July 30, 2021
Ann Arbor, MI – The work of affiliates of the Michigan Alzheimer’s Disease Research Center captured the attention of the Journal of Biological Chemistry last week for their new findings which could change the trajectory of treatment for key neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, frontotemporal dementia and ALS. Currently, these diseases together affected over 6.5 million Americans, making this finding of incredible significance.
In the manuscript, Nate Safren, now Research Assistant Professor of Neurology at Northwestern University, previously a research scientist at U-M; Sami Barmada, Angela Dobson Welch and Lyndon Welch Research Professor of Neurology at U-M; and colleagues at the University of Michigan establish a new and innovative approach to identifying drugs that could someday be used to treat disease such as Alzheimer’s disease, Parkinson’s disease, and frontotemporal dementia. This approach focuses on an essential pathway called autophagy that is capable of degrading the proteins that build up in the brains of individuals with these conditions.
In the past, finding drugs that activate autophagy has been exceptionally difficult. For unknown reasons, brain cells don’t respond well to these treatments, complicating efforts to develop autophagy-related drugs for dementias. Drs. Safren and Barmada used CRISPR to create cells that allow us to ‘see’ autophagy in action for the first time, and used these cells to quickly screen through almost 30,000 drugs to find those that turn on autophagy. They then tested these drugs in human brain cells that were generated from adult stem cells, finding several that were effective.
In the big picture, the study by Drs. Safren and Barmada is important for two reasons: First, they identified a series of drugs that activate autophagy and may be repurposed for treating neurodegenerative conditions. This is exciting in and of itself. Secondly, and perhaps more importantly, they created cells that can be used by any investigator to watch autophagy and measure all aspects of the pathway, paving the way for the discovery of even more promising and potentially therapeutic drugs.
Due to the significance of this research finding, the Journal for Biological Chemistry selected this publication as an “Editors’ Pick” meaning that the faculty’s referees, associate editor, and the Editors’ Pick committee viewed this work as providing an exceptional contribution to the field, as shared by the Journal’s Editor in Chief, F. Peter Guengerich on July 21.
To read the full study, titled “Development of a specific live-cell assay for native autophagic flux” please visit the journal online here.
This work was largely funded by Ann Arbor Active Against ALS and the Robert Packard Center for ALS Research.
The Michigan Alzheimer’s Disease Research Center (Michigan ADRC) is an extension of the Michigan Alzheimer’s Disease Center at the University of Michigan. The Michigan Alzheimer’s Disease Center promotes state-of-the-art clinical care, conducts memory and aging research, and provides education and wellness programs.
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