01 June 2020

Nilotinib against Alzheimer's

Cancer pills have proven effective in treating Alzheimer's disease

Svetlana Maslova, Hi-tech+

American scientists presented the results of the second phase of clinical trials of the drug nilotinib, which is now widely used in the treatment of leukemia. In people with Alzheimer's disease, it has shown not only good tolerance, but also a decrease in the clinical manifestations of the incurable disease. This is the first oral treatment that reduces the signs of the disease, the authors said.

Article by Turner et al. Nilotinib Effects on Safety, Tolerance, and Biomarkers in Alzheimer's Disease is published in the journal Annals of Neurology – VM.

In the second phase of a placebo-controlled clinical trial, scientists from Georgetown University involved 37 patients with mild Alzheimer's disease. For a year, one group of volunteers took the drug nilotinib in tablets: first at a dosage of 150 mg, and then 300 mg once a day. The results were compared with the placebo group.

Nilotinib showed safety and good tolerability in all participants. In some cases, with a higher dosage, sharp mood swings were recorded in the volunteers.

The scientists then analyzed changes in the level of beta-amyloids in the brain using MRI. The accumulation of toxic beta-amyloid plaques is now considered the main sign of the disease and its severity, as well as a probable cause of dementia.

In people who took nilotinib, the level of beta-amylodes in the brain was reduced. When scientists analyzed samples of patients' cerebrospinal fluid, they also found a decrease in their concentration.

"The results are consistent with data from previous studies that showed the modifying properties of nilotinib for the treatment of manifestations of Alzheimer's disease," commented study co–author Charbel Moussa.

It is likely that nilotinib penetrates the blood-brain barrier of the brain and triggers the process of autophagy in neurons, thanks to which there is a purification from "toxic debris", scientists suggest.

"This is the first oral treatment that shows a decrease in the level of beta-amyloids in the brain," added Scott Turner, the main author of the research. At the moment, the team is preparing the next stage of research, where the dosage of 150 mg will be evaluated on a larger sample.

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