Neurologist and senior investigator for this study, Charbel E-H Moussa, MB and PhD, head of the dementia laboratory at Georgetown University stated that when this utilized drug, nilotinib, is used to treat CML, or chronic myelogenous leukemia. When used in high enough and safe doses, it causes the cancer cells to go into a state of autophagy, pushing them to cannibalize their own organelles, which leads to the death of tumor cells.
In the study that was performed, for the first time, cancer drugs were being utilized for a different cause. Mice in the lab that over-expressed a specific protein, known as alpha-Symuclein, were given one Milligram of nilotinib every two days. Previous testing of the drug concluded that it would get rid of the toxic protein found in the brain, the cells would go into a state of autophagy and within a matter of treatments, the lab mice treated with the drug had drastically better movement and functionality than the untreated mice.
At the end of the experiment, Moussa hypothesized that in order for therapy of these neurological diseases to be effective, it must happen as soon as possible. Later usage may result in retardation of further extracellular formation, as well as the accumulation of intracellular proteins such as Lewy bodies, which was the whole point of using the Leukemia drug in the first place.
Michaeline L. Hebron, Irina Lonskaya, and Charbel E.-H. Moussa. Nilotinib reverses loss of dopamine neurons and improves motor behavior via autophagic degradation of α-synuclein in Parkinson's disease models. Hum. Mol. Genet., May 10, 2013 DOI: 10.1093/hmg/ddt192
Georgetown University Medical Center (2013, May 10). Cancer drug prevents build-up of toxic brain protein. ScienceDaily.