Towards a Consensus Potency Assay for Mesenchymal Stromal Cells: A Matrix Analysis of Cell Source, Donor Variability, and Inflammatory Stimuli to Refine Surrogate Markers of Immunomodulation

The document from 2019 presents two separate studies. The first study evaluated the efficacy of ATIR101, a T-cell immunotherapy, in 37 patients undergoing haploidentical hematopoietic stem cell transplantation (haplo-HSCT) compared to a historic control group of 35 patients. The ATIR101 group showed a significantly higher overall survival rate of 58% versus 20% in the control group (p=0.00351), lower non-relapse mortality (33% vs 66%), and lower relapse-related mortality (8% vs 15%). The incidence of total chronic graft-versus-host disease (GVHD) was also lower in the ATIR101 group (3% vs 11%), while acute GVHD grade III-IV was similar between the two groups. The study concluded that ATIR101 improved overall survival and survival without severe complications compared to the control group, supporting its further investigation in a phase III trial. The second study focused on the impact of autologous stem cell interventions in Australia, based on two workshops with 22 participants and 15 interviews conducted in 2016 and 2017. It identified five factors influencing patients' decisions to undergo such interventions and discussed the effects of a new ban by the Australian Therapeutic Goods Administration (TGA) on direct-to-consumer advertising of autologous cell and tissue products. The study found some improvements in marketing practices post-ban but stressed the need for ongoing regulation to protect patients, as the previous lenient regulatory environment had allowed for the exploitation of patients and the marketing of unproven treatments.