Advancing Neonatal Cell Therapy Research

Newborn Cell Therapies Group at Monash University in Melbourne, Australia, is a world leader in the research and translation of cell therapies for newborn health conditions. The group consist of clinicians, scientists, research staff and students working together to find innovative cell therapy solutions for complications associated with preterm and high-risk birth.

Their specific interest is in stem cells and stem cell like cells derived from gestational tissues. These tissues are generally discarded after birth. Gestational tissues including placenta, its membranes (amnion and chorion), umbilical cord blood and cord tissue (wharton jelly) are rich sources of cells that have many potentially beneficial properties, including regenerative properties and cells with pluripotent potential. Some of these cell therapies are already in clinical use for a variety of hematological and oncological conditions (cord blood derived hematopoietic stem cells), but we are now evaluating their use for regenerative medicine purposes especially for conditions related to preterm and other high-risk births.

The cells display a number of paracrine and trophic effects (effects away from the site of cell administration) on injured and vulnerable cells of vital organs like the brain, heart and lungs in preclinical models of newborn health conditions. The premise is that similar effects may be seen when these therapies translated in babies born after preterm and other causes of high-risk birth. Translation requires carefully stepping through early phase trials (establishing safety) to large trials evaluating efficacy, before these therapies could be considered therapeutic for newborn infants.

The group has a very strong track record of human translation already. Monash Newborn clinicians, along with colleagues from The Ritchie Centre, Hudson Institute of Medical Research, conducted the first-in-human trial of allogeneic (cells from a healthy donor) placental amnion epithelial cells for infants with the preterm lung disease bronchopulmonary dysplasia. They are now involved in a dose escalation study of human amnion epithelial cells for the prevention of bronchopulmonary disease.

Cord blood derived cell therapies is a special interest of the group with a number of pre-clinical studies conducted by the group showing benefit in perinatal brain injury models. Cord blood and cord tissue derived therapies are increasingly being evaluated in early phase studies for newborn health conditions. The group is conducting two world–first studies using cord blood derived cells. These include autologous (babies own cord blood derived cells) safety and feasibility studies in extremely preterm infants (CORD-SaFe study) and in infants with fetal stroke (ACTRN12622000662763). While these trials are at an early phase of investigation, the group is confident that they will be able to progress a number of these therapies to larger trials (randomised controlled trials) evaluating efficacy in the near future.

The group prides itself with strong consumer engagement in the design of all its studies. The involvement of parents with lived experiences of preterm or high-risk birth is critical to ensure that studies planned are going to be acceptable to parents of these vulnerable babies with meaningful outcomes being assessed after cell therapy administration.

Newborn Cell Therapies group’s work is supported by funding from a number of national bodies and philanthropic organisations including National Health and Medical Research Council of Australia, Cerebral Palsy Alliance Research Foundation, Inner Wheel Australia, Lions Cord Blood Foundation, and Monash Health Foundation.

The group most recently announced a $50,000 grant from Lions Cord Blood Foundation in August 2022. The group will receive the money over 2 years to support the neonatal cell therapy research.

The group is excited to announce the First Ever Neonatal Cell Therapy Symposium, which be held later this year in Melbourne, Australia. You can follow the Newborn Cell Therapies group’s activities on Social Media via their Twitter account, @Neo_CellTherapy.

REFERENCE

Lions Foundation provides support to neonatal cell therapy research. News release. Monash Unviersity. August 25, 2022. https://www.monash.edu/medicine/news/latest/2022-articles/lions-foundation-provides-support-to-neonatal-cell-therapy-research