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Q: Do clinical trials ever go wrong?

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A test to check for cancer of the cervix, the opening to a woman's womb. It is done by removing cells from the cervix. The cells are then prepared so they can be seen under a microscope.

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 Subject :World First Stem Cell Clinical Trial: Q & A with Johns Hopkin's St.. 2010-10-13 16:07:12 
Joined: 2009-07-13 19:19:42
Posts: 23
Subject :World First Stem Cell Clinical Trial: Q & A with Johns Hopkin's Stem Cell Expert Candace Kerr

  1. What would you tell spinal cord patients about news that a safety study looking at human embryonic stem cells has started? Is this good news?
    This trial is a pivotal step forward for understanding how human embryonic stem cells will be able to treat patients with SCI. Importantly, patients should expect that this is just a first step of many that will be required for such a consuming affliction.
  2. How promising is the approach that Geron reports in this trial? What are key hurdles or challenges it might face from your viewpoint? What approaches do you see as more, or equally, promising?
    It is critical that this trial will test for the first time how human derived stem cells behave in patients. Without knowing the specifics it still appears that cell tracking and cell function will be difficult to determine in live patients. These features will be important to assess whether injected cells move out of the spinal cord or whether they replace and perform the function of the cells damaged by SCI. For instance, many ongoing clinical trials with human mesenchymal stem cells have shown short-term improvement only, and that the improvement is caused by factors the injected cells secrete that help in the recovery of the patient's own tissue or by reducing damage caused by inflammatory responses from the immune system.
  3. What would you tell patients who want to explore a clinical trial such as this one?
    Patients should go into these trials understanding that its purpose is to determine whether the transplantations are safe. The purpose of Geron's animal trials where these cells were tested for FDA approval would have included studying any adverse effects. Main adverse effects would be uncontrolled growth of cells which may lead to tumors or movement of the transplanted cells out of the spinal cord into other areas of the body. In addition, patients would need to understand that they may not see any immediate benefits or recovery, while nonetheless, these trials will provide invaluable information that will pioneer the field of stem cell based therapies. Not just SCI but for other neurological disorders like MS and ALS, where these cells also play a significant role in the disease.
  4. Is it worth reporting the news, or it is unfairly raising expectations just to report it? Why, or why not?
    I do not believe you will be able to ignore this story as it unfolds as many are anticipating a significant outcome from this trial. Whether justifiably so or not, this trial will put the clinical utility of these cells in perspective for not just SCI but for other afflictions, as well. Many believe that until we can show clinical benefit of these cells, their utility and justification for studying them will constantly be under public scrutiny and skepticism. It is important to understand that the purpose of this trial is safety. So what does that really mean? The patients receiving treatment for this trial have the most severe levels of SCI in which they have complete loss of functional and sensory output in the lower torso. Thus, the level of damage may be so severe that the strategy as it is tested for the first time in this trial may not overcome all the obstacles associated with such severe injury or that only minor recovery is achieved. If this occurs the greatest injustice will be to report that the cells do not work. I think this is where scientists are holding their breath, that "what if" lack of improvement will be reported and seen by the public as a failure. This may not be the case at all in that it may significantly improve less severe levels of injury or with improvements in the implementation of the cell based strategy along with other drugs this may produce improved outcomes in further studies.
  5. Are there any key points, or other information worth noting, in a news report about this? Geron's position in the hESC universe has always been interesting to people in the field, but not something the general reader can appreciate, for example.
    Geron's investment in making this clinical trial happen attests to the importance of government funding to support this type of research. For instance, only a handful of laboratories, including our own have demonstrated the ability to generate this specific cell type used in Geron's trial from human embryonic stem cells. Therefore, it is essential that federal funding remain an integral part of supporting the endeavors of nonprofit institutions to study how to grow and to generate the specific cell types from human embryonic stem cells.
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Last Edited On: 2010-10-13 16:07:12 By Leon for the Reason
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