From penicillin to potato chips, some of mankind’s greatest discoveries were found by accident. The story behind a drug called nilotinib appears to be no different.
Originally approved as a treatment for leukemia, nilotinib seems to be having notably positive effects on patients with certain types of dementia, particularly Parkinson’s disease with dementia, and Lewy body dementia.
Nilotinib’s efficacy in treating dementia was discovered in a recent pilot test of the drug, after which researcher’s related the outcomes at the Society for Neuroscience meeting in Chicago on Saturday. According to them, of the 12 patients who were given small doses of nilotinib, movement and mental function improved in 11 of them. The results of the final participant were inconclusive as they didn’t complete the six month trial.
According to Fernando Pagan, an author of the study and director of the Movement Disorders Program at Georgetown University Medical Center, the positive effects for some of the patients were dramatic. One man found he no longer needed to use his walker, one woman could once again feed herself, and three patients who were previously nonverbal began speaking again.
Pagan expressed his positive feelings toward the drug, saying, “After 25 years in Parkinson’s disease research, this is the most excited I’ve ever been.”
From here it is Pagan’s desire to confirm the drug’s efficacy in larger studies controlled with a placebo. If it is proven to protect the brain from the brain cell death that occurs in Parkinson’s, it will be looked at as a possible treatment for other neurodegenerative disorders as well, including Alzheimer’s.
The new face for this campaign is 74-year-old Alan Hoffman who lives in Virginia with his wife Nancy. First diagnosed with Parkinson’s in 1997, Alan describes his first symptom as having trouble moving his arms. His ability to walk gradually lessened and his speech began to slur. Then, a decade after his diagnosis, it began to affect his mind.
He claims his ability to read “dropped off” and that he had “no ability to focus.” His wife Nancy also noted that “He had more and more difficulty making sense.” He also became less active and unable to help with household chores.
A few weeks into his nilotinib trial, though, he began to act and feel more like his old self. According to his wife, he “improved in every way. He began loading the dishwasher, loading the clothes in the dryer, things he had not done in a long time.”
And it wasn’t all physical. Hoffman’s scores on cognitive tests improved as well. He also began making more sense when he talked to his wife and was able to focus and read books again.
Alan Hoffman’s profound breakthrough was thanks to Charbel Moussa, an assistant professor of neurology at Georgetown University and an author of the study.
Moussa had the idea to use the cancer drug specifically for Parkinson’s and Lewy Body dementia. He knew that in both diseases, toxic proteins build up in certain brain cells and kill them. He also knew that while nilotinib killed the cancer cells it came into contact with, it made brain cells healthier.
He tested his theory, first on brain cells in a petri dish and then on mice. It proved to be overwhelmingly effective both times.
And while the prospective efficacy of nilotinib is definitely great news, it may not be wise to put all your hopes into it yet. For one, it sill needs to be tested in a larger study with a control setting. Also, if the drug is confirmed to be effective and approved for widespread usage for Parkinson’s, it is still extraordinarily expensive.
But the hope is finally there for those who have been suffering from the devastating effects of the disease.
Now, it is Georgetown’s hope, a treatment for Alzheimer’s will be addressed.