Health
New drug for stiff person syndrome granted designation to speed up development: ‘Thrilled for the community’
Patients with stiff person syndrome are one step closer to having access to a new treatment.
Kyverna Therapeutics’ new drug, KYV-101, has been designated by the U.S. Food and Drug Administration (FDA) a Regenerative Medicine Advanced Therapy (RMAT), the company announced on Monday.
A drug is eligible for RMAT designation if it is “intended to treat, modify, reverse or cure a serious or life-threatening disease or condition” and if “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition,” according to the FDA’s website.
STIFF PERSON SYNDROME PATIENTS SHARE WHAT IT’S LIKE TO LIVE WITH THE RARE DISEASE
One of the benefits of RMAT is that Kyverna will be able to work closely with the FDA to help support accelerated development, review and approval, according to the company.
The FDA’s decision was based on positive results from clinical trials with patients, a company press release stated.
Carrie Robinette, left, was diagnosed with stiff person syndrome in 2023. The FDA named KYV-101 a Regenerative Medicine Advanced Therapy (RMAT) on Monday. (Carrie Robinette; iStock)
A rare neurological disorder, stiff person syndrome affects only one or two people for every million — including singer Celine Dion.
The disease can have a devastating impact, causing muscle rigidity, pain, spasms and loss of mobility.
LIKE CELINE DION, PENNSYLVANIA MAN IS FIGHTING STIFF PERSON SYNDROME WITH ‘EVERYTHING I HAVE’
Kyverna Therapeutics, based in Emeryville, California, developed the new CAR-T cell therapy, KYV-101, with the goal of “resetting” the immune systems of patients with autoimmune diseases, according to the company.
“As a physician dedicated to optimizing diagnosis and treatments for patients with autoimmune neurological disorders, I am grateful to be able to witness and contribute to the advancement of treatments and patient outcomes in stiff person syndrome via collaborative research efforts with leading players in the field and the support of the FDA oversight,” Amanda Piquet, M.D., director of the Autoimmune Neurology Program at CU Anschutz Medical Campus in Aurora, Colorado, told Fox News Digital via email.
Céline Dion shared her stiff person syndrome diagnosis with the world in Dec. 2022. (Edward Berthelot/GC Images/Getty Images)
Piquet was not involved in the development of KYV-101, but did take part in one of the medical advisory boards.
Peter Maag, PhD, CEO of Kyverna, also commented on the announcement.
“This RMAT designation means science agrees that fast-tracking this treatment could save lives.”
“Kyverna is focused on supporting patients and their clinical unmet needs, building patient-centric clinical trials to allow access to the latest medical advances in the CAR-T space for autoimmune disease,” Maag told Fox News Digital in a written statement.
“We are proud to be able to investigate the potential of safe and effective long-lasting treatment that can lead to meaningful, durable remission for patients suffering from autoimmune diseases like SPS.”
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Patients also reacted to the news with a sense of excitement.
“From the moment I learned what Kyverna’s treatment had done for the SPS patient in Germany, my dream was that it would get FDA-approved quickly so we could all benefit,” Carrie Robinette, 45, from San Diego, California, told Fox News Digital.
A drug is eligible for RMAT designation if it is “intended to treat, modify, reverse or cure a serious or life-threatening disease or condition” and if “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition,” according to the FDA’s website. (REUTERS/Andrew Kelly/File photo)
Robinette, a Navy wife and mother, was diagnosed with stiff person syndrome in spring 2023.
“This RMAT designation means science agrees that fast-tracking this treatment could save lives,” she added. “I’m thrilled for the entire autoimmune disease community.”
During clinical trials, 50 patients with oncological and autoimmune conditions have been treated with KYV-101 in more than 15 locations in Europe and the U.S., according to the company.
Kyverna will now continue collecting data from the drug’s Phase 2 trials for stiff person syndrome, multiple sclerosis and myasthenia gravis.
Carrie Robinette, 45, from San Diego, California, at left, and Corwyn Wilkey, 44, who lives in Anchorage, Alaska, right, are both living with stiff person syndrome. (Carrie Robinette; Corwyn Wilkey)
Phase 1 and 2 trials are also underway for systemic sclerosis and lupus.
“We are eager to begin generating data from our sponsored trial to advance the knowledge on a potential immunological reset of the patient’s immune system,” Maag said in the company’s release.
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Added Piquet, “Stiff-person syndrome has devastating and life-altering effects on patients suffering from this rare autoimmune disease … I look forward to the data that will emerge from the KYSA-8 trial, as this trial could drastically change the treatment landscape for SPS.”
Fox News Digital reached out to the FDA for additional comment.
Health
Origin of deadly cancer affecting young adults revealed in alarming report
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As colorectal cancer (CRC) is now the leading cause of cancer death in adults under 50, a new report reveals some surprising shifts in the incidence of the disease.
Although rates of CRC have been declining among seniors, those 65 and under are facing a rise in diagnoses, according to a report titled Colorectal Cancer Statistics, 2026, from the American Cancer Society.
Adults 65 and younger comprise nearly half (45%) of all new colorectal cancer cases — a significant increase from 27% in 1995, states the report, which was published in CA: A Cancer Journal for Clinicians.
The disease is rising fastest among adults 20 to 49 years old, at a rate of 3% per year.
Colorectal cancer is now the leading cause of cancer death in adults under 50. (iStock)
Among adults 50 and under, 75% of colorectal cancers are diagnosed at an advanced stage. Half of the diagnoses in that age range are made between the ages of 45 and 49. Although that age group is eligible to receive routine screenings, just 37% do so.
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The report also revealed that rectal cancer is on the rise, now accounting for about one-third (32%) of all CRC cases — an increase from 27% in the mid-2000s.
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“After decades of progress, the risk of dying from colorectal cancer is climbing in younger generations of men and women, confirming a real uptick in disease because of something we’re doing or some other exposure,” said Rebecca Siegel, senior scientific director, surveillance research at the American Cancer Society and lead author of the report, in a press release.
Among adults 50 and under, 75% of colorectal cancers are diagnosed at an advanced stage. Half of the diagnoses in that age range are made between the ages of 45 and 49. (iStock)
“We need to redouble research efforts to understand the cause, but also circumvent deaths through earlier detection by educating clinicians and the general public about symptoms and increasing screening in people 45-54 years.”
It is projected that 158,850 new cases of colorectal cancer will be diagnosed this year, and that the disease will cause 55,230 deaths, per the report.
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More than half of CRC cases can be linked to high-risk behaviors, the researchers said. Those include lack of nutrition, high alcohol consumption, smoking, lack of exercise and obesity.
“These findings further underscore that colorectal cancer is worsening among younger generations and highlight the immediate need for eligible adults to begin screening at the recommended age of 45,” said Dr. William Dahut, chief scientific officer at the American Cancer Society.
When the disease is caught at a local (early) stage, the five-year survival rate is 95%. (iStock)
“The report also shines a light on the crucial importance of continued funding for research to help discover new therapies to treat the disease and advance patient care.”
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When the disease is caught at a local (early) stage, the five-year survival rate is 95%, the report stated.
Health
Aging process could accelerate due to ‘forever chemicals’ exposure, study finds
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A new study suggests that middle-aged men may be more vulnerable to faster biological aging, potentially linked to exposure to “forever chemicals.”
The research, published in the journal Frontiers in Aging, examined how perfluoroalkyl and polyfluoroalkyl substances, more commonly known as PFAS, could impact aging at the cellular level.
PFAS are synthetic chemicals commonly used in nonstick cookware, food packaging, water-resistant fabrics and other consumer products, the study noted.
Their chemical structure makes them highly resistant to breaking down, allowing them to accumulate in water, soil and the human body.
Chinese researchers analyzed blood samples from 326 adults enrolled in the U.S. National Health and Nutrition Examination Survey between 1999 and 2000.
A new study suggests that middle-aged men could face accelerated biological aging at the cellular level due to exposure to PFAS. (iStock)
The researchers measured levels of 11 PFAS compounds in participants’ blood and used DNA-based “epigenetic clocks” — tools that analyze chemical changes to DNA to estimate biological age — to determine how quickly their bodies were aging at the cellular level, the study stated.
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Two compounds, perfluorononanoic acid (PFNA) and perfluorooctanesulfonamide (PFOSA), were detected in 95% of participants.
Higher concentrations of those chemicals were associated with faster biological aging in men of certain age groups, but not in women.
“People should not panic.”
The compounds most strongly linked to accelerated aging were not the PFAS chemicals that typically receive the most public attention, the researchers noted.
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“The associations were strongest in adults aged 50 to 64, particularly in men,” Dr. Xiangwei Li, professor at Shanghai Jiao Tong University School of Medicine and the study’s corresponding author, told Fox News Digital.
“While this does not establish that PFAS cause aging, it suggests that these widely present ‘forever chemicals’ may be linked to molecular changes related to long-term health and aging.”
The study found that two of the compounds were detected in 95% of participants, and higher levels were linked to faster biological aging in men ages 50–64. (iStock)
Midlife may represent a more sensitive biological period, when the body becomes more vulnerable to age-related stressors, according to the researchers.
Lifestyle factors, such as smoking, may influence biological aging markers, potentially increasing vulnerability to environmental pollutants.
While Li said “people should not panic,” she does recommend looking for reasonable ways to reduce exposure.
That might mean checking local drinking water reports, using certified water filters designed to reduce PFAS, and limiting the use of stain- or grease-resistant products when alternatives are available.
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Meaningful reductions in PFAS exposure will likely depend on broader regulatory action and environmental cleanup efforts, Li added.
The researchers noted that midlife could be a particularly sensitive stage, when the body is more susceptible to stressors associated with aging. (iStock)
Study limitations
The researchers outlined several important limitations of the research, including that the findings show an association, but do not prove that PFAS directly causes accelerated aging.
“The study is cross-sectional, meaning exposure and aging markers were measured at the same time, so we cannot determine causality,” Li told Fox News Digital.
The study was also relatively small, limited to 326 adults age 50 or older, which means the findings may not apply to younger people or broader populations.
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Researchers measured PFAS levels using data collected between 1999 and 2000, and today’s exposure patterns may differ.
Li added that while PFAS is known to persist in the environment and the body, these results should be validated through larger, more recent studies that follow participants over time.
Health
Melissa Joan Hart, 49, Opens up About Weight Loss in Perimenopause
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