Ampion and Rare Inflammatory Pediatric Diseases
Children with a functional or anatomic single ventricle typically undergo a surgical procedure known as a Fontan operation and are at risk of developing challenging post-operative complications such as protein-losing enteropathy (PLE), plastic bronchitis, chylothorax, and heart failure.
Previous studies have used targeted approaches to elucidate the molecular mechanisms underlying Fontan patient pathophysiology, but investigations into inflammation, metabolism, and protein dysregulation in Fontan patients could provide additional insight into causes of the disease and treatment options for these children.
A dysregulated inflammatory response may play a role in the clinical outcomes of Fontan patients. Using in vitro and in silico characterization models related to Fontan patients and post-Fontan complications could provide insight into the mechanisms of disease in this rare condition, and provide potential therapies, including Ampion, for treating Fontan patients.
Kawasaki Disease (KD) is a disease primarily observed in children less than five years of age, characterized by high fever, rash, conjunctivitis, inflammation in the mouth, lips, and throat, inflamed lymph nodes, and inflammation in the blood vessels, which leads to serious complications including coronary artery dilation and aneurysm.
KD is a rare disorder, affecting only 9 to 20 in a 100,000 children under the age of five in the U.S., but it is a leading cause of acquired heart disease. KD is thought to be triggered by infectious agents and to involve an inappropriate stimulation of the innate immune system. During the recent COVID-19 pandemic, a small but significant number of children infected with the SARS-CoV-2 virus have developed a KD-like syndrome suggesting a relationship between the virus and a dysregulated immune response.
Today, the standard therapy for KD is intravenous immunoglobulin (IVIG). While many children undergoing this treatment recover without permanent damage to their blood vessels, between 15 to 20% of these patients don’t respond to IVIG treatment and have a higher incidence of complications, including coronary artery aneurysms.
Concentrations of inflammatory cytokines (e.g., TNFα, IL-1β) have been shown to decrease with IVIG treatment and prostaglandins (specifically PGE2) may play a role in the disease.
Research and development for Ampion use in KD is being explored based on preclinical pharmacology studies which show Ampion reduces inflammatory cytokines (e.g., TNFα, IL-1β) while promoting the production of prostaglandins (e.g., PGE2).
Ampion regulates the immune response
Ampion uses the power of the immune response and is in development to treat osteoarthritis, respiratory illness due to COVID-19, and a wide range of other debilitating and life-threatening inflammatory conditions.