Mayo Clinic is home to one of the largest and most influential comprehensive cancer centers in the world — the nation’s only National Cancer Institute-Designated Comprehensive Cancer Center with three locations in Arizona, Florida and Minnesota. For more than 50 years, Mayo Clinic has transformed cancer care through multidisciplinary collaboration, finding new and better ways to prevent, intercept, diagnose and treat cancer. 

Ahead, explore powerful examples of how Mayo Clinic is advancing cancer research and treatment, enhancing early detection, designing the next generation of cancer therapies, and expanding access to care.

Smarter Blood Test for Cancer Treatment

Mayo Clinic researchers have developed a blood test that reveals how cancer interacts with its surrounding environment, helping physicians choose the most effective treatment, with strong potential across multiple cancer types.

Today, clinicians often cannot predict who will benefit from immunotherapy because most tests look only at tumor cells. This new test goes further. Researchers mapped key “neighborhoods” of immune and support cells and used artificial intelligence to detect those patterns in DNA fragments circulating in blood.

With a single blood draw, physicians can better predict whether a patient will respond to immunotherapy or if another treatment should be considered. In studies of more than 1,300 patients across several cancers, the test outperformed standard tools. This approach helps physicians guide patients away from ineffective treatments and unnecessary side effects and move them more quickly to therapies that work.

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New Era in Lung Cancer Treatment

Recent years have seen remarkable improvements in lung cancer outcomes driven by advances in diagnostics, novel therapeutics and multidisciplinary care. One of the most significant shifts has been the move toward personalized treatment strategies.

Innovations such as molecular genotyping, genetic testing, radiomics and artificial intelligence are enhancing diagnostic capabilities, offering deeper insight into tumor biology, clinical stage and patient-specific factors. At Mayo Clinic, this diagnostic insight is accelerating the development and application of novel cancer therapeutics, ensuring innovations are translated quickly into personalized patient care.

Lung cancer is not the same disease we treated a decade ago. Outcomes have improved substantially, and a growing number of patients — even those with stage 4 disease — are living longer, better lives. Some may even be cured.

— Vamsi Velcheti, M.D., Mayo Clinic Comprehensive Cancer Center site director of Clinical Trials

Carbon ion radiation therapy represents a new frontier in precision care, delivering highly targeted radiation that may improve outcomes while minimizing side effects. Its potential to synergize with immunotherapy may enhance the immune system’s ability to recognize and attack cancer cells. Mayo Clinic in Florida is building the first carbon ion therapy facility in the Americas, located inside the Duan Family Building, scheduled to begin treating patients in 2028.

Immunotherapy continues to evolve through clinical trials exploring chimeric antigen receptor-T cell (CAR-T) therapy, engineered immune cells and mRNA-based therapeutics. Together, these innovations are redefining lung cancer care, improving outcomes and offering new hope to patients.

Data-Driven Prostate Cancer Surveillance

Traditional follow-up care after prostate cancer treatment relies on a standard schedule and manual review, which can be time-consuming and delay care.

To improve this process, Mayo Clinic investigators developed the PSA Control Tower, an intelligent system designed to transform post-treatment monitoring. After therapy, physicians monitor prostate-specific antigen (PSA) levels and watch for signs the cancer may return. The PSA Control Tower continuously analyzes these results, helping care teams identify changes earlier and allowing them to focus their expertise where it is needed most.

Powered by Mayo Clinic Platform, the system securely analyzes large volumes of de-identified data to build and refine predictive models over time. By combining advanced technology with expert clinical judgment and compassionate care, Mayo Clinic is creating a more reliable, personalized and proactive approach to prostate cancer follow-up.

Bringing World-Class Glioma Care Closer to Home

As a global leader in neurology, neurosurgery and cancer care, Mayo Clinic is advancing new ways to deliver expert glioma care to patients who may not have access to high-volume specialty centers. While care at these centers is linked to better survival, travel can be a significant barrier. To close this gap, Mayo Clinic’s neuro-oncology team is expanding the use of telehealth and remote assessments to bring specialized care closer to home.

Researchers are studying the feasibility of remotely evaluating cognitive function in patients who have received cranial radiation, an essential but potentially cognitively impactful brain tumor treatment. Using Mayo Test Drive, a secure web-based platform, patients complete self-administered assessments while researchers study whether interventions such as metformin and physical activity may help protect cognition. Additional studies are evaluating patient satisfaction with telehealth visits and the feasibility of remotely managing chemotherapy. Together, these efforts reflect Mayo Clinic’s commitment to delivering innovative, high-quality brain tumor treatment to patients anywhere.

First-in-Human Targeted Radiotherapy for Liver Cancer

Mayo Clinic has become the first institution to treat a patient with liver cancer using a promising investigational radioactive therapy in a first-in-human clinical trial. The therapy targets glypican-3, a protein found on liver cancer cells but not on healthy adult liver tissue. By focusing on this protein, the treatment is designed to precisely locate and destroy cancer cells while limiting damage to normal tissue.

This novel approach combines advanced imaging and targeted radiation in a single treatment, allowing physicians to see the cancer and treat it at the same time. Early imaging shows the therapy concentrates in tumors with minimal exposure to healthy organs, an encouraging sign as the study moves forward.

The post 5 Strategies Advancing Cancer Research and Treatment appeared first on Mayo Clinic Magazine.

The Eyes Have It: Retinal Imaging Shows Promise for Transforming Care

Every year, tens of millions of people across the United States receive routine eye exams as part of basic vision care. These retinal scans are quick, noninvasive and already embedded in everyday medical practice. What if, in that same moment, they could reveal not only the health of the eye but also early signs of stroke risk, cognitive decline and neurodegenerative disease?

“That’s the future we’re working toward,” says Oana Dumitrascu, M.D., a Mayo Clinic neurologist and physician-scientist whose research explores how the retina can serve as a window into the brain. “A tool like this could shift neurological care from reactive to proactive, from therapy when clearly symptomatic to prevention in the asymptomatic phase. It could also meaningfully address health equity, because retinal imaging is far more affordable and widely available than many of the alternatives we rely on today.”

Dr. Dumitrascu’s work sits at the intersection of neurology, ophthalmology and artificial intelligence (AI). With colleagues at Mayo Clinic in Arizona and collaborators at Arizona State University, she is applying advanced machine learning techniques to thousands of retinal images — pictures already captured every day in optometry, ophthalmology clinics and sometimes even primary care settings. Her goal is ambitious but pragmatic: to transform routine eye exams into scalable tools for assessing brain health.

In two peer-reviewed papers published in 2024, Dr. Dumitrascu and her team demonstrated that, when analyzed with AI, low-cost retinal scans can identify changes occurring in the brain before cognitive deficits are noticeable — a stage known as preclinical Alzheimer’s disease — with 85% accuracy. That type of information is traditionally obtained through expensive neuroimaging, limiting clinician’s ability to diagnose and treat Alzheimer’s earlier and more effectively.

While the results represent a major scientific advance, the idea itself took shape much earlier, during Dr. Dumitrascu’s medical training.

“One of my medical school professors once told me, ‘The eye is the window to the soul,’” she recalls. “But as I trained, I realized the eye is the window to the brain. The retina is the only place in the body where you can directly visualize blood vessels and neural tissue in living people. That insight changed everything for me.”

That realization led her to focus on what is called the eye-brain axis: the shared vascular and central neurological features of the retina and the brain. To fully understand that connection, she pursued an unconventional training path, earning dual expertise across specialties.

After completing medical school and an ophthalmology residency in Romania, Dr. Dumitrascu received an international award to continue her training in the United States. She completed a neuro-ophthalmology fellowship at UCLA’s Jules Stein Eye Institute, followed by a neurology residency at Cedars-Sinai Medical Center in Los Angeles. She later joined Mayo Clinic in Arizona to complete a fellowship in vascular neurology.

One of my medical school professors once told me, ‘The eye is the window to the soul.' But as I trained, I realized the eye is the window to the brain.

— Oana Dumitrascu, M.D.

“Each stage of my training added something essential,” she says. “I wanted a deep understanding of both the eye and the brain, especially in vascular disease and neurodegeneration, which are far more common than many people realize.”

Supported by National Institutes of Health funding, Arizona state health agencies and a Mayo Clinic-Arizona State University research alliance, Dr. Dumitrascu’s lab now combines retinal imaging, advanced AI modeling and clinical neuroscience to identify early biomarkers of stroke, vascular cognitive impairment and Alzheimer’s disease.

The next phase of the work is focused on validation and scale. Dr. Dumitrascu is planning a collaborative clinical trial that would test her retinal biomarkers prospectively in both urban medical centers and rural locations, including mobile medical settings across Arizona. By studying diverse populations in real-world settings, she hopes to demonstrate not only scientific accuracy but also real clinical utility.

“One of the most exciting aspects of this trial is where it will take place,” she says. “We want to understand how these tools perform in resource-limited environments, where access to advanced neuroimaging is scarce. That’s where the potential for impact is greatest.”

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Mayo Clinic has played a critical role in accelerating that work, providing specialized imaging equipment and access to powerful digital infrastructure and AI capabilities through Mayo Clinic Cloud. The institution’s partnership with Arizona State University has also enabled close collaboration with computer scientists focused on advanced modeling and software development — expertise essential for translating research into usable clinical tools.

Ultimately, Dr. Dumitrascu envisions a future in which retinal imaging becomes an integrated part of neurological risk assessment. An optometrist or primary care provider could one day flag early warning signs of brain disease during a routine eye exam, enabling earlier referrals, targeted prevention strategies and timely interventions, often years before symptoms emerge.

“Success for me would mean that insights from routine medical imaging measurably improve patient outcomes,” she says. “It would mean shifting our focus from treating disease after damage occurs to preventing it or detecting it early enough to change its course.”

The post The Eyes Have It: Retinal Imaging Shows Promise for Transforming Care appeared first on Mayo Clinic Magazine.

Doctors will one day diagnose and treat diseases like Alzheimer’s the same way as many cancers — by precision approaches, says physician-scientist Nilüfer Ertekin-Taner, M.D., Ph.D., a neuroscientist, behavioral neurologist and chair of the Department of Neuroscience at Mayo Clinic. “The end game is classifying patients by their disease type and putting them on the right medicine,” she says. “This is precision medicine, and this is what we’re after.”

Dr. Ertekin-Taner’s mission is to uncover diagnostics and treatments for neurodegenerative diseases that affect millions and that currently take too long to diagnose, have too few treatments and have no cures. Her work conducting groundbreaking research into the genomic underpinnings of Alzheimer’s and related conditions has already led to more accurate diagnostic approaches and more therapeutic targets.

“We have seen the field come a long way,” says Dr. Ertekin-Taner, who has watched basic science discoveries from her lab, and others around the world, advance enough to translate to patient care. “That gives me tremendous hope that the best is yet to come.”

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Tracing Genetic Pathways

Researchers first began to chip away at the genetics of neurodegenerative diseases, Dr. Ertekin-Taner says, when they discovered that certain conditions, including Alzheimer’s, amyotrophic lateral sclerosis (ALS), Lewy body dementia and Parkinson’s disease, sometimes occur in families.

But that only got them so far. The vast majority of neurodegenerative disease cases have some other contributing cause. In fact, there are hundreds of genetic factors related to these diseases. “Each patient with neurodegenerative disease gets their disease for their own set of reasons,” Dr. Ertekin-Taner says.

We have seen the field come a long way. That gives me tremendous hope that the best is yet to come.

— Nilüfer Ertekin-Taner, M.D., Ph.D.

Think of a balance scale. On one side are the genomic and environmental factors, such as exposure to air pollution, that increase one’s risk of developing a neurodegenerative disease. On the other side are protective factors. Depending on which way the scale tips, a person is either predisposed to or protected from the common, complex forms of these diseases. The only way to identify these hundreds of risk factors is by studying broad patient populations.

In her large multisite, multi-institutional projects, Dr. Ertekin-Taner leverages hundreds of thousands of brain samples from the Mayo Clinic Brain Bank and blood specimens from Mayo Clinic patients to generate deep genomic data. Thanks to the power of data repositories and infrastructure like those within Mayo Clinic Platform, she can also access clinical information, such as cognitive data and other diagnoses, to consider alongside a genetic profile. This enabled her to be among the first researchers to apply what’s called an endophenotype approach to genomic studies of Alzheimer’s disease. This approach uses measurable biological traits to trace the molecular pathways that link complex genetic features to Alzheimer’s disease.

For instance, Dr. Ertekin-Taner pioneered the use of a small protein fragment known as an amyloid beta peptide as a biomarker for Alzheimer’s. This paved the way for her and other researchers to gain a deeper understanding of the complex biology connected with different facets of this disease — essential to help diagnose and treat Alzheimer’s.

“The Mayo Clinic Department of Neuroscience leads the nation’s largest studies in understanding the molecular underpinnings of these diseases, which is fundamental for us to find cures and diagnostics,” she says.

Still, Dr. Ertekin-Taner acknowledges that currently there are “an unmanageable number of molecular targets to go after” in the effort to develop therapies for neurodegenerative diseases. That’s why, when she became enterprise chair of the department in 2023, Dr. Ertekin-Taner began the process to modernize Mayo Clinic’s 30-year-old brain bank, which contains specimens from more than 11,000 brain donors. The effort includes digitization that will help researchers connect more dots with the assistance of large-scale, artificial intelligence-enabled analytic models.

“The incredible data from the brain bank will become an incessant factory of finding therapeutic targets against which researchers and clinicians can develop therapies,” she says. “We are at a phenomenal time in research.”

A Patient-Centered Mission

Dr. Ertekin-Taner is the daughter of two neurologists who are clinical neurophysiologists. Her father is also a physician-scientist.

“We joke that everything below the neck is them,” she says, “and everything above the neck is me.”

Deciding medicine was a safer career choice than her first love of acting, she attended medical school in her home country of Turkey before seeking further training in neuroscience. “For a curious person,” Dr. Ertekin-Taner says, “the brain is the best organ to study.”

She came to the United States to continue her studies with her husband, C. Burcin Taner, M.D., now chair of the Department of Transplantation at Mayo Clinic in Jacksonville, Florida, because the country had the best opportunities in brain and transplant medicine. After about a decade at Mayo Clinic in Rochester, Minnesota, where Dr. Ertekin-Taner earned her Ph.D. in molecular neuroscience and completed two residencies and two fellowships, the couple were recruited to join Mayo Clinic in Florida, as staff members. “Mayo’s overall mission aligns with mine: discovery and innovation for the sake of patients,” she says.

Despite the demands of her research, Dr. Ertekin-Taner has continued to serve as a behavioral neurologist in the Memory Disorders Clinic at Mayo Clinic in Florida. There, she evaluates and manages patients with Alzheimer’s and other neurodegenerative diseases.

“There was never a question in my mind that I’d continue seeing patients,” she says. “Because that’s what inspires me.”

The post Pioneering a Precision Approach to Neurological Diseases appeared first on Mayo Clinic Magazine.

How Discovery Science Powers Every Breakthrough at Mayo Clinic

Mayo Clinic’s vision for research is bold: to create a future of personalized, proactive healthcare by integrating pioneering science, data intelligence and clinical impact to accelerate the path from bench to patient bedside. This transformation relies on the essential, often unseen work of pioneering research science — the “bench” side of the equation. Through deep exploration of basic biology, Mayo Clinic scientists are opening pathways to new treatments and cures.

Often, the outcomes of clinical trials are what garner the most attention from the public and from media. Vijay Shah, M.D., the Mr. and Mrs. Ronald F. Kinney Executive Dean of Research, compares clinical trials to “the lead guitarist in a band,” because they are what everyone sees, and what gets featured in major news publications.

“In most bands, there is also a very important bass player, who provides the rhythm that goes through the whole song,” says Dr. Shah, who is also recognized as the Carol M. Gatton Professor of Digestive Diseases Research, Honoring Peter Carryer, M.D. "The rhythm section is like our pioneering science — all the basic, foundational science research that we're doing."

Beginning with a strong foundation — a steady rhythm — is what allows clinicians and researchers to better understand serious and complex diseases, identify new drug targets and biomarkers, and consider new approaches to treating and curing disease.

“Pioneering science doesn't always get all the glory, but it’s so important because that's how we open up the aperture of all the science that we can tap into,” says Dr. Shah.

Without this basic science research, our knowledge will remain critically limited. “If we only do clinical trials on the science that we know, we're so constrained because we know just the tip of the iceberg of the total domain of science that's really there to know,” he says.

Even as Mayo Clinic continues to develop unprecedented new tools and treatments, such as pioneering AI algorithms to diagnose Alzheimer’s disease years earlier and powerful cancer-targeting technologies, many areas of human biology remain poorly understood. True cures for cancer remain frustratingly elusive; the biological underpinnings of psychiatric illness remain mostly unknown. In cases where we have treatments available, without pioneering science, it’s difficult to know if the current approaches are the best or most effective.

This discovery work is increasingly important for complex diseases. While technologies like CRISPR-CAS9 can edit single-gene disorders like sickle cell disease, most diseases are not so simple. Many serious or complex diseases are the result of a combination of factors — everything from a person’s individual genetic make-up to how their proteins are expressed to the environmental exposures they’ve had across their lifetime. To address them, scientists and clinicians need tools that aren’t available yet. By continuing and expanding this foundational research, Mayo Clinic researchers can develop the next generation of treatments.

“Probably a lot of what we think we know, we don't,” says Dr. Shah. “We could be misunderstanding the data or not seeing the full picture without additional research. That's why it's so important to keep doing that pioneering science."

The post How Discovery Science Powers Every Breakthrough at Mayo Clinic appeared first on Mayo Clinic Magazine.

"I'll never forget how difficult the recovery was after that first surgery to remove the tumor," Angela recalls. "I ended up with an infection while still recovering at home with my parents. I tried to go back to college afterward, but being away from my support system made everything so much harder." 

Looking back on those early days before receiving care at Mayo Clinic, she shares, "Doctors didn't know what we were dealing with. I wasn't angry — I just kept going because that's all I could do." 

The Turning Point  

Life regained some normalcy after her initial recovery. She met Chris — her future husband — and started planning a future. But less than a year into their marriage, the cancer returned, this time requiring surgery and 30 days of radiation. Two years later, the disease spread to her pelvic lymph nodes. 

This marked a turning point. Angela sought a second opinion at Mayo Clinic, where her care team removed 13 lymph nodes, sparing her from further therapies. "Mayo became my anchor," she says. "For the first time, I felt like I wasn't fighting this alone." 

Mayo became my anchor. For the first time, I felt like I wasn't fighting this alone.

— Angela Bristow

Over the next two decades, Angela’s life became a cycle of surgeries, treatments and recoveries, as tumors returned every two years. Yet she found strength in her "dream team" at Mayo Clinic, including her palliative care team, who are dedicated to easing suffering by managing symptoms associated with serious illness. Beyond medical care, they provide compassionate support to patients and their loved ones during life's most trying moments. 

"I have the distinct privilege of supporting Angela as she navigates the challenges of living with cancer," says Liz Sokolowski, M.D., Angela’s palliative care physician. "From our first meeting, I was deeply moved by her resilience and strength, and how she continues to maintain hope and her sense of humor. Patients like Angela are true heroes. They teach us daily about courage, and they remind us why we do this work." 

Fighting Forward

Advances in treatment provided new hope. What began with surgeries and radiation evolved into cryoablation, thermal ablation, and more recently, oral chemotherapy. Even when the cancer metastasized to her lungs a decade ago, Angela faced each recurrence with determination, balancing the fear of the unknown with acceptance.  

"Cancer doesn't own me," she affirms. "It's part of my story, but not the whole story. I'm still learning, still growing and still fighting. There's a reason I'm here." 

Angela's journey is one of resilience, self-advocacy and profound partnership — with her care team and her family. Chris has been her unwavering support, whether staying overnight in hospital chairs or managing wound care. Their daughter, now a teenager, has grown up witnessing her mother's courage. "She was just 3 months old when she stayed in my hospital room. She's my light, and Chris is my rock."

Now, Angela speaks with heartfelt wisdom. 

"I wish I could tell my 19-year-old self it will be OK. Cancer doesn't define life — it's just a part of it," Angela says. For those walking a similar path, she offers this advice: "Advocate for yourself, build a trusted care team and don’t let cancer take more than it has to. You are stronger than you think." 

Holding Hope 

Since first sharing her story, Angela’s journey has continued to evolve. She is now living with metastatic ependymoma and recently completed proton beam therapy at Mayo Clinic. While no additional treatment is scheduled at this time, her care continues through close monitoring and regular scans.  

Through it all, Angela remains grounded in the same quiet resilience she’s practiced for decades — focusing on daily life, protecting her sense of joy and holding hope in the moments that matter most. 

A version of this story was published on Mayo Clinic News Network. 

The post Living Beyond Diagnosis appeared first on Mayo Clinic Magazine.

Predicting Alzheimer’s Before It Starts

Mayo Clinic researchers have developed a prediction model that can estimate an individual’s risk of developing memory and thinking problems associated with Alzheimer’s disease years before symptoms appear.

The tool looks at basic health information, including age, sex, genetic makeup and brain imaging of amyloid protein, to predict a person’s likelihood of developing mild cognitive impairment or dementia within 10 years or over their remaining lifetime.

The model is built on data from more than 5,500 participants in the Mayo Clinic Study of Aging, one of the world’s most comprehensive long-running brain health studies. By following participants for years and carefully tracking medical outcomes, researchers were able to create one of the most complete pictures of who is likely to have cognitive decline.

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Brain scan results showing amyloid buildup proved to be the strongest predictor of lifetime risk. The research also revealed that women and people carrying a particular gene variant, known as APOE 4, face higher lifetime risk than the general population.

This breakthrough comes at a pivotal moment: The Food and Drug Administration recently approved therapies that remove amyloid from the brain and can slow disease progression in people with mild cognitive impairment or early dementia.

Knowing who is at risk before symptoms begin could help physicians determine when to begin therapy and guide patients in making proactive lifestyle changes.

This work is part of Mayo Clinic’s broader Precure initiative aimed at intercepting disease before it evolves into complex, hard-to-treat conditions and was supported by the National Institute on Aging, the GHR Foundation, Gates Ventures, and the Alexander Family Foundation.

A version of this story was published on Mayo Clinic News Network.

The post Predicting Alzheimer’s Before It Starts appeared first on Mayo Clinic Magazine.

The System Never Sleeps

Eric Klee, Ph.D., applied to medical school and subsequently pulled his application.

What made all the sense in the world for young Eric to do with his life made none in his own mind. His father was a Mayo Clinic pathologist and his mother a nurse. His siblings were all in the process of becoming clinicians themselves.

For an institution built more than 160 years ago by a family of medical practitioners, the Klees were as close to a modern story of the Mayo family as one could get.

"I was born and raised in Rochester, Minnesota,” Dr. Klee says. “I was surrounded by medicine.”

Dr. Klee was very interested in healing patients, but he didn’t want to study biology as an undergraduate followed by years of medical school.

"I knew once I got into medical school, I’d be doing residency and locked into a long-term career,” Dr. Klee says. “That’s why I pulled my application.”

His quest to find a career led him in the late 1990s to the office of Franklyn Prendergast, M.D., Ph.D., a friend of his father, searching like so many others did for some advice from the visionary clinician.

The conversation with Dr. Prendergast, who passed away in 2023, would chart Dr. Klee’s direction and set the stage for the future.

“I was interested in engineering and technology. I was interested in medicine. I wanted to figure out how it all came together,” Dr. Klee recalls. “Dr. Prendergast said, ‘Eric, there’s an emerging field called bioinformatics. It doesn’t actually exist yet, but I promise you it will in five years, and it’ll be important for at least 30 years to come.’”

Today, Dr. Klee, who first graduated with a degree in electrical engineering, has found his answer to what it means to practice medicine as an engineer. And it’s transforming how Mayo Clinic cares for patients with the rarest diseases.

BUILDING BEHIND THE SCENES

Dr. Klee, who is recognized as the Everett J. and Jane M. Hauck Midwest Associate Director, Research and Innovation, Center for Individualized Medicine, has spent his career building systems that aid doctors in new ways. As a leader in Mayo Clinic’s rare disease research and digital innovation efforts, he and his teams have created platforms that keep diagnosing patients years after their genetic sequencing tests. These platforms automatically watch for new scientific discoveries while families sleep, multiplying the impact of the organization’s expertise far beyond exam rooms.

His work represents a distinctive Mayo Clinic approach. It’s focused on cross-disciplinary collaboration where mindsets like his in engineering meet clinical needs. Infrastructure investments from a decade ago enable today’s artificial intelligence (AI) breakthroughs, and through it all, the institution’s commitment to patients extends even when they’re not physically present.

I was interested in engineering and technology. I was interested in medicine. I wanted to figure out how it all came together.

— ERIC KLEE, PH.D.

From increasing diagnostic success rates for rare diseases to being at the forefront of AI initiatives in the Mayo Clinic Comprehensive Cancer Center, Dr. Klee has found his own path to honoring the family calling — one that feeds his soul in ways a traditional practice would not have.

Choosing to study engineering led to an opportunity with the medical device maker Medtronic before going back to graduate school. After obtaining his doctorate degree, the pull of Mayo Clinic brought him back to Rochester, where he would practice medicine in a different way through systems and infrastructure.

None of this was more apparent than in the rare disease space, where finding answers requires something far beyond traditional clinical care — even at a place steeped in subspecialty expertise.

THE RARE DISEASE PARADOX

There are more than 6,800 identified rare diseases, generally considered to be diseases that affect fewer than 200,000 people in the United States at any time. Altogether, rare diseases affect an estimated 25 million to 30 million Americans, according to the National Human Genome Research Institute. These conditions span so many specialties that even the most skilled clinicians around the world can struggle to see the complete picture.

“A patient might have cardiac symptoms, neurological issues, immune system concerns and physical differences — but when these signs are scattered across different body systems, it creates what we call the ‘elephant problem,’” Dr. Klee explains. “Each specialist sees their part clearly, but few clinicians can observe the whole elephant at once.”

Even further, what happens if the latest breakthrough occurs after an episode of clinical care? What then?

“Our understanding of what genes and what variants cause disease is growing,” Dr. Klee says. “So what we know today is different than what we knew six years ago, six months ago or even six days ago. When you have a static report, you miss out on what we’ve learned since that test.”

To address this challenge, in 2022 Dr. Klee’s teams launched an automated system called RENEW. The tool is built to literally renew clinical test results by looking at what’s been learned since the last time the test was run or the patient’s data was accessed.

The system works with elegant simplicity.

RENEW automatically pulls in newly published discoveries of disease-causing genetic variants from research labs around the world. It compares these findings against Mayo Clinic’s database of unsolved patient cases — nearly 2,000 and growing.

Special filters whittle down the flood of new genetic information to focus only on what’s meaningful and could explain a patient’s symptoms.

When it flags a match, a team of genomic researchers digs deeper by reviewing medical records and clinical histories and correlating DNA alterations with new findings. The question they ask: Could this newly described variant be the answer someone’s been waiting years to hear?

So far, RENEW has delivered dozens of definitive diagnoses for Mayo Clinic patients with rare and previously unexplained diseases. And it takes less than a minute per patient to run the analysis.

“Every time we get a diagnosis, it makes me feel proud of what we’ve been able to accomplish at Mayo,” Dr. Klee says. “It’s this concept that the patients put trust in — not my team, not a single clinician — they put their trust in Mayo Clinic, and Mayo Clinic as an organization has supported this type of really challenging work.

“The parents and families, they believe that someday we will call them with an answer. And it’s incredibly gratifying for everyone when we do.”

VISIONARY LEADERSHIP, VISIONARY BENEFACTORS

A bigger question is why does this happen seemingly so often at Mayo Clinic? While Dr. Klee’s engineering background might seem like an unconventional foundation for rare disease research, at Mayo Clinic, cross-collaboration and diverse skill sets aren’t just accepted — they are championed.

But even the best collaborative culture needs infrastructure to reach its potential. Mayo Clinic leadership invested in this space a decade ago, and it’s paying dividends today.

“One thing that has set Mayo Clinic apart from a lot of other academic medical centers was the foresight of our institution, and now President and CEO Gianrico Farrugia, M.D., to think about data organization,” Dr. Klee says. “While other health systems were still figuring out electronic health records, Mayo Clinic was organizing all its clinical data in the cloud, creating Mayo Clinic Platform, de-identifying patient information and making it available for research.”

It was a massive investment bolstered by visionary benefactors despite an uncertain future, and the foresight it unlocked has set the stage for something much bigger.

“It opened the door for the transformative power of what we’re starting to realize in artificial intelligence, agentic AI and other emerging capabilities,” Dr. Klee says. “That realization is you’re only as successful as the data you have organized and readily available.”

In other words, Mayo Clinic didn’t just build strong and secure data systems. It built the foundation for the AI tools and future breakthroughs that didn’t even exist when the foundation was laid.

This work extends through Mayo Clinic Research enterprise priorities, including the Research Data Atlas, an initiative led by Dr. Klee to unify and mobilize research data at scale. It brings diverse research datasets into a secure, cloud-based environment where they become discoverable, interoperable and ready for advanced analysis. By increasing access to high-quality, harmonized data, the Research Data Atlas strengthens collaboration across the institution and accelerates Mayo Clinic’s ability to advance artificial intelligence-enabled discovery and translational research.

That infrastructure has allowed Dr. Klee to expand his impact beyond rare diseases. When Cheryl Willman, M.D., the Stephen and Barbara Slaggie Executive Director of Mayo Clinic Cancer Programs and Director of the Mayo Clinic Comprehensive Cancer Center¹, approached Dr. Klee about leading data and AI initiatives in oncology, he identified a chance to scale what he’d learned.

“I saw the opportunity in the Cancer Center to take a lot of what we had done in the Center for Individualized Medicine with regard to how we think about our genomic data and other omics data and organizing it to have an impact by breaking down barriers and opening doors to new discoveries.”

The same engineering mindset now applied to a different problem — making the data frictionless, accessible and useful across the entire enterprise to create systems that multiply the impact of every researcher, clinician and discovery.

RETURNING TO HIS ROOTS

Back to that conversation nearly 30 years ago in Dr. Prendergast’s office about the field that didn’t exist.

Bioinformatics employs hundreds of researchers at Mayo Clinic alone. And Dr. Prendergast’s prediction it would be important for at least 30 years has turned out to be conservative.

While Dr. Klee doesn’t have the same deep physician-patient relationships others in his family do, he keeps working on hope and healing in the way only an engineer could — leading teams who are building systems that never stop looking and leveraging infrastructure built with the forethought of what might be to come.

Because of that, in a cloud server room tonight, the system Dr. Klee championed runs. New databases of discovery are being checked against organized data files of patients. Algorithms are filtering results for meaningful changes.

And when tomorrow comes, a multidisciplinary team may get a notice to review results that could contain an answer someone has been waiting years to hear.

What kind of medicine can an engineer build? The kind that practices medicine even when patients aren’t there — because at Mayo Clinic, the needs of the patient come first, whenever and wherever they are.

1. Dr. Willman is also recognized as the David A. Ahlquist, M.D., Professorship in Cancer Research. ↩︎

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Precision Without Compromise

In the mid-2000s, Nadia Laack, M.D., a pediatric radiation oncologist at Mayo Clinic, saw an opportunity for patients. Mayo Clinic in Rochester was in the midst of designing a new facility for proton beam therapy — a powerful form of radiation that uses streams of protons to destroy tumor cells.

Dr. Laack knew that many patients would benefit the most from pencil beam scanning, the newest form of proton beam therapy. With this precise tool, she could direct protons at the exact contours of a tumor without injuring nearby organs.

But there was a catch. With pencil beam scanning, patients must remain perfectly still. For cancers in the lungs or abdomen, even the subtle movement of breathing could throw the beam off target.

And to prevent young children from wiggling, any form of proton therapy requires anesthesia, which greatly lengthens time in the treatment room.

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As a result, existing proton beam facilities only offered pencil beam scanning for cases where movement could be more controlled. These facilities also strictly limited the number of children they would treat each day so that, from a financial perspective, they could reserve more space for adults who could be moved in and out quickly.

Dr. Laack found these compromises unacceptable. Her colleagues agreed. Mayo Clinic would build the first proton beam facility in the United States that would exclusively offer the more advanced pencil beam scanning approach without limiting access for children.

To do so would come with significantly more work for Dr. Laack and her team, who partnered with engineers and physicists to design new tools to adapt to patients’ movements. And she brought on anesthesiologists to help engineer new ways to move kids in and out of treatment more quickly.

The Mayo Clinic Richard O. Jacobson Building housing the state-of-the-art proton beam facility opened in 2015. And Dr. Laack’s team has never had to turn away a child.

“We didn’t want anything to stand in the way of our ability to treat kids who needed our care,” she says.

Promise in Pencil Beam

Dr. Laack was committed to building a pencil beam scanning facility because she saw how much potential the treatment had to reduce the long-term side effects of radiation for many patients.

Traditionally, patients received radiation with photons, X-rays that pass into the body, through the tumor and out the other side of the body.

Photon radiation is an effective treatment for many cancers and is still commonly used today. But its path through the body requires radiation oncologists to limit the dose or risk damaging healthy tissue.

Proton beam therapy works by using a particle accelerator to whip protons up to a super high velocity — nearly the speed of light. A technician then directs these highly energized protons at a tumor. As protons pass into the body, they release most of their energy within the tumor, minimizing the radiation hitting healthy tissue around the cancer.

Pencil beam scanning is an even more targeted form that delivers protons packed into balls the size of pencil erasers, rather than a scattering of protons covering a wider area.

That precision is critical for children who could develop long-term side effects — such as growth, fertility or vision issues — if healthy tissue is damaged along with the tumor. Adults too can benefit from pencil beam scanning when tumors are situated in sensitive areas such as the brain, spinal cord, heart, lungs, liver and other abdominal organs.

Making Pencil Beam Possible

Dr. Laack and her team from the Division of Medical Physics collaborated with colleagues in the Department of Engineering to design tools to follow tumors in real time and only deliver protons when the tumors are on target. The team developed a respiration tracking tool, for example, that they place on a patient’s abdomen to capture the movement of breathing. They can then automatically trigger the proton beam to pause each time a tumor moves above or below the beam’s reach with each breath in and out.

To open access for more children, the team worked with Mayo Clinic anesthesiologists to figure out how to shorten treatment time. Their solution: prepare children for treatment outside of the pencil beam scanning room.

In the pretreatment area, a young patient lies on a specialized mobile table and begins receiving anesthesia from a compact delivery system. Once asleep, the child is then wheeled on the table into the pencil beam treatment room without interruption to anesthesia. Then a robotic arm docks onto the treatment table and moves it into the beam position. The robotic arm also has a compact anesthesia system built into the base, so the transfer of anesthesia tubes and lines is quick and seamless.

When designing this solution, the team was inspired by how anesthesia is delivered on the Mayo Clinic’s medical helicopters, says Dr. Laack. “You have a tight space in a helicopter too, and you don't want anesthesia lines in the way.”

In the proton beam treatment rooms, technicians use advanced imaging techniques to quickly pinpoint the precise location of the tumor. The robotic table adjusts and aligns the child perfectly so that the pencil beam directly hits the tumor.  

Commitment to Research and Care

Dr. Laack’s passion for designing the best possible cancer care began when her grandfather was diagnosed with leukemia. At the time, she was a college student at Colorado State University. She knew she couldn’t learn enough fast enough to help him. But she committed then to studying cancer so that she could help others.

As a medical student at Loma Linda University in California, Dr. Laack conducted research alongside her coursework, earning a master’s in physiology with a focus on breast cancer research.

She envisioned herself as a full-time cancer researcher. But then she began clinical rotations and discovered how much she values working with patients. “I was still passionate about studying cancer biology,” she says, “But the patient interaction was what brought me the most meaning and joy.”

It is so important to have all the tools in our toolbox so we can continue offering these patients the very best care.

— Nadia Laack, M.D.

As a radiation oncology resident at Mayo Clinic, she discovered that she was especially drawn to helping one patient group in particular: children. Working with kids facing difficult diagnoses was emotionally challenging, she says. “But I felt this was where I was needed the most.”

Today, Dr. Laack cares for patients of all ages, with a special focus on children. She also continues her research.

Before launching the proton beam facility, for example, Dr. Laack and her team spent a decade studying proton therapy and developing computer models that could project how effective and safe their new pencil beam facility would be for treating a wide range of cancers, including brain, breast, prostate and lung. After opening, they led more than 70 clinical trials to confirm that their proton therapy treatment resulted in the best outcomes. 

“The safety of our patients is the highest priority,” she says.

Accelerating Radiation Therapy Innovation for Patients

Today, Mayo Clinic’s proton beam therapy program has treated more than 10,000 patients, and soon, Mayo Clinic and Dr. Laack will have yet another radiation tool available for patients.

Within a few years, the recently constructed Duan Family Building at Mayo Clinic in Florida will be the first clinic in North America to offer a new technology: carbon ion therapy.

Carbon ion therapy is precise, much like proton therapy. But because carbon ions are more massive than protons, their impact is even more damaging to a tumor — making this form of radiation especially effective for patients with large or resistant tumors.

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Dr. Laack was an early proponent of bringing carbon ion therapy to Mayo Clinic. She recognized that Mayo Clinic staff were uniquely positioned to develop the new technology because of the knowledge they had already gained in proton therapy.

“We believed strongly that if anybody in North America was going to offer carbon ion therapy to patients, it needed to be Mayo Clinic,” she says. “We have the physics, the engineering and the physician expertise to be able to do it well and safely.”

Few of Dr. Laack’s pediatric patients require carbon ion therapy because pediatric tumors typically respond well to photon or proton therapy. But for some adult patients, and for children with treatment-resistant tumors, the technology could fight off cancer better and faster than existing therapies. Dr. Laack wanted to ensure that carbon ion therapy would be available for these patients.  

“Patients with some of the most difficult cancers — the hardest of the hard — come to us for hope and healing,” Dr. Laack says. “It is so important to have all the tools in our toolbox so we can continue offering these patients the very best care.”

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Leading the Charge in Carbon Ion Therapy

Laura Vallow, M.D., stands at the forefront of a medical milestone: bringing carbon ion therapy to the United States.

As chair of the Department of Radiation Oncology at Mayo Clinic in Florida, Dr. Vallow leads a team of physicians, scientists and international collaborators who are advancing research in carbon ion therapy, an advanced cancer treatment that uses high-energy carbon particle beams to precisely target tumors.

“It’s not just another form of radiation,” says Dr. Vallow. “Carbon ions have unique biological properties. Our goal is to unlock that full potential.”

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Small-Town Roots, Big Ambitions

Raised in a small town 35 miles south of Chicago, Dr. Vallow grew up surrounded by family — including 28 cousins, a mom who served as a nurse, an uncle who was the fire chief, and another uncle who was the police chief. Hard work, helping others and service to the community were constant themes.

“There was always this closeness in my family,” she says. “Work and family were intertwined, and they had fun doing it. No one pushed me toward a specific career. Instead, they taught me to find my passion and work hard at it.”

For Dr. Vallow, that passion was science. She attended the University of Illinois where she earned a degree in biochemistry. During her years as an undergraduate student, she joined Argonne National Laboratory, where she worked alongside Ph.D. scientists and presented at national conferences, building the confidence to envision a future in science.

“I realized I could do this — and succeed,” she says. “I was excited for a life of helping and impacting others through science.”

Finding Medicine

Eager to continue her scientific career, Dr. Vallow entered Stritch School of Medicine, Loyola University Chicago where radiation oncology quickly captured her interest.

“Radiation oncology has this fascinating technology you get to explore,” she says. “It’s the perfect blend of science, technology and patient care.”

These are diagnoses that too often are a death sentence. Despite decades of work, progress has been limited. Carbon ion therapy gives us a real chance to change that.

— Laura Vallow, M.D.

After completing her residency at Rush Presbyterian St. Luke’s University, she joined Mayo Clinic in 2001. Specializing in breast radiation oncology, Dr. Vallow became a leader in clinical trials and advancing innovations to improve outcomes for patients with breast cancer, such as shorter treatment courses and effective positioning for minimal impact on heart and lungs during treatments. In 2021, she became chair of the Department of Radiation Oncology.

“Once I started at Mayo Clinic, I knew that I never wanted to go anywhere else,” she says. “I love the integration of science and patient care. It’s a wonderful thing to take care of patients in this environment where everyone pushes you to be your best.”

Shattering Limitations

Today, Dr. Vallow no longer runs a single lab. Instead, she oversees multiple research efforts, including collaborations with carbon ion centers in Asia and Europe. At the center of her work is Mayo Clinic’s new integrated oncology building — the Duan Family Building — which will house the nation’s first carbon ion treatment facility.

Current radiation therapy applies a one-size-fits-all approach, using general parameters that don’t account for biological differences between tumors and normal cells. Dr. Vallow wants to change that.

“We envision profiling tumors to understand which patients will benefit most, and at which dose,” she says. “That’s how we’ll shatter current limitations to make treatments more personalized — and more powerful.”

Her department is already advancing this vision. One prospective study, led by colleague Bradford Hoppe, M.D., compares outcomes and quality of life for patients with bone sarcoma receiving traditional care at Mayo Clinic versus those treated at international carbon ion centers.

Through studies like this, the team will extend carbon ion benefits to more people with more types of cancer.

Shaping the Future

Dr. Vallow credits Mayo Clinic’s leadership with taking bold steps to bring carbon ion therapy to the U.S.

“Many institutions have talked about it, but no one has done it,” she says. “Our responsibility is to do this so well and lay out the research so impeccably that others can follow, and together we’ll expand access to carbon ion nationwide.”

Under her leadership, the department is poised to continue to grow as Mayo Clinic opens its doors to patients in need of carbon ion therapy. Dr. Vallow’s vision includes tackling some of the most intractable cancers, such as glioblastoma and pancreatic cancer.

“These are diagnoses that too often are a death sentence,” she says. “Despite decades of work, progress has been limited. Carbon ion therapy gives us a real chance to change that.”

And now, with carbon ion therapy on the horizon, Dr. Vallow’s passion continues to drive her to reshape the future of cancer care.

“I didn’t always know I would end up as a physician researcher, but now, I couldn’t imagine doing anything else.”

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A Childhood Dream, A Lifelong Mission

When Bradford Hoppe, M.D., was in middle school, he was told to make a collage using magazine cutouts to visualize his future goals. He created an image of a doctor living near the beach.

Today, his artwork has become a reality. Dr. Hoppe serves as a consultant in the Department of Radiation Oncology at Mayo Clinic and lives with his family in Atlantic Beach, Florida. But it’s not just a childhood dream that drives him. After nearly losing both his wife and his father to cancer, he is more determined than ever to transform the future of cancer care.

Following in His Father’s Footsteps

Raised in Los Altos, California, Dr. Hoppe grew up admiring his father’s lifelong career as a radiation oncologist at Stanford Medicine.

Dr. Hoppe says his dad’s work in Hodgkin lymphoma left a lasting impression that made him eager to follow in his footsteps. While traditional radiation could cure the condition, it could also lead to serious long-term side effects such as second cancers or heart complications decades later.

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“I became interested in this idea of optimizing clinical outcomes while minimizing risk of side effects. I knew I wanted to be part of the next generation of radiation therapy,” says Dr. Hoppe.

After earning a B.S. in biology at Stanford University and an M.D. at Cornell University Medical Center, Dr. Hoppe spent a year in Brazil conducting infectious disease and immunology research. He studied the impact of schistosomiasis, a parasitic disease, on the immune system and volunteered at a leprosy clinic.

“The work in infectious disease was really engaging in Brazil. But when I returned home, I knew I wanted to get back to my first passion: radiation oncology. I wanted to continue to advance the field just like my father had done.”

Shifting Coasts, Deepening Focus

After spending most of his life on the West Coast, Dr. Hoppe moved to the East Coast to pursue a radiation oncology residency at Memorial Sloan Kettering Cancer Center in New York, where he met his future wife, Sonia.

After completing an M.P.H. at Harvard School of Public Health, the couple then moved to Florida where Sonia began working in radiation oncology at Mayo Clinic and Dr. Hoppe became a faculty member at the University of Florida Health Proton Therapy Institute. There, he held the James E. Lockwood Endowed Chair in Proton Therapy and pioneered the development of proton therapy in the management of lymphoma, thymoma and lung cancer before joining Mayo Clinic in 2019.

“My wife had been working at Mayo Clinic as a radiation therapist for about 10 years before I joined,” says Dr. Hoppe. “When Mayo Clinic announced its plans for particle therapy, I knew it was the right move.”

Reimagining Carbon Ion Therapy

Dr. Hoppe is part of a team at Mayo Clinic that is bringing carbon ion radiation therapy to the United States. Similar to proton therapy, carbon ion can be delivered to a specific depth in the body, reducing damage to critical organs. However, unlike proton therapy, carbon ion causes clustered DNA damage, which is more effective in killing cancer cells, particularly with radiation-resistant cancers, and can be completed in less time than a traditional radiation therapy course. 

Mayo Clinic’s Duan Family Building in Florida will provide advanced cancer treatment options that are currently only available in Asia and Europe. The building opened to patients in July 2025, with the first carbon ion treatment expected to be available by 2028.

We are building upon existing strategies and making them better to shape a new future. And we’re getting closer every day.

— Bradford Hoppe, M.D.

Dr. Hoppe and his colleagues have toured and learned from existing carbon ion centers in Japan, Germany, Taiwan, Korea, Italy and Austria. But it’s not a simple copy-and-paste process.

“Mayo Clinic is approaching carbon ion therapy differently than other institutions,” explains Dr. Hoppe. “Traditionally, carbon ion therapy has been limited to rare, hard-to-treat tumors that don’t respond well to other treatments. But with the advances in precision medicine, we are working to identify patients with radioresistant forms of more common cancers who could benefit.”

Leading an International Collaboration

Dr. Hoppe is leading a collaborative clinical trial with centers in Europe and Asia to compare surgical treatment, proton radiation and carbon ion approaches for patients with pelvic bone sarcomas. The team is studying whether patients being treated with carbon ion therapy have higher cure rates compared with proton therapy and better functional quality of life compared with surgery.

Studies like this one will help experts better understand which cancers would benefit from carbon ion therapy.

“The key is knowing which patients will benefit from which treatments,” explains Dr. Hoppe. “It’s difficult for patients who have already undergone radiation unsuccessfully to jump into carbon ion because we don’t want to exceed radiation dose levels to critical structures and cause more problems for the patient. Our goal is to be able to identify the patients who would do better with carbon ion therapy at the time of diagnosis to improve outcomes and spare them from unnecessary side effects.”

A Mission Fueled by Experience

While Dr. Hoppe has realized his childhood dream, his mission has grown even more meaningful.

“My wife and my father both were diagnosed with metastatic cancers more than five years ago that were expected to be terminal,” says Dr. Hoppe. “Both have undergone cutting-edge, personalized treatments and are in remission.”

Dr. Hoppe is building on his father’s legacy — but also creating his own. His research in bone sarcomas is just the beginning.

“I imagine a future where Mayo Clinic will be able to identify patients most suitable for proton therapy and carbon ion radiation therapy through radiomic and genomic signatures. That means better outcomes, fewer side effects and more lives saved. We are building upon existing strategies and making them better to shape a new future. And we’re getting closer every day.”

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