The world’s most exciting Type 1 diabetes experiments are looking for volunteers. A lucky few will be selected to receive an experimental therapy transplant named VX-264. This is a potential landmark on the path to treating type 1 diabetes.
What is Vertex Pharmaceuticals?
Vertex Pharmaceuticals is a biotech startup that develops treatments that many experts believe are the best opportunity to treat type 1 diabetes.
The apex approach is based on islet cell transplantation. For over 20 years, we have known that islet transplantation, a cell cluster containing insulin-producing beta cells that fail in type 1 diabetes – can restore insulin secretion. Some people who have undergone islet transplants have enjoyed healthy blood sugar levels for over 10 years without the need for insulin injections.
However, islet cell transplantation has not been widely used for two important reasons.
- The island cannot donate and must be harvested from the organ donor’s body, strictly limiting the supply.
- Islet transplantation requires anti-rejection drugs, which have negative side effects and may indeed be toxic to the cells that have been transplanted.
As a result, islet transplantation is largely limited to people with serious needs, such as those with a perception of life-threatening hypoglycemia.
The vertices seem to have solved the first problem of these problems. The business has developed a new method for growing islet cells in the laboratory from pluripotent stem cells. And although these cells have been tested in only a few people so far, so far they have been working. Two apex patients currently do not contain insulin, while several others have shown impressive improvements in blood glucose.
The second issue – the need for preservatives – remains unresolved. That’s where the VX-264 comes in.
What is the VX-264?
VX-264 is the name of a new procedure that collects islet cells grown in these labs, encapsulates the body, and protects it from the body’s immune system. Theoretically, shielded cells can sense glucose levels and secrete insulin in a healthy way, but completely avoid the tendency to reject transplanted organs (or autoimmune attacks that cause type 1 diabetes).
Camilo Ricordi, Maryland, told diabetes every day that the apex now has a “polar position” in the race for type 1 diabetes treatment. Dr. Ricordi is honorary director of the Diabetes Institute in Miami and is one of several facilities testing the VX-264.
When and where will the trial be held?
The VX-264 exam is being conducted at many medical centers around the world.
- Miami – Uhealth Diabetes Research Institute
- Chicago – University of Chicago
- Boston – Massachusetts General Hospital
- Pittsburgh – University of Pittsburgh Medical Center Montefiore
- Edmonton, Alberta – University of Alberta, Edmonton
- Toronto – Toronto General Hospital
- Leiden, Netherlands – Leiden University
- Headton, UK – Churchill Hospital
Some facilities may already have registered volunteers. The trial is expected to end in May 2026, but earlier results will be available.
The first volunteers receive only partial doses of islet cells, and researchers primarily check the safety of VX-264 rather than its effectiveness. However, if the trials unfold in the coming years, subsequent patients may be given full doses of islet cells to assess the efficacy of the treatment. The trial lasts for two years for each volunteer.
Who is eligible to participate?
To be clear, this is a very rare opportunity. The vertex is expected to enroll approximately 17 patients. Each participating facility only has 1-3 volunteers.
All volunteers must:
- Between 18 and 65 years old
- I have lived with type 1 diabetes for at least 5 years
- I used a continuous glucose monitor (CGM) for the past 4 months
- I have never had cell therapy or islets or organ cell transplants before
For more information, please visit ClinicalTrials.gov.
Experimental therapy is provided to study volunteers for free. This is the opportunity to become one of the world’s first recipients of a potentially game-changing diabetes treatment. Of course, there is also the risk that VX-264 is ineffective or even harmful, but patients can be expected to be monitored very carefully by their doctors.
Even if VX-264 is not a major advance into the treatment we wanted, this trial promises to contribute meaningfully to our knowledge of diabetes and other efforts to treat.
contact address:
If you are interested in participating, please call (617) 341-6777 or email [email protected].
