Cancer Drug Enables Parkinson's Patients to Walk Again

The drug nilotinib, which is already approved to treat cancer, appears to also directly target the causes of Parkinson’s disease. “We’ve seen patients at end stages of the disease coming back to life,” says Charbel Moussa of Georgetown University Medical Center in Washington DC, who led the trial. The small safety trial at Georgetown University involved 12 volunteers with Parkinson's Disease, or a similar condition called dementia with Lewy bodies. All the volunteers were at an advanced stage of disease at the start of the trial. For six months, they were given a daily oral dose of  the drug nilotinib, which is already approved in the treatment of leukemia. Nilotinib blocks a protein that drives chronic myeloid leukemia. It also blocks a protein that interferes with lysosomes – cell structures that destroy harmful proteins. Once they began taking the drug, all the volunteers began to improve, some as early as three weeks later. One woman who was barely able to move her limbs at the start of the study, was able to feed herself after five months of treatment. “We had people as stiff as a board at the start of the study who were walking around, sitting down and bending their legs by the end,” says Moussa. “You could see the elation on their faces when they saw the improvement. There wasn’t a dry eye in the room." The drug was detectable in the cerebrospinal fluid of the volunteers, which shows that it makes it through the blood-brain barrier and into the brain. The team also monitored the tau, amyloid beta and alpha-synuclein proteins that accumulate as part of Parkinson’s disease, and found that the levels of these proteins either stabilized or declined in all participants. At the same time, dopamine levels increased. Moussa’s team is now enrolling people with a range of disorders that involve accumulating brain proteins, including Alzheimer’s disease and amyotrophic lateral sclerosis, for a larger, placebo-controlled trial.