Medical Breakthroughs at the UVA Health System
From life-saving cancer drugs to artificial intelligence systems redefining chronic disease management, research at the UVA Health System is reshaping the future of medicine. Backed by more than $287 million in annual research funding and major new facilities on the way, UVA Health has become a national leader in turning bold scientific ideas into real-world treatments. The breakthroughs that follow span cancer, neuroscience, heart health, diabetes, and beyond—all offering new hope to patients locally and around the world.
CANCER TREATMENT & RESEARCH
New Leukemia Treatment Approved
While at UVA, researchers Jolanta Grembecka and Tomasz Cierpicki helped develop ziftomenib (brand name Komzifti), a groundbreaking new treatment for acute myeloid leukemia—the deadliest blood cancer—that received FDA approval in November 2025. This once-daily oral medication offers hope for patients with recurring or treatment-resistant disease who have the NPM1 gene mutation and previously had no good treatment options. The drug works by disrupting menin protein interactions that drive leukemia cell growth, allowing cells to mature normally instead of becoming cancerous. Their UVA‑ and University of Michigan–based work pioneered a class of menin inhibitors at a time when much of the pharmaceutical industry was skeptical that targeting protein‑protein interactions was even feasible.
First-in-Virginia Treatment Extends Lives of Small Cell Lung Cancer Patients
UVA Health became the first healthcare provider in Virginia to offer tarlatamab, a revolutionary immunotherapy for small cell lung cancer (SCLC). This “bispecific T-cell engager” supercharges the body’s immune system to find and destroy cancer cells, significantly extending patient survival. In UVA’s published results from 21 patients, nine showed partial tumor response, with an overall response rate of 42.9%—a remarkable outcome for one of the deadliest cancers. In larger clinical trials of tarlatamab, median survival has reached about 14 months, compared to less than 6 months with standard chemotherapy. UVA has developed an outpatient protocol that expands access to this life‑extending therapy across Virginia.
ALZHEIMER’S DISEASE & BRAIN HEALTH
Why Alzheimer’s Patients Forget Loved Ones
Harald Sontheimer, chair of UVA’s Department of Neuroscience, and his research team have discovered why Alzheimer’s patients lose the ability to recognize family members and friends, solving a heartbreaking mystery that has plagued families for generations. Published in October 2025, their research found that protective “perineuronal nets” surrounding neurons in the brain’s social memory center break down in Alzheimer’s disease. Using MMP inhibitor drugs, they successfully preserved these protective nets in mice and protected social memory, offering a completely new therapeutic target. This discovery could lead to treatments that delay one of the most devastating aspects of Alzheimer’s—when patients no longer recognize their own loved ones.
Immune System Link to Alzheimer’s Unveiled
Professor John Lukens, director of the Harrison Family Translational Research Center in Alzheimer’s and Neurodegenerative Diseases, discovered that DNA damage accumulating during aging triggers inflammation in the brain that damages neurons and impairs memory in Alzheimer’s disease. By removing the STING protein in mice, his team dampened harmful immune activation, protected neurons, and improved memory function—revealing a novel pathway for potential treatments that could slow or prevent Alzheimer’s progression.
HEART HEALTH & BLOOD PRESSURE
Hidden Cause of Obesity-Related High Blood Pressure
In breakthrough research announced December 2025, professor Swapnil Sonkusare and his team discovered a previously unknown mechanism causing high blood pressure in obesity—and it comes from within blood vessels themselves. They found that smooth muscle cells in small arteries release an inflammatory molecule called TNF that disrupts neighboring endothelial cells, raising blood pressure. Using a TNF-blocking drug, they successfully lowered blood pressure in obese mice, with early human cell tests showing promise. This discovery opens the door to targeted treatments for the millions of Americans with obesity-related hypertension.
Body’s Natural “Off Switch” for High Blood Pressure
UVA scientists, led by Drs. R. Ariel Gomez and Maria Luisa S. Sequeira-Lopez, revealed how our bodies naturally regulate blood pressure by controlling a key hormone, discovering what amounts to a built-in “off switch” for high blood pressure. This discovery of how calcium activity in kidney cells regulates renin production could lead to new treatment strategies for the millions of Americans struggling with hypertension.
DIABETES INNOVATION
AI-Powered Artificial Pancreas Frees Diabetes Patients from Constant Management
UVA’s Center for Diabetes Technology developed an FDA-approved artificial pancreas that uses advanced algorithms to automatically adjust insulin delivery, significantly improving blood glucose control for people with type 1 diabetes. In a major clinical trial, the system increased time in a healthy glucose range from 72% to 77% compared with standard therapy, while reducing the daily burden of diabetes management. Building on this progress, UVA researchers are now testing next-generation artificial pancreas systems that use artificial neural networks and “digital twin” technology to model individual physiology and move toward fully automated insulin delivery without patient input. These systems remain in clinical trials, while the FDA-approved UVA artificial pancreas already supports patients worldwide.
INSTITUTIONAL EXPANSION
Manning Institute of Biotechnology
UVA marked a key construction milestone with the “topping-out” ceremony in October 2025 for the Paul and Diane Manning Institute of Biotechnology, a 350,000‑square‑foot facility at Fontaine Research Park. Funded by $100 million from Paul and Diane Manning, $100 million from the Commonwealth of Virginia, and $150 million from UVA, the $350 million project is designed to dramatically speed up how quickly new treatments move from the lab to patients.
The institute will function as a kind of “factory for cures” by housing, in one place, scientists discovering new therapies, specialized clean rooms that can manufacture cell and gene therapies, and clinical spaces where patients can receive those experimental treatments. Its work will center on cellular therapies that use a patient’s own cells as medicine, gene therapies that fix or replace faulty genes, immunotherapies that train the immune system to fight cancer, and nanotechnology‑enabled drug delivery that sends medicine directly to diseased cells while sparing healthy tissue. Expected to open in 2027, the institute is projected to create hundreds of high‑skilled jobs and attract biotech partners such as AstraZeneca—already collaborating with UVA on new ways to combat serious infections.
Statewide Clinical Trials Network Expands Access Across Virginia
UVA Health launched its Clinical Trials Network in September 2025, bringing cutting-edge clinical trials to community hospitals across Virginia for the first time. Patients at UVA Health’s Culpeper and Prince William medical centers can now access clinical trials for breast cancer, kidney stones, and other conditions without traveling to Charlottesville—dramatically expanding opportunities for Virginians to participate in groundbreaking research.
MORE BREAKTHROUGHS
Long COVID Treatment Trials Offer Hope
UVA Health joined nationwide NIH RECOVER Initiative trials in March 2025 to test potential long COVID treatments for patients suffering from POTS (postural orthostatic tachycardia syndrome) and sleep disturbances. These trials are testing multiple promising therapies including intravenous immunoglobulin, ivabradine, modafinil, and light therapy to address the debilitating symptoms affecting millions of Americans.
Children with Wheezing Get Better Treatment
Dr. Gerald Teague discovered that nearly a quarter of children with recurrent wheezing have “silent” viral lung infections better treated with antivirals than commonly prescribed steroids. This discovery can immediately improve care and prevent lifelong side effects like stunted growth from unnecessary steroid use—a game-changer for pediatric respiratory care.
World’s Most Advanced Robotic Surgery System
UVA Health became among the first hospitals worldwide—and the first in Virginia—to adopt the da Vinci 5 robotic surgical system in 2024. Featuring 150 design innovations including force feedback technology and improved 3D vision, this cutting-edge system enables more precise minimally invasive procedures across multiple surgical specialties.
Incision-Free Prostate Treatment
UVA Health introduced high-intensity focused ultrasound (HIFU) technology in November 2025 for treating prostate conditions without a single incision. This revolutionary robotic technology offers more precise treatment with smoother recovery and fewer side effects for patients.