Neural Stem Cell Transplantation in Multiple Sclerosis Patients
Neural Stem Cell Transplantation in Multiple Sclerosis Patient: a Phase I Study
What it found
A paper reports the results; we have not read it yet. See Papers below.
What was tested
Treatment tested
- human fetal-derived Neural Stem Cells (hNSCs)
What it measured
Main measures
SHORT TERM (0-24 hours) Overall survival
0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SHORT TERM (0-24 hours) Overall safety and tolerability measured by Adverse Event (AE) recording
0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SafetySHORT TERM (0-24 hours) Changes in neurological conditions not related to disease
0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SHORT TERM (0-24 hours) Proportion of successful intrathecal administration procedure (feasibility)
0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
FeasibilityMID TERM (day 1- day 14) Overall survival
from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
MID TERM (day 1- day 14) Overall safety and tolerability measured by AE recording
from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
SafetyMID TERM (day 1- day 14) Changes in neurological conditions not related to disease
from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
LONG TERM (day 15 - week 96) Overall survival
from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
LONG TERM (day 15 - week 96) Overall safety and tolerability measured by AE recording
from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
SafetyLong term incidence of malignancies
from day 0 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Evaluation of changes in quality of life measures
12, 24, 48, 72 and 96 weeks
Quality of life (reported by participants)LONG TERM (day 15 - week 96) Changes in neurological conditions not related to the disease
from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Who could take part
Progressive MS · 18 to 55
Show the full criteria
Inclusion criteria
1. Signature of the informed consent by the patient or patients' legal tutors 2. Age 18 to 55 years 3. Diagnosis of a. Progressive MS as per the revised MC Donald 2010 criteria with a progressive course according to 2013 Lublin phenotypes classification (PMS) with failure or intolerance to all approved therapies according to the disease course or without any alternative approved therapy 4. Evidence of progression of disease defined by an increase of ≥ 0.5 Expanded Disability Status Scale (EDSS) points in the last 12 months 5. Disease duration 2 to 20 years (included) 6. Expanded Disability Status Scale (EDSS) ≥ 6.5 7. Presence of oligoclonal band in the cerebrospinal fluid (CSF) is required for Primary Progressive MS
Exclusion criteria
They will be excluded from the study patients: 1. with any active or chronic infection or diseases other than MS including but not limited to infection with HIV1-2, Hepatitis B or Hepatitis C and tuberculosis or immune deficiency syndromes; 2. treated with any immunosuppressive therapy, including but not limited to natalizumab and fingolimod, within the 3 months prior to screening; 3. treated with interferon-beta or glatiramer acetate within the 30 days prior to screening; 4. treated with corticosteroids within the 30 days prior to screening; 5. if relapse occurred during the 30 days prior to screening; 6. with contraindications for or intolerance to any medication, treatments and procedures that will be used in the study; 7. pregnant or in lactation or of childbearing age who are not willing to use a contraceptive method effective* for the entire duration of the study; 8. who, in the opinion of the investigator, showing any condition that would preclude study participation. * refer to guideline http://www.hma.eu/fileadmin/dateien/Human_Medicines/01 About_HMA/Working_Groups/CTFG/2014_09_HMA_CTFG_Contraception.pdf
As registered. Criteria can change, so ask the trial team or your neurologist.
About this trial
In the sponsor's words
This is a phase I study evaluating the feasibility, safety and tolerability of intrathecally administered human Neural Stem Cells (hNSCs), at an escalating dose ranging from 0.7x10\^6±10% cells to 5.7x10\^6±10% cells/kg of body weight, in patients affected by Progressive Multiple Sclerosis
Papers
Results
Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study.Nat Med · 2023
Results
Kokaia Z, Martino G, Schwartz M, Lindvall O. Cross-talk between neural stem cells and immune cells: the key to better brain repair? Nat Neurosci. 2012 Jul 26;15(8):1078-87. doi: 10.1038/nn.3163.Results
Martino G, Franklin RJ, Baron Van Evercooren A, Kerr DA; Stem Cells in Multiple Sclerosis (STEMS) Consensus Group. Stem cell transplantation in multiple sclerosis: current status and future prospects. Nat Rev Neurol. 2010 May;6(5):247-55. doi: 10.1038/nrneurol.2010.35. Epub 2010 Apr 20.Results
Pluchino S, Gritti A, Blezer E, Amadio S, Brambilla E, Borsellino G, Cossetti C, Del Carro U, Comi G, 't Hart B, Vescovi A, Martino G. Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates. Ann Neurol. 2009 Sep;66(3):343-54. doi: 10.1002/ana.21745.Results
Pluchino S, Quattrini A, Brambilla E, Gritti A, Salani G, Dina G, Galli R, Del Carro U, Amadio S, Bergami A, Furlan R, Comi G, Vescovi AL, Martino G. Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature. 2003 Apr 17;422(6933):688-94. doi: 10.1038/nature01552.Results
Pluchino S, Zanotti L, Rossi B, Brambilla E, Ottoboni L, Salani G, Martinello M, Cattalini A, Bergami A, Furlan R, Comi G, Constantin G, Martino G. Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism. Nature. 2005 Jul 14;436(7048):266-71. doi: 10.1038/nature03889.
Loading reviews and guidelines…
History
Started
May 2017
Main results due
Jul 2021
Finished
Jul 2021