This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 874839 ISLET.

This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 874839 ISLET.

Advanced
Therapies

An advanced therapy uses new medical products that involve gene therapy, cell therapy, and tissue engineering. This is a fast evolving area with ground-breaking potential for the future treatment of a wide-array of diseases and injuries. Delve into the science of these approaches below.

What are advanced therapies?

Evolving scientific understanding of how our bodies work is revolutionising the way we understand and treat disease. New insights into health and disease can come from a better understanding of our genes and how they control different cell behaviours. Or, through a deeper insight into the way stem cells enable us to grow, repair and renew our organs. Advanced therapies use these breakthroughs to develop targeted treatment approaches.

 

Many disease conditions are caused by damage to, or loss of, cells, tissues or organs in the body. Neurological disorders such as multiple sclerosis (MS), Parkinson’s and Huntington’s disease are caused by damage to cells of the nervous system. Whilst Type 1 Diabetes is caused by deterioration of the insulin-producing beta cells in the pancreas. Cell therapies aim to either replace damaged cells or to encourage the body to repair damaged cells itself.

Are there advanced therapies currently available?

There are just a few treatments available to people suffering from a handful of conditions. Other advanced therapies are still under development and are at various stages along the path from basic research to the clinic.

 

The most well-established and widely used stem cell treatment is the transplantation of blood stem cells. This therapy is used to treat diseases and conditions of the blood and immune system, or, to restore the blood system after specific cancers treatments.

 

Since the 1980s, skin stem cells have been used to grow skin grafts for patients with severe burns covering large areas of the body. Only a few clinical centres are able to carry out this treatment and it is usually reserved for patients with life-threatening burns.

 

A new stem-cell-based treatment to repair damage to the cornea (the surface of the eye) after an injury like a chemical burn, has received conditional marketing approval in Europe.

Find out more about current stem cell treatments and clinical trials on Eurostemcell

 

Advanced therapy
for diabetes

There are currently no approved advanced therapies for diabetes. ISLET aims to change this by pioneering an effective cell-therapy for Type 1 diabetes.

 

In diabetes it is the beta cells in the pancreas that are damaged and their loss, in turn, affects the function of the whole organ. By replacing the damaged cells with healthy lab-grown cells we aim for individuals living with diabetes to regain control of their blood glucose levels.

The ISLET cell-therapy for Diabetes 

Pluripotent stem cells are capable of making nearly all of the cells in our bodies, including pancreatic beta cells. By understanding how beta cells are made from these stem cells in early human development we have learnt to re-create this process in the lab. The result is that we can now make lab-grown beta cells, which have the potential to be used in a therapy for Type 1 Diabetes – our first generation therapy

In ISLET, though, we want to go beyond making just beta cells. In the pancreas, beta cells are located within the islets of Langerhans. These islets contain beta cells and glucagon-producing alpha cells – the main blood sugar regulators. The combination of these two cell types produce a more fine-tuned response to blood-sugar levels than beta cells alone. Again, beginning with pluripotent stem cells we are working out how to make functioning islet-like clusters of alpha and beta cells for cell therapy – our next generation therapy.

Bringing advanced cell therapy to patients with Diabetes

Much of the pipeline to patients for advanced therapies is complex and unexplored, creating many challenges to be overcome for their potential to be fulfilled.

We will pioneer this route, overcoming barriers that will bring both advanced therapy for diabetes to people and a way forged for other therapies to follow.

The ISLET consortium has been designed to consist of experts each highly experienced in different parts of the pipeline from basic science to pre-clinical testing and Good Manufacturing Procedure (GMP) manufacturing. Ultimately, this expertise will enable the generation of a cell-therapy product that is ready for regulatory approval and clinical trials by 2025.

Follow the path from lab bench discovery to working therapy in this enthralling graphic comic Hope Beyond Hype

 

 

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