Multiple sclerosis (MS) is a frequently debilitating disease for which there is no cure. In the early stages of MS, the body develops an abnormal immune response against the central nervous system that damages the protective myelin sheath on nerve cell processes. Later stages are marked by damage to, and loss of, neurons. The body has mechanisms to repair this damage; however, in MS they cannot keep up with the damage that is continually occurring. Mesenchymal stem cells (MSCs) are primitive cells in the bone marrow (the central part of bones that gives rise to blood cells) that have immunologic effects and the ability to promote tissue repair, both of which may be beneficial in MS. In laboratory studies, MSCs have been able to turn into neuron-like cells, encourage other immature cells to become neuron-like, and decrease the activity of specific types of immune cells which are overactive in MS. These characteristics of MSCs have focused attention on these cells as a possible way to reduce immune-mediated damage and increase neural repair in MS.
This proposal details a Phase I study investigating feasibility, safety, and tolerability of MSC transplantation in 24 MS patients. Participants will meet the following criteria: (1) Age 18-55; (2) a relapsing form of MS; (3) Have moderate to severe disability but are still able to walk; (4) Are on, have failed, or have refused FDA-approved therapies; and (5) Have documented evidence of relapse, worsening impairment, or active MRI within the last 2 years. Bone marrow will be removed from the participant's hip, and MSCs will be purified from the marrow. These cells will be grown in a laboratory until a sufficient cell number is achieved. The cells will then be given back to the participant intravenously. The participants will be monitored very closely for 6 months after MSC administration, including physical exams, blood work, and brain MRI scans, to determine how safe the procedure is and how well it is tolerated.
MS disease status will be monitored by neurologic exam, vision testing, MRI, and optical coherence tomography prior to, and for 6 months after, MSC administration to evaluate whether there is indication of benefit on MS disease activity or severity. This evaluation will be undertaken in only a preliminary way as the main focus of the trial is to evaluate the safety of the procedure. If this study demonstrates that MSC transplantation is safe in MS patients, further studies will be developed to more definitively assess the benefit of MSC transplantation in MS.