Research Summary #1: Dr. Opal

Puneet Opal, MD, PhD, Professor of Neurology and of Cell and Developmental Biology at Northwestern University Feinberg School of Medicine, has devoted his career to understanding and treating spinocerebellar ataxias (SCA1 and related inherited movement disorders). His research has fundamentally reshaped how SCA1 is understood by demonstrating that, although symptoms emerge later in life, the disease disrupts neurodevelopment at very early stages, suggesting that many so-called late-onset neurodegenerative disorders have developmental roots. Building on these insights, Dr. Opal’s laboratory has identified growth factor–based therapeutic strategies that improve disease phenotypes in animal models of SCA1 and is advancing these discoveries toward nanoparticle-enabled clinical translation. In parallel, his group leads efforts to identify readily accessible biomarkers needed to enable and de-risk clinical trials. Dr. Opal also founded and directs the Northwestern Ataxia Clinic, a National Ataxia Foundation Center of Excellence, integrating multidisciplinary care with translational research and serving as a hub for global ataxia patients. Why funding now matters: the field has reached a critical inflection point. Foundational biological insights are in hand, therapeutic candidates and delivery strategies are ready for translational development, and biomarker pipelines are poised to support first-in-human trials. Strategic philanthropic investment at this stage can accelerate the path from discovery to treatment, compressing timelines in a way that traditional funding mechanisms cannot and directly impacting patients living with SCA1 and related ataxias.