Calling the human brain “complex” is an understatement. Everything we do, think, and experience is subserved by hundreds of billions of neural cells utilizing trillions of connections. For scientists working to treat neurological conditions like multiple sclerosis (MS), Alzheimer disease (AD), Parkinson disease (PD), and Huntington disease (HD), the many intricacies of the brain amplify the difficulty to find solutions. Developing new treatments for the millions of people worldwide living with neurological conditions1 proves to be challenging at every turn. The path to scientific discovery often fails to follow a straight line.
Advancing the boundaries of scientific understanding
For decades, Genentech, a member of the Roche group, has conducted neuroscience research and clinical trials aimed at exploring areas of the highest medical need. Although we’ve made great strides, the journey has not been without its challenges and setbacks along the way.
But the scientific insights we’ve gleaned along our journey — including around biomarkers, refining dosing approaches and improving clinical trial designs — have all contributed to the overall body of evidence, state of the science of therapeutic development and a better understanding of the disorders themselves. We continue to learn more about when, in whom, and for how long to intervene; and how to measure therapeutic effects on cognition, function and biomarkers at various points in the disease continuum.
For example, converging lines of bench and clinical research have indicated that the earlier patients and providers can intervene in the disease process, the more we can do to slow the progression of decline, neuronal damage, and disability. In our clinical research of neurological conditions such as MS, spinal muscular atrophy, AD and HD, we’re studying medicines at this critical point — in pre-symptomatic patients, in the earliest stages of the disease, and in younger patients with less disease burden. In AD specifically, we recently started a Phase III prevention study to evaluate the potential of an investigational anti-amyloid monoclonal antibody to slow disease progression in people with the earliest biological signs who are clinically asymptomatic. We have also pursued clinical trials in young, genetically predisposed, asymptomatic individuals with autosomal dominant familial forms of AD in an effort to intervene at very early stages to prevent the development of the disease.
Purposefully following the science
With more than a dozen investigational medicines in our pipeline, Genentech is expanding our neuroscience research — helping evaluate people at risk for or in the earliest stages of disease, studying the experiences of diverse and underrepresented populations, and applying our learnings and insights across therapeutic areas. For example, we are currently conducting a dedicated clinical trial in historically underrepresented patient populations to better understand differences in the pathophysiology of multiple sclerosis and response to a disease-modifying treatment.
Our observations, discoveries, and even disappointments over decades of time move us collectively forward — even if, in some instances, that means changing course. By remaining open to any and all insights we collect from what might otherwise be considered “setbacks,” we open the door for new knowledge across our field, with even the smallest discoveries making an immense impact on current and future research. We must maintain this steady trajectory of progress — and persistence — for as long as needed to develop transformational medicines that impact the course of neurological disorders.
To maintain progress, it is essential that we continue to listen and learn from the communities who have firsthand knowledge of the unique challenges of living with neurological conditions. Nearly 90% of our clinical trials have included patient input so we can better meet the needs of the communities.
What’s next in neuroscience?
While neuroscience research hasn’t followed a linear path, we are determined. We are committed. We will continue to ask the tough questions of ourselves, our industry and society to push the boundaries of scientific understanding and discovery. Our unrelenting perseverance fuels our vision to build a future where neurological disorders no longer limit human potential — so we can help preserve the unique qualities that make people who they are.
To learn more about Genentech’s commitment to advancing neuroscience, visit https://www.gene.com/scientists/neuroscience.
 Gooch C L, Pracht E, and Borenstein AR. The burden of neurological disease in the United States: A summary report and call to action. Ann Neurol. 2017: 81:479-484. doi:10.1002/ana.24897.