Health
Children with inherited deafness hear for first time through experimental gene therapy
- Gene therapy has successfully restored hearing in some children born with inherited deafness, health officials say.
- A study in China and another at the Children’s Hospital of Philadelphia documented significant improvements in hearing in treated children.
- Cochlear implants are commonly used for children with hereditary deafness, but gene therapy offers a potential treatment.
Gene therapy has allowed several children born with inherited deafness to hear.
A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar improvements in an 11-year-old boy treated there. And earlier this month, Chinese researchers published a study showing much the same in two other children.
So far, the experimental therapies target only one rare condition. But scientists say similar treatments could someday help many more kids with other types of deafness caused by genes. Globally, 34 million children have deafness or hearing loss, and genes are responsible for up to 60% of cases. Hereditary deafness is the latest condition scientists are targeting with gene therapy, which is already approved to treat illnesses such as sickle cell disease and severe hemophilia.
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Children with hereditary deafness often get a device called a cochlear implant that helps them hear sound.
Dr. Yilai Shu examines a young patient at the Eye & ENT Hospital of Fudan University in Shanghai, China, after a gene therapy procedure for hereditary deafness. Gene therapy has allowed several children born with inherited deafness to hear. (Courtesy Dr. Yilai Shu via AP)
“No treatment could reverse hearing loss … That’s why we were always trying to develop a therapy,” said Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study published Wednesday in the journal Lancet. “We couldn’t be more happy or excited about the results.”
The team captured patients’ progress in videos. One shows a baby, who previously couldn’t hear at all, looking back in response to a doctor’s words six weeks after treatment. Another shows a little girl 13 weeks after treatment repeating father, mother, grandmother, sister and “I love you.”
All the children in the experiments have a condition that accounts for 2% to 8% of inherited deafness. It’s caused by mutations in a gene responsible for an inner ear protein called otoferlin, which helps hair cells transmit sound to the brain. The one-time therapy delivers a functional copy of that gene to the inner ear during a surgical procedure. Most of the kids were treated in one ear, although one child in the two-person study was treated in both ears.
The study with six children took place at Fudan University in Shanghai, co-led by Dr. Yilai Shu, who trained in Chen’s lab, which collaborated on the research. Funders include Chinese science organizations and biotech company Shanghai Refreshgene Therapeutics.
Researchers observed the children for about six months. They don’t know why the treatment didn’t work in one of them. But the five others, who previously had complete deafness, can now hear a regular conversation and talk with others. Chen estimates they now hear at a level around 60% to 70% of normal. The therapy caused no major side effects.
Preliminary results from other research have been just as positive. New York’s Regeneron Pharmaceuticals announced in October that a child under 2 in a study they sponsored with Decibel Therapeutics showed improvements six weeks after gene therapy. The Philadelphia hospital — one of several sites in a test sponsored by a subsidiary of Eli Lilly called Akouos — reported that their patient, Aissam Dam of Spain, heard sounds for the first time after being treated in October. Though they are muffled like he’s wearing foam earplugs, he’s now able to hear his father’s voice and cars on the road, said Dr. John Germiller, who led the research in Philadelphia.
“It was a dramatic improvement,” Germiller said. “His hearing is improved from a state of complete and profound deafness with no sound at all to the level of mild to moderate hearing loss, which you can say is a mild disability. And that’s very exciting for us and for everyone. “
Columbia University’s Dr. Lawrence Lustig, who is involved in the Regeneron trial, said although the children in these studies don’t wind up with perfect hearing, “even a moderate hearing loss recovery in these kids is pretty astounding.”
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Still, he added, many questions remain, such as how long the therapies will last and whether hearing will continue to improve in the kids.
Also, some people consider gene therapy for deafness ethically problematic. Teresa Blankmeyer Burke, a deaf philosophy professor and bioethicist at Gallaudet University, said in an email that there’s no consensus about the need for gene therapy targeting deafness. She also pointed out that deafness doesn’t cause severe or deadly illness like, for example, sickle cell disease. She said it’s important to engage with deaf community members about prioritization of gene therapy, “particularly as this is perceived by many as potentially an existential threat to the flourishing of signing Deaf communities.”
Meanwhile, researchers said their work is moving forward.
“This is real proof showing gene therapy is working,” Chen said. “It opens up the whole field.”
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Feeling lonely may take a toll on older adults’ memory — but it may not speed up cognitive decline, according to a new study.
Researchers from Colombia, Spain and Sweden analyzed data from more than 10,000 adults ages 65 to 94 across 12 European countries and found those who reported higher levels of loneliness did worse on memory tests at the start of the study, according to research published this month in the journal Aging & Mental Health.
Over a seven-year period, however, memory decline occurred at a similar rate regardless of how lonely participants felt.
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“The finding that loneliness significantly impacted memory, but not the speed of decline in memory over time was a surprising outcome,” lead author Dr. Luis Carlos Venegas-Sanabria of the School of Medicine and Health Sciences at the Universidad del Rosario said in a statement.
Loneliness may be linked to memory performance in older adults, a new study suggests. (iStock)
“It suggests that loneliness may play a more prominent role in the initial state of memory than in its progressive decline,” Venegas-Sanabria said, adding that the findings highlight the importance of addressing loneliness as a factor in cognitive performance.
The findings add to debate about whether loneliness contributes to dementia risk. While loneliness and social isolation are often considered risk factors for cognitive decline, research results have been mixed.
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The study looked at data from the long-running Survey of Health, Ageing and Retirement in Europe (SHARE), which tracked 10,217 older adults between 2012 and 2019. Participants were asked to recall words immediately and after a delay to measure memory performance.
Social isolation and loneliness could play a surprising role in cognitive health among seniors. (iStock)
Loneliness was assessed using three questions about how often participants felt isolated, left out or lacking companionship.
About 8% of participants reported high levels of loneliness at the outset. That group tended to be older, more likely to be female and more likely to have conditions such as depression.
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Researchers found that those with higher loneliness had lower scores on both immediate and delayed memory tests at baseline. Still, all groups — regardless of loneliness level — experienced similar declines in memory over time.
The results suggest loneliness may not directly accelerate the progression of memory loss, though it remains linked to poorer cognitive performance overall.
Researchers look at a brain scan at the National Institutes of Health in Bethesda, Maryland. (Saul Loeb/AFP/Getty Images)
Experts warn, however, that the findings should not be interpreted to mean loneliness is harmless.
“The finding that lonely older adults start with worse memory but don’t decline faster is actually the most interesting part of the paper, and I think it’s easy to misread,” said Jordan Weiss, Ph.D., a scientific advisor and aging expert at Assisted Living Magazine and a professor at NYU Grossman School of Medicine.
“It likely means loneliness does its damage earlier in life, well before people show up in a study like this at 65-plus,” Weiss told Fox News Digital.
By older age, long-term social patterns may already be established, making it harder to detect when the effects of loneliness first took hold, an aging expert says. (iStock)
He suggested that by older age, long-term social patterns may already be established, making it harder to detect when the effects of loneliness first took hold.
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“By the time you’re measuring someone in their late 60s, decades of social connection patterns are already baked in,” he said.
Weiss, who was not involved in the research, added that loneliness may coincide with other health conditions, and noted that participants who felt more isolated also had higher rates of depression, high-blood pressure and diabetes. The link, he said, may reflect a cluster of health risks rather than a direct cause.
“While they can go hand-in-hand, it’s not clear that loneliness contributes to dementia,” a psychotherapist says. (iStock)
Amy Morin, a Florida-based psychotherapist and author, said the findings reflect a broader pattern in research on loneliness and brain health, and that the relationship may be more complex than it appears.
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“The evidence shows there’s a link between loneliness and cognitive decline but there’s no direct evidence of a cause and effect relationship,” she said. “So while they can go hand-in-hand, it’s not clear that loneliness contributes to dementia.”
Morin added that loneliness, which can fluctuate, may not be the root of the problem, but rather a symptom of other underlying mental or physical health issues.
Researchers suggested screening for loneliness be incorporated into routine cognitive assessments as one way to support healthy aging. (iStock)
She said staying socially and mentally engaged is crucial for overall brain health.
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“It’s important to be proactive about social activities,” Morin said. “Joining a book club, having coffee with a friend, or attending faith-based services can be a powerful way to maintain connections in older age.”
The researchers also suggested screening for loneliness be incorporated into routine cognitive assessments as one way to support healthy aging.
Fox News Digital reached out to the researchers for comment.
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Research continues to uncover new details on how fasting may help extend life.
A new study published in the journal Nature Communications investigated how intermittent fasting can boost longevity in small worms often used in aging research.
Researchers from the University of Texas Southwestern Medical Center in Dallas compared worms that were fed normally to those that underwent a 24-hour fast in early adulthood and were then fed again, according to a press release.
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The scientists measured a variety of factors, including stored fat, gene activity related to fat metabolism and lifespan.
The results showed that the life-boosting benefit did not depend on the fasting itself but on the body’s behavior after eating again.
Experts say sustainability is key when choosing a long-term weight-loss strategy. (iStock)
Study lead Peter Douglas, associate professor of molecular biology and a member of the Hamon Center for Regenerative Science and Medicine at UT Southwestern, suggested that these discoveries “shift the focus toward a neglected side of the metabolic coin – the re-feeding phase.”
“Our data suggest that the health-promoting effects of intermittent fasting are not merely a product of the fast itself, but are dependent on how the metabolic machinery recalibrates during the subsequent transition back to a fed state,” he said.
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“Our findings bridge a gap between lipid metabolism and aging research,” he added. “By targeting aging, the single greatest risk factor for human disease, we move beyond treating isolated conditions toward a preventive model of medicine that enhances quality of life for all individuals.”
Lauri Wright, director of nutrition programs at the University of South Florida’s College of Public Health, called this a “high-quality” study that adds an “important nuance to how we think about fasting and longevity.”
Intermittent fasting typically involves limiting meals to an eight-hour daily window or fasting every other day. (iStock)
The benefits of the refeeding phase after fasting were “especially interesting,” Wright, who was not involved in the study, told Fox News Digital.
“The researchers showed that longevity was linked to the body’s ability to turn off fat breakdown after fasting, allowing cells to restore energy balance,” she reiterated.
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“From a scientific standpoint, that’s a meaningful shift because it suggests fasting is not just about burning fat, but about metabolic flexibility.”
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Fasting may support longevity through triggering metabolic switching, enhancing cellular repair and stress resistance and improving markers like insulin sensitivity, research shows.
Limitations and cautions
Although this study provides “important insight” on the power of refeeding, Wright noted that the findings should be approached with caution, as the study was done on worms and cannot always be translated to humans.
“Additionally, it explains how a process might work in a controlled lab condition rather than real-world eating behaviors,” she added as a limitation. “Finally, the study is short-term and doesn’t give us the long-term translation on lifespan outcomes.”
The review found intermittent fasting was barely more effective than doing nothing, according to the study authors. (iStock)
Wright cautioned that fasting is “not a magic solution for longevity, and how you eat overall matters more than when you eat.”
“I advise, first and foremost, to focus on diet quality, including a variety of fruits and vegetables, healthy fats and minimally processed foods,” she said.
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For those who are considering fasting, it’s better to stick with a moderate plan — like a 12- to 14-hour overnight fast — rather than going to extremes, Wright said. After fasting, she recommends focusing on well-balanced meals.
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Several groups of people should be cautioned against fasting, according to Wright, including those with diabetes who are on insulin or hypoglycemic medications, those who are pregnant or breastfeeding, anyone with a history of eating disorders and older adults at risk of malnutrition.
Anyone considering intermittent fasting should consult with a doctor before starting.
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