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Could a Drug That is Used to Treat Cancer Reverse the Effects of Parkinson’s with Dementia?

A pilot study using a drug that already has approval for treating cancer appears to have had a dramatic effect on the symptoms suffered by people with Parkinson’s disease with dementia, and a related condition called Lewy body dementia.

During the 6-month pilot at Georgetown University Medical Center, 12 patients were given nilotinib, a drug normally used to treat leukaemia. The results showed that there was an improvement in both mental function and movement of the 11 people who completed the trial, with some patients experiencing dramatic improvements. Fernando Pagan, one of the authors of the study, explained how one female patient was able to feed herself once more, while one male patient was able to walk without the aid of a walker. Furthermore, three patients began to speak again.

The idea that it might be possible to use nilotinib to treat patients with neurodegenerative diseases

The idea that it might be possible to use nilotinib to treat patients with neurodegenerative diseases was the brainchild of Charbel Moussa, an assistant professor at the university. He already understood that toxic proteins build up in certain brain cells in people who have Parkinson’s with dementia, or Lewy body dementia, and thought that this process could be reversed with nilotinib, as it has the ability to activate a system that cleans up cells by clearing out unwanted proteins. Furthermore, when exposed to nilotinib, brain cells actually appear to become healthier.

Following tests on brain cells in a Petri dish, Moussa tested his theory on transgenic mice that were almost completely paralysed as a result of Parkinson’s. The results were staggering, allowing them to regain their movement until they moved almost as well as healthy mice. As a result of these findings, Pagan, who is director of the university’s Movement Disorders Program, authorised a pilot study to determine whether the treatment was safe for patients with Parkinson’s, and to find out how much of the drug reached the brain.

While larger, placebo-controlled studies will need to be carried out in order to confirm the effectiveness of nilotinib, this cancer drug could turn out to be the first treatment that is able to stop the process that kills brain cells in a range of neurodegenerative diseases, including Parkinson’s and Alzheimer’s.

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