New Alzheimer's drug signals
breakthrough in human clinical trial
Published time: 3
Nov, 2016 20:13
© Edgard Garrido /
Reuters
A groundbreaking
new drug has been shown to clear away toxic clusters of protein material in the
brain thought to be a primary cause of Alzheimer's, suggesting a cure to the
disease could soon be a reality.
Alzheimer’s theory
suggests the plaque kills the healthy neurons, leading to memory loss. Results
from a small Phase 1 study confirm that the drug verubecestat can reduce levels
of beta-amyloid, a protein fragment that accumulates in sticky deposits or "plaques" in
the brains of Alzheimer's sufferers.
The trial was
primarily seeking to determine safety of verubecestat , but found it worked to
prevent the production of enzymes that cause plaque on the brain’s neurons.
A study published
in Science Translational Medicine by
Merck Research Laboratories details the results of the verubecestat trial,
which found promising results and importantly, no severe side effects. However,
conclusions won’t be drawn until phase III of the clinical trial has been
completed, which tests effectiveness and safety on a larger scale.
“It represents
well over a decade of investment in this project by many, many scientists,” team
leader Matthew Kennedy told Scientific American. “Today
there are very limited therapeutic options available for people with Alzheimer’s
disease, and those that exist provide only short-term improvement to the
cognitive and functional symptoms. They do not directly target the underlying
disease processes. There is an urgent need for [these].”
The trial involved
giving 32 early-stage Alzheimer’s patients the drug for seven days, with some
taking it for two weeks. The short term trial couldn’t render visible changes
to the plaques, but fluid samples show reduced levels of the compounds that make
up the proteins.
Verubecestat
inhibits BACE1 (Beta-site Amyloid precursor protein Cleaving Enzyme 1), an
enzyme which produces a protein (amyloid beta) that clumps together and forms
the plaques around the neurons, causing Alzheimer's.
Phase III will
trial 2,000 patients with early Alzheimer’s and 1,500 with mild to moderate
stages of the disease. The results are expected in June 2017.