Phase II double blind trial to investigate the efficacy and the optimal way of administration (clinical, neurophysiological and neuroradiological effects) of autologous mesenchymal bone marrow stem cells in active and progressive Multiple Sclerosis
Author(s): ,
P Petrou
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
,
I Kassis
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
,
N Levin
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
,
N Raz
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
,
T Ben Hur
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah, Jerusalem, Israel
,
A Ginzberg
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
D Karussis
Affiliations:
Neuroimmunology and Cell Therapy Unit, Neurology Department, Hadassah University Hospital, Ein Kerem
ECTRIMS Online Library. Petrou P. Sep 16, 2016; 145972; P1290
Panayiota Petrou
Panayiota Petrou
Contributions
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Abstract

Abstract: P1290

Type: Poster

Abstract Category: Therapy - disease modifying - Others

Objective: To evaluate the safety and efficacy of transplantation of autologous bone marrow-derived mesenchymal stem cells (MSC), in MS.

Background: MSCs have been shown to possess neurotrophic and neuroprotective effects. Two prior pilot studies in our center showed that a single intrathecal(IT), or IV administration of MSCs was safe and well tolerated, and provided indications of clinical efficacy in MS and ALS.

Design/Methods: The current study involves a double-blind crossover design that will enroll upon completion 48 progressive MS-patients. The study started in February-2015 and till the day of this abstract, 29 patients have already been included. During the 2-month run-in period, functional evaluations (EDSS, MSFC, neurocognitive evaluation, quantitative/functional MRI, OCT, dynamic visual and neurophysiological tests) were performed monthly before the transplantation. Bone marrow-derived MSC (1x106/Kg) or placebo were injected to the patients, IT or IV. At 6-months the patients are treated with a second injection of MSC/placebo and are followed for safety and all the efficacy measures for 12 months. The study was approved by the Ethics committee and MOH and is monitored by an external CRO and a safety committee.

Results: No serious treatment-related adverse events have been observed this far. Until today 25 patients received the first transplantation and 13 also the second treatment. One patient already completed study.

Conclusions: IT and IM administration of autologous MSCs seems till now to be well-tolerated. Our trial, uniquely, uses the intrathecal way of administration and for the first time a rigorous double blind design utilizing extensive surrogate markers to detect possible clinical effects of regeneration. In addition, this study is the first to try to evaluate the optimal way of administration of stem cells in MS and to evaluate the possible additional benefit of a repeated injection. Detailed and updated safety data will be provided upon presentation (NCT02166021).

Keywords: Multiple Sclerosis (MS), Stem Cells, Mesenchymal Stromal Cells (MSC), Clinical Trial, Transplantation, Autologous, Intrathecal

Disclosure: Petrou Panayiota: Nothing to disclose

Kassis Ibrahim:Nothing to disclose

Levin Neta:Nothing to disclose

Raz Noah:Nothing to disclose

Hallimi Michelle:Nothing to disclose

Ben Hur Tamir:Nothing to disclose

Ariel Ginzberg:Nothing to disclose

Karussis Dimitrios: Nothing to disclose



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