A near-complete immunoablation, followed by immune cell depleted autologous haemopoietic stem-cell transplantation (aHSCT), fully halted all detectable inflammatory activity in patients with multiple sclerosis (MS), according to researchers involved in a phase 2 single-arm trial at 3 hospitals. Furthermore, they noted, many of the patients had substantial recovery of neurological function despite their disease’s aggressive nature. In this study, they enrolled patients with MS, aged 18 to 50 years with poor prognosis, ongoing disease activity, and an Expanded Disability Status Scale (EDSS) of 3·0-6·0. They found:

• 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.

• The primary outcome, MS activity-free survival at 3 years after transplantation was 69·6%.

• 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 341 MRI sequential scans.

• 35% of patients had a sustained improvement in their EDSS score.

Citation: Atkins HL, Bowman M, Allan D, et al. Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: A multicenter single-group phase 2 trial. [Published online ahead of print June 9, 2016]. Lancet. doi:10.1016/S0140-6736(16)30169-6.