Haematopoietic stem cell transplantation (HSCT) has been used as treatment for MS since the mid-nineties. The aim of this treatment is to reboot the immune system using chemotherapy which destroys the defective immune cells driving the inflammation in the brain and spinal cord and rebuilds a regenerated immune system from the patient's own blood stem cells, with greater numbers of naïve and regulatory immune cells.
Within the European Society for Bone Marrow Transplantation Registry, more than 800 patients have undergone this treatment, and elsewhere around the world prospective clinical trials have been reporting.
The largest reported case series is from Northwestern University in Chicago which included 145 patients (123 with RRMS and 28 with SPMS). There was not a control treatment arm but in patients with RRMS, AHSCT was linked to improved disability in 64% of the patients who were followed up. Of those patients who were followed for 4 years, 80% were relapse-free and 87% were disability progression-free. There was a significant decrease in T2 lesion volume and a significant improvement in the quality of life scores. There was no treatment related mortality. The treatment was less effective for patients with secondary progressive MS or those who had MS for more than ten years, highlighting that this treatment works most effectively in patients with early inflammatory disease.
The Chicago group, led by Dr Richard Burt, are now leading an international multicentre randomised clinical trial (the MIST trial) to compare AHSCT against FDA approved disease modifying agents as a second line treatment in patients with RRMS who have had two or more significant relapses in the previous 12 months despite the use of disease modifying agents. The Royal Hallamshire Hospital in Sheffield is one of four international centres running this trial.
Basil and John
Burt RK et al. Association of nonmyeloablative hematopoietic stem cel transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA. 2015; 313(3):275-284.
CoI: None declared
For further information on ASCT treatment and the research trial, please see www.sth.nhs.uk/panorama (CLICK) or call 0114 2715934.