There is great interest in the MS community around autologous haematopoietic stem cell transplantation or AHSCT. This treatment has been used to treat highly active inflammatory MS for over two decades. Evidence suggests that it can be effective in treating people with relapsing remitting MS where they have failed to respond to more conventional disease modifying treatments. While it does appear to be an effective treatment, AHSCT it is also associated with greater short-term risks which have limited its use in MS.
AHSCT is an immunosuppressive chemotherapy treatment combined with reinfusion of blood stem cells to help re-build the immune system. It has a broad suppressive effect on all immune cells, whereas many of the more conventional treatment options are more selective and targeted to a small subset of immune cells.
One important question that is yet to be definitively answered is how AHSCT stacks up as a treatment against the new generation, highly efficacious therapies available for relapsing MS. However, to date there have been limited clinical trials comparing the success rate of AHSCT with more conventional treatments of relapsing remitting MS.
Associate Professor Tomas Kalincik is utilising data gathered through the world’s largest MS clinical database, MSBase as well as other international clinical registers to compare the clinical outcomes of people treated with ASHCT compared with those of similar patients who have been treated with natalizumab, alemtuzumab, fingolimod or mitoxantrone. This will help build a more complete picture of the outcomes of AHSCT.
Associate Professor Tomas Kalincik and his team have made significant strides in their analyses. Their findings revealed that AHSCT is a highly effective treatment option for those with aggressive MS. When compared to the strongest disease modifying therapies (DMTs), AHSCT demonstrated a superior ability to prevent relapses compared to mitoxantrone and fingolimod and showed similar relapse prevention potential to natalizumab (Tysabri®) and potentially alemtuzumab (Lemtrada®). The research team did not identify significant differences in the likelihood of disability worsening between AHSCT and the other therapies studied. However, they did uncover a higher rate of recovery from disability in two key comparisons: against natalizumab and fingolimod (Gilenya®). They have recently published their findings in JAMA Neurology.
Associate Professor Kalincik and his team are now investigating AHSCT further by comparing it with other treatments that work differently, especially those targeting B cells, such as cladribine (Mavenclad®). They also intend to explore the characteristics of individuals that may influence how effective AHSCT is in comparison to other therapies. In addition, they will investigate the role of AHSCT in treating progressive forms of MS in more detail.
Kalincik T, Sharmin S, Roos I, et al. Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis. JAMA Neurol. 2023;80(7):702–713. doi:10.1001/jamaneurol.2023.1184
Updated 23 May 2022
Updated: 06 October, 2020