Stem-cell therapy for type 1 diabetes reduced patient’s insulin needs by 91%


Editor’s Note: The news shared below is from a recent and promising clinical trial. For more information on Type 1 diabetes research visit our research portal; to learn more about getting involved with clinical trials, click here.

The first patient dosed with VX-880 — a stem cell-derived potential therapy to treat Type 1 diabetes — has experienced a 91% decrease in their daily insulin doses, according to a recent press release.

In Phase 1/2 of its clinical trial, Vertex Pharmaceuticals Incorporated shared the staggering results after only 90 days of its ongoing investigational study, with the patient’s insulin needs reducing from 34 daily units down to 3 daily units.

VX-880 is manufactured using proprietary technology from Vertex Pharmaceuticals, a global biotechnology company that has multiple FDA-approved medications for cystic fibrosis. With their sights set on a breakthrough treatment for people with Type 1 diabetes, VX-880 is stem cell therapy approach that aims to restore insulin production and stable blood sugar levels.


To understand how VX-880 works, we must first understand the mechanics of Type 1 diabetes. In people with T1D, the immune system attacks and destroys the insulin-producing beta-cells in the pancreas.

Without these healthy cells, the pancreas cannot produce normal amounts of insulin. Insulin is the hormone that converts food to fuel. Without insulin, the body cannot use sugar from the bloodstream for fuel, causing blood sugar levels to rise to life-threatening levels.

Without insulin, the human body cannot survive. People with Type 1 diabetes must take manufactured insulin daily via injections, pump, or inhalation in an effort to maintain safe blood sugar levels.

While there are a variety of methods to deliver insulin and monitor blood sugar levels available today, they do not prevent those living with the disease from experiencing immense daily stress, life-threatening blood sugar fluctuations, rising healthcare costs, and an overall impact on their quality of life.


VX-880 works by essentially replacing the damaged insulin-producing cells in a person with Type 1 diabetes with healthy transplanted beta-cells. Because these are “allogeneic” cells — which means they come from outside the patient’s body — the patient must also undergo immunosuppression therapy to protect these transplanted cells from their body’s immune system.

VX-880 is delivered by an infusion into the patient’s ​​hepatic portal vein, which is located near the pancreas. The patient in this ongoing study received a single infusion of VX-880 — but only half the dose that Vertex anticipates a patient might need — in conjunction with ongoing immunosuppressive therapy to protect the transplanted cells.

By providing the body with these allogeneic islet beta-cells and immunosuppression, the transplanted cells are able to successfully produce insulin and manage safe and normal blood sugar levels.

“As a surgeon who has worked in the field of islet cell transplantation for decades, this approach, which obviates the need for an organ donor, could be a game changer,” said James Markmann, M.D., Ph.D., Professor of Surgery and Chief of the Division of Transplant Surgery at Massachusetts General Hospital.


It’s important to remember that these early results mean they cannot fully reflect the potential of this therapy’s ability to manage blood sugar levels.

The patient in the trial has lived with Type 1 diabetes for over 40 years. The duration of this person’s diabetes is significant since many of today’s ongoing efforts to effectively cure Type 1 diabetes are focused on those who are newly diagnosed.

“In the one year prior to treatment, the patient experienced five severe, potentially life-threatening hypoglycemic episodes,” explained the press release.

The patient not only tolerated both the infusion and immunosuppression well — with no serious adverse effects — they also experienced “robust improvements” in many areas related to diabetes health.

“These results from the first patient treated with VX-880 are unprecedented. What makes these results truly remarkable is that they were achieved with treatment at half the target dose,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex.

“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.”

Vertex’s encapsulation technology is also in early trial stages and offers the potential to protect the transplanted insulin-producing cells without immunosuppression.

“More than a decade ago our lab had a vision for developing an islet cell replacement therapy to provide a functional cure to people suffering from T1D,” said Doug Melton, Ph.D., Xander University Professor at Harvard and an Investigator of the Howard Hughes Medical Institute. “These promising results bring great hope that stem cell-derived, fully differentiated islet cells could deliver a life-changing therapy for people who suffer from the relentless life-long burden of T1D.”

With multiple active sites in the United States — and soon, Canada — Vertex will continue this phase of the trial with hopes for continued success.


How difficult do you think this is? Bad?

My interest screeched to halt when this requirement was finally revealed in the eighth paragraph. I halfway expected it but I hoped immunosuppression was not needed.


Encapsulate perhaps via Sernova process, take away immunosuppressive drugs, and I’d be there in a heartbeat! How much $$?

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What is that? I remember reading years ago about encapsulating beta cells in a glass capsule that would protect the cells from the auto-immune attack while allowing the cells food and the ability to sense BG.

Like a lot of these breaking advancement, we often don’t here about why they failed to pan out.

Like you, I wouldn’t want to have to trade insulin for immunosuppressive drugs.

But still undergoing trials and last I read they also required immunosuppression.

Edit: I have invested in this company but not made any money so far!!

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I considered this and the encapsulated cells too, because they make you suppress your immune system on that too.

The completely encapsulated implants failed miserably, so now they are only partially encapsulated and require the immune drugs

Yeah, this same thing happened with the ViaCtye. The device the cells were in just couldn’t handle the body defense systems. The body just doesn’t like foreign objects inside it.

I did do a small amount of suppression drugs to see if it would help and I was off all of them after the first month. But it made no difference.

I just don’t know if I would love to be diabetes free but than have to worry about catching every bug that is floating around me. Yes, I would love to not have diabetes but I do love living my life to its fullest. And being afraid of everyone coughing around me would make life pretty hard.

I will continue to wait. I do think stem cells will be the answer. I just don’t think I will see it in my lifetime. The great way our body works will make this very hard to overcome. But I continue to watch these studies and they sound great except I want it all. I want easy and careful, just like all the other “normals” who go about their life not even thinking about what their body is doing every moment of everyday.

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Agree that immunosuppressive make stem cell treatment undesirable. There would be danger of all sorts of infection not to mention your own body attacking you with cancer, etc. ugh No Thanks!! They have many years to go before this is viable.

The people who are being put into these studies are people who have major hypo issues. Like they are low most of the day, type of people. These people are very rare and most doctors never see a patient like this.

That, or they have complete hypoglycaemia unawareness and are constantly going comatose. Much less of an issue in the age of CGMs.

That wasn’t the goal though. The encapsulated islets themselves need to be brought to a stable state. This part needs to mature, otherwise any efforts at shielding them from the immune system are pointless. Efforts to achieve such shielding are still happening in parallel. Also check out this explanation from a researcher in that field.

I remain cautiously optimistic, also because such an encapsulation technology could be applied to other tissues as well, increasing its value. Think of encapsulated adrenal cells, or encapsulated thyroid cells, or encapsulated hepatic cells etc. And as the researcher said, the real news are that stem cells were used instead of cells harvested from cadavers. That’s quite big.

I kept my daughters cord block from birth, she is now 8 and T1D. I am wondering if there would be a need for immunosupression if she is using her own cells???

anyone have more information about this?

Yes there is, since T1D is caused by an autoimmune reaction, meaning that even if the islets from the same person, the immune system still erroneously attacks the beta cells inside those islets.

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