A revolutionary medicine for extreme hunger offers indications of the complexity of obesity – KFF Health News

Ali Foley Shenk still remembers the panic when her 10-year-old son, Dean, ended up a 20 ounce box with raisins in the seconds that the cupboard was unlocked. They hurried to first aid and feared a dangerous intestinal impact action.

The irony stabbed: when Dean was born, he was so weak and floppy that he only survived with food tubes because he could not suck or swallow. He was diagnosed as a baby with the Prader-Willi syndrome-a rare condition fueled by a genetic defect. He remained free of food in food for years. But doctors warned that as Dean grew, his hunger would eventually become so uncontrollable that he could get dangerous amounts of weight and even eat until his stomach tore.

“It’s crazy,” said Foley Shenk, who lives in Richmond, Virginia. “They suddenly turn around.”

Prader-Willi syndrome affects a maximum of 20,000 people in the US The most striking symptom is the most life-threatening: an insatiable hunger known as hyperfagie that encourages caregivers to install cupboards and refrigerators, chain waste cans and install cameras. Until recently, the only treatment of growth hormone therapy to help patients stay slimmer and growing bigger, but it did not respond to the appetite.

In March, the Food and Drug Administration Approved Vykat XRA version with an extensive release of the existing Diazoxide choline, which enlightens the ruthless hunger and can offer insight into the biology of extreme appetite and binge eating. This breakthrough for these patients is because other medicines trigger a revolution in how doctors treat obesity that influences More than 40% From American adults. GLP-1 Agonistic Medicines Ozempic, Wegovy and others also deliver dramatic results for millions.

But what becomes clear is that obesity is not one disease – it’s a lot, said Jack YanovskiA senior obesity researcher at the National Institutes of Health, which was the co-author of some of the Vykat XR studies. Researchers learn that the directors of obesity can be environmental, family or genetic. “It is only logical that it is complex to treat,” said Yanovski.

Obesity medicine probably leads the path of high blood pressure or diabetes treatments, with three to five effective options for different types of patients. For example, up to 15% of patients in the GLP-1 investigations did not respond to those medicines, and at least One study found The medicines do not help significantly Prader-Willi patients.

Nevertheless, researchers, efforts say to understand how they can treat the many causes of obesity and the paths are now in doubt, because the Trump government dismantles the infrastructure of the nation for medical discovery.

While Secretary of Health and Human Services Robert F. Kennedy Jr. an agenda “Make America Healthy Remory” promotes, focused on nutrition and lifestyle, Federal Financing for Health Research is cut, including some subsidies that support the Obesity study. University laboratories Face cuts, FDA employees are massively fired and researchers of rare diseases fear the wrinkle effects in all medical progress. Even with biotech partnerships-as the work that led to Vykat XR Hangs the progress of laboratories and university researchers funded by NIH.

“That whole thing will probably be disturbed now,” said Theresa Strongresearch director of the Foundation for Prader-Willi Research.

HHS spokesperson Andrew Nixon said in a statement that no NIH prices for Prader-Willi Syndrome Research have been cut. “We continue to commit ourselves to supporting critical research into rare diseases and genetic disorders,” he said.

But Strong said that some of the contacts at the FDA she had spent almost 15 years with teaching the condition left the agency. She has heard that some research groups are considering moving their laboratories to Europe.

Early progress in hunger and obesity research changes the life of Dean Shenk. During the trial for Vykat XR, his fear of food disguised so much that his parents had cabinets unlocked.

Jennifer MillerA pediatric endocrinologist at the University of Florida who co-has the Vykat XR investigations treats around 600 Prader-Willi patients, including Dean. She said that the impact she has seen is life -changing. Since the drug study started in 2018, some of her adult patients have started to live independently, go to university and start jobs – milestones that were once impossible. “It opens their world in so many ways.”

For more than 26 years in practice, she has also seen how serious the disease patients hurts. One patient ate a bag of four pounds of dried out potato flakes; Another took all 10 frozen pizzas from a costco package taken; Some ate pets food. Others climbed out of windows, dived into waste containers, even died after being hit by a car while they walked away from home in search of food.

Low muscle tension, development delays, cognitive disabilities and behavioral challenges are also common characteristics of the condition.

Dean attends a special educational program, his mother said. He also has narcolepsy and cataplexia – a sudden loss of muscle control caused by strong emotions. His once regulated collapses and skin picking, which led to deep, infected lesions, were bound to fear over his obsessive, almost painful urge to eat.

In the process, however, his hyperfagia 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 strongest hunger signals in the body. “For most people, if you stop excreting NPY, hunger disappears,” said Anish Bhatnagar, CEO of Solo therapiesWhat makes the medication, the first medicine of the company. “That off-switch does not exist in Prader-Willi. It is literally that your brain tells you: ‘you hungry’ while you eat.”

GLP-1 drugs, on the other hand, simulating a intestinal hormone that helps people feel full by delaying digestion and signaling saturation to the brain.

The possible side effects of Vykat XR include high blood sugar levels, increased hair growth and liquid retention or swelling, but these are considerations that many patients are willing to get some relief from the most devastating symptom of the disorder.

Yet the average price of the drug of $ 466,200 a year is amazing, even for treatments with rare diseases. Soleno said in a statement that the broad cover is expected from both private and public insurers and that the copayments will be ‘minimal’. Until more insurers start repaying the costs, the company will provide the medicine to trial participants for free.

The shares of Soleno rose 40% after the FDA nod and have kept quite stable since then, with the company that is almost appreciated $ 4 billion From the beginning of June.

Although Vykat XR can be limited with who can help with appetite control, obesity researchers hope that the research behind it can help them decode the complexity of hunger and identify other treatment options.

“Insight into how more focused therapies work in rare genetic obesity helps us to better understand the brain paths behind the appetite,” said Jesse RichardsAn internal doctor and the director of obesity medicine at the University of Oklahoma-Tulsa’s School of Community Medicine.

That future may already take shape. For Prader-Willi, two other remarkable phase 3 clinical studies are underway, led by Acadia Pharmaceuticals and Aardvark therapeutics, each focused on different routes. In the meantime, hundreds of tests for general obesity are currently recruiting, despite the uncertainties in American medical research financing.

That brings more hope to patients like Dean. Almost six years after starting treatment, the now 16-year-old is a quieter, happier child, his mother said. He is more social, has friends and can concentrate better at school. With the impulse to eat too much that no longer dominates his thought, he has room for other interests – Star Wars, American Ninja Warrior and a healthy appreciation for avocados among them.

“Before the medicine it just felt like a dead end. My child was miserable,” said Foley Shenk. “Now we have our son back.”