Accelerated Funding Advances Potential for Revolutionizing Therapy for Infrequent Brain-Related Ailments
The University of Rochester Medical Center (URMC) is set to extend its participation in the National Institute of Neurological Disorders and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), marking a significant step forward in advancing gene therapy clinical trials for rare neurological diseases.
URMC has made notable contributions in the field of gene and cellular therapies, particularly in immune-based cell and gene therapies for diseases such as cancers. While the medical center has received considerable attention for its work in cancer treatments, such as multiple myeloma through CAR T-cell therapy trials, this expertise provides a strong foundation for expanding into gene therapy clinical trials for rare neurological diseases.
The URMC is a member of the Empire State Cellular Therapy Consortium, a collaboration among five New York research centers aiming to expand and improve access to early-stage gene and cell therapy clinical trials. Through this consortium, URMC contributes to shared resources, expertise, and collaborative clinical trials that encompass not only cancer but also non-malignant and potentially neurological and rare diseases.
One of the lab-developed technologies that URMC has pioneered is StitchR, a new RNA technology that could reshape gene therapy approaches. This innovative technology, developed by Dr. Douglas Anderson's lab, reflects the cutting-edge research capabilities that may translate into therapies for rare neurological disorders.
Although specific published clinical trials of URMC exclusively focused on rare neurological diseases are not detailed in the current search results, their participation in cutting-edge CAR T-cell therapy trials for multiple myeloma—showing promising long-term remission outcomes—and their involvement in a collaborative consortium indicate a robust clinical trial environment and infrastructure likely supportive of gene therapy trials for rare neurological diseases in the near future.
The new funding for the program will focus on projects aimed to accelerate the study and approval of gene therapies for neurological diseases. The new funding will bring the medical center's total federal funding for the program to $5.7 million. Researchers at the University of Rochester Medical Center are poised to help accelerate the development of these drugs.
The field of neurology is undergoing a transformation, as new gene therapies could offer treatments for a wide range of rare and more common neurological and neuropsychiatric diseases. The researchers at URMC aim to overcome the recruitment and retention barriers associated with rare diseases and measure the impact of treatments based on outcomes that are important to patients and their families.
Dr. Robert Holloway, MD, MPH, serves as the principal investigator of the Rochester site, which is named UR-NEXT. Christine Annis is the program coordinator for UR-NEXT. Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White also serve as UR-NEXT co-investigators.
In summary, URMC's role in gene therapy clinical trials encompasses pioneering cancer immunotherapies and expanding into broader cell and gene therapies through a major New York state consortium. This positioning sets URMC up for meaningful contributions to rare neurological disease trials, supported by innovative technologies developed in their labs. The new funding will further bolster their efforts in this crucial area of medical research.
References: - URMC as part of the Empire State Cellular Therapy Consortium expanding gene and cell therapy clinical trials[1][2]. - URMC's CAR T-cell therapy trials in multiple myeloma showing significant clinical progress[3]. - Development of StitchR RNA technology for advancing gene therapy at URMC[4]. - Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White serve as UR-NEXT co-investigators. - Christine Annis is the program coordinator for UR-NEXT. - The focus of the new funding will be on projects aimed to accelerate the study and approval of gene therapies for neurological diseases. - The new funding will bring the medical center's total federal funding for the program to $5.7 million. - Researchers at the University of Rochester Medical Center are poised to help accelerate the development of these drugs. - The field of neurology is undergoing a transformation, as new gene therapies could offer treatments for a wide range of rare and more common neurological and neuropsychiatric diseases. - Robert Holloway, MD, MPH, is the principal investigator of the Rochester site, which is named UR-NEXT.
- The University of Rochester Medical Center (URMC) is participating in the National Institute of Neurological Disorders and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), indicating their commitment to advancing gene therapy clinical trials for rare neurological diseases.
- URMC's work in immune-based cell and gene therapies for diseases such as cancers provides a strong foundation for expanding into gene therapy clinical trials for rare neurological diseases, demonstrating the potential for cross-disciplinary advancements in science.
- The URMC's pioneering technology, StitchR, could reshape gene therapy approaches and may translate into therapies for rare neurological disorders, showcasing the importance of education-and-self-development and innovative online-education in medical research.
- Researchers at the URMC are striving to overcome recruitment and retention barriers associated with rare diseases, emphasizing the role of health-and-wellness, fitness-and-exercise, and learning in their approach to improving the lives of patients and their families facing neurological disorders.
