The world’s most exciting type 1 diabetes experiment is looking for volunteers right now. A lucky few will be selected to receive transplants of an experimental therapy named VX-264 — a potential landmark on the road to a cure for type 1 diabetes.
What Is Vertex Pharmaceuticals?
Vertex Pharmaceuticals is the biotech startup that is developing the therapy that many experts believe is the best chance we have of a type 1 diabetes cure.
Vertex’s approach is based on islet cell transplants. We have known for over 20 years that transplants of pancreatic islets — cell clusters that contain the insulin-producing beta cells that fail in type 1 diabetes — can restore insulin secretion. Some people who have received islet transplants have enjoyed over a decade of healthy blood sugars without the need for insulin injections.
But islet cell transplants haven’t spread widely for two important reasons:
Islets cannot be donated and must be harvested from the body of an organ donor, strictly limiting the supply.
Islet transplants require anti-rejection medications, which come with negative side effects and can actually be toxic to the transplanted cells.
As a result, islet transplants have mostly been limited to people with a severe need, such as those with life-threatening hypoglycemia unawareness.
Vertex appears to have solved the first of these problems. The business has developed a new method of growing islet cells in a laboratory from pluripotent stem cells. And though these cells have been tested in only a handful of people to date, so far, they have worked: Two Vertex patients are now insulin-free, and several others have shown impressive glycemic improvements.
The second problem — the need for antirejection medications — remains unsolved. That’s where VX-264 comes in.
What Is VX-264?
VX-264 is the name of a new procedure that will take those lab-grown islet cells and physically encapsulate them, shielding them from the body’s immune system. In theory, the shielded cells will still be able to sense glucose levels and secrete insulin in a healthy way, all while completely evading the body’s tendency to reject transplanted organs (or the autoimmune attack that causes type 1 diabetes to begin with).
Camillo Ricordi, MD, told Diabetes Daily that Vertex currently has the “pole position” in the race for a type 1 diabetes cure. Dr. Ricordi is the director emeritus of Miami’s Diabetes Research Institute, one of several facilities that will test VX-264.
Where and When Is the Trial Taking Place?
The VX-264 trial is taking place at a number of medical centers across the globe:
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
Headington, England — Churchill Hospital
Some facilities may have already enrolled volunteers. The trial is predicted to end in May 2026, but we’ll have early results before then.
The first volunteers will receive only a partial dose of islet cells, and researchers will primarily be checking the safety of VX-264 rather than its efficacy. As the trial unfolds over the next several years, however, subsequent patients may be given full doses of islet cells in order to assess the potency of the therapy. The trial will last as long as two years for each volunteer.
Who Is Eligible to Participate?
To be clear, this is an extraordinarily rare opportunity. Vertex intends to enroll about 17 patients. Each participating facility will likely have only one to three volunteers each.
All volunteers must:
Be between the ages of 18 and 65
Have lived with type 1 diabetes for at least five years
Have used a continuous glucose monitor (CGM) during the last four months
Never before received cell therapy or an islet or organ cell transplant
You can find more details at ClinicalTrials.gov.
The experimental therapy is provided to study volunteers for free. It’s an opportunity to be one of the world’s first recipients of a potentially game-changing diabetes treatment. Of course, there is also a risk that VX-264 is ineffective or even harmful, though patients can expect to be monitored by doctors very carefully.
Even if VX-264 is not the big step forward to the cure we’ve been hoping for, the trial promises to meaningfully contribute to our knowledge of diabetes and other efforts for a cure.
If you’re interested in participating, call (617) 341-6777 or send an email to firstname.lastname@example.org.