Leukemia Drug Safe and Effective as Treatment for Dementia with Lewy Bodies

Tasigna (nilotinib; Novartis, East Hanover, NJ) treatment was safe and effective in individuals with dementia with Lewy bodies (DLB) according to study results presented at the 17th annual Clinical Trials on Alzheimer’s Disease (CTAD) conference. Tasigna inhibits the Abelson tyrosine kinase protein, which is linked to pathways associated with alpha-synucleinopathies. Tasigna is Food and Drug Administration (FDA)-approved for the treatment of chronic phase and accelerated phase Philadelphia chromosome positive chronic myelogenous leukemia. 

The single-center, randomized, double-blind, placebo-controlled phase 2 study included 43 participants with DLB who were randomized 1:1 to receive oral Tasigna 200 mg or matched placebo once daily for 6 months, followed by a 1-month washout. 

• There were a reduced number of falls in the Tasigna group (6) vs the placebo group (21) (95% CI, 1.30 to 10.12; P=.006). 
• Scores on the 14-item cognitive subscale of the Alzheimer’s Disease Assessment Scale (ADAS-Cog14) improved by 2.8 points in the Tasigna group vs the placebo group (95% CI, 0 to 6.34; P=.037). 
• There was an improvement in the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part I (mentation, behavior, mood) scores in the Tasigna group vs the placebo group (0.9 pts; 95% CI, 0 to -2, P=.044). No differences were observed in MDS-UPDRS part II (activities of daily living) and III (motor examination) scores. 
• More adverse events occurred in the placebo group (74) vs the Tasigna group (37) (95% CI, 0.98 to 2.32; P=.054). 

"We're looking at repositioning or repurposing tyrosine kinase inhibitors for neurodegenerative diseases, including Lewy body dementia," said study author R. Scott Turner, MD, PhD, Professor at Georgetown University School of Medicine in Washington, DC. "Due to its promotion of autophagy and perhaps other potential targets, nilotinib may be useful in neurodegenerative diseases."