An international team has reported results of a small, early clinical trial involving 10 people with relapsing or secondary-progressive MS that tested the feasibility and preliminary safety of using a patient’s own altered blood cells to reduce immune responses against specific components of myelin, the nerve covering that is a key target of immune attacks in MS. They report that the treatment appeared safe and showed signs of reducing immune responses to myelin. Larger, longer trials of this approach would be needed to evaluate its safety and potential to treat multiple sclerosis. The study, by Roland Martin, MD (Institute for Neuroimmunology and Clinical MS Research, Hamburg and University Hospital Zürick) and colleagues including Stephen D. Miller, PhD (Northwestern University, Chicago), was published in the June 5, 2013 issue of Science Translational Medicine
(5, 188ra75 (2013). The trial was largely funded by the German Federal Ministry for Education and Research.
MS involves misdirected immune attacks against the brain and spinal cord. For many years researchers, including many supported by the National MS Society, have been searching for ways to disarm only the immune cells responsible for this attack, leaving the rest of the immune system intact to fight infection. Read more
about research to stop MS immune attacks (.pdf). The major stumbling blocks for this strategy have been that we don’t know the primary targets of MS immune attacks, and that the specific myelin targets are different among people with MS, and can change over time.
Dr. Martin and colleagues launched a clinical trial based on preclinical work by Dr. Miller, a longtime grantee of the Society. The team selected seven components, or peptides, of myelin proteins that have shown high immune reactivity in people with MS. In the laboratory, these peptides are coupled by way of a chemical process to a patient’s carrier cells taken from their blood. For this clinical trial, only people whose immune responses showed reactions to at least one of the myelin peptides were entered into the study.
This phase I trial was designed to test the feasibility and safety of the treatment, and to detect whether any of the doses tested could alter immune cell reactions to the myelin peptides. All told, ten people with relapsing-remitting or secondary-progressive MS were enrolled. Each received a single infusion of their own carrier blood cells chemically coupled with the myelin peptides, and were monitored clinically and with MRI scans for six months.
The team reports that the treatment was well tolerated, and at higher doses appeared to reduce immune reactions to the myelin peptides that were administered. Two participants who had shown high disease activity before the study experienced MS relapses 10 to 16 days after therapy. The investigators note that this is an important safety signal that will require monitoring in any future clinical trials of this approach, but that overall the treatment was well tolerated. A small study like this is not designed to detect the effectiveness of an experimental treatment.
This is the first time this chemically coupled myelin peptide treatment has been tested in people. The team reports that it is a feasible approach to take to larger trials, and that it appeared to be well tolerated. Larger and longer trials will be required to determine the safety and effectiveness of this treatment in people with MS.
about research for MS (.pdf).