From Concept to Clinic

Bertarelli Symposium highlights collaborative neuroscience efforts

At the 2016 Bertarelli Symposium, researchers discussed the promise of combining engineering and targeted basic biology research for rapid advancement in treatment of neurological and psychiatric disease. Confocal microscope image of sensory hair cells in the cochlea of a Beethoven mouse treated with gene therapy. Image: Charles Askew

At the 2016 Bertarelli Symposium, researchers discussed the promise of combining engineering and targeted basic biology research for rapid advancement in treatment of neurological and psychiatric disease. Confocal microscope image of sensory hair cells in the cochlea of a Beethoven mouse treated with gene therapy. Image: Charles Askew

Extraordinary progress has been made in the basic understanding of how the brain and nervous system work. Getting this knowledge to the bedside—to make a difference in the care of devastating neurological and psychiatric disorders—has been slower. But Harvard Medical School and its scientific neighbors have teams of basic researchers, engineers and clinicians who are coming together to collaborate in new ways to move neuroscience from concept to clinic.

At the 2016 Bertarelli Symposium, held on April 16 at Harvard Medical School, neuroscientists and neuroengineers from around the world gathered to hear about advances in translational neuroscience. Talks covered the spectrum from creating interdisciplinary research groups to the final stages of getting a drug to market with a special focus on two areas: autism spectrum disorders and hearing loss.

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Keynote speaker Susan Hockfield, past president of the Massachusetts Institute of Technology, described a wave of transformative science that she said is rising from the 21st century convergence of the physical and life sciences with emerging engineering and technology.

Dean of the Faculty of Medicine at Harvard University Jeffrey S. Flier agreed. “The interface of engineering and medicine is helping to bring basic biology research to the clinic at an ever faster pace,” he said. “We at Harvard Medical School are fortunate to have collaborations with our colleagues at Harvard-affiliated hospitals, at the Harvard John A. Paulson School of Engineering and Applied Sciences, and with outstanding local institutions like MIT. We especially value the collaboration with the École Polytechnique Fédérale de Lausanne in Switzerland, made possible by the Bertarelli Foundation, which is developing new treatments for neurological and psychiatric disorders.”

Symposium speakers highlighted new research supported by the Bertarelli Program in Translational Neuroscience and Neuroengineering, a joint program of HMS and EPFL.

Launched in 2010, the program is designed not just to initiate new research but also to establish cross-institutional working groups that bring complementary skills in neurobiology and engineering.

“By focusing on engineering solutions for neurological disorders, and on basic research that can be immediately applicable to patient care, these teams have made remarkable progress in just a few short years. The result of these partnerships has been translational research that neither institution would have done alone,” said David Corey, Bertarelli Professor of Translational Medical Science and HMS director of the program.

In the morning session, researchers reported on efforts to find new genetic clues to autism, to develop better imaging tools for visualizing the brain network of autistic children, and to test new treatment options for autism spectrum disorders.

In the afternoon, presenters described their work on next generation diagnostics and screening tools that could help identify hearing loss in newborns, on gene therapy strategies that could be used to treat hereditary hearing loss, and on new electronic and optical implants that could deliver sound signals directly to the auditory brainstem.

“The strength of this program results from the empowerment of scientists and physicians from diverse disciplines and backgrounds to work together,” said Ernesto Bertarelli, whose foundation funded the symposium and the joint HMS-EPFL program.

“I am excited to see how these two great universities have come together with the common goal of solving complex problems in neuroscience for the benefit of patients. This kind of partnership is, I believe, fundamental to translating great science into solutions for humanity,” Bertarelli said.

Speakers at the symposium reiterated these themes, emphasizing the importance of collaboration between the university and the biotech industry.

Additional keynote speakers Ricardo Dolmetsch, global head of neuroscience at Novartis Institutes for Biomedical Research, and Alfred Sandrock, executive vice president for neurology discovery and chief medical officer at Biogen, explored how this transformation is changing the relationships between academia, biotech and hospitals.