Vertex Pharmaceuticals has launched a clinical trial of VX-880, a stem cell-derived beta cell therapy in type 1 diabetes (T1D) at Virginia Commonwealth University (VCU) Medical Center in Richmond, VA and four other sites across the United States.

Formal study title: A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes (T1D)

Brief background: Quietly, and perhaps without enough fanfare, a watershed moment has arrived for those of us living with T1D and JDRF’s leadership has helped make this moment possible. In the coming months, for the first time ever, people living with T1D will have new islets implanted in them that were grown in a lab – this represents the fulfillment of a dream that began two decades ago.

So, what happened 20 years ago? Researchers in Edmonton, Canada (and elsewhere) were able to transplant islets derived from a recently deceased person into a person with T1D and the patient woke up from the procedure no longer needing to take insulin shots – essentially “cured” of their T1D. But, of course, there were a few challenges with the Edmonton Protocol (as it came to be known.) Two main difficulties arose:

• Immune suppression: For those newly-implanted islets to survive, the recipients needed to take immune suppression medication (just like anyone who has had an organ transplant plus to stop the autoimmune attack on the islet that caused the T1D in the first place).
• Islets: It is very technically challenging and expensive to separate islets from cadavers. Despite major efforts, there are simply not enough islets from this source to meet the need. In any given year fewer than 100 islet cell transplants happened in the US because the supply of islets simply isn’t there.

JDRF’s research investments set about to systematically overcome these two barriers. The good news is that calibrating and adjusting the immune suppression requirements has allowed those who do receive new islets to see great survival rates of the new cells while taking a simplified regimen of immune suppressing drugs. Research continues as we try to figure out ways to avoid this need altogether, such as JDRF’s encapsulation research. But the really big news came about five to seven years ago when multiple JDRF supported researchers announced they had been able to grow islets in their laboratories. Stem cell research had finally fulfilled its promise!

Fast forward to today and the company Vertex has received FDA approval to start implanting these lab-grown cells in patients. The most transformational moments still need to start at their beginning – this first-ever trial marks (hopefully) such a beginning.

Target Patient Population: Vertex’s trial seeks to implant these cells in individuals aged 18 -65 who experience severe, documented, low blood-sugar unawareness. Read more about the trial’s full details including the specific inclusion criteria.

Trial Locations: At this time only a few sites are enrolling but luckily for those in the Mid-Atlantic, one site is at VCU Medical Center in Richmond, VA. Other sites include City of Hope (Los Angeles, CA), Diabetes Research Institute (Miami, FL), Massachusetts General Hospital (Boston, MA), and University of Pennsylvania (Philadelphia, PA).

VCU Trial Coordinator: Mary Baldecchi, mary.baldecchi@vcuhealth.org

Study: NCT04786262 – A Safety, Tolerability, and Efficacy Study of VX-880 in Subjects With Type 1 Diabetes

Interventions: Biological: VX-880; drug: Vx-880

Conditions: Diabetes Mellitus, Type 1; Impaired Hypoglycemic Awareness; Severe Hypoglycemia; Endocrine, Nutritional, Metabolic Diseases; Immunity Disorders (240-279)

If you or someone you know might be interested in this trial, encourage them to learn more by contacting Vertex or reach out to Mid-Atlantic Clinical Trials Education Volunteer, Tom Brobson, to be connected with a local trial coordinator.