Stem Cells May Become Routine Therapy Against Multiple Sclerosis
Multiple sclerosis (MS) is a potentially debilitating condition in which the immune system targets the protective myelin sheath surrounding nerve cells.
There is currently no cure for the condition and existing treatments focus on accelerating recovery from attacks, altering the disease’s progression, and alleviating symptoms.
Among the promising emerging therapies for MS is stem cell therapy. It works by essentially resetting the immune system and preventing it from attacking myelin or causing inflammation in the brain and spinal cord.
Due to the potential that stem cell therapy is showing in this area, numerous medical professionals are recommending more liberal use, particularly for patients with severe MS.
In particular, several studies are suggesting that autologous haematopoietic stem cell transplantation (AHSCT) is ready to move from a last resort to a first-line treatment for MS.
From Third-Line to Routine Therapy
While AHSCT is part of the standard-of-care treatment for MS, the NHS England Treatment Algorithm for Multiple Sclerosis Disease-Modifying Therapies recommends it as a third-line therapy, only after first- and second-line disease-modifying treatments (DMTs) fail to control MS symptoms.
However, a large body of data is now available to compare AHSCT’s efficacy to other DMTs.
The European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party maintains a registry of MS patients treated with AHSCT. According to Raffaella Greco, MD, the EBMT registry contains data on over 4,100 patients from more than 40 countries who have undergone AHSCT treatment for autoimmune diseases.
Of the 2,100 patients treated for MS in the registry, the low incidence of death and relapses among the group supports the establishment of AHSCT as a standard practice.
Gavin Giovannoni, professor of neurology at Barts and the London School of Medicine and Dentistry, has found that younger patients with fewer prior DMTs have better outcomes with AHSCT. He advocates for offering the most effective treatments, such as AHSCT and monoclonal antibody-based therapies, to newly diagnosed patients to prevent organ damage.
Growing the Evidence Base
At Uppsala University in Sweden, open trials of AHSCT with relapsing-remitting MS (RRMS) patients have shown that more than 60% have had no evidence of disease activity (NEDA) over six years. NEDA is characterised by no increase in disability, no relapses, and no brain lesions seen with MRI scans.
The trial’s adverse events were also low, with a mortality rate of around 0.19%—less than the 0.3% rate for hip replacements. On top of this, AHSCT demonstrated a positive effect on brain health, slowing brain damage and potentially halting neuronal damage altogether.
Joachim Burman, PhD, from Uppsala University, highlighted that AHSCT can become cost-neutral within two to three years. This is particularly significant as MS patients typically require lifelong, expensive DMTs.
The StarMS trial, led by MD Basil Sharrack is another trial currently underway in Sheffield in the UK. This randomised controlled trial aims to generate more data proving that AHSCT provides better outcomes than DMT options for RRMS patients.
StarMS aims to recruit 198 patients aged 16–55 at 19 sites across the United Kingdom. John Snowden, director of the BMT Programme at Sheffield Teaching Hospitals NHS Foundation Trust, hopes to have 40 people in the trial by April 2023.
StarMS is funded by the UK National Institute for Health and Care Research. Snowdon says that, in the UK, they can perform AHSCT for around £35,000 ($43,000) per patient. So, from a health economics point of view, AHSCT to treat RRMS is cost-effective compared to modern DMTs.
The use of AHSCT as a first- or second-line therapy for severe RRMS patients, instead of a last resort, is being supported by increasing evidence of its safety, efficacy, and cost-effectiveness.
Ongoing trials like StarMS will further contribute to this evidence base over the coming months and years, building the foundation for making stem cell therapy a routine treatment for MS and other autoimmune disorders