A novel cell therapy trial for individuals with diabetes has produced encouraging results, according to researchers. The NEPHSTROM clinical research is starting to look at the possibility of a novel cell therapy to treat persons with type 2 diabetes who are still experiencing kidney impairment after getting extensive medical care. At the American Society of Nephrology’s Kidney Week conference in Orlando, Florida, the findings of the NEPHSTROM clinical study were presented in November. It demonstrated that a single intravenous dosage of ORBCEL-M was safe and linked with greater renal function preservation than a placebo when administered to carefully chosen persons with progressive kidney disease owing to diabetes.
After receiving ORBCEL-M, study participants were continuously monitored for an additional 18 months. Orbsen Therapeutics Ltd., a spinout firm from the University of Galway, discovered and developed the ORBCEL-M cell treatment in Galway using mesenchymal stromal cells (MSC) prepared from healthy bone marrow. The Mario Negri Institute for Pharmacological Research IRCCS in Bergamo, Italy, is overseeing the clinical trial, which is being conducted in collaboration with top medical facilities in Galway, Bergamo, Birmingham, and Belfast.
Professor Matt Griffin, the trial’s principal investigator and a consultant nephrologist at Galway University Hospitals’ Regenerative Medicine Institute (REMEDI), said: “In Ireland, there are close to a quarter of a million diabetics, and we know that more than 40% of them have kidney disease, also known as diabetic kidney disease or DKD.
“In type 2 diabetes, as many as one-third of those with DKD have worsening kidney function despite the best medical therapy we can offer. These people are at high risk for requiring dialysis or kidney transplantation in the years ahead – both of which are complex treatments with potentially serious complications.
“In NEPHSTROM, our goal is to secure evidence that a cell therapy, such as ORBCEL-M, is safe and can slow the course of DKD to help more people with diabetes avoid the need for dialysis or transplantation. It was exciting to report that our first analysis of results from the trial supports that goal.”
Dr Steve Elliman, who discovered the ORBCEL-M therapy, is Chief Scientific Officer for Orbsen Therapeutics. Dr Elliman said: “At Orbsen Therapeutics we are motivated by improving patient care. Diabetic patients with progressive kidney disease eventually require dialysis and often a kidney transplant. While dialysis improves the quality of life of patients with kidney failure, it is expensive and does not prevent further decline of kidney function.
Additionally, dialysis takes four hours per session and three times a week – creating logistic and economic challenges for the patient. Our goal with ORBCEL-M is to resolve systemic inflammation and improve kidney function, so that patients will not require dialysis or a kidney transplant. We’re encouraged by the safety profile and the preliminary efficacy signals in patients with DKD reported by the NEPHSTROM trial. We look forward to continued collaboration with our University of Galway and NEPHSTROM partners to advance this new medicine through Phase 3 efficacy trials and a market approval.”
Dr Veronica McInerney, Administrative Director at the HRB Clinical Research Facility at University of Galway said: “Without patient involvement in clinical trials, advances in new treatments are simply not possible. We are fortunate to have the HRB Clinical Research Facility Galway, a clinically equipped space to see and treat patients on trials. We are hopeful that future generations will benefit from the willingness of patients to participate in trials, such as the NEPHSTROM trial.”
Professor Timothy O’Brien, Director of the Regenerative Medicine Institute (REMEDI), at the University of Galway and Consultant Physician in Endocrinology at Galway University Hospitals and the overall lead of the NEPHSTROM project, said: “University of Galway’s ecosystem is set up to facilitate and lead international trials of this nature.
The Cell Therapy GMP manufacturing facilities at the Centre for Cell Manufacturing Ireland, located in the University, along with the HRB Clinical Research Facility, the close partnership with Saolta University Heath Care Group and REMEDI have been instrumental in making the progression of this potential new therapy possible. Funding from SFI, the Higher Education Authority and the Health Research Board has supported and helped build this ecosystem and along with European Commission funding has made the advancement of this research possible.”