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Clinical Trial Tests Stem Cell Therapy to Cure People with Type 1 Diabetes

Published: 3/15/21
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By Matthew Garza

Vertex's new cell therapy, made from stem cells, aims to replace insulin-producing cells in people with type 1 diabetes. The therapy will soon begin testing in clinical trials to determine its safety and efficacy.

Over thirty years ago, Dr. Doug Melton’s son was diagnosed with type 1 diabetes at just 6 months. Dr. Melton was a prominent scientist and this life-changing event triggered him to begin searching for a cure to type 1 diabetes, not just a treatment. His research lab has been working for years to discover a way to turn stem cells into working beta cells (the cells in your pancreas that produce insulin). 

Moving forward in time, Semma Therapeutics was a biotechnology company that pioneered the use of stem cell-derived human islets as a potentially curative treatment for type 1 diabetes. They made several major scientific advances, including the ability to produce large quantities of working human beta cells that restore insulin secretion, and they were acquired by Vertex in 2019.

Vertex Pharmaceuticals has since built on all of this groundbreaking and foundational work that began in Dr. Melton’s lab and Semma Therapeutics. Vertex recently announced that it will soon launch a clinical trial for VX-880, a therapy for type 1 diabetes. VX-880 is the first “fully differentiated stem cell-derived islet cell therapy,” but what does this mean?

Type 1 diabetes occurs when the immune system, a complex network of cells and proteins that defend the body against infection and keep you healthy, attacks the beta cells in the islets of the pancreas that produce insulin, called islet cells. Researchers believe that it is possible to replace the damaged islet cells with new healthy cells, but this might also require a way to prevent your body’s immune system from attacking and destroying these new cells. This would essentially be a possible cure for type 1 diabetes. 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 the cells in the bones, heart, brain, and essentially every part of the body. Stem cells 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 VX-880, stem cells are grown in a lab and instructed to become islet cells that are able to produce insulin. They can then be transplanted into someone with type 1 diabetes. Type 1 diabetes is not the only disease in which stem cells might be a cure, whether they become specialized in a lab or inside a person.

This is an exciting area of research. If scientists can get stem cells to turn into safe, working islet cells and then successfully transplant them into a person with diabetes, they may be able to restore a person to a healthy state. Scientists have long known that islet transplantations can effectively ‘cure’ diabetes, but donor material has been insufficient. For the VX-880 therapy, because the stem cells are not a person’s own stem cells, a person will also receive an immunosuppressive therapy that will act to turn off the immune system and keep it from attacking these new cells – this is similar to a person who receives an organ transplant.

This clinical trial aims to show whether the VX-880 therapy could work and whether it is safe. If it is successful, a follow-up phase 3 clinical trial would enroll more people to further investigate its efficacy and safety. 

Clinical Trials Identifier: NCT04786262

Trial name: A Safety, Tolerability, and Efficacy Study of VX-880 in Participants with Type 1 Diabetes

Diabetes type: Type 1

What is the trial testing? The study will look at the safety, tolerability, and efficacy of different doses of Vertex’s VX-880 therapy. The trial will include about 17 people with type 1 diabetes who have experienced impaired hypoglycemia awareness and severe hypoglycemic events. The participants will receive an infusion of VX-880 (delivered through a vein) – fully mature and working islet cells, which were initially stem cells. 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. Each person is different and the answer may vary. Participants will also receive ongoing immunosuppressive medication; this will protect the new islet cells from being attacked by the immune system. All transplants, including those who obtain a kidney and/or a pancreas for type 1 diabetes must take immunosuppression medication, and for this first clinical trial of the VX-880 cell therapy, this will also be true.

What is the trial measuring? 

Vertex will evaluate several different outcomes:

  • The number of “adverse events” or “severe adverse events” from the start of the therapy up to five years 

  • The number of participants with an A1C below 7% (or a one percentage point decrease in A1C from their starting point) 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 is separated from insulin as it is released from the pancreas. If a person’s levels improve, it could indicate functioning of the VX-880 cells.

Trial location: Sites currently include University of Miami Health System, the University of Pennsylvania, and Massachusetts General Hospital. More sites may be added.

Are you interested?

You may be eligible to join the study 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 transplant, organ transplant, or cell therapy

For more information: contact Medical Information at Vertex at 617-341-6777 or medicalinfo@vrtx.com.

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