New drug holds promise for patients

Published: March 3, 2016

Science & Tech Editor

Since the recognition of Alzheimer’s disease in 1906, scientists have raced to find a solution to this debilitating disease. Now, 110 years later, we may finally have some promising results.

A new drug, LM11A-31, has recently come to light that has proven to be successful in murine test subjects and now is entering phase II, human testing, setting it on the path for approval from the Food and Drug Administration. The drug is just one of hundreds of possible treatments that have come out in the last 15 years, but this one takes an unorthodox approach to treating Alzheimer’s disease.

Traditionally, many of the drugs designed to treat Alzheimer’s disease go after one, or both, of the major proteins involved in causing the neurological damage. Amyloid and tau are two proteins that get misfolded by neurons. Improper folding causes these proteins to accumulate in cells. They then accumulate in the cell forming structures called plaques. These plaques damage the synapses and neurons. They also can activate several neurodegenerative pathways in the brain. Overall, the damage to the cells accumulate leading to widespread damage to the brain in the frontal, parietal and temporal lobe.

Up until now, the main perogative of drug treatments was to attack the amyloid plaques and destroy as much of them as they possibly could. While the treatment works to a certain degree, none have had results that served as a cure for Alzheimer’s disease. This is where LM11A-31 comes in.

A study published in the Public Library of Science, PLoS, discusses the underlying mechanism by which drug works. Danielle Simmons, Ph.D., shows how the drug reverses the neurodegenerative effects seen in mid to late stage Alzheimer’s disease. The drug does not target plaques directly, but rather counteracts the effects that the amyloids plaques have on the cell. The drug instead alters and interferes with the amyloid-beta induced degenerative signaling pathway. Amyloid plaques activate this pathway and induce apoptosis in the cell. LM11A-31 works to inhibit this pathway by acting upon one or more of the signaling molecules involved in the pathway.

Alzheimer’s research is not a new topic, especially here on campus. For years our own Timothy Foley, Ph.D., focused on understanding Alzheimer’s disease and related neurological disorders. Foley’s research focused on understanding the biochemical side of the disease. He focused on the role of reactive oxygen species in the propagation of the disease.

Whatever the solution to preventing or treating Alzheimer’s, you can be sure it will be a combination of treatment techniques. Even now the treatment protocol outlook is that it will likely be combined with anti-amyloid and anti-tau drug therapies. The drug holds promise for providing hope to those afflicted with Alzheimer’s.

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