#Stem cell treatment for diabetes type 1 cost full#

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. The participants will receive an infusion of VX-880 (delivered through a vein) – fully mature and working islet cells, which were initially stem cells.

#Stem cell treatment for diabetes type 1 cost trial#

The trial will include about 17 people with type 1 diabetes who have experienced impaired hypoglycemia awareness and severe hypoglycemic events. What is the trial testing? The study will look at the safety, tolerability, and efficacy of different doses of Vertex’s VX-880 therapy. Trial name: A Safety, Tolerability, and Efficacy Study of VX-880 in Participants with Type 1 Diabetes If it is successful, a follow-up phase 3 clinical trial would enroll more people to further investigate its efficacy and safety. This clinical trial aims to show whether the VX-880 therapy could work and whether it is safe.

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. Scientists have long known that islet transplantations can effectively ‘cure’ diabetes, but donor material has been insufficient. 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. 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.

They can then be transplanted into someone with type 1 diabetes. 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. For example, a stem cell can become a new blood cell under the right conditions and with the right biological instructions. 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. 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. This would essentially be a possible cure for type 1 diabetes. 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. 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. VX-880 is the first “fully differentiated stem cell-derived islet cell therapy,” but what does this mean? Vertex recently announced that it will soon launch a clinical trial for VX-880, a therapy for type 1 diabetes. Vertex Pharmaceuticals has since built on all of this groundbreaking and foundational work that began in Dr. 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. 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. 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). 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. Doug Melton’s son was diagnosed with type 1 diabetes at just 6 months. The therapy will soon begin testing in clinical trials to determine its safety and efficacy. Vertex's new cell therapy, made from stem cells, aims to replace insulin-producing cells in people with type 1 diabetes.