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October 2018 Newsletter: Spotlight

October 2018

Dr. Andrew Lieberman is the Co-Chair of the NNPDF Reinvention Advisory Committee. He is the Gerald D. Abrams Professor in the Department of Pathology and Director of Neuropathology at the University of Michigan Medical School. He received his BS from Duke University and his MD, PhD from the University of Maryland Medical School. He trained as a research fellow in the Neurogenetics Branch of NINDS at the National Institutes of Health and then joined the University of Michigan Medical School faculty in 2001. Dr. Lieberman’s research focuses on the mechanism of neurodegeneration in inherited neurological disorders. His laboratory uses cell culture and mouse models to explore the pathogenesis of Niemann-Pick C disease and Kennedy’s disease, a degenerative disorder of the neuromuscular system.

What inspired you to begin researching NPC?

I started working on NPC after moving to Michigan. The faculty member in my department who led the recruitment that brought me to Ann Arbor is a former member of the scientific advisory board of the Parseghian Foundation. He encouraged me to get into the field. At the time, there were many excellent cell biologists interested in NPC, but relatively few neurobiologists and no neuropathologists. He thought that we could make an important contribution to NPC research by bringing a different perspective.

What changes have you seen in the field of NPC research throughout your career?

The transformation in our understanding of NPC disease has been tremendous. Over the last decade, we have developed a deep understanding of how the NPC1 and NPC2 proteins function together as a molecular machine to facilitate the movement of cholesterol from inside lysosomes to other points within the cells. This knowledge complements the emerging realization of the importance of lysosomes, not only as cellular degradation depots, but also as critical nodes that regulate cellular energy metabolism. Our knowledge of disease mechanisms also has been facilitated by the development and characterization of an array of model systems, including a number of important mouse models and the cat model. These have played critical roles in preclinical testing of therapeutic strategies. Thanks to the work in model systems, the community now has several drugs in clinical trial.

How did you come to be involved with the NNPDF?

I was invited to join the NNPDF scientific advisory board while Dan Ory was Chair. I was delighted to get involved with a patient advocacy organization that provides critical support for patients and families. Over the years, the SAB has reviewed applications for grant funding and provided advice to the NNPDF leadership on a variety of topics. When Dan stepped down as SAB Chair, I was asked to take over that role and I was happy to do so.

What do you enjoy must about your professional role and your volunteer role with the NNPDF?

Without question, the most enjoyable aspect of working with the NNPDF has been getting to know the members. Interacting with patients who suffer with the disease we study and talking with parents and caregivers is tremendously impactful. One of the great privileges of performing disease-related research is interacting with individuals whose lives are touched by the diseases we study.

What do you think the future of NPD looks like?

From a therapeutic standpoint, this is an incredibly hopeful time. Drugs are in clinical trial that may be disease modifying for both NPC and ASMD. These activities have spurred interest from a number of additional companies that may bring alternative strategies forward for testing in patients. Moreover, the NPC and ASMD research communities are flourishing, and because of this, new ideas are being tested in preclinical model systems. Complementing all of this activity is the fact that the NPD community is fortunate to have a number of important resources. These include a reinvigorated patient advocacy organization in the NNPDF that will facilitate interaction with drug companies and government officials including federal regulators, and the development of a patient registry, which is an incredibly important resource that will be controlled by NPD families.