Microscopic Cells

Vertex Pharmaceuticals, a biotech company that develops stem cell-derived therapies, announced 90-day data on their first patient to receive VX-880, a novel, human stem cell-derived replacement therapy for people living with type 1 diabetes (T1D) — and the results were incredibly promising.

“This is the first demonstration of a patient with T1D achieving robust restoration of islet cell function from such a cell therapy,” explained Vertex in their press release.

Patient 1 had measurable increases in insulin production, improved blood sugars, and a 91% decrease in exogenous (injected) insulin needs, according to Vertex. In terms of insulin dosing, this means that the insulin needs of patient 1 decreased from 34 units a day to 3 units a day. Study results are most impressive, as this individual only received half of the targeted dose. VX-880 is in clinical phase 1/2, and it’s ongoing.

What Cell Therapy Is and Why It's Effective For Type 1 Diabetes

Type 1 diabetes is an autoimmune disease. This means that the body becomes confused and it destroys its insulin making beta-cells. People living with T1D must take insulin for life. There are only medications and tools to help manage T1D, but there’s nothing to help what caused T1D in the first place.

This is where the potential for VX-880 comes into play. VX-880 is an investigational stem cell-derived therapy that is being used to re-establish insulin production in the human body, potentially providing a functional cure for T1D.

The most important study outcomes thus far may possibly be how well the patient has tolerated both VX-880 and the required immune suppressing medications, according to Vertex. There have been no adverse effects — and there have been measurable improvements in the patient’s health (see below).

Noted Improvements

Improvements noted at the 90-day mark in patient 1 are:

  • Decreases in daily exogenous (injected) insulin needs by 91%

  • Increases in c-peptide levels (indicating that the cells are making insulin as expected)

  • Decreases in HbA1c levels (indicating stabilizations in blood sugar)

“While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. 

How VX-880 Is Administered

This therapy is injected into the portal vein which leads into the liver. The cells settle in and are nourished from the surrounding vessels. When working as expected, the cells can detect an individual’s blood sugar and adjust insulin as needed. So, changes noted in injected insulin needs, blood sugars, and c-peptide levels are all indicative that the cells are doing their job. 

Why Participants Need to Take Immune Suppressing Medication

The cells haven’t come directly from the patient’s body, so they need protection. Immune suppressing medications are given in tandem with VX-880 to ensure that the body doesn’t destroy them. In 2022, Vertex will be filing with the FDA in hopes of putting these same cells into a protective encapsulation device. This means that in this next clinical phase, immune suppressing medication may not be necessary. 

There are two types of cells used for various treatments: 

  • Allogeneic cells - “allo” means other. The cells come from a source other than the patient.

  • Autologous cells - “auto” means self. The cell comes from the same person who will get the transplant. The patient is their own donor. 

What is c-peptide?

A c-peptide test can show how much insulin the body is making. A low c-peptide level, or no level, means that a person is making little to no insulin. This test can be used to help tell the difference about what type of diabetes a person has. With T1D, a person has little to no c-peptide level, while with type 2 diabetes, a person has higher c-peptide levels, but the body isn’t using the insulin as expected. 

Can I be a participant in this study?

Visit Vertex to learn more, and to see if you may qualify as a future research participant. Enrollment is ongoing; approximately 17 people will participate in the study.

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