The first ever 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 in the year before joining the trial.
During the trial, researchers recruited 110 people with relapsing remitting MS and frequent relapses on convention drug therapy.
Key findings:
- 110 people 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 period, disability improved significantly after AHSCT.
- The EDSS score of patients 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 EDSS scores declined from an average score of 3.3 to 3.9.
- Within a year of joining 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.
- With a mean follow up of three years, treatment failure measured by disability progression was six per cent in the HSCT arm and 60 per cent in drug treatment arm.
- 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.
- No person in the AHSCT arm suffered any significant side effects.
AHSCT is an intensive treatment which essentially rebuilds a patient’s immune system using stem cells harvested from their own 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.
The results, have been hailed as “hugely encouraging” by researchers Professor Basil Sharrack and Professor John Snowden from Sheffield’s Royal Hallamshire Hospital, the sole UK site involved in this landmark trial which is being led by Dr Richard Burt, of North Western University in Chicago. The trial has closed to any further patient recruitment.