral dystrophy, inherited neuropathies, and Batten's disease, demonstrating its commitment to advancing neurological research and improving patient outcomes.
- The new funding for the URMC's NINDS T32 Postdoctoral Training Program will be utilized to accelerate gene therapy studies and approvals, particularly focusing on neurological disorders such as Batten disease, neuromuscular diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and myotonic dystrophy.
- In health-and-wellness, URMC's Centre for Health + Technology (CHeT) is harnessing digital tools to enable participants in remote clinical trials and are leaders in developing patient-reported outcome measures, specifically for rare diseases.
- The URMC's participation in NeuroNEXT, a network for conducting clinical trials in neurological conditions, is a testament to their commitment to improving the efficiency and effectiveness of genetic therapies and treatments, aiming to improve patient outcomes for neurological-disorders.
