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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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Scientists make startling discovery when examining prostate cancer tissue

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Scientists make startling discovery when examining prostate cancer tissue

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Small fragments of plastic were found in the tumors of most prostate cancer patients, according to a new study from NYU Langone Health. 

In past studies, microplastics have been found in almost every human organ and in bodily fluids, but their impact on human health still isn’t fully understood.

The researchers analyzed tissue samples from 10 patients with prostate cancer who underwent surgery to remove the entire organ. 

Using visuals of both benign samples and tumor samples, as well as specialized equipment, the scientists identified plastic particles in 90% of the tumor samples and 70% of benign tissue samples, according to the study press release.

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In past studies, microplastics were found in almost every single human organ along with bodily fluids, even the placenta. (iStock)

The cancerous tissue contained on average more than double the amount of plastic as healthy prostate tissue samples, the study found. This equates to about 40 micrograms of plastic per gram of tissue compared to 16 micrograms.

Researchers avoided contaminating the samples with other plastics by substituting standard tools with those made of aluminum, cotton and other non-plastic material, the release noted.

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The scientists say this is the first direct evidence linking microplastics to prostate cancer.

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“By uncovering yet another potential health concern posed by plastic, our findings highlight the need for stricter regulatory measures to limit the public’s exposure to these substances, which are everywhere in the environment,” said senior study author Vittorio Albergamo, assistant professor in the department of pediatrics at NYU Grossman School of Medicine, in the release.

Using visuals of both benign samples and tumor samples, as well as specialized equipment, the scientists identified plastic particles in 90% of the tumor samples and 70% of benign tissue samples. (iStock)

The study findings were presented during the American Society of Clinical Oncology’s Genitourinary Cancers Symposium in San Francisco on Feb. 26.

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“What is most striking is not that microplastics were detected, but that they were found embedded within tumor tissue itself,” Dr. David Sidransky, oncologist and medical advisor at SpotitEarly, a startup that offers an at-home breath-based test to detect early-stage cancer, told Fox News Digital.

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“While complete avoidance is unrealistic, people can take practical steps to reduce exposure.”

“We already know microplastics are present in water, air, blood and even placental tissue. Their detection in prostate tumors suggests systemic distribution and long-term bioaccumulation,” added Maryland-based Sidransky, who was not involved in the study.

Study limitations

Albergamo cautioned that a larger sample is needed to confirm the findings. Additionally, Sidransky noted that the presence of microplastics alone does not prove they cause cancer.

“Tumors can act as ‘biologic sinks,’ meaning they may accumulate circulating particles simply because of altered vasculature and permeability,” he said.

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A key unanswered question, according to the doctor, is whether microplastics are biologically active in ways that “promote DNA damage, immune modulation or chronic inflammation within the prostate.”

About one in eight men in the U.S. will be diagnosed with prostate cancer at some point in their lifetime, according to the Centers for Disease Control and Prevention.

The most actionable step men can take is appropriate screening and early detection, according to doctors. (iStock)

For those concerned about microplastics, Sidransky offered some insights.

“I believe the appropriate response is curiosity, not panic, and a commitment to understand more,” he said.

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“While complete avoidance is unrealistic, people can take practical steps to reduce exposure, such as minimizing heating food in plastic containers, reducing bottled water consumption when possible, and favoring glass or stainless steel alternatives.”

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The most actionable step men can take, however, is getting appropriate screenings to help ensure early detection, according to the doctor. Screening discussions should be individualized based on age, family history and other risk factors.

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How a Vegan Diet Can Help You Lose Weight 8X Faster

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How a Vegan Diet Can Help You Lose Weight 8X Faster


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Cancer-linked herbicide in the spotlight after controversial order: ‘Toxic by design’

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Cancer-linked herbicide in the spotlight after controversial order: ‘Toxic by design’

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There has been a shake-up in the Make America Healthy Again movement regarding glyphosate, a widely used herbicide that has been the subject of significant controversy.

The debate follows an executive order signed by President Donald Trump that ensures an adequate supply of elemental phosphorus and glyphosate-based herbicides related to national defense.

MAHA supporters have previously pushed a pesticide-free agenda, warning of potential health harms caused by glyphosate.

Dr. Marc Siegel, Fox News senior medical analyst, said he believes there is sufficient evidence linking glyphosate to neurodegenerative diseases, including ALS, Parkinson’s and multiple sclerosis, to warrant limiting exposure.

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President Donald Trump signed an executive order that ensures an adequate supply of elemental phosphorus and glyphosate-based herbicides related to national defense. (AP Photo/Charlie Neibergall)

“With Parkinson’s, this association appears to be due to the gut, vagus nerve and brain axis, where the exposure affects the microbiome in the gut, which then ascends slowly up to the brain, causing the neurodegenerative disease years later,” Siegel told Fox News Digital.

“There is also a growing association being found between high-dose glyphosate or occupational exposure and metabolic disorders, liver disease and some cancers, specifically lymphoma.”

He added, “Growing research backs this. I favor limiting it.”

“When we apply them across millions of acres and allow them into our food system, we put Americans at risk.”

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Studies have shown that glyphosate, which is used in products such as Roundup, owned by Monsanto, could raise cancer risk.

In one University of Washington study published in the journal Mutation Research, researchers found that exposure to it increased the risk of non-Hodgkin lymphoma by 41%.

The nonprofit Investigate Midwest, which analyzed data from both the U.S. Geological Survey and the National Cancer Institute, also recently found that pesticides may contribute to cancer rates.

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Among the top 500 counties for per-square-mile pesticide use, more than 60% had cancer rates above the national average of 460 cases per 100,000 people, according to the report.

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Investigate Midwest, which is based in Illinois, interviewed more than 100 farmers, environmentalists, lawmakers and scientists as part of a partnership with the Pulitzer Center’s StoryReach U.S. Fellowship.

Among the top 500 counties for per-square-mile pesticide use, more than 60% had cancer rates above the national average of 460 cases per 100,000 people, according to one study. (iStock)

Iowa, which used 53 million pounds of pesticides last year, holds the nation’s title for second-highest cancer rate.

Bill Billings, a resident of Red Oak, Iowa, was diagnosed with cancer in 2014. 

“The cancer specialist said, very directly, (my) cancer is a result of being exposed to chemicals,” Billings said in the report.

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Kelly Ryerson, founder of Glyphosate Facts and owner of the Instagram account @glyphosategirl, told Fox News Digital her journey researching the herbicide began with her own health struggles.

Ryerson, who is based in California, previously struggled with chronic illness and autoimmune issues, which she said improved when she stopped eating gluten. 

Iowa, which used 53 million pounds of pesticides last year, holds the nation’s title for second-highest cancer rate. (iStock)

After attending a medical conference at Columbia University’s Celiac Disease Center, Ryerson began to question modern farming practices rather than the gluten itself.

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“A lot of times, farmers are spraying Roundup on our grains right before harvest to facilitate an easier harvest,” she said. “After that easier harvest, because everything’s dry at the same time, those crops go directly to the mill and may end up in our food supply, at alarmingly high levels.”

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In 2015, the International Agency for Research on Cancer (IARC), part of the World Health Organization framework, classified glyphosate as “probably carcinogenic to humans.”

The classification was based on limited evidence of cancer in humans (notably non-Hodgkin lymphoma in some studies) and sufficient evidence in experimental animals.

“President Trump’s executive order reinforces the critical need for U.S. farmers to have access to essential, domestically produced crop protection tools, such as glyphosate,” a Monsanto spokesperson said. (Wolf von Dewitz/picture alliance via Getty Images)

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A spokesperson for Monsanto told Fox News Digital it will comply with Trump’s order to produce glyphosate and elemental phosphorus.

“President Trump’s executive order reinforces the critical need for U.S. farmers to have access to essential, domestically produced crop protection tools, such as glyphosate,” the spokesperson said.

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HHS Secretary Robert F. Kennedy Jr. has long been a vocal critic of Roundup, working with his legal team in 2018 to award $289 million to a man who alleged the weed killer caused his non-Hodgkin lymphoma, according to reports.

Following backlash to Trump’s executive order, Kennedy said he supports the order but acknowledged that “pesticides and herbicides are toxic by design, engineered to kill living organisms.”

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“When we apply them across millions of acres and allow them into our food system, we put Americans at risk,” he posted on X. “Chemical manufacturers have paid tens of billions of dollars to settle cancer claims linked to their products, and many agricultural communities report elevated cancer rates and chronic disease.”

Fox News Digital reached out to the White House for comment.

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