Insulin-Producing Therapy Shows Continued Promise for Type 1 Diabetes in Human Trials - T1D Exchange

In recent years, research has made impressive strides in developing potentially life-changing treatments for people with type 1 diabetes (T1D). A major player driving this progress forward is Vertex Pharmaceuticals. Building on their 2019 acquisition of Semma Therapeutics, the development of Vertex’s VX-880 and VX-264 continues to result in groundbreaking progress. 

On October 3, 2023, Vertex shared an update on the status of the first six patients to receive VX-880 — here’s a closer look at the news. 

First, what are VX-880 and VX-264? 

VX:880: This investigational therapy uses insulin-producing stem cells delivered directly into the patient’s hepatic portal vein. This vein is key in transporting blood through several organs, including the pancreas. There is one catch, however; to ensure these newly introduced cells can thrive and produce insulin, the treatment requires immunosuppression to protect them from the patient’s immune system. 

VX-264: This next step using Vertex’s proprietary product involves surgically implanting the insulin-producing stem cells into the patient with a protective device, ensuring the cells are shielded from the patient’s immune system. The best part? This approach completely removes the need for immunosuppression therapy. 

Update on the first six patients to receive VX-880 

• Patient 1: This individual has been living with T1D for nearly 42 years. Before the trial, they required an average of 34 units of daily exogenous (outside the body) insulin via injections or pump. By day 270 post-infusion of VX-880, the patient achieved insulin independence through Month 24. 
• Patient 2: With a history of T1D for 19 years and an initial daily requirement of 45 units of exogenous insulin, this patient achieved insulin independence by Day 180, sustaining it through Month 12. However, starting at Month 15, the patient resumed a regimen of four units of basal insulin daily, based on the investigator’s discretion. 
• Patient 3: After the data cut-off date, this patient achieved insulin independence on Day 180.
• Patients 4-6: Still earlier in the trial, all three of these patients, like their counterparts, displayed significant improvement in their glucose levels post-VX-880 treatment. By Day 90, they showed signs of islet cell integration and the ability to produce insulin in response to glucose levels. There was a notable drop in HbA1c levels, increased time-in-range, and a reduction or complete elimination of the need for insulin via injection or pump.

VX-880: a beacon of hope 

For those living with T1D, the name VX-880 might soon become synonymous with hope. All participants in the Phase 1/2 clinical trial of this novel treatment demonstrated a significant improvement in glucose levels and reduced exogenous insulin needs. 

The idea of daily immunosuppression therapy might daunt many, but this treatment is intended for those struggling with recurring severe hypoglycemia. For that group of people with T1D, this trade-off might be a small price for a significantly improved quality of life. 

It also serves as a stepping stone for VX-264. 

The promise of VX-264 

If VX-880 is the vanguard, VX-264 might as well be the future. Building on the advancements made with VX-880, VX-264 is a potential treatment approach that offers endogenous insulin without the need for immunosuppression.  

The initial success of VX-880 isn’t just a standalone victory; it’s proof of concept that paves the way for VX-264. It is tangible evidence that these insulin-producing cells have the potential to make a real difference in the lives of those with T1D. 

The dedicated researchers at Vertex 

Vertex’s progress is the result of a deeply dedicated team with leaders determined to develop cures for T1D, having already made significant progress in diseases like cystic fibrosis and others.  

In the mission to cure T1D, this work began with Doug Melton, Ph.D. at the Harvard Stem Cell Institute, eventually founding Semma Therapeutics in 2014. Felicia Pagliuca, Ph.D., joined Melton to help lead this groundbreaking biotech company. Their vision was clear – take this academic achievement and turn it into a life-changing therapy for T1D patients.  

Vertex’s acquisition of Semma Therapeutics was a testament to its dedication to curing T1D. It brought together the founders with a global biotechnology powerhouse. This collaboration pushes the boundaries of T1D research, exploring new and innovative ways to address the challenges of the disease. 

Vertex Pharmaceuticals’ endeavors in T1D treatment are more than just scientific experiments; they represent a beacon of hope for countless individuals worldwide. The advancements with VX-880 and VX-264 are prime examples of how innovation, dedication, and a deep understanding of human biology can pave the way for transformative treatments.  

As the world watches with bated breath, one thing is clear: the journey towards a potential cure for type 1 diabetes is in good hands with the dedicated researchers at Vertex Pharmaceuticals. 

READ MORE ABOUT VERTEX PHARMACEUTICALS & VX-880/VX-264

• Stem-Cell Therapy Gets Greenlight for Human Trials
• The Journey to a Potential Islet Cell Replacement Therapy for T1D
• Vertex partners with Lonza to build a manufacturing facility