It also significantly changed biomarkers, the toxic proteins linked to disease progression, accord to Dr. Charbel Moussa, PhD, and Dr. Fernando Pagan, PhD, both of Georgetown University Medical Center in Washington (GUMC), who conducted the preclinical research.
All patients experienced benefits, and 10 of them reported meaningful clinical improvements, but the most dramatic was in observable behavior.
Most patients experienced an improvement in cognition, motor skills and non-motor function improvement (such as constipation). One individual confined to a wheelchair was able to walk again; three others who could not talk were able to hold conversations.
On stopping the nilotinib treatment, despite restarting their L-dopa therapies, patients again underwent cognitive and motor decline.
Alan Hoffman, PhD, who participated in the study, had fallen eight times in the 6 weeks before the trial, but during the 6 months of the study, fell only once. He says that his speech and thinking are improved.
He adds:
"Before the nilotinib, I did almost nothing around the house. Now, I empty the garbage, unload the dishwasher, load the washer and the dryer, set the table, even take responsibility for grilling. My wife says it's life-changing for her and for my children and grandchildren. To say that nilotinib has made a change in our lives is a huge understatement."