Insights Blog

Stem Cell Therapy: A Medical Breakthrough—But How Do We Keep It Safe?​

Stem Cell Therapy: A Medical Breakthrough—But How Do We Keep It Safe?​

Stem Cell Therapy: A Medical Breakthrough—But How Do We Keep It Safe?​

Insights from the Network

May 2025

By Jon Draper, Ph.D., VP, Research & Training, Stem Cell Network
Dr. Jon Draper
Dr. Ivana Barbaric
Dr. Nika Shakiba

 

In this month’s Insights blog, SCN’s Dr. Jon Draper sits down with co-authors Dr. Ivana Barbaric and Dr. Nika Shakiba to reflect on their recently published paper in Cell Stem Cell. Together, they explore the key findings, the broader implications for stem cell science, and their excitement about how this research could shape the future of the field.

Introduction: Advancing Stem Cell Therapies with an Eye on Safety

Stem cell therapies – particularly those using human pluripotent stem cells (hPSCs) – are one of the most exciting frontiers in modern medicine. These cells can turn into any cell type in the body, making them ideal for regenerating damaged tissues, restoring lost function, and potentially curing chronic or life-threatening diseases.

But there may be a catch. As these powerful cells are grown and prepared in the lab, they can pick up genetic mutations. Most of these mutations are harmless. But others? Researchers are working hard to know if they increase the risk of tumor formation or affect how well the treatment works.

In May 2024, a major workshop in the UK brought together experts from stem cell biology, cancer research, genetics, bioengineering, industry representatives, research funders and regulatory science to tackle this very question:

How do we make stem cell therapies safer for patients?

The outcome was a detailed roadmap – published as an article in Cell Stem Cell in April 2025 – calling for new ways to detect, assess, and manage risky genetic variants in hPSCs. The authors outlined practical strategies, from better screening tools to smarter biomanufacturing protocols and “fail-safe” genetic systems to stop cells from causing problems.

UK Regenerative Medicine Platform (UKRMP) Genetic Stability Workshop group photo
UK Regenerative Medicine Platform (UKRMP) Genetic Stability Workshop​​
Q&A with the Experts:

Why are genetic variants in stem cells such a big deal? Aren’t mutations a normal part of biology? (Ivana)​

Indeed, mutations happen all the time in our cells. While many of these have no significant effect, some mutations, like those seen in cancer, can cause cells to divide and grow uncontrollably. Therefore, understanding the risks associated with these mutations is crucial to ensure patient safety.

Is this just a theoretical concern, or have there been real-world problems? (Nika)

The clinical results to date are quite positive, with over 1,200 patients having received hPSC-derived cell therapies, there has only been one confirmed case of a tumour forming. However, we need to be cautiously optimistic in interpreting these results. As the clinical demand for these therapies increases, the need to make more and more high-quality batches of cells will also increase. With this comes an increased risk that we could see emerging genetic variants. Therefore, it’s important that we anticipate these challenges now so that we can stay a step ahead of them.

What are you proposing to make these therapies safer? (Ivana)

To make these therapies safer, we are focusing on several key areas. First, we propose having a centralized database where researchers can share and track information about the mutations they find in the batches of stem cells we are working with. This way, we can all learn from each other’s findings and identify potential risks faster. Second, we need to develop improved tools to test whether a mutation affects safety or function, as our current predictions are based on incomplete information. Third, we think that the way we grow stem cells in the lab can be improved, and this may lead to fewer mutations forming. Finally, beyond preventing mutations, we are also exploring building-in safety mechanisms into the cells themselves – a “kill switch” that may help eliminate risky cells if needed.

How close are we to implementing these solutions? (Nika)​

There’s a lot of momentum already that we can build on towards implementing solutions. We’ve already seen exciting advancements in AI models to predict the effect of harmful mutations in diseases. At the same time, labs around the world have created protocols for making lab-grown organoids that mimic the tissues in our body. These are powerful tools we can wield to predict how mutations in hPSC-derived cell therapies will impact their safety in the body. There are also safety technologies we are looking to implement, such as kill switches we can embed in cell therapies so that we can shut them off if we need to. These are all technologies that provide us a running start to get ahead of the mutation risk in hPSC-derived cell therapies, but we also need to keep the momentum going towards developing global systems for data sharing and standardized testing, which are in their early stages. To fully implement all of these solutions, we need collaboration between scientists, regulators, and industry partners.

Should patients be worried? Or excited? (Ivana & Nika)

Definitely excited! Stem cell therapies are already transforming lives, and the risk is very low when these therapies are done carefully and under regulation. We are working to ensure long-term safety and trust in these amazing technologies. We are hoping this call to action will rally interdisciplinary experts to ensure we build safe and robust pipelines for hPSC-derived cell therapies now so that we can see their clinical impact take off as they move through clinical trials and into our healthcare systems.

Final Thoughts​

Stem cell therapies that start with human pluripotent stem cells have the power to change the way we treat disease. Thanks to global efforts like the one led by Dr. Ivana Barbaric and her colleagues, the field is moving toward a future where we can minimize risks and develop safer, more effective therapies using hPSCs for life threatening diseases.

If you are a researcher working with hPSCs or developing stem cell-based therapies, I encourage you to read the roadmap published in Cell Stem Cell and consider how its recommendations can be applied in your own lab, institution, or clinical translation efforts. Advancing safety isn’t just a scientific imperative—it’s a shared responsibility that will shape the future of regenerative medicine.