The passive immunization that we are developing is based on the administration of monoclonal antibodies, antibodies specific to a protein called SOD1. A small number of patients possess mutations of this gene. It is believed that administering these antibodies to patients will eliminate the protein's toxic effect.
As we know, the majority of ALS cases are sporadic. Recent, unpublished studies have shown that sporadic patients can acquire malformations of this protein even if there is no mutation in the SOD1 gene. Environmental toxins can cause misfolding of the protein's tertiary structure and create toxicity. The treatment we are developing could be used with patients who do not have a genetic mutation, which represents the vast majority of people suffering from ALS.