Potential Found for Cancer Drug as Oral Therapy for
Previous work with mice found cognition improved,
nerve cell loss was reduced when microtubule protein stabilized
23, 2014 - Scientists are reporting new progress on a set of compounds
initially developed for cancer treatment that shows promise as a
potential oral treatment for Alzheimer's disease. Currently, no cure
exists for the devastating neurological disease that affects more than 5
million Americans – primarily senior citizens.
In a healthy brain, the protein known as tau binds
to and stabilizes microtubules, which are cellular components made of
protein inside cells. Microtubules are critical for performing many
processes in the cell, such as growth and division.
In the brain, they are particularly important for
transporting molecules or other "cargo," such as nutrients. But in
people with Alzheimer's disease, tau doesn't bind well to microtubules
and clumps up in the brain. That leaves microtubules in disarray.
Scientists believe that this process leads to the
mental problems associated with Alzheimer’s disease, including memory
loss, dementia and ultimately nerve cell death.
These researchers, Carlo Ballatore, Kurt R. Brunden
and colleagues have previously shown that when agents that can stabilize
microtubules are given to mice with Alzheimer-like traits, their
cognition has improved, and nerve cell loss is reduced. But the
experimental compounds so far have not been good drug candidates, mainly
because they must be injected, which can be painful.
The Ballatore and Brunden teams wanted to test a
series of compounds, already identified as potential anti-fungal and
anti-cancer agents, that patients might one day be able to take orally.
They gave the compounds to mice by mouth and found
that the drugs reached the brain, took on the role of tau and stabilized
microtubules in the animals' brains. This led the scientists to conclude
that the molecules from these classes could be good oral therapy
candidates for treating Alzheimer's and related disorders.
Their study appears in ACS' Journal of Medicinal
The authors acknowledge funding from the National
Institutes of Health, the Marian S. Ware Alzheimer's program and the
Penn Medicine Neuroscience Center.
The American Chemical Society
is a nonprofit organization chartered by the U.S. Congress. With more
than 161,000 members, the ACS is the world's largest scientific society
and a global leader in providing access to chemistry-related research
through its multiple databases, peer-reviewed journals, and scientific
conferences. Its main offices are in Washington, D.C., and Columbus,
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