C34 triols in the treatment or prevention of inflammatory disorders in children and adults with a focus on necrotizing enterocolitis

Unmet Need
Necrotizing enterocolitis (NEC) is the leading cause of death and disability in premature infants. The disease is characterized by a bacterial infection in the intestines that quickly progresses to organ death if untreated. Current treatment of NEC entails surgically removing the diseased parts of the intestine. However, this procedure leads to death in half of all patients and complications later in life. Poor outcomes in treating NEC has led Hopkins researchers to investigate non-invasive treatments of this disease.
 
Technology Overview 
Recently, Hopkins researchers have identified that the premature infant intestine contains a “switch” called Toll Like Receptor 4 (or TLR4), which is “turned on” in premature babies by bacteria and leads to NEC. Using computer assisted drug design, Hopkins researchers recently identified a novel molecule, called “C34”, which can safely “shut off” the TLR4 switch after oral administration. The invention disclosed herein presents additional C34-related structures that show efficacy in relevant mouse models for the treatment or prevention of inflammatory disorders with a focus on NEC.
 
Stage of Development
Hopkins researchers have shown that the oral administration of a C34 analog successfully “shuts off” TLR4 in well validated mice and piglet models.
 
Publications
Hackman et al., Journal of Pediatric Surgery, 2018.

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