CZI panelists say basic science holds key to cures for neurodegenerative diseases

A panel discussion, “The Challenge of Neurodegenerative Diseases: Will Cell Biology Hold the Answer?” hosted by the Chan Zuckerberg Initiative (CZI), yielded lively discussion about the direction of basic scientific research. The discussion was one of several satellite events held during the 2017 ASCB|EMBO meeting in Philadelphia, and panelists included 2017 ASCB President Pietro De Camilli (Yale), Frank Bradke (German Center for Neurodegenerative Diseases), Erika Holzbaur (University of Pennsylvania), Jennifer Lippincott-Schwartz (HHMI), and Steven Scherer (University of Pennsylvania).

Katja Brose, CZI’s Science Program Officer, introduced the panel and told the packed room that “there is huge potential in cell biology” to help find treatments and cures for this broad class of disorders. “Neurodegenerative disorders tend to be treated as disease subsets, but they are not,” Brose said. “There are major gaps in our understanding; we need new approaches, new ideas, new people, and new perspectives.”

Brose announced that CZI would soon be inviting scientists to apply to its Neurodegenerative Challenge Network, which will provide funding for projects that bring researchers together from across many disciplines to solve problems related to these diseases. (For information about funding opportunities through CZI visit https://chanzuckerberg.com/initiatives/rfa.)

The panelists emphasized the need for those working directly with patients to collaborate with cell biologists and to apply the principles of basic science in clinical trials. “Most clinical trials are not embedded in cell biology, and perhaps cell biologists are intimidated by neurobiologists and clinicians,” remarked De Camilli.

Lippincott-Schwartz noted that her research, conducted at HHMI’s Janelia Research Campus, “is heavy with neurobiologists but not clinicians—we need to have both.”

“We also have the ability to visualize the whole brain down to the synaptic and cellular level, but you can only understand what you are looking at if you have a deep understanding of classical cell biology,” she said. Bradke added that “basic scientific research and the clinical structures have different cultures, and we have to learn to communicate, how to ask new questions.”

Scherer, the only practicing physician on the panel, noted that clinicians are gathering myriad patient data via information technology and bioinformatics and that “cell biologists ought to jump in” and analyze it.

Holzbaur remarked that ASCB is the “absolute most exciting place to facilitate discoveries in cell biology and neurodegenerative diseases.”

The panel concluded with a Q&A session, where it became evident that not all those in attendance agreed with CZI’s ambitious goals or the practicalities of funding cross-divisional, cross-institutional research initiatives. However, many agreed that more discussion on means of breaking down research silos was needed.

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Mary Spiro is ASCB's Strategic Communications Manager.