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Stem cell treatment in people with active multiple sclerosis stabilises the disease and improves disability. International large scale randomised trial into autologous haematopoietic stem cell transplantation (AHSCT) in relapsing remitting multiple sclerosis (MS) has shown that the treatment stabilised the disease and improved disability in people who had experienced two or more relapses.
Researchers recruited 110 participants with relapsing remitting MS and frequent relapses on convention drug therapy during the trial. Fifty percent of the participants on the trial were randomized to AHSCT, and the rest were randomized to the best available drug treatment . Disability was measured using a standard assessment tool known as the Expanded Disability Status Scale (EDSS) was used to test disability to see if the disease had improved, progressed or remained the same.
The lower levels of the scale (1.0 to 4.0) capture people who are still able to walk but have some difficulties with their vision, movement, sensation, coordination and bladder control. The middle levels (4.5 to 6.5) capture people who have difficulties with their mobility. The higher levels of the scale capture people with more severe disability. Participants with active, relapsing, remitting MS despite being treated with disease modifying drugs were randomised to receive either the best available drug treatment or AHSCT.
During the treatment follow up, disability improved after AHSCT treatment.Participants receiving the transplantation improved from an average of 3.5 to 2.4, which is unprecedented in MS treatment trials. This contrasted significantly with those receiving standard drug treatment whose scores declined from an average score of 3.3 to 3.9. During twelve months of the trial, only one patient in the transplant arm of the trial suffered a relapse compared to 39 relapses observed in the drug treatment arm.
Treatment failure measured by disability progression was six per cent in the HSCT arm and 60 per cent in drug treatment arm after three years. 30 people who were originally randomly allocated into the drug treatment arm of the trial were moved over to the transplant arm during the trial period after they had a decline in their EDSS scores. After AHSCT their scores improved from 5.2 to 2.6. None of the participants in the AHSCT arm suffered any side effects.
AHSCT is an intensive treatment which rebuilds patient’s immune system using stem cells harvested from their blood and bone marrow to reset it to a point before it caused MS. After having their stem cells harvested and frozen, the patient is then given a high dose of chemotherapy before the stem cells are thawed and re-infused into the patient’s blood to reboot their immune system. It is currently only suitable for patients with the relapsing remitting form of the disease who have failed to respond to standard treatments and who have lived with the disease for ten years or less. In the study, patients receiving autologous haematopoietic stem cell transplantation showed no disease and improves levels of disabilities after the treatment.
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