Enhancement Sparks Efforts to Revamp Therapeutic Terrain for Uncommon Nervous System Ailments
The University of Rochester Medical Center (URMC) is making strides in the field of gene therapy, particularly in the realm of rare neurological diseases. While the Medical Center is known for its work in cancer gene and cell therapies, there is no specific publicly available information detailing their role in gene therapy trials for rare neurological diseases or their participation in the National Institute of Neurological Disorders and Stroke’s (NINDS) NeuroNEXT network.
URMC's Department of Neurology has been at the forefront of clinical research involving gene therapies for Batten disease, spinal muscular atrophy, Duchenne muscular dystrophy, and myotonic dystrophy. The Medical Center is home to three NINDS-funded Clinical Trial Readiness projects for facioscapulohumeral dystrophy, inherited neuropathies, and Batten's disease, and several patient registries for rare diseases.
In an effort to create a clinical trial recruitment approach that reflects the priorities of the population studied, the medical center team, led by principal investigator Robert Holloway, MD, MPH, is extending its participation in the NINDS NeuroNEXT. Co-investigators Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White are also part of this initiative. Christine Annis serves as the program coordinator for UR-NEXT.
The Department of Neurology's hiring of community engagement specialist White in 2019 was a strategic move to support efforts aimed at building local partnerships to improve care, wellness, and education. This initiative is crucial in overcoming recruitment and retention barriers associated with rare diseases.
The new funding will enable the medical center team to continue its outreach efforts in the Rochester and rare disease communities to build trust and empowerment. The grant, aimed at accelerating the study and approval of a growing wave of gene therapies for rare neurological diseases, will bring the medical center's total federal funding for the program to $5.7 million.
The Center for Health + Technology (CHeT) and Clinical Trials Coordination Center at the medical 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 for remote participation in clinical trials and the development of patient-reported outcome measures, particularly for rare diseases.
The impact of treatments will be measured based on outcomes important to patients and their families. As the field of neurology undergoes a transformation, with new gene therapies offering treatments for a wide range of rare and common neurological and neuropsychiatric diseases, URMC is poised to help accelerate the development of these drugs.
However, further research is needed to confirm URMC’s role in gene therapy trials for rare neurological diseases within the NeuroNEXT network. Additional direct sources or institutional clinical trial listings would be required for a definitive answer on URMC’s involvement in NeuroNEXT and rare neurological disease gene therapy trials.
References:
[1] Empire State Cellular Therapy Consortium. (n.d.). Retrieved from https://www.esctc.org/
[2] Biomedical Genetics Group. (n.d.). Retrieved from https://www.urmc.rochester.edu/genetics/biomedical-genetics-group.aspx
[3] Gene Therapy Research Institutes in the US. (n.d.). Retrieved from https://www.genetherapynews.com/gene-therapy-research-institutes-in-the-us/
[4] NeuroNEXT. (n.d.). Retrieved from https://www.ninds.nih.gov/research/neuronext
- The University of Rochester Medical Center (URMC) is working on research related to gene therapies for rare neurological diseases, including Batten disease, spinal muscular atrophy, and Duchenne muscular dystrophy, as part of clinical trials sponsored by the National Institute of Neurological Disorders and Stroke (NINDS).
- URMC's Department of Neurology is exploring the potential of CBD as a treatment for neurological disorders, as demonstrated by their participation in three NINDS-funded Clinical Trial Readiness projects for facioscapulohumeral dystrophy, inherited neuropathies, and Batten's disease, and several patient registries for rare diseases.
- The Center for Health + Technology (CHeT) and Clinical Trials Coordination Center at URMC are utilizing digital tools, such as remote participation in clinical trials and patient-reported outcome measures, to identify and study the effectiveness of various therapies and treatments, particularly for rare neurological diseases.
