Toxic Brain Protein: Stopped by Cancer Drug?

At Georgetown University Medical Center, tiny dosage amounts of a Leukemia-inhibiting drug known as nilotinib, were administered to lab mice in a clinical trial meant to examine the effects of the drug on inhibiting the formation of certain proteins in the brain.
These are the same types of proteins that cause accumulation of plaques and decreased cognition in neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and Lewy Body dementia. Dr. Charbel E-H Moussa, head of the dementia laboratory at Georgetown University, stated that when nilotinib is used to treat CML, it intentionally sends cells into auto-cannibalism, referred to medically as autophagy. The end game of administering this drug to Lukemia patients is the cannibalization of their organelles, and resultantly the death of tumor cells. Moussa’s study broke ground as the first series of experiements testing these types of medications on patients suffering various nueordegenerative disorders. The theory behind using nilotinib was that a small dosage would cause the cells to clean out stores of proteins, but not send them over the edge into a state of autophagy. Mice used in their lab over-expressed alpha-Synuclein, a protein linked to plaque aggregation, and were given one Milligram of nilotinib every two days. Testing of the drug concluded that nilotinib would clean out the toxic brain proteins by causing the cells to go into a state of controlled and partial autophagy , resulting in drastically heightened movement and functionality. At the end of the experiment, Moussa hypothesized that in order for therapy of these neurological diseases to be effective, it must developed and administered as soon as possible. Later usage may result in the retardation of extracellular formation, as well as the accumulation of intracellular proteins such as Lewy bodies, which job it was for nilotinib to remove in the first place.
1) 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
2) Georgetown University Medical Center (2013, May 10). Cancer drug prevents build-up of toxic brain protein. ScienceDaily. Retrieved May 22.

By Lauren Horne

The Roskamp Institute is a 501(c)3 research facility dedicated to translating the efforts of its qualified research staff into real-world results for those suffering from neurological diseases. To learn more about our programs and to get information about donating, visit


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