Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS)

University of Cambridge10 enrolled

Overview

Hypothesis: Intravenous administration of bone marrow-derived autologous adult human mesenchymal stem cells is a safe novel therapeutic approach for patients with multiple sclerosis. Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS) is a phase I/IIA trial designed to establish the safety of intravenous administration of bone marrow-derived autologous adult human mesenchymal stem cells to patients with multiple sclerosis.

Disease under investigation: Multiple Sclerosis Phase: I/IIA Number of patients: 10 Design: 18 month cross over, single treatment at 6 months Intervention: Administration of bone marrow-derived autologous mesenchymal stem cells Route of administration: Intravenous Dose: Up to 2,000,000 Mesenchymal Stem Cells per kilogram Source of patients: Referrals accepted from Neurologists in East Anglia and North London, UK Referral Criteria: (all 3 required) 1. Clinically definite multiple sclerosis 2. Expanded Kurtzke Disability Status Score 2.0 - 6.5 (inclusive) 3. Evidence of optic nerve damage by * history of optic neuritis, or * relative afferent pupillary defect, or * optic atrophy on fundoscopy, or * abnormal visual evoked potential from either or both eyes suggestive of demyelination Primary Objective: Establish the safety of intravenously administered bone marrow-derived autologous mesenchymal stem cells at a dose of up to 2,000,000 cells/kg over 12 months by monitoring adverse reactions. Secondary Objectives: Explore the efficacy of intravenously administered bone marrow-derived autologous mesenchymal stem cells at a dose of up to 2,000,000 cells/kg over 12 months on visual function by clinical, neurophysiological, and imaging assessments. Outcome Measures: 1. Primary * Adverse events 2. Secondary * Visual function (acuity and colour) * Visual evoked potential latency * Optic nerve Magnetisation Transfer Ratio * Retinal nerve fibre layer thickness (by optical coherence tomography) * Brain lesion Magnetisation Transfer Ratio * MRI brain T1 hypointensity load * T cell response suppression 3. Tertiary * Multiple Sclerosis Functional Composite Score * Expanded Kurtzke Disability Status Score

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Multiple Sclerosis

Interventions

MSC TreatmentPROCEDURE

Intravenous administration of up to 2x10\^6 autologous MSCs per kg

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Clinically definite multiple sclerosis * Expanded Kurtzke Disability Status Score 2.0 - 6.5 (inclusive) * Evidence of optic nerve damage by: * history of optic neuritis, or * relative afferent pupillary defect, or * optic atrophy on fundoscopy, or * abnormal visual evoked potential from either or both eyes suggestive of demyelination * Prolonged visual evoked potential P100 latency with preserved waveform * T2 lesion on MRI optic nerve * Retinal nerve fibre layer thickness on optical coherence tomography \> 40 microns Exclusion Criteria: * Age \< 18 years * Age \> 65 years * Patient lacks capacity to give informed consent * Presence of a severe bleeding disorder * Planning a pregnancy during the trial period * Current MS disease modifying therapy

Locations (2)

University of Cambridge Dept of Clinical Neurosciences

Cambridge, Cambridgeshire, United Kingdom

University College London Institute of Neurology

London, London, United Kingdom

Outcomes

Primary Outcomes

Adverse events

Time frame: 0,1,2,3,4,12 and 52 weeks post treatment