Scientific success stories can sometimes occur when therapies being studied for one disease can be used to treat another.
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.
Nevertheless, it is now a good time to move promising drugs to the clinic given the recent failures of many drug trials for Alzheimer’s disease.The first step before even being aware of the cancer drug was a difficult and risky experiment we conducted that had a surprising outcome. We collected skin cells from patients with a form of dementia called frontotemporal dementia that has only neurofibrillary tangles but no senile plaques.
To test this drug we used genetically engineered mice that develop human tau tangles and then dementia. Such animals often run in circles. But when we fed these animals lonafarnib, the drug blocked the formation of the tau tangles in the brain and the abnormal behavior.
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