Session Details

Plenary Session 1 – Mesenchymal and Progenitor Stem Cells: Results from the Clinic and the Future of Living Drugs
Friday, September 13, 2019 08:45 AM - 10:15 AM
Plenary Hall
Mesenchymal and Progenitor Stem Cells have begun to show clear evidence of efficacy outstanding safety profile in early clinical trials. This session aims to bring together some of those trials that are in the most advanced stages of testing. The presenters will each highlight the results from their efforts to with an emphasis on the lessons learned from these trials and their approach toward answering critical questions for the future of these technologies including purity, potency and logistics considerations.

Chair: Allan Dietz, PhD, Mayo Clinic, USA

Speakers:

Development of autologous adipose derived mesenchymal stromal cell therapy; Selected clinical results and lessons learned from treating more than 200 patients under IND
Allan Dietz, PhD, Mayo Clinic, USA
Mayo Clinic Rochester has actively been investigating the use of autologous adipose derived mesenchymal stromal cells for more than 10 years. We have applied these cells in more than 16 clinical trials focused on understanding the safety and feasibility of these cells in a variety of clinical conditions mostly representing patients who are refractory to any available clinical therapy. Strikingly, we have shown evidence of efficacy in some of these small (usually 20 patient) clinical trials. As the number of patients treated under IND grows to over 200, we are now focusing on developing assays to better characterize the purity and potency of our products in anticipation of testing these therapies in large, definitive trials.  

Addressing Major Areas of Unmet Medical Need with ""Off The Shelf"" Cell Therapy
Gil Van Bokkelen, PhD, Athersys, USA
Cell therapy has shown tremendous potential for addressing areas of substantial unmet medical need. The keys to unlocking this potential include achieving true scalability, demonstration of consistent safety and efficacy, and a corresponding understanding of key mechanisms of action. MultiStem represents a proprietary investigational allogeneic cell therapy that has shown promise in treating multiple critical care indications where current standard of care is limited or unavailable to many patients. Example indications where promising clinical results have been generated include the treatment of ischemic stroke (currently in Phase 3 with Fast Track and RMAT designations), acute respiratory distress syndrome (ARDS - also with Fast Track designation) and other indications.

Human Allogeneic Liver Progenitor Cells (HALPC) (HepasStem®) clinical development for Acute on Chronic Liver Failure and NASH
Etienne Sokal, MD, PhD, Promethera Université Catholique de Louvain, Institut de Recherche Clinique et Expérimentale (IREC), Laboratory of Pediatric Hepatology and Cell Therapy, Belgium
Progenitor Cells (HALPC) (HepasStem®, Promethera BiosciencesBsc, Mont-Saint-GuibertMt St Guibert, Belgium) have a specific well defined anti-inflammatory activity anti inflammatory activity mediated by PGE2 and HGF. They home to the liver and do not express HLA class II proteins. Preclinical models confirm anti-inflammatory and anti-fibrotic activities. In a phase IIa clinical trial, safety of ascending doses of Hepastem was demonstrated, and preliminary efficacy was shown on bilirubin, MELD and Child-Pugh score in Acute on Chronic Liver Failure. A large Phase II placebo randomized controlled trial is initiated. Phase I is also in progress in late stage NASH patients. Cells are delivered worldwide in dry shippers and given on site after simple reconstitution.