Taking cell therapies from the lab to the clinic

At the end of last year, we attended the ‘Advancing Cell Therapy for Type 1 Diabetes: Overcoming Barriers to Developing Clinical Trials in Europe’ event in Belgium.

Organised to bring together key stakeholders including people with lived experience, health care professionals, researchers and decision makers from across Europe, this one-day conference looked at different, innovative routes and technologies used to develop cell therapies for type 1 diabetes (T1D), and what it will take to make these advances a reality. These types of cell therapies for T1D aim to replace the damaged, insulin-producing cells in the pancreas with cells from other sources, which would remove the need for insulin therapy.

1 – Lived experience is essential to understand attitudes towards cell therapies

It’s vital that the perspectives, opinions, and concerns of everyone affected by T1D remain at the heart of all T1D research, and studying cell therapies is no exception.

To help understand these opinions and perspectives better, three Italian-based institutes (The Vita-Salute San Raffaele University, the Diabetes Research Institute of the IRCCS San Raffaele Hospital and Diabetes Italia) have launched the Diabetes Cells Citizens (DICCI) project.

This citizen science initiative will gather opinions and data on cell therapies from the T1D community. This information can then be used to improve the care of people living with T1D and help translate breakthroughs from the lab to the clinic.

Find out more about how you can get involved in T1D research.

2 – Multiple cell therapy routes are being explored

We heard talks on some of the different routes, and different technologies, being explored in the cell therapy area.

One method involves taking stem cells, a type of cell that can develop into other cell types, and turning them into the different cells found in the pancreas in clusters called islets. These islets include the beta cells, which make insulin, and alpha cells, which make glucagon. Both of these hormones are needed to manage blood glucose levels. We also heard about research using islets from pigs as a source of replacement cells for people with T1D.

Using cells from alternative sources like these would allow cells to be produced in the large quantities needed for a sustainable therapy. At the moment, islet transplants are received from organ donors, and the need far exceeds the available donations.

We also heard about different technologies being developed to ‘hide’ cells from the immune system. These technologies would protect the cells from further immune attack and could mean that drugs to limit the immune system (immunosuppressants) would not be needed.

Currently, people who receive islet transplants from organ donors need to take immunosuppressants because their body recognises that the transplanted cells are from a different person. Your immune system then thinks these cells shouldn’t be there and may reject the transplant. Immunosuppressants help to prevent this, but their use comes with an increased risk of infection and cancer. Protecting cells by hiding them, or genetically modifying them to stop this rejection, could mean that lifelong immunosuppression wouldn’t be needed.

3 – Collaboration and communication are key to moving forward

Collaboration and working across different scientific areas are essential parts of the research process; bringing in experts from different specialisms provides varied viewpoints and expertise, which can help to drive projects forward.

We heard how collaboration with engineers and manufacturing experts could improve the ways in which new islets can be made from stem cells in high quantities. Making sure this process is efficient in delivering only the sorts of cells suitable for transplantation will be vital to enable this therapy to move into the clinic.

We also heard how the agencies that license new cell therapies can support researchers throughout the research process, not just during the regulatory and approval stages. For example, the European Medicines Agency can help researchers by providing scientific advice and protocol support. The MHRA (Medicines and Healthcare products Regulatory Agency) Innovation Office in the UK provides similar support for researchers. Engaging with these agencies earlier streamlines the research process, meaning that potential new therapies could move out of the lab and into the clinic sooner.

The talks and discussions were an insight into the incredible research that is moving us towards cures for T1D. It is inspiring to see how these experts are coming together to share knowledge and push boundaries to overcome the challenges ahead. With continued support, the future for cell therapies in T1D looks exciting, and we cannot wait to see how these research breakthroughs will transform what it means to live with T1D.