New Treatment for Rare Obesity Alleviated, Yet Further Studies Questionable due to Budget Reductions
In a stunning turn of events, a 10-year-old boy named Dean Shenk, who once survived only with feeding tubes due to Prader-Willi syndrome, found himself in the hospital, not for his life-threatening disorder, but due to an insatiable hunger that led him to devour an entire box of raisins. This ironic scenario highlights the cruel twist that this rare genetic disorder triggers – hyperphagia, aka uncontrollable hunger, sparking a desperate need for careful monitoring and food restriction.
Until March, the only options for Prader-Willi patients were growth hormone therapy and strict dietary control. But things changed with the FDA's approval of Vykat XR – an extended-release version of the existing drug diazoxide choline – which eases relentless hunger. This breakthrough comes at a time when other drugs, like GLP-1 agonists Ozempic, Wegovy, and others, are revolutionizing the treatment of obesity, which affects over 40% of American adults.
Researchers now understand that obesity is a complex mix of environmental, familial, and genetic factors, pointing towards multiple possible courses of treatment. Obesity medicine might eventually follow in the footsteps of treatments for high blood pressure or diabetes, offering various effective options tailored to different types of patients.
However, advancements in understanding the causes and pathways of obesity are now threatened by the Trump administration's dismantling of the nation's health research infrastructure. Rare disease researchers fear the ripple effects across all medical advancements, as federal funding for health research is being slashed, university labs face cuts, and the FDA experiences massive layoffs. Even with partnerships, such as the work that led to Vykat XR, progress depends heavily on NIH-funded labs and university researchers – let's hope they don't have to move their labs to Europe.
Vykat XR's possible side effects include high blood sugar, increased hair growth, and fluid retention, but for many patients, these trade-offs are worth the relief from their most devastating symptom. However, the drug's average price of $466,200 a year is astronomical, leaving some wondering who will foot the bill.
For Dean Shenk, the future looks brighter. During the trial for Vykat XR, his anxiety about food eased, allowing his parents to leave cupboards unlocked. With his hyperphagia under control, he gained lean muscle mass, lost body fat, and his bone mineral density increased – major improvements that have made a significant difference in his life and the lives of about 600 other Prader-Willi patients treated by pediatric endocrinologist Jennifer Miller at the University of Florida.
While Vykat XR may be limited in whom it can help with appetite control, researchers remain hopeful that the research behind it will further our understanding of the complexity of hunger and uncover other treatment options. Two Phase 3 clinical trials targeting different pathways are currently underway, and numerous general obesity trials are ongoing despite the uncertainties in U.S. medical research funding. So let's keep the hope alive for patients like Dean, who finally have a glimmer of a normal life. Will the future bring more affordable treatments, insights into the brain pathways behind appetite, and eventually, a cure for Prader-Willi syndrome? Here's to hoping!
KFF Health News is one of the core operating programs at KFF – an independent source for health policy research, polling, and journalism.
- Prader-Willi Syndrome
- Obesity Management
Enrichment Data:
Prader-Willi Syndrome (PWS): A rare genetic disorder, PWS is characterized by low muscle tone, developmental delays, cognitive disabilities, excessive hunger, and related complications such as rapid weight gain, behavioral issues, and sleep problems.
Treatments: Current treatments include growth hormone therapy, strict dietary control, and behavioral therapy; however, recent advancements have led to the FDA approval of Vykat XR, an extended-release drug that addresses excessive hunger in PWS patients.
Ongoing Research: Research is focusing on understanding the underlying neurological mechanisms of excessive hunger in PWS, as well as exploring epigenetic therapies, gene editing, and regenerative medicine as potential cures for the disorder. Several clinical trials for novel drugs targeting appetite are underway,Targeting different pathways such as Acadia Pharmaceuticals and Aardvark Therapeutics.
- The FDA's approval of Vykat XR, an extended-release drug, offers hope for Prader-Willi patients fighting excessive hunger, known as hyperphagia.
- Ongoing research focuses on understanding the neurological mechanisms of excessive hunger in Prader-Willi Syndrome (PWS), potentially leading to epigenetic therapies, gene editing, and regenerative medicine.
- Obesity medicine, similar to treatments for high blood pressure or diabetes, may eventually provide various effective options tailored to different types of patients, as researchers understand its complex mix of environmental, familial, and genetic factors.
- With Vykat XR, some patients experience side effects like high blood sugar, increased hair growth, and fluid retention, but for many, the relief from hyperphagia is worth the trade-offs.
- In the realm of health and wellness, nutrition plays a crucial role in managing conditions like Prader-Willi Syndrome, obesity, and associated chronic diseases such as type-2 diabetes and chronic kidney disease.
- KFF Health News is a core operating program at KFF, an independent source for health policy research, polling, and journalism, contributing significantly to the understanding of complex health conditions and treatments like Prader-Willi Syndrome and obesity management.
