A Failed Cancer Drug Shows Promise Treating Animal Models of Dementia

According to a publication from Medical News Today, in animal studies, a failed cancer drug showed signs of promise in treating frontotemporal dementia.

Frontotemporal Dementia

Dementia is a blanket term for a collection of symptoms that profoundly affecting memory, thinking, and social abilities.

Frontotemporal dementia is a blanket term (not unlike “dementia” itself) for a group of conditions affecting the brain’s frontal and temporal lobes. In cases of true frontotemporal dementia, parts of these lobes will shrink and atrophy, resulting in the characteristic changes of dementia.

Because the frontal and temporal lobes are associated with personality, behavior, and language, this form of dementia rarely affects the patient’s memory. Instead, patients with this form of dementia commonly experience changes to their behavior, emotions, and decision-making abilities. Often people with the condition will become quite impulsive and engage in inappropriate behaviors, according to Dr. Kenneth Kosik – a professor of neuroscience at University of California, Santa Barbara.

About Lonafarnib

Lonafarnib is a still-experimental drug that was originally designed to fight cancer. It wasn’t very good at that, though.

What researchers later discovered, though, was that Lonafarnib acted on the same pathways as proteins coded by the gene RASD2. RASD2 has been discussed for some time as a potentially link to Huntington’s disease, another neurodegenerative condition. Researchers, on a whim and admittedly large leap of faith, decided to test the drug in mouse dementia models. Shockingly, they found that mice behaving “erratically” ten weeks into the study were behaving normally after week twenty. The team found that Lonafarnib had eased inflammation of the brain, and relieved some tissue damage.

Mice models are not always an accurate predictor of human response to a treatment, however. Mice and humans are pretty different, after all – as many important scientists have noted. But the results encountered during the Lonafarnib mouse trial piqued the interest of some physicians and dementia patients. People want to find out more.

However, anyone hoping for the drug to make its way into clinical trials for dementia will be, for now, sorely disappointed. That’s because Lonafarnib is already being tested by creator Eiger Biopharmaceuticals for use in progeria – a rare disease in its own right that causes rapid physical aging. Until the results for that trial come in and Lonafarnib is approved or rejected, no other human testing for the drug is to be allowed.


Do you think it’s right that an investigative drug can only undergo one trial at a time? Why or why not? Patient Worthy wants to hear from you!

Share this post

Follow us