How one study is changing the way cell therapy is delivered for premature babies

Research into a chronic lung disease in premature babies is leading Hudson Institute researchers to develop new procedures on how to treat infants with cell-based therapies.

Associate Professor Rebecca Lim, Cell Therapy Researcher, Hudson Institute of Medical Research
A/Prof Rebecca Lim

Key points

  • Cell therapy works by treating, regrowing or replacing diseased or dysfunctional cells with healthy, functioning ones at prescribed doses
  • This study found that the method of infusing the cells in premature babies affects how many cells they give
  • This is the first time a protocol or procedure for cell infusion has been created for premature babies, paving the way for future cell therapy treatment.

The discovery came as researchers were investigating how to deliver amnion cells, a cutting-edge cell therapy for the treatment of the chronic lung disease in premature babies called bronchopulmonary dysplasia (BPD). This promising therapy involves slowly infusing a small volume of amnion cells to preterm babies at high risk of BPD.

During the clinical study PhD candidate Dr Liz Baker, from the Royal Women’s Hospital, showed for the first time that the simple changes to infusion methods can significantly alter the dose of cells delivered to extremely premature infants.

What happened during the cell therapy trial

Dr Elizabeth Baker, Neonatal Research Fellow and Cell Therapy Researcher, The Royal Women's Hospital
Dr Elizabeth Baker

During the trial Dr Baker noticed something unusual. “As the Primary Investigator on this early phase safety study, I stand at the bedside and watch the infusion like a hawk. I could see the cells settling and accumulating in the equipment that we use to deliver the therapy to babies. I had to ask myself the question, “What influence does that have on the dose the babies are actually getting?”

 

This prompted the research team to pause the trial and examine how many of the cells were actually being delivered and how they could change this. They were surprised to learn that only around 20 per cent of the intended cell dosage was actually getting through.

The study found that simple measures—small-volume IV lines, increased albumin concentration and a vertically positioned syringe and IV line—improved cell delivery to 99 per cent of the intended dose. These findings have been published in Stem Cells Translational Medicine.

Hudson Institute researcher Associate Professor Rebecca Lim said, “In hindsight we shouldn’t have been surprised by the findingspremature infants are not simply smaller humans. There are unique challenges when dealing with this patient population. They are tiny, their organs haven’t fully matured, the equipment is tiny and every single intervention can significantly affect the infant so we have to be extremely careful.”

How will this research move cell therapy in newborns forward?

Cell therapy is neonatal medicine’s new frontier. While the challenges of translating research discoveries are apparent, a simple yet fundamental hurdle had previously been overlooked—a protocol for the intravenous delivery of cell therapy in newborns.

The research team at Hudson Institute aims to develop protocols for intravenous (IV) delivery of cell therapies to newborns, to ensure dosages of cells in cell-based therapies are correct—this is the first time that a cell infusion protocol has been specifically engineered and evaluated for newborns.

“Good science always stirs the pot a bit—I am proud that our team had the courage to ask the tough questions about the delivery method and our findings have implications for all future research in the cell therapy space, in particular those involving infants. Undoubtedly, it has implications for past cell therapy trials in infants, including our own,” said A/Prof Rebecca Lim.

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