Join HDSA TODAY at 12PM (ET) for the next HDSA Research Webinar! HDSA Human Biology Project Fellows Dr. Michael Placzek (Massachusetts General Hospital) and Dr. Simon Laganiere (Beth Israel Deaconess Hospital) will present their work on neuroimaging biomarkers in HD. Click here to register.
SAGE-718 Earns FDA Fast Track Designation
In a recent press release, Sage Therapeutics announced FDA Fast Track Designation for SAGE-718, a novel experimental therapeutic that aims to treat cognitive decline in HD and related neurodegenerative diseases. This designation is reserved to speed up development and review of experimental medications that have the potential to improve treatment options for dire health conditions and fill unmet medical needs. Fast Track designation will provide Sage the opportunity to collaborate with the FDA for comprehensive development of SAGE-718 for treatment of HD.
Cognitive decline is often an early symptom of HD that inhibits independence, day-to-day-functioning, and quality of life in affected individuals. SAGE-718 is a first-in-class neuropsychiatric drug candidate that is designed to increase activity of specific brain receptors, NMDA receptors, by mimicking the molecule that stimulates their function, 24(S)-HC, which is known to decline with onset of HD. NMDA receptors are critical for tasks like combining and linking memories, effective decision making, and others that fall under the umbrella of cognition. Sage plans to begin a Phase 2 clinical study of SAGE-718 later this year.
PREVENT-HD: Preparing for Prevention of HD
A new study at the University of Wisconsin, Madison is looking to find and track subtle but measurable changes in behavior, cognition, and emotional responses that occur before the more visible HD symptoms appear. Individuals who have been genetically tested for HD and do or do not carry the HD mutation and individuals who have recently been diagnosed with HD and are in the very early stages of the disease are invited to join PREVENT-HD, Preparing for Prevention of HD. Trial participants will complete cognitive, motor, and quality of life tasks, have a brain scan with imaging tools, and undergo a lumbar puncture for collection of Cerebrospinal Fluid (CSF). Volunteers will be compensated for their time and any travel or hotel expenses will be covered. This is not a treatment study; the results of the study will help assess whether new treatments can be tested earlier in people who are at risk for developing HD. To learn more, view the study listing in HD Trialfinder, contact the research team at hdresearch@neurology.wisc.edu, or join HDSA for a research webinar on November 3rd for a presentation from the investigators—registration available here.
