November 14, 2024
Clinical trial results reported at a recent scientific conference suggest that the drug nilotinib may improve outcomes in people with dementia with Lewy bodies (DLB). Nilotinib is already approved by the FDA for use in a specific kind of cancer, but its use in DLB is experimental.
What was the trial about?
Due to the kinds of molecules that nilotinib interacts with in the body, it has been suspected that it may be useful in treating DLB. In this Phase 2 trial, researchers put this idea to the test. Forty-three volunteers with DLB were given either the study drug or placebo (an inactive “sugar pill”) for 6 months. At the beginning of the study, the volunteers had mild to moderate dementia and mild parkinsonism. Researchers then measured several important health outcomes, including cognition, day-to-day functioning, falls, and lab tests, to check the safety and effectiveness of the experimental treatment.
What did the study find?
Nilotinib treatment was found to be safe and well tolerated by those who took it. In comparison to the placebo group, the group treated with the study drug did not experience more side effects.
In addition, nilotinib treatment appeared to improve cognition in comparison to the placebo group. People taking the study drug had better scores on several cognitive tests after three months of treatment, though the differences between the treatment group and placebo group were smaller after six months of treatment. Some of the changes were relatively large, but others were small enough that researchers cannot be sure yet they are not due to random chance in this relatively small group of volunteers.
Notably, the treatment group had fewer falls, a major driver of health problems and healthcare costs for people with DLB.
The researchers also asked the care partners of the study volunteers to rate the volunteers’ irritability, apathy, and cognitive fluctuations – symptoms that greatly affect quality of life. Care partners reported that people in the nilotinib treatment group had lower irritability, apathy, and cognitive fluctuations than the placebo group by the end of the trial.
The researchers also wanted to understand what is happening inside the body that might explain these improvements. Volunteers provided samples of cerebrospinal fluid, which is the fluid that surrounds and supports the brain within the skull. Proteins in this fluid can provide clues about what is happening inside the brain. The researchers found changes in the levels of several proteins in this fluid that are associated with DLB, supporting the idea that the study drug works through the brain to improve cognition.
The researchers also found more dopamine in this fluid in the active treatment group, suggesting that the treatment may have a beneficial effect on the parts of the brain that use dopamine to communicate with other brain areas. These parts of the brain are vulnerable in people with DLB.
Will this change medical practice?
Not yet. This study is promising, but it is too early to know whether nilotinib will be useful in DLB. This study was a Phase 2 trial with a small group of volunteers, and many drugs that show promising results in Phase 2 do not perform as expected in Phase 3 with larger groups of volunteers. However, the positive results from this study provide evidence that nilotinib should be further studied in Phase 3 trials for people with DLB.
How can I get involved?
All kinds of research studies are actively seeking volunteers today. These range from complex drug trials, like the one described here, to brief online studies that can be completed from the comfort of home. To see studies that are currently recruiting, visit the LBDA clinical trials page. If you would like to be updated by email when new studies are launched, you can sign up for the Lewy Trial Tracker.