Three people with type 1 diabetes who received Vertex Pharma’s islet cell therapy VX-880 have been able to come off insulin altogether within a year, raising the prospect of a life without regular injections.
The new data from Vertex’s phase 1/2 trial of VX-880, presented at the American Diabetes Association (ADA) annual congress in Florida, suggest that the therapy can restore the physiological activity of the insulin-producing islet cells of the pancreas and restore glucose control.
The study enrolled 17 people with type 1 diabetes and impaired hypoglycemia awareness, meaning they find it hard to spot the telltale signs of declining blood sugar levels and are at increased risk of severe hypoglycemic events (SHEs) that can cause confusion, coma, seizures, cardiovascular events, and even death.
The new data came from a group of 12 treated with the full dose of VX-880 given as a single infusion, three of whom had been followed up for at least 12 months and so could be evaluated for efficacy.
All three met the study’s primary endpoint of elimination of SHEs and a reduction of haemoglobin A1c levels – a marker of blood glucose control – to 7% or less, along with becoming insulin-independent at this time point.
Moreover, all 12 showed evidence that the islet cells had grafted and were producing insulin in response to glucose levels in the body within three months, and 11 of them had been able to reduce or eliminate insulin use by the data cutoff point. Of 10 participants who completed a follow-up visit on day 180, seven were no longer using insulin therapy and two had approximately cut their daily insulin use by around 70%.