Philadelphia, PA -- The Internet is rife with provider Web sites and patient testimonials touting exciting new stem cell treatments for paralysis, Parkinson’s disease, heart failure and cancer. And every year, desperately ill patients travel thousands of miles to seek experimental stem cell therapies with bold claims of cures for such intractable diseases.
Few, if any, of these putative treatments are based on sound pre-clinical data that have been published in peer-reviewed journals. Today, there are no proven treatments based on stem cells other than those for blood disorders, like leukemia and immune deficiencies. All other stem cell treatments are still highly experimental.
At the sixth annual meeting of the International Society for Stem Cell Research, a task force of leaders in the field announced a draft set of guidelines to ensure that rigorous best practices are applied to the clinical translation of stem cell research from the laboratory to human subjects. The guidelines are the culmination of months of discussion by scientists, ethicists, public policymakers, clinicians, industry representatives and members of the public from 13 countries. (The full guidelines will be posted at www.isscr.org.)
“These guidelines are critically important to the future success of the field,” according to Dr. George Q. Daley, ISSCR president and associate director of the Stem Cell Program at Children’s Hospital Boston. “Not only does the use of untested therapies put patients at risk, it jeopardizes the legitimate practice of all translational stem cell research.”
In strongly worded language, the guidelines “condemn” the use of stem cell therapies outside of an established clinical trial, particularly when patients are charged for “advertised medical services that constitute experimental, unproven and unestablished interventions.” The guidelines call upon regulators in countries where these putative therapies are offered to regulate such practices to prevent exploitation of vulnerable patients. The guidelines call upon scientists and clinicians to conduct research with transparency and adherence to rigorous independent scientific and ethical review.
The guidelines address three major areas of the clinical translational process involving all types of human stem cells and their direct derivatives, including cell processing and manufacturing; pre-clinical studies; and clinical research. The guidelines also make specific recommendations for ethical oversight, peer review of studies, informed consent and protection of subjects, avoidance of conflict of interest, clinical trial design and reporting, and long-term follow up of patients. Also addressed are issues of social justice, such as public engagement in policy making and fair access to treatment, in particular, offering affordable treatments to patients in resource-poor countries.
The guidelines do allow for treating a specific patient or small numbers of patients outside of a clinical trial in “exceptional circumstances of justified medical need” where reasonable scientific evidence exists that the patient or patients might benefit and not be harmed. All clinical trial regulations related to informed consent, clinical follow-up, independent expert review and institutional support and accountability still apply.
Following a solicitation of comment period from June 12 to Sept. 15, 2008, the task force will develop a final version of the guidelines, expected to be released by the end of the year. At that time, the ISSCR will release an advisory for patients considering stem cell therapy to help guide them in evaluating programs and treatments. The advisory will be posted on the ISSCR Web site, www.isscr.org.
The guideline’s task force was chaired by Dr. Olle Lindvall and co-chaired by Dr. Insoo Hyun. Four working groups participated in their development: the Quality Control and Manufacturing Subcommittee, chaired by Dr. Mahendra Rao; the Preclinical Testing Subcommittee, chaired by Dr. Giulio Cossu; the Clinical Trials Subcommittee, chaired by Dr. Ira J. Fox; and the Social Justice Subcommittee, chaired by Dr. Laurie S. Zoloth.
The International Society for Stem Cell Research (ISSCR) is an independent, nonprofit membership organization established to promote and foster the exchange and dissemination of information and ideas relating to stem cells, to encourage the general field of research involving stem cells and to promote professional and public education in all areas of stem cell research and application.
Posted June 16, 2008