Thread: ChinaSCINet Update

  1. #1361

    Searching for Miracles

    "We get too little from medical innovation because we expect too much."

    The New York Times reports on a remarkable trial of a new gene-based therapy for leukemia, which reprograms the patient's own immune system to fight the rogue cells. The results have been nothing short of remarkable:

    Three adults with chronic leukemia treated at the University of Pennsylvania have also had complete remissions, with no signs of disease; two of them have been well for more than two years, said Dr. David Porter. Four adults improved but did not have full remissions, and one was treated too recently to evaluate. A child improved and then relapsed. In two adults, the treatment did not work at all. The Pennsylvania researchers were presenting their results on Sunday and Monday in Atlanta at a meeting of the American Society of Hematology.
    "Despite the mixed results," the story goes on, "cancer experts not involved with the research say it has tremendous promise, because even in this early phase of testing it has worked in seemingly hopeless cases."

    "Mixed results?" These are fantastic results. Twelve people were treated for end-stage, multi-drug resistant leukemia; one third are in full remission. More than one third have at least slowed the progress of their disease. These are people who had one foot, and the toes on the other foot, in the grave. Even if this is only a temporary respite, it's a major success.

    It strikes me that medical research is haunted by the memory of penicillin, and the other antibiotics that immediately followed. For a period of ten or fifteen years, "miracle cures" were the stuff of everyday life rather than television movies and late night infomercials: you took a pill, and something that had previously been fatal, like pneumonia or tuberculosis, simply went away. Often, you went from death's door to the picture of health in hours or days.

    As a consequence, people think that this is how medical discoveries are supposed to work: you find a cure for a fatal or crippling disease, everyone gets better instantly, and the world is a better place. I suspect that this is Big Pharma critic Marcia Angell's mental model. The reason she's decided that drug companies are about as useful as a third buttock is that they're no longer delivering the miracle pills on schedule.

    But this is not how innovation works most of the time. Innovation is not "Problem solved forever!" Instead it's mostly "We've reduced the incidence of this problem by 11.3%." But if you do that over and over again, eventually you can produce a pretty good fascimile of the miracle cure you thought you were supposed to get in the first place.

    Unfortunately, Penicillin Envy can make it difficult to achieve those lesser innovations. A while back, Michael Mandel pointed out that the FDA frequently makes the perfect the enemy of the "needs improvement"--and thereby kills promising technologies. A company that developed a computer system for diagnosing melanoma was turned down on the grounds that it didn't do as good a job as the best dermatologists. Of course, not everyone has access to the best dermatologists--and more importantly, there's no reason to think that the first-generation system will do as good a job as the eighth generation. But if you kill the first generation, you also kill the eighth.

    The FDA eventually reversed itself, but the mentality remains. We expect too many miracle cures, and as a result, we get fewer medical miracles.
    The Daily Beast
    Last edited by crabbyshark; 12-11-2012 at 03:41 AM.

  2. #1362
    Quote Originally Posted by crabbyshark View Post
    "We get too little from medical innovation because we expect too much."
    An interesting point of view. Thanks for posting.

    Clayton
    "Wheelie Wanna Walk!"

  3. #1363
    There are two reasons for excluding ASIA B and C patients from the trials. The first is that transplanting cells into the spinal cord may jeopardize surviving axons that are crossing the injury site. The second is that ASIA B and C subjects have better prognoses for recovery than ASIA A subjects. The second reason is not applicable to people with chronic spinal cord injury. I may be able to convince my colleagues to include patients with sacral sparing and no other motor or sensory function in the lower limbs. We shall see


    I was under the impression the surgery required was already proven safe in China and "better prognoses" is a good thing. Aren't those two factors enough to expand the inclusion criteria in at least a few people who understand the risk?

    Brad
    Last edited by Brad; 12-11-2012 at 03:58 PM. Reason: misspelled word
    jbs

  4. #1364
    "Cells Harvested From Human Urine Used To Make Stem Cells"

    Anyone recall Dr. Wise mentioning this in the June open house? via Wired

    Biologists in China have published a study detailing how they transformed common cells found in human urine into neural stem cells that can be used to create neurons and glial brain cells. The find holds huge potential for the rapid testing and development of new treatments for neurodegenerative disorders.
    Prediction: When the Kunming results are finally released to the public this month they will be a headline story on the evening news, front page of cnn.com, trending on Twitter, upvoted on reddit, and covered by sites like Huffington Post.

  5. #1365
    [QUOTE=Wise Young;1623654]
    Quote Originally Posted by paolocipolla View Post

    Paolo,

    Yes, you seem to have gotten the explanation right. I should clarify that I reported that some patients are showing improvements in locomotion scores without significant increases in their motor scores, which reflect voluntary motor strength of legs. To explain this, I said that "the patients appear to be able to activate their central pattern generators without increasing their voluntary motor control or strength." I don't think that I said that people are walking without any voluntary movement. Certainly, that was not my intent.

    It is all right if you don't believe my explanation of the phenomenon. If you are interested, there is ample evidence in the literature of people who are walking through activation of the central pattern generator. If you watch the youtube of Reggie Edgerton's lecture posted by Grammy earlier, he actually shows a person doing "air walking" with stimulation of the central pattern generator. However, you will have to wait until we publish the study to see all the data from our study and decide for yourself. I hope, of course, that our subjects will show improved motor and sensory scores on followup examinations. Six months may still be too early. Thanks.

    http://spinalcordresearchandadvocacy...otor-function/

    Wise.
    Dear Wise,
    One more question: if I understood correctly, there was a comparison between people who got the treatment only, and some other group who received both the treatment + locomotor training in Kunming.
    What about comparing people who had treatment only to people who received locomotor training only? has this been done ?

    If I remember well, you have always been quite positive about the intensive locomotor training given in Kunming. What I am trying to figure out through this question is whether the treatment has (so far) influenced the ambulation capabilities of patients in combination with locomotor training, or whether that progress was only or mainly due to intensive locomotor training itself.

    I understand it is too early for any conclusion and further functional progress might still happen should the white matter growth lead to actual new connections and regained motor control, but I am just trying to understand the current results better.

    thanks a lot in advance for your explanations. Corinne

  6. #1366
    Quote Originally Posted by crabbyshark View Post
    "Cells Harvested From Human Urine Used To Make Stem Cells"

    Anyone recall Dr. Wise mentioning this in the June open house? via Wired



    Prediction: When the Kunming results are finally released to the public this month they will be a headline story on the evening news, front page of cnn.com, trending on Twitter, upvoted on reddit, and covered by sites like Huffington Post.
    Lets hope so!!!
    "I'm manic as hell-
    But I'm goin' strong-
    Left my meds on the sink again-
    My head will be racing by lunchtime"

    <----Scott Weiland---->

  7. #1367
    Quote Originally Posted by havok View Post
    I didnt think much progress was being made with scaffolds? Also who was doing periferial nerve transplants?
    For the latest results using periferial nerve see the second presentation of Jerry Silver at W2W as soon as it comes on line.

    For biodegrdable scaffolds here are some random examples:

    http://www.nanotope.com/

    http://www.invivotherapeutics.com/

    http://www.eni-net.org/organization/...of-eva-sykova/

    http://pubs.acs.org/action/doSearch?...ation=40025957

    I wonder why Wise doesn't seem to consider any scaffold for future clinical trials in ChinaSCINet

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

  8. #1368
    Quote Originally Posted by paolocipolla View Post
    For the latest results using periferial nerve see the second presentation of Jerry Silver at W2W as soon as it comes on line.
    Paolo
    Actually another good presentation that will come up online shortly is by
    Ravi Bellamkonda, PhD, Walter H. Coulter Dept. of Biomedical Engineering, Georgia Institute of Technology. Dr. Bellamkonda’s lab focuses on peripheral nerve regeneration and interfacing, as well as overcoming the CSPG (Chondroitin Sulfate Proteoglycan) contribution to regenerative failure in the central nervous system. He also looks at interfacing technologies that might better integrate electronics into the nervous system.

    Ravi has worked on the delivery of a thermostabilized Ch'ase via scaffolding.

  9. #1369
    Quote Originally Posted by paolocipolla View Post
    For the latest results using periferial nerve see the second presentation of Jerry Silver at W2W as soon as it comes on line.

    For biodegrdable scaffolds here are some random examples:

    http://www.nanotope.com/

    http://www.invivotherapeutics.com/

    http://www.eni-net.org/organization/...of-eva-sykova/

    http://pubs.acs.org/action/doSearch?...ation=40025957

    I wonder why Wise doesn't seem to consider any scaffold for future clinical trials in ChinaSCINet

    Paolo
    Do you know for a fact he hasn't considered this as a possible future avenue?

  10. #1370
    Quote Originally Posted by cripwalk View Post
    Do you know for a fact he hasn't considered this as a possible future avenue?
    I said "doesn't seem", so what does that mean to you?

    In other words "no", I hope Wise can comment.

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

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