Toward an oral therapy for treating Alzheimer's disease: Using a cancer drug

July 23, 2014, American Chemical Society

Currently, no cure exists for Alzheimer's disease, the devastating neurological disease affecting more than 5 million Americans. But scientists are now reporting new progress on a set of compounds, initially developed for cancer treatment, that shows promise as a potential oral therapy for Alzheimer's. Their study appears in ACS' Journal of Medicinal Chemistry.

Carlo Ballatore, Kurt R. Brunden and colleagues explain that in a healthy brain, the protein known as tau binds to and stabilizes , 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 the disease, including memory loss, dementia and ultimately nerve cell death. The researchers 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.

Explore further: Brain cell activity regulates Alzheimer's protein

More information: J. Med. Chem., Article ASAP. DOI: 10.1021/jm5005623

Related Stories

Brain cell activity regulates Alzheimer's protein

February 26, 2014
Increased brain cell activity boosts brain fluid levels of a protein linked to Alzheimer's disease, according to new research from scientists at Washington University School of Medicine in St. Louis.

Cancer drug improves memory in mouse model of Alzheimer's disease

March 13, 2012
A compound that previously progressed to Phase II clinical trials for cancer treatment slows neurological damage and improves brain function in an animal model of Alzheimer's disease, according to a study in the March 14 ...

Anti-tau drug improves cognition, decreases tau tangles in Alzheimer's disease models

July 19, 2012
While clinical trial results are being released regarding drugs intended to decrease amyloid production - thought to contribute to decline in Alzheimer's disease - clinical trials of drugs targeting other disease proteins, ...

Novel protein fragments may protect against Alzheimer's

May 13, 2014
The devastating loss of memory and consciousness in Alzheimer's disease is caused by plaque accumulations and tangles in neurons, which kill brain cells. Alzheimer's research has centered on trying to understand the pathology ...

New brain protein tied to Alzheimer's disease

July 16, 2014
Scientists have linked a new protein to Alzheimer's disease, different from the amyloid and tau that make up the sticky brain plaques and tangles long known to be its hallmarks.

Recommended for you

Anxiety: An early indicator of Alzheimer's disease?

January 12, 2018
A new study suggests an association between elevated amyloid beta levels and the worsening of anxiety symptoms. The findings support the hypothesis that neuropsychiatric symptoms could represent the early manifestation of ...

One of the most promising drugs for Alzheimer's disease fails in clinical trials

January 11, 2018
To the roughly 400 clinical trials that have tested some experimental treatment for Alzheimer's disease and come up short, we can now add three more.

Different disease types associated with distinct amyloid-beta prion strains found in Alzheimer's patients

January 9, 2018
An international team of researchers has found different disease type associations with distinct amyloid-beta prion strains in the brains of dead Alzheimer's patients. In their paper published in Proceedings of the National ...

Advances in brain imaging settle debate over spread of key protein in Alzheimer's

January 5, 2018
Recent advances in brain imaging have enabled scientists to show for the first time that a key protein which causes nerve cell death spreads throughout the brain in Alzheimer's disease - and hence that blocking its spread ...

Molecular mechanism behind HIV-associated dementia revealed

January 5, 2018
For the first time, scientists have identified and inhibited a molecular process that can lead to neurodegeneration in patients with HIV, according to a Northwestern Medicine study published in Nature Communications.

Mice with frequent flier miles advance the Alzheimer's cause

January 4, 2018
Alzheimer's disease wreaks emotional havoc on patients, who are robbed of their memories, their dignity, and their lives. It's financially devastating as well: care for Alzheimer's patients is predicted to top $1 trillion ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.