Sheffield Teaching Hospitals NHS Foundation Trust has become the UK’s sole centre for a pioneering international research trial that could transform treatment for patients with a rare and life-limiting autoimmune condition.
The trial – named UPSIDE – is the first to investigate whether autologous haematopoietic stem cell transplantation should be offered as a first-line treatment for diffuse cutaneous systemic sclerosis, a severe form of scleroderma. The disease causes the immune system to attack healthy tissue, leading to skin hardening, inflammation, and damage to vital organs including the heart, lungs and kidneys.
UPSIDE is being conducted across 11 European sites, with Sheffield the only UK location involved. The study will recruit 60 patients to compare early stem cell transplantation with standard immunosuppressive therapy, followed by rescue transplantation if initial treatment fails.
Autologous haematopoietic stem cell transplantation involves collecting, storing and reinfusing a patient’s own stem cells after high-dose chemotherapy. It has already shown promise in advanced cases, improving survival and quality of life while reducing deterioration of skin and lung function.
Researchers now hope that offering the treatment earlier could yield even better outcomes. As the trial begins, experts are optimistic about its potential to reshape clinical practice.
Dr John Snowden, Consultant Haematologist and Director of Blood and Marrow Transplant at Sheffield Teaching Hospitals, said: “This is a landmark trial which could change the way we treat systemic sclerosis. The hope is that by intervening earlier with stem cell transplantation, we can improve long-term outcomes and reduce the burden of disease.”
If successful, UPSIDE could pave the way for stem cell therapy to become a standard early intervention for patients diagnosed with this aggressive condition.
