Proven and unproven cell therapies – what we have learned so far?

Abstract

The exciting advancement in cell therapies in both cancer and regenerative medicine provides alternative therapeutic opportunities for patients with advanced diseases who have exhausted all treatment options. However, with the emergence of new technologies and innovations this also brings about “unproven” cell‐ and tissue‐based interventions that have not been fully evaluated in clinical trials. Such interventions have resulted in injuries and deaths in patients. Collectively, these practices increase the potential for patient exploitation and harm through the imposition of medical and financial risks. Recognizing this worrying situation, in April 2020, European Medicine Agency (EMA), committee for Advances Therapies advised patients and the general public to be cautious unproven cell‐based therapies. Likewise, the Food and Drug Administration (FDA) has posted information about products marketed as stem cells, mesenchymal stromal cells (MSCs) or other regenerative medicine products, including the conditions for which they are approved, and which products are not approved. Though both EMA and FDA recognize the promise of cell‐based therapies for treating patients with serious diseases, both concur that well‐designed clinical trials and regulatory oversight are necessary to ensure safety, efficacy and quality of the therapies. In this review, I will mention proven cell therapies in the haematopoietic stem cell transplant setting and in the form of chimeric antigen receptor (CAR) T‐cell therapies and briefly mention how these therapies have evolved over time, the learning curve and scientific progress in these therapies. I will then mention briefly about MSCs and focus on both the proven and unproven aspects of MSC.