Connecting the dots in neuroscience

There are approximately 86 billion neurons in the human brain. Each of them exchanging signals with thousands of other neurons creating a network of connections and circuits forming the central nervous system, which controls almost every function of the body. When neurons stop functioning and die, the network in that part of the brain stops working properly, resulting in devastating neurodegenerative diseases. 

In the past few decades, we have seen enormous advancements in our understanding of the brain, central nervous system and neurodegeneration, with treatments becoming available for a number of neurological diseases. But with the brain, the most complex organ in the body, which governs every emotion, thought and movement, is it realistic to think our success in understanding some areas of neurology could be applied to others?

For Chris Henderson, Ph.D., Head of Research at Biogen, it is a resounding yes. “But it’s not an ephemeral exploration of the brain,” he says. “It’s about experience both in research but also in developing treatments. When you’ve done this multiple times for a number of diseases, you are able to carry that knowledge forward and start connecting the dots.”

Chris Henderson, Ph.D.
Senior Vice President and Head of Research at Biogen

Biogen’s research organization consists of research units for Neurodegenerative Diseases, Multiple Sclerosis & Neurorepair, Neuromuscular & Muscle Diseases, Ophthalmology, and Genetic & Neurodevelopmental Disorders.

“In our work, we have two types of jobs," says Chris. “One is to support the disease areas where we already have marketed drugs that are helping patients, and the other is to think to the future, to areas where we're not yet involved but where we think we could make a difference. For this second focus, we believe our greatest chance of success lies in exploiting synergistic overlap between what we have learned about disease mechanisms and therapeutic strategies in Disease A and applying it, where appropriate, to related Disease B.”

From SMA to ALS and beyond

As a biotechnology company specializing in neuroscience, Biogen is known for the development of treatments for three neurological diseases: multiple sclerosis (MS), spinal muscular atrophy (SMA) and Alzheimer’s disease.

For each of these conditions, Chris says, the company is building “a pipeline of medications we hope can more fully address patients' needs, either by taking different mechanistic approaches or by improving patient convenience and access. As one example, we are proud of the disease-modifying benefits for patients shown by the first generation of anti-inflammatory drugs for relapsing-remitting forms of MS. But we are open in saying that the progressive forms of the disease – which are closer to neurodegeneration – remain a major unmet clinical need and will likely require innovation around new targets and new biologies that are emerging from our 'human-first' strategy across all neurological diseases that show neurodegeneration.”

Another cause for optimism at Biogen is the many connections between SMA and amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. Like SMA, ALS is a neuromuscular disorder affecting neurons that control voluntary muscles.

“Our appreciation of the importance of understanding the underlying genetic elements of SMA led us to focus first on the rare genetic variants of ALS: SOD1 and C9orf72, for example. Subsequently, our deep learnings from our ALS trials at scientific, clinical and executional levels are important guideposts in planning our programs for the more common non-genetic forms of the disease as well,” Chris says. Similarly, what they've learned about SMA “has made it clear there are many other diseases, from genetic epilepsies to neurodevelopmental disorders where, although the specific genetic drivers are different, our successful experience in the rare-disease field should hopefully allow us to tailor therapeutic approaches that have a significant impact on patients.”

From Alzheimer’s to Parkinson’s disease

What can the successes and challenges encountered in the clinical development of therapies for Alzheimer’s disease tell us about the best approach to Parkinson’s disease: the next most common neurodegenerative disease? “It's a logical step for us,” Chris says. “In both cases, neurons are dying, and these are diseases that affect huge numbers of people.”

But beyond clinical trials, advances in the field of biomarkers, the physiological measurements that provide insight into biological processes in our bodies, give cause for optimism. For Alzheimer's, Biogen scientists are using biomarkers to track the efficacy of treatments targeting misfolded proteins in the brain. Taking the same approach for Parkinson's, which is similarly characterized by malfunctioning proteins, has the potential to optimize development of treatments in this area.

However, using biomarkers to advance Parkinson's solutions, “is currently more technically challenging than it is in Alzheimer's,” Chris says, “but it has also become a real focus of ours because we believe it holds a lot of promise.”

Another critical need in Parkinson’s disease is the development of clinical endpoints that can more closely and reliably measure the clinically meaningful effects of potential disease-modifying therapies. The whole industry is exploring the most productive way to combine clinical, digital and imaging data in a way that could hopefully speed the delivery of effective medicines to patients and eliminate at an early stage those that are unlikely to succeed.

From collaboration to true innovation

The fact that their work might benefit patients—including, potentially, close friends and family—has led many of the world's best scientists to join the Biogen R&D team. At the same time, though, according to Chris, most of those who come to the company are also there for its culture of collaboration. This extends beyond the Biogen walls to experts in other organizations.

“No one can do this work alone,” Chris says. “You can have all the internal know-how in the world, but you still need to have external collaborators" to make real progress in neuroscience.”

“Academic scientists, clinicians at leading medical centers, other biotech companies with specialized expertise, patients themselves—they're all critical to the work we do,” Chris says.

And because science is not owned by any one organization or individual, every success is celebrated by everyone, and every challenge is a lesson for all. “It has to be that way," he says. “These connections have gotten us to where we are today, and they're going to take us into the future.”

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