How are the cells used?
The FORWARD-101 trial for the therapy, known as VX-880 or zimislecel, takes lab grown beta cells and infuses them into the liver of the participants. These cells are then able to mimic the destroyed cells in the pancreas which produce insulin (pancreatic islets), removing the need for insulin injections. Zimislecel requires long-term medication to stop the immune system from rejecting the cells.
How did the cells perform?
In this study, 12 out of 12 patients were free of severe low blood sugar (hypoglycaemia) and spent 70% of the time within their glucose target range. 10 out of 12 participants had insulin independence and were not injecting any insulin after 365 days.
One of two doses of the cellular therapy were given in this trial (either one half dose or one full dose) and their success was measured using a biomarker called C-peptide. The presence of C-peptide levels in the blood directly relate to insulin activity. All 12 participants in the full-dose cohort had detectable C-peptide levels before and after meals, showing that the implanted cells were successfully producing insulin.
Why are cell therapies so important?
Cellular therapies like zimislecel represent a significant advancement toward a potential cure for T1D. By restoring the body’s ability to produce insulin, these therapies address the underlying cause of the condition rather than just managing its symptoms. This clinical trial marks a promising step toward enabling individuals with T1D to achieve insulin independence.
What are the next steps?
These new results are promising for cell-therapies in T1D. Further investigations are required with larger, more diverse populations to increase the evidence that lab grown beta cells can be given to patients to successfully treat T1D and help millions of people. Additional studies would also need to assess any long-term impacts of therapies like zimislecel.
Breakthrough T1D’s role in advancing research
Breakthrough T1D has played a key role in the early stages of this progress. In 2000, we funded Professor Doug Melton’s work to create insulin-producing beta cells from stem cells, which he successfully achieved in 2014. He went on to form Semma Therapeutics to develop these cells into potential cures for type 1 diabetes. Our T1D Fund later invested in Semma, which was acquired by Vertex. In 2021, their therapy VX-880 received fast-track designation from the FDA.
Rachel Connor, Director of Research Partnerships at Breakthrough T1D UK, says:
Today’s news brings a new wave of hope to those with T1D, as the journey toward cures for T1D reaches another new milestone. The results presented at the ADA conference are so exciting because they tell the story of people with T1D regaining the ability to live without the need for insulin therapy. This represents a landmark moment in T1D research. At Breakthrough T1D, we are proud to support the foundational research that made this innovation possible, and we remain committed to our ultimate goal – a world without T1D.
