Type 1 diabetes (T1D) is an autoimmune disease in which the immune system mistakenly attacks beta cells. As a result, these insulin-producing cells can’t produce as much as the body needs. This means that people with T1D must administer exogenous insulin, which is insulin made outside the body, for the rest of their lives.  

 What if we could replace dysfunctional beta cells with new insulin-producing cells? You might not have to give yourself as much insulin or any at all — for as long as the cells stay healthy. This is the idea behind cell therapy for T1D.  


Cell therapy for T1D 

Scientists have been working for years to make cell therapy possible. To do that, they must accomplish several challenging tasks:  

  • Obtain or generate insulin-producing cells, 
  • Deliver them safely into your body, 
  • Keep them alive and functioning for as long as possible, and  
  • Protect them from your immune system. 

Thanks to their hard work, we’ve made great progress toward solving these challenges. Today, three promising types of cell therapy are available or in development. Let’s take a closer look at them. 


Lantidra, approved for some adults 

Lantidra, made by CellTrans, is the first cell therapy for T1D approved by the FDA. It is only available to adults with T1D who have severe hypoglycemia. 

Lantidra uses insulin-producing cells from deceased donors, much like organ transplants. The cells are administered into the bloodstream and travel to the liver. The insulin they produce is then carried to other organs, including the pancreas. People who receive Lantidra must take medication to suppress their immune system, so it doesn’t attack the new cells.  

In clinical testing, 30 people received Lantidra. About one-third of them didn’t need insulin for 1–5 years, and another third didn’t need insulin for more than 5 years. However, five participants still had to take insulin every day.  

There are some drawbacks to using Lantidra. Most of the study participants experienced unwanted side effects. Some of these, such as infections, were due to the use of immune-suppressing medications. Suppressing the immune system can also raise the risk of other health complications and possibly life-threatening conditions. 

Moreover, Lantidra relies on insulin-producing cells from donors. Because of this, their availability is limited, and their quality cannot be guaranteed. 

With these drawbacks, the FDA has only approved the use of Lantidra in people living with T1D who experience severe hypoglycemic events. 


VX-880, now in clinical trials 

VX-880 is an experimental cell therapy from Vertex Pharmaceuticals. It uses donated stem cells, modifying them to produce insulin. These cells are more readily available than the cells used in Lantidra.  

Aside from that, VX-880 is somewhat like Lantidra. The cells are delivered into the bloodstream in a similar way, and immune-suppressing medications are needed to protect them. 

A clinical trial of VX-880 began in 2021 for people living with T1D and severe hypoglycemia. Early in 2024, the study was paused for an independent committee review of the data, and it has since resumed.  

At the ADA Scientific Sessions last month, Vertex reported that 14 people had received VX-880 so far, with 12 of them receiving a full dose. All 12 were producing some of their own insulin. Some participants produced enough insulin to meet their body’s needs and no longer had to administer it themselves. All participants had glucose levels in the target range, and none experienced severe hypoglycemic events. Based on the data so far, the trial has been expanded to 37 participants. 


VX-264, also in clinical trials 

Vertex is also working on a second approach to cell therapy called VX-264. This investigational therapy uses modified stem cells to produce insulin, like VX-880, but the administration is different. The cells are placed, or encapsulated,  inside a protective device to shield them from the immune system. Then, the device it’s surgically implanted in the abdominal wall. Because this mechanism protects the cells, VX-264 is used without immune-suppressing drugs.  

In May 2023, Vertex launched a clinical trial of VX-264. They plan to enroll 17 participants with T1D from around the world. As of January, multiple people were already enrolled and had received VX-264. The trial should conclude in 2026, but preliminary results may come out sooner. 


The next generation of cell therapy 

Cell therapy for T1D has come a long way in the past decade. It can help people who have T1D produce their own insulin, transforming their daily lives. But today’s approaches still leave room for improvement. 

Researchers are working to improve cell therapy so it can benefit more people living with T1D in the future. Physical encapsulation is one example of an important innovation. Another option might be to use immune or gene therapy to protect insulin-producing cells from the immune system.  

While it’s not yet available, with continued development and testing, cell therapy has the potential to positively affect the lives of everyone living with T1D.