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VX-880: A New Stem Cell-Derived Experimental Therapy for People With Type 1 Diabetes

Type 1 diabetes (T1D) happens when the immune system destroys the pancreas’s beta cells, which produce insulin and regulate blood sugar levels. As a result, people with T1D must take insulin, a vital hormone, multiple times a day. Taking insulin and managing blood sugar levels are a significant burden for people with T1D.  

Although there are modern treatments and technology that can help alleviate some of that burden, there is still no cure for T1D.

A recent news release from the Vortex pharmaceutical company revealed the first results of an ongoing clinical trial of its stem cell-derived treatment for T1D. This new therapy, called VX-880, achieved a 91% decrease in daily insulin requirements for the first patient after only 90 days. Although the study is still in its preliminary phase, it offers a glimmer of hope for a potential cure for T1D in the future.

Stem cell therapy

Stem cells are found in most tissue; they are the source from which different types of tissue-specific cells are formed. These cells can multiply indefinitely and form different cells. For instance, stem cells from the bone marrow can develop into red blood cells (which carry oxygen), white blood cells (which fight infections), blood platelets (which allow for coagulation), etc. Same goes for the pancreas’s beta cells, where stem cells can develop into insulin-producing beta cells. 

This is the basis of stem cell therapy: replacing sick or defective body cells with healthy cells. These treatments are being studied for a number of diseases.   

In T1D, the goal would be to replace the insulin-producing beta cells that were destroyed by the immune system with healthy beta cells so that the pancreas can once again produce insulin to meet the body’s requirements. 

VX-880: How does it work, and how is it administered? 

VX-880 is currently being studied in the United States. It works by replacing the damaged beta cells with healthy cells made in a lab. It is administered by perfusion through a liver vein located near the pancreas.  

Since this treatment uses stem cells that come from another human organism, the patient must also take an oral immunosuppressive medication that prevents the body from rejecting the cells. This way, the healthy transplanted cells can produce insulin and maintain blood sugar levels in range.

However, immunosuppressive drugs and are a lifelong commitment that often comes with many adverse events. For instance, they may increase the risk of serious infections and cancer. 

Promising results

The press release indicates that the first patient in this study has been living with T1D for 40 years and had experienced many severe hypoglycemia episodes in the year before receiving the stem cell treatment.

The patient received a half dose of the VX-880 treatment, along with immunosuppressive medication.

After 90 days, their daily insulin requirements decreased by 91%, going from 34 units to 3 units.

The press release also mentions that there were no serious adverse events—only minor to moderate adverse reactions—following the treatment and the use of immunosuppressive medications, but there aren’t many details on this issue. Obviously, any risks associated with this treatment will need to be considered carefully.

What’s next?

These impressive results are to be taken with a grain of salt: they were observed on one patient only, and the study is only starting.

It is also important to know that people with T1D have “memory” immune cells that can destroy the new beta cells. These memory cells are very hard to suppress, even with immunosuppressive medication. They are the reason why stem cell transplants generally don’t last in the long term.

If successful, stem cell-derived therapy could be a great alternative to pancreas transplantatiuon, which involves replacing the whole pancreas and is not suitable for every patient (heavy therapy with risk of mortality). Pancreases are also harder to come by than stem cells.

The clinical trials for VX-880 are expected to continue. There are already many recruiting sites in the United States, and more will be coming to Canada soon, where the company just received approval to begin clinical trials. 

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