IV/IT hUC-MSCs Infusion in RRMS and NMO: A 10-Year Follow-Up Study.

Zhengjuan Lu,Zhuo Liu,Jiayong Wu,Cun-Jin Zhang,Lin Zhu,Yun Xu

doi:10.3389/fneur.2020.00967

Abstract

Background: Stem cell transplantation is emerging as a potential therapeutic strategy in several autoimmune diseases. However, the safety and feasibility of long-term combined intravenous (IV) and intrathecal (IT) administration of hUC-MSCs in relapse remitting multiple sclerosis (RRMS) and neuromyelitis optica (NMO) is largely unknown.Objectives: In this study, we followed up the long-term safety and feasibility of combined IV and IT human umbilical cord mesenchymal stem cells (hUC-MSCs) transplantation in patients with RRMS and NMO.Methods: Five NMO patients and 5 RRMS patients were treated intravenously (4 times) and intrathecally (3 times) over a 21-day period with low-dose allogeneic umbilical cord blood–derived MSCs. All of the patients were monitored regularly by an investigator in a blinded manner to access the Expanded Disability Status Scale, MRI characteristics, and adverse events every 3 months within 12 months and once every year thereafter for 10 years after transplantation.Results: During the long-term follow-up, our data suggested that combined IV and IT administration of hUC-MSCs transplantation is safe and feasible. None of the intolerant adverse events, such as tumor formation and peripheral organ/tissue disorders, were observed throughout the 10-year follow-up.Conclusions: These data suggest that combined intravenous and intrathecal low-dose hUC-MSCs transplantation is safe and feasible in RRMS and NMO patients in the long term. The conclusion requires confirmation by future clinical trials in a larger cohort.

Highlights

Multiple sclerosis (MS) is a chronic, immune-mediated, demyelinating disorder of the central nervous system (CNS) [1]
Baseline Characteristics, Treatments of Patients, and Follow-Up. Because both neuromyelitis optica (NMO) and MS diseases were affected more in women, all of the patients enrolled in the current follow-up study were female and the mean disease duration of participants was 6.4 years
Patients 1–5 had a diagnosis of NMO, whose mean Expand Disability Status Scale (EDSS) score was 5, and patients 6–10 were diagnosed relapse remitting multiple sclerosis (RRMS) with mean EDSS score of 4.7 at baseline (Table 1)

Summary

Introduction

Multiple sclerosis (MS) is a chronic, immune-mediated, demyelinating disorder of the central nervous system (CNS) [1]. The disease predominantly affects individuals in their early adult lives. There are about 2.3 million people living with multiple sclerosis globally, which crucially increases the burden of family and society. The firstline treatments mainly depend on steroids for acute episodes and disease-modifying drugs (DMDs) hUC-MSCs Therapy in RRMS and NMO for chronic stage; these therapeutic approaches share some common characteristics, such as suppressed immune system and compromised host defense to bacterial and virus infection. Stem cell transplantation is emerging as a potential therapeutic strategy in several autoimmune diseases. The safety and feasibility of long-term combined intravenous (IV) and intrathecal (IT) administration of hUC-MSCs in relapse remitting multiple sclerosis (RRMS) and neuromyelitis optica (NMO) is largely unknown

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