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To Wise Young re: compassionate use

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    To Wise Young re: compassionate use

    Hi I know you've been in contact with him in the past, and his company and work were tightly sidelined by the FDA. I started investigating and coMmunicating directly with Dr.Ahfors back in ''06. It seemed his work was being streamlined to a quick clinical approach, then company restructure took place amongst other derailing objects. To cut to the chase, on the New World Labs website it says Regeneration Matrix has been or used ''compassionate use'' Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available.
    Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug). Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use.

    I was wondering if you're aware of the outcome or details of any of these studys or if you could try to contact him and check since they wont answer anything.

    I am not your rolling wheels
    I am the highway
    I am not your carpet ride
    I am the sky
    I am not your blowing wind
    I am the lightning
    I am not your autumn moon
    I am the night, the night..

    #2
    Stormy, thanks. Yes, I have spoke to Jan-Eric Ahlfors several times and was fascinated by what he is trying to do. He was developing ways to differentiate cells consistently and providing scaffolds that would support their growth when transplanted. I have not heard of any clinical trials yet, at least not for spinal cord injury. Unfortunately, I have been spending most of my time in Asia and have not been to meetings that he has been attending.

    Unlike some people who are looking for villains in the slow progress in spinal cord injury trials, I don't consider the FDA to be a major obstacle. In fact, I regard them an ally and not an opponent. If you have a therapy that restores function in chronic spinal cord injury, the FDA will treat it with high priority and give the therapy fast-track and "compassionate use" consideration. In fact, they did that with a Purdue device to stimulate regeneration in the spinal cord, after only phase II trials. This is because there is nothing out there that is effective. However, the moment the first therapy has been approved by the FDA, subsequent therapies will not be as rapidly approved.

    In my opinion, the real obstacles in the United States are the high cost of clinical trials and requirements for safety before you can take something to clinical trials. For example, to transplant cells into the spinal cord and to rehabilitate the people after the surgery, it will cost us $150,000 per person, i.e. at least $50,000 for the surgery, $50,000 for 6-week outpatient rehabilitation, and then $50,000 for 3-6 months of locomotor training. If I want to study the effects of umbilical cord blood mononuclear cells in 40 subjects with spinal cord injury, the clinical costs will cost $6 million. That is not including the cost of hiring the CRO (clinical research organization) to monitor the trial, the company that is required to make clinical grade cells that can be transplanted, clinical trial insurance, the training, shipping, and the personnel needed to apply to the FDA. I am conservatively expecting the total costs of the trial to exceed $200,000 per patient or about $8-10 million. By the way, this is cheap.

    Comment


      #3
      "however, the moment the first therapy has been Approved by the FDA, subsequent trials will 'not' be as rapidly Approved ".... Was that a typo?

      I thought it will speed up
      "That's not smog! It's SMUG!! " - randy marsh, southpark

      "what???? , you don't 'all' wear a poop sac?.... DAMNIT BONNIE, YOU LIED TO ME ABOUT THE POOP SAC!!!! "


      2010 SCINet Clinical Trial Support Squad Member
      Please join me and donate a dollar a day at http://justadollarplease.org and copy and paste this message to the bottom of your signature

      Comment


        #4
        Yes it is quite apparent throughout the history of CareCure, mosts opinion of the FDA is a negative one. Some countries more/less lenient. His site even states, "with realistic FDA hurdles" in regards to the obvious. Given why he upped and went back to Canada if they're any more of the emphasized. I realize you have to comply with every standard they establish into the framework and you being an accredited M.D. along side their policys aren't going to bash them.
        As far as "If you have a therapy that restores function in chronic spinal cord injury, the FDA will treat it with high priority and give the therapy fast-track and "compassionate use" consideration" I don't want to go on a rant but theres been an array of therapies shown to do that, come on man!
        I respect and thank you for your diligent work. Thanks for your reponse.
        I am not your rolling wheels
        I am the highway
        I am not your carpet ride
        I am the sky
        I am not your blowing wind
        I am the lightning
        I am not your autumn moon
        I am the night, the night..

        Comment


          #5
          Actually Wise is right on the money on this. Once any regenerative therapy is found that restores ANY function and is reasonably safe than the next therapy is competing against the first one instead of against a baseline injury especially in chronic cases. In acutes the most a new therapy need beat is methylprednisolone and that still has never been proven to not help preserve the function that the trials showed and the FDA approved. So if new therapy one regenerates 3 neurological levels or 20% of the cord then the next up has to not only equal or better the safety of therapy one but cause substantially better performance like 6 neuro levels or 40% of the cord or to do the same in much better time.

          The trial creations may speed up as hospitals and rehabs get better at the surgeries, tasking of rehab and data collection and correlating but the FDA will look for a better outcome. Right now all we have is methylprednisolone at the acute stage of the first 8 and preferably 3 hour mark. There is no approved treatment for chronic injuries right now.

          I think funding may speed up as countries realize and charitable donors see that if partial cures are doable than full cures should be also. People like to back a winner.
          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.

          Comment


            #6
            what happened with that field oscilating implant unit that showed great success/?
            I am not your rolling wheels
            I am the highway
            I am not your carpet ride
            I am the sky
            I am not your blowing wind
            I am the lightning
            I am not your autumn moon
            I am the night, the night..

            Comment


              #7
              It is available for prescription under the compassionate use program, after just a phase II study. Wise.

              Comment


                #8
                How do you qualify for compassionate use of a drug or treatment? I've never heard of this in Canada.

                Comment


                  #9
                  Compassionate use is a program through the FDA (USA). I don't know if Canada has anything comparable:

                  http://www.fda.gov/ForConsumers/ByAu.../ucm176098.htm

                  (KLD)
                  The SCI-Nurses are advanced practice nurses specializing in SCI/D care. They are available to answer questions, provide education, and make suggestions which you should always discuss with your physician/primary health care provider before implementing. Medical diagnosis is not provided, nor do the SCI-Nurses provide nursing or medical care through their responses on the CareCure forums.

                  Comment


                    #10
                    James, if Canada has something like our compassionate use program you might try searching for Fidia Inc and their drug Sygen or GM-1. It remained available under compassionate use after going through phase 2 for SCI until the US part of the company ran out of money. It is an Italian company I think and Sygen had a good safety record for other uses in Europe. It should lead you to the Canadian agency in charge of such things.
                    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.

                    Comment


                      #11
                      Sygen was also associated with higher rates of Guillian-Barre syndrome in those who were on the clinical trials in the USA, and as I remember, while those on this drug did get faster return, the amount of return over the long run was no more than with the control group.

                      (KLD)
                      The SCI-Nurses are advanced practice nurses specializing in SCI/D care. They are available to answer questions, provide education, and make suggestions which you should always discuss with your physician/primary health care provider before implementing. Medical diagnosis is not provided, nor do the SCI-Nurses provide nursing or medical care through their responses on the CareCure forums.

                      Comment


                        #12
                        The "association" of GM1 treatment and Guillain-Barré syndrome *GBS) is spurious. For many years, Sygen or GM1 was used to treat patients with GBS. There is no clinical trial evidence that I am aware of that shows that Sygen causes GBS. On the other hand, the multicenter randomized placebo-controlled double-blind clinical trial showed no significant effect of Sygen on neurological recovery, except transiently at 8 weeks. Yet the authors concluded, "Although not proven in the primary efficacy analysis of this trial, Sygen appears to be beneficial in patients with severe spinal cord injury." This statement is paradoxical.

                        Geisler FH, Coleman WP, Grieco G, Poonian D and Sygen Study G (2001). The Sygen multicenter acute spinal cord injury study. Spine (Phila Pa 1976) 26: S87-98. Medical Group, S.C., Chicago Institute of Neurosurgery and Neuroresearch, 2515 North Clark Street, Suite 800, Chicago, Illinois 60614, USA. fgeisler@concentric.net. STUDY DESIGN: Randomized, double-blind, sequential, multicenter clinical trial of two doses of Sygen versus placebo. OBJECTIVES: To determine efficacy and safety of Sygen in acute spinal cord injury. SUMMARY OF BACKGROUND DATA: An earlier, single-center trial in 28 patients showed an improvement (50.0% vs. 7.1%, P = 0.034) in marked recovery with Sygen. METHODS: Standard clinical trial techniques. RESULTS: The prospectively planned analysis at the prespecified endpoint time for all patients was negative. There was a significant effect in all patients in the primary outcome variable (the percentage of marked recovery) at week 8, the end of the dosing period. There was a significant effect in all patients in the time at which marked recovery is first achieved. Restricted to severity Group B, which has small sample size, the primary efficacy analysis showed a trend but did not reach significance. There is a large, consistent and, at some time points, significant effect in the primary outcome variable in the nonoperated patients through week 26. The American Spinal Injury Association motor, light touch, and pinprick scores showed a consistent trend in favor of Sygen, as also did bowel function, bladder function, sacral sensation, and anal contraction. The less severely injured patients appeared to have a greater beneficial drug effect. Evidence against an effect of Sygen was minimal and scattered. CONCLUSIONS: Although not proven in the primary efficacy analysis of this trial, Sygen appears to be beneficial in patients with severe spinal cord injury.

                        Comment


                          #13
                          Originally posted by Wise Young
                          It is available for prescription under the compassionate use program, after just a phase II study. Wise.
                          oh wow. so is there any provided documented journal therapy outcomes of restoration or you know of firsthand with this device. much appreciated. and when is phase 2. so it sounds like its already being an approved therapy yes...what would guidelines be for an approved prescription//a SCI of any type..[srry typing one handed/no question marks]

                          Since NWL site states Regeneration matrix has been used in ''compassionate use'' or study,, i'm curious to know was there outcome data or did he tell you directly of the results of this.. TY
                          I am not your rolling wheels
                          I am the highway
                          I am not your carpet ride
                          I am the sky
                          I am not your blowing wind
                          I am the lightning
                          I am not your autumn moon
                          I am the night, the night..

                          Comment


                            #14
                            Originally posted by Stormycoon

                            oh wow. so is there any provided documented journal therapy outcomes of restoration or you know of firsthand with this device. much appreciated. and when is phase 2. so it sounds like its already being an approved therapy yes...what would guidelines be for an approved prescription//a SCI of any type..[srry typing one handed/no question marks]

                            Since NWL site states Regeneration matrix has been used in ''compassionate use'' or study,, i'm curious to know was there outcome data or did he tell you directly of the results of this.. TY
                            To my knowledge, this device passes alternating current into the spinal cord and no convincing efficacy data is available but surgeons are able to implant it for subacute spinal cord injury. I don't know of any "regeneration matrix" that has been used under "compassionate use" guidelines. Wise.

                            Comment


                              #15
                              Interesting. I was in the Sygen study when I was injured in 1996. They followed me for a few years. Initially I was first entered into it at the University of Albuquerque, New Mexico. They did follow me when I was transferred to Ohio State University, and later on. Does this mean that they a study was done in the 90s as well as 2001?

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