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Thread: Which cell types hold the most promise for SCI therapy?

  1. #11
    Quote Originally Posted by Christopher Paddon View Post
    And yet, Wise, you are very optimistic as we saw in your interview in New Zealand. I can only deduce then that you believe simply throwing money at a problem is not the only way of solving it (if that makes sense). You are running your trials on an absolute shoestring because supposedly the animal results have provided enough evidence to make them worthwhile.
    Chris,

    I wouldn't turn down money if it were offered. While it is true that throwing money at the problem will not solve it, money is necessary and will markedly accelerate the process. A lot of people are asking why we are doing the trial in Hong Kong why it takes so long. It is taking so long because the trial is being sponsored by the medical authorities in Hong Kong and participation is limited to residents of Hong Kong.

    Wise.

  2. #12
    Quote Originally Posted by Wise Young View Post
    Pelican,

    I am actually preparing a lecture on this subject for tomorrow and thought that I might chip in my views.

    In my opinion, regulatory agencies are not the problem and they are not the reason why clinical trials are not being done in the United States and Europe. The central problem is the failure of our society to invest in development of therapies for spinal cord injury. Let me explain why.

    Thousands of clinical trials are going on for many other conditions. For example, almost every person who gets a cancer can join dozens of clinical trial comparing some experimental chemotherapy or other experimental therapy against the best standard therapy.

    How do they do it for these other conditions? Is it just the fact that cancer is a more common condition and therefore a larger market? Small market size is often blamed for why so few companies invest in spinal cord injury clinical trials. I hope that the following examples will persuade you that this argument is false.
    • Cancer is not a single condition. Many cancers are more rare than spinal cord injury and yet there are often clinical trials for rare cancers.
    • Multiple sclerosis is not a common condition. In fact, the number of people with multiple sclerosis is similar to those with chronic spinal cord injury and many of them are not so severely disabled. Yet, they have many more clinical trials going on for multiple sclerosis than for spinal cord injury, to the point that centers are competing with each other from multiple sclerosis patients.
    • Brain injury is a very common condition, probably affecting several million people in the United States alone but no clinical trials are going on for restoring function of people with brain injury, memory loss, and other devastating disabilities associated with brain injury.


    The above three examples (and there ar many similar ones) tells me the following. Market size is not the main reason why biotechs or other companies develop therapies for certain conditions. In my opinion, the main reasons for biotech investment are whether promising therapies are available for the condition, whether similar products that have already been developed for the condition, and subsidy that the government has put into the field. All these reduce the cost of developing therapy. Let me explain.

    Whenever I say "promising therapies", I refer to therapies that show promise in animal or even initial clinical trials but have not yet been proven or approved. If therapies have been proven or approved, they would no longer be just "promising". Promising therapies mean that somebody has invested in and harvested the fruits of preclinical research. The fact that people know that they are "promising" already indicates the research has been published and the results are widely accepted. The other side of "promising" is "controversial". In other words, the research is not widely accepted. So, this means that a major cost and hurdle of therapy development has already been crossed.

    Whether other products already exist in the field is important because the cost of clinical trials depend on whether the therapy is "first-in-class" as opposed to a "me-too" therapy. The latter is when another similar product has already been shown to be safe and effective. In that case, all the preclinical and clinical trials must do is to show biological equivalence and that is a much cheaper, faster, and less onerous route to travel than to get approval of a product that is "first-in-class". Many companies wait until another company spends a wad of getting the "first-in-class" treatment approved by the FDA and then announcement a whole slew of "me-too" products.

    Finally, subsidy by the government is a very important factor in therapy development. If the NIH is funding clinical trials, this takes a huge burden of cost from the companies. For example, the National Cancer Institute currently funds thousands of trials in their NCI Centers of Excellence. Yes, the U.S. government is spending billions funding a network of hundreds of cancer centers that are taking products from companies and testing them. If you were a company, would you not want to invest in those products that are being subsidized by NIH funded clinical trials?

    Failure of companies to invest in spinal cord injury is often attributed to the small number of people with spinal cord injury. This is not true and not the main reason for the failure of investment. While the number of acute spinal cord injuries is indeed small, i.e. about 10000 people per year, the number of people with chronic spinal cord injury is much greater. The Christopher Reeve Foundation, for example, has funded studies indicating that over a million people in the United States suffer from chronic spinal cord injury.

    The main reason why so few companies have invested into therapies for chronic spinal cord injury is because there is a dearth of promising therapies (therapies that the companies believe will work) for chronic spinal cord injury, there are few or no therapies that have already approved in the field, and virtually no government subsidy for spinal cord injury clinical trials in the United States. The NIH is not subsidizing spinal cord injury research or clinical trials in the United States.

    On top of these reasons, we are also coming out of the deepest and longest recession in the United States since the Depression of the 1930's. Private donations have fallen to less than half of what private donations were in 2000. Interest rates are nearly a third of what it was in 2000. State funding for spinal cord injury research has essentially been stopped. NIH funding has been frozen for a decade. The amount of spinal cord injury research funded by the NIH has fallen to below 1995 levels.

    For the past decade, most biotech companies in the United States have been unable to raise money for clinical trials. I know because I was on the Board of Directors of Acorda Therapeutics and can attest to firsthand knowledge concerning how difficult it has been to raise money from Wall Street. Stemcyte, the company that is supporting our clinical trials of umbilical cord blood, had to go to the Middle East and Asia to raise funds because there is simply so little biotechn funding available in the United States and Europe.

    So, the problem is not the regulatory agencies. The problem is money. Spinal cord injury research and clinical trials funding is at its lowest ebb in over 30 years. I have been working in this field since 1979 and have helped organized the funding of many spinal cord injury clinical trials. It has never been so bad and the end of the tunnel is not yet in sight. I am surprised that nobody seems to realize this and people are blaming regulatory agencies. Believe me, the FDA and the EMA are not the problems or obstacles. The lack of funding is problem.

    People are expecting miracles. Indeed, the fact that we do have any clinical trials at all is a miracle. In China, we are doing clinical trials by pulling up our own bootstraps, relying on the generosity of clinicians to donate their time and effort, and getting trials going based on the generosity of a few families who have donated several million dollars for the trials.

    I am writing this because I know that you understand money

    Wise.
    Im not blaming the lack of progress in the field on the FDA alone. Yes money is a massive issue and unfortunately with many Western governments in brutal deficit cutting mode this is not likely to change in the coming years.

    However, a fertile and flexible environment with for biotechs with a possibility of lower burn-rate during regulatory application phase would be a more attractive proposition for entry.

    Im guessing if you asked Geron if the $25m 'award' they have just received is enough to cover the burn-rate they have taken whilst dealing with the FDA over the last 5 years they will probably say, no way! In hindsight, would they have gone ahead with this trial in the US or even the UK? I bet they wouldnt!

    With second and third generation cures likely to be combinatory the burn-rate during regulatory application will surely be a major reason for deciding in which domicile to run Phase I/II trials.

    It would be interesting to know where the UCB+Lithium trial would be right now if you had applied for a Phase I/II in the US. I guess we will never know

    As for a dearth of promising therapies for chronic SCI - I totally agree.

  3. #13
    What about cooperation with the The International Society for Stem Cell Research (ISSCR)
    Cannot there be combined trials or research that spreads funding over a wider venue.

  4. #14
    Quote Originally Posted by Wise Young View Post
    Pelican,

    I am actually preparing a lecture on this subject for tomorrow and thought that I might chip in my views.

    In my opinion, regulatory agencies are not the problem and they are not the reason why clinical trials are not being done in the United States and Europe.

    ............

    Wise.
    Wise,

    what is the reason you couldn't start yet the trials in the SCINetUSA with UCB?
    I thought it was because the FDA was asking more preclinical studies with UCB on mouse?

    Paolo
    In God we trust; all others bring data. - Edwards Deming

  5. #15
    Quote Originally Posted by Wise Young View Post

    The main reason why so few companies have invested into therapies for chronic spinal cord injury is because there is a dearth of promising therapies (therapies that the companies believe will work) for chronic spinal cord injury, there are few or no therapies that have already approved in the field, and virtually no government subsidy for spinal cord injury clinical trials in the United States. The NIH is not subsidizing spinal cord injury research or clinical trials in the United States.

    Wise.
    So would you agree that SCI foundations like CDRF should direct part of their $$ on chronic SCI research?
    I year ago I got a bit mad in a scientific meeting because I keep seeing sci reserch on acute SCI, while there is still the endless discussion about the "scar" in chronic SCI that I believe it should be easy to clear.
    I think that if finally some $$ would be directed just to chronic SCI research we could make some significant progress soon.

    What about the preclinical studies? Do you think that this is also a velley of death of many therapies?
    I am thinking at chondroitinase, for example. As I understand the problem now is the preclinical development of it that is going to take several years and many millions if it will ever happen.

    Paolo
    In God we trust; all others bring data. - Edwards Deming

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