The 67-year-old patient had run out of options.
The bioprosthetic aortic valve in his heart was failing and needed to be replaced. But the opening of his left coronary artery was so close to the valve that it would probably get blocked by a valve replacement. A history of vascular disease and other interventions made the patient’s heart too weak for an open-heart bypass procedure.
Two surgeons found a way around the obstacle, literally: creating a bypass without opening the patient’s chest. The surgeons “cut” the heart muscle using an electrified wire at the end of a catheter, which allowed them to replace the aortic valve. The challenge was to divert blood flow safely through the patient’s heart — a procedure they had tried only on animal models.
It worked.
That is just one of many recent pioneering achievements that are improving care and saving lives. A baby born with a life-threatening metabolic disorder that could harm his brain and liver enjoyed Christmas at home because of gene editing. A bladder and kidney transplant allowed a patient to discontinue dialysis and urinate normally for the first time in years. A blood test for Alzheimer's disease will help doctors start treatments sooner.
Read more about these life-saving achievements.
The first minimally invasive coronary bypass surgery
Adam Greenbaum, MD, and Vasilis Babaliaros, MD, co-directors of the Structural Heart and Valve Center at Emory University Health, along with colleagues at the National Heart, Lung, and Blood Institute of the National Institutes of Health, had been preparing for just such a circumstance: a patient who needed a bypass operation but could not sustain traditional open heart surgery. The team had previously developed a novel technique of accessing the heart muscle with an electrified wire at the end of a catheter, allowing them to replace a mitral valve without having to open the chest. Safely diverting blood flow through the patient’s heart was a new challenge.
With a traditional coronary bypass, surgeons open the chest, typically by cutting through the breastbone, and might place the patient on a heart-lung machine. The surgeons sew a spare blood vessel, usually taken from the chest or leg, around the blocked blood vessel to create a new route for blood to reach the heart muscle. It’s a highly effective but major operation, with a significant incision and recovery period.
In contrast, the Emory team planned to create a bypass without opening the chest at all. Working entirely through catheters threaded through blood vessels, they used a covered metal stent as the bypass conduit instead of a harvested vein or artery. Using transcatheter electrosurgical tools, they connected the aorta — the main artery leaving the heart — to one of the heart’s own arteries through the space around the heart and then used the covered stent to ensure that the new channel would remain open.
The surgeons had successfully practiced the procedure, called ventriculo-coronary transcatheter outward navigation and re-entry (VECTOR), in animal models. In this case, a human candidate benefited by receiving the procedure on the basis of compassionate use, when a patient has a life-threatening condition for which there is no alternative treatment and thereby gains access to an investigational therapy.
“What’s unique about this is that it was a fully percutaneous bypass,” Greenbaum says of the procedure, performed in spring of 2025. “We basically relocated the left main artery up high enough in the ascending aorta that it would not be blocked.”
A second patient underwent the procedure later in the year. In that case, both the right and left main arteries were bypassed.
“Now having done three vessels in two patients, we’ve shown it’s reproducible,” Greenbaum says of the delicate feat. “Sometimes when you do something once, you think, ‘Did we just get lucky?’ Although it needs to be refined, this procedure is certainly doable.”
Although the procedure remains limited to patients whose conditions make them poor candidates for traditional bypass, Greenbaum says, the minimally invasive electrosurgical approach could eventually become an option for healthier patients, allowing them to avoid the pain, infection risk, and lengthy recovery of open-heart surgery.
The first personalized CRIPSR-based gene editing therapy
Within days of his birth in August of 2024, a baby named KJ developed symptoms that pointed to a metabolic disorder. Dangerously ill, he was transferred to the Children’s Hospital of Pennsylvania (CHOP), where tests revealed a rare urea cycle disorder, caused by a deficiency in the carbamoyl phosphate synthetase 1 (CPS1) gene. He lacked an enzyme in the liver needed to convert ammonia to urea, which is normally excreted from the body as urine. The disorder can lead to a lethal buildup of ammonia in the body, particularly in the brain and liver.
KJ was put on dialysis to filter out the ammonia, which put him out of the woods temporarily. His parents learned, however, that the only treatment for the devastating disease was a liver transplant, assuming that KJ was healthy enough to endure the procedure.
KJ’s birth coincided with the efforts of two physician scientists: Rebecca Ahrens-Nicklas, MD, PhD, an assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania and director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP, and Kiran Musunuru, MD, PhD, MPH, professor for translational research at the school and co-director of the Orphan Disease Center, a joint CHOP-Penn center. Their team had been researching how to use CRISPR technology to develop customized treatments for individuals with diseases like CPS1 deficiency.
Previous gene-editing techniques had focused on diseases that affect thousands of patients, specifically sickle cell disease and beta thalassemia, for which there are CRISPR therapies approved by the U.S. Food and Drug Administration (FDA).
Ahrens-Nicklas and Musunuru went to work designing and manufacturing a base editing therapy to be delivered via lipid nanoparticles to the liver to correct KJ’s faulty enzyme. In February 2025, just six months after the work began, KJ received his first infusion of the investigational therapy and became the first person to receive a CRISPR-based treatment customized to his specific genetic variant.
KJ’s condition rapidly improved and he received additional doses in March and April with no serious side effects. In June he was able to go home, and in December he took his first steps and enjoyed his first Christmas at home with his family.
KJ has done well post-treatment but will need to be closely monitored. The breakthrough, as described in the New England Journal of Medicine, could pave the way for gene-editing technology to be used to treat people with other rare diseases that currently lack effective therapies.
“We hope this is the first of many,” says Ahrens-Nicklas. “I really hope that 10 years from now, targeted, personalized therapies are available for most of my patients.”
The first bladder transplant in a human
Up to now, patients with a nonfunctioning, or “terminal,” bladder required surgery to augment or replace it. Such surgeries involve using part of the patient’s intestine to create either a new reservoir (“ileal neobladder”) or a pathway for urine to exit the body (“ileal conduit”). While these procedures are effective, they can lead to considerable potential for infections, compromised kidney function, and digestive problems, and might require patients to self-catheterize.
Now a more natural option is on the horizon for select patients: a bladder transplant.
In May of 2025, Inderbir Gill, MD, chair and distinguished professor in the Catherine and Joseph Aresty department of urology and the Shirley and Donald Skinner Chair of Urologic Cancer Surgery at the Keck School of Medicine of the University of Southern California (USC), and Nima Nassiri, MD, assistant professor of urology and kidney transplantation at University of California, Los Angeles, David Geffen School of Medicine, performed the world’s first bladder transplant in a human patient. They followed with a second transplant months later.
“This could be a game-changer for appropriately selected patients,” says Gill. “It’s exciting to be able to offer this new potential option.”
Harvesting and transplanting a bladder is no easy task. The organ lies deep in the pelvis and has a complex network of blood vessels.
“The bladder has a significant multi-directional blood supply coming from both the right side and the left side of the deep pelvis,” Gill says. “These are small blood vessels, which are difficult to fully identify during surgery. We had to develop a new technique to remove the bladder with all its vasculature intact to have a shot at transplanting it successfully and keeping it alive.”
Gill and Nassiri refined the technique using animal models, followed by cadaveric models, and then research donors — a four-year process.
Transplanting a bladder has been challenging, in part because transplant patients must take immunosuppression drugs for the rest of their lives to minimize the risk of rejection.
“If somebody has a critical organ that is non-functioning, such as a kidney, heart, lung, or liver, you can justify immunosuppression,” Gill says. “These are critical organs, without which we cannot live. But immunosuppression in the context of noncritical transplants [including the bladder] is a different matter.”
That’s why the new transplant procedure is an option best suited for patients who are already on immunosuppressant drugs (e.g., have a preexisting transplant) or those who have kidney failure and a terminal bladder, thus justifying a combined kidney and bladder transplant. Eventually, however, the indications for bladder transplantation could be expanded for other conditions.
The team’s first transplant recipient had lost most of his bladder to cancer and subsequently had his kidneys removed for cancer as well, more than five years earlier. After an eight-hour operation to transplant a new kidney and the bladder and connect the two organs, the patient was able to stop dialysis and urinate normally for the first time in seven years, which significantly improved his quality of life.
“Hopefully, the bladder transplantation will eliminate the complications arising from the use of intestine and help these patients lead normal, healthier lives,” Gill says.
The first blood test cleared for Alzheimer’s disease
An estimated 7.2 million Americans age 65 and older live with Alzheimer’s dementia. But getting an accurate diagnosis has not been easy or affordable.
Until recently, a positive diagnosis required a positron emission tomography (PET) scan of the brain to visualize the telltale abnormalities, known as amyloid plaques and tau tangles, a test that can cost as much as $6,000. Alternatively, clinicians can look for disease biomarkers in the patient’s cerebral spinal fluid (CSF), which requires lumbar puncture (also known as a spinal tap).
Now a simple blood test can quickly and cost effectively confirm the diagnosis. In 2025, the FDA cleared the first Alzheimer’s disease blood test, called Lumipulse, which could dramatically improve the outlook for thousands of people.
“This changes the game,” says Suzanne Schindler, MD, PhD, associate professor of neurology and principal investigator at the University of Washington School of Medicine in St. Louis, who has researched the test and others like it. “We already have a lot of data showing that the blood tests work well, but the FDA’s stamp of approval carries considerable weight and increases the confidence of clinicians that these tests can be used as part of clinical care.”
The timing is critical, Schindler says:
“Now that there are treatments for Alzheimer’s disease available that are specific, and that work best the earlier they're given, there's actually some urgency to testing. Primary care doctors are increasingly doing these tests, and they want to get patients diagnosed more quickly so they can get started on treatments earlier.”
Although six blood tests have been developed for Alzheimer’s disease, Lumipulse is the first one cleared by the FDA. It measures the ratio of two proteins called p-tau217 and amyloid-beta 42 that reflect brain changes that define Alzheimer’s disease.
Research shows that 91% of people who had positive results from the test had the presence of amyloid plaques indicated by PET scan or CSF test, and 97% of people with negative results had a negative amyloid PET scan or CSF test result.
Lumipulse is not a stand-alone test. “It’s one part of a clinical assessment that includes a history and examination and other tests,” Schindler says. “It’s not diagnostic by itself.”
The test is cleared for people 50 and older who have experienced memory or thinking problems and “is not recommended for people who are cognitively unimpaired,” Schindler says.
A positive result could lead to a prescription for an Alzheimer’s therapy, and it may also propel patients to take steps that delay the progression of the disease, such as a better diet and exercise. A negative result, on the other hand, helps clinicians zero in on the root of patients’ symptoms, including other neurological disorders or another cause altogether, such as obstructive sleep apnea.
“The FDA clearance of a blood test heralds a new era in Alzheimer’s disease diagnosis and treatment,” says Schindler.
