T1D Cure Research Breakthroughs from Vertex and Sana Biotechnology

1. Vertex Pharmaceuticals shared the two-year update on the 12 participants in their VX-880 study using zimislecel. 
2. Sana Biotechnology shared the six-month update on two participants in their proprietary therapy, UP421. 

Here’s a closer look at these exciting type 1 diabetes (T1D) cure research updates:

Vertex’s Quest for a T1D Cure

Vertex shared a very encouraging update about its VX-880 clinical trial, which is using their proprietary therapy, zimislecel.

Zimislecel was first made using a single stem cell, but Vertex has since developed a method for manufacturing an endless supply of these insulin-producing cells. The therapy is administered as a single infusion into the participant’s hepatic portal vein, which is located in the liver, a common location for this type of therapy. 

How does it work?

• First, they turned the original stem cell into a cell that produces insulin.
• Normally, if you were to simply infuse these cells into the body of a person with T1D, they would be attacked and destroyed by your immune system.
• To protect these cells from the T1D immune system, participants must follow a daily immunosuppression regimen — just three pills daily.
• The participants involved 12 people with T1D, with impaired hypoglycemia awareness, and a history of severe hypoglycemic events.

Two-year update

• All 12 participants demonstrated “glucose-responsive” c-peptide levels. C-peptide levels indicate natural insulin production, which means the cells are producing insulin in response to rising blood glucose levels.
• 10 out of 12 participants are insulin-independent! This means they are no longer required to take daily injections of insulin to manage their blood glucose levels.
• The remaining two participants on daily insulin therapy have experienced significant decreases in daily insulin needs — by an average reduction of 92% — and A1C levels.
• The therapy is considered to be “generally well-tolerated” with most side effects classified as mild or moderate.
• Neutropenia (low neutrophils) was the most notable serious side effect, observed in three patients.

Keep in mind

• Yes, this is groundbreaking! In fact, Vertex is so hopeful about the long-term success of the VX-880 clinical trial that they’ve already begun building a manufacturing facility in NH to produce larger quantities of zimislecel.

• This therapy is only being considered in people who’ve experienced recurring severe hypoglycemia (when you need assistance regularly to treat low blood sugar) and live with “hypoglycemia unawareness” because of the necessity of immunosuppression therapy.
• This is considered a “functional cure” for T1D because it enables a person with T1D to no longer require daily insulin therapy and blood glucose monitoring despite not actually fixing the deeper dysfunctional issues within the T1D body. 
• The need for immunosuppression is obviously a key drawback and limitation of the therapy, but it could still be life-changing for people struggling to get through the day safely.
• They are expanding enrollment in the VX-880 study to 50 participants in the next phase.
• Vertex’s clinical trial VX-264 was cancelled earlier this year. This trial involved striving to protect the cells using a surgically implanted device, but it quickly proved to be ineffective.

Bottom line

Vertex is a clear leader and visionary in the pursuit of a functional cure for T1D! This update on VX-880 is truly exciting and a first of its kind. Still, there is more work to be done as they continue to monitor these participants’ results and work to bring this therapy to the market with full FDA approval.

Learn more about Vertex in Ginger’s videos:

• Will There be a Cure for Type 1 Diabetes? 
• Vertex UPDATE: VX264 & VX264
• Vertex Announces VX-264 Failure

Sana’s Quest for a T1D Cure

Sana Biotechnology announced very exciting results from the first-ever human trial using “hypoimmune” islet cells in people with T1D.

Their therapy, UP421, involves cells that can produce insulin while also hiding from your immune system. This means they’ve eliminated the need for immunosuppression therapy.

Islet cells produce beta cells — the cells in your pancreas that produce insulin. 

The study involves “intramuscular surgical transplantation of primary, or donor-derived, HIP-engineered islet cells into the participants’ forearms.

How does it work?

• First, they transformed stem cells into pancreatic islet cells, which include beta cells that produce insulin.
• Normally, if you were to simply infuse these cells into the body of a person with T1D, they would be attacked and destroyed by your immune system.
• By making the cells hypoimmune, the immune system does not attack and destroy them.
• The therapy was administered to two people with T1D who had undetectable C-peptide levels, which means they were not producing any of their own insulin.

Six-month update

• After six months, both participants have measurable C-peptide levels, which means their bodies have started making some of their own insulin again.
• Participants also produced insulin directly in response to meals, which means the cells are responding properly to the immediate presence of digested food entering the bloodstream.
• This means the cells are working and the hypoimmune protection is successfully warding off the T1D immune system!

Keep in mind

• This is early data! And it only includes two participants.
• This is the first step on a long road. Yes, it works so far, but that doesn’t mean they can rush this through the FDA approval process and put it on the market. 
• They’ll need to show results in larger, longer studies to demonstrate critical details, like: How long can these cells survive? Are there any adverse side effects? Will they need an additional dose after a few years? Etc. 

Bottom line

Sana's therapy is a huge step toward a functional cure for T1D—restoring the body's ability to make insulin. It’s very exciting and very hopeful! But there’s still a lot of work to be done!

Learn more about Sana in Ginger’s video:

• 4 Top Cure Efforts for Type 1 Diabetes