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Thread: Ten frequently asked questions concerning cure of spinal cord injury

  1. #561
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    Dr Wise since your first entry on the 07-21-2004 about trials and cures six years ago. I was just wondering if things have progressed from that point as you thought back then or have they progressed futher than you expected in developing a cure and do you think that it would be possiable by 2020 given the current rate of medical advancements, a cure will be in our life time?

    Just your thoughts on the issue as mankind has appeared to have moved at faster rate from the year 1900's especially the fiftys, or do you think things are slowly being hampered by patents.

  2. #562
    Quote Originally Posted by CAS View Post
    Dr Wise since your first entry on the 07-21-2004 about trials and cures six years ago. I was just wondering if things have progressed from that point as you thought back then or have they progressed futher than you expected in developing a cure and do you think that it would be possiable by 2020 given the current rate of medical advancements, a cure will be in our life time?

    Just your thoughts on the issue as mankind has appeared to have moved at faster rate from the year 1900's especially the fiftys, or do you think things are slowly being hampered by patents.
    Things have of course been slower than I would have wished. That is why I formed the ChinaSCINet to accelerate the process and to set an example. Clinical trials are beginning to happen all around the world and hopefully will increase the likelihood of discovering therapies that restore function. I don't think that patents have been an obstacle. The three main obstacles have been:
    1. Lack of funding
    2. Fear of failure
    3. Ignorant politics


    The first is obvious. Spinal cord injury clinical trials have been a low priority, even now, for over a decade. It bothers me a great deal that health care reform doesn't include clinical trials, that our government spent a trillion do,lars of stimulating the economy but deliberately excluded clinical trials.

    The second is clear whenever I attend a meeting for clinical trials. There are always people who get up to pontificate about how difficult clinical trials are. Many companies and investigators are consequently afraid to invest. A trial does not fail if it shows that a therapy does not work. It only fails when the trial doesn't show whether the treatment works or not.

    The third is best exemplified by the situation concerning stem cell research in the United States. The people who oppose the research have a totally wrong idea about the therapies they are against. Yet, they have shut down embryonic stem cell research for over decade.

    Wise.
    Last edited by Wise Young; 09-20-2010 at 01:04 AM. Reason: corrected errors (in red)

  3. #563
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    Quote Originally Posted by Wise Young View Post
    Things have of course been slower than I would have wished. At is why I formed the ChinaSCINet to accelerate the process and to set an example. Clinical trials are beginning to happen all around the world and hopefully will increase the likelihood of discovering therapies that restore function. I do 't think that patents have been an obstacle. The three main obstacles have been:
    1. . Lack of funding
    2. . Fear of failure
    3. . Ignorant politics

    The first is obvious. Spinal cord injury clinical trials have been a low priority, even now, for over a decade. It bothers me a great deal that health care reform doesn't include clinical trials, that our government spent a trillion do,lars of stimulating the economy but deliberately excluded clinical trials.

    The second is clear whenever I attend a meeting for clinical trials. There are always people who get up to pontificate about how difficult clinical trials are. Many companies and investigators are consequently afraid to invest. a trial does not fail if it shows that a therapy does not work. It only fails when the trial doesn't show whether the treatment works or not.

    The third is best exemplified by the situation concerning stem cell research in the United States. The people who oppose the research have a totally wrong die about heat they are against. Yet, they have shut down embryonic stem cell research for over decade.

    Wise.
    Thank-you for the info
    In regards to failure i think that mankind only fails to achive things that need to be done to improve the quality of the future or life is;

    one - that it appear impossiable or
    two - fail to move on because of a set back.

    Therefore I (and I assume the rest off us) hope that you and others like don't ever give up because we appreciate what you do very much even though it possibly do's not appear that way at time - cheers

  4. #564
    No question about it, Wise is leading the way into the future. I hope and pray that his trials are successful. If so, they will lead us out of the wilderness.

  5. #565
    Quote Originally Posted by CAS View Post
    Thank-you for the info
    In regards to failure i think that mankind only fails to achive things that need to be done to improve the quality of the future or life is;

    one - that it appear impossiable or
    two - fail to move on because of a set back.

    Therefore I (and I assume the rest off us) hope that you and others like don't ever give up because we appreciate what you do very much even though it possibly do's not appear that way at time - cheers
    CAS,

    Thanks. When we first started organizing ChinaSCINet, many people thought that there would be two insurmountable obstacles:
    1. We would not be able to raise funds to do clinical trials in China.
    2. We would not be able to get Chinese doctors to collaborate & participate.

    Everybody thought that it would be easy to get regulatory approval in China to do whatever clinical trials we wanted to do.

    It turned out that the two anticipated obstacles were not as difficult as people thought and the one "easy" task was the most difficult. It is true that China does not have many (only one) non-governmental charities to raise donations for clinical trials. However, we were able to raise the funds in Hong Kong for for establishing the clinical trial network and then substantially reduced the funds needed for the trials by helping participating institutions write and get grants from their provinces and Beijing for the trials.

    The second obstacle of getting Chinese clinicians to work together turned out to be not problematical at all. After an initial period of reluctance to work together, Chinese doctors are committing to the trials, working closely together and deciding to adopt standard outcome measures and to share information concerning their work. If there is one thing that we can be proud of, ChinaSCINet has standardized neurological examination and assessment of people with spinal cord injury in China. Many of the doctors are working with each other and the standards of spinal cord injury care has improved.

    An unexpected obstacle was the difficulty of getting governmental approval of therapies. We started the network at the time of tightening restrictions on clinical trials and imposition of international standards on clinical trial centers in China. We have to obtain approval not only from the sFDA and Ministry of Health but also from provincial and military departments of health. We have to get permission to import cells. Phase 1 trials of new therapies from overseas as not allowed. Unlike the U.S. FDA which must provide an answer to IND (clinical trial applications) within 3 months, the chinese bureaucracies do not have to answer you. They may keep you waiting for a year or longer. In China, anything new requires time and a lot of discussion. Bureaucrats are often unwilling to take chances on anything new or something where there has not been a precedent decision.

    Due to the experience of the Chinese surgeons in transplanting cells to human spinal cord, we had little difficulty reaching a consensus concerning the best way to inject the cells into the spinal cord. Likewise, there was virtually no opposition to the concept of doing a laminectomy and exposing the spinal cord to make the cell injections. Finally, because the surgeons have injected many different kinds of cells into the spinal cord, they actually thought that umbilical cord blood mononuclear cells are safer and better than fetal or even bone marrow cells.

    Once the infrastructure and procedures for cellular and drug therapies has been established, it should be relatively easy to substitute different kinds of cells and drugs to give to patients. We can then compare the results of different cells and regenerative therapies. There should be less lag time in getting the next therapy tested. The first and most difficult hurdle is getting the trials going. Therefore, it should be easier and less expensive, both in terms of labor and costs.

    Wise.

  6. #566
    Quote Originally Posted by Wise Young View Post
    The three main obstacles have been:
    1. Lack of funding
    2. Fear of failure
    3. Ignorant politics
    The first is obvious. Spinal cord injury clinical trials have been a low priority, even now, for over a decade. It bothers me a great deal that health care reform doesn't include clinical trials, that our government spent a trillion do,lars of stimulating the economy but deliberately excluded clinical trials.

    The second is clear whenever I attend a meeting for clinical trials. There are always people who get up to pontificate about how difficult clinical trials are. Many companies and investigators are consequently afraid to invest. A trial does not fail if it shows that a therapy does not work. It only fails when the trial doesn't show whether the treatment works or not.

    The third is best exemplified by the situation concerning stem cell research in the United States. The people who oppose the research have a totally wrong idea about the therapies they are against. Yet, they have shut down embryonic stem cell research for over decade.

    Wise.
    Dr. Young,

    I have a couple questions regarding this assessment:

    1. When you spoke at the 2009 World Stem Cell Summit here in Baltimore this past fall, you mentioned a change in FDA policy regarding clinical trial funding. Apologies if I do not get this right, but let me try to paraphrase your words as I understood them: The FDA removed some restrictions on patients paying for an investigational treatment when it is part of a clinical trial. As such, you were proposing a “scholarship program” in order to help fund the research without leaving patients out who could not afford it. In addition, since the most expensive part of the trial is rehabilitation, a patient’s insurance plan may cover these costs, even though it part of the treatment protocol. Is this accurate? If so, are you planning to apply it to SCINetUSA trials?

    2. I am curious how often funding for human clinical trials comes from public funds when evaluating a brand new drug. I know comparative effectiveness and trials with existing therapies take place, but has the federal government ever sponsored human evaluation of a novel therapy, such as umbilical cord blood for SCI? I know that the STAAR Act may potentially fund human trials with new antibiotics, but is this common? I agree that health spending is backwards for expensive chronic conditions like SCI. Medicare pays billions in claims for management of these chronic diseases while the NIH and other health agencies ignore funding opportunities, like clinical trials, to potentially end the disease acutely and prevent secondary conditions.

    Thanks for any insight.

  7. #567
    Quote Originally Posted by walderness View Post
    Dr. Young,

    I have a couple questions regarding this assessment:

    1. When you spoke at the 2009 World Stem Cell Summit here in Baltimore this past fall, you mentioned a change in FDA policy regarding clinical trial funding. Apologies if I do not get this right, but let me try to paraphrase your words as I understood them: The FDA removed some restrictions on patients paying for an investigational treatment when it is part of a clinical trial. As such, you were proposing a “scholarship program” in order to help fund the research without leaving patients out who could not afford it. In addition, since the most expensive part of the trial is rehabilitation, a patient’s insurance plan may cover these costs, even though it part of the treatment protocol. Is this accurate? If so, are you planning to apply it to SCINetUSA trials?

    2. I am curious how often funding for human clinical trials comes from public funds when evaluating a brand new drug. I know comparative effectiveness and trials with existing therapies take place, but has the federal government ever sponsored human evaluation of a novel therapy, such as umbilical cord blood for SCI? I know that the STAAR Act may potentially fund human trials with new antibiotics, but is this common? I agree that health spending is backwards for expensive chronic conditions like SCI. Medicare pays billions in claims for management of these chronic diseases while the NIH and other health agencies ignore funding opportunities, like clinical trials, to potentially end the disease acutely and prevent secondary conditions.

    Thanks for any insight.
    Dear Walderness,

    Thank you for your post and sorry for the delay in my response. Indeed, I pointed out that the FDA issued last August new regulations that may allow investigators to charge patients for some of the costs of clinical trials. As I understand the regulation, they must involve therapies that are already approved and accepted as beneficial for the patient (such as rehabilitation) and the protocol and payment must be approved by the FDA in the IND (the application for the clinical trial). As this is a relatively new regulation, I do not know how many trials have been approved by the FDA that allows patients to pay for the rehabilitation phase of the trial. I was obviously interested in this development because rehabilitation accounts for about half of the cost of our planned trial.

    The federal government has long sponsored clinical trials in various fields. For example, many cancer therapies are tested in clinical trials funded by the the National Cancer Institute (part of the NIH). Likewise, NIH sponsored the first clinical double-blind randomized trial of a treatment for spinal cord injury, the National Acute Spinal Cord Injury Study (NASCIS) which showed that methylprednisolone given within a few hours after injury improves recovery. However, in recent years, due to escalating costs of clinical trials, NIH has been funding fewer spinal cord injury clinical trials. Most clinical trials are sponsored by companies, who stand to profit if the trial shows that the treatment is safe and effective.

    You are correct in pointing out that the federal government's limited funding of clinical trials for expensive conditions such as spinal cord injury does not make sense. Also, certain therapies are unlikely to make a profit for any company. For example, when we studied methylprednisolone for spinal cord injury, the patent for the treatment had already lapsed and the drug was already generic, i.e. could be made by any company who chose to do so. So, the likelihood of major profit from the treatment is unlikely and most companies would not be willing to sponsor a clinical trial under such circumstances. Who is mostly likely to benefit from a therapy that restores function to people with spinal cord injury? The answer is clear, the people are most likely to benefit.

    I am not sure that the NIH or other federal agencies will be willing or able to come up with the billions of dollars needed to fund clinical trials, particularly "small-market" conditions with relatively few people, such as spinal cord injury. Spinal cord injury now belongs in that category of condition where there are just enough people so that the condition may not qualify as an orphan disease or condition (<200,000). But, it is also not common enough to convince a company to invest tens of millions of dollars into clinical trials. That is the reason why we have started a new program to try to fund clinical trials for spinal cord injury: http://justadollarplease.org.

    Wise.

  8. #568
    Quote Originally Posted by Wise Young View Post
    Dear Walderness,

    Thank you for your post and sorry for the delay in my response. Indeed, I pointed out that the FDA issued last August new regulations that may allow investigators to charge patients for some of the costs of clinical trials. As I understand the regulation, they must involve therapies that are already approved and accepted as beneficial for the patient (such as rehabilitation) and the protocol and payment must be approved by the FDA in the IND (the application for the clinical trial). As this is a relatively new regulation, I do not know how many trials have been approved by the FDA that allows patients to pay for the rehabilitation phase of the trial. I was obviously interested in this development because rehabilitation accounts for about half of the cost of our planned trial.

    The federal government has long sponsored clinical trials in various fields. For example, many cancer therapies are tested in clinical trials funded by the the National Cancer Institute (part of the NIH). Likewise, NIH sponsored the first clinical double-blind randomized trial of a treatment for spinal cord injury, the National Acute Spinal Cord Injury Study (NASCIS) which showed that methylprednisolone given within a few hours after injury improves recovery. However, in recent years, due to escalating costs of clinical trials, NIH has been funding fewer spinal cord injury clinical trials. Most clinical trials are sponsored by companies, who stand to profit if the trial shows that the treatment is safe and effective.

    You are correct in pointing out that the federal government's limited funding of clinical trials for expensive conditions such as spinal cord injury does not make sense. Also, certain therapies are unlikely to make a profit for any company. For example, when we studied methylprednisolone for spinal cord injury, the patent for the treatment had already lapsed and the drug was already generic, i.e. could be made by any company who chose to do so. So, the likelihood of major profit from the treatment is unlikely and most companies would not be willing to sponsor a clinical trial under such circumstances. Who is mostly likely to benefit from a therapy that restores function to people with spinal cord injury? The answer is clear, the people are most likely to benefit.

    I am not sure that the NIH or other federal agencies will be willing or able to come up with the billions of dollars needed to fund clinical trials, particularly "small-market" conditions with relatively few people, such as spinal cord injury. Spinal cord injury now belongs in that category of condition where there are just enough people so that the condition may not qualify as an orphan disease or condition (<200,000). But, it is also not common enough to convince a company to invest tens of millions of dollars into clinical trials. That is the reason why we have started a new program to try to fund clinical trials for spinal cord injury: http://justadollarplease.org.

    Wise.
    Thanks for the reply…can’t find your insight on this subject elsewhere given your longstanding career. I am rounding up topics to discuss for an event sponsored by a student health policy interest group I am in, and I’m presenting it to as many Hopkins med students as I can coerce to come. A big part of me wants to use it as a forum to release the more frustrating aspects of funding SCI researc, but I’ve resigned to pull it back a little. Anyway, I did support the trial a couple months ago with a dollar a day, $365, and I am a poor student who never budgets.

    I absolutely understand that a biotech would resist the risk investment in a therapy that has few applications. When we’re talking about public investment, however, a successful therapy would mean huge public savings: 1. Decreased health care costs for Medicare 2. Decreased public funds for SSDI and other entitlement programs 3. Increased productivity and tax revenue, etc. This means billions…all you need to do is compare estimated savings to estimated costs.

    I suppose it would have made the tea partiers furious if the government had included expenditures for HCTs. Just once, I would love to see them put down their signs declaring Obama a Nazi and pick up a laptop to put together a simple cost analysis. Savings takes investment!

  9. #569
    Quote Originally Posted by walderness View Post
    Thanks for the reply…can’t find your insight on this subject elsewhere given your longstanding career. I am rounding up topics to discuss for an event sponsored by a student health policy interest group I am in, and I’m presenting it to as many Hopkins med students as I can coerce to come. A big part of me wants to use it as a forum to release the more frustrating aspects of funding SCI researc, but I’ve resigned to pull it back a little. Anyway, I did support the trial a couple months ago with a dollar a day, $365, and I am a poor student who never budgets.

    I absolutely understand that a biotech would resist the risk investment in a therapy that has few applications. When we’re talking about public investment, however, a successful therapy would mean huge public savings: 1. Decreased health care costs for Medicare 2. Decreased public funds for SSDI and other entitlement programs 3. Increased productivity and tax revenue, etc. This means billions…all you need to do is compare estimated savings to estimated costs.

    I suppose it would have made the tea partiers furious if the government had included expenditures for HCTs. Just once, I would love to see them put down their signs declaring Obama a Nazi and pick up a laptop to put together a simple cost analysis. Savings takes investment!
    The issue of patient funding of clinical trials has come up recently in the TCA clinical trials of bone marrow mesenchymal stem cell therapy of spinal cord injury in Louisiana. They apparently have an approved IND (application to the FDA to do a clinical trial) but have offered patients the same therapy for $20,000 outside of the trial. I was taken aback by this information. This is not approved therapy and I don't know whether they have permission for a second trial where the patients pay for the therapy. The only way that I think that may be allowed is if they have obtained permission to apply a "compassionate use" therapy. This practice was actually started by the FDA in the 1990's for AIDS drugs and I remember that GM1 was approved to be used in this way for patients who could pay for it.

    I know that the FDA has been struggling to find ways to allow ethical funding of clinical trials. In the SCINetUSA, we have struggled with this as well. One of the questions that we have is whether or not we should be applying for an IND to the FDA that would allow patients to pay for the rehabilitation part of the trial. This cost is about $50,000 for 6 weeks of intensive walking training. After having though about it for a while, it became clear that the major drawback of charging patients is that the trial may become limited only to patients who can afford to join them. In other words, poor people may not be able to join clinical trials. To circumvent this, I think that it will be necessary for us to offer clinical trial "scholarships" to families that can't afford it. Of course, the best option is to pay for all the costs of the trial. After all, the subject is providing his/her time, effort, and body for the trial. The least that we can do is to provide the treatment for free.

    What the FDA is doing is the right thing, in my opinion. If a clinical trial charges the patients, the protocol including the payment must be approved by the IRB (Institutional Review Board) and the FDA. This will prevent most of the abuses, which occur in other countries when doctors are allowed to charge for non-approved therapies without review by regulatory agencies.

    Wise.

  10. #570
    Quote Originally Posted by Wise Young View Post
    The issue of patient funding of clinical trials has come up recently in the TCA clinical trials of bone marrow mesenchymal stem cell therapy of spinal cord injury in Louisiana. They apparently have an approved IND (application to the FDA to do a clinical trial) but have offered patients the same therapy for $20,000 outside of the trial. I was taken aback by this information. This is not approved therapy and I don't know whether they have permission for a second trial where the patients pay for the therapy. The only way that I think that may be allowed is if they have obtained permission to apply a "compassionate use" therapy. This practice was actually started by the FDA in the 1990's for AIDS drugs and I remember that GM1 was approved to be used in this way for patients who could pay for it.

    I know that the FDA has been struggling to find ways to allow ethical funding of clinical trials. In the SCINetUSA, we have struggled with this as well. One of the questions that we have is whether or not we should be applying for an IND to the FDA that would allow patients to pay for the rehabilitation part of the trial. This cost is about $50,000 for 6 weeks of intensive walking training. After having though about it for a while, it became clear that the major drawback of charging patients is that the trial may become limited only to patients who can afford to join them. In other words, poor people may not be able to join clinical trials. To circumvent this, I think that it will be necessary for us to offer clinical trial "scholarships" to families that can't afford it. Of course, the best option is to pay for all the costs of the trial. After all, the subject is providing his/her time, effort, and body for the trial. The least that we can do is to provide the treatment for free.

    What the FDA is doing is the right thing, in my opinion. If a clinical trial charges the patients, the protocol including the payment must be approved by the IRB (Institutional Review Board) and the FDA. This will prevent most of the abuses, which occur in other countries when doctors are allowed to charge for non-approved therapies without review by regulatory agencies.

    Wise.
    Hi Wise - rehabilitation costs of $50k for 6 weeks sounds like an awful lot. Even at a kingly sum of $200 an hour for 3 hours a day, 7 days a week for 6 weeks we are talking about $25k. Throw in accomodation and some care then maybe it gets closer to $35k.

    Also, 3 hours a day everyday is a LOT of training. From personal experience 2 hours a day 4 times a week is optimum for most people and there are many who dont have the physical stamina to do even that.

    Maybe it would be prudent to discuss tailoring a specific rehabilitation programme with the more cost-effective organisations such as Project Walk (and affiliates)? They have the experience and existing national coverage to allow you to outsource the physiotherapy at a far more competitive rate.

    PS I am not a sales representitive of PW. Im just throwing an idea out there.

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