- AHSCT has emerged as a potential treatment option for highly active relapsing-remitting MS. AHSCT involves using the individual’s own stem cells to ‘reset’ the immune system.
- MS Australia-funded researchers investigated how AHSCT performs in comparison to other commonly used disease modifying therapies (DMTs) for relapsing-remitting MS.
- When comparing against three DMTs registered in Australia, AHSCT was associated with significantly fewer relapses when compared to fingolimod, with a lower relapse rate compared to natalizumab, but did not find evidence for a difference in the effectiveness of AHSCT and ocrelizumab over a shorter available follow-up time.
Advancing MS Treatment: Exploring the Potential of Stem Cell Therapy for Treatment-Resistant MS
In recent years, autologous hematopoietic stem cell transplant (AHSCT) has emerged as a potential treatment option for highly active relapsing-remitting MS. This form of MS is characterised by frequent relapses and worsening disability.
AHSCT involves using the patient’s own stem cells to replace the faulty immune system with a new one, aiming to ‘reset’ the immune response and halt the progression of the disease.
MS Australia-funded researcher Professor Tomas Kalincik, and his team at the Royal Melbourne Hospital recently published their findings in JAMA Neurology, indicating that stem cell therapy was effective for patients with treatment-resistant MS, offering hope to those with few options and aggressive disease.
What Did the Researchers Do?
Professor Kalincik and his team conducted a comparative treatment effectiveness study to evaluate how AHSCT performs in comparison to other commonly used DMTs for relapsing-remitting MS.
The study was conducted at six specialist MS centres with AHSCT programs and utilised data from the international MSBase registry. They analysed data from patients who had been treated with either AHSCT, fingolimod, natalizumab, or ocrelizumab and had a follow-up period of at least two years with at least two disability assessments.
Participants were matched based on their clinical and demographic characteristics. This matching process helped to reduce potential biases and ensured that the groups being compared were similar in terms of relevant factors.
What Did the Researchers Find?
The study included a total of 4915 individuals, with 167 receiving AHSCT, 2558 treated with fingolimod, 1490 with natalizumab, and 700 with ocrelizumab.
Before matching, the AHSCT group tended to be younger and had more severe disability compared to the other treatment groups. However, after matching, the groups were more comparable in terms of their characteristics.
The researchers found that AHSCT was associated with significantly fewer relapses when compared to fingolimod, and it showed a marginally lower annualised relapse rate compared to natalizumab.
However, AHSCT was found to have similar efficacy to ocrelizumab in preventing relapses over a shorter follow-up time (ocrelizumab has had a shorter time registered in Australia than the other two DMTs).
In terms of disability, AHSCT had a similar risk of disability worsening compared to the other treatments, but it showed a higher chance of disability improvement when compared to fingolimod and natalizumab.
The study reported only one case of AHSCT-related mortality out of 159 patients, indicating that, in general, AHSCT is safe. Still, as with any medical procedure, it carries some degree of risk.
What Does This Mean for People with MS?
The findings from this study suggest that AHSCT may be a promising treatment option for individuals with highly active relapsing-remitting MS.
Compared to some of the widely used DMTs, such as fingolimod and natalizumab, AHSCT demonstrated superior effectiveness in reducing relapse rates and promoting recovery from disability.
Professor Kalincik commented that less than 0.2% of individuals have “highly active, relapsing” MS, making it more difficult to conduct clinical trials to develop new treatments in this group.
He added that MS clinicians only resort to the riskier AHSCT for patients after other therapies are unsuccessful, with about a 1% mortality rate during the chemotherapy phase of the procedure.
It is important to consider that AHSCT is not suitable for everyone with MS. The decision to pursue this treatment should be made in close consultation with neurologists and/or MS specialists who can assess the specific condition and overall health of the individual.
As advances in MS treatments continue to evolve, MS Australia remains committed to keeping people with MS informed about the latest developments. Our goal is to empower people with MS to work closely with their healthcare providers to find the most suitable and effective treatments for their individual needs.
