Wednesday, April 27, 2005

Autism Spectrum Disorder experimental drug

An experimental immunoglobulin drug called Oralgam may help gastrointestinal dysfunction in kids with autism.
The immunoglobulin drug is FDA approved for intravenous use in immunodeficiency disorders where patients have lower-than-normal levels of immunoglobulin (a protein produced by plasma cells and lymphocytes). It is also used in chronic lymphoid leukemia and chronic idiopathic purpura. It has only been FDA approved for clinical trials administering it orally to treat gastrointestinal dysfunction in autism.
According to Cameron Durrant, M.D., president of PediaMed (Oralgam's manufacturer), between 20 percent and 50 percent of autistic children have gastrointestinal dysfunction. This includes diarrhea, constipation, bloating, and/or abdominal cramping. He says this is a problem that needs to be addressed and hasn't been until now.
In a pilot phase 1/phase 2 study, 12 children with autism were given Oralgam daily for eight weeks. Response to treatment was measured using a Gastrointestinal Severity Index (GSI). The scale goes from zero to 15, with numbers increasing with gastrointestinal symptom severity. Before treatment, the range of participants' GI went from seven to 13. After four weeks, the range was between three and seven, and after eight weeks, the range was from two and six.
Side effects associated with Oralgam include nausea and some abdominal discomfort, but Dr. Durrant says it is difficult to separate that from the condition itself.
Researchers are unsure why autistic children tend to have GI problems, but it may be that they have lower than normal levels of immunoglobulin in their gut, causing localized inflammation. Some children benefit from exclusion diets, where gluten or casein is eliminated, for example, but there is no approved treatment that works in the majority of children.
Dr. Durrant tells Ivanhoe, "We're very excited about the potential with this project. We think it meets a very important unmet need, but it is still early days with the study, and we don't want to claim anything that isn’t proven. We don't want to give any false or misleading hope to families that have children with this condition."
Dr. Durrant adds if proven effective and approved, Oralgam could be used to treat other GI or autoimmune conditions.
A second phase 2 study has recently begun of 120 patients. It will include a longer period of active treatment. Dr. Durrant says 15 hospital units across the country are awaiting approval for involvement, including UCLA, Cincinnati Children's Hospital, and Indiana University.