Vertex Releases New Data on Potential Cure for Type 1 Diabetes
By Anna BrooksAndrew Briskin
For many years, a cure for type 1 diabetes has only been a pipe dream for the millions of people living with type 1 diabetes. New data from Vertex Pharmaceuticals on the first people to receive a revolutionary cell therapy for type 1 suggests a significant step toward a cure.
A third person with type 1 diabetes has achieved insulin independence after treatment with VX-880, a novel stem cell-derived beta cell therapy developed by Vertex Pharmaceuticals.
While Vertex’s clinical trial is still in early phases, the new data supports VX-880 as a potentially revolutionary treatment for people with type 1 diabetes. In addition, all six participants in the trial experienced significant improvements in A1C, time in range, as well as a reduced need for insulin injections.
The new findings were presented at the 2023 European Association for the Study of Diabetes (EASD) conference in Hamburg, Germany. Before VX-880 treatment, all study participants produced zero insulin and had a history of severe, recurring episodes of hypoglycemia (low blood sugar). Three months after cell transplantation, participants exhibited insulin production that successfully responded to glucose.
“These data are particularly meaningful in the context of our overall investigational type 1 diabetes program,” said Dr. Carmen Bozic, chief medical officer at Vertex, in a press release. “We are moving with urgency to bring these potentially transformative therapies to patients who are waiting.”
The third person in the trial to attain insulin dependence joins two others, who after 12 months of follow-up, maintained an A1C lower than 7% and no longer experienced severe hypoglycemia. Such encouraging results position VX-880 as a “functional cure” for type 1 diabetes.
So far, VX-880 has been well-tolerated with no serious reported adverse events. The data also shows promise for VX-264, another new Vertex stem cell treatment that does not require immunosuppression currently being investigated in clinical trials.
What is VX-880 therapy?
A type of experimental stem cell therapy, VX-880 aims to restore natural insulin production in people with type 1 diabetes. This would result in a drastic improvement in quality of life as those with the disease would no longer require insulin or experience severe, sometimes life-threatening, episodes of hypoglycemia.
Stem cell therapies like VX-880 work by transplanting healthy insulin-producing cells into the body. If successful, the new cells would replace non-functioning ones and ultimately restore insulin production.
Similar to people who receive organ transplants, those treated with VX-880 must take immunosuppressants to prevent the immune system from rejecting new beta cells.
Because of the risk of infection associated with immunosuppressants, only those with severe hypoglycemia unawareness have been eligible for Vertex’s trial. This is because these candidates have the most to gain from this type of therapy, given the risks associated with hypoglycemia unawareness.
Recruitment for the Vertex clinical trial is currently ongoing.
The history behind VX-880
Vertex first announced the development of VX-880 in 2021. Data from the first two participants in the trial were presented by Dr. James Markmann, chief of the division of transplant surgery at Massachusetts General Hospital, at the 82nd American Diabetes Association’s (ADA) Scientific Sessions.
In the first phase of the trial, each participant was given half the dose of beta cells estimated to be required. A common practice in these types of studies is starting with half the expected dose and increasing it as the trial progresses.
The following results show astonishing improvements in A1C, time in range, and reduced insulin needs for each participant. For reference, the ADA 2023 Standards of Care recommends a target time in range of 70% (70-180 mg/dL). There is no “standard” insulin dose, as daily insulin doses are determined by a healthcare provider based on the needs of each individual.
Participant 1
|
Study day |
Day 0 (baseline) |
Day 121-150 |
Day 241-270 |
|
Daily Insulin dose |
34 units |
2.6 units |
0 units |
|
Time in Range |
40.1% |
81.4% |
99.9% |
|
A1C |
8.6% |
6.7% |
5.2% |
Glucose data generated by a continuous glucose monitor (CGM) worn by participant 1.
Participant 2
|
Study day |
Day 0 (baseline) |
Day 121-150 |
|
Daily Insulin dose |
25.9 units |
18.2 units |
|
Time in Range |
35.9% |
51.9% |
|
A1C |
7.5% |
7.1% |
Glucose data generated by a continuous glucose monitor (CGM) worn by participant 2.
When will this become available to more people with type 1 diabetes?
Unlike other interventions, such as pancreas transplants or beta cell islet transplants from human donors, VX-880 has the potential to be used on a much larger scale.
“Right now in the U.S., there are only about 1,000 pancreas transplants available, so supply is a significant problem,” Markmann said. “One of the most important aspects of this work is that there can be an unlimited supply of beta cells for transplantation going forward.”
While the results so far are exciting, research is in early stages. The current trial is anticipated to wrap up in 2028, and additional studies with large sample sizes would need to follow. After all of the studies are completed, Vertex needs to submit an application to the FDA before it can be approved for use.
“This is groundbreaking work and a real leap forward for the field,” said Markmann. “While we cannot say exactly when this will become available, we are much closer to a functional cure for type 1 diabetes than we were before this approach.”
Learn more about treatments for type 1 diabetes here:
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FDA Approves Lantidra, the First Ever Cell Therapy for Type 1 Diabetes
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Ozempic May Reduce Insulin Needs in People With Type 1 Diabetes
