VerTex Stem Cell Therapy for Type 1 Diabetes Shares Early Results

Vertex Pharmaceuticals announced last year that the first Type 1 diabetes (T1D) patient was about to be dosed in their Phase I/II clinical trial that investigates stem cell therapy, VX-880. They announced that this patient had a reduction in insulin use of about 91% just 90 days after receiving an infusion of the fully differentiated cells.

According to a meta-analysis published in the journal Health Promotion Perspectives the incidence of type 1 diabetes is about 15 per 100,000 people and has a prevalence of 9.5% globally. 

In 2016, the prevalence of diagnosed type 1 diabetes was 0.55%, representing 1.3 million US adults.

The efficacy of the therapy according to the company is being evaluated using different metrics. One of them is by measuring the levels of C-peptide, which directly indicates endogenous beta-cell function. The patient that received the therapy had no detectable C-peptide pre-infusion and after the therapy with stem cells, the patient had a significant increase in C-peptide levels, which directly indicates the presence of insulin secretion. 

The patient also saw a significant decrease in HbA1c, which indicates an average of glucose levels in the last two to three months. The patient improved from 8.6% to 7.2% without severe hypoglycemic events, he also didn’t experience any severe adverse events.

For the trial, the company is using immunosuppressive therapies prior to administering stem cell therapy. 

The research team is planning to enroll around 17 patients into its early-stage trial. In its current form, VX-880 requires recipients to go on life-long immunosuppressants. Therefore the therapy’s risk-to-benefit ratio may only be viable for those with the severest form of the disease.

The company is also progressing IND-enabling studies for its encapsulated islet cell program, which would potentially eliminate the requirement for immunosuppression, and is planning to file an investigational new drug (IND) application to the FDA for this program this year. 

The therapy has incredible potential if proven safe and effective, and could have an amazing impact on people’s lives and also decrease the economic burden for patients and healthcare systems, like the US where the cost of insulin is high and often requires patients to pay out of pocket. 


Kezia Parkins. (2021, Oct 19). Impressive early results for Vertex’s potentially curative stem cell diabetes therapy. Clinical Trials Arena. Retrieved from: 

Brian Herrick. (2021, Oct 18). Vertex Shares Data from Their Stem Cell-Derived Therapy- and It’s Oustanding. JDFR. Retrieved from: 

Mobasseri M, Shirmohammadi M, Amiri T, Vahed N, Hosseini Fard H, Ghojazadeh M. Prevalence and incidence of type 1 diabetes in the world: a systematic review and meta-analysis. Health Promot Perspect. 2020;10(2):98-115. Published 2020 Mar 30. doi:10.34172/hpp.2020.18,diabetes%20than%20did%20Hispanic%20adults.

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