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Mesenchymal stem cells for multiple sclerosis: hype or hope?

Lancet Neurol. 2021 Nov;20(11):881-882. doi: 10.1016/S1474-4422(21)00324-0. PMID: 34687621.

Authors/Editors: Hohlfeld R.
Publication Date: 2021


In the search for new approaches to treat or even cure multiple sclerosis, several forms of cell-based therapies have been explored, including haematopoietic stem cells, mesenchymal stem cells, and oligodendrocyte progenitor cells. 1 In the past 15 years, several small uncontrolled trials have assessed the safety and feasibility of approaches using mesenchymal stem cells derived from diverse tissues (eg, bone marrow, adipose tissue, or placenta), with different routes of administration (eg, intravenous or intrathecal), and various outcome measures (eg, clinical, neurophysiological, or MRI-based measures). Petrou and colleagues did a randomised, double-blind, placebo-controlled trial to investigate the safety and efficacy of bone marrow-derived mesenchymal stem cells in patients with progressive multiple sclerosis. Following a complex crossover design, 48 patients with secondary progressive (n=41) or primary progressive (n=7) multiple sclerosis received mesenchymal stem cells or placebo via intravenous or intrathecal injection. The results were positive in that the primary efficacy outcome based on the Expanded Disability Status Scale was met. Significantly fewer patients had treatment failure in the groups treated with mesenchymal stem cells intrathecally (6-7%) or intravenously (9·7%) than those in the sham-treated group (41·9%; p=0·0003 vs intrathecal administration; p=0·0008 vs intravenous administration). Patients with MRI activity and those who received mesenchymal stem cells intrathecally showed the largest benefit

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