Intrathecal Gammaglobulin update. While we are still not in a position to assess the effectiveness of intrathecal gamma globulin as treatment, ALS TDF believes that there is sufficient merit to the approach to warrant substantial and immediate investigation. Scott of ALS-TDF has traveled to Puerto Rico to interview patients who have received the treatment and collect independent information. Gamma Globulin is a very complex treatment and its exact mechanism of action is unknown. There are several possible ways it might impact ALS including deactivation of certain receptors on microglia as well as modifying antibody signaling.
Microglia are a subcomponent of the immune system located in the brains that respond to injury.
There is evidence that they are activated in ALS and that it might be involved in the pathology of the disease. Gamma Globulin also can have anti-inflammatory effects. It should be noted that several clinical trials have been done on the use of intravenous gamma globulin in ALS and that they have found no effect. Because gamma globulin does not cross the blood brain barrier efficiently it is possible that intrathecal delivery shows a benefit where the earlier trials did not. We are working quickly to try to answer these questions.