A new Georgetown University Medical Center clinical trial found that the cancer drug Tasigna (nilotinib) alters Alzheimer’s disease biomarkers while being safe and well-tolerated by Alzheimer’s patients.
Georgetown’s researchers were already aware that Tasigna can trigger autophagy, a process which clears out Tau, beta-amyloid and other toxic proteins from neurons. These proteins are biomarkers of Alzheimer’s as well as other neurodegenerative diseases.
The trial’s objective was to determine whether Tasigna can clear out these proteins from neurons in a safe manner which Alzheimer’s patients tolerate well. The researchers found that the drug “significantly reduced” the central nervous system amyloid burden in a safe manner which Alzheimer’s patients tolerated well.
“Nilotinib is safe and achieves pharmacologically relevant cerebrospinal fluid concentrations. Biomarkers of disease were altered in response to nilotinib treatment,” the trial’s abstract states.
Adverse events such as agitation, irritation and mood swings were noted with some higher dosages. People with irregular heartbeats were excluded from the trial since irregular heartbeat is a known side effect of Tasigna. None of the trial participants experienced any cardiac events.
Prior Georgetown studies has found Tasigna may be able to treat Parkinson’s disease and Lewy Body dementia as well.
“The data across the PD, AD and LBD studies are consistent, and demonstrate that nilotinib enters the brain and it is a potential disease-modifying drug that significantly increases the level of brain dopamine and reduces neurotoxic protein levels, including amyloid plaques and toxic tau (tangles),” said Georgetown’s Dr. Charbel Moussa.
The trial involved 37 people with mild Alzheimer’s-related dementia. Participants were selected randomly to be part of a group which received Tasigna or a group which received a placebo. The group receiving Tasigna received a 150 mg dose every day for 26 weeks, followed by a 300 mg dose every day for 26 weeks.
Georgetown researchers started exploring Tasigna’s effects on people with cognitive impairment from Parkinson’s or Lewy Body dementia in 2014. The researchers found that the drug effectively crossed the blood-brain barrier, improving motor and non-motor symptoms and raising dopamine levels in a safe way which patients tolerated well.
The next step in terms of Tasigna and Alzheimers will be a Phase 3 trial which will be larger than the recent trial. It will be a placebo-controlled, double-blind study regarding early Alzheimer’s and is currently planned to begin in the next year or two.