In patients with multiple sclerosis, the immune system incorrectly reacts to myelin, the insulating sheath that covers nerves in the brain, optic nerve, and spinal cord, and then destroys it, disrupting normal transmission of electrical signals. This results in symptoms from partial limb numbness to paralysis and blindness.
The new human trial is part of a collaboration between Northwestern’s Feinberg School, University Hospital Zurich in Switzerland and University Medical Center Hamburg-Eppendorf in Germany.
During the phase 1 clinical trial, nine MS patients in Hamburg, Germany were treated by introducing billions of myelin antigens into their bodies through their own white blood cells, reprogramming the immune system to treat myelin as harmless.
By filtering out white blood cells from each patient, researchers coupled the cells with myelin antigens through a complex GMP manufacturing process before reintroducing them into the blood stream intravenously. With the large number of myelin antigens suddenly present, immune cells began to recognize myelin as safe and cease attacking it.
The primary goal of the study was to determine the safety of the treatment. It showed that there were no adverse effects in MS patients with the injection of up to 3 billion of these specially treated white blood cells. Most significantly, the treatment did not affect the immune system’s response to other cells and real pathogens, unlike current MS treatments that suppress the entire immune system, making patients more susceptible to other infections.
To demonstrate this, researchers tested the patients’ immune response to tetanus one month later. All patients had received tetanus shots in their lifetime, and their immune systems continued to show strong, normal response to tetanus after the treatment. The immune effect of the blood cell treatment was thus only in response to myelin.
The size of the study was too small to statistically prove the treatment’s success in preventing the progression of MS. However, the phase 1 safety trial lays the groundwork for a phase 2 trial, which has already been approved in Switzerland and awaits the $1.5 million necessary to begin the study.
The hope, after further testing of this treatment, is that it may be applied not only to MS patients but also to a wide range of other autoimmune diseases and allergic reactions by coupling appropriate antigens to the cells. The treatment may also be tested with the use of nanoparticles to carry the antigens rather than the more difficult and costly procedure using white blood cells, an option which is currently in preclinical development.
Source: Northwestern University Press Release 6/4/2013 https://www.northwestern.edu/newscenter/stories/2013/06/big-multiple-sclerosis-breakthrough.html