NEPHSTROM, a research project to explore the use of stem cells to treat Diabetic Kidney Disease (DKD), is being funded by the European Union’s Horizon 2020 Program http://ec.suropa.eu with Ireland acting as coordinator. DKD is the leading cause of End-Stage Renal Disease (ESRD) in Europe and the U.S where about 40 percent of all ESRD patients die within 5 years plus the fact that DKD is linked to other diseases.
In most cases of kidney damage, there is no effective medical treatment. The main treatments are drugs, dialysis, and kidney transplants but these treatments have significant costs and only provide limited protection against adverse outcomes.
A promising stem cell treatment for DKD is going to be assessed in a clinical trial. NEPHSTROM is exploring a novel therapy using a special type of stem cell known as a Mesenchymal Stromal Cells (MSC). Total costs for the project will be € 6 million and will run until April 2019.
Stromal stem cells have the ability to help regulate the body’s immune responses. MSCs have already demonstrated the potential to target major aspects of DKD including hypertension, inflammation, and fibrosis. Partners on the project believe that MSC therapy is a promising approach to slowing or halting the progression of DKD.
The trial will take place in Ireland, UK, and Italy on a particular type of stromal cell therapy called Orbcel-M which was developed by Orbsen Therapeutics http://orbsentherapeutics.com, a project partner. NEPHSTROM will conduct the Clinical Trial in 48 adult patients with progressive DKD to evaluate the safety and efficacy of an MSC treatment.
The patients as they are recruited, are pre-screened and randomized into four groups where the low-dose patients are treated first, medium dose patients are treated following verification of medium-dose safety, and lastly, the high dose patients are treated. All patients are followed-up for an extended period.
NEPHSTROM will also develop and validate a new combined manufacturing approach that would improve the production of the MSC drug and reduce costs. This approach will involve the creation of a network of four European cell production centers. The technology developed by the project will enable the network to scale up production and maintain quality.
A successful trial, demonstrating safety and indicating efficacy would provide the evidence needed to progress to a more advanced trial with a larger patient population. There is also strong research that suggests that stem cells can be beneficial in treating other chronic non-communicable diseases.