A Cell Therapy for Necrotising Enterocolitis

Project: Research

Project Description

This Application is about developing a cell therapy for necrotising enterocolitis (NEC) - the most common gastrointestinal emergency in premature babies. The proposed work arises principally from two programs of research at The Ritchie Centre, both targeting diseases of the preterm infant: a program of cell therapy/regenerative medicine research focussed on amnion (placental) cells, and a program of research in anti-inflammatory therapeutics in NEC. NEC predominantly affects babies of very low birthweight and is most common in very premature babies, affecting about 1 in 10. Of these babies, as many as 30% will die. Of those that survive, about 1 in 4 will develop brain injury due to overwhelming inflammation, leading to lifelong disability. There is currently no targeted cure for NEC. Instead, "treatment" is purely supportive, resting the baby's gut and empirically treating with antibiotics. Not surprisingly, such non-targeted care fails in almost half of cases.

Here we propose a potentially game-changing approach, namely the use of a cell-based therapy - amnion epithelial cells (hAECs) - to mitigate the gut injury associated with overwhelming inflammation in NEC and to accelerate gut repair by stimulating replication of the gut's own endogenous stem cells. We will assess the efficacy of hAECs and their nano sized extracellular vesicle products, called exosomes, in our mouse model of NEC. If successful, the results will set the stage for an early phase clinical trial. We have assembled arguably the world's leading team of experts in the different components of biology required for this work: cell and exosome therapy, innate immunology, gut stem cell biology, and perinatal medicine
Short titleNecrotising Enterocolitis
StatusActive
Effective start/end date1/01/1831/12/20

Funding

  • National Health & Medical Research Council (NHMRC): AUD659,428.00