Scientists are hoping that a single drug can treat two devastating brain diseases: Parkinson's and Alzheimer's.
The drug is nilotinib, which is approved to treat a form of leukemia.
In late 2015, researchers at Georgetown University Medical Center found that small doses of the drug appeared to help a handful of people with Parkinson's disease and a related form of dementia. They'd tried the unlikely treatment because they knew nilotinib triggered cells to get rid of faulty components — including the ones associated with several brain diseases.
Results of that preliminary study generated a lot of excitement because there is currently no treatment that can slow or halt the brain damage caused by either Parkinson's or Alzheimer's.
"Our phones were basically (ringing) off the hook," says Fernando Pagan, medical director of the translational neurotherapeutics program at Georgetown.
Many researchers were cautious, though. "It was such a small trial, there was no placebo control and it really wasn't designed to assess efficacy," says J. Paul Taylor, chair of the cell and molecular biology department at St. Jude Children's Research Hospital in Memphis.
So Georgetown is launching two larger and more rigorous trials of nilotinib, both designed with input from the Food and Drug Administration. One of the trials will enroll 75 patients with Parkinson's disease, the other will enroll 42 patients with Alzheimer's.