The world’s most enjoyable type 1 diabetes experiment is on the lookout for volunteers right away. A lucky few might be chosen to receive transplants of an experimental therapy named VX-264 — a possible landmark on the road to a cure for type 1 diabetes.
What Is Vertex Pharmaceuticals?
Vertex Pharmaceuticals is the biotech startup that’s developing the therapy that many experts consider is the most effective likelihood we’ve of a kind 1 diabetes cure.
Vertex’s approach is predicated on islet cell transplants. We now 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 individuals who have received islet transplants have enjoyed over a decade of healthy blood sugars without the necessity for insulin injections.
But islet cell transplants haven’t spread widely for 2 necessary reasons:
- Islets can’t be donated and have to be harvested from the body of an organ donor, strictly limiting the availability.
- Islet transplants require anti-rejection medications, which include negative uncomfortable side effects and may actually be toxic to the transplanted cells.
Consequently, islet transplants have mostly been limited to individuals with a severe need, similar to those with life-threatening hypoglycemia unawareness.
Vertex appears to have solved the primary of those problems. The business has developed a brand new approach to growing islet cells in a laboratory from pluripotent stem cells. And though these cells have been tested in just a handful of individuals so far, to this point, they’ve worked: Two Vertex patients at the moment are insulin-free, and a number of other others have shown impressive glycemic improvements.
The second problem — the necessity for antirejection medications — stays unsolved. That’s where VX-264 is available in.
What Is VX-264?
VX-264 is the name of a brand new procedure that may 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 have the opportunity 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 start with).
Camillo Ricordi, MD, told Diabetes Each day that Vertex currently has the “pole position” within the race for a kind 1 diabetes cure. Dr. Ricordi is the director emeritus of Miami’s Diabetes Research Institute, considered one of several facilities that may test VX-264.
Where and When Is the Trial Taking Place?
The VX-264 trial is going down at plenty 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 could have already enrolled volunteers. The trial is predicted to finish in May 2026, but we’ll have early results before then.
The primary volunteers will receive only a partial dose of islet cells, and researchers will primarily be checking the protection of VX-264 reasonably than its efficacy. Because the trial unfolds over the following several years, nevertheless, subsequent patients could also be given full doses of islet cells with a view to assess the potency of the therapy. The trial will last so long as two years for every volunteer.
Who Is Eligible to Participate?
To be clear, that is a very rare opportunity. Vertex intends to enroll about 17 patients. Each participating facility will likely have just one to 3 volunteers each.
All volunteers must:
- Be between the ages of 18 and 65
- Have lived with type 1 diabetes for not less than five years
- Have used a continuous glucose monitor (CGM) throughout the last 4 months
- Never before received cell therapy or an islet or organ cell transplant
You could find more details at ClinicalTrials.gov.
The experimental therapy is provided to review volunteers free of charge. It’s a chance to be considered one of the world’s first recipients of a potentially game-changing diabetes treatment. In fact, there may be also a risk that VX-264 is ineffective and even harmful, though patients can expect to be monitored by doctors very rigorously.
Even when VX-264 just isn’t the large step forward to the cure we’ve been hoping for, the trial guarantees to meaningfully contribute to our knowledge of diabetes and other efforts for a cure.
Contact Information:
When you’re keen on participating, call (617) 341-6777 or send an email to medicalinfo@vrtx.com.