JDRFNewsPromising stem cell therapy for type 1 given green light to progress

Promising stem cell therapy for type 1 given green light to progress

Posted on 06 July 2022

The research trial has restarted after a pause and will now be extended to a small group of new participants.

An image of pancreatic cells grown from stem cells stained blue and green.
A picture of beta cells made in a lab from stem cells, which can be grown into any cell.

VX-880 is an exciting potential treatment for type 1 diabetes involving transplanting lab grown insulin-producing beta cells into people with T1D. This allows the body to produce its own insulin again, reducing the need for insulin injections.

Testing stem cells in people

A clinical trial is currently underway in the United States to test the therapy in participants with type 1. Read more about what clinical trials are on this page of our research section.

All the participants recruited for the trial of VX-880 have impaired hypoglycemic awareness, meaning they struggle to tell when they are having a hypo, which results in severe hypoglycemia.

Promising results so far

So far, two people have been given half the maximum planned dose of new beta cells. They have all shown a significant improvement in their diabetes management, measured by more time in range and less insulin needed.

One participant has even become insulin-independent, meaning they can now produce all their required insulin themselves and no longer need to inject.

Read more about the first results of the trial in our news piece.

The clinical trial was paused…

This improvement with just half the intended dose led the FDA – the organisation that licenses treatments in the US – to put the trial on hold because they did not see enough evidence for increasing the dose.

…and has now restarted again!

The FDA has just lifted this hold on VX-880, allowing the clinical trial to continue. This means the study can recruit more participants to receive the beta cell transplant, this time at the full dose.

The safety and effectiveness of the full dose of VX-880 will first be assessed in five new participants before being extended to a handful more people.

Where did the beta cells come from?

The beta cells used in VX-880 are made in a lab by scientists from stem cells. Making the cells in this way means no donors are needed to donate their healthy beta cells. The lack of available donors has previously limited the number of people with type 1 who could benefit from transplants, which will no longer be an issue if the trial of VX-880 is successful.

Watch this video to hear from scientists who are growing beta cells in their lab.

Research funded by JDRF

Since 2000, we have been funding a scientist called Doug Melton to make beta cells from stem cells. He succeeded and we invested more money in his company Semma to turn these cells into a cure for type 1. Another company called Vertex took over Semma, and Doug is now working at Vertex to bring his original research closer to a functional cure for type 1. Read Doug’s inspiring story about why and how he developed VX-880.

It’s thanks to our incredible supporters that we were able to fund this initial research. Find out how you can support us.

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