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Stem cell biology is developing rapidly, as are the number of clinical trials in the regenerative medicine space that leverage cellular therapies. This growth created a compelling need for information to assess the science and safety of these approaches. Stem Cell Based Clinical Trials: Practical Advice for Physicians and Ethics / Institutional Review Boards offers physicians and institutional review boards a set of practical questions to evaluate early stage, first-in-human stem cell-based clinical approaches in order to protect patients while supporting robust research. The questions and criteria are designed to assist in the assessment of a proposed stem cell trial to ensure it is based on strong and rational preclinical evidence, and is reasonably expected to be safe, effective, and meet patient needs.
Stem cell-derived cellular interventions require a unique set of considerations that differ from those used to assess pharmacological drugs.
Stem Cell Based Clinical Trials: Practical Advice for Physicians and Ethics / Institutional Review Boards,” a report released by the International Society for Stem Cell Research
Because stem cells can give rise to all the cell types of the adult body, stem cell therapies have the potential to repair or rebuild failing organs or networks of cells that can restore and prolong human health. As translational research projects mature, the number of clinical trials using stem cell-derived approaches is increasing worldwide.
In this therapeutic area, national and international guidelines and standards are still developing, therefore each stakeholder has a responsibility to rigorously assess these trials to ensure that they are well-designed and based on strong and rational preclinical evidence. Further, stakeholders, such as physicians and institutional review boards, must determine that the potential therapy will be safe, effective, and meet patient needs. The risk of poorly designed or ineffective trials, or of testing unsafe interventions, could put patients at risk and undermine the progress of the entire field.
It became clear that many physicians and ethics / institutional review boards had little independent guidance in this emerging field to make critical assessments about stem cell-based clinical trials. Because, stem cell-derived cellular interventions require a unique set of considerations that differ from those used to evaluate pharmacological drugs, it is essential that physicians who run them, and the ethics / institutional review boards that approve them, know the appropriate questions to ask.
The ISSCR’s Stem Cell-Based Clinical Trials: Practical Advice for Physicians and Ethics/Institutional Review Committees addresses the knowledge gap generated by rapid growth in the regenerative medicine field, by offering guidance on how to assess stem cell-based clinical trials. It establishes a series of questions and criteria to determine whether a particular early-phase cell-based clinical trial is based on strong and rational preclinical evidence and is reasonably expected to meet patient needs and to be safe and effective, regardless of disease area or approach. The resource was developed by practicing physician-scientists and stem cell professionals on the ISSCR’s Clinical Translation Committee.
The ISSCR’s Stem Cell-Based Clinical Trials guide is available as a free download to anyone, though is designed specifically for practicing physicians who are conducting clinical trials and for research institutions.