The Institute has been studying the use of a chemotherapy drug called methotrexate to attack immune cells in the brain. The preliminary findings show patients who were progressively developing more symptoms, actually
What are the most common treatments for MS?
Approved disease-modifying therapies include Betaseron®, Avonex®, Rebif®, Novantrone®, and Copaxone®. These therapies work on the peripheral immune system, but do not act inside the central nervous system. Several other treatments are being clinically tested at locations around the country, including the methotrexate study at the MidAmerica Neuroscience Research Foundation.
The methotrexate study is a different type of treatment because it works inside the blood-brain barrier inside the central nervous system.
What is the blood-brain barrier and what does it mean to people with MS?
A century ago, researchers discovered if they injected dye into the bloodstream, it would cause all the tissues of the body except the brain and spinal cord to change color. They now understand the blood-brain barrier (BBB) protects the brain against foreign substances by keeping materials in the blood from entering the brain. The BBB creates a problem in treating MS because drugs do not reach abnormal immune cells inside the brain. As long as the treatment does not penetrate the BBB, the disease will have enough cells to regenerate even after treatment has wiped out the abnormal cells in the rest of the body.
Doctors treating cancer first discovered some materials are able cross the BBB to attack disease cells in the brain. They have used the chemotherapy drug methotrexate to cross the BBB. Since 1997, neurologists at the Institute have been testing the use of methotrexate to cross the blood-brain barrier in MS patients.
How does methotrexate work?
The scientists theorize that Methotrexate attacks the MS disease at its primary site, in the CNS, by suppressing the MS inflammatory process. Results from the study show there were significant reductions in inflammatory markers. The Foundation is also exploring other possible mechanisms of action of the therapy.
What kind of studies is the Foundation doing to test the effectiveness of methotrexate in treating MS?
In 1997, Foundation founder Dr. Vernon Rowe learned about methotrexate’s use in cancer treatment and theorized it would work in MS treatment as well. That year, Rowe set up the first of three drug studies to test the safety and effectiveness of methotrexate treatment with Leucovorin rescue. Interim analysis in late 2003 of the third study shows the treatment holds promise for people with progressing MS.
What further research does the Foundation plan to do?
The Foundation hopes to begin a larger controlled study in multiple locations across the country, in consultation with the FDA. This is the first step toward gaining FDA approval so that this therapy can be made available to all MS patients.
The Foundation also is studying the relationship between sleep disorders and MS through its sleep laboratory. Many MS patients suffer from extreme fatigue. Preliminary findings suggest sleep disorders are present in 49 percent of MS patients, much higher than the general population. The Foundation is planning further studies to test whether treatment of sleep disorders could reduce fatigue in MS patients.
