Aggressive chemotherapy followed by a stem cell transplant can halt the progression of multiple sclerosis (MS), a small study has suggested.
The research, published in The Lancet, looked at 24 patients aged between 18 and 50 from three hospitals in Canada.
For 23 patients the treatment greatly reduced the onset of the disease, but in one case a person died.
An MS Society spokeswoman said this type of treatment does “offer hope” but also comes with “significant risks”.
Around 100,000 people in the UK have MS, which is an incurable neurological disease.
MS causes the immune system to attack the lining of nerves in the brain and spinal cord. Most patients are diagnosed in their 20s and 30s.
One existing treatment is for the immune system to be suppressed with chemotherapy and then stem cells are introduced to the patient’s bloodstream – this procedure is known as an autologous haematopoietic stem cell transplant (HSCT).
The Canadian researchers went further – not just suppressing the immune system, but destroying it altogether.
It is then rebuilt with stem cells harvested from the patient’s own blood which are at such an early stage, they have not developed the flaws that trigger MS.
The authors said that among the survivors, over a period of up to 13 years, there were no relapses and no new detectable disease activity.
All the patients who took part in the trial had a “poor prognosis” and had previously undergone standard immunosuppressive therapy which had not controlled the MS – which affects around two million people worldwide.
Strong immunosuppression, including chemotherapy and immune-depleting antibodies followed by autologous haemopoietic stem-cell transplantation (aHSCT), has been used to treat patients with multiple sclerosis, improving control of relapsing disease. We addressed whether near-complete immunoablation followed by immune cell depleted aHSCT would result in long-term control of multiple sclerosis.
They did this phase 2 single-arm trial at three hospitals in Canada. They enrolled patients with multiple sclerosis, aged 18–50 years with poor prognosis, ongoing disease activity, and an Expanded Disability Status Scale of 3·0–6·0. Autologous CD34 selected haemopoietic stem-cell grafts were collected after mobilisation with cyclophosphamide and filgrastim. Immunoablation with busulfan, cyclophosphamide, and rabbit anti-thymocyte globulin was followed by aHSCT. The primary outcome was multiple sclerosis activity-free survival (events were clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, and sustained progression of Expanded Disability Status Scale score). This study was registered at ClinicalTrials.gov, NCT01099930.
Between diagnosis and aHSCT, 24 patients had 167 clinical relapses over 140 patient-years with 188 Gd-enhancing lesions on 48 pre-aHSCT MRI scans. Median follow-up was 6·7 years (range 3·9–12·7). The primary outcome, multiple sclerosis activity-free survival at 3 years after transplantation was 69·6% (95% CI 46·6–84·2). With up to 13 years of follow-up after aHSCT, no relapses occurred and no Gd enhancing lesions or new T2 lesions were seen on 314 MRI sequential scans. The rate of brain atrophy decreased to that expected for healthy controls. One of 24 patients died of transplantation-related complications. 35% of patients had a sustained improvement in their Expanded Disability Status Scale score.
They describe the first treatment to fully halt all detectable CNS inflammatory activity in patients with multiple sclerosis for a prolonged period in the absence of any ongoing disease-modifying drugs. Furthermore, many of the patients had substantial recovery of neurological function despite their disease’s aggressive nature.
SOURCES- BBC News, The Lancet, Wikipedia
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