Royal Papworth Hospital NHS Foundation Trust has begun enrolling patients in a pioneering global research trial testing a new treatment approach for sarcoidosis, a challenging lung disease that causes long-term health problems for thousands of patients worldwide.
The trial, the first advanced phase global study for sarcoidosis, is evaluating whether the protein efzofitimod can help reduce steroid dependency in patients, potentially minimizing the significant side effects associated with long-term steroid use.
Understanding Sarcoidosis: A Complex Immune Disorder
Sarcoidosis affects approximately 7,000 people in the UK and is characterized by the formation of granulomas—small clumps of inflammatory cells—that typically develop in the lungs but can affect multiple organs including the eyes, skin, brain, and heart.
Despite being identified over 150 years ago, the exact cause of sarcoidosis remains unknown. Medical experts believe it stems from an immune system abnormality where the body essentially attacks itself.
"Even after 150 years, we still don't know what causes sarcoidosis," explained Dr. Muhunthan Thillai, Principal Investigator for the study and Consultant Physician at Royal Papworth Hospital. "It is an immune disease where the body fights itself."
Unlike many respiratory conditions that affect older populations, sarcoidosis typically strikes younger adults in their 30s and 40s, creating significant impacts on quality of life, employment, and long-term health outcomes.
Current Treatment Challenges
The standard treatment for sarcoidosis involves corticosteroids, which can effectively reduce inflammation but come with substantial side effects when used long-term. These include weight gain, increased risk of diabetes, and bone weakness.
"Sarcoidosis is very tricky to treat," said Dr. Thillai, whose center at Royal Papworth Hospital manages over 300 sarcoidosis patients, making it one of the largest specialized centers in the UK. "Steroids can be effective but also create other problems. This makes it a very complicated disease to manage as it can cause frailty and a big impact on people's quality of life, health risks, and ability to work."
The Efzofitimod Trial
The new clinical trial is testing efzofitimod, a protein that researchers hope will provide an alternative approach to managing sarcoidosis. Approximately 200 patients across Europe, North America, and Asia are participating in this large-scale study.
"The idea of trials such as this is to find alternative drugs to get people off the steroids," Dr. Thillai noted.
Patient Experience: Simon Osborne's Story
Simon Osborne, a 48-year-old former plasterer from Cambridge, is the first patient from Royal Papworth Hospital to join the trial. Diagnosed with pulmonary sarcoidosis 20 years ago, Osborne has experienced firsthand the life-altering effects of both the disease and its treatment.
Once physically active and employed in a demanding trade, Osborne first noticed symptoms when he became unusually breathless climbing stairs. His condition deteriorated rapidly, culminating in a collapse in 2004 that led to his diagnosis after a month-long hospital stay.
"I was taken to Addenbrooke's Hospital where I stayed for a month before being diagnosed with pulmonary sarcoidosis and transferred to Royal Papworth Hospital for my ongoing, long-term care," Osborne explained.
The disease progressively limited his ability to work. After leaving his plastering career, he attempted factory assembly work but found even that too demanding. By 2011, he had to stop working entirely.
"I loved playing sport and working but it got too much, I miss doing it but I can't do it, it is not nice," he said. "Now I get out of breath doing housework, I get very tired very quickly."
The Impact of Long-Term Steroid Treatment
Two decades of steroid treatment have taken a significant toll on Osborne's health beyond the effects of sarcoidosis itself.
"The side-effects of steroids for 20 years has seen me put weight on and my bones feel weak," he said. "When I got a phone call to invite me to this trial, I said yes straightaway."
Osborne's motivation for participating extends beyond potential personal benefit. "If there's a new drug that helps me feel better, provides me with a better quality of life and also could help others like me in the future, then I'm happy to help."
Significance of the Trial
This research represents a significant step forward in sarcoidosis treatment. As the first advanced phase global trial for the condition, it could potentially establish a new standard of care that reduces reliance on steroids while effectively managing the disease.
For the thousands of patients living with sarcoidosis worldwide, many of whom are in their prime working years, an effective steroid-sparing treatment could dramatically improve quality of life and reduce long-term health complications.
The trial's international scope—spanning three continents—underscores the global medical community's commitment to addressing this challenging condition and finding better solutions for patients like Simon Osborne.
