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  1. #1

    Off Topic Discussion

    Quote Originally Posted by Nowhere Man View Post
    So hypothetically, let's say that in the Neuralstem trial, that it is shown to be safe in the Asia A patients, but had no signs of efficacy. Do you think they should then abandon any future trials for other types of SCI injuries (ex: incomplete and/or sub-acute) with this same treatment? Even though it has shown signs of modest efficacy in the other injury type animal models?
    Quote Originally Posted by tomsonite View Post
    No, I don't think that at all. Where did you get the idea that I would?
    I don't think that animal research should be completely ignored. But it should also not be the only thing that decides how research is done in humans, or what research does or doesn't get done in humans.
    This is a general opinion of mine and not directed at any specific human trial:

    You gave the logical answer. You wouldn't let a lack of efficacy in chronic, complete human patients stop you from starting other trials in different injury types. My point is that current trials in chronic, complete SCI are not about efficacy in any meaningful way, they are truly about safety. Sure, if it shows some signs of efficacy it would be great for the given company, but that's not what the trial is about. They're about safety. Essentially, we chronic & complete human patients are just the guinea pigs to make sure there are no harmful effects.

    My main concern is that the chronic, complete patients are putting their bodies at high risk and are wasting their time (months) just to prove safety of these cells. If each patient knew that going in, then I have no issues with that (maybe they do know). It's a free society. However, most SCI are not knowledgeable in the science arena and drool at the term "stem cells". They are unaware of what they are and what they can do. They probably think that they might get substantial recovery, even though there is absolutely no animal study to justify that thought. Especially those within a few years of injury. Meanwhile, the chronic & complete population get all sorts of hope that a human trial is underway that is testing on someone with their injury type; unaware that it is basically just for safety and that there has been no efficacy shown in chronic, complete animals, nor is that the company's goal to achieve. I'm not saying this is the case in reality, but I'm saying that it worries me that it might be the case.

    If companies actually wanted to try to treat the human chronic & complete population, why not do an animal study in chronic & complete animals first? (Maybe they have but had no positive results.) We know it can be done. Who in their right mind would go from straight from the drawing boards to a human clinical trial, without any relevant animal studies? What company would waste tens of millions on a human trial without a successful animal trial? None.
    Last edited by Nowhere Man; 05-17-2014 at 06:38 PM.

  2. #2
    Senior Member khmorgan's Avatar
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    Quote Originally Posted by Nowhere Man View Post
    This is a general opinion of mine and not directed at any specific human trial:

    You gave the logical answer. You wouldn't let a lack of efficacy in chronic, complete human patients stop you from starting other trials in different injury types. My point is that current trials in chronic, complete SCI are not about efficacy in any meaningful way, they are truly about safety. Sure, if it shows some signs of efficacy it would be great for the given company, but that's not what the trial is about. They're about safety. Essentially, we chronic & complete human patients are just the guinea pigs to make sure there are no harmful effects.

    My main concern is that the chronic, complete patients are putting their bodies at high risk and are wasting their time (months) just to prove safety of these cells. If each patient knew that going in, then I have no issues with that (maybe they do know). It's a free society. However, most SCI are not knowledgeable in the science arena and drool at the term "stem cells". They are unaware of what they are and what they can do. They probably think that they might get substantial recovery, even though there is absolutely no animal study to justify that thought. Especially those within a few years of injury. Meanwhile, the chronic & complete population get all sorts of hope that a human trial is underway that is testing on someone with their injury type; unaware that it is basically just for safety and that there has been no efficacy shown in chronic, complete animals, nor is that the company's goal to achieve. I'm not saying this is the case in reality, but I'm saying that it worries me that it might be the case.

    If companies actually wanted to try to treat the human chronic & complete population, why not do an animal study in chronic & complete animals first? (Maybe they have but had no positive results.) We know it can be done. Who in their right mind would go from straight from the drawing boards to a human clinical trial, without any relevant animal studies? What company would waste tens of millions on a human trial without a successful animal trial? None.
    Maybe I'm missing something you are saying, but all human trials consist of 3 phases. See http://www.fda.gov/drugs/resourcesfo.../ucm143534.htm

    Phase 1 studies are usually conducted in healthy volunteers. The goal here is to determine what the drug's most frequent side effects are and, often, how the drug is metabolized and excreted. The number of subjects typically ranges from 20 to 80.
    (In other words, is it safe.)

    Phase 2 studies begin if Phase 1 studies don't reveal unacceptable toxicity. While the emphasis in Phase 1 is on safety, the emphasis in Phase 2 is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. For controlled trials, patients receiving the drug are compared with similar patients receiving a different treatment--usually an inactive substance (placebo), or a different drug. Safety continues to be evaluated, and short-term side effects are studied. Typically, the number of subjects in Phase 2 studies ranges from a few dozen to about 300.
    (Is it safe and effective.)

    Phase 3 studies begin if evidence of effectiveness is shown in Phase 2. These studies gather more information about safety and effectiveness, studying different populations and different dosages and using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.
    (Looking for non-obvious side-effects.)

    So, all tri
    als must first demonstrate they are safe before they try to demonstrate that they are effective.

    With respect to Neuralstem's trial:
    Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI, it says the Official Title of the study is:

    A Phase 1, Open-label, Multi-site, Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI.

    So, they are not just testing on chronic completes like guinea pigs. This is just Phase 1 of a three Phase trial, and if some of the test patients don't see some improvements, I doubt that there will be a Phase 2.

    Finally, how do you know that no animal study was done? In the previous thread, you said:

    Quote Originally Posted by Nowhere Man View Post
    The patients in this trial will be chronic & complete (Asia A)? I wonder if there are any chronic & complete animal studies using this treatment that would justify using these patients.

    Now you say:
    Who in their right mind would go from straight from the drawing boards to a human clinical trial, without any relevant animal studies? What company would waste tens of millions on a human trial without a successful animal trial? None.

    You must have found that they did not do animal trials, or you are saying that maybe they didn't, and you want to crucify them because you don't know for sure that they did?

    Nice attitude about research.
    Last edited by khmorgan; 05-18-2014 at 04:02 PM. Reason: Add explanation

  3. #3
    Quote Originally Posted by Nowhere Man View Post
    This is a general opinion of mine and not directed at any specific human trial:

    You gave the logical answer. You wouldn't let a lack of efficacy in chronic, complete human patients stop you from starting other trials in different injury types. My point is that current trials in chronic, complete SCI are not about efficacy in any meaningful way, they are truly about safety. Sure, if it shows some signs of efficacy it would be great for the given company, but that's not what the trial is about. They're about safety. Essentially, we chronic & complete human patients are just the guinea pigs to make sure there are no harmful effects.

    My main concern is that the chronic, complete patients are putting their bodies at high risk and are wasting their time (months) just to prove safety of these cells. If each patient knew that going in, then I have no issues with that (maybe they do know). It's a free society. However, most SCI are not knowledgeable in the science arena and drool at the term "stem cells". They are unaware of what they are and what they can do. They probably think that they might get substantial recovery, even though there is absolutely no animal study to justify that thought. Especially those within a few years of injury. Meanwhile, the chronic & complete population get all sorts of hope that a human trial is underway that is testing on someone with their injury type; unaware that it is basically just for safety and that there has been no efficacy shown in chronic, complete animals, nor is that the company's goal to achieve. I'm not saying this is the case in reality, but I'm saying that it worries me that it might be the case.

    If companies actually wanted to try to treat the human chronic & complete population, why not do an animal study in chronic & complete animals first? (Maybe they have but had no positive results.) We know it can be done. Who in their right mind would go from straight from the drawing boards to a human clinical trial, without any relevant animal studies? What company would waste tens of millions on a human trial without a successful animal trial? None.
    Seems like you are proposing a lot of hypothetical situations there...are you asking me in particular a specific question, or just making a statement in general?

  4. #4
    Quote Originally Posted by khmorgan View Post
    Maybe I'm missing something you are saying, but all human trials consist of 3 phases. See http://www.fda.gov/drugs/resourcesfo.../ucm143534.htm

    Phase 1 studies are usually conducted in healthy volunteers. The goal here is to determine what the drug's most frequent side effects are and, often, how the drug is metabolized and excreted. The number of subjects typically ranges from 20 to 80.
    (In other words, is it safe.)

    Phase 2 studies begin if Phase 1 studies don't reveal unacceptable toxicity. While the emphasis in Phase 1 is on safety, the emphasis in Phase 2 is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. For controlled trials, patients receiving the drug are compared with similar patients receiving a different treatment--usually an inactive substance (placebo), or a different drug. Safety continues to be evaluated, and short-term side effects are studied. Typically, the number of subjects in Phase 2 studies ranges from a few dozen to about 300.
    (Is it safe and effective.)

    Phase 3 studies begin if evidence of effectiveness is shown in Phase 2. These studies gather more information about safety and effectiveness, studying different populations and different dosages and using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.
    (Looking for non-obvious side-effects.)

    So, all tri
    als must first demonstrate they are safe before they try to demonstrate that they are effective.

    With respect to Neuralstem's trial:
    Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI, it says the Official Title of the study is:

    A Phase 1, Open-label, Multi-site, Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI.

    So, they are not just testing on chronic completes like guinea pigs. This is just Phase 1 of a three Phase trial, and if some of the test patients don't see some improvements, I doubt that there will be a Phase 2.

    Finally, how do you know that no animal study was done? In the previous thread, you said:


    Now you say:

    You must have found that they did not do animal trials, or you are saying that maybe they didn't, and you want to crucify them because you don't know for sure that they did?

    Nice attitude about research.
    Some clinical trials combine phase I & 2 into the same trial. So by definition they are looking at both safety and efficacy. However, I have doubt about them looking for efficacy in chronic, complete patients. Why would you expect a treatment to work in these patients when you have no successful animal data in similar injury? Furthermore, the only successful animal data are in incomplete and/or acute animals.

    Secondly, even in just a phase I safety trial only. Yes, it is clear that it is only testing for safety. But common sense would tell the patients that if there is a human safety clinical trial, that there must be animal studies that show success in similar injury. But for chronic, complete patients, there are not! I'm not sure what these companies tell the patients beforehand. So I do worry for these patients. That they are just being used.

    On Neuralstem's website, they posted animal study publications for their cells. I found none that were in a chronic and complete injury model. Can you find one?

    http://www.neuralstem.com/pharmaceuticals?id=120

  5. #5
    Super Moderator Sue Pendleton's Avatar
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    Just because you cannot find a study doesn't mean that it was not done and that the results were part of the company's IND. When companies realize that they may have a drug or treatment that could change their company, the medical specialties that would use it and all the patients that could benefit two things happen. Yes, some see dollar signs but some want their name on that discovery. The FDA will hold things back that are considered proprietary (like the spices in KFC chicken) until the trials are done and whether it makes the grade for release to doctors and patients. If a company published every paper before they had enough to claim exclusive rights to trial and possibly market it nothing would make it to market as other companies and countries would read what was done and try and beat the first company. Break throughs in science make journals. Break throughs in treatments make patents and profits.
    Courage doesn't always roar. Sometimes courage is the quiet voice at the end of the day saying, "I will try again tomorrow."

    Disclaimer: Answers, suggestions, and/or comments do not constitute medical advice expressed or implied and are based solely on my experiences as a SCI patient. Please consult your attending physician for medical advise and treatment. In the event of a medical emergency please call 911.

  6. #6
    Senior Member khmorgan's Avatar
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    Right Sue. The company I worked for published technical papers and patents (thousands every year). They also had trade secrets. The trade secrets were only shared internally on a "need to know" basis. They were eventually published, but only after they had been fully exploited.

    Basically, big companies publish papers/patents when they want to profit from an invention through royalties. If they plan to exploit an invention internally, they simply document when they invented/discovered something so they can prove that they did not steal the idea if someone else patents it first. As long as they don't steal an idea, they can continue to use it royalty free, even if someone else patents it.
    Last edited by khmorgan; 05-21-2014 at 07:46 PM.

  7. #7
    Quote Originally Posted by Sue Pendleton View Post
    Just because you cannot find a study doesn't mean that it was not done and that the results were part of the company's IND. When companies realize that they may have a drug or treatment that could change their company, the medical specialties that would use it and all the patients that could benefit two things happen. Yes, some see dollar signs but some want their name on that discovery. The FDA will hold things back that are considered proprietary (like the spices in KFC chicken) until the trials are done and whether it makes the grade for release to doctors and patients. If a company published every paper before they had enough to claim exclusive rights to trial and possibly market it nothing would make it to market as other companies and countries would read what was done and try and beat the first company. Break throughs in science make journals. Break throughs in treatments make patents and profits.
    Maybe they did a chronic, complete rat study and the rats recovered the ability to walk with a bbb score of 20; full return of bladder, bowel, and sexual function. Maybe they did primate studies and got the same fantastic results.

  8. #8
    Maybe we should just stick with experimenting on rats, it's not so bad being a quad.

  9. #9
    Senior Member Tbone57's Avatar
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    Agreed, I'm happy as a clam sitting in a wheelchair watching the world go by, peeing in my pants and digging shit out of my ass on a daily basis.

  10. #10
    Quote Originally Posted by Jim View Post
    Maybe we should just stick with experimenting on rats, it's not so bad being a quad.
    Maybe we should START experimenting on rats. Show me ONE (reputable) successful chronic, complete animal study.

    So you think animal efficacy studies are an unnecessary step in clinical process?

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