Stem Cell Therapy for Type 1 Diabetes – A Potential Breakthrough Treatment

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Type 1 diabetes (T1D) is an autoimmune disease where the immune system attacks and destroys the insulin-producing beta cells in the pancreas. This results in the inability to produce insulin, the hormone needed to regulate blood sugar. Without insulin, blood sugar rises unchecked leading to complications like heart disease, nerve damage, kidney failure, vision loss and more. People with T1D face a lifetime of injecting themselves with insulin to survive. It is a relentless daily burden. But now, new stem cell research may offer hope for a functional cure.

Stem Cell-Derived Pancreatic Cells Restore Insulin Production  

In an exciting clinical trial by Vertex Pharmaceuticals, a patient with longstanding T1D was treated with an investigational stem cell therapy called VX-880. This therapy contains fully differentiated insulin-producing pancreatic islet cells that were derived from human embryonic stem cells.

Unlike previous stem cell attempts at treating diabetes, VX-880 provides the patient with replacement cells capable of sensing blood sugar levels and producing insulin on demand, just as a healthy pancreas does. This means patients may no longer need injectable insulin to control their blood sugar.

So how does it work? The stem cell-derived islet cells are infused into the patient along with immunosuppressive drugs to prevent rejection. In the trial, after a single infusion of VX-880, the patient’s blood work showed rapidly increased levels of C-peptide, a marker of natural insulin production. The patient also had improved HbA1c, reduced exogenous (injected) insulin requirements, and better glycemic control overall.

In essence, the infused islet cells acted like a mini-pancreas, restoring the body’s ability to produce insulin again. This is the first time stem cell therapy has achieved robust islet cell engraftment and function in humans.

A Decades-Long Vision Becomes Reality  

This potential diabetes breakthrough originated over a decade ago in the lab of Harvard scientist Doug Melton, Ph.D. Melton has a personal connection to T1D – both his children were diagnosed with the disease in childhood.

He became determined to find a better treatment, and in the 2000’s he began working on using stem cell science to create insulin-producing islet cells. It was still early days for the stem cell field back then. But Melton had a vision for translating embryonic stem cells into a cell replacement therapy for diabetes.

Now, years later, his vision is becoming a reality. The VX-880 clinical trial leveraged the protocols Melton’s lab developed to produce functional pancreatic islet cells from stem cells. For Melton, “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.”

A Potential Game Changer for Islet Transplantation  

Currently, the only way to perform islet transplantation is to isolate islet cells from deceased organ donors – a severe limitation. VX-880 overcomes this by creating an unlimited supply of islet cells from stem cells.

According to Dr. James Markmann, Chief of Transplant Surgery at Massachusetts General Hospital who has performed islet transplants for decades, “This approach, which obviates the need for an organ donor, could be a game changer.

“Not only does VX-880 solve the donor shortage problem, but since the islet cells are grown from stem cells, they can be carefully quality controlled for purity, function, and safety. No more relying on variable deceased donor organs. VX-880 offers standardized, reproducible islets capable of restoring natural insulin production.

A Functional Cure on the Horizon  

While it’s still early days, the success seen with VX-880 makes scientists optimistic that stem cell-derived islet replacement may one day free T1D patients from the shackles of daily insulin injections. By providing long-term functioning islet cells, this therapy offers the possibility of a functional cure.

Dr. Bastiano Sanna of Vertex Pharmaceuticals called the initial trial results “unprecedented” and stated that VX-880 supports “the continued progression of our clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression.

“Immunosuppressive drugs have been required to prevent rejection of transplanted cells. However, encapsulation technologies may overcome this need by hiding the islet cells in a protective coating. Encapsulated stem cell-derived islets could become a completely curative therapy.

Bringing Relief to Those Burdened by Diabetes  

For people with T1D, the daily hardship of managing this disease cannot be overstated. The promise of being liberated from insulin injections through regenerative medicine brings tremendous hope. While more clinical trials are needed, VX-880 represents a giant leap down the path toward a cure.

Here at Stem Cell Medical Center, we specialize in providing leading-edge stem cell therapies from around the world. Our expert physicians continuously evaluate the latest innovations to determine how they may benefit our patients. If VX-880 continues to show positive results, we will look to make it available to help relieve the suffering of those living with diabetes.

The Future is Bright  

Thanks to pioneering stem cell scientists, the future looks brighter for finding a cure for type 1 diabetes. We encourage anyone struggling with this disease to have hope. The regular insulin injections you endure today may soon be replaced by a regenerative therapy that frees you from this burden forever. Stem cell technology is advancing rapidly, so please check our website or schedule a consultation to learn about the latest options. We are here to help.