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
Highly contagious stomach bug spreads fast, hitting certain patients hardest
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A highly contagious digestive virus is surging across the U.S., experts warn.
Rotavirus, a double-stranded RNA virus, causes acute gastroenteritis — inflammation of the stomach and intestines — which can lead to severe diarrhea, vomiting, fever and stomach pain.
The virus primarily affects infants and young children, but there have also been outbreaks in elderly populations, such as nursing homes.
AT LEAST 46 CHILDREN DEAD AMID MEASLES OUTBREAK AS VIRUS SPREADS GLOBALLY
Data from the Centers for Disease Control and Prevention shows that in the week ending April 4, out of 2,329 rotavirus tests, 7.3% were positive for the infection. Last year’s highest infection rate was 6.77% as of the week ending April 19.
Rotavirus, a double-stranded RNA virus, causes acute gastroenteritis — inflammation of the stomach and intestines — which can lead to severe diarrhea, vomiting, fever and stomach pain. (iStock)
“We’re seeing a lot of rotavirus in the wastewater right now,” Dr. Marc Siegel, Fox News senior medical analyst, confirmed to Fox News Digital. “Testing for rotavirus is way down, but the percentage of positive tests is up.”
While the virus typically peaks in the spring, it is not currently slowing down, he noted.
Why cases may be rising
Patricia Pinto-Garcia, M.D., a medical editor at GoodRx who is based in California, said there are several possible reasons for the rotavirus spike.
“Vaccine rates are down overall among young children, as they decreased during COVID,” she told Fox News Digital. “This means there’s a growing number of infants and young children who are vulnerable to infection.”
The rotavirus vaccine series must be completed by the time a child is 8 months old, she noted.
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As a result of the declining vaccinations, herd immunity isn’t protecting vulnerable children, according to Pinto-Garcia. “Children who haven’t finished the vaccine series yet, are too young to get vaccinated, or can’t get the vaccine due to medical illness are more likely to get exposed to the illness because other children aren’t vaccinated,” she said.
Siegel noted that before the vaccine became available, rotavirus resulted in 55,000 to 70,000 in the U.S. per year.
“Vaccine rates are down overall among young children, as they decreased during COVID,” a doctor told Fox News Digital. “This means there’s a growing number of infants and young children who are vulnerable to infection.” (iStock)
“I am concerned that the vaccination rate has been declining over the past seven years and is continuing to decline in the current climate of vaccine skepticism,” he said.
Surveillance methods are also much better than they used to be, Pinto-Garcia noted, which means public health experts are able to pick up and track cases better than ever before.
HOW LONG YOU’RE CONTAGIOUS WITH THE FLU — AND WHEN IT’S SAFE TO GO OUT
“But we see that rotavirus-related healthcare visits are also up, so improved detection is not the only reason we are seeing this spike,” she said.
The COVID pandemic also disrupted the pattern of infections, according to Pinto-Garcia, so it’s “tricky” to compare the current levels against older cycles.
“It’s possible that what we are seeing is still some post-pandemic rebound, but it’s unlikely that this year’s pattern is fully explained by just this factor,” she added.
Transmission and risk
Dr. Zachary Hoy, a pediatric infectious disease specialist at Pediatrix Medical Group based in Nashville, Tennessee, often sees young patients with rotavirus.
“Rotavirus is spread via the fecal-oral route, meaning that a person comes into contact with virus droplets from contact with other children or adults, or from contact with objects such as toys that have been contaminated with the virus from someone who is sick,” he told Fox News Digital. “This can lead to outbreaks, especially at schools where many young children share the same toys.”
“It’s possible that what we are seeing is still some post-pandemic rebound, but it’s unlikely that this year’s pattern is fully explained by just this factor.”
Rotavirus is associated with many dehydration cases in the hospital due to the degree of diarrhea, according to Hoy.
In some severe cases, the virus can lead to seizures due to electrolyte imbalances from dehydration and loss of electrolytes in the stool.
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“Younger children do not have the reserves that older children and adults have, so they can become more dehydrated quicker and develop more severe electrolyte imbalances, leading to more severe infections,” Hoy said.
“Patients with problems with their immune systems or on medications that can decrease their immune systems can have more severe and prolonged infections, too.”
Treatment and care
Because rotavirus is a viral infection, antibiotics are not effective against it. There is no specific antiviral treatment for the condition, with doctors typically recommending supportive care.
“The mainstay of treatment is hospitalization for rehydration via intravenous (IV) fluids,” Hoy told Fox News Digital. “Sometimes it can take up to two to three days of IV fluids to help get patients rehydrated.”
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Blood draws are often necessary to evaluate patients’ electrolyte levels, such as sodium, potassium, calcium and magnesium, according to the doctor.
“If these electrolyte levels are significantly low, sometimes patients need special IV solutions or individual electrolyte medications,” he added.
“The mainstay of treatment is hospitalization for rehydration via intravenous (IV) fluids,” a doctor told Fox News Digital. (iStock)
Dr. Daniel Park, medical director of the Pediatric Emergency Department at UNC Health in Chapel Hill, North Carolina, noted that most children recover with supportive care, but parents should seek medical attention if a child shows signs of dehydration. Those include decreased urination, lethargy or inability to keep fluids down.
“While rare, rotavirus can be life-threatening in vulnerable populations, especially very young infants or children with underlying medical conditions,” Park told Fox News Digital.
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Prevention strategies
Given the lack of antiviral medications for rotavirus, doctors emphasize the importance of prevention, primarily the vaccine.
There are two rotavirus vaccines – Rotateq (a three-dose series) and Rotarix (a two-dose series). They are given starting at age 2 months as oral drops, not injections, according to Hoy.
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“It’s important to get the rotavirus vaccines on schedule, because these younger infants are at greatest risk if they get rotavirus,” he advised.
Other recommended prevention methods include handwashing with soap and water.
Health
How Well Will You Age? Take Our Quiz to Find Out.
Every day we’re faced with a zillion small choices: Go to sleep early, or watch one more episode of that Netflix drama. Call an old friend to catch up, or cruise social media. Of course, no single action will guarantee a long, healthy life or doom you to an early grave. But those little daily decisions do add up, and over the long term they can make a difference when it comes to both your longevity and your health span, the amount of life spent in relatively good health.
Scroll through this theoretical “day in the life” and select the option that best fits your typical day. Not every situation will apply perfectly, but think about which choice you’d be most likely to make. This isn’t a formal scientific assessment. The goal here isn’t to assign you a “good” or “bad” score, but to help you understand the central factors that shape the way we age and how long we live.
Health
Red hair may be increasing as study points to surprising evolution trend
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A study from Harvard Medical School indicates natural selection has favored the red hair gene, resulting in a potential increase in the number of redheaded people as humanity continues to evolve.
By analyzing nearly 16,000 ancient genomes spanning 10,000 years, researchers identified a list of traits that nature is actively pushing forward. Among the most prominent were the genetic variants for red hair.
“Perhaps having red hair was beneficial 4,000 years ago, or perhaps it came along for the ride with a more important trait,” the authors noted.
22 HEALTH CARE PREDICTIONS FOR 2025 FROM MEDICAL RESEARCHERS
The study, published in the journal Nature, relied on a large database of ancient DNA from West Eurasia. Using new computing methods, the team was able to filter out random fluctuations in DNA to identify what it called “directional selection.”
Directional selection happens when a particular version of a gene gives an organism a strong survival or reproductive advantage, causing it to become more common in a population faster than it would by chance, according to experts.
Directional selection is when a specific gene provides such significant benefits that it rises in frequency across a population much faster than random chance. (iStock)
Prior to this study, scientists only knew of about 21 such instances in human history, one of which was lactose tolerance. This new research uncovered hundreds more.
“With these new techniques and a large amount of ancient genomic data, we can now watch how selection shaped biology in real time,” Ali Akbari, first author of the study and senior staff scientist in the lab of Harvard geneticist David Reich, said in a press release.
COMMON EATING HABIT MAY TRIGGER PREMATURE IMMUNE SYSTEM AGING, STUDY FINDS
The data showed that genetic markers for red hair are among 479 gene variants that have been strongly favored over the past 10,000 years. One likely explanation, the researchers said, is a major shift in human history: the transition to farming.
Scientists have long pointed to vitamin D synthesis as a likely driver for the rise of traits like fair skin and light hair. (iStock)
As humans moved away from hunting and gathering and settled into agricultural societies, their environment and behavior changed radically, triggering an evolutionary “acceleration.”
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While the Harvard study provides the first definitive statistical proof that red hair was actively selected during the rise of farming, the researchers noted that the exact prehistoric benefit still requires more study.
However, scientists have long pointed to vitamin D synthesis as a likely driver for the rise of these light-pigmented traits in northern climates.
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While redheads remain a minority of the global population today, the Harvard study’s analysis suggests that they may not be an evolutionary accident.
While redheads remain a minority of the global population today, the Harvard study’s analysis suggests they may not be an evolutionary accident. (iStock)
Instead, the red hair trait was “boosted” by natural selection as humans adapted to the challenges of a modern world, according to the researchers.
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The researchers urged caution in how these findings are interpreted.
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“What a variant is associated with now is not necessarily why an allele propagated,” the authors noted.
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