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New drug for stiff person syndrome granted designation to speed up development: ‘Thrilled for the community’

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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.

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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’

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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.

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“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.

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“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.”

                                            

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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.

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Goodbye, Late-Night Cravings! How To Curb Hunger and Make Weight Loss Easier

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Goodbye, Late-Night Cravings! How To Curb Hunger and Make Weight Loss Easier


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Lurking dementia risk exposed by breakthrough test 25 years before symptoms

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Lurking dementia risk exposed by breakthrough test 25 years before symptoms

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A new blood test could determine a woman’s dementia risk as early as 25 years before symptoms emerge.

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That’s according to new research from the University of California San Diego, which found that a specific biomarker protein associated with early pathological processes of Alzheimer’s disease was “strongly linked” to future dementia risk.

The researchers analyzed blood samples from 2,766 participants in the Women’s Health Initiative Memory Study in the late 1990s, according to the study’s press release. 

KEY FITNESS MEASURE IS STRONG PREDICTOR OF LONGEVITY AFTER CERTAIN AGE, STUDY FINDS

The women ranged from 65 to 79 years of age and showed no signs of cognitive decline at the start of the study.

After tracking the participants for up to 25 years, the researchers concluded that the biomarker phosphorylated tau 217 (p-tau217) was “strongly associated” with future mild cognitive impairment and dementia. 

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A new blood test could determine a woman’s dementia risk as early as 25 years before symptoms emerge. (iStock)

Women who had higher levels of p-tau217 at the beginning of the study were “much more likely” to develop the disease. The findings were published today in JAMA Network Open.

“The key takeaway is that our study suggests it may be possible to detect risk of dementia two decades in advance using a simple blood test in older women,” first author Aladdin H. Shadyab, a UC San Diego associate professor of public health and medicine, told Fox News Digital. 

“These biomarkers may help us identify who is at greatest risk and develop strategies to delay or prevent dementia.”

“Our findings show that the blood biomarker p-tau217 could help identify individuals at higher risk for dementia long before symptoms begin,” he added.

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This long lead time could open the door to earlier prevention strategies and more targeted monitoring, rather than waiting until memory problems are already affecting daily life, according to Shadyab.

A specific biomarker protein associated with early pathological processes of Alzheimer’s disease was “strongly linked” to future dementia risk. (iStock)

“As the research advances, these biomarkers may help us identify who is at greatest risk and develop strategies to delay or prevent dementia,” he said.

This risk relationship wasn’t the same across the board, however. Women over 70 with higher p-tau217 levels had “poorer cognitive outcomes” compared to those under 70, as did those with the APOE ε4 gene, which is a known risk factor for Alzheimer’s disease.

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The study also found that p-tau217 was a stronger predictor of dementia in women who were randomly assigned to receive estrogen and progestin hormone therapy compared to those who received a placebo.

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“Blood-based biomarkers like p-tau217 are especially promising because they are far less invasive and potentially more accessible than brain imaging or spinal fluid tests,” said senior author Linda K. McEvoy, senior investigator at Kaiser Permanente Washington Health Research Institute and professor emeritus at the Herbert Wertheim School of Public Health, in the release. 

“Blood-based biomarkers like p-tau217 are especially promising because they are far less invasive and potentially more accessible than brain imaging or spinal fluid tests,” a researcher said. (iStock)

“This is important for accelerating research into the factors that affect the risk of dementia and for evaluating strategies that may reduce risk.”

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Blood tests for Alzheimer’s disease are still being studied and are not recommended for routine screening in people without symptoms, Shadyab noted. 

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More research is needed before this approach can be considered for clinical use prior to cognitive symptoms. 

Future studies should investigate how other factors — like genetics, hormone therapy and age-related medical conditions — might interact with plasma p-tau217, the researchers added.

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“The study examined only older women, so the findings may not necessarily apply to men or younger populations,” Shadyab noted. “We also examined overall dementia outcomes rather than specific subtypes such as Alzheimer’s disease.”

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Key fitness measure is strong predictor of longevity after certain age, study finds

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Key fitness measure is strong predictor of longevity after certain age, study finds

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For women over 60, muscle strength plays a critical role in longevity, a new study confirms.

Researchers at the University at Buffalo, New York, followed more than 5,000 women between the ages of 63 and 99, finding that those with greater muscle strength had a significantly lower risk of death over an eight-year period.

The findings were published in JAMA Network Open.

EXERCISE AFFECTS THE HEART IN A HIDDEN, POWERFUL WAY BY REWIRING NERVES, STUDY FINDS

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Muscle function was measured using grip strength and how quickly participants could complete five unassisted sit-to-stand chair raises. 

These are two tests commonly used in clinical settings to evaluate muscle function in older adults, the researchers noted.

A recent study shows that stronger muscle strength in women over 60 is linked to a lower risk of death over eight years. (iStock)

“In a community cohort of ambulatory older women, muscular strength was associated with significantly lower mortality rates, even when we accounted for usual physical activity and sedentary time measured using a wearable monitor, gait speed and blood C-reactive protein levels,” study lead author Michael LaMonte, research professor of epidemiology and environmental health at the University at Buffalo, told Fox News Digital.

“Movement is the key — just move more and sit less.”

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Many earlier studies did not include those objective measurements, making it difficult to determine whether muscle strength itself was linked to longevity, according to LaMonte. “Our study was able to better isolate the association between strength and death in later life,” he added.

Even for women who don’t get the recommended amount of aerobic physical activity, which is at least 150 minutes per week, muscle strength remained important for longevity, the researchers found.

Women with greater muscle strength were more likely to live longer, even if they did not meet the recommended amount of aerobic exercise. (iStock)

“The findings of lower mortality in those who had higher strength but were not meeting current national guidelines on aerobic activity were somewhat intriguing,” LaMonte said.

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Federal guidelines recommend strengthening activities one to two days per week, targeting major muscle groups.

Resistance training does not have to require a gym membership, LaMonte noted. These exercises can be performed using free weights, resistance bands, bodyweight movements or even household items, such as soup cans.

Experts recommend working major muscle groups one or two days a week using weights, bands or bodyweight exercises. (iStock)

“Movement is the key — just move more and sit less,” he said. “When we can no longer get out of the chair and move around, we are in trouble.”

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LaMonte acknowledged several limitations of the study. The researchers assessed muscle strength in older age but did not explore how earlier levels in adulthood might influence long-term health outcomes.

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“We were not able to understand how strength and mortality relate in younger ages,” he said, noting that future research should explore whether building strength earlier could have an even greater impact on longevity.

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