ISSCR Guidelines for the Clinical Translation of Stem Cells

A commentary published in the December 4 issue of Cell Stem Cell discusses the new International Society for Stem Cell Research (ISSCR) guidelines, which underscore major principals for responsible translational stem cell research.

A copy of the paper is available on the SMC website. To access this, please register and login to the Resource Library.

We asked Bronwen Connor, Associate Professor in Pharmacology & Clinical Pharmacology at the University of Auckland to comment on the new guidelines:

“New discoveries with clinical implications are continually being made in basic stem cell research, and stem-cell based approaches are advancing rapidly towards clinical application.  However, the general public may not fully understand how many years of preclinical and clinical research will be required to bring novel stem cell-based therapies to clinic.  Unfortunately, there are some clinics around the world already exploiting patient’s hopes by purporting to offer effective stem cell therapies for seriously ill patients but without credible scientific rationale, transparency, oversight and patient protections. The International Society for Stem Cell Research (ISSCR) is concerned that administering unproven stem cell therapies outside carefully regulated clinical trials puts individual patients at risk and jeopardizes the legitimate progress of stem cell therapies to clinic. The ISSCR has therefore developed the Guidelines for the Clinical Translation of Stem Cells to provide a framework for the responsible and timely development of clinically useful stem cell-based therapies.  These guidelines present 5 major principles, summaries below:

Independent Review and Oversight: Due to the novelty and unpredictability of early stem cell-based therapies, the ISSCR guidelines stress the importance of having stem cell experts involved throughout the development and review of stem cell-based clinical trials.  This will ensure investigators and human research review committees have stem cell-specific expertise to assess the science of preclinical studies, the proposed clinical trial and any risks that may occur. The guidelines also endorse rigorous preclinical testing in animal models where appropriate to determine the mechanisms by which stem cells may act and to identify associated risks such as tumor formation.  However the guidelines acknowledge the limitations of animal experiments in predicting how stem cells will behave in humans due to the unique facets of human physiology.  

Voluntary Informed Consent: When obtaining informed consent from research volunteers (patients), the guidelines recommend that special emphasis be placed on the unique risks of stem cell-based clinical research.  Research volunteers must be educated about the realistic potential for therapeutic benefit as they may have recourse to alternative therapies and because they may harbor misconceptions about the potential for a cure from stem cell-based therapy.

Patient Monitoring and Adverse-Event Monitoring: The guidelines state that the welfare of the research participants is of utmost importance and that their health must be carefully monitored throughout the duration of the stem cell-based clinical trial.  Given the unknowns of stem cell-based clinical research, it is very important that all positive, negative and adverse results are presented / published and assessed by experts on demand to prevent others being exposed to unnecessary risk in future clinical trials. 

Medical Innovation: The guidelines acknowledge that there may be exceptional circumstances that allow clinicians to attempt medical innovation care using stem cells outside the context of a formal clinical trial in a very small number of seriously patients, subject to stringent oversight criteria including independent expert review to assess the scientific rationale, informed consent procedures and close patient monitoring.

Social Justice and Other Aspirational Goals: The guidelines raise the issues of justice that are traditionally considered in clinical research, including the fairness of subject selection and whether the participants will receive the benefits of research discoveries and therapies.

In summary, the ISSCR guidelines are an excellent and timely set of recommendations to ensure the advancement of stem cell-based therapies to clinical and therapeutic use are undertaken in a responsible and scientifically rigorous manner.  Due to the novelty and unpredictability of stem cell-based therapies it is very important that these guidelines are adhered to in order to attend to the safety and welfare of patients / research volunteers and to ensure the field of stem cell therapy continues to advance the progress of stem cell research.  The ISSCR has stated it is committed to ongoing review of these guidelines to keep pace with new developments in the field and to protect the interests of individuals and communities.”  

Reference: Hyun I, et al. New ISSCR Guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell, 2008; 354: 607-609. 

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Further information

For information on research and ethical issues, and New Zealanders views of stem cell research, see the Ministry of Research Science and Technology website. 

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