A radical and risky (adult) stem cell therapy has been shown to halt and even reverse some of the symptoms of those worst affected by multiple sclerosis, a disease that in many people has proved untreatable.
Doctors in Canada conducted an experimental (adult) stem cell transplant with 24 patients who were expected to be confined to a wheelchair within 10 years. After receiving the treatment most of the patients regained control of their lives, becoming able to walk, play sport and drive.
To succeed, the transplant required the destruction and rebooting of each person’s immune system – such a high risk approach that one of the patients died. But the others, followed up for between four and 13 years, had no further progression of the disease.
Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial
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.
We 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.