Clinical Trial Tests Cell Therapy To Cure People With Type 1 Diabetes
Vertex is recruiting participants for its zimislecel (VX-880) cell therapy trial. Early study results show promise that zimislecel may be the first functional cure for type 1 diabetes.
Trial Status: Recruiting
Clinical Trials Identifier: NCT04786262
Trial Name: A Safety, Tolerability, and Efficacy Study of VX-880 in Participants with Type 1 Diabetes
Diabetes Type: Adults 18-65 years of age with type 1 diabetes
Trial Sponsor: Vertex Pharmaceuticals
Why is this trial important?
This clinical trial is testing zimislecel (VX-880), the first fully differentiated cell-derived islet cell therapy for type 1 diabetes. Early study results so far are promising, with 12 participants no longer needing daily insulin after one year of therapy. Though immunosuppressants are required to prevent the body’s immune system from attacking and destroying the new cells (similar to when someone receives an organ transplant), research so far indicates that zimislecel could be the first functional cure for type 1 diabetes.
A little background: Type 1 diabetes occurs when the immune system, a complex network of cells and proteins that defend the body against infection, attacks the beta cells in the pancreas that produce insulin. These specialized cells are called islet cells.
Researchers believe that it is possible to replace damaged islet cells with new healthy cells. Traditionally, islet cells were transplanted from deceased individuals – the problem being that there is a very limited supply of cells from cadavers. This is where stem cells come in.
Stem cells have not yet developed into the mature cells that make up our organs and tissues, such as in the bones, heart, brain – essentially every part of the body. They form the pool of cells in the body that can turn into one or another of these specialized cells, but to do this, they must receive specialized instructions. For example, a stem cell can become a new blood cell under the right conditions and with the right biological instructions. To make cell therapies like zimislecel, stem cells are grown in a lab and instructed to become islet cells able to produce insulin. They can then be transplanted into someone with type 1 diabetes.
If scientists can get stem cells to turn into safe, working islet cells and then successfully transplant them into someone with diabetes, they may be able to restore a person to a healthy state.
How does the trial work?
This clinical trial is unique in that it’s a combination phase 1, 2, and 3 trial. That means participants are being recruited to receive the therapy for the first time, as well as following people who have been in the study for years to see how zimislecel is working longer term.
The study will look at the safety, tolerability, and efficacy of different doses of Vertex’s zimislecel therapy. The trial will include about 52 people with type 1 diabetes who have experienced impaired hypoglycemia awareness and severe hypoglycemic events.
The participants will receive an infusion of zimislecel (fully mature and working islet cells) delivered into through the portal vein of the liver. Each person will go through a full review of the inclusion and exclusion criteria with their care team to determine whether participation and maintenance of previous and ongoing therapies (such as insulin) is appropriate for them, given the constraints of the trial.
Participants will also receive ongoing long-term immunosuppressive medication to protect the new islet cells from being attacked by the immune system. It is important to know that there are serious risks associated with the use of immunosuppressive medicines, including an increased risk of infection and cancer.
How was VX-880 therapy developed?
Dr. Doug Melton, a prominent scientist at Harvard, learned his son had type 1 diabetes at just 6 months old. This life-changing event triggered him to begin searching for a cure to type 1 diabetes – not just a treatment. Melton and his team made several major scientific advances, including the ability to produce large quantities of working human beta cells that restore insulin secretion. Vertex Pharmaceuticals acquired this technology in 2019 and, using research from the Melton Lab, developed VX-880 – now known as zimislecel.
What outcomes will this clinical trial be measuring?
Vertex will evaluate several different outcomes during the trial:
- The number of adverse events (including severe adverse events) from the start of the therapy up to five years
- The number of participants with an A1C below 7% who do not experience severe hypoglycemic events after one year
- The number of participants who do not require insulin one year after treatment
- Changes in stimulated C-peptide levels one year after treatment. Measuring C-peptide is a widely used method for scientists to assess how much insulin someone is producing on their own, even if they are taking insulin. C-peptide is a molecule that is attached to the insulin a person produces on their own and separates from insulin as it’s released from the pancreas. If a person’s levels improve, it could indicate the new zimislecel cells are functioning.
Are you interested?
You may be eligible to participate if you:
- Are 18-65 years old
- Have been diagnosed with type 1 diabetes for at least five years
- Have had at least two episodes of documented severe hypoglycemia in the past year
- Are on a stable diabetes treatment plan
- Have used a continuous glucose monitor (CGM) consistently for at least three months prior to screening for the trial, and are willing to use the CGM during the trial
- Have not had any prior islet cell transplants, organ transplants, or cell therapy
This trial is currently recruiting in 29 locations worldwide, including the U.S., Canada, France, Germany, the Netherlands, Norway, Switzerland, and more. Learn more about this study here or contact the Vertex team at medicalinfo@vrtx.com or call (+)1-617-341-6777.
Learn more about emerging treatments for type 1 diabetes here: