A cancer drug has shown promise in treating an entirely different ailment -- Parkinson's Disease -- in an early stage trial, researchers say.
A drug developed by pharmaceutical company Novartis AG for treating leukemia may also help treat Parkinson's patients as evidence from the trial suggests.
Doctors from the Georgetown University Medical Center, who had hoped the drug Tasigna might halt Parkinson's unstoppable progression, reported that it appears to do even more, reversing some of the worst symptoms in 10 of the 12 patients involved in the trial.
The drug, already approved by the FDA for cancer treatment, improved cognition, motor skills and non-motor function in patients with Parkinson's disease, the university researchers reported.
"To my knowledge, this study represents the first time a therapy appears to reverse -- to a greater or lesser degree depending on stage of disease -- cognitive and motor decline in patients with these neurodegenerative disorders," said Dr. Fernando Pagan, an associate professor of neurology.
One patient confined to a wheelchair regained the ability to walk and three others individuals who were unable to speak before the trial were able to conduct conversations. However, the researchers noted that larger and more comprehensive studies and trials would be required to gauge the drug's true potential impact.
Tasigna -- generic name nilotinib -- is used in treating chronic myelogenous leukemia, and given in high doses can kill leukemia cells. At lower doses, it seemed able to help damaged brain cells flush out toxic elements that keep them from functioning properly, as seen in Parkinson's.
Parkinson's, a degenerative condition affecting an estimated 10 million people around the globe that causes tremors and motor impairment, is associated with dysfunctions in the dopamine system. Tasigna increased to the production of the brain chemical dopamine.
Because of the trial's small size, there was no control group for comparison now was there a comparison based on placebos or other existing Parkinson's drugs. The trial's main goal was to test the drug for safety.
"The use of nilotinib in doses much smaller than are used to treat cancer, which is up to 800 milligrams daily, was well tolerated with no serious side effects," Pagan explains.
For the trial the doses were kept between 150 and 300 milligrams daily, he added.
According to the researchers, one of the reasons for the trial's success was that Tasigna was able to penetrate the blood-brain barrier more effectively than existing Parkinson's drugs. Moreover, they are already planning larger clinical trials with the drug for Parkinson's and other diseases including Alzheimer's.
