Giving Compass' Take:

• Kenneth S. Kosik explains how early research suggests that a drug currently used in cancer treatment has potential as a treatment for neurodegenerative diseases like Alzheimer’s.

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Scientific success stories can sometimes occur when therapies being studied for one disease can be used to treat another.

In the case of the drug we have been studying in my lab, this is especially important because it could be used to develop a drug for Alzheimer’s. This cancer drug, lonafarnib, may be able to rid the cell of abnormal tau proteins and the clumps of tau protein called tangles that, together with other abnormal proteins called senile plaques, are the hallmarks of Alzheimer’s disease. Plaques and tangles are two structures easily observed under the microscope in the brains of deceased Alzheimer’s patients.

We used mice engineered to develop tau tangles, and showed in my lab that lonafarnib prevents their formation. This approach, called repurposing, is the use of a drug originally invented to treat one disease, but is unexpectedly effective in another. Because tau in the neurofibrillary tangles is a critical feature of Alzheimer’s, a disease that has risen to pandemic proportions worldwide, I believe this repurposed drug should be a high priority for testing in clinical trials.

One thing that is exciting about repurposing this cancer drug is that testing has clearly demonstrated its safety in humans. Society does not currently have any medication that can modify or prevent Alzheimer’s – which underscores the importance of following our results from mice to patient testing. However, many details need to be worked out before a trial can proceed and the most appropriate study subjects chosen.

Read the full article about a cancer drug as a treatment for neurodegenerative diseases by Kenneth S. Kosik at The Conversation.