The Royal Free London has recruited the first UK patient to a global research trial of a new drug for a rare autoimmune condition.
Scleroderma is a rare disease caused by the immune system attacking the connective tissue of the skin, internal organs and blood vessels, leading to scarring and thickening of the tissue in these areas.
Those with the disease often experience a range of uncomfortable symptoms including skin thickening, joint pain, finger ulcers, stiffness and difficulty swallowing linked to weight loss. Some patients find it difficult to move their hands and arms. Currently there is no cure for sufferers and those living with the condition are offered treatments that target the various symptoms of the disease.
Now a drug trial is offering patients new hope. The drug blocks particular proteins in the body, so they aren’t able to send a signal to the immune cells that cause scleroderma. Without this signal, these immune cells aren’t able to attack the connective tissues in the body.
In this trial the new drug is administered to half of the participants over a course of 24 weeks. Thirteen countries are involved with this study and eight countries are currently recruiting to the trial. The Royal Free is the only participating hospital in the UK.
Fred Whitby, 57, from Feltham in west London, has become the first patient in the UK to take part in the trial.
Fred said: “At the start of 2016 I began experiencing sensitivity in my fingers and my fingertips became discoloured. I visited my GP who initially suspected a condition called Raynaud’s. I was referred to a specialist at the Royal Free and it was later confirmed I had scleroderma. I have been given treatments to manage some of the symptoms I’m experiencing but at the moment there is no cure.
“When the team at the Royal Free offered me the opportunity to take part in a new clinical trial that aims to find a cure, I signed up. I’m given an injection every week by a nurse, either at the hospital or in my home. The treatment lasts for six months. Although it is a big time commitment, I know that without research a cure will never be found. Even if participating in this trial doesn’t benefit me in the short term, my hope is that it will benefit me and others with my condition in the future.”
At the Royal Free, the trial is being led by Professor Chris Denton and his team.
Notes to editors
About the Royal Free London NHS Foundation Trust
The Royal Free began as a pioneering organisation and continues to play a leading role in the care of patients. Our mission is to provide world class expertise and local care. In the 21st century, the Royal Free London continues to lead improvements in healthcare.
The Royal Free London attracts patients from across the country and beyond to its specialist services in liver and kidney transplantation, haemophilia, renal care, HIV, infectious diseases, plastic surgery, immunology, Parkinson's disease, vascular surgery, cardiology, amyloidosis and scleroderma and we are a member of the academic health science partnership UCLPartners.