Understanding clinical development of chimeric antigen receptor T cell therapies

A decade of marketing approval of gene and cell-based therapies in the United States, European Union and Japan: An evaluation of regulatory decision-making

“…Trial design challenges hinder GCT approval [5], which has also been shown for promising candidates such as CAR-T [51]. It is a complex task to incorporate benefits, risks and sources of uncertainties into benefit-risk assessments, which is further complicated when endpoints differ between patient populations or over time, or when data is pooled from various studies [52].…”

Spanish Cell Therapy Network (TerCel): 15 years of successful collaborative translational research

“…These include the ARTROCELL trial (a randomized phase 3 trial comparing intra-articular administration of autologous versus allogeneic MSCs versus hyaluronic acid for knee osteoarthrosis), the NOMA (No More Amputations) trial (another phase 3 trial that compares different doses of MSCs in the treatment of diabetic foot), the AGTPII trial (a phase 2 trial that evaluates the effects of the adipose-derived graft transposition in myocardial postischemic scars), the CaSeC-HF trial (a phase 2 study assessing the safety and efficacy of intracoronary allogeneic cardiac stem cells in ischemic chronic heart failure) and a phase 2 trial that explores the role of intramuscular autologous mononuclear cells in amyotrophic lateral sclerosis. Finally, for the sixth trial (CART19-BE-02), some Ter-Cel groups have started to collaborate in new approaches to cover academic immunotherapy trials with CAR technology [16]. This trial will explore the role of ARI-001 (an academic CAR-T developed at the Hospital Clinic in Barcelona) in patients with CD19+ relapsed or refractory acute lymphoid leukemia.…”