Health
Heart failure is reversed with new gene therapy in animal study: ‘Unprecedented recovery’
Heart failure has historically been irreversible, but the outcome of a new study suggests that could someday change.
At the University of Utah, scientists used a new gene therapy that was shown to reverse the effects of heart failure in a large animal study.
In the study, pigs with heart failure were found to have low levels of cardiac bridging integrator 1 (cBIN1), a critical heart protein.
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The scientists injected a harmless virus into the pigs’ bloodstreams to carry the cBIN1 gene into their heart cells, according to a university press release.
The pigs survived for the six-month duration of the study, while they would have been expected to die from heart failure without the gene therapy.
Senior study co-authors Robin Shaw, MD, PhD (left) and TingTing Hong, MD, PhD (right) are pictured in the lab. (Charlie Ehlert / University of Utah Health.)
In what the researchers called an “unprecedented recovery of cardiac function,” the IV injection appeared to improve heart function by increasing the amount of blood it can pump, which “dramatically improves survival.”
The pigs’ hearts also appeared to be “less dilated and less thinned out” after the therapy, “closer in appearance to that of non-failing hearts.”
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While previous attempts to treat heart failure improved function by just 5% to 10%, the gene therapy used in the new study resulted in 30% improvement, according to the researchers.
The study, which was funded by the National Institutes of Health, was published on Tuesday in the journal npj Regenerative Medicine.
Microscope images of failing heart cells (top) and heart cells that received gene therapy (bottom). Cell boundaries, labeled in magenta, are more organized after gene therapy, and the level of cBIN1 protein (green) is higher. (Hong Lab)
“Even though the animals are still facing stress on the heart to induce heart failure, in animals that got the treatment, we saw recovery of heart function and that the heart also stabilizes or shrinks,” said TingTing Hong, MD, PhD, associate professor of pharmacology and toxicology at the University of Utah, in the release.
“We call this reverse remodeling. It’s going back to what the normal heart should look like.”
“A possible new therapy to cure heart failure is on the way,” Hong told Fox News Digital in a statement.
“A possible new therapy to cure heart failure is on the way.”
The researchers were surprised to find that the gene therapy worked so well in large animals at such a low dose, Hong added.
Co-senior author Robin Shaw, MD, PhD, director of the Nora Eccles Harrison Cardiovascular Research and Training Institute at the University of Utah, said the “unprecedented” study ushers in a “new paradigm” for heart failure treatments.
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“Given our treatment efficacy, the complex multi-organ syndrome of heart failure can be reduced to a treatable disease of failing heart muscle,” he told Fox News Digital via email.
“The toxicity of gene therapy increases with dose, so our low dose suggests that our gene therapy approach will be safe for patients.”
While gene therapy has historically been used for rare diseases, the study results suggest that it could also be an effective approach for “acquired disease,” a researcher said. (iStock)
While gene therapy has historically been used for rare diseases, the study results suggest that it could also be an effective approach for “acquired disease,” according to Shaw.
The study did have some limitations, the researchers acknowledged.
“Dose escalation and toxicology studies are still needed for the therapy to move to the next step [toward FDA approval],” Hong noted.
It is also uncertain whether the gene therapy will work for people who have obtained a natural immunity to the virus that carries the therapy, the researchers said.
The toxicology study is currently underway, and the team plans to start human clinical trials in the fall of 2025, Hong said.
Cardiologists weigh in
Dr. Jasdeep Dalawari, interventional cardiologist and regional chief medical officer at VitalSolution, an Ingenovis Health Company based in Ohio, was not involved in the research but shared his reaction to the findings.
“Gene therapy, precision medicine and personalized health care is the future.”
“Research in the animal phase is always interesting, but the application to human test subjects is necessary in terms of understanding if this approach would have the same effect in humans,” he told Fox News Digital.
While previous attempts to treat heart failure improved function by just 5% to 10%, the gene therapy used in the new study resulted in 30% improvement, according to the researchers. (iStock)
“That said, there are many genetic modifications happening in different diseases, like cystic fibrosis and muscular dystrophy, that are looking at a similar intervention – injecting healthy genes in the hopes of finding cures,” he went on.
“Gene therapy, precision medicine and personalized health care is the future, and I look forward to learning more about this.”
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Dr. Johanna Contreras, advanced heart failure and transplant cardiologist at the Mount Sinai Fuster Heart Hospital in New York City, noted that conventional pharmacological interventions can help to alleviate cardiac stress and “systemic congestion,” but “for the most part, they do not address the pathogenic remodeling of failing heart muscle.”
About 6.7 million adults in the U.S. have heart failure, statistics show. (iStock)
“Gene therapy has emerged as a new modality that could interfere or modify the expression of several proteins, thus it could alter the pathologic remodeling of the heart muscle that exists in heart failure,” Contreras, who also was not involved in the study, told Fox News Digital.
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Contreras reiterated that human trials are needed to determine whether this therapy will be effective outside animal models and to identify any “downstream effects.”
“I will look forward to learning more and eventually learning if this could one day be applied to humans with heart failure.”
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A new injectable therapy is showing positive results in reducing melanoma throughout a five-year period.
The personalized mRNA cancer therapy, called intismeran autogene, combined with the cancer immunotherapy drug KEYTRUDA (pembrolizumab), is a collaboration between Merck and Moderna.
The results from the phase 2b KEYNOTE-942 study were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago on May 27.
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After about a five-year follow-up, the combo drug was found to reduce the risk of melanoma recurrence or death by 49% compared to pembrolizumab alone.
The researchers analyzed data from 157 patients with high-risk stage 3 and 4 melanoma whose cancer had been removed via surgery. The participants were split into two groups — one received the combo therapy and the other only received pembrolizumab, according to a press release.
The therapy was found to reduce the risk of melanoma recurrence or death by 49% compared to pembrolizumab alone after a five-year follow-up. (iStock)
The findings revealed that the combination group saw benefits that were “sustained and durable over time.”
Intismeran autogene is designed using mutations identified in a patient’s own tumor, with the intention of teaching the immune system what the cancer looks like so that it can recognize and attack it.
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According to the researchers, intismeran is “well-tolerated” with a “manageable” safety profile.
The most commonly cited side effects of the personalized mRNA vaccine plus KEYTRUDA were fatigue, injection-site pain, chills, fever and headache. The researchers reported no new long-term safety concerns and no severe vaccine-related adverse events.
The combination therapy is currently being evaluated in a phase 3 study — the final confirmation stage.
Patients with late-stage melanoma have a “significant risk” of cancer recurrence, according to an expert. (iStock)
In a Merck press release from January, Kyle Holen, MD, Moderna’s senior vice president and head of development, oncology and therapeutics, noted that this data highlights the “potential of a prolonged benefit … in patients with resected high-risk melanoma.”
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“We continue to invest in our platform in oncology because of encouraging outcomes like these, which illustrate mRNA’s potential in cancer care,” he said.
Dr. Marjorie Green, senior vice president and head of oncology, global clinical development at Merck Research Laboratories, also commented that for many patients with stage 3 or 4 melanoma, there is a “significant risk of recurrence following surgery.”
Researchers confirmed that the combination therapy is currently being evaluated in a phase 3 study. (iStock)
“As such, demonstrating the longer-term potential of intismeran autogene and KEYTRUDA to reduce the risk of recurrence for certain patients with melanoma is a meaningful milestone,” she said.
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The company cited encouraging five-year follow-up data and pointed to upcoming late-stage INTerpath trial results with Moderna in several hard-to-treat cancers.
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An accidental lab discovery has opened the door to entirely new ways of preventing the flu.
While investigating how influenza replicates, researchers discovered that different flu strains use completely different strategies to infiltrate human cells, SWNS reported.
By targeting the specific molecules the viruses rely on, scientists found that they could block them from entering new cells and halt their replication altogether.
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Researchers say these “fundamental insights” into seasonal influenza highlight a clear path toward developing better preventive medications.
“The hope is that fundamental, curiosity-based research like this helps to pave the way for novel strategies to treat and prevent influenza infections,” principal investigator Dr. Emily Bruce, from the University of Vermont’s Larner College of Medicine, said in the SWNS report.
While investigating how influenza replicates, researchers discovered that different flu strains use completely different strategies to infiltrate human cells. (iStock)
While several flu strains cause illness, H1N1 and H3N2 influenza A viruses are the most common. However, current flu tests cannot differentiate between them, and clinical treatments are identical for both.
Although vaccines and antivirals are available, Bruce noted a “dire” need for better medications to stop the virus from spreading cell to xxcell.
“You don’t get sick when a virus is in one cell,” he noted. “You get sick because a virus replicates itself and goes into many more cells.”
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The study, which was published in The Journal of Virology, originally aimed to map how viral RNA segments are transported within cells to create new viral particles.
The team used H1N1 and H3N2 viruses isolated from the nasal passages of positive patients in 2022.
Clinical treatments remain identical for both primary strains of the flu virus. (iStock)
During the investigation, the team unexpectedly stumbled upon a cellular pathway that blocked the virus from entering lung cells, SWNS reported.
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The data revealed that when a specific human protein called Rab11B was depleted, H3N2 viruses failed to enter human lung cells. H1N1 viruses were completely unaffected.
Using reverse genetics, the team mapped this defect and uncovered a brand-new, H3N2-specific role for Rab11B during viral entry.
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This discovery challenged the scientific assumption that all flu viruses enter cells the same way.
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“Viruses are like pirates from different countries hijacking someone’s ship,” Bruce said. “Different viruses, like different types of pirates, use different methods to get onboard.”
This discovery challenged the scientific assumption that all flu viruses enter cells the same way. (iStock)
“We had previously thought that all flu viruses used the same way to get into a cell, but we discovered that this is not true,” she went on. “H1N1 and H3N2 need different proteins to get in, and if you get rid of the right protein, a specific virus can’t get in.”
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While these findings identify a critical cellular pathway for viral entry, the study was conducted using isolated cells, the researchers acknowledged.
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Further research is needed to determine whether blocking the protein is safe and effective within a live, complex human respiratory system.
Bruce and the team hope to conduct further research to determine whether this Rab11B-dependency is a fundamental property of H3N2, or if it’s a trait unique to currently circulating flu strains.
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Eating processed meat like ham, sausage and bacon may be linked to a higher risk of certain types of cancer, according to new research.
While health organizations have already confirmed that processed meat can contribute to colon cancer, this study looked closer at cancers in the upper digestive tract, where the link has historically been less clear.
To understand these connections, researchers from the European Prospective Investigation into Cancer and Nutrition (EPIC), one of the world’s largest long-term nutrition and cancer cohorts, tracked the health and diets of 450,112 people across Europe for an average of 14 years.
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The study group included 131,426 men and 318,686 women, according to the study’s press release.
During the follow-up period, 876 people developed stomach cancer and 215 people developed esophageal adenocarcinoma, which is cancer of the tube connecting the mouth to the stomach.
For female participants, eating both processed meat and white meat was linked to an increased risk of developing the disease. (iStock)
Researchers tracked where the stomach cancers grew, separating them into the upper part of the stomach near the throat and the lower part of the stomach.
The researchers also sorted the tumors into two categories based on how the cancer cells appeared under a microscope: intestinal, which forms more organized structures, and diffuse, in which the cells are more scattered throughout the tissue.
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After adjusting for other lifestyle factors, the researchers found that for every extra 30 grams of processed meat a person ate per day, their overall risk of stomach cancer went up by 9%. Eating that same extra 30 grams a day was also linked to a 13% higher risk of esophageal adenocarcinoma.
A standard single slice of regular deli-sliced ham or lunch meat averages around 28 grams, according to USDA data and nutritional tracking databases.
An extra 20 grams of white meat, such as chicken and turkey, was linked to a 12% higher risk of cancer in the main body of the stomach. (iStock)
An extra 20 grams of white meat, such as chicken or turkey, was linked to a 12% higher risk of cancer in the main body of the stomach, the researchers noted.
The study also revealed differences between men and women. For male participants, only processed meat showed a clear, statistically significant link to a higher risk of stomach cancer. For female participants, however, eating both processed meat and white meat was linked to an increased risk.
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These findings align with global health benchmarks, particularly those established by the World Health Organization’s International Agency for Research on Cancer.
The agency has long classified processed meat as a known human carcinogen, primarily due to its strong, well-documented links to colorectal cancer.
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However, health organizations have also consistently pointed to a potential, yet less definitive, relationship between these meats and cancers of the stomach.
Eating 30 grams of processed meat a day, or the equivalent to one slice of ham, was linked to a 13% higher risk of esophageal adenocarcinoma. (iStock)
Further scientific investigation is needed to confirm the findings and to account for other underlying risk factors, such as certain stomach infections, which could interact with dietary habits.
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A key limitation of the study is its reliance on self-reported diets, which can sometimes lead to inaccuracies in how participants recall their meat consumption over time, the researchers noted.
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The findings were published in the International Journal of Cancer.
Fox News Digital reached out to the researchers requesting comment.
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