For many years, it has been clear that one possible path to a cure is to transplant new beta cells and also in some way prevent the body from rejecting those new, transplanted cells. Since the 1980s many different companies have tried encapsulated beta cells as a path to a cure. The idea is that beta cells cure T1D and encapsulation means both that the person does not need to take anti-rejection drugs and that the person's autoantibodies cannot destroy the new beta cells. Unfortunately, even after 40 years of research done by many different companies, this line of research has not been successful.
The JDRF has a good timeline of their support for stem cell therapies here:
Note that they have funded several different research projects, and ViaCyte is just one of them.
ViaCyte is one of these "old school" beta cell transplantation companies. They have developed an infrastructure to grow large numbers of beta cells from stem cells. However, recently they have partnered with a CRISPR company (called CRISPR Therapeutics). CRISPR is a technology to edit genes. In this case, they are editing genes in their newly grown beta cells, so that the beta cells are invisible to the patient's immune system. The hope is that this is going to have two important effects: First, the body will not reject the transplanted beta cells. Second, the bodies' autoimmune attack will not be able to target the transplanted beta cells.
Obviously, either one of these effects would result in a huge step forward on the path to curing T1D, and both of them together could result in an outright cure. This combination is what they have just started testing in a Phase-I clinical trial.
The Phase-I Clinical Trial
This is an open label (no blinding) clinical trial with 10 participants who are adults (18-65 years old) and have established T1D (diagnosed at least 5 years prior). All 10 people will get the treatment; there is no control group.
The gene edited, stem cell derived beta cells are loaded into a device and then implanted into a person. The person is then followed for 6 months. The trial's end points involve safety and several scientific measures of transplant success. The researchers will check for bad side effects, if the body is generating antibodies against the transplanted cells, and new autoantibodies related to T1D. They will also see if the stem cells are growing properly. However, none of the end points measure success in treating or curing T1D. There are no measures of C-peptides (the body generating its own insulin) or lowered requirements of insulin, or improved A1c. This study is really focused on testing safety and ability to be tolerated, not effectiveness.
Because patients are only followed for 6 months, they hope to run this study from January 2022 to December 2022. This includes the time needed to recruit the patients and the time to actually run the study on each patient.
This trial is being run in Canada, and you can get more information on participating here:
Contact: Clinical Trials +1 (877) 214-4634 MedicalAffairs@crisprtx.com
The study locations include:
- University of Alberta, Edmonton, Alberta, Canada
Contact: Parastoo Dinyari +1 (780) 407-1501 email@example.com
- University of British Columbia, Vancouver, British Columbia, Canada
Not yet recruiting
- LMC Manna, Toronto, Ontario, Canada
Enrolling by invitation
Clinical Trial Record: https://www.clinicaltrials.gov/ct2/show/NCT05210530
Discussion and Opinions
I'm very happy that a company is testing a combination cure, which (in theory) will both generate new beta cells and prevent the autoimmune process from destroying the new beta cells. This is something I've been hoping for for 10 years.
Normally, I'd would be happy with the speed of this trial. Finishing this year means we will get our first data very quickly. However, this clinical trial is only measuring safety outcomes, not effectiveness results. Although phase-I trials are sometimes safety only, in T1D research it is normal to include some effectiveness outcomes. They can give an early signal of success. Not having effectiveness measures makes the short duration and quick results of this study less important, because (at best) it can only show us the treatment is safe, and not that it actually works (much less, works well). We might need to wait to the end of the phase-II trial to see if it works, which will be very frustrating.
Here are two very good articles which cover this clinical trial:
And here is the official press release:
Some notes on names and terminology:
- ViaCyte refers to this product as PEC-QT, but CRISPR Theraputics calls it VCTX210 in the press release and VCTX210A in the clinical trial record.
- The stem cells are created using CRISPR/Cas9, which is an improved form of CRISPR gene editing.
- The clinical trial is called VCTX210A-101 by the companies running it, and has the clinical trial registration number NCT05210530.