Precision Medicine in Alzheimer's Disease: Mayo Clinic Expert Outlines Future of Targeted Treatments
Key Insights
Current Alzheimer's treatments follow a one-size-fits-all approach despite the disease's complex biology, failing to address diverse molecular pathways driving progression in individual patients.
Precision medicine aims to classify patients by disease subtype and stage, tailoring therapies to maximize benefits and minimize risks based on biological markers, similar to modern cancer treatment approaches.
Multidisciplinary collaboration, particularly involving pharmacists, will be essential as treatment strategies evolve toward multimodal therapies that must adapt throughout the chronic progression of neurodegenerative diseases (search).
Dr. Nilufer Ertekin-Taner, a neurologist at Mayo Clinic, presented a compelling vision for the future of Alzheimer's disease (search) (AD (search)) treatment at the American Academy of Neurology (search) 2025 Annual Meeting in San Diego. Her plenary session, "A Roadmap to Precision Medicine in Neurodegenerative Diseases (search)," highlighted the urgent need to move beyond current one-size-fits-all approaches toward personalized treatment strategies.
"Even though we know that diseases like AD (search) 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, we still treat these diseases like they were a single entity," Dr. Ertekin-Taner explained in an interview with Pharmacy Times.
The Limitations of Current Treatment Approaches
Current Alzheimer's treatments primarily fall into two categories: decades-old symptomatic medications and newer anti-amyloid (search) therapies. Neither approach fully addresses the disease's biological complexity or the individual factors that influence disease progression.
"Despite the fact that each of these patients with AD (search) likely got their disease for their own set of reasons, for their own set of biological reasons, combined with their own set of environmental risk and protective factors, we treat them as though they are identical," Dr. Ertekin-Taner noted.
The recently approved anti-amyloid treatments (search) represent a step forward but still target only one of many disrupted pathways in Alzheimer's disease (search). This limited approach fails to account for the diverse molecular changes occurring in patients' brains.
Defining Precision Medicine for Neurodegenerative Diseases
Dr. Ertekin-Taner defines precision medicine as "treating the right patient with the right therapy at the right time." This approach, already transforming cancer care, aims to classify patients based on their unique biology, disease stage, and environmental factors.
"For a future treatment, we envision that this will be a situation where we can actually biologically classify the patient, we can classify the stage at which they are at, and ideally also be able to predict their responses as well as the potential side effects that they may have based on their biology," she explained.
This personalized approach would allow clinicians to:
- Target specific biological pathways disrupted in individual patients
- Match treatments to disease subtypes and stages
- Predict treatment responses and potential side effects
- Consider environmental factors affecting treatment adherence and efficacy
The Dynamic Nature of Treatment
A critical aspect of precision medicine for neurodegenerative diseases (search) is recognizing their chronic, progressive nature. Unlike static conditions, Alzheimer's evolves over time, requiring treatment strategies that adapt accordingly.
"We need to also consider the notion that a set of therapies that may be most useful at an earlier stage may not be very useful at a later stage of the disease," Dr. Ertekin-Taner emphasized. "Precision therapies and the notion of precision medicine need to take into account that treatments, treatment strategies, are expected to change along the disease continuum."
The Essential Role of Multidisciplinary Collaboration
As treatment approaches become more complex, collaboration across healthcare disciplines becomes increasingly vital. Dr. Ertekin-Taner highlighted the particular importance of pharmacists in this evolving landscape.
"For us to be able to optimize care of complex patients, like AD (search) patients and others with related neurodegenerative diseases (search), it's going to take a village," she stated. "The physicians need to work hand in hand with the pharmacist, hand in hand with other health care providers in the team, hand in hand with the patients, and hand in hand with their care providers and caregivers."
This collaborative approach will be especially crucial as multimodal therapies—potentially involving different administration routes and complex medication interactions—become more common. Pharmacists will play a central role in managing these complexities and optimizing patient care.
Early Signs of Precision Medicine in Practice
While comprehensive precision medicine for Alzheimer's remains a future goal, some elements are already emerging in clinical practice. For instance, genetic information is increasingly informing patient counseling and treatment decisions.
"We are utilizing a little bit of precision medicine principles in how we counsel our patients," Dr. Ertekin-Taner noted, referring to how genetic risk factors like APOE4 (search) status might influence discussions about anti-amyloid treatments (search).
A Future of Hope
Despite the challenges ahead, Dr. Ertekin-Taner expressed optimism about the future of neurodegenerative disease treatment. Decades of research have yielded tremendous insights into the complex biology of these conditions, identifying hundreds of potential treatment targets.
"My message is one of hope in that we expect that, in the coming decade and decades, we are going to see a significant increase in the types of treatments that we can offer our patients with neurodegenerative diseases (search), including AD (search)," she concluded. "And that we can hope and expect to practice precision approaches for our patients with these conditions, much like we are doing for cancer."
As the field advances toward this vision of personalized care, the collaboration between specialists, pharmacists, patients, and caregivers will remain essential to navigating the increasing complexity of treatment options and optimizing outcomes for those affected by these devastating diseases.