Barrow Alzheimer’s Researcher Sees Reason for Optimism

A Barrow researcher is working to identify measurable indicators – known as biomarkers – of early-onset Alzheimer’s disease, which he hopes will lead to the development of novel drug treatments.

“In order to prevent or even slow the disease, you have to treat people at the earliest stages of the disease,” said Elliott Mufson, a professor in Barrow’s Division of Neurobiology. “One way of doing that is to come up with a biomarker.”

Much of Mufson’s research into biomarkers for Alzheimer’s disease has been through studies using human brain tissue from the Religious Orders Study, which began in 1994. More than 1,100 priests, nuns, and brothers agreed to undergo a clinical evaluation each year and donate their brains to the study after death.

By studying human brain tissue rather than that of animals, Mufson and the other researchers are able to compare the biological changes in the brain with cognitive changes observed in the clinic. This provides the researchers with a better idea of how to target the onset of Alzheimer’s disease.

In order to prevent or even slow the disease, you have to treat people at the earliest stages of the disease. One way of doing that is to come up with a biomarker.

-Elliott Mufson, PhD
Professor of Neurobiology

“It’s a uniquely human disorder,” Mufson said. “We can’t just recreate it in a dish or in an animal. There are no models that truly replicate all of the changes that you see in Alzheimer’s disease, which makes it harder to study. If we really want to understand the disease, we have to study the human condition.”

One of the recent findings from his laboratory was the identification of the protein proNGF as a possible biomarker. Early in the onset of Alzheimer’s disease, the levels of proNGF/NGF signaling pathways are increased within brain regions associated with memory. Changes in this protein can be measured in the cerebrospinal fluid and mark the transition from mild cognitive impairment to frank Alzheimer’s disease.

“Until we actually define the initiating factor, it’s difficult to treat the disease because all you’re doing is going after targets that appear after the disease starts,” Mufson said. “Once you find those initiating factors, you can really start to attack the disease.”

Mufson is also known for being a pioneer in the application of single-cell gene array technology to study the genetic signature of neurons during the progression of Alzheimer’s disease. He said understanding those particular genes involved in the progression of the disease could lead to targeted therapies designed to alter the dysfunctional genes.

“It’s a tricky business,” he said. “There’s no silver bullet that we have seen so far.”

However, Mufson said the progress made in Alzheimer’s research over the past 10 to 15 years is impressive. He is optimistic that within another 10 to 15 years, there will be a way to diagnose and treat people when they begin to develop mild cognitive impairment, which precedes Alzheimer’s disease.

“We’re trying to come at it through multiple approaches,” he said. “These pieces are all going to come together and eventually lead us to some treatment approach.”

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