Research and Pipeline
Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.
These studies are investigating treatments or outcomes that have not all received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority.
What is type 1 diabetes? Type 1 diabetes (T1D) is a metabolic, autoimmune disease where the cells in the pancreas (pancreatic islets) that produce insulin are destroyed. Insulin is a hormone that the body needs to process glucose, a key source of energy. Without insulin, no cell in the body can use or store glucose normally. This leads to high glucose levels in the blood, known as hyperglycemia, associated with severe, multi-systemic, acute and chronic complications that can accumulate over decades. Due to these high blood sugar levels, people with T1D develop symptoms including increased thirst or hunger, frequent urination and unexplained weight loss. Unable to use blood glucose for energy, the body will attempt to compensate by breaking down muscle and fat, leading to muscle wasting and buildup of ketones in the blood, which becomes toxic. This can result in diabetic ketoacidosis, which may lead to death.
Living with T1D requires lifelong treatment with insulin. People with T1D need to give themselves multiple injections of insulin per day or use an insulin pump that is worn on the body. They must also check their blood sugar levels frequently and manage their diet and exercise closely. There’s a delicate balance between too much insulin and not enough insulin. Without enough insulin, uncontrolled high blood sugars can, over time, lead to complications such as kidney disease, eye disease (including vision loss), nerve damage and even death. Too much insulin can cause low blood sugar, which can lead to symptoms of severe hypoglycemia like loss of consciousness, coma or even death.
A small number of patients have been made free of insulin therapy with a transplant of insulin-producing cells (pancreatic islets) from organ donors. This experimental procedure is only available to a few dozen people each year in the U.S. and requires lifelong treatment to prevent rejection of the donor tissue.
How is type 1 diabetes diagnosed? T1D is initially diagnosed when people develop the characteristic symptoms of increased thirst/hunger, frequent urination and unexplained weight loss. Blood tests are used to assess blood sugar levels and confirm the diagnosis. This type of diabetes used to be known as “juvenile-onset diabetes” or “insulin-dependent diabetes” but can be diagnosed in childhood or adulthood.
What is the underlying cause of disease? In T1D, the body’s own immune system destroys the beta cells in the pancreatic islets leading to a loss of insulin production. When beta cells function normally, they produce insulin to respond to increases in blood sugar (e.g., after eating) and maintain normal blood sugar (e.g., during sleep and between meals). Without enough beta cells to make the right amount of insulin, blood sugar levels are not regulated, and this leads to symptoms and long-term complications. Genetic and environmental factors are thought to play a role in the development of T1D, but the exact triggers for autoimmune cell destruction are not known and may vary between people.
We’re investigating and advancing cell therapies aimed at treating an underlying cause of T1D – the absence of insulin producing cells. In the 1970s, scientists developed an understanding that T1D is caused by the destruction of insulin-producing beta cells by the body’s own immune system. For 100 years, improvements in insulin and technologies for insulin management have helped people with T1D better balance their condition and live fuller lives. However, there are still no approved treatments that address a root cause of T1D – the absence of insulin producing cells in the pancreatic islets.
Our investigational approach is to replace the insulin-producing cells that have been destroyed in people with T1D. We’re evaluating multiple approaches to deliver the insulin-producing cells, including a transplant approach that would require immunosuppression similar to an organ transplant, and a device approach that is intended to protect the transplanted cells from the immune system. Both approaches utilize our proprietary, fully differentiated, insulin-producing islet cells.
VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.
VX-880 involves an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.
In addition, we are researching a second approach that would use a device to potentially protect the transplanted cells from the immune system.
In 2021, Vertex initiated a Phase 1/2 study evaluating VX-880 in participants with type 1 diabetes mellitus (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia. To learn more, visit clinicaltrials.gov.