Thread: ChinaSCINet Update

  1. #1331
    Quote Originally Posted by ay2012 View Post
    Dr. Young,
    This is a big hypothetical but: should the treatment prove effective but "incomplete" and there is reason to believe receiving multiple injections of umbilical cord blood would lead to further improvements, what's the protocol with respect to this? Once safety (and efficacy) is shown for a given amount, can that amount be used as a therapy repeatedly? Or would more, longer term, data be necessary for the FDA to approve? Feel free to answer in the most general way as, again, it's all hypothetical. Thanks!
    Dear ay2012,

    This is a decision that we are already facing and have been discussing. What do we do in the 2013 trial? We have many choices. Should we go on to test another cell, treat the patients with more repeated transplants with longer courses of lithium, add another therapy to the umbilical cord blood mononuclear cell and lithium therapy? I am not sure and we have held some meetings with many of the investigators of the network to discuss such issues. We need more data and the phase III trial will provide that data. However, as we know already, the recovery is likely to be slow and we won't know the final extent of recovery until a year or more from the start date of the trial in 2013. So, we will be heading into 2014 without as much data to make a decision. That is one of the reasons why we have been considering several options.

    The first option is to add another therapy to the umbilical cord blood mononuclear cell and lithium combination. Lisa McKerracher and her company Bioaxone had completed a Phase I/II showing that Cethrin (an analog of C3 that blocks rho, the intracellular messenger responsible for Nogo and CSPG inhibition of axonal growth) is not only safe but appears to convert more people from ASIA A to C than would be expected. What we would like to do is to do a trial of 20 subjects, all of whom would receive the umbilical cord blood mononuclear cell transplants and lithium but half would get Cethrin as well at the time of the transplant. If that trial suggests (by the way, a small trial with those numbers can only suggest and will not provide convincing statistical evidence) that triple combination is more effective than the double combination, we can consider the combination therapy. We are starting animal studies to look at the triple combination now and raising money for that trial.

    A second option is to transplant a cellular component of umbilical cord blood mononuclear cells. Many of the animal studies that showed beneficial effects used CD34+ cells, which are typically about 1% of the umbilical cord blood mononuclear cells. There are also CD133+ cells in cord blood that are said to be pluripotent stem cells and so we are interested in those as well. Finally, we have been studying on Muse cells, CD105+ and SSEA3+ cells that are pluripotent. Depending on the animal study results, we may choose one or another of these cells for the trials in 2014.

    A third option is to try some of the newer therapies therapies that are currently being assessed in animals, including a small molecule blocker of the CSPG receptor, the soluble Nogo decoy receptor, a pharmacological blockade of PTEN, or combinations of these. We are obviously watching the field very carefully and hoping for promising data. Many of these therapies are not quite ready to be taken to clinical trials and need both large animla safety studies and validated GMP production status. But, now is the time to start working on them. It sometimes takes years to get a therapy up to the standards required to give the treatment to humans.

    I think that we would want to wait at least 2 years before even considering giving a second course of umbilical cord blood and lithium therapy. In any case, we are still discussing all these options. It is possible that we will do a series of smaller phase II trials before embarking on another phase III. I don't think that we have enough data yet for a phase III in 2014. It would be really great if there were other chronic spinal cord injury trials going on.

    Wise.

  2. #1332
    Quote Originally Posted by beuty View Post
    Dear beuty,

    Thanks for posting this youtube interview of Patrick Rummerfield. He is a great man. By the way, the interview was done before he actually broke the world landspeed record for electric cars in the Utah salt flats. That record stood over a decade and was broken only last year.

    Wise.

  3. #1333
    Quote Originally Posted by KyleP2112 View Post
    Dr. Young,

    As I lay here in bed unable to move about 75% of my body (though strangely a few months ago I started to be able to move my right thumb) I'm wondering...what are the real prospects of research resulting in a reversal of paralysis? What might a "cure" look like? Will therapies for new and chronic injuries be completely different?

    I apologize if you've answered similar questions...the research is dense and a little hard to get an overview of. From my perspective it's easy to be impatient and want a "cure" to happen yesterday, get depressed about getting out of these chairs, dependence on others and to wonder if a cure will happen in our lifetime.

    Thanks for your time and effort,
    Kyle
    Dear Kyle,

    How interesting that you started moving your right thumb! As you may remember, Christopher Reeve recovered his ability to move his left index finger several years after his injury. I think that is from spontaneous regeneration.

    Believe me, I want to see therapies that restore function to people with chronic spinal cord injury to be available within our lifetime. I have now been working on spinal cord injury research since 1979, over 30 years. For 15 of those years (since 1997 when I moved to Rutgers), I have been focussing on chronic spinal cord injury therapies.

    While things have progressed slower than I had thought, still a great deal has happened in the chronic spinal cord injury field in the last 15 years. In 1997, we didn't know about adult stem cells and all hopes rested on embryonic stem cells. While we knew that the spinal cord could regenerate, we did not have any practical therapies that could be tried in humans. Now, therapies are going into clinical trial.

    We need more clinical trials testing therapies in parallel for chronic spinal cord injury. I will push as hard as I can from Rutgers to do so and continue to do trials. Every trial is giving us important information with which we can improve the trials and therapies. By next year, we will know a lot more than we do now. There are a lot of promising therapies waiting in the wings. So, I am optimistic.

    Wise.

  4. #1334
    Quote Originally Posted by Jim View Post
    The 6 month data will be made public this month.
    Thats what I thought was said, when in this month ? In Detroit it was said we would know in Novemeber? Either way thanks for the hard work and dedication Dr. Wise
    Last edited by allenstevens; 12-07-2012 at 10:12 AM.

  5. #1335
    Dr. Young,

    Thank you. You are an incredible man and you give hope to so many people. The first glimmer of hope that my husband got after his accident was from watching some of your interviews on youtube, and me reading him some of your articles. I feel so fortunate to have someone like you fighting for my husband, and all other SCI patients. For the last 3 months since my husband's injury I have consumed myself with SCI information and your research. I am incredibly thankful for you, and the time that you take to repsond to people like me...knowing that you have answered some of these questions hundreds, maybe thousands of times before. You are truly an angel! Thank you!

  6. #1336

    Incompletes?????

    Dr. Young,

    Is there any hope / chance that we incompletes might be able to take part in any of your upcoming trials? i'm a c4-c5 walking quad. Jan 2013 will be my 23rd anniversary since my injury. i walk with a cane but am starting to struggle more and more with overall strength, balance and stamina. The only positive in my life in recent years has been complete return of bowel, i no longer use suppositories and go normally like before injury. bladder is still a big problem and can only hold for bout 1 minute.

    in the past 5 years the pain levels have sky rocketed. everything hurts terribly and its never ending. the pain has seriously affected my overall health and state of mind, theres just never any rest or break from the pain!

    i don't take ANY type of medicines because to this day i haven't found anything that helps.

    thank you for all you do Dr Young.
    "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. #1337
    Quote Originally Posted by Wise Young View Post

    I think that we would want to wait at least 2 years before even considering giving a second course of umbilical cord blood and lithium therapy. In any case, we are still discussing all these options. It is possible that we will do a series of smaller phase II trials before embarking on another phase III. I don't think that we have enough data yet for a phase III in 2014. It would be really great if there were other chronic spinal cord injury trials going on.

    Wise.
    Could multiple trials be run simultaneously if the funding was available?

  8. #1338
    Senior Member
    Join Date
    Jan 2009
    Location
    Baldwinsville, N.Y.
    Posts
    645
    Dr. Young,
    You mentioned Cethrin should asia improvements. Has their been any positive results in bowel and blatter functions?

  9. #1339
    Senior Member
    Join Date
    Jan 2009
    Location
    Baldwinsville, N.Y.
    Posts
    645
    I meant Had shown Positive Asia improvements.

  10. #1340
    [QUOTE=Wise Young;1623188]
    Quote Originally Posted by paolocipolla View Post

    Paolo,

    I disagree. It is neither necessary nor useful to throw a tantrum and question somebody's intentions or motivations simply because you do not understand something. Such behavior is detrimental to communication. You had questioned me when I said that people are showing improved locomotor scores even though they are not showing changes in motor or sensory scores. That is a legitimate concern and I tried my best to explain it to you. Apparently my explanation was confusing to you. So, let me try again.

    Would you not agree with the following statement? If a person has no direct connections to the motoneurons controlling muscles of the legs but has some connections to the central pattern generator that connects to the motoneurons, that person can initiate walking and stop walking but cannot activate movement of individual leg muscles. If you don't agree with the statement, don't read any further and we should discuss why you don't agree with it. If you do agree with the statement, then please continue reading.
    ......

    Wise.
    Wise,

    back in June you said that "some people are walking", then a few weeks ago you said people are walking without having recovered any voluntary movements, so if I get it right, these people have been walking for a few months just by activating the CPG.

    I would like you to show me a person that can only activate the CPG walking, because I don't think that many people would agree to call that "walking", but I give you the benefit of the doubt untill I can see people like that.

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

Similar Threads

  1. ChinaSCINET Update
    By Schmeky in forum Cure
    Replies: 11
    Last Post: 06-16-2008, 06:25 PM
  2. ChinaSCINET on Schedule?
    By Schmeky in forum Cure
    Replies: 11
    Last Post: 01-27-2008, 05:53 AM
  3. Dr. Young and ChinaSciNet
    By Imight in forum Cure
    Replies: 17
    Last Post: 01-14-2008, 12:51 AM
  4. ChinaSCINET Schedule
    By Schmeky in forum Cure
    Replies: 4
    Last Post: 07-11-2007, 04:30 AM
  5. ChinaSCINET, On Schedule?
    By Schmeky in forum Cure
    Replies: 56
    Last Post: 01-30-2007, 03:46 PM

Posting Permissions

  • You may not post new threads
  • You may not post replies
  • You may not post attachments
  • You may not edit your posts
  •