Researchers Identify Promising Therapy for Rare Immune Disorder

Thursday, July 23, 2015

NIAID Scientists and Colleagues Determine Disorder’s Cause

Researchers from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and the Cincinnati Children’s Hospital Medical Center report on a promising therapy for people with LRBA deficiency, a rare immune disorder that lacks effective treatments and is caused by mutations in the LRBA gene. The researchers found that the drug abatacept, which is FDA-approved for treating rheumatoid arthritis, may be an effective long-term therapy for LRBA deficiency. Furthermore, the team has discovered that LRBA protein plays a role in limiting the activity of immune cells.