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FDA OKs First Human Trial of Neural Stem Cell Therapy 149

Posted by kdawson from the spine-enhancing dept.
An anonymous reader sends word that the FDA has approved a phase 1 trial for Neuralstem, a company with a patented stem cell procedure targeting ALS (Lou Gehrig's disease) and other spinal conditions. The company's CEO said in a press release, "While this trial aims to primarily establish safety and feasibility data in treating ALS patients, we also hope to be able to measure a slowing down of the ALS degenerative process." Results are expected in 2 years. The trial will involve 12 ALS patients who will receive stem cell injections in the lumbar area of the spinal cord. An information site for the disabled community adds hopefully: "If it makes it through all stages of testing, we will see if doctors are willing to [use] it on subjects that have injuries coming from physical injuries like diving accidents."
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FDA OKs First Human Trial of Neural Stem Cell Therapy More

FDA OKs First Human Trial of Neural Stem Cell Therapy

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  • Dr. Stephen Hawking (Score:3, Insightful)

    by Anonymous Coward on Tuesday September 22, 2009 @06:23PM (#29509561)

    Any chance that this could be passed through quick enough to prolong a certain genius' life?

    • Re: (Score:2, Interesting)

      by reverseengineer ( 580922 )
      Probably not, to be honest. If they're just starting Phase I right now, figure on at best 6-8 years or more before a possible approval by regulatory agencies, which naturally assumes that this treatment would be demonstrated as both safe and efficacious. Phase I doesn't take much time, but Phases II and III can easily take years- particularly for a disorder like ALS, where patients would need to be monitored for months to determine the treatment's effects. In Stephen Hawking's particular and remarkable c

      • Keep in mind that Hawking lives in England, not the United States. The FDA has no jurisdiction over him. Now it may be in practice that the British health service does follow FDA recommendations in large part, so it's not likely that the treatment would be available any too soon there. But it is certainly possible for him to travel to Europe or even to Asia in order to get treatment if he wants it. There are clinics in Germany, Mexico and China at least that are doing experimental stem cell treatments for A

    • Well Dr. Hawking it will take two years just on the test subjects to see if there is any progress. As a man of science you should know that these sort of things cannot be rushed and the scientific method has to be followed and the work peer reviewed.

      You've made it this far, chances are you can make it until they find a cure for ALS. I am certain after the trials are done and the procedure is ready for the public, you'll be on the top of the list because of your scientific importance.

      Some in your situation m

    • Re: (Score:2)

      by bobbuck ( 675253 )
      If it were that promising he could leave the country to have the treatment before the approval.

    • Re: (Score:3, Insightful)

      by Bruce Perens ( 3872 ) *
      He's 67. It might not be ALS that kills him.

    • Re: (Score:2, Interesting)

      by tunapez ( 1161697 )

      If it proves to be effective early in the trials, he can afford to meet his doc in "some hospitals in the far east"(better yet in Cabo) for the treatments. It happens every day, at great expense to the sick and great risk to the providers. The FDA may not approve, but the people who can't afford to wait for the bureaucratic trials to inch along do.

  • Big News? (Score:4, Insightful)

    by lobiusmoop ( 305328 ) on Tuesday September 22, 2009 @06:25PM (#29509577) Homepage

    It makes me sad that this is news in 2009. This should really have been commonplace research by now.

    • Re: (Score:2)

      by Trepidity ( 597 )

      How so? The earliest versions of the technique only date back to about 2002, and I don't see any evidence that research has been slower than one might expect.

  • I read TFA and I still don't understand if these are embryonic stem cells or adult stem cells... anyone?

    • Re: (Score:1, Informative)

      by Anonymous Coward

      As far as I can tell from the radio interview at the top of their news page [neuralstem.com], they are adult stem cells from the brain.

      • Re:What kind of stem cells? (Score:4, Informative)

        by interkin3tic ( 1469267 ) on Tuesday September 22, 2009 @07:26PM (#29510047)

        That's what I'm guessing too. TFA is ridiculously underinformative. Neuralstem doesn't seem to be talking specifics for some reason.

        A video on their website [neuralstem.com] was -slightly- more informative. They make lines of neural stem cells and inject them into the damaged part. That to me was somewhat questionable. Injecting undifferentiated, replicating cells into your central nervous system, even if they're neural stem cells sounds dangerous. You want the specific type of neuron there, and enough glial cells. Without directing their differentiation, I would expect you'd end up with a random mix of cells, or possibly a glioma [wikipedia.org].

        It mentioned that these were patented methods. I don't know much about patents, but I did find this patent [patentstorm.us] issued to neuralstem biopharmecuticals ltd (same company?).

        The abstract to that patent:

        A systematic and efficient method for establishing stable neural stem cell lines and neuronal progenitor lines is described. The resulting cell lines provide robust, simple, and reproducible cultures of human and other mammalian neurons in commercially useful mass quantities while maintaining normal karyotypes and normal neuronal phenotypes.

        What it actually seems to cover is nothing revoultionary. They isolate a neuronal stem cell, culture it in a wide variety of commonly used growth factors for 30 divisions, transfect the C myc gene, and then culture it in the same growth factors and/or whole serum. That's to make a line of cells. C-myc by the way was one of the transcription factors used to make induced pluripotent stem cells, and is associated with many cancers, which is worrisome. Nothing in that really suprises me. I'd be interested to hear from slashdot's armchair lawyers (or real lawyers) as to whether or not you can simply patent a combination of common techniques to make a line of stem cells.

        What is more interesting to me is another patent that Neuralstem has [patentstorm.us], Use of fuse nicotinamides to promote neurogenesis.
        The abstract for that one:

        The present invention provides a group of compounds found to increase the number of neurons derived from stem cells for use as a therapeutic agent in neurological conditions or diseases. In one embodiment of the present invention, the compounds are used to detect the mechanism by which the number of neurons is increased.

        I'm less of an expert on this, it's a lot of biochemistry I'm not familiar with, but from the summary:

        the present invention is related to classes of compound structures that are shown to be particularly effective in promoting neurogenesis includingcompounds of the type, fused imidazoles, aminopyrimidines, nicotinamides, aminomethyl phenoxypiperidines and aryloxypiperidines

        It seems they have a patent on compounds which have been shown to nudge stem cells towards making neurons. This might be their answer to the first problem I mentioned: not wanting to inject undifferentiated cells into your spine or brain.

        I'm guessing their technique involves 1. Surgery to get tissue samples which would be enriched in neural stem cells (I've heard the cells next to the ventricles in your brain are good spots for that) 2. They take those cells and put them in their culture media that causes the stem cells to divide 3. They transfect c-myc to increase the yeild 4. They harvest the undifferentiated cells and incubate them with their differentiation compounds before or as they 5. Inject the mix into your damaged spinal cord.

        If they moved on to humans, I'm guessing they've already demonstrated this works to a degree and doesn't cause a lot of cancers in mice or other animal models. The results on that are probably published, but I've wasted enough time here.

        • That's what I'm guessing too. TFA is ridiculously underinformative. Neuralstem doesn't seem to be talking specifics for some reason.

          I'm guessing their technique involves 1. Surgery to get tissue samples which would be enriched in neural stem cells (I've heard the cells next to the ventricles in your brain are good spots for that)

          So, if I understand you correctly, you're guessing that they're adult stem cells, not embryonic stem cells? That might answer your implied question of why Neuralstem isn't talking specifics -- stem cell research is only cool when it's embryonic stem cell research... ;)

          • Well not really guessing, the video says something like "takes your stem cells."

            Anyway, the current buzzword is induced pluripotent stem cell... and terrorism. Nano. Biofuel.

    • Re: (Score:3, Funny)

      by geekoid ( 135745 )

      Why does it matter?

      • Re: (Score:2, Interesting)

        by Anonymous Coward

        Why does it matter?

        One reason it matters is that we are forever being told how embryonic stem cell research is going to find the cure for every disease under the sun, and anyone who thinks the money should be diverted to research into stem cells from alternative sources is a religious nut who should shut up, yet of nearly 100 current treatments using stem cells, there is not a single one that uses embryonic stem cells. If this were better known, then all the resources being wasted on this dead-end research could perhaps be u

        • Re: (Score:3, Insightful)

          by BlueParrot ( 965239 )

          One reason it matters is that we are forever being told how embryonic stem cell research is going to find the cure for every disease under the sun

          Bullshit.

          There are a number of people who repeat that straw-man for political and religious motives, but what promoters of embryonic stem cell research usually argue is something more along the lines of:

          "Embryonic stem cells are worthy of research not only because understanding how they differentiate can help us understand how to better use adult stem cells, but a

        • One reason it matters is that we are forever being told how embryonic stem cell research is going to find the cure for every disease under the sun,

          The new line of thinking is that Induced pluripotent stem cells (IPSC) [google.com] will do all that ESC was promised to do. IPSC comes from your own cells which have been modified to be like ESC.

          Stem cells were never said to be a cure for all diseases. Cancer and infectious diseases, for example, were never potential diseases to be treated with stem cells. Regenerative medicine only.

          ...and anyone who thinks the money should be diverted to research into stem cells from alternative sources is a religious nut who should shut up

          Frankly, some of you guys are seriously misinformed. It's easy to write off people who honestly have ethical concerns, when others ar

          • Re: (Score:3, Interesting)

            by HanClinto ( 621615 )

            And none of you guys seem to realize that ESC research is done on embryos that were headed for the incincerator anyway.

            If ESC treatments become viable, IVF leftovers do not provide a sufficient supply for more than a tiny fraction of the people who would request treatment

            Besides, many people will still have ethical problems with forcibly "harvesting" parts / cells from people, even if they are headed for the incinerator. Didn't we just have this same debate a few years ago with harvesting condemned criminals for organs? "And none of you guys seem to realize that these organs are being taken from people who were headed for

            • If ESC treatments become viable, IVF leftovers do not provide a sufficient supply for more than a tiny fraction of the people who would request treatment

              Citation needed. These are self-renewing cells. It's not like you need to sacrifice one embryo for each patient.

              Besides, many people will still have ethical problems with forcibly "harvesting" parts / cells from people, even if they are headed for the incinerator. Didn't we just have this same debate a few years ago with harvesting condemned criminals for organs? "And none of you guys seem to realize that these organs are being taken from people who were headed for the electric chair anyway."

              You slipped "people" in there. You realize of course that's a not exactly a clear cut issue. It's a major difference between the execution and ESC.

              In a word, if you want it, spend your own money on it, and don't just get upset and call people ignorant when they don't share your priorities. Other people have legitimate needs for this cash, and there's only so much of it to go around.

              I don't think enough people would be willing to spend their tax money on defense to actually support an army. I don't want any of my tax money going overseas to Iraq or Israel, yet that's not an option. The idea that you should get to decide where

              • If ESC treatments become viable, IVF leftovers do not provide a sufficient supply for more than a tiny fraction of the people who would request treatment

                Citation needed. These are self-renewing cells. It's not like you need to sacrifice one embryo for each patient.

                True -- in some previous treatments, each patient may be implanted with brain tissue from as many as seven aborted donors [nytimes.com]. Granted, that was before isolated stem cells were in use, but for ESCs to move from the laboratory into the doctor's office, unless we plan on keeping all of our patients on immunosuppressants, I was under the impression that SCNT or some other form of "therapeutic cloning" is necessary to obtain ESCs that are usable cells that won't cause rejection. Ultimately you're right -- it's ha

                • These are self-renewing cells. It's not like you need to sacrifice one embryo for each patient.

                  True -- in some previous treatments, each patient may be implanted with brain tissue from as many as seven aborted donors [nytimes.com]. Granted, that was before isolated stem cells were in use,

                  The article there was talking about fetal brain tissue, not embryonic stem cells. Fetal brain tissue cannot be expanded in vitro, ESC do. That 1992 study used seven fetuses presumably for that reason: there is so little tissue per fetus and you can't just grow it to as much as you need.

                  but for ESCs to move from the laboratory into the doctor's office, unless we plan on keeping all of our patients on immunosuppressants, I was under the impression that SCNT or some other form of "therapeutic cloning" is necessary to obtain ESCs that are usable cells that won't cause rejection.

                  Right, which is one reason why IPSC is more promising. And that reminds me we've gotten off on a tangent. When you implied that IVF would not supply enough ESC for -therapy- I should have pointed out that ESC probably are

                  • Re: (Score:3, Informative)

                    by HanClinto ( 621615 )

                    Right, which is one reason why IPSC is more promising.

                    FWIW, I fully support IPSC. There already exist over a hundred treatments using them, and I see every reason to concentrate effort on expanding this research line.

                    When you implied that IVF would not supply enough ESC for -therapy- I should have pointed out that ESC probably aren't going to be used for therapy, they're used for research.

                    But you said, "These are self-renewing cells. It's not like you need to sacrifice one embryo for each patient." -- by saying "patient", we were continuing on the line of "treatment", not "research". If ESCs are to be used on patients, and we don't intend to keep them on immunosuppressants, then SCNT requires that we don't just create and sacrifi

    • psst let me let you in on a little secret if it's an article about stem cells actually being used in some treatment they are adult stem cells

  • Existing Conditions? (Score:4, Insightful)

    by BJ_Covert_Action ( 1499847 ) on Tuesday September 22, 2009 @06:47PM (#29509741) Homepage Journal
    The article states that the clinical trials are being conducted on patients with various levels of the disease. It also states that they are hoping to see the degenerative rate of the disease slow due to the treatments. It does not, however, talk about whether or not this stem cell treatment, or a similar one, could be used to treat patients with a developed case of ALS. For instance, to the /.er that talked about saving Hawking's life, Hawking has had the disease long enough that many of his motor neurons have probably already died out. Can this treatment be used to restore or replace said neurons? For those ALS patients that are already severely disabled, treatment needs to go beyond the stage of slowing the disease down. I would love to see ALS patients walking and talking again that couldn't previously.

    Neuralstem's own website [neuralstem.com] also seems rather scant in details on therapy for highly developed levels of ALS. Does anyone know of any research being conducted to treat the latter stages of ALS or how relevant this treatment is for those stages?

  • Stem cells = Cancer (Score:4, Informative)

    by sonnejw0 ( 1114901 ) on Tuesday September 22, 2009 @06:50PM (#29509757)
    Stem cells have the potential to reproduce exponentially. Give these stem cells to a patient that has a mutation in growth factor production or secretion, like many cancer or precancerous patients, and you have an unmitigated tumor. I do research with growth factors and development. This, in my opinion, is not a good idea.
    But those are the problems this research will address. I'll be eager to see the results in two years.

    • What they need is stem cells that grown in presence of a bio-compound not found naturally in the body. Give stem cells and drug them until stem cells are at the "just right" stage, stop taking the drug.

      • That's actually a great idea. An exogenous stem cell could be genetically engineered to intrinsically express a pro-apoptotic protein in an undifferentiated state while concurrently expressing a receptor that activates an anti-apoptotic protein. Inject the stem cells along with the signal that activates the receptor and the stem cells can proliferate until they differentiate. If they migrate outside the area that the signal is, the cells automatically die. If they do not differentiate, the cells automat

    • Do tumors REALLY reproduce exponentially, or do the "message" other sells to become part of the tumor? Isn't the heart of a cancer tumor dead cells?

      I've got my own theory that Cancer and Viruses in most cases are broken messaging systems in the body. A genetic message is sent and something like a Telomere gets snipped and eventually, the message is not reproduced and copied. Cancer and Viruses cannot do anything on their own -- they use the bodies on mechanisms against it.

      So stem cells are not going to beco

  • we will see if doctors are willing to [use] it on subjects that have injuries coming from physical injuries like diving accidents.

    First you have to convince crazy religious idiots, then you have to convince the crazy government idiots, and yet you still have to convince the crazy doctor idiots!? Is there no end to this insanity?!

    • Gee, here's a novel idea -- maybe this isn't the sort of thing we want to try to use the government for?
  • Neural Stem Cell Therapy - It Tickles!!! (tee hee .......eyes go glassy .... drool begins)

  • Sample Size? (Score:3, Interesting)

    by JSBiff ( 87824 ) on Tuesday September 22, 2009 @09:08PM (#29510725) Journal

    Can someone who understands statistics and FDA trial phases explain something to me. . . Is a sample size of 12 really big enough to be a reasonable 'safety' trial? Or do they start with a small trial, just to find out if there's any problems so severe that they would affect almost *anyone*, then in future phases, increase the sample sizes to more and more test subjects, looking for those problems that only affect 1/1000 or 1/100000 patients?

    • Can someone who understands statistics and FDA trial phases explain something to me. . . Is a sample size of 12 really big enough to be a reasonable 'safety' trial? Or do they start with a small trial, just to find out if there's any problems so severe that they would affect almost *anyone*, then in future phases, increase the sample sizes to more and more test subjects, looking for those problems that only affect 1/1000 or 1/100000 patients?

      A phase 1 is testing for safety to show it should be allowed to progress to phase 2 trials, not safety nor efficacy with respect to the population. More than you every wanted to know about clinical trials, but you did ask: http://en.wikipedia.org/wiki/Clinical_trial [wikipedia.org]

  • Misleading Title (Score:5, Informative)

    by DynaSoar ( 714234 ) on Wednesday September 23, 2009 @05:23AM (#29513155) Journal

    The first US phase 1 trial, yes. The FDA couldn't have approved the first neural stem cell trial because it was conducted in Sweden by Hakan Widner in 1982 http://www.nytimes.com/1992/11/26/us/success-reported-using-fetal-tissue-to-repair-a-brain.html [nytimes.com]

    George Carillo was the first recipient. He was the first and worst of the 'frozen addicts' covered in J William Langston's "The Case of the Frozen Addicts". His and others' poisoning by MPTP contaminated home made fentanyl resulted in Parkinsonism, which was partially reversed by fetal neural cell grafting http://en.wikipedia.org/wiki/MPTP [wikipedia.org]

    Their misfortune and subsequent treatment contributed to our now extensive understanding of Parkinson's and of the dopamine system, understanding that contributed to the success of Drs Arvid Carlsson, Paul Greengard, and Eric R. Kandel, recipients of the 2000 Nobel Prize in Medicine and Physiology. It also contributed to the discovery of endogenous MPTP, and that its conversion to MPP+ in neural mitochondria could be blocked in a majority of cases by trimethylnaphthoquinone, an MAO inhibitor found in tobacco.

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