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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.

Celine Dion in a white turtleneck smiles on the carpet in France

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.”

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“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.

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FDA Building

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.

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Stiff person syndrome split

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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Video: About Our Investigation Into Cord Blood Banks

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Video: About Our Investigation Into Cord Blood Banks

Some families pay thousands of dollars to store their children’s stem cells with the hope of a healthier future for them. But Sarah Kliff, an investigative health care reporter for The New York Times, explains that the cells are rarely useful and are sometimes contaminated.

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Rare sleep disorder causes people to cook and eat food while they're asleep

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Rare sleep disorder causes people to cook and eat food while they're asleep

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A rare health condition could be causing some people to eat while they’re sound asleep. 

Sleep-related eating disorder (SRED) is an abnormal behavior called parasomnia that occurs during sleep.

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The condition causes a person to eat — and in some cases even prepare food — while asleep, according to experts.

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Kara Becker, a certified eating disorders specialist and national director of eating disorder programs for Newport Healthcare in California, told Fox News Digital that people who experience this sleepy snacking usually have no recollection of it the next morning.

A person can have more than one sleep-eating event per night — and it can happen even if the person isn’t hungry, Becker noted.

The person is “generally not aware” of the sleepwalking or “might only have a hazy awareness,” said a clinical psychologist. (iStock)

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Certain medications can cause SRED, as can smoking cessation, alcohol and drug use, stress, narcolepsy and dieting, according to the expert.

Drug-induced SRED can also occur after taking sedative-hypnotic drugs to treat insomnia, Cleveland Clinic noted on its website.

FEELING HUNGRIER THAN USUAL? YOUR SLEEP SCHEDULE COULD BE THE CULPRIT, AN EXPERT SAYS

Symptoms of the disorder can include repeated episodes of “out-of-control” eating and drinking when asleep, eating strange combinations of foods, or loss of appetite in the morning, Becker said.

The expert also flagged the dangers that can come with cooking or eating during the night, such as fires, burns and cuts.

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Woman tired fridge

Symptoms of the disorder can include repeated episodes of “out-of-control” eating and drinking when asleep, eating strange combinations of foods, or loss of appetite in the morning.  (iStock)

Fox News Digital also spoke to clinical psychologist Kelly Baron, PhD, director of the behavioral sleep medicine lab at the University of Utah, about symptoms and dangers of non-REM parasomnia.

SRED can cause dental issues and can interfere with weight-loss goals, Baron noted.

AMERICANS NEED MORE SLEEP, LESS STRESS, EXPERTS SAY, AS GALLUP POLL REVEALS TROUBLING FINDINGS

“In some cases, the person could cook and injure themselves or run into things,” she warned. 

“People can eat non-food items at times,” Baron said. “I wrote a case series with a colleague and one patient had eaten dog food in his sleep or a casserole with his hands, making a big mess.”

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man preparing lunch in a pan in the kitchen.

People with SRED could “fall or bump into things” or cut or burn themselves during an episode, an expert warned. (iStock)

Consuming large amounts of calories without mindfully eating is “not recommended for anyone and can be very disruptive to adequate nutrition consumption,” Becker added.

“Some may even consume hazardous substances at night without knowing, putting their health immediately at risk.”

So who is at risk?

SRED episodes are more likely to occur with lack of sleep, so conditions like snoring and obstructive sleep apnea can be risk factors, according to Cleveland Clinic’s website.

People are more likely to have SRED if they’re closely related to someone who sleepwalks, if they feel stressed out or sleep-deprived during the day, if they suffer from an eating disorder, or if they have anxiety or depression.

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“For other people, having another sleep disorder can trigger sleepwalking behavior, including night eating, so having a sleep evaluation is really important,” Baron advised.

A woman standing in front of the refrigerator

Those suffering from this condition should be evaluated at a sleep center, an expert said. (iStock)

For some people, sleepwalking is an inherited risk that continues from childhood into adulthood, according to Baron.

Treatment options

There are several treatment options for SRED, including reducing and eliminating triggers and addressing other sleep disorders, Baron told Fox News Digital.

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People can also implement techniques to improve sleep behaviors and try taking medication, the expert said.

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Becker also suggested implementing safety precautions at home, like installing locks on kitchen cabinets and refrigerators and removing dangerous obstacles.

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People with SRED should also get “plenty of regular sleep,” she encouraged, as the condition can heighten with sleep deprivation.

“It’s also recommended to participate in a sleep study so that your health care provider can better understand your nighttime activity,” she said.

Woman eating pizza slice in front of the refrigerator late night

SRED is most common in women under 20 years old, according to Cleveland Clinic. (iStock)

SRED is a condition separate from night eating syndrome (NES), which causes people to feel compelled to eat before bed or to wake up multiple times in the night to eat and then return to bed, Baron said.

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SRED is most common in women under 20 years old, according to Cleveland Clinic, but it can occur in anyone. 

Between 1% and 5% of adults experience parasomnia.

Experts suggest seeking a health care provider if SRED episodes are suspected.

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Chemotherapy drug could have a serious and surprising side effect, study finds

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Chemotherapy drug could have a serious and surprising side effect, study finds

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A chemotherapy drug could cause “significant” hearing loss among cancer survivors, according to a study from the University of South Florida and Indiana University.

Researchers tracked 100 testicular cancer survivors who received a chemo drug called cisplatin for an average of 14 years, as a press release from USF noted.

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Among the participants, who averaged 48 years of age, 78% of them reported experiencing “significant difficulties in everyday listening situations.”

HALF OF CANCER DEATHS COULD HAVE BEEN PREVENTED THROUGH LIFESTYLE CHANGES, SAYS AMERICAN CANCER SOCIETY REPORT

This was reportedly the first study to assess potential hearing loss among cancer survivors.

“Patients receiving cisplatin-based chemotherapy are at high risk for permanent hearing loss, and for some, that hearing loss will progress years after chemo treatment,” lead author Victoria Sanchez, associate professor in the USF Health Department of Otolaryngology Head & Neck Surgery, told Fox News Digital in an email. 

A chemotherapy drug could cause “significant” hearing loss among cancer survivors, according to a study. (iStock)

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“This hearing loss affects how people hear in everyday life, like a noisy restaurant or other social gatherings.”

Cisplatin is a type of chemotherapy drug that contains the metal platinum, according to the National Cancer Institute’s website.

ANCIENT EGYPTIANS ATTEMPTED TO SURGICALLY REMOVE BRAIN CANCER FROM SKULL 4,000 YEARS AGO, STUDY FINDS

The IV-administered drug is approved to treat bladder cancer, ovarian cancer and testicular cancer, the NCI states, either alone or in combination with other medications.

Higher doses of cisplatin were linked to more severe cases of hearing loss, the researchers found.

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woman receives chemo

Cisplatin is a type of chemotherapy drug that contains the metal platinum, according to the National Cancer Institute’s website. (iStock)

Those at highest risk included patients with poor heart health or high blood pressure.

“It was surprising to see that cardiovascular conditions are related to increased hearing loss and progression of hearing loss, which speaks to the need for patients to consider healthy lifestyle choices to help protect their ears,” Sanchez said.

MELANOMA PATIENTS REVEAL DRAMATIC STORIES FOR SKIN CANCER AWARENESS MONTH: ‘I THOUGHT I WAS CAREFUL’

The ears are especially vulnerable to the drug because they cannot filter it out, according to the USF researchers.

“This leads to inflammation and the destruction of sensory cells that are critical for coding sound, causing permanent hearing loss that can progressively get worse well after cisplatin treatments are completed,” the release stated.

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Researchers tracked 100 testicular cancer survivors who received a chemo drug called cisplatin for an average of 14 years. (iStock)

The U.S. Food and Drug Administration (FDA) approved cisplatin for testicular cancer treatment in 1978, according to the NIH.

Packaging labels for the drug warn of the potential for ototoxicity, which involves damage to the inner ear as a side effect of medication.

CHILDREN WITH TOTAL DEAFNESS REGAIN HEARING AFTER ‘GROUNDBREAKING’ GENE THERAPY: ‘LIKE A MIRACLE’

Hearing loss can be unilateral or bilateral and tends to become more frequent and severe with repeated doses,” the packaging stated.

“Decreased ability to hear normal conversational tones may occur.”

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It is unclear whether the drug-induced ototoxicity is reversible, according to the packaging.

Woman with hearing aid

“If a hearing loss is identified, treatment and help for hearing loss is available,” the lead study author said. “Health care providers can talk to patients about their risk of hearing loss and a plan for survivorship.” (iStock)

Based on the findings, the researcher recommends that cancer patients talk to their health care providers about possible hearing loss as a side effect of treatment and to receive hearing evaluations while receiving chemotherapy. 

“Check your hearing if you have any concerns,” Sanchez advised.  

“If hearing loss is identified, treatment and help for hearing loss is available. Health care providers can talk to patients about their risk of hearing loss and a plan for survivorship.”

Human ear anatomy

The ears are especially vulnerable to the drug because they cannot filter it out, according to the USF researchers. (iStock)

Dr. Marc Siegel, clinical professor of medicine at NYU Langone Medical Center and a Fox News medical contributor, noted that cisplatin is a “highly effective treatment” for testicular cancer, improving the cure rate from 10% to 90% when used in combination with other drugs. 

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“[The drug has a] very high degree of side effects, [including] hearing loss, which is very well-known, in up to 80% of patients,” Siegel, who was not involved in the study, told Fox News Digital via email. 

A cost-benefit analysis should be performed for all treatments, the doctor advised.

Audiologist

“High doses of chemotherapy (especially ones that are platinum-based) and radiation to the head, ear or brain can cause damage and hearing problems in one or both ears,” the ACS stated.  (iStock)

“Since this is a potentially deadly cancer, the benefits are generally worth the side effects, until newer, less toxic and equally effective treatments are developed,” Siegel said.

The main limitation of the study, according to Sanchez, is that all of the patients were very similar — males treated for testicular cancer.

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“We need to conduct additional studies to understand women treated with cisplatin and for other types of cancers,” she said.

The goal is that the research will lead to alternatives in chemotherapy treatment plans and preventative medications to reduce the risk of hearing loss, according to USF.

“Hearing loss related to cancer treatment is often permanent (doesn’t go away), but a hearing aid might help.”

The American Cancer Society warns on its website of the potential for chemotherapy drugs to impact hearing.

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“High doses of chemotherapy (especially ones that are platinum-based) and radiation to the head, ear or brain can cause damage and hearing problems in one or both ears,” the ACS stated. 

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“Hearing loss related to cancer treatment is often permanent (doesn’t go away), but a hearing aid might help.”

Fox News Digital reached out to the FDA, the ACS and several manufacturers of branded cisplatin medications requesting comment.

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