Vertex Pharma's stem cell therapy may have potential to cure type-1 diabetes, shows clinical trial

Type 1 Diabetes (T1D) is an autoimmune disease in which the body’s immune system mistakenly attacks and destroys the insulin-producing beta cells in the pancreas. As a result, patients must rely on lifelong external insulin injections or pumps to regulate their blood sugar levels. Despite advances in insulin delivery and glucose monitoring, many people with T1D struggle with glycemic control and face the constant threat of severe hypoglycemic events (SHEs)—dangerously low blood sugar episodes that can lead to seizures, coma, or even death.

Zimislecel is a lab-created version of these cells, derived from stem cells. These new cells can naturally produce insulin, similar to a healthy pancreas. The therapy is based on allogeneic (off-the-shelf) stem cells, meaning it does not depend on scarce donor pancreases for islet cell transplantation. Instead, Vertex is producing these essential cells in the lab from pluripotent stem cells (a type of cell that can develop into almost any type of cell in the body). The breakthrough lies in its ability to replace the damaged beta cells, allowing the body to produce its own insulin in response to glucose levels.

How does Zimislecel work?

The therapy involves a single infusion whereby doctors inject lab-grown cells into the liver of the patient, where they begin to function like normal insulin-producing cells. These cells release glucose-responsive insulin, meaning they provide insulin to the bloodstream in proportion to the body’s sugar levels, mimicking the natural process in non-diabetic individuals. Patients also take immunosuppressive medication alongside Zimislecel to prevent their immune system from attacking the new cells.

What does the data say?

The recent data from the Phase 1/2 portion of the FORWARD-101 clinical trial have been remarkable, according to experts. In a study involving patients with severe T1D who experienced impaired hypoglycemic awareness and recurrent severe hypoglycemic events, it was found that:
Around 83% (10 out of 12) of patients who received a full dose of Zimislecel achieved complete insulin independence within one year of treatment. The other two patients experienced significant reductions in their daily insulin needs (70% and 36%, respectively), resulting in an overall mean reduction of 92%.

All 12 patients with at least one year of follow-up met the American Diabetes Association's recommended target HbA1c levels of less than 7%, spending over 70% of their time within the healthy blood sugar range (70-180 mg/dL). Notably, none experienced dangerous low blood sugar episodes after 90 days, and tests indicated that the new cells were functioning properly. The data is published in the New England Journal of Medicine.

Paradigm shift in Type 1 Diabetes treatment?

If approved, the Zimislecel therapy could allow people with T1D to live without daily insulin injections or constant blood sugar monitoring. The "off-the-shelf" nature of the therapy, which does not rely on donor organs, makes it scalable for manufacturing and potentially accessible to a broader population.

Read More: Prevent Type 2 Diabetes with these small daily lifestyle and diet changes

Any Impact on Type 2 Diabetes treatment

Vertex is currently testing Zimislecel in larger trials and hopes to receive regulatory approval soon. While designed for T1D, scientists are also considering how similar treatments might benefit people with Type 2 Diabetes in the future. In T2D, the body either doesn’t produce enough insulin or doesn’t use insulin effectively (insulin resistance). While T2D is often linked to lifestyle factors and can sometimes be managed with diet, exercise, and oral medications, many patients eventually require insulin therapy as their pancreatic beta cells become exhausted.