Research towards a product
FP7 call HEALTH.2013.0-1 target
“Boosting the translation of health research projects’ results into innovative applications for health”
and the HESUB Collaborative Project
“High Efficient Single-Use-Bioreactor simulating mammalian tissue conditions for expression and proliferation”
did over from May 2013 to April 2016 develop the here show product and procedures
for stem cell mass production for therapeutic treatments
The presented HEUSB project combines several individual technologies from previous FP6 projects into one product that is capable of producing enough stem cells for one therapeutic treatment per day per unit. The HESUB product concept is a Single-Use-Bioreactor, which integrates a nano-fibre porous scaffold optimised for the proliferation of mammalian cells and a sensor package that measures a range of key parameters, and which provides cost-efficient production of human stem cells for therapeutic treatment of a range of diseases including heart disease, diabetes, neurodegenerative diseases, musculoskeletal disorders, spinal cord injury, stroke and autoimmune diseases.
The Proof-of-Concept (PoC) of the new bioreactor focuses first on cell therapeutic application to Muscular Dystrophies (MD) including Duchenne Muscular Dystrophy (DMD). For DMD treatment as approach in a previous European project, MYOAMP, injection of adult myoblasts has led to the development of new muscle fibres, but several limitations have been identified, such as poor cell survival and limited migratory ability and limited injectable cell numbers. As an alternative to myoblasts, myogenic stem cells are preferable for therapeutic applications due to their capacity for self-renewal and myogenic differentiation potential.
Approaches in muscular dystrophy treatment are multiple nowadays; stem cell-based replacement therapy is one of these. Other methods targeting at gene level, e.g. adeno-associated virus treatment correcting mal-functioning dystrophin (exon-skipping) for DMD treatment, have become highly promising. Stem cell-based therapy is viewed today as a solution for these patients or as a complementary treatment for instance in the case of insufficient precursor cell pool in the patient. Large-scale stem cell production is a key for therapy today.