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A Vision for Research at Mayo Clinic

By Alison Caldwell, Ph.D. Photography by Paul Flessland and Matt Meyer Illustration by Alan Daniels

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The message arrived on a Thursday evening through the patient portal.

It wasn't a typical medical question or appointment request. Instead, a patient with alcohol-related liver disease was reaching out in a moment of crisis, seeking help with his addiction to alcohol.

For Vijay Shah, M.D., the Mr. and Mrs. Ronald F. Kinney Executive Dean of Research, that message crystallized everything wrong with current approaches to healthcare and everything that needed to change.

“That patient reached out for support when and where he needed it most, but our current technologies couldn't provide that support,” says Dr. Shah.

In that moment, the patient didn’t need a prescription or a blood panel. They needed a medical team who saw them as more than a name and a set of numbers on a chart. They needed care that was designed just for them and their specific struggles and needs, including the challenge of managing alcohol cravings.

The experience led Dr. Shah to reflect on the current state of medical care, and particularly the way it prevents patients from receiving the care they need.

“That patient message drove me to ask key questions: How do we serve patients better?” he says. “Imagine if that patient had a wearable device that sent an alert to his care team when he had a craving, so someone could reach out proactively and ensure that he had the support that he needed right in that moment. How do we create those sorts of technologies, to help people before they reach a crisis point?”

To answer these questions, Dr. Shah and his team have created a vision for research at Mayo Clinic that will drive the transformation of medicine from a reactive, one-size-fits-all pipeline to a platform where healthcare is a proactive, personalized journey throughout life.

The Challenge in Healthcare Today

While medical institutions like Mayo Clinic excel at providing expert care, the broader healthcare system still operates largely in reactive mode. Patients develop symptoms, seek care and receive treatments based on population-level guidelines rather than their individual biology and circumstances.

Even when care is accessible in a timely manner, the fundamental approach remains the same: respond to disease after it manifests rather than prevent it from occurring. This model, while effective for many conditions, falls short for patients facing serious or complex diseases that might be intercepted or prevented entirely with the right tools and insights.

The solution required rethinking everything. As leader of Mayo Clinic's research enterprise, Dr. Shah developed a vision that transforms healthcare from reactive treatment to predictive prevention, aligning the institution’s discovery and translational science efforts with Mayo Clinic’s Bold. Forward. transformation of healthcare to accelerate access to new treatments and cures for patients everywhere.

“At Mayo Clinic, our research and practice are intertwined,” he says. “Everything we do must serve our primary value of putting the needs of the patient first, so that's where we focused our vision for research. We are addressing the fundamental challenge that our current system doesn’t have the cures our patients need for most serious or complex diseases.”

His perspectives have been shaped by his career working on liver disease.

“I’ve been interested in liver disease my whole life,” Dr. Shah says. “The liver is an organ of serious and complex diseases — such as cirrhosis. It’s a very complicated organ, and ripe with data, which is critical to our approach. Alcoholic liver disease holds such power over people’s lives, and our current medical technologies and approaches are not what patients need.”

Heidi Dieter, chair of Research Administration, works alongside Dr. Shah, serving as his administrative partner and overseeing many of the primary business functions of the Research shield.

“What excites me most about this vision is how it fundamentally changes our relationship with patients," says Heidi. "We're not just treating disease anymore. We're partnering with people throughout their entire life journey, using data and technology to help them remain healthy and prevent illness before it starts.”

And with 25 years of experience as a clinician and researcher, Dr. Shah brings a wealth of expertise using leading-edge digital tools, including artificial intelligence (AI), to bridge discovery science to clinical trials and beyond.

The most dramatic example of this patient-first transformation is already taking shape in how Mayo Clinic conducts clinical trials.

Revolutionizing Clinical Trials

One of the most transformative aspects of this vision involves completely reimagining clinical trials. Traditional trials face a fundamental ethical dilemma: half of participants receive an inactive treatment known as a placebo. This provides a baseline against which the effectiveness of the actual treatment is measured.

“No one participating in a trial wants to be in the placebo group, but we need to collect this data to have the most rigorous study design and truly understand treatment efficacy,” says Dr. Shah. “But what if we could change that?”

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Through AI tools and the power of Mayo Clinic Platform, a vast repository containing millions of patient records from Mayo Clinic and beyond, clinical trial teams can now conduct analyses on data from other patients with the same condition to determine the natural course of the disease without any treatment. These AI-powered control groups are called synthetic placebo arms.

“With AI and our data intelligence, we're starting to reach the point where we can collect real-world data, make synthetic placebo arms, and thereby allow our clinical trials to focus on the active intervention for all of the human patients,” says Dr. Shah. “We're not fully there yet, but we're well on our way.”

This tactic will also include the use of “digital twins” — AI-powered replicas built from individual patient data that can simulate hundreds of different treatments to determine which options might work best for any given patient. This means that clinicians will be able to match patients to the trials that are most likely to succeed for them, while simultaneously identifying which patients will benefit the most from a new treatment or trial.

As these technologies expand, there will also be opportunities to improve access to treatments by developing scalable approaches for decentralized clinical trials, bringing these opportunities into patients' own homes and communities. In short, patients anywhere around the world will be able to access Mayo Clinic-level care, where and when they need it.

“These clinical trials of the future are more patient-centric, they go faster, and they're less expensive,” Dr. Shah says. “With these advances, I believe we can reduce the time it takes to go from discovery to clinical treatment by tenfold — from 17 years down to 17 months.”

This acceleration sits within a broader framework built on three interconnected strategies that work together to deliver cures faster.  

Through the seamless integration of pioneering science discoveries, AI-powered data intelligence, and revolutionary clinical impact approaches, Mayo Clinic is creating a self-reinforcing cycle that not only accelerates the path from laboratory bench to patient bedside but also fundamentally transforms healthcare from a reactive system into a globally accessible platform for preventing and curing serious or complex diseases.

Driving Toward Cures

While these approaches represent the long-term vision for 2045, the foundation is being built today.

The Research shield has already launched two key initiatives — Precure and Genesis. Precure is aimed at intervening before patients get sick, connecting patients to critical insights that can intercept serious diseases before they manifest. Genesis’ goal is cures — using cell therapy technologies and AI-driven solutions to predict organ failure, restore organ function and eliminate the need for transplant.

As part of this effort, research teams are advancing bioengineering and manufacturing efforts, in partnership with industry experts, to design and test new therapies. Scientists are also working on advancing biosensing technology, and new trials are being launched for early detection and treatment across organ systems.

Through it all, the team is building the infrastructure and relationships necessary to make this vision a reality with strategic funding and industry partnerships to turn promising developments into new treatments and cures.

“If a researcher wants to explore a disease under Genesis, we have all the infrastructure set,” says Dr. Shah. “If a company wants to work with us to explore a new biomanufacturing approach to developing cell therapies, we can do that. It’s a scalable process.”

For Dr. Shah and his team, this isn't just about advancing medicine. It's about fundamentally changing what's possible for every patient who walks through Mayo Clinic's doors.

Detecting Cancer Risk Decades Earlier

The work of hematologist Mrinal Patnaik, M.B.B.S., with clonal hematopoiesis of indeterminate potential (CHIP) exemplifies Mayo Clinic's Precure initiative in action, shifting from reactive treatment to proactive prevention.

As we age, the DNA in our blood cells mutates due to environmental exposures like radiation, chemicals and stress. While most damaged cells are eliminated, some survive and multiply. When numbers of these mutated clones grow large enough to detect, they're classified as CHIP. This precancer stage causes inflammation and over time, significantly increases risk of blood cancers and the risk of dying from all causes, especially cardiovascular disease.

Using advanced DNA sequencing, Dr. Patnaik's team in the Center for Individualized Medicine and Division of Hematology detects CHIP mutations from a simple blood draw. AI-driven software analyzes the data and translates it into actionable insights.

“We're creating tools that can identify precancer decades before it would traditionally be diagnosed,” says Dr. Patnaik. “This gives us a crucial window to intervene.”

Since 2016, Mayo Clinic has monitored over 1,000 patients with CHIP. Under Precure, the goal is accelerating the work and scaling to 100,000 patients. His research examines why people develop CHIP, including hereditary and environmental factors, and understanding which interception strategies have the greatest impact.

CHIP research demonstrates Precure's approach: early detection followed by interception. For patients, this means knowing cancer risk decades in advance and having concrete steps to reduce it, representing a fundamental shift from treating disease to preventing it entirely.

A Legacy Worth Building

These efforts represent more than just a vision for research at Mayo Clinic. They’re part of the Bold. Forward. blueprint for a research-driven, transformed global healthcare system.

“When I think about the legacy we're building, I imagine a world where no patient has to endure what that patient with alcohol liver disease wrote to me about in his portal message, struggling alone when he needed help the most,” says Dr. Shah. “We're creating a future where serious or complex diseases don't define the end of someone's story but become preventable chapters we can rewrite.”

By 2045, Mayo Clinic envisions a new approach to medical care where serious or complex diseases are identified and intercepted before they manifest, and where interventions are tailored to each patient’s unique profile and needs.

“This research vision isn't just about Mayo Clinic becoming the global authority in healthcare innovation,” says Dr. Shah. “It's about ensuring that every person, everywhere, has access to the tools and insights they need to thrive. That's a legacy worth dedicating our lives to building.”