Diabetes is a global epidemic. The disease and its complications and co-morbidities kill one European every minute. More than 10% of these deaths are directly linked to diabetic kidney disease (DKD). DKD and End-Stage Renal Disease consume between 10% and 15% of global healthcare spending. Outcomes for patients with diabetes, hypertension and cardio- vascular disease are much less positive, if they also have DKD. There is no cure for DKD. Even with optimal pharmacological care, patients typically progress to End-Stage Renal Disease, dialysis, transplant (where feasible) and death.
The NEPHSTROM project was designed to test and validate a novel stem cell therapy (ORBCEL-M, from Orbsen Therapeutics) For DKD. This therapy has already been shown to improve four key indicators of DKD (Glomerular Filtration Rate, or GFR; proteinurea; glomerulosclerosis; and inflammation) in mouse models. This excellent evidence, from the FP7 REDDSTAR project, has enabled us to progress to clinical and regulatory submissions for a first-in-man trial of this novel cell therapy ORBCEL-M for DKD.
As part of NEPHSTROM, clinicians in Ireland, the U.K. and Italy evaluated the clinical safety and efficacy of GMP-compliant ORBCEL-M in a Phase 1b/2a clinical trial in patients suffering from DKD. While safety was the primary endpoint, we were also looking for initial indications of efficacy, to encourage further trials.
NEPHSTROM is also addressing the challenge of scaling cell production to meet increased clinical demand, as soon as clear positive clinical results are published. This is a key challenge for any cell therapy, including ORBCEL-M. In NEPHSTROM, we are establishing and validating a network of cell production centres, using common cell stock, across Europe. This is a key enabler for any later stage clinical trial, and for clinical use. The cell production and clinical work carried out within the NEPHSTROM project was supplemented and supported by a programme of investigation into the efficacy, mechanism of action, immune response and bio-distribution of ORBCEL-M in animal models. In addition, the project evaluated the economic benefit of this novel cell therapy relative to current treatment scenarios. The information gained here will be critical for planning for future later-stage clinical trials.
