19TH Global Cardio-Vascular Clinical Trialists Forum / 1-3 December 2021
Session : THE INNOVATION LANDSCAPE Part 2: GENE, CELL AND RARE DISEASE THERAPIES. PATHWAYS FOR APPROVAL
The Potential of Expanded CD34+ Stem Cells
By Ibon GARITAONANDIA – CSO Cellprothera, France
We have shown in a pilot study that injection of autologous CD34+ cells in patients with acute myocardial infarction is safe and leads to long term improvement with an average follow-up of 14 years. Left ventricular ejection fraction values significantly increased more than 18 percentage points two years after injection, much higher than the standard of care. Three patients who were initially scheduled for early heart transplantation, no longer required the heart transplant after cell injection. Patients also experienced New York Heart Association grade improvement and myocardial structure regeneration and revascularization with recovery of contractility in the previous akinetic area. After the successful results of the pilot study, CellProthera decided to conduct a multicenter, randomized, controlled Phase 2 clinical trial in severe acute myocardial infarction to investigate the safety and efficacy of expanded autologous CD34+ cells, also known as Protheracytes® (ClinicalTrials.gov Identifier: NCT02669810). Protheracytes® are registered by European Medicines Agency as an advanced therapeutic medicinal product (ATMP) and are manufactured using CellProthera’s proprietary StemXpand system, which is an automated device for expanding and manufacturing autologous peripheral blood CD34+ cells. The Phase 2 clinical trial is currently enrolling patients but preliminary results indicate that the delivery of Protheracytes® directly into the myocardium is feasible and safe and the efficacy data is promising, in line with the pilot study results.