Work

Understanding the Patient Experience and Potential Barriers to Enrollment and Participation in Cell and Gene Therapy Trials in AML

Year: 2025

Type: article

Abstract: Introduction Cell and gene therapies (CGT) are new and emerging treatments in oncology . Unlike traditional trials, CGT involves complex mechanisms involving cell and gene modification, which can impact the patient experience in unique ways. This inherent complexity may result in lack of understanding, emotional concerns, logistical barriers, or various other issues that affect a patient's willingness to participate. Clinical trial site staff, including social workers and research coordinators/nurses, play a key role in supporting patients from enrollment through treatment and offer valuable insights into the patient experience. Method In this initial pilot study, we conducted a multiple-choice survey and interviews with seven research coordinators/nurses and interviews with two social workers at treatment centers across North America that offer cell and gene therapy trials in AML to learn about the patients’ experiences and potential barriers to trial enrollment. The multiple-choice survey and interviews included questions related to time and resources required to explain CGT trials, patient concerns, barriers to enrollment, and psychosocial issues that may hinder trial participation. Results Most coordinators and nurses believe CGT trials take longer or are more difficult to explain compared to a non-CGT trial. One participant reported difficulty explaining a CAR-T trial as there are no similar, approved therapies in AML . Additional concerns noted by most respondents included side effects , extensive follow-up, and financial constraints, which were identified as the main barriers to CGT trial enrollment. One respondent noted that patients often worry about increased study follow up demands when compared with standard of care or non-CGT trials. Social workers identified the same barriers, but added that psychosocial and caregiver concerns also play a significant role due to the increased need for monitoring and extended housing. Both social workers noted that patients worry about committing to a CGT trial because the financial and time burden to their family may outweigh the unknown benefits of the trial. Conclusion This pilot study revealed that patients considering CGT trials encounter many of the same barriers as non-CGT trials, but may face additional challenges due to the complexity of these novel therapies. These findings lay the groundwork for expanding our research to conduct interviews with more social workers and broader patient care teams. Gathering insights from these key stakeholders will facilitate the development of resources to support both patients and staff and address logistical and socioeconomic barriers that limit access to CGT trials. By overcoming these challenges, we can potentially improve the design of future studies to ensure that more eligible patients have equal ability to participate in clinical trials evaluating these advanced therapies.

Source: Transplantation and Cellular Therapy

DOI: 10.1016/j.jtct.2025.01.554