Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Patients With Multiple Sclerosis

Tisch Multiple Sclerosis Research Center of New York

Status and phase

Completed

Phase 1

Conditions

Multiple Sclerosis

Treatments

Biological: intrathecal administration of autologous MSC-NP

Study type

Interventional

Funder types

Other

Identifiers

NCT01933802

TISCHMS-MSCNP-001

IND 13889 (Other Identifier)

Details and patient eligibility

About

The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Study participants will receive three intrathecal injections of culture-expanded autologous MSC-NPs at three month intervals.

Enrollment

20 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of MS as defined by the McDonald criteria
Diagnosis of primary progressive or secondary progressive MS
Between the ages of 18-70 years
Significant disability shown by an Expanded Disability Status Score (EDSS) of 3.0 or greater that was not acquired within the last 12 months
Stable disease state as evidenced by a lack of gadolinium-enhancing lesions on an MRI and by a stable MRI disease burden (number of T2 lesions and size of lesions) in the last six months and no significant change in EDSS (1 point or more) in the last 12 months
Must agree to undergo MRIs at the time of enrollment, 2 months after the first treatment, and 27 months after the last treatment
Live in northern New Jersey, southern New York, or southwestern Connecticut during the study period, or patients must be able to arrange reliable travel accommodations to be present for every study visit if they live farther away.

Exclusion criteria

Pregnant or nursing mothers or any woman intending to become pregnant in the next three years
All patients will have pre-study liver function tests, PT/PPT, platelets, hematocrit, and renal function laboratory tests done. Only patients whose values are in the normal range as determined by the laboratory norms based on age and sex will be allowed to participate.
Use of systemic chemotherapeutic or anti-mitotic medications within three months of study start date due to the possibility of interference with bone marrow procedure
Any patients with a history of or with active malignancy
Use of steroids within three months of the study start date, as this would suggest a highly active disease state
History of cirrhosis due to increased risk of central nervous system (CNS) infection
Poorly controlled hypertension because of increased risk for stroke or CNS hemorrhage. Specifically, any patient with a systolic blood pressure value of ≥ 145 mm/Hg or a diastolic blood pressure value of ≥ 95 mm/Hg will be excluded from study participation.
History of thyroid disorders or other endocrine disorders because of hormone influence on cell growth
History of central nervous system infection or immunodeficiency syndromes due to increased risk of CNS infection
Preexisting blood disease (such as bone marrow hypoplasia, leukopenia, thrombocytopenia, or significant anemia) due to invasive nature of bone-marrow aspiration
Previous or current history of a coagulation disorder
Any metal in the body, which is contraindicated for MRI studies
Allergy to any of the antibiotics used in this study, e.g. tobramycin, vancomycin, or gentamicin
Patients with alcohol or other substance abuse problems
Other major disease that, in the opinion of the Principal Investigator, would preclude participation in the study
Patients with HBV, HCV, syphilis, HIV-1, or HIV-2.
Any evidence of significant cognitive dysfunction based on a screening history and physical examination because it would preclude giving a truly informed consent
Patients who are enrolled in another clinical trial for MS treatment or who have received any study drug/biologics within the last 6 months