VX-880 clinical trial of lab-grown islet cells with immunosuppressants

What is the VX-880 clinical trial?

In the trial, researchers give people with type 1 diabetes a dose of islet cells (containing insulin-making beta cells) via a plastic tube into a vein that goes to the liver. The participants also need to take immunosuppressant drugs to avoid the immune system destroying the new islet cells. After receiving VX-880, the participants are monitored for up to five years.

Because it is the first time these cells have been given to humans, the clinical trial began slowly, with people treated one by one to ensure the treatment was safe. As more data becomes available, the trial continues to expand to enrol more participants.

Participants free from taking insulin

Vertex presented the latest data from the VX-880 clinical trial at the American Diabetes Association’s 2024 conference. They reported that 12 people with type 1 have now received the full dose of the islets.

Dr Piotr Witkowski, one of the researchers on the study, said: “The data we’ve seen to-date in this study are extremely exciting. Stem cell-derived islets regulate blood glucose control as well as natural human islets. The marked improvements seen…have the potential to fundamentally change the treatment landscape for T1D and alleviate the significant burden this disease carries for patients.”

The transplanted cells were accepted by all 12 participants’ bodies and began making insulin in response to glucose within 90 days. The participants also achieved HbA1c levels of below 7.0%, time-in-range above 70%, and no longer experienced any severe hypos. In addition, 11 out of the 12 patients either now require less insulin or don’t need to take any at all, achieving what’s known as insulin independence.

What are the next steps?

Dr Carmen Bozic, Chief Medical Officer at Vertex, said: “These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for T1D. As we plan toward pivotal development, we are pleased to have secured regulatory approval to expand study enrolment and look forward to advancing this programme for patients who have long awaited a transformative therapy.”

Is the VX-880 islet cell treatment safe?

One of the aims of this clinical trial is to assess how safe the VX-880 therapy is. This is why so few participants are involved and why they are being so closely monitored. So far, the trial has not led to serious adverse effects. The most common symptoms people had include dehydration, diarrhoea, low magnesium levels and rashes.

Immunosuppressant drugs have the potential to cause anaemia, reduced kidney function and increased risk of cancer and infections. This is because immunosuppressants suppress the beneficial parts of the immune system as well as the part that attacks beta cells in type 1. International scientists are developing other ways to keep the lab-grown islets safe from the immune system to remove the need for immunosuppressants.

Who can take part in this clinical trial?

The participants in the VX-880 clinical trial are all people with type 1 who experience severe hypos and hypoglycaemia unawareness. This is because the risks and lifestyle adjustments involved in taking immunosuppressants outweigh the benefits of the exploratory therapy for healthier people with type 1.

The people taking part also need to be either blood type A or AB. As the treatment develops, the researchers hope to grow islet cells that are suitable for people of all blood types.

The current total of participants needed is 37 people, but this may increase as more results come in. Find out if you’re eligible to take part in the VX-880 clinical trial.

Where is the VX-880 clinical trial taking place?

There are currently nine trial sites throughout the US, and eight across Canada, Norway, Switzerland and the Netherlands. This is due to expand to more sites and countries.