“This is the epidemic of the century… where the number of cases will exponentially increase without cures.”
Dr. Nilüfer Ertekin-Taner, Professor and Enterprise Chair of Mayo Clinic’s Department of Neuroscience, explains the very latest research and treatments for Alzheimer’s and other neurodegenerative diseases before an April 2, 2024 meeting of The Economic Club of Florida.
Dr. Ertekin-Taner gave the club some shocking figures on dementia and Alzheimer’s disease:
At Mayo Florida, she has more than 200 investigators working in her lab and supervises more than 250-scientists and trainees at the Florida campus.
“Our collective mission is to find cures and diagnostics for currently incurable and undiagnosable neurologic illnesses, like dementias, like Alzheimer’s disease,” she said. “Dementia is the umbrella term, it means a person is having problems in their thinking, and it’s interfering with their day-to-day life.”
She pointed out that while Alzheimer’s was first identified more than 100-years ago, it has only recently become recognized clinically. It was thought to be a normal part of aging, but now we know it is not.
Dr. Ertekin-Taner and her lab are looking for cures not only for Alzheimer’s but also Progressive Supranuclear Palsy (PSP), a disease that progresses faster and at a younger age than Alzheimer’s.
The investigators use genetics to get information about possible risk factors, but it does not give specific information.
“Basically, think of it like the general address. There is a fire so the firefighters are called, and we say there’s a fire in this general region. That’s the kind of information that these kinds of studies give us. But they don’t tell us exactly which house is on fire. And they don’t tell us what caused the fire.”
While diagnosis has gotten better, there are still few cures.
“The answer again, lies in in the economy. The cost of putting a single drug on the market is on average over a billion dollars. So pharmaceutical companies cannot commit unless those drug targets are de-risked for them. And the groups that are going to really identify those drug targets and de-risk it for big pharmaceutical companies are academic groups. Places like Mayo Clinic and other places.”
Dr. Ertekin-Taner said Alzheimer’s, PSP, and the other forms of dementia differ from person to person. While Alzheimer’s is now treated as a single disease, she says it should be treated similarly to cancer with specific therapies for specific patients. Doctors need to apply precision medicine, which is diagnosing and treating the right patient with the right treatment at the right time.
Genes are not all researchers look to.
“There’s also a big emphasis now on environment, the exposed zone. Where you were born, what you eat, what are your other risk factors, and education? What are the things that enrich your brain, and what are those that take away from your brain? Their relationship with genes matters. And this combination either puts you at risk for diseases like Alzheimer’s disease, or may protect you against it.”
These differences in people means differences in how the disease progresses.
“If you are at risk, there are a myriad of changes in your brain. These changes occur in different pathways, like your immune system, like your vascular system. Cardiovascular health is important in preserving our brain in other areas.”
The brain changes can be detected with biomarkers in the spinal fluid and in the blood.
“What’s surprising is that there are brain perturbations that are common to both (Alzheimer’s and PSP) of these diseases. Why is that important? That’s important because that tells us that there are common pathways that affect both of these diseases, meaning we can join forces, meaning that findings in one can help the other one.”
Most dementia research has been done on people of European ancestry. But Latin Americans have half-again the risk and Blacks have double. Because of the diversity of Florida, a great deal of new research is being done here. A grant from the Florida Department of Health enabled Mayo to conduct research on biomarkers – particularly in African-Americans.
Mayo is in a unique position for such research. More than 11,000 people have donated their brains for scientific research. Additionally, the lab has thousands of blood samples and millions of patient records to provide data, and help them find solutions.
“We can combine these molecular data to identify not just genes, but how they act. How are they beneficial to us? And how are they detrimental to us? We need to combine brain studies with blood studies because the brain isn’t accessible (to us) until it’s too late.”
Dr. Ertekin-Taner said all of this data needs to be translated to knowledge.
“There are widespread brain molecular changes. We can find genes and their variants. So, the house and how the house came to be on fire, we can find cell specific disease pathways. But that’s still a way to go from finding therapies and biomarkers.”
The research has produced results though.
“These new types of biomarkers increase the diagnostic accuracy of being able to say this patient has Alzheimer’s disease by 7% or 8%,” she said.
Mayo researchers are now growing brain cells from skin cells in dishes. They also experiment with fruit flies. They apply therapeutic modalities to thousands of genes then choose a therapeutic target and apply a specific RNA treatment. Dr. Ertekin-Taner says the results give the kind of specific information drug companies need.
“We have a new drug that targets the amyloid in the brain. It’s not a silver bullet but a start of more to come.”
She said we should all take responsibility for our brain health, and she gave some guidelines:
(You can also view the entire Club meeting on YouTube.)
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