Scientists in the United States have announced early results from a pioneering trial testing a new stem cell treatment designed to replace insulin-producing beta cells in people with type 1 diabetes. In a small clinical trial, which is still underway, the first person to be given a transplant of new beta cells made from stem cells could reduce the amount of insulin they were injecting by around 90%.
The results have created a buzz, but what does this research mean for you and for the future of type 1 treatments? Here we take a deeper dive into research to replace lost beta cells and this latest news.
Making strides with stem cells
The stem cell therapy research is being led by a biotech company called Vertex. They’re running an early-stage clinical trial in the US testing a new approach to replacing the beta cells that are destroyed by the immune system in type 1 diabetes.
Transplants of cells taken from donor pancreas already exist, called islet transplants. But islet transplants rely on donated pancreas cells, which are in really short supply. So not many people can be given an islet transplant.
To solve the supply issue, scientists have turned to stem cells. Most cells in our body have one particular job to do, but stem cells are different. They can be coaxed into becoming different types of cells, including beta cells.
Researchers at Vertex are testing how well beta cells made in the lab from stem cells work. Their treatment, called VX-880, involves injecting these cells into a vein that sits near the pancreas. Once inside the body, the scientists hope the cells will start making the right amount insulin to keep blood sugar levels in a healthy range, just like real beta cells do.
The Vertex researchers are early on in their initial trial and so far have looked at the effects of VX-880 in just one person. Before the study, the individual – who has lived with type 1 diabetes for 40 years – was taking an average of 34 units of insulin per day. Three months after the transplant they were making their own supply of insulin and had reduced the amount of insulin they were injecting to just three units a day. That's a drop of 91%. Their HbA1c also dropped from 8.6% to 7.2%.
Safety and side effects
Being able to make new beta cells in the lab that can help people with type 1 to make their own insulin again is hugely exciting. We’re fast approaching exactly 100 years since insulin was discovered, on 10th November 1921, and these results are a moment to celebrate and offer up real hope for the day when can put insulin injections and infusions into retirement. But there a few things to keep in mind.
These are early findings from just one person and there’s much more work to do before we can be confident that the treatment is safe and effective. The researchers plan to test 15 more people in this trial to assess the treatment’s safety and work out the right dose to use. Then larger trials, involving hundreds of people, will be needed to test how effective stem cell therapy is at helping to manage blood sugar levels and how long the benefits last for.
The first participant to receive the VX-880 treatment didn’t have any unexpected side effects, but the immune system in people with type 1 diabetes wants to find and destroy beta cells. Because of this, people on the VX-880 trial need to take immunosuppression drugs that blunt the immune system to prevent it from attacking the transplanted cells. Immunosuppression drugs come with risk of serious side effects, like raising the risk of infections and a small but increased risk of certain cancers.
This means there’s a careful balance of risks versus benefits to consider for treatments like VX-880. The researchers are only testing it in people with type 1 diabetes who have regular severe hypos and can’t detect the symptoms of low blood sugars. This can be extremely dangerous. People can lose consciousness and, in extreme cases, go into a coma. For this group of people, the risks of hypos outweigh the risks of immunosuppression.
The quest to keep cells safe
For stem cell therapy to offer hope for everyone with living with type 1 diabetes, we need treatments other than immunosuppression drugs to protect transplanted cells from the immune system. That’s why we’re funding research to develop and test treatments called immunotherapies. Unlike immunosuppression, which affects the whole immune system, immunotherapies work to retrain the specific parts of the immune system responsible for attacking the pancreas in type 1 diabetes. This means they don’t have the same side effects.
Find out more about how immunotherapies work and how we’re driving forward research to make them a reality sooner.
Another possible way to protect beta cells from the immune system’s attack is to transplant them inside a protective barrier. This is called beta cell encapsulation. Barriers are being tested that allow new beta cells to sense blood sugar levels and let in important nutrients they need to survive, while at the same time shielding them from attacking immune cells.
We also know that beta cells scientists make from stem cells in the lab aren’t perfect yet, and don’t work as well as ‘real’ beta cells. But our researchers, like Dr Natasha Hill and Dr Ildem Akerman, are on the case and figuring out how to improve the way we grow beta cells in the lab, so that they’ll make more insulin and respond better to blood sugar levels.
What about type 2 diabetes?
Stem cell therapies at the moment are being tested in people with type 1 diabetes and frequent hypos and hypo unawareness, for whom this pioneering treatment could be life-saving. But as research advances, we hope stem cells-turned-beta cells could also be used to boost beta cell supply in people with type 2 diabetes and help them to make enough of their own insulin to perfectly control blood sugar levels.
Can I take part?
Vertex is only recruiting people to take part in their study in the US and Canada. But if you’re interested in getting involved in research, you can search for studies near you on our take part in research page.