New Drug May Cure Alzheimer's Disease

An investigational drug that prevents inflammation and removes protein aggregates in the brain shows great promise for treating Alzheimer’s disease.

Amyloid fibrils tangle with each other and form abnormal clumps in the brain, which eventually cause inflammation and damage. The formation of the initial amyloid fibrils takes decades, but the process is much more rapid following the initial formation. But the process in which these fibrils can replicate without additional assistance is not fully understood.

This damage causes the traditional symptoms of Alzheimer’s disease: memory impairment, confusion, and dementia. While many clinical and preclinical trials focus on Alzheimer’s disease, there is currently no cure available for patients. Patients may be treated for different behavioral and cognition problems, but the underlying cause is not addressed.

This new drug, NTRX-07, was shown to decrease inflammation and preserve neurons and regenerative cells in the brains of mice models, according to a study presented at the Anesthesiology 2016 annual meeting.

“This drug may reduce inflammation in the brain, which is linked to Alzheimer's disease,” said lead researcher Mohamed Naguib, MD. “NTRX-07 uses a different mechanism than many other Alzheimer's drugs currently available, as it targets the cause of the disease, not just the symptoms."

The drug was originally developed to treat neuropathic pain, which has similar chronic inflammation.

“Patients who have neuropathic pain have chronic neuroinflammation,” Dr Naguib said. “This is a compound that blunts that inflammation.”

In the study, scientists tested the drug in mice models that have neurodegeneration similar to Alzheimer’s disease. They discovered that inflammation changed microglial cells in the brain, which are immune cells that remove amyloid plaques in the brain.

As the amyloid plaques aggregated, the microglia cells were not able to remove them. This was shown to cause inflammation and damage nerve cells, resulting in decreased cognitive function, according to the study.

Microglia cells have CB2 receptors on their surface, and these receptors are able to create an anti-inflammatory response. When NRTX-07 was administered to mice, removal of abnormal amyloid plaques was observed. The mice also had better memory performance and improved cognitive skills.

The drug also increases levels of SOX2, which is a protein that helps new brain cells develop, and can protect the brain in patients with Alzheimer’s disease, according to the study. In mice treated with NTRX-07, normal SOX2 levels were restored.

Mice treated with a placebo had decreased levels of the protein, inflammation, decreased removal of amyloid plaques, and poor memory performance. If these findings translate to humans, NTRX-07 could offer patients with Alzheimer’s disease a much needed treatment option.