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07-28-2007, 07:10 AM | #11 | |||
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Dottie - you are a great example; I admire what you are doing!
I am glad that the biotech involved and the FDA were so forthcoming so quickly with the news of this death. That's the "transparency" we always talk about but rarely see. It would have been easy for them to say nothing at this time. What was objectionable was the statement in the NY Times: "The case could be another setback for gene therapy, a field with a troubled history and numerous treatment failures, including the death of a teenager in 1999 in an experiment." Pure speculation, tagged on to a statement of truth, designed, I'm sure, so that the Times is being fully "transparent" when really they are just being inflammatory. QUESTION: If indeed the treatment is found to be the reason, then will the trial be stopped? Will they look carefully at what triggered the event enough to know if AAV is fundamentaly flawed, or perhaps, a mistake to use in this particular person? What is the standard for success or "effectiveness"? Does every treatment have to work for every person in order to be approved? My daughter is allergic to sulfa; she was head to toe in hives at the age of 3 when we discovered this. It seems it was just as experimental to give a toddler a drug that has a history of being allergenic to many patients. "Whoops! Now we know only to give penicillin!". She could have just as easily stopped breathing. Every drug or surgical procedure comes with a written warning to be observant for the unintended consequences of an "SAE"; if it's surgery, you must sign a statement that you have been informed and assume the risk (any surgery alone is risky regardless of its reason). It's hard to understand why adverse effects would not be expected in clinical trials when they are anticipated in every drug that is already approved and used by millions. Thanks!
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Carey “Cautious, careful people, always casting about to preserve their reputation and social standing, never can bring about a reform. Those who are really in earnest must be willing to be anything or nothing in the world’s estimation, and publicly and privately, in season and out, avow their sympathy with despised and persecuted ideas and their advocates, and bear the consequences.” — Susan B. Anthony |
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07-28-2007, 07:36 AM | #12 | |||
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Member aka Dianna Wood
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My post was very definitly on topic. Please click on link below:
http://www.caringbridge.org/cb/viewJ...od=executeInit I support stem cell research which does not kill unborn life. This was a personal story for me to share with others on this site to show a positive way to do research. This child risked as much as Caroline for research. Just because it was not for Parkinson's specifically, does not make her courageous act any less. Perhaps you would like to sign her guestbook and tell her how her sacrafice was off topic and not worth her life. Vicky Quote:
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07-28-2007, 07:42 AM | #13 | |||
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Senior Member
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Vicky -
this thread is about gene therapy, not stem cells. No embryos or stem cells were involved. It's a different thing entirely. your link doesn't work because a subscription is necessary for access. also, want to add: stem cell treatment occurs daily in the USA; it's more commonly known as "bone marrow transplant." It involves the use of adult stem cells, not embryonic. Researchers have had great success with adult stem cells, especially in blood-related diseases. It has not found success for other diseases such as Parkinson's. Thus the great debate and the need for research.
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Carey “Cautious, careful people, always casting about to preserve their reputation and social standing, never can bring about a reform. Those who are really in earnest must be willing to be anything or nothing in the world’s estimation, and publicly and privately, in season and out, avow their sympathy with despised and persecuted ideas and their advocates, and bear the consequences.” — Susan B. Anthony |
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07-28-2007, 08:04 AM | #14 | |||
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Senior Member
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I don't think anyone has suggested that the only heroes are Parkinson's patients who participate in clinical trials - they all are.
The letter from the church representative is one perspective, agreed to by many people, while many others disagree, but in any case OFF TOPIC for this particular thread.
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Carey “Cautious, careful people, always casting about to preserve their reputation and social standing, never can bring about a reform. Those who are really in earnest must be willing to be anything or nothing in the world’s estimation, and publicly and privately, in season and out, avow their sympathy with despised and persecuted ideas and their advocates, and bear the consequences.” — Susan B. Anthony |
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07-28-2007, 08:23 AM | #15 | ||
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In Remembrance
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Carey,
Good point about the New York Times. Aftermathman, thanks for the articles. Here's the statement that came into question with pipeliners - "Targeted Genetics is cooperating with FDA's investigation and has agreed to provide the agency with ongoing results from various tests and all other information it is compiling that may help determine the cause of this patient's death. FDA is also coordinating with the National Institutes of Health in an effort to acquire a better understanding of the potential scientific and safety implications of this event. These matters will be discussed at the September meeting of the NIH Recombinant DNA Advisory Committee. " Why not sooner? These are the delays that add up to years eventually. I'm sure they must be thorough in their investigation and information gathering. But it behooves us to question every delay for it's length of time. Thank you for commenting Dottie. You two are frequently in the back of my mind now, and we are here to support you every step of the way. paula
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paula "Time is not neutral for those who have pd or for those who will get it." |
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07-28-2007, 08:25 AM | #16 | |||
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Senior Member
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It's so good to hear from you.
Todd, how are you doing?
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Jean B This isn't the life I wished for, but it is the life I have. So I'm doing my best. |
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07-28-2007, 08:28 AM | #17 | ||
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In Remembrance
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Todd,
I didn't mean to exclude anyone. Did I miss a post about you being part of this trial? I am assuming that this is not Todd from CA. Sorry if I missed it and please feel free to share with us. paula
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paula "Time is not neutral for those who have pd or for those who will get it." |
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07-28-2007, 08:31 AM | #18 | |||
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Senior Member
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Treatment that consists of introducing into a patient a normal copy of one or more defective genes responsible for the patient’s disease.
When a gene is damaged, the blueprint for the construction of the protein it codes for is imperfect: the resulting protein is therefore abnormal. The goal of gene therapy is to cure a genetic disease by repairing the damaged gene responsible for the disease. It involves introducing a normal copy of the gene into cells containing the damaged version. The cells then can produce the normal protein.
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Jean B This isn't the life I wished for, but it is the life I have. So I'm doing my best. Last edited by jeanb; 07-28-2007 at 08:32 AM. Reason: missed part initially |
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07-28-2007, 08:39 AM | #19 | |||
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Member aka Dianna Wood
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Dear Indigogo,
Respectful of your degree in psychology, gene therapy is used in conjunction with stem cells in the bones when attempting to reverse the disease Arrian suffers from. Bone stem cells are mixed with a virus and the gene Arrian needs to produce the protein that is now (or was) killing her bone cells ability to make white blood cells. The link I gave is accessable if you choose to sign on to the site. There is no cost. However, since you are not interested in knowing the facts about this 10 year old girl, perhaps this local news link might be easier for you: http://kstp.com/article/stories/S150050.shtml God is in every part of our lives, espiecially at the time of our death. To refer to God's grace is never "Off Topic." Peace, Vicky Quote:
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07-28-2007, 08:56 AM | #20 | |||
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Senior Member
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Bone marrow cell transplants use ADULT stem cells.
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Jean B This isn't the life I wished for, but it is the life I have. So I'm doing my best. Last edited by jeanb; 07-28-2007 at 09:15 AM. Reason: clarity |
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