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Children with inherited deafness hear for first time through experimental gene therapy

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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)

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“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.

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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.

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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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Doctors thought man had brain cancer — they found live tapeworms instead

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Doctors thought man had brain cancer — they found live tapeworms instead

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Doctors thought a 60-year-old man had metastatic brain cancer after scans revealed multiple tumors – but further testing revealed a shocking diagnosis.

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The Spanish man, who was not named, was found to have a parasitic tapeworm larvae lodged in his brain — a case of neurocysticercosis, a parasitic infection of the central nervous system caused by the pork tapeworm (Taenia solium).

The patient, a lifelong resident of Castellón, Spain, had not traveled to any regions where the disease is endemic, according to the case report published in the CDC journal Emerging Infectious Diseases.

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The man had initially come to the hospital after suffering two weeks of progressive headaches and mild behavioral changes.

Radiologic findings from a study of autochthonous neurocysticercosis brain lesions mimicking metastatic disease. (Emerging Infectious Diseases)

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CT scans showed multiple abnormal spots that looked like tumors that had spread from cancer elsewhere in the body, leading doctors to suspect advanced brain cancer.

However, whole-body scans, a colonoscopy and specialized imaging failed to identify cancer anywhere in the patient’s body, the case report stated.

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When doctors performed a more detailed MRI, they discovered several fluid-filled cysts in the brain, some of which contained the head of a tapeworm. A blood test confirmed the diagnosis of neurocysticercosis.

The man was treated with a combination of albendazole and praziquantel (two antiparasitic medications), as well as corticosteroids to reduce inflammation. He successfully recovered with no complications, according to the case report.

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CT scans (not pictured) showed multiple abnormal spots that looked like tumors that had spread from cancer elsewhere in the body, leading doctors to suspect advanced brain cancer. (iStock)

The authors suggested that the patient may have gotten the infection after accidentally ingesting microscopic tapeworm eggs years earlier.

The exposure may have occurred while working a construction job with migrant coworkers from regions where neurocysticercosis is endemic.

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People can catch the pork tapeworm in two different ways. Eating undercooked infected pork usually leads to an intestinal tapeworm, but accidentally swallowing the parasite’s eggs — typically through food or water contaminated with feces — can send the larvae into the bloodstream, where they may form cysts in the brain and other organs. This can cause the disease the patient in the case report acquired.

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A photomicrograph shows histopathologic features in a brain tissue specimen from a case of neurocysticercosis caused by the pork tapeworm Taenia solium. The image documents pathological changes associated with this parasitic infection. (Smith Collection/Gado/Getty Images)

As this was just a single case, the source of transmission could not be proven, the researchers acknowledged, and the findings cannot be generalized to a wider population. The report shows that there is a possibility of local transmission in non-endemic settings, but cannot establish how often this occurs.

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In the United States, less than 2% of neurocysticercosis cases are considered domestically acquired, according to the case report.

A prior systematic review identified only 18 confirmed locally acquired cases in Western Europe between 1990 and 2011.

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The authors suggested that the patient may have gotten the infection after accidentally ingesting microscopic tapeworm eggs years earlier. (Associated Press)

In some cases, NCC can cause serious symptoms, including seizures, stroke, neurological deficits and cognitive decline.

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“Our case emphasizes that the absence of travel history should not preclude NCC from the differential diagnosis of multiple ring-enhancing brain lesions, even in regions where metastatic cancer is statistically much more likely,” the researchers concluded in the case study.

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Detecting the worms earlier could have prevented “unnecessary invasive oncologic procedures and led to prompt, targeted antiparasitic therapy,” they added.

Fox News Digital reached out to the researchers for comment.

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Rare tick-borne virus turns deadly fast as US cases reach record high, experts warn

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Rare tick-borne virus turns deadly fast as US cases reach record high, experts warn

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A rare and potentially fatal tick-borne illness currently spreading across the United States can be traced back to a 1958 case involving a young boy on a farm.

The disease, known as Powassan virus, was named for the Ontario town near where it was first discovered.

At the time of his death, Lincoln Byers, a 4-year-old living in Canada, had a condition that medical professionals could not explain, the Boston Globe reported.

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Years later, researchers discovered a tick harboring the same virus on a dead squirrel, finally providing an answer to the tragedy, but foreshadowing a growing public health challenge.

While once considered an obscure medical anomaly, Powassan virus cases have reached historic highs in the U.S., data show.

Powassan is most prevalent from late spring through mid-fall, when tick populations peak and outdoor activity increases. (iStock)

According to the Centers for Disease Control and Prevention, 76 Americans were diagnosed with the virus in 2025, the highest annual total on record. Previously, the U.S. averaged just seven to eight diagnoses each year.

The virus is primarily transmitted to humans through the bite of an infected woodchuck tick or deer tick. Like other tick-borne illnesses, Powassan is most prevalent from late spring through mid-fall, when tick populations peak and outdoor activity increases.

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Public health experts warn that the virus’s transmission speed makes it uniquely dangerous.

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“One of the most dangerous aspects is its rapid transmission,” Dr. Jorge P. Parada, a medical advisor at the National Pest Management Association in Chicago, told Fox News Digital.

“Powassan can be transmitted in as little as 15 minutes after the infected tick bites, while Lyme disease usually requires a 36- to 48-hour attachment time for transmission.”

Powassan carries an incubation period of one to four weeks before symptoms manifest. (iStock)

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Parada noted that while Powassan remains rare compared to Lyme disease, it poses clinical concern.

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Dr. Marc Siegel, senior medical analyst for Fox News, confirmed that the virus transmits significantly faster than Lyme disease. He noted that Powassan carries an incubation period of one to four weeks before symptoms manifest.

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Initial symptoms include fever, headache, vomiting and weakness, though some infected individuals remain asymptomatic, according to the CDC.

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There are currently no specific medications or vaccines to treat or prevent Powassan virus. (iStock)

The virus can progress to severe neurological complications, including encephalitis (inflammation of the brain) and meningitis (inflammation of the spinal cord membranes).

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In severe cases, patients may experience confusion, loss of coordination, difficulty speaking and seizures, per the CDC.

“One of the most dangerous aspects of it is its rapid transmission.”

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Approximately 10% of Powassan cases involving severe neurological disease are fatal, and many survivors experience long-term neurological issues.

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There are currently no specific medications or vaccines to treat or prevent Powassan virus; clinical care is limited to supportive therapy, such as intravenous fluids and respiratory support.

While anyone can develop severe illness, those at the highest risk include children, older adults and immunocompromised individuals, experts caution.

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America’s lifespan has doubled since 1776 — experts reveal what changed

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America’s lifespan has doubled since 1776 — experts reveal what changed

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Americans today live roughly twice as long, on average, as they did when the Declaration of Independence was signed.

When the nation was founded in 1776, life expectancy was around 35 to 40 years old, historians estimate. However, someone who survived childhood in colonial America often lived into their 60s or even 70s.

Today, the average lifespan is about 79 years old, according to data from the Centers for Disease Control and Prevention.

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The improvement in lifespan over the centuries has been largely attributed to reduced deaths in infancy and from infectious diseases, multiple researchers have stated. Advances in sanitation, clean water, nutrition, vaccination and medical care have also contributed to lower mortality rates.

“Much of this vast discrepancy is related to the extremely high rates of infant, childhood and maternal mortality,” Dr. Omer Awan, physician and professor at the University of Maryland School of Medicine, told Fox News Digital.

Americans today live roughly twice as long, on average, as they did when the Declaration of Independence was signed. (Milan Markovic/iStock)

“Childbirth was dangerous, and without antibiotics and vaccines, many infectious diseases, such as measles, smallpox and pneumonia, were deadly,” he went on. “Now we have cleaner water and sanitation, vaccines and antibiotics that have significantly prolonged life.”

Advances in treatments of chronic diseases such as high blood pressure, cancer and diabetes have also significantly prolonged life, the Harvard-trained doctor noted.

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According to the CDC, improved prevention and treatment of high blood pressure has helped reduce deaths from heart disease and stroke, two of the nation’s leading causes of death.

Mia Kazanjian, MD, a Stanford-trained body and breast radiologist with an interest in longevity who is based in Greenwich, Connecticut, attributes the shorter life expectancy in the 1700s to suboptimal sanitation, poor hygiene and limited medical treatments.

Today, the average lifespan is about 79 years old, according to data from the Centers for Disease Control and Prevention. (iStock)

“Many babies and children died from infections like dysentery, diphtheria, scarlet fever and pneumonia,” she told Fox News Digital. Children who survived into adulthood often succumbed to infections like tuberculosis, cholera and typhoid fever.

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Maternal mortality has also fallen dramatically over the past century due to advances in antibiotics, blood transfusions and safer obstetric care, according to the CDC.

Milestones in public health

Kazanjian pointed to several key advancements over the centuries that contributed to longevity improvements, including the development of early municipal water systems that provided cleaner drinking sources.

“Sewer system networks were built, the first in Brooklyn in 1857,” she said. “These allowed people to drink clean water and dispose of waste. Indoor plumbing with toilets and bathrooms became more widespread.”

“Without antibiotics and vaccines, many infectious diseases, such as measles, smallpox and pneumonia, were deadly.”

At this time, people’s understanding of disease started to improve, and public health measures were developed to minimize risk.

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During the late 1800s, germ theory became widely accepted in medicine and public health, helping shape the Sanitary Era, the expert said.

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“The Federal Quarantine Act of 1878 allowed the government to prevent spread of infection from out of the country, from epidemics like yellow fever,” she said. “Food safety regulations went into effect in 1906, when the Pure Food and Drug Act and Federal Meat Inspection Act were passed.”

By 1900, the average life expectancy was about 49 years old, according to the National Vitals Statistics Report.

Another major landmark in increasing lifespan came with the development of vaccines and antibiotics to prevent and treat disease, Kazanjian noted.

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One major landmark in increasing lifespan came with the development of vaccines and antibiotics to prevent and treat disease, experts say. (iStock)

“Jenner developed the smallpox vaccine in 1796, Pasteur created vaccines for rabies and anthrax in the 1880s, and several scientists created vaccines for polio, measles, influenza, mumps and rubella in the mid 1900s,” she said.

“Antibiotics proliferated in the 1940s, specifically penicillin and tetracycline. By 1950, the US life expectancy was about 68 years old.”

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From the mid-20th century to 2014, life expectancy continued to rise, Kazanjian said, largely due to “major gains” in medical knowledge of ways to prevent heart disease and stroke.

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Public health campaigns promoting smoking cessation also played a role, as declining smoking rates helped reduce deaths from lung cancer, heart disease and stroke, according to the CDC.

“Motor vehicles became safer and carseats became staples,” Kazanjian noted.

Modern longevity is more focused on preventing chronic disease and less about surviving childhood infections. (iStock)

According to the National Institutes of Health, advances in emergency medical services and trauma care have substantially reduced deaths after serious injuries.

Development of pharmaceuticals for cardiovascular disease and cancer also contributed to longer lives, according to Kazanjian.

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Today’s longevity challenges

Modern longevity is more focused on preventing chronic disease and less about surviving childhood infections, noted Nneoma Oparaji, MD, a triple board-certified media physician specializing in obesity, lifestyle and internal medicine.

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“The next frontier will be less about living longer, but more about living healthier longer,” Houston-based Oparaji told Fox News Digital.

Kazanjian pointed out that between 2014 and 2026, there has been a fall and a rise in lifespan.

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“The fall was due to young adult deaths from drug overdoses, particularly the opioid epidemic, suicides and alcohol-related deaths,” she told Fox News Digital.

The COVID-19 pandemic reduced U.S. life expectancy by more than two years between 2019 and 2021 before it began recovering, CDC data shows.

Advances in sanitation, clean water, nutrition, vaccination and medical care have contributed to lower mortality rates. (iStock)

Although U.S. life expectancy has rebounded since the pandemic, it remains below that of other high-income countries, largely because of higher death rates from chronic diseases, substance use and other preventable causes, according to KFF.

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Obesity rates also continue to climb, contributing to higher numbers of cardiovascular disease, diabetes and cancer, Kazanjian said.

“Most concerning is the rise in obesity in children,” she added.

“The next frontier will be less about living longer, but more about living healthier longer.”

Changing cancer trends are also affecting lifespan among younger adults, data shows.

“My generation, the millennials, has seen an unprecedented rise in young adult cancers, particularly colon and breast,” Kazanjian said, citing factors that include sedentary lifestyles, poor diet, alcohol, obesity and smoking, among others.

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The doctor said she aims to raise public health awareness of ways to improve lifespan.

“We need to get off our screens, move around more, eat a whole food, plant-based diet, sleep seven hours a night, do our screening exams, and avoid toxins like alcohol and cigarettes.”

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