Accelerated Funding Bolsters Mission to Revolutionize Therapies for Infrequent Brain-Related Conditions
University of Rochester Medical Center Advances Gene Therapy Research for Neurological Diseases
The field of neurology is witnessing a significant transformation, with the development of new gene therapies for a wide range of rare and common neurological and neuropsychiatric diseases. At the forefront of this revolution is the University of Rochester Medical Center (URMC), which is making strides in gene therapy clinical trials, particularly in the realm of oncology.
URMC is a founding member of the Empire State Cellular Therapy Consortium, a multi-institution collaboration in New York aimed at expanding access to gene and cellular therapy clinical trials. This consortium, which shares resources, expertise, and trials, is not only accelerating the development and availability of gene therapies for cancer but also broadening these approaches towards other diseases, including neurological, autoimmune, and metabolic conditions.
URMC's contributions to the field include conducting clinical trials of gene and cellular therapies, especially CAR T-cell therapies for blood cancers such as multiple myeloma. Data from these trials shows prolonged remissions and improved survival for patients. Additionally, URMC is innovating new RNA-based gene therapy technologies, such as StitchR, which could transform gene therapy approaches, potentially including rare neurological disorders.
In the realm of rare neurological diseases, URMC is participating in a national network for conducting clinical trials. The university is one of the original institutions selected for the National Institute of Neurological Disorders and Stroke Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) in 2011. The new award brings the medical center's total federal funding for the program to $5.7 million.
Robert Holloway, MD, MPH, serves as the principal investigator of the Rochester site, which is named UR-NEXT. Emma Ciafaloni, MD, Jennifer Vermillion, MD, and Charles White serve as co-investigators for UR-NEXT. The researchers at URMC aim to help accelerate the development of these drugs by overcoming recruitment and retention barriers associated with rare diseases.
The impact of treatments will be measured based on outcomes that are important to patients and their families. Christine Annis is the program coordinator for UR-NEXT at the University of Rochester Medical Center. The new funding will focus on projects aimed to accelerate the study and approval of gene therapies.
In summary, URMC's role in gene therapy clinical trials involves conducting trials of gene and cellular therapies, participating in a state-wide consortium to expand clinical trials access, and innovating new RNA-based gene therapy technologies. Their involvement in the consortium and RNA technology development suggests they are well-positioned to contribute to the field of rare neurological diseases in the broader context of gene and cellular therapies.
- Science in the field of neurology is advancing significantly, with the University of Rochester Medical Center (URMC) playing a crucial role through conducting clinical trials for gene and cellular therapies, especially in rare neurological disorders.
- The URMC's involvement in the Empire State Cellular Therapy Consortium is not only accelerating the development of gene therapies for cancer but also broadening these approaches towards other medical-conditions like neurological, autoimmune, and metabolic conditions.
- URMC's innovative RNA-based gene therapy technologies, such as StitchR, hold potential for transforming gene therapy approaches, potentially including therapies-and-treatments for neurological disorders.
