By Elisa Roldan-Galvan and Tobias Alexander, Secretary and Chair of the Autoimmune Diseases Working Party (ADWP) and Gavin Brittain, member of the ADWP neurology subcommittee.
Multiple sclerosis (MS) is a lifelong condition where the immune system mistakenly attacks the brain and spinal cord. Symptoms can include problems with walking, vision, balance, fatigue, thinking, and hand function. For many patients, living with MS means uncertainty about how their condition may change over time.
Over the past twenty years, there have been major advances in treatment. Among the most important has been the development of autologous haematopoietic stem cell transplantation (aHSCT).
Today, aHSCT is recognised as a standard treatment option for carefully selected people with aggressive inflammatory MS who continue to have disease activity despite other treatments. This represents an enormous change from only a few years ago, when transplantation in MS was considered experimental.
What is aHSCT?
AHSCT is a procedure that aims to “reset” the immune system.
The process involves collecting a patient’s own blood stem cells, followed by chemotherapy to remove the immune cells driving MS inflammation. The stored stem cells are then returned to help rebuild the new immune system.
This treatment reduces relapses and stops the abnormal immune attack that causes ongoing damage in MS. For some people, aHSCT can lead to long periods without relapses, new MRI lesions, or worsening disability.
While aHSCT is not suitable for everyone with MS, outcomes have improved significantly over time thanks to better patient selection, growing clinical experience, and better supportive care during the procedure.
The importance of research and international collaboration
One of the reasons outcomes continue to improve is the strong international research effort led by clinicians, scientists, nurses, allied health professionals, and people with MS.
The EBMT Registry has played a major role in this progress. By collecting data from over 2,600 transplant procedures for people with MS across Europe, the Registry has helped researchers better understand safety, effectiveness, long-term outcomes, and best practice in MS transplantation.
The efforts of the EBMT community have led to international recommendations and have helped establish aHSCT as an accepted treatment option MS care.
Several important clinical studies led by ADWP members are also helping answer key questions about when and how transplantation should be used: StarMS in the United Kingdom, NET-MS in Italy, RAM-MS in Sweden, and BEAT-MS in the United States. Together, these studies are helping clinicians and patients make more informed treatment decisions and shaping the future of care.
Looking ahead: new cellular therapies
Alongside aHSCT, researchers are now exploring innovative approaches such as CAR-T cell therapy and other immune-targeted cellular treatments. These therapies aim to precisely target the harmful immune cells involved in autoimmune diseases, potentially offering new options for people whose disease remains difficult to control.
Although these approaches are still at an earlier stage of development in MS, early results are encouraging and have generated significant interest within the scientific community. The topic will also feature in dedicated sessions at upcoming EBMT educational meetings, reflecting the growing momentum in this field.
Keeping patients at the centre
While the scientific advances that have been made are exciting, it is important to remember the people behind the research. Living with MS affects every part of life and that is why patient involvement, education, and shared decision-making are essential. People with MS should have access to clear information about benefits, risks, uncertainties, and available treatment options so they can make informed choices together with their healthcare teams.
The MS community has also benefited enormously from the voices of those who act as advocates and continue to raise awareness and improve understanding of the lived experience of MS.
A message of hope
There is still no cure for MS, and many challenges remain. However, the progress seen in recent years gives real reason for optimism.
AHSCT has moved from an experimental approach to an established treatment option for selected patients. International studies and registry data continue to improve understanding and outcomes. New cellular therapies are opening additional exciting treatment possibilities for the future.
Most importantly, collaboration across the global MS and transplant communities continues to drive progress forward.