Latest news with #PraderWilliSyndrome
Medscape
3 days ago
- Health
- Medscape
A Drug for Hunger Offers Clues to Obesity's Complexity
Ali Foley Shenk still remembers the panic when her 10-year-old son, Dean, finished a 20-ounce box of raisins in the seconds the cupboard was left unlocked. They rushed to the emergency room, fearing a dangerous bowel impaction. The irony stung: When Dean was born, he was so weak and floppy he survived only with feeding tubes because he couldn't suck or swallow. He was diagnosed as a baby with Prader-Willi syndrome — a rare disorder sparked by a genetic abnormality. He continued to be disinterested in food for years. But doctors warned that as Dean grew, his hunger would eventually become so uncontrollable he could gain dangerous amounts of weight and even eat until his stomach ruptured. 'It's crazy,' said Foley Shenk, who lives in Richmond, Virginia. 'All of a sudden, they flip.' Prader-Willi syndrome affects up to 20,000 people in the US. The most striking symptom is its most life-threatening: An insatiable hunger known as hyperphagia that prompts caregivers to padlock cupboards and fridges, chain garbage cans, and install cameras. Until recently, the only treatment was growth hormone therapy to help patients stay leaner and grow taller, but it didn't address appetite. In March, the Food and Drug Administration approved Vykat XR, an extended-release version of the existing drug diazoxide choline, which eases the relentless hunger and may offer insights into the biology of extreme appetite and binge eating. This breakthrough for these patients comes as other drugs are revolutionizing how doctors treat obesity, which affects more than 40% of American adults. GLP-1 agonist medications Ozempic, Wegovy, and others also are delivering dramatic results for millions. But what's becoming clear is that obesity isn't one disease — it's many, said Jack Yanovski, a senior obesity researcher at the National Institutes of Health, who co-authored some of the Vykat XR studies. Researchers are learning that obesity's drivers can be environmental, familial, or genetic. 'It only makes sense that it's complex to treat,' Yanovski said. Obesity medicine is likely heading the way of treatments for high blood pressure or diabetes, with three to five effective options for different types of patients. For example, up to 15% of patients in the GLP-1 trials didn't respond to those drugs, and at least one study found the medications didn't significantly help Prader-Willi patients. Yet, researchers say, efforts to understand how to treat obesity's many causes and pathways are now in question as the Trump administration is dismantling the nation's infrastructure for medical discovery. While Health and Human Services Secretary Robert F. Kennedy, Jr, promotes a 'Make America Healthy Again' agenda centered on diet and lifestyle, federal funding for health research is being slashed, including some grants that support the study of obesity. University labs face cuts, FDA staffers are being laid off en masse, and rare disease researchers fear the ripple effects across all medical advances. Even with biotech partnerships — such as the work that led to Vykat XR — progress depends on NIH-funded labs and university researchers. 'That whole thing is likely to get disrupted now,' said Theresa Strong, research director for the Foundation for Prader-Willi Research. HHS spokesperson Andrew Nixon said in a statement that no NIH awards for Prader-Willi syndrome research have been cut. 'We remain committed to supporting critical research into rare diseases and genetic conditions,' he said. But Strong said that already some of the contacts at the FDA she'd spent nearly 15 years educating about the disorder have left the agency. She's heard that some research groups are considering moving their labs to Europe. Early progress in hunger and obesity research is transforming the life of Dean Shenk. During the trial for Vykat XR, his anxiety about food eased so much that his parents began leaving cupboards unlocked. Jennifer Miller, a pediatric endocrinologist at the University of Florida who co-led the Vykat XR trials, treats around 600 Prader-Willi patients, including Dean. She said the impact she's seen is life-changing. Since the drug trial started in 2018, some of her adult patients have begun living independently, getting into college, and starting jobs — milestones that once felt impossible. 'It opens up their world in so many ways.' Over 26 years in practice, she's also seen just how severely the disease hurts patients. One patient ate a 4-lb bag of dehydrated potato flakes; another ingested all 10 frozen pizzas from a Costco pack; some ate pet food. Others have climbed out of windows, dived into dumpsters, even died after being hit by a car while running away from home in search of food. Low muscle tone, developmental delays, cognitive disabilities, and behavioral challenges are also common features of the disorder. Dean attends a special education program, his mother said. He also has narcolepsy and cataplexy — a sudden loss of muscle control triggered by strong emotions. His once-regular meltdowns and skin-picking, which led to deep, infected lesions, were tied to anxiety over his obsessive, almost painful urge to eat. In the trial, though, his hyperphagia was under control, according to Miller and Dean's mother. His lean muscle mass quadrupled, his body fat went down, and his bone mineral density increased. Even the skin-picking stopped, Foley Shenk said. Vykat XR is not a cure for the disease. Instead, it calms overactive neurons in the hypothalamus that release neuropeptide Y — one of the body's strongest hunger signals. 'In most people, if you stop secreting NPY, hunger goes away,' said Anish Bhatnagar, CEO of Soleno Therapeutics, which makes the medication, the company's first drug. 'In Prader-Willi, that off switch doesn't exist. It's literally your brain telling you, 'You're starving,' as you eat.' GLP-1 drugs, by contrast, mimic a gut hormone that helps people feel full by slowing digestion and signaling satiety to the brain. Vykat XR's possible side effects include high blood sugar, increased hair growth, and fluid retention or swelling, but those are trade-offs that many patients are willing to make to get some relief from the most devastating symptom of the condition. Still, the drug's average price of $466,200 a year is staggering even for rare-disease treatments. Soleno said in a statement it expects broad coverage from both private and public insurers and that the copayments will be 'minimal.' Until more insurers start reimbursing the cost, the company is providing the drug free of charge to trial participants. Soleno's stock soared 40% after the FDA nod and has held fairly steady since, with the company valued at nearly $4 billion as of early June. While Vykat XR may be limited in whom it can help with appetite control, obesity researchers are hoping the research behind it may help them decode the complexity of hunger and identify other treatment options. 'Understanding how more targeted therapies work in rare genetic obesity helps us better understand the brain pathways behind appetite,' said Jesse Richards, an internal medicine physician and the director of obesity medicine at the University of Oklahoma-Tulsa's School of Community Medicine. That future may already be taking shape. For Prader-Willi, two other notable phase 3 clinical trials are underway, led by Acadia Pharmaceuticals and Aardvark Therapeutics, each targeting different pathways. Meanwhile, hundreds of trials for general obesity are currently recruiting despite the uncertainties in US medical research funding. That brings more hope to patients like Dean. Nearly 6 years after starting treatment, the now-16-year-old is a calmer, happier kid, his mom said. He's more social, has friends, and can focus better in school. With the impulse to overeat no longer dominating his every thought, he has space for other interests — Star Wars, American Ninja Warrior, and a healthy appreciation for avocados among them. 'Before the drug, it just felt like a dead end. My child was miserable,' Foley Shenk said. 'Now, we have our son back.'
CBS News
4 days ago
- Health
- CBS News
Breakthrough drug helps rare obesity condition, but other research uncertain amid cuts
Ali Foley Shenk still remembers the panic when her 10-year-old son, Dean, finished a 20-ounce box of raisins in the seconds the cupboard was left unlocked. They rushed to the emergency room, fearing a dangerous bowel impaction. The irony stung: When Dean was born, he was so weak and floppy he survived only with feeding tubes because he couldn't suck or swallow. He was diagnosed as a baby with Prader-Willi syndrome — a rare disorder sparked by a genetic abnormality. He continued to be disinterested in food for years. But doctors warned that as Dean grew, his hunger would eventually become so uncontrollable he could gain dangerous amounts of weight and even eat until his stomach ruptured. "It's crazy," said Foley Shenk, who lives in Richmond, Virginia. "All of a sudden, they flip." Prader-Willi syndrome affects up to 20,000 people in the U.S. The most striking symptom is its most life-threatening: an insatiable hunger known as hyperphagia that prompts caregivers to padlock cupboards and fridges, chain garbage cans, and install cameras. Until recently, the only treatment was growth hormone therapy to help patients stay leaner and grow taller, but it didn't address appetite. In March, the Food and Drug Administration approved Vykat XR, an extended-release version of the existing drug diazoxide choline, which eases the relentless hunger and may offer insights into the biology of extreme appetite and binge eating. This breakthrough for these patients comes as other drugs are revolutionizing how doctors treat obesity, which affects more than 40% of American adults. GLP-1 agonist medications Ozempic, Wegovy, and others also are delivering dramatic results for millions. But what's becoming clear is that obesity isn't one disease — it's many, said Jack Yanovski, a senior obesity researcher at the National Institutes of Health, who co-authored some of the Vykat XR studies. Researchers are learning that obesity's drivers can be environmental, familial, or genetic. "It only makes sense that it's complex to treat," Yanovski said. Obesity medicine is likely heading the way of treatments for high blood pressure or diabetes, with three to five effective options for different types of patients. For example, up to 15% of patients in the GLP-1 trials didn't respond to those drugs, and at least one study found the medications didn't significantly help Prader-Willi patients. Yet, researchers say, efforts to understand how to treat obesity's many causes and pathways are now in question as the Trump administration is dismantling the nation's infrastructure for medical discovery. While Health and Human Services Secretary Robert F. Kennedy Jr. promotes a "Make America Healthy Again" agenda centered on diet and lifestyle, federal funding for health research is being slashed, including some grants that support the study of obesity. University labs face cuts, FDA staffers are being laid off en masse, and rare disease researchers fear the ripple effects across all medical advances. Even with biotech partnerships — such as the work that led to Vykat XR — progress depends on NIH-funded labs and university researchers. "That whole thing is likely to get disrupted now," said Theresa Strong, research director for the Foundation for Prader-Willi Research. HHS spokesperson Andrew Nixon said in a statement that no NIH awards for Prader-Willi syndrome research have been cut. "We remain committed to supporting critical research into rare diseases and genetic conditions," he said. But Strong said that already some of the contacts at the FDA she'd spent nearly 15 years educating about the disorder have left the agency. She's heard that some research groups are considering moving their labs to Europe. Early progress in hunger and obesity research is transforming the life of Dean Shenk. During the trial for Vykat XR, his anxiety about food eased so much that his parents began leaving cupboards unlocked. Jennifer Miller, a pediatric endocrinologist at the University of Florida who co-led the Vykat XR trials, treats around 600 Prader-Willi patients, including Dean. She said the impact she's seen is life-changing. Since the drug trial started in 2018, some of her adult patients have begun living independently, getting into college, and starting jobs — milestones that once felt impossible. "It opens up their world in so many ways." Over 26 years in practice, she's also seen just how severely the disease hurts patients. One patient ate a four-pound bag of dehydrated potato flakes; another ingested all 10 frozen pizzas from a Costco pack; some ate pet food. Others have climbed out of windows, dived into dumpsters, even died after being hit by a car while running away from home in search of food. Low muscle tone, developmental delays, cognitive disabilities, and behavioral challenges are also common features of the disorder. Dean attends a special education program, his mother said. He also has narcolepsy and cataplexy — a sudden loss of muscle control triggered by strong emotions. His once-regular meltdowns and skin-picking, which led to deep, infected lesions, were tied to anxiety over his obsessive, almost painful urge to eat. In the trial, though, his hyperphagia was under control, according to Miller and Dean's mother. His lean muscle mass quadrupled, his body fat went down, and his bone mineral density increased. Even the skin-picking stopped, Foley Shenk said. Ali Foley Shenk (right) and her son Dean, at their home in Richmond, Virginia. Dean was born with Prader-Willi syndrome — a rare disorder that causes hyperphagia, a medical term for insatiable hunger. Since starting on a new medication as part of a clinical trial, his symptoms have improved, his mother says. Parker Michels-Boyce for KFF Health News Vykat XR is not a cure for the disease. Instead, it calms overactive neurons in the hypothalamus that release neuropeptide Y — one of the body's strongest hunger signals. "In most people, if you stop secreting NPY, hunger goes away," said Anish Bhatnagar, CEO of Soleno Therapeutics, which makes the medication, the company's first drug. "In Prader-Willi, that off switch doesn't exist. It's literally your brain telling you, 'You're starving,' as you eat." GLP-1 drugs, by contrast, mimic a gut hormone that helps people feel full by slowing digestion and signaling satiety to the brain. Vykat XR's possible side effects include high blood sugar, increased hair growth, and fluid retention or swelling, but those are trade-offs that many patients are willing to make to get some relief from the most devastating symptom of the condition. Still, the drug's average price of $466,200 a year is staggering even for rare-disease treatments. Soleno said in a statement it expects broad coverage from both private and public insurers and that the copayments will be "minimal." Until more insurers start reimbursing the cost, the company is providing the drug free of charge to trial participants. Soleno's stock soared 40% after the FDA nod and has held fairly steady since, with the company valued at nearly $4 billion as of early June. While Vykat XR may be limited in whom it can help with appetite control, obesity researchers are hoping the research behind it may help them decode the complexity of hunger and identify other treatment options. "Understanding how more targeted therapies work in rare genetic obesity helps us better understand the brain pathways behind appetite," said Jesse Richards, an internal medicine physician and the director of obesity medicine at the University of Oklahoma-Tulsa's School of Community Medicine. That future may already be taking shape. For Prader-Willi, two other notable phase 3 clinical trials are underway, led by Acadia Pharmaceuticals and Aardvark Therapeutics, each targeting different pathways. Meanwhile, hundreds of trials for general obesity are currently recruiting despite the uncertainties in U.S. medical research funding. That brings more hope to patients like Dean. Nearly six years after starting treatment, the now-16-year-old is a calmer, happier kid, his mom said. He's more social, has friends, and can focus better in school. With the impulse to overeat no longer dominating his every thought, he has space for other interests — Star Wars, American Ninja Warrior, and a healthy appreciation for avocados among them. "Before the drug, it just felt like a dead end. My child was miserable," Foley Shenk said. "Now, we have our son back." KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — the independent source for health policy research, polling, and journalism.
The Sun
05-06-2025
- Entertainment
- The Sun
Katie Price gives update on Harvey's weight loss jabs after revealing he weighs almost 30st
KATIE Price has revealed son Harvey will "hopefully" start weight loss injections this week in an attempt to save his life. The former glamour model is worried about the disabled 22-year-old's health after his weight reached 30st. 5 5 Katie, 47, gave her fans an update on Harvey's weight loss journey on her podcast, The Katie Price Show, which she hosts with her sister, Sophie. Revealing her plans for the weekend, she said Harvey will be joining her on Saturday when she performs at Portsmouth Pride. Katie then added: "Hopefully Harvey starts his Mounjaro this week, but we'll talk about that next week and I'll go through all of what's happening about that." The mum-of-five previously opened up on how Harvey's life was at risk because of his size. The TV personality's eldest child has Prader-Willi syndrome, which sparks a constant desire to eat food and a permanent feeling of hunger which leads to obesity. In a video posted in April, Katie revealed: 'I'm so heartbroken and gutted that his weight is just going up. 'I just googled it in stones, 188kg is just a few kg of being 30 stone. 'It's so life-threatening now, I'm still waiting for the doctors to get back to me starting on the mounjaro and his journey to a healthy life.' She continued: 'It's so sad his quality of life at the moment where he's so big, he just can't really do much. Katie Price drops huge hint daughter Princess Andre is in talks for Love Island after boyfriend split 'It's just another thing I have to deal with because he's at high risk of having a heart attack, he struggles to put his trainers or struggles to walk anywhere but I love him and I'm going to help him through this. 'So sad, obesity and his condition is sad, it's sad to see someone go through it and he doesn't understand.' 5 Everything you need to know about fat jabs Weight loss jabs are all the rage as studies and patient stories reveal they help people shed flab at almost unbelievable rates, as well as appearing to reduce the risk of serious diseases. Wegovy – a modified version of type 2 diabetes drug Ozempic – and Mounjaro are the leading weight loss injections used in the UK. Wegovy, real name semaglutide, has been used on the NHS for years while Mounjaro (tirzepatide) is a newer and more powerful addition to the market. Mounjaro accounts for most private prescriptions for weight loss and is set to join Wegovy as an NHS staple this year. How do they work? The jabs work by suppressing your appetite, making you eat less so your body burns fat for energy instead and you lose weight. They do this my mimicking a hormone called GLP-1, which signals to the brain when the stomach is full, so the drugs are officially called GLP-1 receptor agonists. They slow down digestion and increase insulin production, lowering blood sugar, which is why they were first developed to treat type 2 diabetes in which patients' sugar levels are too high. Can I get them? NHS prescriptions of weight loss drugs, mainly Wegovy and an older version called Saxenda (chemical name liraglutide), are controlled through specialist weight loss clinics. Typically a patient will have to have a body mass index (BMI) of 30 or higher, classifying them as medically obese, and also have a weight-related health condition such as high blood pressure. GPs generally do not prescribe the drugs for weight loss. Private prescribers offer the jabs, most commonly Mounjaro, to anyone who is obese (BMI of 30+) or overweight (BMI 25-30) with a weight-related health risk. Private pharmacies have been rapped for handing them out too easily and video calls or face-to-face appointments are now mandatory to check a patient is being truthful about their size and health. Are there any risks? Yes – side effects are common but most are relatively mild. Around half of people taking the drug experience gut issues, including sickness, bloating, acid reflux, constipation and diarrhoea. Dr Sarah Jarvis, GP and clinical consultant at said: 'One of the more uncommon side effects is severe acute pancreatitis, which is extremely painful and happens to one in 500 people.' Other uncommon side effects include altered taste, kidney problems, allergic reactions, gallbladder problems and hypoglycemia. Evidence has so far been inconclusive about whether the injections are damaging to patients' mental health. Figures obtained by The Sun show that, up to January 2025, 85 patient deaths in the UK were suspected to be linked to the medicines. In February Katie told The Sun she consulted top doctors who suggested starting Harvey on the jabs in a bid to improve his chances of living longer. Harvey's biological dad is former footballer Dwight, 53, dated for a short period between 2000 and 2001, but split shortly after Katie told him she was pregnant. 5 5
The Sun
31-05-2025
- General
- The Sun
UK landmarks glow orange to raise awareness of rare genetic condition
Some of the UK's most iconic landmarks were glowing orange last night - to raise awareness of a rare complex genetic condition that affects approximately 2,000 people in the UK. Lon don's Tower 42, Cardiff Castle, The Mersey Gateway Bridge and Green's Windmill in Nottingham were among the buildings to 'Glow Orange' to highlight Prader-Willi Syndrome (PWS). 4 4 4 Southend Civic Centre, Worcester's Guild Hall and Edinburgh's Camera Obscura and World of Illusions were also illuminated in an orange. The glow up lasted from sundown until sunrise, across 17 different landmarks, to mark the end of Prader-Willi Awareness month in May. Nigel Birrell, CEO of which was behind the activation and a proud charity partner with a scratchcard dedicated to the cause said: 'The Prader-Willi Syndrome Association (PWSA UK) is a cause that's very personal to me, and I've been a long-time admirer of the incredible work they carry out. "It's been truly inspiring to witness so many landmarks across the country light up in support of PWS awareness - the level of engagement has really touched us.' PWSA UK and FPWR UK (Foundation for Prader-Willi Research UK) have come together for a second year to plan and organise several events, which included one at the House of Lords on May 14th. Hosted in the capital city, it included speeches from both charities and the PWS community who shared their experience of living with the condition. The event also provided information to help people understand the complexities of PWS – the hope being that a better understanding will lead to more effective support which will save money and lives. The aim of the various events was to generate awareness and understanding of this rare condition which causes an overwhelming and uncontrollable drive to eat that can be life-limiting, as well as learning and physical difficulties. Together the charities are using the condition's initials to help people recognise key issues associated with the disorder. P - Persistent hunger which can lead to life-threatening obesity. W - Warning signs absent, typical health warning signs are absent for people with PWS. S – Specialist Care - people with the condition need specialist care, with round-the-clock supervision. Jackie Lodge of PWSA UK and Catherine Shaw of FPWR UK issued a joint statement. They said: 'We're delighted that PWSA UK and FPWR UK have come together again for PWS month to raise awareness for such an important cause. 'We want to generate as much awareness about PWS as possible and by these buildings and landmarks lighting up orange it is a fantastic way to do so, on top of the other events that have taken place this month. 'We're really grateful to these buildings for shining a light on this important cause.' 4
Yahoo
24-05-2025
- Business
- Yahoo
Soleno Therapeutics, Inc. (SLNO) Secures EMA Validation for PWS Treatment VYKAT XR
Soleno Therapeutics, Inc. (NASDAQ:SLNO) has achieved a major regulatory milestone as the European Medicines Agency (EMA) validated its Marketing Authorization Application for Diazoxide Choline Prolonged-Release Tablets (formerly DCCR) to treat hyperphagia in Prader-Willi syndrome (PWS) for patients aged four and older. This follows the recent U.S. FDA approval of the drug, now marketed as VYKAT XR, marking it as the first approved therapy specifically targeting hyperphagia, the most life-limiting symptom of PWS, in both adults and children. A researcher studying cellular therapeutics under a microscope. PWS is a rare genetic disorder characterized by insatiable hunger, leading to dangerous food-seeking behaviors and severe health complications. Soleno Therapeutics, Inc. (NASDAQ:SLNO) estimates that about 9,500 PWS patients in the UK, France, Germany, Italy, and Spain could benefit from this therapy if approved in Europe. The company has also secured Orphan Drug Designation in the EU, potentially granting up to 10 years of market exclusivity. Soleno Therapeutics, Inc. (NASDAQ:SLNO)'s CEO, Dr. Anish Bhatnagar, emphasized the importance of this milestone in expanding access to a much-needed treatment for the PWS community across Europe. The company is working closely with regulators to expedite the availability of the therapy, which has shown promise in addressing a critical unmet need for patients and families. SLNO surged by over 61% in 2025 so far. While we acknowledge the potential of SLNO to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SLNO and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Sign in to access your portfolio
