“This is a problem we can potentially address with a straightforward manufacturing strategy by removing these cells before therapy production.”
Katy Rezvani, M.D., Ph.D.
Institute for Cell Therapy Discovery & Innovation

  • CAR NK cell therapy is a novel immunotherapy made from natural killer (NK) cells engineered to better recognize and eliminate cancer cells
  • At UT MD Anderson, CAR NK cell therapies are created from NK cells collected from donated umbilical cord blood
  • MD Anderson researchers identified a small population of immature NK cells associated with poorer treatment outcomes
  • These immature cells can pick up cancer proteins through a process known as trogocytosis and become false targets, causing therapeutic NK cells to attack one another
  • Removing the immature cells before manufacturing improved CAR NK cell function and improved tumor control in preclinical models

July 29, 2026 – The composition of donor cord blood may play a critical role in the success of chimeric antigen receptor (CAR) natural killer (NK) cell therapies. A small population of immature NK cells present in donor cord blood can undermine the activity of CAR NK cell therapy by redirecting potent immune cells away from tumors and causing them to attack one another, according to researchers from The University of Texas MD Anderson Cancer Center.

The study, published in Cancer Cell, identifies a previously unrecognized mechanism of CAR NK cell dysfunction and offers a practical strategy to improve the potency and consistency of future off-the-shelf NK cell therapies. Researchers found that cord blood units enriched for mature NK cells were linked to stronger treatment responses and better patient outcomes, while higher levels of immature NK cells were associated with reduced effectiveness. The team discovered that these immature cells actively disrupted the function of more potent CAR NK cells.

“Our findings show that a relatively small population of immature NK cells can have a disproportionately harmful effect on the entire therapeutic product,” said principal investigator Katy Rezvani, M.D., Ph.D., vice president and head of the Institute for Cell Therapy Discovery & Innovation, and professor of Stem Cell Transplantation and Cellular Therapy. “Importantly, this is a problem we can potentially address with a straightforward manufacturing strategy by removing these cells before therapy production.”

The study was led by first and co-corresponding author Ye Ethan Li, M.D., Ph.D., an assistant professor at UT MD Anderson whose work helped define the biological differences between these NK cell populations and uncover the mechanism responsible for impaired CAR NK cell function.

What are CAR NK cell therapies? 

CAR NK cells are an investigational ‘off-the-shelf’ immunotherapy in which natural killer cells are engineered to better recognize and eliminate cancer cells. At UT MD Anderson, researchers have pioneered the development of off-the-shelf CAR NK cell therapies manufactured from donated umbilical cord blood.

Unlike patient-specific cell therapies, cord blood-derived CAR NK cells can be manufactured in advance, cryopreserved and potentially made readily available to patients when needed.

However, donor cord blood units contain biologically diverse populations of NK cells. The researchers sought to understand whether this cellular composition could influence the quality and effectiveness of the final CAR NK cell product.

What are the key findings of the study? 

Researchers discovered that immature NK cells can pick up proteins from cancer cells after coming into contact with them. This process is known as trogocytosis.

As a result, the immature NK cells begin to display cancer proteins on their own surface. The engineered CAR NK cells can then mistake these immature immune cells for cancer cells and attack them. In effect, the immature NK cells create false targets that distract the therapeutic cells from the cancer.

These interactions reduced the fitness and persistence of the stronger NK cells and weakened their ability to control the tumor.

Can removing immature NK cells improve CAR NK cell therapy?

When the immature NK cells were removed before manufacturing, the remaining cells were more effective, persisted longer and showed stronger antitumor activity. This strategy enhanced tumor control and survival in preclinical models of lymphoma and ovarian cancer.

The study provides a new framework for selecting donor cord blood and manufacturing CAR NK cell therapies. Researchers now are working to incorporate these findings into next-generation CAR NK cell platforms with the goal of producing more consistent, potent and durable therapies for patients.

***

This research was supported by philanthropic contributions to the Institute for Cell Therapy Discovery & Innovation at UT MD Anderson. For a full list of collaborating authors, disclosures and funding sources, see the full paper in Cancer Cell.



JULY 28, 2026 — The U.S. Department of Health and Human Services (HHS) today applauds the release of the Trump Administration’s new United States Government Policy for Stopping High-Risk Life Sciences Research. This comprehensive framework prohibits federally supported dangerous gain-of-function (DGOF) research while establishing stronger oversight for life sciences research that could pose significant risks to public health, biosecurity, or national security. The policy fulfills President Trump’s Executive Order aimed at improving the safety and security of biological research, and it ensures that taxpayer-funded research advances scientific discovery and medical innovation without exposing Americans to biological risks.

“The federal government has a duty to protect the American people—not fund research that could put them at risk,” said HHS Secretary Robert F. Kennedy, Jr. “Today, we are ending federal support for dangerous gain-of-function research and replacing weak oversight with clear, enforceable safeguards. This policy delivers on President Trump’s commitment to put safety first while ensuring America remains the global leader in ethical, responsible, and lifesaving biomedical research.”

Specifically, the new policy prohibits federal support for dangerous gain-of-function research conducted in the United States and abroad, establishes rigorous independent review for certain high-risk life sciences research, and restricts federal funding for research conducted in countries or institutions that lack appropriate biosafety, biosecurity, and oversight standards. At the same time, the policy preserves critical biomedical research — including the development of vaccines, therapeutics, diagnostics, and other medical countermeasures — under strong safety and security safeguards.

The policy replaces previous federal oversight frameworks with a unified, risk-based approach focused on the potential consequences of research rather than a limited list of pathogens. It establishes clear responsibilities for researchers, research institutions, and federal agencies to identify, assess, and manage the highest-risk life sciences research before taxpayer dollars are awarded.

“This policy reflects a fundamental principle of science: the pursuit of knowledge must be matched by a commitment to responsibility,” said NIH Director Dr. Jay Bhattacharya. “The overwhelming majority of biomedical research provides enormous benefits to society, and this framework helps ensure that research with the potential to create extraordinary biological risks receives the highest level of scrutiny. It protects scientific innovation while strengthening public trust that federally supported research is conducted safely, transparently, and in the best interests of the American people.”

The United States also is calling on international partners to strengthen biosafety and biosecurity standards and join the effort to end dangerous gain-of-function research that could threaten global health and security. By establishing clear guardrails for federally supported research, the new policy reinforces America’s leadership in responsible science while protecting the health, safety, and security of future generations.

The United States Government Policy for Stopping High-Risk Life Sciences Research is available here [PDF, 1.50 MB].



A panel of proteins could help match ideal candidates with preventative trials.

July 27, 2026 – Months to several years before amyotrophic lateral sclerosis (ALS) symptoms arise, levels of certain blood proteins may dramatically shift. Researchers analyzed data from the long-running, National Institutes of Health (NIH)-funded Pre-symptomatic Familial ALS (Pre-fALS) study, to identify a lineup of key proteins that may predict the emergence of clinically manifest ALS. By anticipating the arrival of symptoms, investigators could intervene with preventative therapies before the irreversible motor neuron damage that is characteristic of ALS sets in.

“If someone carrying an ALS-associated genetic variant had asked me in the past when they would become symptomatic, I would have struggled to provide a reasonable estimate,” said senior author Michael Benatar, M.D., Ph.D., a professor of neurology and public health sciences at the University of Miami. “These biomarkers give us a far better idea of the timing, allowing us to estimate the time to symptom onset with an average error of about 18 months. That’s something we can work with.”

For nearly 20 years, the Pre-fALS study, led by Benatar and Joanne Wuu, Sc.M., research associate professor of neurology and public health sciences at the University of Miami, has collected data and biological samples from people who are at significantly elevated genetic risk for ALS but have not yet progressed, or phenoconverted, to the disease. While this cohort is unique, permitting the examination of presymptomatic ALS, recent studies suggest that findings from Pre-fALS are likely relevant to the broader population.

In 2017, an analysis of ten Pre-fALS participants who had developed symptoms showed that neurofilament light chain (NfL), a structural protein in neurons, spiked in their blood in the months preceding ALS phenoconversion.

As more study participants have begun showing symptoms or signs of disease, new opportunities to search for other pre-symptomatic ALS biomarkers have emerged.

Now, the investigators applied a high-throughput protein, or proteomic, analysis method called Olink to plasma samples from 137 study participants, 33 of whom had demonstrated clinical manifestations of ALS or frontotemporal dementia. Having collected data on the levels of more than 5,000 proteins, the team identified 92 whose levels differed in people before they eventually showed symptoms.

Using machine-learning techniques, the authors tested how various combinations of proteins could predict future risk of phenoconversion. They settled on a select panel of 19, including NfL, that maximized predictive accuracy across time horizons ranging from six months to five years. The researchers demonstrated that, with data on these 19 proteins, their predictive models could estimate a patient’s onset within less than two years of the actual time that they showed signs of disease.

They also produced similar results using data from the UK Biobank, which, despite some limitations, is more representative of the general population than the genetically predisposed cohort of Pre-fALS.

“With preventative gene-targeting treatments now becoming available, there is a particularly urgent need for reliable biofluid-based signatures that indicate near-term onset in individuals that carry ALS risk genes,” said Amy Bany Adams, Ph.D., acting director of NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

Tofersen, a drug approved for symptomatic ALS, is currently being evaluated as a preventative therapeutic in pre-symptmoatic ALS through ATLAS, a clinical trial designed by Benatar in partnership with the company Biogen. ATLAS will test whether starting treatment shortly before symptoms appear could avert or delay the onset of ALS.

“This is all possible because of the members of the carrier community who believe in our mission of preventing ALS and have supported and participated in our research. It has been one of my life’s greatest privileges to give something back,” Benatar said.

This research was supported by NIH through NINDS grants R01NS105479 and U54NS092091.

About the National Institute of Neurological Disorders and Stroke (NINDS): NINDS is the nation’s leading funder of research on the brain and nervous system. The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease. https://www.ninds.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

NIH…Turning Discovery Into Health®

Reference

Ximing Ran, Joanne Wuu (co-first authors), Zhaohui S. Qin et al. Longitudinal plasma proteomics predict phenoconversion to clinically manifest ALS. Nature Medicine. July 27, 2026. DOI: 10.1038/s41591-026-04528-x



Through Tampa General Hospital’s Global Medicine Program, Mercedes Marmorek traveled from Argentina for specialized parathyroid care and found relief from years of symptoms caused by primary hyperparathyroidism.

July 27, 2026 – After years of undiagnosed symptoms from primary hyperparathyroidism, 49-year-old Mercedes Marmorek traveled from Argentina to Tampa General Hospital (TGH) for complex specialized care she could not find anywhere else in the world. Through TGH’s Global Medicine Program, she connected with Dr. Jose Lopez and underwent a life-changing parathyroidectomy August 18, 2025.

For years, Mercedes endured exhaustion, bone pain and anxiety. “I felt like I was living in a fog,” she said. Despite consulting multiple specialists, she couldn’t get answers. Misdiagnosed and sent for psychiatric treatment, her actual condition, primary hyperparathyroidism, was only identified after years of searching for a proper diagnosis. But no surgeon in Argentina could perform the minimally invasive procedure she needed.

“I knew I had to look elsewhere,” Mercedes explained.

Searching online, Mercedes discovered Tampa General Hospital’s Parathyroid & Thyroid Institute. With more than 13,000 parathyroidectomies performed, Dr. Jose Lopez and Dr. Douglas Politz are among the most experienced parathyroid surgeons in the world.

“When I read about the success rates, I knew I could trust them,” Mercedes said.

TGH’s Global Medicine Program offered a clear path forward. A virtual consultation with Dr. Lopez confirmed she was a candidate for surgery, and the program’s transparent self-pay packages made planning her care simple.

A Surgery That Changed Everything

Mercedes flew to Tampa for her procedure on August 18, 2025. Using his years of advanced surgical expertise and intraoperative nuclear mapping, Dr. Lopez located two malfunctioning parathyroid glands that previous scans had missed. “If I had gone elsewhere, one of the glands might have been left untreated,” Mercedes shared.

At TGH, surgeons examine all four parathyroid glands during every procedure to ensure comprehensive care. This thorough approach set TGH apart and made all the difference for Mercedes.

Life After Surgery: A New Beginning

Post-surgery, Mercedes felt like herself again. Her fatigue, anxiety and pain vanished. “I feel like I’ve been given my life back,” she said. Mercedes returned to leading her opera company, grateful for the advanced care, compassionate team and seamless experience at TGH.

“From my first call to follow-up, everything was easy and clear,” Mercedes said. “I finally feel free.”

Geography Shouldn’t Limit Care

Mercedes is one of many international patients benefiting from TGH’s expertise. Tampa General’s Global Medicine Program supports patients worldwide with support with travel logistics, transparent financial options and personalized care coordination.

Take the First Step Toward Answers

If you or a loved one struggles with unexplained symptoms or a diagnosed parathyroid condition, TGH’s Global Medicine Program can help. Call (866) 839-9828 or complete the Patient Intake Form to start your journey to better care.



BY: Rebecca Bromelkamp

july 28, 2026  — An international phase 3 study performed at all three Mayo Clinic destination medical centers found that a new immunotherapy helped many patients with an aggressive form of bladder cancer keep their bladder instead of having it surgically removed. The treatment also kept many patients cancer-free for more than two years and caused mostly mild side effects. The findings were published in The Lancet Oncology.

Patients in the study had high-risk bladder cancer that had returned despite standard treatment with bacillus Calmette-Guérin (BCG). For these patients, surgery to remove the bladder is typically the recommended next step. The new immunotherapy treatment, called cretostimogene grenadenorepvec, produced a complete response, with no detectable signs of cancer in 75% of patients, with many of those responses lasting beyond two years.

Portrait of Dr. Mark Tyson
Mark Tyson II, M.D.

“Until now, patients whose cancer returned after BCG therapy have had few effective options besides bladder removal,” says Mark Tyson II, M.D., lead author of the study and a urologist at Mayo Clinic in Arizona. “This study shows we may be able to offer many of those patients another option without compromising cancer control.”

Among patients whose cancer completely disappeared after treatment, about 60% remained cancer-free two years later. In one patient, the cancer has not returned for more than four years after completing treatment. Most treatment-related side effects were mild, temporary bladder symptoms, and researchers reported no severe treatment-related side effects.

“For patients, this isn’t just about treating cancer — it’s about preserving quality of life,” says Dr. Tyson. “Avoiding bladder removal can have a profound impact on daily living. Seeing patients remain cancer-free for years while keeping their bladder is exactly the outcome we’ve been hoping to achieve.”

The study included 115 patients treated at 41 medical centers across North America, Asia and Australia. Two years after starting treatment, an estimated 81% of patients had not needed surgery to remove their bladder. The researchers concluded that cretostimogene grenadenorepvec could become an important bladder-sparing treatment option for patients whose high-risk bladder cancer has returned after standard BCG therapy. They also noted that the treatment has not been directly compared with other bladder-sparing therapies in clinical trials.

The study was funded by CG Oncology. For a complete list of authors, disclosures and funding, review the study. The therapy remains investigational.

Mayo Clinic
Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news.