Modulating PN aggregase pathway may offer hope for Alzheimer’s disease

Proteostasis Therapeutics announced today that modulating a key Proteostasis Network (PN) pathway may offer a novel approach to Alzheimer’s disease, a finding that was published in the journal Cell by its scientific founders Andrew Dillin, Ph.D., and Jeffery Kelly, Ph.D. The publication describes how regulation of an aggregase pathway reduced the neurodegenerative effects of beta amyloid aggregates that are a hallmark of the disease. Proteostasis has exclusively licensed the intellectual property for modulating this pathway from the Salk Institute and is integrating it into its proprietary technology platform directed to the discovery of a new class of therapeutics, Proteostasis Regulators (PR), small molecules designed to correct defects in the PN.

Christopher Mirabelli, Ph.D., Chairman of the Board of Proteostasis, said, “Novel insights such as those described in the Cell publication provide Proteostasis with proprietary capabilities for discovering novel Proteostasis Regulators to treat neurodegenerative diseases where few treatment options currently exist, such as Alzheimer’s disease. We are working with leading scientists to help create a new field of biology defined by the Proteostasis Network. By leveraging our relationships with top academic laboratories elucidating network pathways, we are able to rapidly integrate such findings into our proprietary platform to selectively guide our drug discovery efforts.”

Proteostasis co-founder, Andrew Dillin, Ph.D., Howard Hughes Medical Institute Investigator and associate professor of molecular and cell biology at the Salk Institute for Biological Studies, said, “Our research not only demonstrates that an important pathway may be involved in protecting against Alzheimer’s disease, but also that this pathway can be regulated and manipulated.” Dr. Dillin is the author of the publication, which appears in the December 11, 2009 issue of Cell, and co-founder Jeffery Kelly, Ph.D., Lita Annenberg Hazen Professor of Chemistry at the Scripps Research Institute, is a co-author.

The team of researchers led by Dr. Dillin were able slow the cellular aging process in Alzheimer’s mice. By modulating the activity of a PN aggregase pathway which plays an important role in regulating lifespan, the scientists showed that Alzheimer’s mice were able to live up to 35% longer than controls. The experimental mice were chronologically old but biologically young and had nearly normal cognition and behavior. The study also showed that by modulating the pathway to delay the cellular aging process, the mice became more efficient at dealing with toxic beta amyloid accumulations.

Online copies of the article can be obtained at


Proteostasis Therapeutics


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