Swedish scientists are calling for a stem cell therapy to become standard practice for treating patients with multiple sclerosis, despite known risks.
MS is a leading autoimmune disease in young adults where the body’s lymphocytes – white blood cells – attack the fatty myelin sheath that protects nerves. This degradation exposes the nerve to scarring, which can lead to neurological symptoms ranging from motor issues, pain, numbness and tingling, fatigue, incontinence, vision and cognitive problems.
There is no single cause of MS and symptoms are often unique from patient to patient.
Publishing research in the British Medical Journal today, the research group from Uppsala University found the use of a stem cell therapy usually deployed to treat blood cancer is effective at slowing MS symptoms when used in healthcare settings.
They suggest that while further research is required to determine which patient groups would most benefit from the therapy, it should be considered as “a standard of care for highly active MS”.
Studies increasingly show benefit of bone marrow
For more than a decade, autologous haematopoietic stem cell transplantation – aHSCT, or more commonly known as a bone marrow transplant – has been tested as an MS treatment.
It involves the extraction of stem cells in bone marrow in the bloodstream which are returned to the patient after a course of chemotherapy ‘deactivates’ their immune system. The transplanted stem cells reactivate the immune system over several months.
It’s not without risks – as with any bone marrow transplant – with the immunocompromised nature of chemotherapy leaving patients susceptible to infection with other diseases. But research over the past 10 years has shown patients can experience positive outcomes, including slowing disease progression.
The Swedish study reviewed records of 174 people experiencing relapsing-remitting MS before 2020 – where a person relapses and experiences symptoms, followed by periods of symptomless recovery – and found no disease activity five years after treatment in about 73% of patients. That had reduced to 65% after a decade. Half of patients with disability prior to treatment saw improvement in their condition, although about a third experienced no change.
As an observational study with no control group for comparison, the researchers acknowledge no substantive conclusions can be drawn between the use of aHSCT and favourable outcomes detected.
But given the improvements observed, they say the treatment “has the potential to benefit a larger number of MS patients”. They consider the data sufficient for consideration as standard care therapy for patients.
In 2019, researchers from Northwestern University in Illinois performed a randomised clinical trial of 110 people with relapsing-remitting MS and investigated the benefit of aHSCT as opposed to other immunotherapies. It found progression of MS occurred in nearly three-quarters of patients not treated with bone marrow stem cells, while only 5% of those receiving the transplant saw similar outcomes.
The researchers behind the BMJ study say their findings support the Northwestern study’s outcomes.
“This [Northwestern] trial demonstrated a significant advantage of aHSCT over standard disease-modifying therapiesin terms of time to progression and neurological disability after 2 years,” they write.
“[Our] study supporters and strengthens the evidence from the sole randomised controlled trial.
“The incidence of severe adverse events was low, and there was no record of treatment-related mortality, suggesting that aHSCT can be safely implemented within routine healthcare.”
A cautious approach
MS Australia is the nation’s peak advocate for the disease. It currently supports a registry of MS patients who are undergoing – or who have already received – bone marrow transplant as a treatment.
The Uppsala study itself drew its patients for analysis from such a registry in Sweden.
But MS Australia is wary of recommendations for aHSCT to be adopted as a standard of care for particular groups with the disease.
At present, the therapy is only offered through clinical trials at St Vincent’s Hospital in Sydney, Austin Health and the Alfred Hospital in Melbourne. These trials have rigorous screening and require referral by a neurologist to participate.
In a statement, MS Australia told Cosmos it takes a “cautious approach” to the provision of aHSCT.
“AHSCT is not currently recommended as routine MS therapy but can be considered when high-efficacy DMTs fail or are contraindicated due to other health conditions or risk factors,” it says.
“AHSCT carries higher risks and potentially life-threatening complications than most approved therapies, including severe infections, fertility effects, and increased cancer risk. The risk of death 100 days after the procedure has gradually decreased, with recent research reporting a risk of less than 1% compared to about 3% a decade ago.
“Most studies show that the risk of disease activity returning gradually increases over time following AHSCT treatment. As with other MS therapies, in some people undergoing AHSCT, there may be a reversal of disability, but it is not widespread. Other people may continue to experience disease activity and disability progression (worsening) despite treatment with AHSCT.”
While MS Australia does not recommend specific therapy for people with MS, it supports careful consultation between a patient and their neurologist and healthcare providers to develop care plans, particularly as symptoms vary from person to person. It is supportive of patients having access to medications effective in Phase III clinical trials.