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Future trials and treatments - cautions and guidelines
Clinical trials will likely be starting up in the next two years. Offshore clinics are already offering 'treatments' but are providing no peer reviewed research articles to suggest they have overcome exisitng challenges. In this section we provide cautions and guidelines for patients thinking about participating in future trials and treatment opportunities.
Challenges and risks
As promising as stem cell research appears to be, there are a number of challenges to be met before stem cells could become a safe and effective therapy. Pre-clinical studies are needed to demonstrate efficacy and safety, but researchers are aware that animal models do not always predict how humans will respond to the same intervention. One major concern is whether the use of stem cells would cause the development of cancerous tumors over time.
The International Society for Stem Cell Research (ISSCR) has identified a number of areas that need to be addressed in their Guidelines for the Clinical Translation of Stem Cell Research which were developed by "stem cell researchers, clinicians, ethicists and regulatory officials in thirteen countries." (see link below). Thirty-nine recommendations address areas such as the need to demonstrate efficacy and safety in preclinical studies, for peer review of research, for independent review and oversight of proposed trials, the communication of potential risks and benefits and obtaining informed consent, and for frank reporting of failures and adverse events.
Offshore stem cell clinics
In the last few years, stem cell clinics have been established in China, the Soviet Union, and other countries and are being marketed to patients through the Internet. Is it possible that their researchers have somehow leapfrogged over those from the United States and other countries where the researchers are still involved in preclinical research and come up with safe and effective treatments? They provide no scientific evidence to suggest that this is so.
An article which appeared in the journal Cell Stem Cell reviewed the claims on the 19 websites the authors were able to find. Sixteen of these websites offered treatment for neurological problems. The authors found that the websites inaccurately portrayed the treatments as "safe and effective for a broad range of diseases in the context of routine clinical use." Potential side effects were downplayed and testimonials were presented instead of citations to research articles in reputable scientific journals. The treatments were expensive, averaging around $25,000. The authors searched the literature but could not find peer-reviewed research which supported the treatments.
The International Society for Stem Cell Research has expressed concerns about these clinics:
Numerous clinics around the world are exploiting patients' hopes by purporting to offer new and effective stem cell therapies for seriously ill patients, typically for large sums of money and without credible scientific rationale, transparency, oversight, or patient protections. The ISSCR is deeply concerned about the potential physical, psychological, and financial harm to those who pursue unproven stem cell ‘therapies' and the general lack of scientific transparency and professional accountability of those engaged in these activities.
A disturbing case report
An article has been published in PLoS Medicine which reports on the case of a young Israeli boy with ataxia telangiectasis , a neurodegenerative disorder. Against the advice of his doctors, his parents took him to a Russian stem cell clinic where beginning in 2001 he received several injections of human fetal neural stem cells into the brain and the surrounding fluid. No improvement was seen in his condition. In 2005 he complained of headaches and an MRI revealed tumors in the brain and on the spinal cord. The tumors in the brain were slow growing and have not been removed but the growth on the spinal cord was removed.
The tumor was analyzed and it contained DNA from at least two donors, one of whom was female. This shows that the tumor was not something that the boy might have developed anyway but was caused by the stem cell injections.
The ISSCR issued a press release about the case. press release
If ongoing preclinical research suggests that some form of stem cell treatments are likely to be safe and effective, researchers may proceed to clinical trials. The progress to date has been exciting and hopeful but challenges remain. When trials do begin they will involve patient volunteers who are fully informed about the trial and the risks and carefully monitored throughout the trial and afterwards. Safety data will be continually analyzed and patients will not be charged for their participation.
At this time, if a clinic is charging for unproven ‘treatments' or trials, promising ‘cures', and citing testimonials rather than solid scientific research and if details about procedures and ‘formulas' are clouded in secrecy, potential patients should recognize that these are warning signs that the clinic or trial is not legitimate.
Guidelines and cautions
Dr. Leslie Thompson, stem cell researcher and Coalition for the Cure member, has urged caution. In a letter writen with colleagues she notes that extensive animal research is required before clinical trials can proceed safely and recommends that ISSCR guideline be followed. Dr. Thompson's letter : link
In addition to the guidelines, the ISSCR has written a helpful patient handbook.
Ninette Amariglio, Abraham Hirschberg, Bernd W. Scheithausen, Yoram Cohen, Ron Loewenthal, Luba Trakhtenbrot, Nurit Paz, Maya Koren-Michowitz, Dalia Waldman, Leonor Leider-Trajo, Amos Toren,Shlomi Constanti, and Gideon Rechavi. "Donor Derived Brain Tumor Following Neural Cell Transplantation in an Ataxia Telangiectasia Patient." PLos Medicine Vol 6:2 (February 2009): 221-31.
Darren Lau, Ubaka Ogbogu, Benjamin Taylor, Tania Stafinski, Devidas Menon, and Timothy Caufield. "Stem Cell Clinics Online: The Direct-to-Consumer Portrayal of Stem Cell Medicine." Cell Stem Cell Vol. 3 (December 4, 2008):591-4.