Health In Focus
  • Alzheimer’s disease is a progressive neurodegenerative disease that currently has no known cure.
  • New treatments are being evaluated and research is ongoing to try and halt or reverse its progression.
  • One such treatment being evaluated in a new clinical trial is Nilotinib, a cancer drug used in the treatment of chronic myeloid leukemia (CML)

A new clinical trial to evaluate Nilotinib an anti-cancer drug as a possible treatment for Alzheimer's disease is being planned by researchers at Georgetown University Medical Center.

Processes Underlying Alzheimer's Disease

To understand the reason behind research exploring new treatments for Alzheimer's in general and this study in particular, it is essential to know how this disease is caused.
Cancer Drug Nilotinib Being Tested As Treatment For Alzheimer’s In New Clinical Trial

It is believed that the destruction and death of neurons (nerve cells) are responsible for most of the symptoms of the disease. Neuronal or nerve cell death is believed to be caused by the deposition of two abnormal proteins namely, beta-amyloid and tau proteins. While beta-amyloid accumulates in spaces between the nerve cells i.e. extracellularly (forming plaques), tau protein accumulates within the nerve cells (forming tangles).

Scientists are unsure what role plaques and tangles perform in Alzheimer's disease. It is hypothesized that they somehow interrupt communication between nerve cells and disrupt critical processes necessary for cells to survive.

Possible Role For Nilotinib In Alzheimer's Disease

Nilotinib minimized cognitive dysfunction by targeting two major underlying mechanisms of Alzheimer's—neuroinflammation and misfolded proteins. Nilotinib stimulates a process termed autophagy which removes the abnormal proteins beta-amyloid and tau before they have a chance to accumulate forming plaques and tangles respectively.

Dr. Turner, co-medical director of Georgetown University Medical Center's Translational Neurotherapeutics Program (TNP) and director of the Georgetown Memory Disorders Program, says, "By stimulating the brain's normal autophagic process, which clears out these misfolded proteins in cells, we hope to prevent or slow the progression of Alzheimer's. In fact, nilotinib may be a first—a broad-spectrum anti-neurodegenerative drug that targets all misfolded protein aggregates that accumulate in the brain of Alzheimer's patients. By targeting both amyloid and tau, this study may point the way to a new strategy in Alzheimer's disease treatment."

The preclinical study was done by Charbel Moussa, MD, PhD, scientific and clinical research director for Georgetown's TNP, who explains, "Nilotinib seems to activate the cell's garbage disposal machine, reduce plaques and tangles and reverse cognitive decline in animal models of Alzheimer's disease. We hope that this trial will clarify the effects of nilotinib in Alzheimer's patients." Moussa will act as the co-investigator on the Alzheimer's clinical trial. 

Outline of The Clinical Trial

The phase II clinical trial being conducted at Georgetown University Medical Center under the leadership of  R. Scott Turner, MD, PhD will be testing Nilotinib (marketed as Tasigna® for use in CML).

The clinical trial is due to begin this year, with 42 patients participating. Half of them, randomly chosen, will get an escalating dose of nilotinib, while the other half will receive a placebo. The chief aim of the study will be to test the patient safety and tolerability of the drug, and to analyze how well nilotinib decreases inflammation and to detect the presence of beta-amyloid and tau proteins in the spinal fluid. In addition, Dr. Turner and his colleagues in the Georgetown Memory Disorders Program will also be conducting cognitive and functional abilities tests on the participants.

About Alzheimer's Disease

Alzheimer's disease is a progressive, irreversible disorder of the brain, affecting memory, thought process and behavior. It is the commonest cause of dementia (impairment of cognitive process) in the elderly. Typically symptoms begin to appear around 65 years of age.

At present around 44 million people worldwide are living with Alzheimer's disease. In the United States, it is estimated to be the sixth leading cause of mortality.

In the initial stages, the disease symptoms are mild and include
  • Forgetting recent messages, names of objects or places
  • Misplacing things,
  • Asking the same question repeatedly,
  • Lack of judgment and difficulty in making decisions
  • Reluctance to try out new things
With progression, when the disease becomes severe, the person is unable to perform the simplest of tasks, and is utterly dependent on others for activities of daily living.

Presently, there is no cure for the disease but drugs can control the behavioral symptoms.

Scope Of The Clinical Trial

If the clinical trial proves successful, it may become possible to retard or possibly even reverse the cognitive decline seen in Alzheimer's and offer hope and a new lease of life for patients.

References :
  1. Alzheimer's Disease and Dementia - (
  2. Alzheimer's Disease Fact Sheet - (
  3. Alzheimer's Disease - Symptoms - (
Source: Medindia

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