Stem cell therapy
Stem cell therapies are being explored as a potential way to address the unmet need for more effective treatments for MS
Last updated: 1st August 2025
The following information should not be relied on to suggest a course of treatment for a particular individual, and it should not be used in place of a visit, call, consultation or the advice of a physician or other qualified healthcare provider.
There is continued need for more effective treatments for MS. Stem cell therapies are being explored as a potential way to address this unmet need. The unique properties of stem cells make them promising candidates for treatments that can slow MS disease activity.
Autologous haematopoietic stem cell transplantation (aHSCT) is another type of treatment for MS. Hematopoietic stem cells are a type of adult stem cell made in the bone marrow, which have the ability to produce the different cells found in the blood, including immune cells. In aHSCT, stem cells from a person’s own bone marrow are collected and stored, while a significant portion of the rest of an individual’s immune cells are reduced by chemotherapy. Following chemotherapy, the stored stem cells are reintroduced into the body and over time they produce new cells that repopulate the body with all types of blood cells including immune cells.
This treatment attempts to ‘reformat’ the person’s immune system, and stop the inflammation that contributes to active, relapsing MS or active, progressive MS. However, these stem cells are not able to regenerate damaged nerves or other parts of the brain and spinal cord.
MS is a very varied disease and therefore this treatment is not suitable for all people nor all types of MS. It is important to note that aHSCT is an intensive treatment with significant risks, including life-threatening for some people, and the chemotherapy itself has several known side effects.
Whilst aHSCT is considered to be a treatment for highly active MS in a few countries, the conditions under which it is made available may differ between, or even within, countries. In some cases, even if aHSCT is approved for use, it may not be available due to lack of specialist expertise or resources.
Crucially, there is a need for further large, gold-standard, randomised controlled trials specifically for MS, which compare aHSCT to currently available MS therapies.