Despite great advances in understanding how the human brain works, psychiatric conditions, neurodegenerative disorders, and brain injuries are on the rise. Progress in the development of new diagnostic and treatment approaches appears to have stalled. In a special issue of the Cell Press journal Neuron, experts look at the challenges associated with "translational neuroscience," or efforts to bring advances in the lab to the patients who need them.
"A variety of global impact studies have identified brain disorders as a leading contributor to disabilities and morbidity worldwide with a critical economic, public health, and societal impact," notes Neuron Editor Dr. Katja Brose. "There is resounding agreement that we need new approaches and strategies, and there are active efforts, discussion, and experimentation aimed at making the process of therapeutic development more efficient and effective."
One paper in the issue notes that there are limited venues for stakeholders to come together in a coordinated way to address the challenges ahead. An Institute of Medicine sponsored workshop brought together leaders from industry, academia, government, and nonprofit agencies to discuss the challenges associated with creating effective treatments for brain disorders. Steve Hyman, of the Broad Institute of MIT and Harvard, and colleagues present the results of this workshop and review the challenges associated with the translational process and propose opportunities and solutions for increased collaborations to accelerate the development of needed treatments. "To ensure continued advances in brain science, partnerships between government, industry, and academic scientists are needed," says Dr. Hyman.
Another paper points to recent decisions by several large pharmaceutical companies to downsize their neuroscience research divisions, reflecting a growing view that developing drugs to treat brain diseases is more difficult and often more time consuming and expensive than developing drugs for other therapeutic areas. Changing the policies that regulate market returns for the most needed, breakthrough drugs may be necessary, according to Dr. Dennis Choi, of SUNY Stony Brook, and his co-authors. "The broader neuroscience community and patient stakeholders should advocate for the crafting and implementation of these policy changes," says Dr. Choi. "Scientific and patient group activism has been successful in keeping the development of therapies in other areas—such as HIV and cancer—appropriately on track, but this type of sector-wide activism would be a novel step for the neuroscience community."
While the challenges related to brain research are great, significant progress continues to be made. For example, one paper in the special issue examines the impressive amount of research to date regarding various protein markers—including amyloid and tau—that indicate the presence of neurodegeneration and an increased risk of developing Alzheimer's disease. "This paper highlights the remarkable advances in our ability to detect evidence of Alzheimer's disease in the brain, prior to clinical symptoms of the disease, and to predict those at greatest risk for cognitive decline," says lead author Dr. Reisa Sperling, of Brigham and Women's Hospital, Massachusetts General Hospital, and Harvard Medical School. "These new findings have implications for ongoing and future clinical trials aimed at preventing memory loss associated with Alzheimer's disease." She notes that research suggests that while amyloid accumulation is necessary but not sufficient to cause cognitive decline along the trajectory of Alzheimer's disease, emerging data suggest that amyloid may accelerate spreading of tau pathology, synaptic dysfunction, nerve cell loss, and other disruptions that can lead to cognitive impairment.
If allowed to continue with robust funding and collaboration, research on Alzheimer's disease and other neurological conditions may eventually lead to ways to prevent memory loss, learning disabilities, and a host of other devastating symptoms caused by alterations in the brain.
Neuron, Brose: Bridging the Gap
Neuron, Choi et al.: "Medicines for the Mind: Policy-Based ''Pull'' Incentives for Creating Breakthrough CNS Drugs"
Neuron, Pankevich et al. "Improving and Accelerating Drug Development for Nervous System Disorders"
Neuron, Sperling et al. "The Evolution of Preclinical Alzheimer's Disease: Implications for Prevention Trials"