An FDA-approved drug that was meant to treat leukemia appears to also have other therapeutic effects. In its pilot clinical study, the drug was shown to have reduced the symptoms and improved mental health of patients with Parkinson's disease and dementia.
Nilotinib, the drug commercially available under the brand name Tasigna, was tested in a small sample population consisting 12 patients who received a small dose of the drug in the course of six months.
It was indeed the first time that a therapy has effectively reversed cognitive dysfunction in patients who suffer neurodegenerative diseases, according to Fernan Pagan, M.D., researcher and associate professor of neurology in Georgetown University Medical Center (GUMC), as shared by IBTimes. He added that, however, there is still a need to conduct larger studies before confirming the true impact of the drug, as there were only a few patients who received the treatment.
As stated in the press release, the daily dose of nilotinib was set within the range of 150 to 300 mg. Among all patients who completed the trial, 11 people reported benefits and improvements. Patients with Parkinson's disease showed positive changes in terms of the level of biomarkers measured in their cerebrospinal fluid (CSF). These biomarkers include alpha-synuclein, amyloid beta-40/42, dopamine, Tau and p-Tau.
According to previous studies, a decrease in alpha-synuclein and amyloid beta-40/42 indicate worse Parkinson's, while an increase in Tau and p-Tau in CSF would signify a likely onset of dementia. However in the study's findings, the levels of these biomarkers had gone the opposite, suggesting harmful accumulative proteins in the brain were cleared out, and that the patients experienced neurological improvements.
NPR reports that the dose used, which was set below the required dose of 800 mg daily to treat cancer, was found well tolerated in patients with no observable side effects. But above all these, the observed efficacy in mental health, motor and non-motor skills was the most promising. Researchers noted that three patients who have difficulties in speech were able to initiate talks, and one patient who initially was restrained in a wheelchair was able to walk again.
One great testimony of the drug's efficacy was Alan Hoffman, Ph.D., a retired professor from Georgia Stae University, who was diagnosed with Parkinson's disease in 1997. He joined the study and after trying, he said: "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 the responsibility for grilling."
Now, Hoffman and other patients were allowed to continue taking the drug as part of the extended research. Georgetown researchers are now heading towards larger clinical trials for nilotinib. They hope to use the drug with other patients with other neurodegenerative diseases such as Alzheimer's, most likely to be pursued in 2016.
