In 2010, over 634,000 EU citizens died as a result of diabetic complications and co-morbidities (~1 death every minute), totalling 11% of all deaths in EU adults aged 20-79 years. It is estimated that 10-20% of these deaths occurred due to renal failure caused by diabetic kidney disease (DKD). DKD comprises both diabetic nephropathy (DN) and other renal lesions associated with progression of chronic kidney disease (CKD).
DKD is the single leading cause of end stage renal disease (ESRD) in the industrialised world, accounting for ~40% of new cases of ESRD in the US and EU. The five year mortality rate is 39% – a rate comparable to many cancers. The economic cost of DKD and its progression to ESRD represents a staggering 13.4% of the US healthcare (Medicare) budget.
In spite of this enormous social and economic cost, there have been no specific therapies successfully developed for DKD in the past 25 years.
NEPHSTROM proposes a new therapy that addresses DKD, but that also shows great promise for diabetes, its resultant complications, and cardiovascular disease.
To address the critical unmet need for a therapy for DKD, NEPHSTROM brings together key leaders in the clinical treatment of chronic kidney disease, leading researchers of the mechanisms of diabetic nephropathy, and experts in stromal cell therapy development in a multi-disciplinary team.
The NEPHSTROM team will carry out a first in man Phase 1b/2a clinical trial of a novel stromal cell therapy (Orbcel-M) for DKD. This treatment has shown exciting efficacy in pre-clinical models of diabetic nephropathy and, unlike single biological or pharmacological therapies, improves several facets of this chronic disease. We have demonstrated in a current FP7 project, REDDSTAR, that a single intravenous injection of stromal cells can simultaneously improve GFR, decrease proteinuria, reduce glomerulosclerosis and limit associated inflammation in preclinical models of type 2 (db/db) diabetic nephropathy – the major lesion that is associated with DKD. Our REDDSTAR data is also consistent with a variety of independent preclinical studies on the effects of stromal cells in pre-clinical models of diabetic nephropathy. This data is compelling and provides the rationale for undertaking the proposed clinical trial.
NEPHSTROM will establish the clinical safety and preliminary efficacy profile of Orbcel-M – a next generation, antibody-purified population of Mesenchymal Stromal Cells (MSCs) from human bone marrow – for the treatment of DKD in order to develop an entirely new treatment option for patients with this devastating disease.