A study published in April 2017 shows two drugs—one already on the market and one being tested for cancer treatment—may be able to stop the progression of certain neurodegenerative diseases, and perhaps even improve disease-related symptoms. The drugs work by preventing a natural, but potentially destructive, self-defense on the cellular level. This protective response by the body, if not kept in check by the drugs, leads to the neurological symptoms, and eventually death.
Fighting the Body’s Own Self-Defense
Many neurodegenerative diseases develop similarly to viruses that attack a cell’s own natural proteins. For example, a cell attacked by a virus halts all protein production to protect itself, while a virus generates its viral proteins. Likewise, in many neurodegenerative diseases, damaged or “unfolded” proteins begin to overtake the cell, and the cell continues to fight back by not creating any new proteins—but for a much more extended period of time, causing the cells to die. When neurons follow this same process and die off, symptoms of impaired movement, memory loss or even death can occur.
Professor Giovanna Mallucci, of University of Cambridge and head of the studies leading to this discovery, refers to this defense mechanism—through which the cell reacts to the growth of unfolded proteins by largely stopping further production of additional proteins—as “the unfolded protein response.” She told the Alzheimer’s Society: “The response suspends much protein-making activity in the cell while it fixes the problem. If it can’t be fixed, the cell will die.”
Mallucci’s team first sought to block the unfolded protein response in mice, and prevent brain degeneration. She said, “If we can find a way to block this response with drugs, then we might be able to develop a treatment that works for people with all these different conditions.”
First Compound to Prevent Neurodegeneration
In a breakthrough study in 2013, mice with prion disease—a group of neurodegenerative conditions that cause dementia, changes in behavior, and coordination—were given an oral compound to halt the auto-defense mechanism to the unfolded proteins. According to the
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