Stem Cell Transplant in Type 1 Diabetes

Could Vertex Pharmaceutical’s VX-880, a procedure using lab-grown pancreatic islet cells, be a potential cure for type 1 diabetes?

The biotech firm Vertex has announced results for the first patient in the clinical trial of their new procedure known as VX-880. The innovator of this procedure, Dr. Doug Melton, is convinced of the procedure’s curing capability. 

As most know, stem cell treatment for type 1 diabetes is not a new remedy. However, previously the stem cells were harvested from organ donors. Due to low availability, this is not the most popular approach. So how is the VX-880 different? This procedure utilizes pancreatic islet cells grown in a laboratory from pluripotent stem cells, avoiding the need to harvest the cells from an organ donor. Once the cells are ready, they are placed into a patient’s portal vein. This allows for the new islet cell to monitor blood glucose levels and secrete insulin accordingly. 

The new release from Vertex has shown the results from a patient who had had type 1 diabetes for over 40 years, had experienced several life-threatening complications to the disease, and showed zero natural insulin production. This patient was utilizing approximately 34 units of insulin per day. They also reported an A1C of 8.6%. 

Results after 90 days post-procedure showed the patient had decreased their insulin usage 91%, from 34 units per day down to 2.9 units per day. They also had a significant decrease in their A1C. In addition, the patient dropped from their previous 8.6% A1C to a much more recommended 7.2%. The patient was only reported to have received half of the total clinical dose that Vertex plans to utilize, implying that the patient may have had better results given the implementation of the total dose. 

A potential downside to this treatment is that, as with most transplantations, immunosuppressive therapy is needed to help in the prevention of rejection. The risks and benefits of changing between one disease state to a state of immunosuppression with all its side effects is a serious issue. Vertex has reported their involvement in generating a solution to the immunosuppression issue in these patients. They are exploring the idea of creating an encapsulated islet cell that could potentially allow blood and insulin to filter through while simultaneously preventing the influx of immune cells. 

Although the results of only one patient have been presented, this is not the only patient involved in this study. At least 16 more patients are involved in this clinical trial, with more being enrolled. After one year, the patients will be reevaluated and then followed for up to 5 years. The goal of the reevaluation is to note the patents’ blood glucose control, insulin usage, and hypoglycemic occurrences. The results are hoped to show better blood glucose, overall decreased insulin usage, and reduced hypoglycemic events. 

This procedure is a long way from being available to the public. However, if the potential of VX-880 is demonstrated to be real, it could provide a possible cure and change the course of treatment for type 1 diabetes.

Practice Pearls

• VX-880 is a new way of approaching stem cell treatment in patients with type 1 diabetes. 
• Vertex is planning to generate a solution to the use of immunosuppressive therapy. 
• An astonishing 91% decrease in insulin usage was reported three months post-operation. 

“A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes.” ClinicalTrials.gov.

“Vertex Announces Positive Day 90 Data for the First Patient in the Phase 1/2 Clinical Trial Dosed With VX-880, a Novel Investigational Stem Cell-Derived Therapy for the Treatment of Type 1 Diabetes.” Press release, Vertex Pharmaceuticals Incorporated.

Wollen, Ross. “A New Type 1 Diabetes Stem Cell Treatment Just Showed Incredible Results.” Diabetes Daily, 19 Oct. 2021,

Quinlynn Champ, PharmD Candidate Class of 2022, South College School of Pharmacy