Evaluating Autologous Cord Blood Derived Cells for the Treatment of Preterm Infants

This is the second part of an interview with Atul Malhotra, MD, PhD. For the first part, click here. 

Atul Malhotra, MD, PhD

Extremely preterm infants, those who are born after less than 28 weeks of gestation, are vulnerable to serious complications. Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, and his colleagues, are currently investigating the potential of autologous neonatal cell therapy products, derived from these infants’ own cord blood, to help protect them from such complications.

CGTLive® recently spoke with Malhotra to learn more about his lab’s research and the broader field of neonatal cell therapy. In addition to discussing some early positive findings and plans for the future, Malhotra emphasized the need for collaboration in this field and highlighted the upcoming 2024 Neonatal Cell Therapies Symposium, which will be held June 24-25 in Sydney, Australia.

CGTLive: Can you tell us about your lab’s work in the field of neonatal cell therapy?

Atul Malhotra, MD, PhD: Our lab is the Newborn Cell Therapies Lab at Monash University. This is in partnership with Monash Health, which is the health network, along with Hudson Institute of Medical Research, which is a research institute linked to Monash Health and Monash University. [These are] world leaders in some types of cell therapies, specifically umbilical cord blood and cord tissue-derived cell therapies.

The umbilical cord blood is a waste product after the baby is born. Basically, the umbilical cord is a lifeline for babies when they're in the womb, and once the baby is born, the umbilical cord is cut and along with the placenta, it is disposed of. We have been harvesting and harnessing the properties of umbilical cord and tissue for extracting those cells for therapeutic indications. For example, the umbilical cord is a very rich source of stem cells called hematopoietic stem cells (CD34+ cells), and also a number of progenitor cells. We believe that these cells have the potential to repair and protect vital organs and tissues, especially for newborn conditions. We've been studying these cells for more than a decade in the lab and over the last 4 or 5 years, we've been translating a number of these cell therapies derived from umbilical cord blood into the clinic.

For example, we have completed 2 trials on umbilical cord blood in our hospital. One of them relates to extremely preterm infants. These are Infants who are born after less than 28 weeks of gestation, and are generally less than 1 kilo. These are the most fragile and vulnerable babies. Before our study, no one had tested the feasibility of actually collecting and extracting these cord blood derived cells from these extremely preterm infants. For the first time, we have shown that it is feasible in close to 80% of cases to derive enough stem cells and cell therapy options from these babies' own cord blood. Then we concentrate, harvest, and then are able to produce a cell therapeutic option for giving back to the baby. This is called autologous transplantation or autologous administration of cells from the baby's own cord blood. We'll be sharing the results of this early phase trial in the Pediatric Academic Societies Meeting in Toronto in May, but what I can report is that they are definitely feasible and they are shown to be safe.

Along with studying their safety and feasibility, we also looked at an exploration of what happens to the body of those preterm infants once these cells are given. We have some exciting results, which could play an important role in the mechanistic properties of what these cells do once infused into an individual. This is really important because you can study this cell therapy as much as you like in preclinical models, but when you translate them into humans, it's important to actually show what they're doing. So watch this space for exciting work on that.

Additionally, we are now progressing to a randomized control trial, a phase 2/3 trial, where we will be administering autologous cord blood-derived cells to 1 group of babies and the other group will get a placebo. A randomized control trial is the best way to show the efficacy of these cells. We'll be following around 300 of those babies in an international trial, including sites in North America, Asia, and Australia, where we will recruit those infants and follow them up for 2 years. The main outcome we'll be looking at is: “Does this cell therapy derived from the baby's own cord blood improve their chances of survival without disability?" The idea is to protect their brain by giving them a cell therapy derived from their own cord blood at an early stage of life.

Is there anything else you want to share with the audience?

I think neonatal cell therapy is a growing area of research. There are a number of groups working on this. I personally chair a group of neonatal stem cell enthusiasts around Europe and Australia. We know that there's a lot of advances happening in this. But what it also highlights is that it's important for all of us groups working in this area to work together so that we can have the collective push towards moving these therapies into the clinic and testing them through rigorous clinical trials and rigorous preclinical methodology to ensure the patients get the best outcomes. What I can say is that we've been bringing this group of people together in a symposia. We held our first Neonatal Cell Therapies Symposium anywhere in the world in Melbourne in 2022. Very soon, in Sydney in June of 2024, we are hosting our second Neonatal Cell Therapies Symposium, with experts from around the world converging in Sydney, Australia to talk about neonatal cell therapies and how the advances we are making in our own labs can be used collectively to move the field forward. I look forward to engaging with people there. We will be reporting what the advances are in neonatal cell therapies in a journal article soon, as well. I think this space is really exciting. I just want to call to action people who are working in this space to come together to connect with us. You can connect with us through the X platform, or through email, and through the symposia, like I mentioned. We look forward to moving this field of neonatal cell therapies forward.

This transcript has been edited for clarity.

Click here for information about the 2024 Neonatal Cell Therapies Symposium.