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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Big moves are continuing in the weight loss landscape in the new year following breakthrough research of GLP-1 medications and other methods.
Weight-loss experts spoke with Fox News Digital about their predictions for the most major changes to come in 2026.
No. 1: Shift to whole-body treatment
Dr. Peter Balazs, a hormone and weight loss specialist in New York and New Jersey, shared that the most important shift is likely to label GLP-1 drugs as “multi-system metabolic modulators” rather than “simple weight loss drugs.”
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“The treatment goal is no longer just BMI reduction, but total cardiometabolic risk mitigation, with effects now documented across the liver, heart, kidneys and vasculature,” he said.
“We are seeing a significant reduction in major adverse cardiovascular events … and progression of renal disease,” he went on.
The focus of GLP-1 drugs will widen beyond weight loss and diabetes, according to experts’ predictions. (iStock)
Philip Rabito, M.D., a specialist in endocrinology, weight loss and wellness in New York City, also shared that “exciting” advancements lie ahead for weight-loss drugs, including GLP-1s and GIPs.
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“These next‑generation agents, along with novel combinations that include glucagon and amylin agonists, are demonstrating even more impressive weight‑loss outcomes than currently available therapies, with the potential for better tolerability and sustained results,” he told Fox News Digital.
“There is also tremendous optimism around new federal agreements with manufacturers that aim to make these medications more widely accessible and affordable for the broad population of patients who need them most.”
No. 2: More convenient dosing
The typical prescription for a GLP-1 medication is a weekly injection, but delivery and dosing may be changing to more convenient methods in 2026, according to Balazs.
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A daily 25 mg pill version of Novo Nordisk’s Wegovy, a semaglutide designed to treat obesity, is now approved and available for chronic weight management, offering a non-injectable option for some patients.
A once-weekly oral GLP-1 is currently in phase 2 trials, as well as an implant that aims for three to six months of drug delivery, Balazs noted.
Incisionless weight-loss procedures will rise as a lower-risk option, according to experts. (iStock)
No. 3: Less invasive surgery
In addition to decreased risk during surgery for GLP-1 users, Balazs also predicted that metabolic surgery without incision will rise as a better option.
“Incisionless endoscopic procedures — like endoscopic sleeve gastroplasty (non-surgical weight-loss procedure that makes the stomach smaller from the inside) and duodenal mucosal resurfacing (non-surgical procedure that resets part of the small intestine to help the body better handle blood sugar) — [may become] more durable and widely available,” he said.
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“These offer significant metabolic benefits with shorter recovery and lower risk than traditional surgery.”
Rabito agreed that “rapid progress” in minimally invasive weight‑loss procedures is “opening powerful new options for patients who are hesitant to pursue traditional bariatric surgery.”
Bariatric surgery remains the most effective weight loss method, one specialist says. (iStock)
This avenue offers “meaningful and durable weight reduction with less risk, shorter recovery times and no external incisions,” the expert added.
Dr. Muhammad Ghanem, bariatric surgeon at the Orlando Health Weight Loss & Bariatric Surgery Institute, reiterated that surgery remains “the most successful modality for the treatment of obesity … with the highest weight loss and most durable outcomes as of yet.”
No. 4: Younger GLP-1 users
As Novo Nordisk’s Wegovy has been indicated for adolescents over 12 years old as an obesity treatment, Balazs commented that pediatric use of weight-loss drugs is “now a clinical reality.”
He predicted that other alternatives are likely to be approved in 2026 for younger users.
No. 5: High-tech, personalized access
Amid the growth of artificial intelligence, Balazs predicted an expansion in the clinical implementation of AI-driven weight-loss methods.
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This could include categorizing obesity into sub-types like “hungry brain,” “emotional hunger” and “slow burn” to personalize how therapy is prescribed while moving away from “trial and error,” he said.
Ghanem agreed that there will likely be a “big focus” on individualized testing for causes of obesity in 2026, as it’s a disease that can have “different causes in different people,” thus requiring different treatments.
AI and other digital opportunities will drive more access for weight-loss patients, experts say. (iStock)
The doctor anticipates that more patients will seek combinations of comprehensive treatments and programs.
“Patients are more aware that now we have a few weapons in our arsenal to combat obesity, and [they] are seeking a multidisciplinary and holistic approach,” Ghanem said.
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Treatment options will also turn digital with the rise of prescription digital therapeutics (PDTs) for weight loss, Balazs predicted.
“These are software applications delivering cognitive behavioral therapy, personalized nutrition and metabolic coaching through algorithms, often integrated with continuous glucose monitors, and reimbursed as medical treatments,” he said.
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Ghanem added that body composition analyzers, like DEXA scans, will likely be more widely used as awareness grows about the limitations of BMI and weight in assessing obesity.
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