From the Editors — Collaboration Begets Progress: Recent Updates in Umbilical Cord Blood

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Jessica Sue
Junior Associate Editor
Telegraft/ISCT ESP Leadership Development Program
Quality Manager, Sydney Cord Blood Bank, Sydney Children’s Hospitals Network
Junior Associate Editor
ISCT ESP Leadership Development Program

Having worked in cord blood (CB) banking for much of my career and reflecting as we approach 37 years since the first ever CB transplant (6th October 1988), I’ve always been an advocate for its use. CB is collected with minimal to no interruption to the routine birth process and has a low donor attrition rate. Following processing (minimal manipulation in a closed system), CB stem cells are cryopreserved for decades and made available as an off-the-shelf product which, compared to other cell sources, is a benefit to clinicians in certain scenarios.

For example, in a CB transplant, the naivety of the cells lowers the risk of graft-versus-host disease and allows for less stringent matching than donor cells from peripheral blood or bone marrow. These unique characteristics allow CB to be considered as a donor source where other donors are not available, particularly relevant for ethnic minority groups who are under-represented on donor registries. Public CB banks can strategically position collection sites at maternity hospitals with desired demographics and the Sydney Cord Blood Bank successfully did so for a number of years to capture CB collections from Indigenous Australians[1].

Despite challenges around the sustainability of CB banks, a global reduction in CB transplants attributable to known disadvantages of CB as a cell source such as slower engraftment and lower stem cell dose per patient kilogram, together with increased use of alternative therapies (e.g. haploidentical transplants), there is still progress and evolution in the field of CB banking in recent years.

CB Derived Stem Cell Therapy: Aiding Patients with Hereditary Conditions

In November 2024, the FDA approved Regenecyte, an allogeneic umbilical CB derived stem cell therapy for transplantation in patients with inherited or acquired conditions affecting the hematopoietic system[2]. Regenecyte is not the first CB FDA-approved therapy, but is the first under a Biologicals License Application associated with a commercial company (StemCyte) [3], and may contribute to a leverageable regulatory and manufacturing framework for CB in the future, both in the US and worldwide. The following month saw the announcement of the National Cord Blood Network: a consortium of transplant programs in the US that aims to increase access to CB transplantation and optimize practices, with recognition of the existing high quality inventory of CB and potential for advancements in the field[4]. Demonstrated commitment to progress like this indicates CB will continue to be a desirable cell source for treatment of traditional indications, especially for pediatric patients, when an urgent transplant is warranted, and for patients with rare HLA. Investigations into the use of CB in new clinical indications will serve to further increase its therapeutic value: increasing utilization rates may contribute to the future sustainability of the field and see more partnerships in the commercial and research space.

Translational Pathway in Australia

Here in Australia, we have a strong presence in the CB field, with the national network of public CB banks known as AusCord and private CB banking offered from Cell Care[5]. With the use of CB to treat hematological malignancies and disorders, immunodeficiencies and metabolic disorders well established, pathways to the use of CB in new indications (e.g. neurological and neurodevelopmental disorders, autoimmune diseases, and cardiovascular and ischemic diseases) has experienced some challenges. The regulatory pathway (under the Therapeutic Goods Administration; TGA) for CB for investigational conditions is – compared to CB for indicated uses – less defined and, whether this creates hesitancy amongst stakeholders (researchers, clinicians, commercial), there are comparatively less clinical trials in these areas in Australia compared to the US and other countries. This does not negate the ongoing commitment to advancement of CB therapies and access for patients here in Australia, and there have been some recent significant developments. In early 2025, results from the Australian, single center, prospective CORD-SaFe study (phase-1, open-label) were published by researchers from Cerebral Palsy Alliance (CPA), Monash University and Hudson Institute of Medical Research. Following animal modelling that displayed the neuroprotective effects of CB infusion, the study aimed to demonstrate that collection of CB from extremely pre-term infants, and intravenous autologous infusion of the GMP-grade cells, was both feasible (sufficient volume and cellularity was achieved) and safe (no adverse events related to the infusion). With a feasibility study necessary for advancement to assessment of efficacy, further safety and dosing optimization, the authors concluded that the results justified progression to this next phase of the study, known as CORD-CELL[6]. Similarly, Monash Children’s Hospital, in collaboration with Hudson Institute of Medical Research and Monash University, ran a world-first phase-1 trial, titled the STELLAR trial, to investigate the feasibility and safety of CB collection and autologous infusion within the first few weeks of life, in patients suffering prenatal stroke[7].

In addition to the advancements described above, a significant milestone has recently been achieved in Australia for the use of CB for cerebral palsy. In May 2025, CPA and Monash Health reported that they successfully infused an autologous CB product into a 6-year-old patient who had their CB privately stored. This was made possible via the TGA’s Special Access Scheme (SAS), which allows clinicians to apply to the TGA to provide individual patients treatment on compassionate grounds using unapproved therapeutic goods, with each application assessed on a case-by-case basis. Whilst this has the potential to model future utilization of CB in clinical research and improve equity of access for Australian patients it is important to note that researchers were only able to overcome major challenges through a truly ‘multidisciplinary collaboration’[8,9]. Also, it was the concept of collaboration that caught my eye when thinking about this editorial and gave me pause to reflect on how many opportunities for collaboration and connections ISCT provides both regionally and globally for its members, through meetings, committees, training courses and other educational events.

Takeaways for ISCT Members

These opportunities are not limited or defined by discipline, subspeciality in the CGT field, or career stage. The ESP Leadership Development Program is a fantastic example of how ESPs can contribute across a range of committees. ISCT members are given the chance to form networks that transcend their immediate professional circles. In my experience, the benefits are two-fold: there is the ability to collaborate with those individuals and organizations you connect with through ISCT, but also the ability to take the skills and mindset needed to effectively collaborate in all areas of one’s profession: with that collaboration being one of the key factors to treating patients with cell therapies – new and old – safely and effectively.