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he goal is always the same: help people live longer, happier, healthier lives. But the path to developing treatments is often winding and marred with roadblocks and red herrings. The keys to success in medical research lie in curiosity, persistence and innovation.

That’s how University of Florida College of Medicine experts have built a legacy of lasting impact over 70 years, from the creation of Gatorade to finding solutions for aggressive cancers, dangerous infections and inherited diseases.

Across campus and across the state, from Gainesville to Jacksonville and beyond, physician-scientists, veterinarians, engineers, data experts and more are combining their expertise and infrastructure to tackle some of the most urgent challenges in human health. Together, they are transforming bold ideas into discoveries that change patient care.

UF College of Medicine researchers are looking beyond conventional treatments and forging new paths. Because if the answer doesn’t already exist, Gators make it possible. This is where breakthroughs begin.

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ike in people, a bone tumor or brain tumor in a dog is all too often a brutal diagnosis, and for decades, the same basic treatments have made up veterinarians’ limited arsenal.

So, when pediatric oncologist Elias Sayour, MD, PhD, approached veterinary oncologist Rowan Milner, BVSc, MMedVet, PhD, to suggest testing an experimental mRNA cancer vaccine in canine patients, Milner saw a chance to change dismal outcomes.

Across both species, naturally occurring bone and brain tumors are strikingly similar at the molecular and pathological levels. What’s more, Sayour and Milner were drawn together by UF’s exceptional infrastructure. With the College of Medicine just steps away from the College of Veterinary Medicine, their partnership has led to remarkable results for canine patients and a first-of-its-kind clinical trial for human patients.

“The connection Rowan and I have, which has now spanned many years, is a foundation of everything that’s been achieved up to this point,” said Sayour, the Stop Children’s Cancer/Bonnie R. Freeman Professor for Pediatric Oncology Research.

The translational work first began in Sayour’s lab, where he pioneered and tested a personalized mRNA-lipid nanoparticle vaccine in mouse models and cells derived from human tumor biopsies.

Then, co-led by UF veterinary neurology service chief Sheila Carrera-Justiz, DVM, the research teams ran a canine clinical trial with 10 boxer dogs and French bulldogs that had developed terminal brain tumors similar to those in humans. The pet patients, whose owners approved of their participation as they had no other treatment options, lived a median of 139 days, compared with a median survival of 30 to 60 days typical for dogs with the condition.

“There’s a whole backroom operation for a tumor that’s taken from a dog, processed by us and then sent to Elias’ lab in a way that is sterile and maintains the integrity of the tumor so they can make the vaccine,” said Milner, the Hill’s Endowed Professor of Oncology, oncology service chief and head of the clinical trials program at the College of Veterinary Medicine.

The canine clinical trial paved the way for a first-of-its-kind human trial of four adult patients with glioblastoma, a treatment-resistant brain tumor with a prognosis of 12 to 18 months. And those results were equally stunning: The vaccine rapidly reprogrammed the immune system to attack the tumor.

Since then, Sayour and collaborators have built upon the results and other pivotal mRNA vaccine research to produce a paper that grabbed attention worldwide last fall: In a retrospective analysis of medical records, a UF-University of Texas MD Anderson Cancer Center team found that people with advanced lung or skin cancer who received the COVID-19 mRNA vaccine within 100 days of starting common immunotherapy drugs lived longer than those who didn’t get the vaccine.

If confirmed in a prospective trial, it would mean that a widely available vaccine capable of jump-starting a patient’s response to immunotherapy already exists — and that scientists could start developing a universal cancer vaccine.
Taken together, these encouraging findings culminate years of work, reminding Sayour and Milner why they joined forces in the first place: to make a difference.

“On any given day, we could be treating mice, dogs, cats and humans,” Sayour said. “Rowan will call me and say, ‘Look, this dog’s got an issue, and we’ve got to figure this out.’ Being co-located allows this type of work to move forward in a way that would be very hard at other centers.”

Now, the vaccine from the dog/human glioblastoma trial is advancing to a Phase 1 human trial for children with brain cancer. Additionally, the collaborators have expanded their animal trials to test an mRNA vaccine in cats with a type of head and neck cancer called squamous cell carcinoma. Next up, they will test a variation of the COVID-19 vaccines in cats with that disease.

“There are publications showing that zoo cats were susceptible to COVID, so we feel this is a natural direction to go,” Milner said.

“And,” Sayour said, “we’re just getting started.”

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hilip Efron, MD, spends a lot of time thinking about sepsis and the patients who battle it. As medical director of the UF Sepsis and Critical Illness Research Center, or SCIRC, he has seen that battleground change dramatically over the course of his career.

Sepsis, life-threatening organ dysfunction in response to infection, is one of the leading causes of in-hospital deaths in the United States. Patients who survive this syndrome often experience extremely morbid outcomes in the long term, including cognitive issues, frailty and death within a year of their initial infection.

“Medical practitioners have done a good job of early recognition and immediate treatment,” said Efron, the Cracchiolo Family Professor of Surgery in the Department of Surgery. “Patients are surviving the initial sepsis incident much better than they did decades ago.”

At the UF SCIRC, experts uniquely engage in sepsis work, from early-stage research through patient outcomes. Physician-scientists like Efron and their interdisciplinary research partners and trainees are searching not only for new ways to keep patients alive but also to improve their quality of life — and long-term health — in the months and years after sepsis.

Guoshuai Cai, PhD, an assistant professor in the Department of Surgery and director of the UF Surgery Genomics Core, is at the forefront of the discovery effort.

In the lab, his team is building a massive database of human cell samples from sepsis patients to generate “predictive power.” They recently received a $2.1 million National Institutes of Health grant to develop statistical and AI models that turn data into actionable outcomes, using tools like UF’s HiPerGator, the fastest university-owned supercomputer in the country.

“In the past, we could only measure aggregate features across cells,” Cai said. “With technology like advanced single-cell profiling machines, we can now obtain high-resolution data from individual cells.”

By examining sepsis patients at the genetic level, UF research teams can develop new models to help predict how patients will react to sepsis in the long run. Physicians like Efron then bring the models to the hospital, tailoring treatment to each patient to address the impact of sepsis incidents and the challenges of long-term recovery.

“We can’t keep doing the same thing over and over again,” Efron said. “Our work is shifting toward precision therapies designed to improve not just survival but also the quality of recovery, helping patients get home and regain their health after critical illness. This work would not be possible without our dedicated multidisciplinary team, whose collaboration and commitment continue to drive meaningful advances in patient care and recovery.”

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atients with rare diseases often struggle to receive a quick diagnosis. For most, there are few effective treatments. An overwhelming majority of the illnesses — about 80% — are genetic. Naturally, so are their most effective therapies.

Late College of Medicine Dean Kenneth Berns, MD, PhD, and his seminal drug delivery model of adeno-associated viruses, or AAV, put UF on the map for gene therapy three decades ago. Years of collaboration between the university’s experts in gene therapy and rare diseases solidified UF’s position as a top-ranked institution for the field. Now experts are building on decades of discovery to advance treatments for patients facing Duchenne muscular dystrophy, Pompe disease and more.

“Our contribution has been not just using these tools in early models of disease but actually implementing them in a new field of genetic medicine, which didn’t even exist five years ago,” said Barry Byrne, MD, PhD, the associate chair of the Department of Pediatrics and director of the Powell Gene Therapy Center.

“Rare diseases give us a unique way to look at biology and sometimes provide insights into the more common and chronic diseases we otherwise wouldn’t be able to understand,” said Matthew Gentry, PhD, a professor and chair of the Department of Biochemistry and Molecular Biology at the UF College of Medicine.

As an expert on glycogen storage and metabolism, Gentry first collaborated with Byrne on Pompe disease, an illness heavily impacted by the mechanism Gentry honed his work in. Since then, they’ve tackled other diseases, developing a gene therapy strategy in which a lifesaving enzyme doesn’t need to be repeatedly delivered. Instead, the gene therapy vector delivers the corrective gene to both the brain and spinal cord and the body’s musculature.

“Some of the challenges for these therapies are that they tend to be something delivered once in a lifetime,” said Byrne, the Earl and Christy Powell University Chair in Gene Therapy and Genetics Research. “For example, the immune response to the AAV product used to deliver the gene prevents us from using it again without reducing or eliminating the antibodies that are present.”

To combat this, researchers have developed a means of blocking the antibodies that would make the treatment less effective as the patient grows older.

Gentry and Byrne have pushed to use leading-edge metabolomics resources to collect samples and give accurate prognoses. Biomarkers and genetic sequencing help clinicians understand not only which rare disease they’re dealing with but also how aggressive a patient’s particular case is.

Byrne and Gentry have collaborated together and with others to push multiple drugs into clinical trials.

“We always want to learn more about foundational biology and understand cellular mechanisms,” Gentry said, “but at the end of the day, we’re doing this to make an impact on as many people as possible.”