Mesoblast (ASX:MSB) has announced that results of the Phase 2 trial of its intravenously-delivered Mesenchymal Precursor Cells (MPCs) for the treatment of type 2 diabetes have been published online ahead of full print in the peer-reviewed journal of the American Diabetes Association, Diabetes Care.
The article is entitled 'Allogeneic Mesenchymal Precursor Cells in Type 2 Diabetes: A Randomized, Placebo-Controlled, Dose Escalation Safety and Tolerability Pilot Study'.
Trial investigator and co-author, Dr Vivian Fonseca, Professor of Medicine and Chief of Endocrinology at Tulane University, and past President of Medicine and Science for the American Diabetes Association, said: "We are very encouraged by the safety profile and suggestion of efficacy of Mesoblast's cell therapy in patients with type 2 diabetes."
Type 2 diabetes is associated with aberrant immune activation and inflammation of various organs, including kidney, liver and fat tissues, resulting in resistance to the effects of insulin in the fat tissues, and poor glucose control.
The most frequent end-organ complication involves the kidney, a condition termed diabetic nephropathy, which affects 40-50 per cent of patients with type 2 diabetes.
Mesoblast's product candidate MPC-300-IV is being developed to treat systemic conditions of excessive inflammation, including type 2 diabetes and its complications, by down-regulating the production of pro-inflammatory cytokines and increasing the production of anti-inflammatory cytokines.
As a first step in developing an MPC-based immunomodulatory therapy for the treatment of type 2 diabetes and its complications, a study was performed which evaluated three escalating doses in patients with type 2 diabetes and poor glycemic control, without kidney disease or other organ involvement. The Phase 2 randomised, single-blind, placebo-controlled trial was conducted across 18 sites in the US and evaluated the effects of a single intravenous infusion of 0.3, 1.0 or 2.0 million MPCs/kilogram (kg) or placebo over 12 weeks in 61 patients with a mean diabetes duration of 10 years.
According to the company, the primary objective of the study was to assess the safety and tolerability of a single intravenous infusion of Mesoblast's MPC therapy (rexlemestrocel-L) in patients with type 2 diabetes. Secondary objectives included evaluation of treatment effect on glycemic control, defined by hemoglobin A1c (HbA1c) measurement, the gold-standard measure of long-term blood sugar control.
On the primary endpoint of safety, the study found that a single MPC infusion ranging from 0.3 to 2.0 million MPCs/kg was safe and well tolerated, with no treatment-related adverse events, and no serious adverse events over the 12-week study period.
On the secondary efficacy endpoints, the study found that, following a single intravenous MPC infusion, there was an improvement in glycemic control as evidenced by a decrease at all timepoints after week 1 in HbA1c in MPC-treated patients compared with an increase in HbA1c in placebo-controlled patients.
"The highest dose (2.0 million MPCs/kg) showed the greatest overall reduction in HbA1c, with a peak decrease of 0.4% at 8 weeks compared with placebo (p less than 0.05)," the company said. "The clinical target of good glycemic control, HbA1c less than 7%, was achieved by 5/15 (33%) subjects in the MPC 2.0 million/kg group vs. 0/15 (0%) in the placebo group, (p less than 0.05)."
Professor Paul Zimmet, Director Emeritus of the Baker IDI Heart and Diabetes Institute, and Foundation Director of the International Diabetes Institute, said: "The treatment of type 2 diabetes with existing pharmaceutical therapies remains a major challenge to obtain optimal metabolic control for both wellbeing and reducing the risk of serious complications such as heart, kidney and eye disease, and amputations.
"This study is important and encouraging in the context of the search for better treatment for this ubiquitous and serious disorder. Given the pressing need for new developments in the treatment of type 2 diabetes, these findings are encouraging and support further studies on the possible use of adult stem cells for treating diabetes and its complications in future."