How do I: Evaluate the safety and legitimacy of unproven cellular therapies?. Issue 3 (10th February 2022)

• Record Type: Journal Article
• Authors: Guleria, Indira; de Los Angeles Muñiz, Maria; Wilgo, Matthew; Bapat, Asawari; Cui, Wanxing; Hsu, Yen‐Michael Sheng; Jeyaraman, Madhan; Muthu, Sathish; Rodriguez, Federico; Fesnak, Andrew; Celluzzi, Christina; Sesok‐Pizzini, Deborah; Reich‐Slotky, Ronit; Spitzer, Thomas
• Is Part Of: Transfusion. Volume 62:Issue 3(2022)
• Page Start: 518
• Page End: 532
• Publication Date: 2022-02-10
• Subjects: adverse events -- biological products -- Food and Drug Administration (FDA) -- hematopoietic stem cells -- investigational new drug application (IND) -- regulatory agencies -- stem cell tourism -- toolkit -- unapproved cellular therapies -- unproven cellular therapies; Hematology -- Periodicals; Blood -- Transfusion -- Periodicals; Blood Group Antigens -- Periodicals; Blood Preservation -- Periodicals; Blood Transfusion -- Periodicals; 615
• DOI: 10.1111/trf.16814
• Languages: English
• ISSNs: 0041-1132

Abstract

Abstract: Background: Unproven cellular therapies are being offered to patients for a variety of conditions and diseases for which other treatments have failed. The use of untested cellular therapies is a worldwide problem. Practitioners (e.g., physicians, scientists, QA/QI facility managers, and policy advocates) are perhaps unaware of the risks involved with such therapies. Therefore, a critical need exists to bring attention to the potential limitations and adverse effects of these therapies to inform and limit misinformation. Study Design and Methods: We describe the extent of the unproven cellular therapy problem through a search of scientific literature and social media coverage. We also describe the regulatory framework that can be used by the practitioner to review and evaluate both proven and unproven cellular therapies. Results: We report on the current state of unproven cellular therapies across the globe. A workflow to facilitate an understanding of the regulatory processes involved in the approval of cellular therapies is provided as well as a list of warnings required by regulatory agencies on various products. It is hoped that this article will serve as a tool kit to educate the practitioner on navigating the field of unproven cellular therapy products. Discussion: Increasing awareness of the issues associated with unproven therapies through education is important to help in reducing misinformation and risks to patients.