What’s trending in neurological drug development?

Advanced MRI scans of the brain showing neural network connections
Credit: Boston Children’s Hospital

Momentum has been growing in the field of neuroscience in our ability to understand and treat various disorders affecting the brain, central nervous system, neuromuscular network and more. So what are the key ways that researchers and drug industry collaborators are discovering new therapies for preventing or reversing neurological disease?

Experts weighed in recently to offer their insights.

This week, neuroscientists from Boston Children’s Hospital and Massachusetts therapeutics companies convened a panel to discuss trends in neurological drug development. With support from MassBio, the panel was held during a research showcase organized by the Boston Children’s Technology and Innovation Development Office, the F.M. Kirby Neurobiology Center and the Translational Neuroscience Center.

Panelists were Clifford Woolf, MB, BCh, PhD, of Boston Children’s, Lamya Shihabuddin, PhD, of Sanofi, Jim Doherty, PhD, of SAGE Therapeutics, and Christopher Walsh, MD, PhD, of Boston Children’s.

They say that the biggest opportunities in neurological drug development include:

Shifting focus from one drug target to many

Traditionally, academic and industry researchers have been locked in this mentality of searching for specific drug targets and then finding the right chemical combination to regulate that target’s function. But this approach ignores the complexity of disease — we must shift our focus to understanding how multiple targets interact with each other to contribute to health or disease.

Developing organoids 

Moving to laboratory models that utilize patient-derived, diseased cells — including lab-grown organoids — will allow researchers to work with actual disease phenotypes rather than approximations of human disease created in mouse and other animal models.

Leveraging machine learning

The rise of “omics” has generated large datasets that have just begun to be mined for patterns related to disease development. New machine learning algorithms designed to process and interpret this data will reveal new patterns and insights.

Understanding molecular domino effects

There’s been a trend of focusing on the molecular understanding of disease – it’s not enough to cure a disease in an animal model and then develop a drug based on that target. It’s about understanding all the molecular domino effects that take place in humans and how drugs can influence this complex process to prevent or roll back disease progression.

Teasing apart the individual cells of the brain

The human brain and central nervous system represents a huge variety of cells and tissue types that must be separately investigated. This is essential for us to understand such fundamental questions like: Why do neurons die? As in, what exactly happens on a molecular level between a neuron and its surrounding cells that causes that neuronal cell to die?

Reaching the human psyche 

We must develop new ways for preclinical evaluation of psychiatric drugs because the elements of the human brain and its cognitive capacity are simply not present in mice. This principle also extends to the development of new pain management drugs — in animal models, pain is measured as a reflex, but we know that pain is not a reflex, it is a sensation interpreted by the brain.

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