New research from Dr Paolo Muraro, of the Department of Medicine at Imperial College London and his colleagues recently reported further evidence of autologous hematopoietic stem cell transplantation (AHSCT) as an effective treatment for multiple sclerosis (MS).
They found that the procedure halted disease progression for five years in almost half of patients.
These results come just after another recent study revealed the success of a similar treatment in a small group of patients with relapsing-remitting MS (RRMS).
However, eight patients died within 100 days of treatment and Dr Muraro and his team warned that further trials are needed to determine the efficacy and safety of AHSCT.
When a patient undergoes AHSCT treatment, the process effectively resets their immune system. Firstly, their stem cells are harvested. They are then subjected to a high-dose chemotherapy to eliminate any diseased cells and the harvested stem cells are then returned to the patient’s bloodstream, with an aim of restarting blood cell production.
In this study, 46 per cent of patients saw no MS progression five years after receiving AHSCT treatment.
Dr Muraro noted: “We previously knew this treatment reboots or resets the immune system – and that it carried risks – but we didn’t know how long the benefits lasted.”
The researchers of this study assessed data from 25 treatment centres across 13 countries, identifying 281 patients with multiple sclerosis (MS) who underwent AHSCT 1995-2006. Of these patients, 78 per cent had a progressive form of MS.
Using the Expanded Disability Status Scale (EDSS), the team evaluated patients' progression-free survival at 5 years after treatment and any improvements in MS symptoms.
An EDSS score of zero represents no disability, seven represents the use of a wheelchair, while 10 represents death from MS. At the beginning of the study, patients had an average EDSS score of 6.5.
Patients with RRMS - characterised by inflammatory attacks, or "flare-ups," followed by periods of remission - had the best outcomes, with 73 per cent experiencing no worsening of symptoms in the five years after AHSCT.
Additionally, patients experienced small improvements in MS symptoms after AHSCT. Patients with progressive MS saw their EDSS score rise by 0.14 a year after treatment, while patients with RRMS experienced a 0.76 increase in their EDSS score.
Patients with a younger age, few immunotherapies prior to AHSCT, and a lower EDSS score at study baseline also showed better outcomes with AHSCT.
This research shows real promise, but with eight deaths which were thought to be treatment related there is still a risk involved.
"In this study, which is the largest long-term follow-up study of this procedure, we've shown we can 'freeze' a patient's disease and stop it from becoming worse for up to 5 years.
“However, we must take into account that the treatment carries a small risk of death, and this is a disease that is not immediately life-threatening." Dr. Paolo Muraro said.
Dr. Muraro notes that, importantly, this study did not include a group of MS patients who did not receive treatment, further highlighting the need for more studies assessing the safety and efficacy of AHSCT.
Source: MS-UK (01/03/17)