Commentary
Video
Nilufer Ertekin-Taner outlines how embracing the biological complexity of neurodegenerative diseases can guide the development of precision therapies akin to those used in oncology.
In an interview with Pharmacy Times®, Nilufer Ertekin-Taner, MD, PhD, FAAN, a member of the department of neurology at Mayo Clinic, discusses her plenary session "A Roadmap to Precision Medicine in Neurodegenerative Diseases," which she will be presenting at the American Academy of Neurology 2025 Annual Meeting, taking place from April 5 to April 9 in San Diego, California.
Ertekin-Taner explains the need for a precision medicine approach to neurodegenerative diseases, particularly Alzheimer disease (AD), which remains challenging due to its biological complexity. Ertekin-Taner discusses how current treatments do not account for the diverse molecular changes driving disease progression, treating all patients as if they have the same condition. However, precision medicine—already transforming cancer treatment—offers a model for tailoring therapies based on individual biological markers, according to Ertekin-Taner.
Pharmacy Times: Can you discuss the research you plan to present during your plenary session at AAN, titled “A Roadmap to Precision Medicine in Neurodegenerative Diseases”?
1. Alzheimer disease involves complex molecular changes that current one-size-fits-all treatments fail to address effectively.
2. Precision medicine—already in use for cancers—is beginning to influence Alzheimer’s care, including patient counseling based on genetic risk factors like APOE4.
3. Future therapies aim to target disease subtypes and stages, personalizing treatment to improve outcomes in neurodegenerative conditions.
Nilufer Ertekin-Taner, MD, PhD: In my talk, I'm going to discuss the complex biology of neurodegenerative diseases. The prototype for neurodegenerative diseases is, of course, Alzheimer disease (AD), the most common cause of dementia, affecting tens of millions of individuals. Through the decades, we learned quite a lot about the biology of AD and related disorders. We have learned that they go together with a lot of molecular changes in the brain, and we think that those molecular changes lead to a lot of perturbations in how the brain functions, thereby leading to the disease itself. Even though we know that diseases like AD and related disorders have such great complexity in how they present themselves in the brain, in their pathology, and in how these patients have symptoms and how we see them in the clinic, we still treat these diseases like they were a single entity because we do not have treatments that specifically address these different perturbed biological pathways. My talk is about emphasizing the biological complexity of diseases like AD and talking about the gaps that we have in the way that we are currently treating these disorders and then talking about the opportunities that we have to develop precision therapies and precision biomarkers for these disorders. By precision medicine, what I'm talking about is treating the right patient with the right therapy at the right time. We're not there yet, but the talk is about emphasizing where we're at and talking about some pathways, a roadmap of how we will arrive in the future of precision therapies for these disorders.
Pharmacy Times: Can you describe the benefits for patients from the approach of precision medicine? Are there diseases that may be most suitable for this treatment?
Nilufer Ertekin-Taner, MD, PhD: Indeed. When we talk about precision medicine, sometimes it sounds like this science fiction concept. However, even today, we are applying principles of precision medicine in disciplines like cancer. We know that today we're not talking about treating cancer with a single therapy. In fact, we're talking about treating a specific type of cancer; we're talking about treating a specific stage of cancer; and moreover, we're talking about treating cancer that has a specific molecular signature. We are therefore defining very deeply what we mean by the subtype of cancer, in fact, its molecular subtype, and we're matching that molecular subtype to the available therapies. I think it's important to emphasize that, today, we are practicing precision medicine for certain conditions. Even for AD, we have begun to utilize precision medicine principles in the context of the newly approved anti-amyloid treatments. We know that patients who have 2 copies of the risk gene APOE4 are, for example, at a higher risk of side effects from the newly approved anti-amyloid therapies. Therefore, today, we are counseling patients who may want to get on these treatments according to their apolipoprotein E genotype—that is, taking a principle out of the playbook of precision medicine and applying it today. The future for the treatment of AD may look very much like how we are treating cancers today, which means we are going to identify the biological subtypes of AD, identify the stage of disease that the patient is at, and then match treatments accordingly to get the most effective outcome.
