Published: 02 December 2022
A world-first trial will test if stem cell transplantation could be used as a first-line treatment for patients with 'highly active' and ‘aggressive’ forms of multiple sclerosis (MS).
The £2.3m StarMS study is funded by an NIHR and Medical Research Council partnership. It is the first to compare the effectiveness and safety of autologous hematopoietic stem cell transplantation (AHSCT) with four leading highly effective drug treatments.
Experts at Sheffield Teaching Hospitals NHS Foundation Trust and the University of Sheffield’s Clinical Trials Research Unit are leading the research.
The study builds on results of the landmark MIST trial. MIST found stem cell transplantation could reverse disability in certain patients with MS. It also showed AHSCT worked better than MS drugs available at the time in reducing the risk of disability in patients with highly ‘active’ MS.
In StarMS, experts will test how AHSCT affects patients with highly ‘active’ MS failing drug treatment, or as a first-line treatment for patients with the ‘aggressive’ MS.
The StarMS team aims to recruit up to 200 patients. Each participant will be randomly allocated to receive a stem cell transplant, or one of the four highly effective drugs. These are Alemtuzumab, ocrelizumab, ofatumumab and cladribine. They weren't around during the MIST trial, but have since shown great promise in clinical trials.
The findings could revolutionise care for thousands of people who have MS. It is the most common chronic inflammatory and neurodegenerative disease of the central nervous system in young adults. It affects 100,000 people in the UK, and 2.3 million globally.
Chief Investigator Professor John Snowden, Consultant Haematologist at Sheffield Teaching Hospitals NHS Foundation Trust, said: “AHSCT has been shown to be highly effective in stabilising, and even reversing disability, in certain patients with MS.
“But the treatment landscape in this condition has shifted since the original MIST trial. The trial will measure how good and safe AHSCT is when compared head-to-head with the latest leading treatments for MS. In this way, we hope to determine the exact place of AHSCT in the modern treatment pathways for patients with severe MS.
“Such translational research may also offer important insights into the fundamental immune system abnormalities that cause MS in the first place.”
Lead Trial Neurologist, Professor Basil Sharrack, who is also a Principal Investigator at NIHR Sheffield Biomedical Research Centre (BRC), said: “Currently, there is no cure for multiple sclerosis, but huge advances have been made in recent years, with the MIST trial offering renewed hope for people living with this devastating condition.
“We now want to bring this research up to date, by taking into account all the latest advances in treatments. This could also provide us with the solid evidence we need to demonstrate that AHSCT can be offered as a first line treatment for those with the aggressive form of the condition. We are delighted to be using our internationally renowned expertise in stem cell transplantation to bring this latest research to the potential benefit of thousands of patients.”
The Efficacy and Mechanism Evaluation (EME) Programme - an NIHR and MRC partnership - is funding the study.
NIHR’s Clinical Research Network (CRN) is delivering the trial at 19 UK sites. Sheffield NIHR Clinical Research Facility (CRF) and Sheffield Biomedical Research Centre (BRC) are also supporting the study.