Vertex Pharmaceuticals Stem Cell Therapy Shows Functional Potential for Cure Diabetes

On October 18, 2021, Vertex Pharmaceuticals announced that its fully differentiated islet cell replacement therapy derived from stem cells has obtained positive data in the first case of type 1 diabetes (T1D) in a phase 1/2 clinical trial
.
On the 90th day after receiving the single-dose treatment, the patient resumed insulin production and reduced daily insulin usage by 91%

T1D is caused by the immune system destroying insulin-producing islet cells in the pancreas, resulting in decreased insulin production and high blood sugar
.
High blood sugar can lead to diabetic ketoacidosis, and over time, it can cause complications such as kidney disease/failure, eye disease, heart disease, stroke, nerve damage, and even death

VX-880 is a fully differentiated islet cell therapy derived from allogeneic stem cells.
It is possible to restore the body's ability to regulate glucose levels by restoring the function of islet cells, including the production of glucose-responsive insulin
.
VX-880 is administered by hepatic portal vein infusion and needs to be combined with chronic immunosuppressive therapy to protect islet cells from immune rejection

The first patient in the trial was diagnosed with T1D about 40 years ago and has been dependent on exogenous insulin therapy
.
Within 1 year before receiving VX-880 treatment, the patient had 5 severe and potentially life-threatening episodes of hypoglycemia

The test results showed that on the 90th day after VX-880 treatment, the patient’s fasting C-peptide was 280 pmol/L, reflecting the recovery of basal insulin production, and it increased to a peak of 560 after the mixed meal tolerance test (MMTT) stimulation.
pmol/L, indicating that VX-880 restored the patient's glucose-responsive insulin production
.
Also on the 90th day, the glycosylated hemoglobin (HbA1c) improved from 8.

Picture source: reference [1]

Reference materials:

[1] 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.