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.

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, who is also recognized as the Carol M. Gatton Professor of Digestive Diseases Research, Honoring Peter Carryer, M.D. 

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.”

Open image in lightbox

Open image in lightbox

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.”

The post N of 1 appeared first on Mayo Clinic Magazine.

These “mini brains” allow researchers to witness in real time how different types of brain cells respond to different drugs, offering insights into psychiatric illnesses that were impossible to observe just a few years ago.

For millions of people worldwide struggling with poorly understood and difficult-to-treat alcohol and substance use disorders, these remarkable mini brains may revolutionize medicine’s understanding and treatment of addiction. Thanks to organoids, Ming-Fen Ho, Ph.D., and other researchers at Mayo Clinic are putting together the addiction puzzle, one piece at a time.

The Addiction Challenge

Substance use disorders and addiction have a wide range of impacts, spanning from the social and psychological to the physical. In 2020, over 14% of people in the U.S. aged 12 or older had experienced a substance use disorder in the prior year.

Addiction is extremely difficult to treat, not only because of the social stigma connected with the condition, but also because to scientists and clinicians the brain remains mostly a “black box.”

As neuroscientists make strides in understanding the molecular underpinnings of the brain via preclinical models, mental illnesses remain difficult to address.

"In psychiatry we don't have access to living human brain tissue,” says Dr. Ho, a stem cell biologist in the Department of Psychiatry and Psychology. “This is a major challenge for our research. It's not like cancer.

“When a patient has breast cancer, you can do a biopsy, you can study the tumor itself, but this is not what's happening in psychiatry. When patients come in with depression, we can’t ask them to give us a chunk of brain tissue to study."

As a result, clinicians remain in the dark about why some medications can work extremely well for certain patients with addiction while proving to be totally ineffective for others. But technologies like organoids are opening new avenues for understanding conditions like addiction, providing hope for these patients.

Understanding Organoids

Organoids represent a step beyond most preclinical models, allowing researchers to use a patient’s own cells to generate tiny organ-like models for research and testing. The process begins with a blood sample from a patient, which is used to generate induced pluripotent stem cells (iPSCs) — cells that are reprogrammed to an embryonic-like state, with the potential to develop into almost any cell type.

The iPSCs are guided into becoming early precursors of brain cells through specific chemical signals, then transferred into a special three-dimensional culture environment to allow them to organize into shapes similar to developing brain tissue.

HOW MINI BRAINS ARE MADE

STEP 1

Patients provide blood samples, which are sent to the laboratory for processing.

STEP 2

Blood cells are reprogrammed to an embryonic-like state, with the potential to develop into almost any cell type.

STEP 3

Using specific chemical signals, the stem cells are guided into becoming precursors to brain cells.

STEP 4

The cells are transferred into a specialized environment, where they develop into different types of brain cells and self-organize into three-dimensional brain-like structures, dubbed “mini brains.”

STEP 5

Under carefully controlled laboratory conditions and with daily feeding and monitoring, the organoids develop into more mature brain-like tissue, where they can then be used in research.

Over the span of a few weeks or months, the organoids continue to develop and become more complex, containing a variety of brain cell types including neurons, astrocytes, oligodendrocytes, microglia and more. Collectively, these cells interact in ways similar to real brain tissue, providing a more relevant model than traditional two-dimensional cell cultures. In turn, this provides deeper insights when studying complex neurological conditions such as addiction, depression and schizophrenia.

The technology behind these models is complex, and Dr. Ho says it wouldn’t be possible without the collaborative Mayo Clinic environment.

"Mayo has a very unique culture and environment to make this possible,” she says. “This research is truly a team effort — it doesn’t come from just one person overnight. We start small, but we move really fast because the technology changes so quickly.”

Building the Foundation for Discovery

Mayo Clinic’s comprehensive infrastructure for organoid development enables the groundbreaking addictions work. This includes a massive biobank of blood samples from patients with a variety of mental health disorders, which has generated an iPSC biobank with hundreds of cell lines, each linked to comprehensive clinical data. In addition, Mayo Clinic’s cross-disciplinary approach to care and research has psychiatrists, bioinformaticians and biologists working together to care for patients, collect clinical data, and translate that data into new discoveries that lead to therapies and cures.

Combined with advanced technology platforms, this ecosystem allows scientists like Dr. Ho unprecedented access to a breadth and depth of data on conditions like addiction. And Dr. Ho is dedicated to turning those insights into meaningful changes for patients.

A Winding Road

Dr. Ho brings an unusual perspective to her work, as she originally pursued training as a registered nurse, where she worked directly with patients. In her interactions with patients, she says, “I saw that modern medicine is not perfect, and there was so much we could improve.” Concurrently, she noticed the engaging work of her physician partners who conducted research alongside their clinical responsibilities.

Though she had no formal scientific background, after practicing as a nurse for two years, she made a leap into research, moving to Australia to pursue a Ph.D. in molecular genetics. Following her dissertation, she came to Mayo Clinic as a research fellow in rheumatology but eventually found her way to neuropsychiatric disorders like depression and addiction.

“I was interested in the complexity of these conditions,” she says. “In addition to the overall complexity of the brain and the challenges we face with understanding it, patients rarely experience a single mental health disorder. Half of patients with alcohol addiction are depressed, and often people with depression struggle with substance abuse. The underlying biology is intertwined. There is a lot to discover here.”

It took Dr. Ho over a year of daily practice and experimentation to master the process of turning stem cells into brain organoids. Now, she and her team manage over 500 cell lines derived from patients.

Decoding Drug Responses

One major area of focus in Dr. Ho’s work is understanding the neurobiology of alcohol disorder and identifying biomarkers that may help predict treatment response.

In a 2024 study, under senior author and pharmacologist Richard Weinshilboum, M.D., Dr. Ho and a team of researchers discovered that certain genetic variants can influence how well patients respond to acamprosate, a medication used to treat alcohol disorder. This discovery could help clinicians predict which patients are most likely to benefit from this treatment.

The power of this research lies in its "multi-omics" approach. "Our work moves beyond traditional pharmacogenomics, which looks at how genes affect drug response, to a broader approach," says Dr. Weinshilboum, from the Center for Individualized Medicine and Department of Molecular Pharmacology and Experimental Therapeutics and Mary Lou and John H. Dasburg Professor of Cancer Genomics Research. "We examine proteins, genes and RNA all together, a multipronged strategy that enhances our understanding of this disorder."

Combining these types of insights with patient-derived organoids, researchers can better understand how each patient’s unique biology influences their neural responses to substances like alcohol or opioids, and how they respond to different treatments.

In one of her earlier studies, Dr. Ho discovered surprising differences between drugs that were expected to work similarly. She examined how mini brains responded to oxycodone (a common opioid pain killer) and buprenorphine (a drug used to treat opioid addiction). Both drugs target the same receptors in the brain but behaved completely differently at the cellular level. While oxycodone primarily affected neurons and activated immune-related signaling pathways, buprenorphine affected other types of brain cells without increasing immune signaling. 

“This approach allows us to examine both the activity of living cells and gene expression after treatment," says Dr. Ho. "We can observe changes that would be impossible to study safely in living humans.”

Ultimately, this research could lead to personalized treatment approaches.

“Every drug has a unique signature in the brain,” says Dr. Ho. “Patient-specific organoids will allow us to predict how a given drug might affect that person’s brain and determine the best therapeutic approach for each person.”

Precision Medicine in Action

This approach to research and treatment exemplifies Mayo Clinic's Bold. Forward. strategy to transform healthcare, moving away from population-based, one-size-fits-all approaches and toward proactive, personalized medicine.

The implications extend far beyond addiction treatment. Dr. Ho envisions a future where organoids become standard tools for drug discovery across psychiatry. “In cancer, they have clinical trials every day and new drugs almost every year,” she says. “But to develop one medication from the day you identify a compound to the day you get Food and Drug Administration approval requires at least 10 to 15 years on average. In psychiatry, we don't have as many targets for clinical trials, and for most addictions, we don't even have medications for treatment.”

Our research has the potential to predict whether a treatment will help or potentially cause harm before we even give the medication to a patient. That's the kind of precision medicine that can truly change lives.

— Ming-Fen Ho, Ph.D.

Patient-derived brain organoids could accelerate this timeline by serving as screening platforms for potential therapies. Rather than spending years testing compounds that may ultimately fail in clinical trials, researchers could identify promising candidates much earlier in the process.

The technology also holds promise beyond substance abuse, with potential applications for depression, schizophrenia and neurodegenerative diseases like dementia. As artificial intelligence and machine learning capabilities continue to advance, researchers will be better equipped to analyze the vast amounts of data these models generate, potentially uncovering patterns invisible to the human eye.

A Vision of Hope

Back in the laboratory, the tiny clusters of brain cells continue their rhythmic pulsing. Each recorded signal represents a step closer to understanding the human brain, one of medicine's most challenging frontiers. What once seemed like an insurmountable black box is slowly yielding its secrets.

This critical work has been made possible with support from the Terrance and Bette Noble Foundation, Donna M. Giordano, and other generous benefactors whose philanthropy has helped advance Dr. Ho’s organoid research and its applications in addiction treatment.

For the millions of people and families affected by addiction and other mental health conditions, these mini brains represent more than scientific curiosity. They embody hope for a future where psychiatric care moves beyond trial and error, where treatments can be tailored to individual biology, and where the stigma surrounding mental illness gives way to the recognition that these are medical conditions deserving of precision treatment.

For Dr. Ho, whose journey from nursing to neuroscience research was driven by a desire to improve patient care, this work represents the culmination of years spent seeking better answers.

“As a nurse, I followed protocols created by others,” she says. “But as a scientist, I can create new knowledge that can truly help patients. Our research has the potential to predict whether a treatment will help or potentially cause harm before we even give the medication to a patient. That's the kind of precision medicine that can truly change lives.”

The post Mini Brains, Major Breakthroughs appeared first on Mayo Clinic Magazine.

Alpha vs. Beta Particles

Radiopharmaceutical therapies for treating cancer rely on the particle emission properties of radioactive isotopes for their effectiveness. By combining radioactive isotopes with a targeting molecule, such as an antibody, these treatments can directly target and bind to cancer cells, where they release high-energy particles to kill the tumor. However, not every radioactive isotope releases the same type of particle.

“From a physics standpoint, the beta particles — which are emitted by most currently approved radioisotopes — are basically electrons, while an alpha particle is two neutrons and two protons,” says Oliver Sartor, M.D., medical oncologist and director of Radiopharmaceutical Clinical Trials for Mayo Clinic Comprehensive Cancer Center.

Different kinds of particles have different properties. Understanding these differences can help clinicians and patients make decisions about the most appropriate treatments for an individual’s needs.

“An alpha particle is almost 8,000 times larger than a beta particle," says Dr. Sartor. "In some studies, just one hit from an alpha particle was enough to kill a cancer cell, while it takes many, many hits from beta particles to sufficiently damage a cell’s DNA to kill it.”

To put that into perspective, the impact of an alpha versus a beta particle can be compared to a fully loaded semitruck versus a 10-pound dumbbell.

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THERANOSTICS:
Old Tech, New Tricks

Learn more about radiopharmaceutical therapies and how clinicians and scientists at Mayo Clinic are leading the world in developing and implementing theranostics for cancer care.

Read More

Precision and Power

Due to their strength, alpha-emitting radioisotopes may benefit patients even when beta-emitting options have failed, and the fact that alpha particles are much larger means that they don’t penetrate as deeply into surrounding tissues, reducing the potential for off-target radiation effects.

As researchers continue to develop new targeted alpha particle treatments and build out the next generation of beta particle therapies, Mayo Clinic is at the forefront of advancing these highly targeted therapies to provide new treatment options for patients. These cutting-edge radiopharmaceuticals hold the potential to significantly improve outcomes for those facing a cancer diagnosis.

The post Alpha vs. Beta Particles: Bigger Particles for Bigger Impact appeared first on Mayo Clinic Magazine.

Oncologist Oliver Sartor, M.D., remembers the exact moment that changed his career. In 1993, he was working in oncology, focusing on experimental trials for treating prostate cancer.

“Experimental cancer medicine is a struggle,” he says. “It’s not easy to go out and cure cancer. It’s hard work, with a lot of opportunity for failure.”

At a conference, he met a nuclear chemist who held the patent for a radioactive isotope of the element samarium, a silvery metal. “He told me, ‘I’m going to take this radiation and put it right on the tumor in the bone where the cancer is,’” recalls Dr. Sartor.

Dr. Sartor was shocked — he couldn’t under-stand how the radiation could target the cancer so precisely. But then the chemist explained that the radioactive isotope known as samarium 153 had a structure that would cause it to bind within tumors of metastatic bone cancer. Once attached, the isotope would decay, releasing radiation directly into the tumor.

It was a eureka moment.

“That was my introduction to theranostics,” says Dr. Sartor. Since that moment, he’s been on a decades-long journey into the field, working on developing therapies that are more precisely targeted and more powerful. Now, he’s part of a dedicated team of researchers and clinicians who are expanding our knowledge about theranostics and working to magnify their use and reach in cancer care.

An Opportunity to Be Truly Bench-to-Bedside

Mayo Clinic is the highest-volume radiopharmaceutical practice in the world. “We treat more than two times as many patients as the next largest practice,” says Geoffrey Johnson, M.D., Ph.D. And he should know — he’s leading the charge working to bring new technologies, studies and experts such as Dr. Sartor to Mayo Clinic.

For Dr. Johnson, his career path led him to nuclear medicine as his specialty because he saw so much potential in theranostics for cancer care.

“It involves all the kinds of technologies that I like,” he says. “This brings together my background in chemistry and my love of advanced nuclear physics, along with advanced scanning technology, to let me develop new imaging technologies and therapy options that we couldn’t have imagined before. I get to work on developing new drugs, running clinical trials and applying my clinical expertise to patient care.”

So far, he’s seen his instinct prove true: Theranostics are becoming a topic of increasing interest in cancer care with new drugs approved in the last decade for neuroendocrine tumors and prostate cancer.

He also sees Mayo Clinic as a key player in the field. Mayo Clinic is the only National Cancer Institute-Designated Comprehensive Cancer Center with integrated yet distinct campuses across three states. All sites are also Society of Nuclear Medicine & Molecular Imaging-designated Comprehensive Radiopharmaceutical Therapy Centers of Excellence.

“Theranostics aren’t an option that we ‘also’ have,” says Dr. Johnson. “They’re integrated into our cancer center, and our approach to cancer care.”

What's in a Name

Theranostics, also called theragnostics, is a blending of the words “therapeutics” and “diagnostics,” and in oncology, it refers to the use of radioactive drugs that can target specific molecules on tumor cells. The radioactive component of the drug, called a radioisotope, can emit low levels of radiation, allowing clinicians to find the locations of tumors using a positron emission tomography (PET) scanner (diagnostics), or they can emit higher levels of radiation to kill the cancer cells they bind to (therapeutics). This allows doctors to use the same targeting molecule to both image and kill cancer cells.

“Being able to see the tumors with the same molecule we use to attack them lets us know exactly where the treatment will go and helps us determine if a given radioisotope therapy will be effective for an individual patient,” says Thor Halfdanarson, M.D., an oncologist who specializes in treating neuroendocrine and pancreatic cancers. “It’s not often in cancer care that we’re able to see the target before we send in the treatment.”

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The Science Behind
Theranostics

A groundbreaking approach called theranostics is emerging as a powerful tool in the fight against cancer. Combining the words "therapeutic" and "diagnostic," theranostics represent a significant leap forward in precision medicine, offering hope to patients who may have exhausted traditional treatment options.

How do theranostics combine therapy and diagnosis, and how do they compare to other cancer treatments?

Read More

Radiopharmaceutical therapy can be beneficial for patients with advanced cancers whose tumors aren’t responding to or can’t be readily addressed by other traditional treatment options. It is also comparatively gentle, according to Dr. Johnson.

“The therapies already on the market have been very effective. They’re not perfect, but they have low toxicity, and in my experience, patients have loved them,” he says. “It’s a very rewarding field, because we love to see patients happy with their care.”

This type of technology isn’t new. It has its origins in the use of radioactive iodine to treat thyroid cancer. In recent years, researchers and clinicians have been developing new theranostic technologies for better imaging and cancer treatment. There are now several Food and Drug Administration-approved radiopharmaceuticals available for treating neuroendocrine and prostate cancer tumors, but the field remains ripe for identifying new cancer targets and new radioisotopes.

Leading the Field

This investment can be seen not only in the new clinical expertise on campus but also in new facilities and partnerships. Mayo Clinic is building new tools and collaborating with industry partners to develop the next generation of theranostic imaging and radioisotope technologies, as well as addressing access issues for patients.

These collaborations lay the foundation for the testing and application of new theranostic treatments for patients with cancer, something that Mayo Clinic continues to put front and center in its practice. One major recent advance is the licensing of a Mayo-developed technology, called PSMA Alpha-PET DoubLET, to Perspective Therapeutics.

“Alpha-emitting radioisotopes have so much more potential to be powerful, and targeted, but they come with a long list of challenges,” says Dr. Johnson. “One of the biggest challenges is that we can’t easily see exactly where they go in the body. The technology we’ve developed really flips the script. We’ve taken what was the main issue with alpha-emitters and made it even better than what we see with beta-emitters.”

The technology uses a targeting molecule that binds to a chemical found on the surface of prostate cancer cells called prostate-specific membrane antigen (PSMA). The unique design allows these targeting molecules to carry either copper-64, which can be used to image the prostate cancer cells on a PET scanner, or lead-212, which releases alpha particles to treat the cancer. This allows researchers to use the same base molecular structure for both radioisotopes, which is not otherwise an option.

Dr. Johnson and a team of researchers at Mayo Clinic devised the idea and technology behind PSMA Alpha-PET DoubLET in the lab and ran it through promising preclinical testing before the technology was licensed to Perspective Therapeutics. The company is now further developing the platform to test in human clinical trials.

Theranostics isn’t an option that we ‘also’ have. It’s integrated into our cancer center, and our approach to cancer care.

— Geoffrey Johnson, M.D., Ph.D.

Progress also has been made on identifying and testing new and better cellular targets for radioisotopes. The team at Mayo Clinic is currently investigating a new targeting molecule for multiple cancers, called fibroblast activation protein inhibitor (FAPI). This molecule has already been used to successfully image pancreatic cancer tumors. Now researchers are investigating its use for delivering alpha-emitting radiation therapy to these notoriously hard-to-target tumors.

Teams at Mayo Clinic also are supporting a multitude of clinical trials in the theranostic space, including applying alpha-emitting radioisotopes to treat melanoma and next-generation beta-emitters for treating prostate cancer. Some clinical trials are investigating whether theranostic approaches may be beneficial even in earlier cancer stages before other types of treatment are implemented. In the lab, researchers are working to understand who the best candidates for radiopharma-ceutical treatments might be and developing new molecules that are longer lasting, are better at targeting cancer and have fewer side effects.

“This has always been a dream of mine,” says Dr. Johnson. “With my research background, I’m working as a clinician, but I also want to see what’s going on in the clinic, take it to the lab, design something new and bring it back to clinical trials so that we can get it to the patients. Doing that whole loop — it’s something that’s on my bucket list, and we might just pull it off.”

While many of these studies and trials are still in their earliest phases, the team is already looking toward a bright future for theranostics.

“Right now we’re in the ‘blue sky’ period,” says Dr. Sartor. “We have a limited number of successful therapies already available to patients, but now that we’ve demonstrated the proof of principle, we know that if we can get the radioisotope to the right spot, we can kill the tumor. We’re in a mad rush to identify new targets, carriers and efficient ways to engineer these treatments end to end.”

The post Theranostics: Old Tech, New Tricks appeared first on Mayo Clinic Magazine.

However, while thousands of Americans have lost their ability to speak, swallow and breathe independently due to damage to or the loss of their larynx, a lack of available donor organs makes it challenging for medical teams to provide this cutting-edge treatment.

Researchers at Mayo Clinic like Cheryl Myers, Ph.D., an immunobiologist who studies regenerative science applications, envision a future in which electrospinning can help address this shortage and provide hope to patients everywhere in need of organ transplants.

Electrospinning New Solutions

Researchers supported by the Mayo Clinic Center for Regenerative Biotherapeutics are using high-tech machines called electrospinners to create the building blocks of regenerative biotherapeutics. One of their most promising areas of research is the creation of 3D-printed, patient-specific larynges, which involves regenerating tissue types that include cartilage, muscle and epithelial tissue. 3D-printed larynges pose far less chance of rejection by the recipient, reducing the need for immunosuppressant drugs.

“If we can use electrospinning to mimic the extracellular matrix of the larynx and provide the scaffolding for cells to grow on, we potentially could improve the healing process in a way that’s controlled and safe,” Dr. Myers says. “Electrospun nanofibers play a crucial role in tissue regeneration and integration because they provide a scaffold for cell growth and mimic the natural extracellular matrix. By optimizing the various components that fabricate the 3D larynx, we could give a patient’s voice back to them.”

How Does an Electrospinner Work?

Electrospinners operate much like medical spinning wheels. They whip biotherapeutic fibers into a scaffold — or platform — using electrical forces that spin chemical solutions into nano- or micrometer-long fibers. In turn, that creates a porous base favorable for growing replacement cells.

“Electrospinning can produce scaffolds that closely mimic the physical environment cells naturally interact with,” explains Dr. Myers. “By customizing the electrospinning formula, it is possible to match the biochemical properties of the extracellular matrix, thereby influencing cell behavior. This is essential for tissue development and regeneration, making electrospinning an ideal platform for tissue engineering and regenerative medicine applications.”

A Look Inside the Center for Regenerative Biotherapeutics

The Center for Regenerative Biotherapeutics is Mayo Clinic’s hub for biomanufacturing, a type of manufacturing that uses sources from the human body — cells, blood, enzymes, tissues, genes or genetically engineered cells — for use in medicines. Biotherapies have the potential for targeted healing with fewer side effects than traditional drugs.

The center plays a critical role in early-stage product development for clinical use at Mayo Clinic and collaborates with biotechnology companies to further accelerate the advancement of regenerative technologies.

Regenerative medicine has changed the focus of healthcare from fighting disease to restoring and building health. Through its biotherapeutics strategy, Mayo Clinic is advancing groundbreaking discoveries toward curative products.

Philanthropy in support of the Center for Regenerative Biotherapeutics speeds up the timeline for early-stage therapies as they work through the development pipeline. Philanthropy also promotes healthcare equity by helping contain the cost of these therapies, a high priority for the center.

Make a gift now to help transform the future of healthcare.

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The Future of Tissue Engineering

Tissue engineering is an emerging technology that offers hope for replacing and repairing damaged cells, tissue or organs. It may offer solutions for body tissue that does not regenerate, such as tissue that is not connected to blood supply, like cartilage.

Artificial joint replacements are often the only long-term solutions for areas like knees and hips after they have developed osteoarthritis as a result of no longer having cartilage.

In addition to work being done on 3D-printed larynges, Mayo Clinic researchers are using electrospinners to create skin patches that heal chronic wounds that have not responded to other treatments and to develop orthopedic patches to regenerate damaged cartilage around the rotator cuff in the shoulder.

Navigating Complexities

Researchers must address several challenges as they bring new tools like this to clinical care. Growing tissue requires researchers to identify the proper chemical compounds, growth factors and cells. Once that happens, clinical trials will be needed to advance electrospinning-developed tissue toward patient care.

Among the necessary trials, Dr. Myers explains, will be those that compare 3D-printed implants that do not use electrospinner-generated material with those that do, to confirm they enhance patients’ recovery.

“Electrospinning’s capability to produce fibers with tailored properties and functionalities makes it a valuable tool for advancing healthcare technologies,” says Dr. Myers. “This versatile technique can tackle several critical healthcare challenges, including tissue engineering, wound healing and drug delivery systems.”

The post Electrospinning the Building Blocks for Regenerative Medicine appeared first on Mayo Clinic Magazine.

How do theranostics combine therapy and diagnosis, and how do they compare to other cancer treatments?

How Do Theranostics Work?

Radioactive pharmaceutical agents used in theranostics have two key components: a targeting component, such as an antibody, that binds to specific molecules or proteins on cancer cells, and a radioactive compound that can be used either for imaging the cancer or for destroying it.

First, the patient is given a radiopharmaceutical agent for imaging the cancer. The targeting molecule attaches to the surface of the cancer cells, while the radioactive isotope emits low-energy radiation that can be visualized and mapped using imaging techniques like PET scans.

Once the tumor locations are identified, the same targeting molecule is then coupled with a higher-energy radioactive isotope. When given to a patient, this targeted radiation selectively kills the cancer cells that the drug binds to, while minimizing damage to surrounding healthy tissues.

The key advantages of theranostics are that they enable precision medicine by binding to specific molecular targets on a patient's tumors. They then use that binding to allow for both diagnosis and selective treatment of the cancer with radiation therapy. Clinicians can determine exactly where the treatment will go and assess if the radioisotope therapy will be effective for an individual patient because both the imaging solution and the treatment use the same molecule. This personalized approach aims to improve outcomes and reduce side effects compared to traditional cancer treatments.

While theranostics may seem cutting-edge, their roots trace back to the 1940s with the use of radioactive iodine to treat thyroid cancer. However, recent advances have dramatically expanded the technology’s potential. The field now combines chemistry, advanced nuclear physics, and state-of-the-art scanning technology to develop new imaging techniques and treatment options previously thought impossible.

Open image in lightbox

THERANOSTICS:
Old Tech, New Tricks

Learn more about radiopharmaceutical therapies and how clinicians and scientists at Mayo Clinic are leading the world in developing and implementing theranostics for cancer care.

Read More

How Do Theranostics Compare to Other Kinds of Cancer Treatments?

Compared to traditional cancer therapies, theranostics offer an innovative approach that combines diagnostic and therapeutic capabilities in a single agent. Conventional treatments such as chemotherapy, external beam radiation and surgery have been the mainstays of cancer treatment for decades. However, these methods often lack the precision of theranostics. Chemotherapy, for instance, circulates throughout the entire body, potentially causing widespread side effects as it targets both cancerous and healthy rapidly dividing cells. External beam radiation, while more localized than chemotherapy, still affects a defined area that may include healthy tissue surrounding the tumor. Surgery, though precise in removing visible tumors, may not address microscopic spread of cancer cells and is not always feasible depending on the tumor's location.

By using a targeting molecule that specifically binds to cancer cells, radiopharmaceutical agents can deliver diagnostic imaging capabilities and therapeutic radiation directly to the tumor sites, wherever they may be in the body. This targeted approach could potentially reduce side effects on healthy tissues and allow for real-time monitoring of treatment efficacy. Furthermore, the diagnostic component of theranostics enables personalized treatment planning, as clinicians can assess whether a patient's cancer expresses the target molecule before proceeding with therapy, while traditional chemotherapy requires a waiting period following treatment to determine if it’s been effective. While theranostics are not a replacement for all existing cancer therapies and may be used in combination with other treatments, they represent a significant advancement in the pursuit of more effective and less toxic cancer care.

How Is Mayo Clinic Driving Innovation in Theranostics?

Mayo Clinic's approach to theranostics is multifaceted, encompassing clinical expertise, research and development, and collaborative partnerships, expanding treatment options and personalized medicine. The Mayo Clinic Comprehensive Cancer Center has recruited top talent, including pioneers in the field with decades of experience in cancer treatment and research. Mayo Clinic is actively developing new technologies and conducting clinical trials, and studies are underway to understand how a person's genetic profile may influence their susceptibility to side effects from radiopharmaceutical treatments, paving the way for more tailored treatment approaches.

In addition to internal efforts, Mayo Clinic has forged strategic alliances with industry leaders, and researchers are investigating new targeting molecules, such as fibroblast activation protein inhibitor (FAPI). FAPI shows promise for imaging and potentially treating pancreatic cancer.

The applications for theranostics are expected to expand beyond current Food and Drug Administration-approved treatments for neuroendocrine tumors and prostate cancer. Clinical trials are underway to explore its potential in melanoma, earlier cancer stages, and other hard-to-treat cancers.

The future of cancer treatment looks more promising than ever. Theranostics represent a significant step forward in the quest for more effective, personalized cancer care, offering hope to patients and pushing the boundaries of what is possible in modern medicine. The ongoing advancements in theranostics highlight the potential for transformative changes in cancer diagnosis and treatment, positioning this field at the forefront of precision medicine.

The post The Science Behind Theranostics appeared first on Mayo Clinic Magazine.

"My feet would be killing me. It would hurt to walk," recalls Chris. Then the pain moved to his knees and shoulders, and Chris' ligaments and tendons were seemingly always tight.

Military doctors diagnosed Chris with scleroderma, an autoimmune disease that affects multiple systems in the body, including the skin, lungs and gastrointestinal track. Scleroderma causes progressive disability and in some cases death. Although rare, the disorder is more common in certain populations, such as African Americans, but it can affect any ethnic group.

"Unknowingly, the latent TB (tuberculosis) medication had caused this seemingly dormant condition to rear its ugly head," says Chris, who was 39 at the time.

"I'll never forget talking to the rheumatologist, and she was telling me what comes along with the condition. I just remember looking at her, and then I sat back and repeated the basics of it: 'So I have a condition that has no cure. And it could potentially kill me.' It was a shock," says Chris, a father of two young children who lives in Brunswick, Georgia.

A downward spiral

Over time, Chris' condition worsened. "I dropped a significant amount of weight. I started to have skin discoloration all over my face, and I was always cold. It got to the point where I was unable to reach for things in the cabinet. It was difficult to do daily activities, like reaching down to put shoes and socks on," says Chris.

In December 2019, at the suggestion of family members — who "always praised the service and quality of Mayo Clinic health care" — Chris traveled south to meet with Andy Abril, M.D., a rheumatologist at Mayo Clinic in Florida.

"When Chris came to see us, his scleroderma was rapidly progressing. So he came to see what alternatives we had for the treatment. Unfortunately, with this condition, there are not a whole lot of options," says Dr. Abril.

Innovative research

One area of innovation though is autologous bone marrow transplantation, where a patient’s own healthy stem cells are used to replace diseased or damaged bone marrow.

Chris had heard this type of transplant was being investigated for patients with autoimmune disorders. But he says he was still shocked when Dr. Abril recommended he visit with a hematologist.

In early 2020, he met with Ernesto Ayala, M.D., a member of Mayo Clinic Cancer Center's Cellular Therapy program.

"Autoimmune disorders target people at the most productive stage of life, usually within age 20 and 40, so they have a huge impact. They are complex and difficult to treat in that there are many patients who fail to respond. But bone marrow transplantation, or cellular therapy, has emerged as a powerful alternative to treat these disorders," Dr. Ayala explains.

"In the southern United States, Mayo Clinic is the only center that is offering this type of treatment as standard of care for patients with severe autoimmune disorders," Dr. Ayala adds.

Chris was admitted to the hospital in April 2020. In less than a week, he received his transplant.

Recovery has been slow but well worth the wait, Chris says.

In the eight months since his transplant, Chris has seen his symptoms almost reverse. He's back at the gym, his skin has softened, and much of the discoloration on his face has resolved. Gastrointestinal issues also have abated. "I'm hoping to regain weight and strength in the next six months and beyond," Chris says.

"It's so exciting to see the results of our work. I can see a family together again as a whole, raising their children, going back to work. And to me, it's like the prize of what we do," says Dr. Ayala.

Transforming medicine

Chris’ experience is the kind of outcome that is driving Mayo Clinic to expand. Ambitious construction projects across Mayo Clinic’s major campuses in Florida, Arizona and Minnesota are providing the expertise, space and technology to deliver new answers to patients and revolutionize the future of medicine. And in Florida, cancer care and related specialties are some of the top priorities for growth.

"In the last few years, Mayo Clinic in Florida has invested significantly in people, technology and space to be able to lead the treatment of solid tumors and hematological cancers, but also to lead in the development of cutting-edge cellular therapies for other diseases, such as autoimmune diseases and neurological diseases," says Roxana Dronca, M.D., chair of the Division of Hematology and Medical Oncology at Mayo Clinic in Florida.

"Seeing the success of this patient gives me hope that we can continue to advance the medical field and make a difference in the lives of patients, not just with cancer, but with other rare and incurable diseases," Dr. Dronca says.

Make a gift now to help transform the future of health care today.

The post Innovative Transplant Offers Hope for Patient With Rare Autoimmune Disorder appeared first on Mayo Clinic Magazine.