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FDA OKs First Human Trial of Neural Stem Cell Therapy
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."
Any chance that this could be passed through quick enough to prolong a certain genius' life?
It makes me sad that this is news in 2009. This should really have been commonplace research by now.
"I bless every day that I continue to live, for every day is pure profit."
Kids eat too much anyway - it'll do them good. How much does a sandwich and an apple cost, anyway? You're not going to fund much research for that.
Ah, does that really matter?
Kid goes to school, then has to spend every cent taking care of a failing parent. Parent dies anyway, kid broke for life.
Kid doesn't go to school, gets a job, puts self thru school, and both parent and kid come out better.
Sig Battery depleted. Reverting to safe mode.
I see you did well at Economic Fallacy School.
-jcr
The only title of honor that a tyrant can grant is "Enemy of the State."
Wonder how much the treatment will cost? How many kids don't get to eat at school so that someone gets this treatment.
Don't worry, the people who can't afford lunch for their kids will be the same ones who can't afford this treatment. So nothing you would be concerned with.
The enemies of Democracy are
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 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?
Motorcycles, Robots, Space Gossip and More!
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.
Why does it matter?
The Kruger Dunning explains most post on
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 used more profitably.
There have been several studies involving prayer and healing, with extensive double-blinds.
It only make the person who prays feel better, which is no small thing, but praying for someone else does not promote healing.
In the case of certain sects who believe in prayer instead of medical treatment, it actually promotes death.
You are welcome on my lawn.
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 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.
It mentioned that these were patented methods. I don't know much about patents, but I did find this patent issued to neuralstem biopharmecuticals ltd (same company?).
The abstract to that patent:
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, Use of fuse nicotinamides to promote neurogenesis.
The abstract for that one:
I'm less of an expert on this, it's a lot of biochemistry I'm not familiar with, but from the summary:
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.
WTF?
That website is horrible and factually wrong.
What's that you say?! A troll has a link in his sig to a website that is wrong!?! Rally the Internet Justice League - this evil cannot be allowed to stand unopposed!!!
Now that I think about it, I'm pretty sure everything I just said is completely wrong.
Neural Stem Cell Therapy - It Tickles!!! (tee hee .......eyes go glassy .... drool begins)
L'esperienza de questa dolce vita (The experience of this sweet life) - Dante Alighieri, The Divine Comedy
That's a hell of a sandwich if you're using the whole tub of Mayo, a pound of tomatoes, a whole loaf of bread, a pound of bacon, a whole head of lettuce, and alongside it a 10 serving bag of fritos, and then drinking a whole quart of milk? Can I ask what your BMI is?
In reality, it's more like this. You use 1/25th of the tub of mayo, so 1/25 of $2 is really more like 8 cents. Hell, let's say you're generous. 25 cents. We'll assume it's one of the nice loaves of bread that only has like 10 slices. so 1/10th of the $4.50, so .45. Let's assume you use 2 romas (they're kinda small) on the sandwich. In my experience that's at most around a half pound. So we'll round and say .40. 3 leaves of lettuce is about 1/10th of a head if it's a medium sized head of lettuce, so rounding up to .13. We'll go with the bacon since you told us volume there, heck, let's make a bacony BLT. 4 thick slices accounts for .30 pounds I'd wager, so that's $1.35 (most expensive item so far!) A single serving bag of Fritos runs 40 cents after tax (they're those 3 for a dollar bags). We'll assume you eat the whole fruit. Finally, 2 servings of milk (we'll even go with the good dairy one you mentioned) would only be half of the quart, so round that up to 88 cents.
The REAL cost of your sandwich would be $4.61. Give or take.
Now if you bought that sandwich at a sandwich shop, yeah it'd be like 15 bucks. Mmmm, capitalism.
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?
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 also because they have a number of unique features that make them promising to be useful for a number of conditions where adult stem cells would not suffice (such as tissue types that lack adults stem cells, like the pancreas). In any case history suggests that understanding how the body functions is absolutely essential for modern medicine and thus embryonic stem cell research is worth pursuing if for no other reason than its academic value."
Calling research into fundamental aspects of how our bodies develop "dead-end" is pretty much a strong display of profound ignorance about modern medicine.
There's also the fact that taking care of somebody who is slowly dying of ALS isn't exactly free. Nor(on average) are the lost years of life.
There are certainly medical treatments that will never be viable in economic terms, that are available(or not) basically for ethical/humanitarian reasons. However, cures for diseases that would otherwise involve a number of years of expensive decline and an early death may well not fall into that category. Because R&D is expensive, the per-case cost of the first round is going to be crazy; but volume use could end up being a win in purely financial terms, not to mention the obvious non-monetary benefits of less painful lingering death.
Do you mean like McDonalds that runs the Ronalds MacDonalds Houses so that the parents of sick children have a place to stay or Eli Lilly that has funded millions of dollars in scholarships? Get a clue.
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
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
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.
"I may be synthetic, but I'm not stupid." -- Bishop 341-B
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 the electric chair anyway."
All I mean is that the ethics of ESC are severe -- and even amplified when humans are in play. If your argument for "research only -- no treatments expected" holds, then would using animal ESCs sidestep any of this for you, while still gaining many of the research benefits?
On the funding issue too, I'm wary of the uninformed public making choices as to what needs to be spent where when they rarely have the context or understanding of what the research is supposed to accomplish.
I suppose I think that when I give the government money, I am hiring them to provide a service for me. If I don't want my car mechanic adding a spoiler onto my car, I don't intend to pay him for it. I may not understand the implications of the performance and whatnot, but if I would rather it be spent on something like better roads or schools, why shouldn't I have a say in where my money goes? Yes, I'm sure that these research dollars will have paid off in the future for our children and their children, but if my child is flunking basic math right now because their school can't afford good teachers, I think it's a bit unfair to call them an ignorant hick just because they don't place quite the priority on stem cell research as you do, and would rather see the government shuttle that money elsewhere.
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.
It shouldn't be a democratic process because then we'd end up spending half our money on "Does prayer heal?" and we'd all be smoking like chimneys wondering why we were dropping dead.
Well it's good that we're not a democracy here -- we're a republic. Sadly, it seems that we're trending ever and ever closer to a democracy though, which has never been a stable (or productive) form of government.
I don't mean to sound elitist, I think most people are -capable- of learning enough to make good decisions on funding of stem cells, but they clearly haven't.
Then if people aren't willing to give you their money, then in the meantime, spend your own. Again, you seem upset that people aren't willing to just give you their limited funds for your priorities. Noone's stopping you from spending your own -- they're just not happy when you try to reach into their pockets for your interests. Plenty of disease research groups (such as March of Dimes, etc) evangelize and raise awareness and gather donations for a cause or for research. It's a bit awkward when such evangelism is skipped and the conclusion is mandated through legislation on the federal level. Yes, fundraising is work -- but at least you're respecting people and their basic liberties. I know it's tempting to want Big Brother to handle all of this for us, but when the government looks like a hammer, all of our little problems start looking like nails.
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 sacrifice one human embryo per patient -- SCNT requires tens (if not hundreds) of embryos to be created per patient. So in the context of ESCs being used on patients, I disagree with you in that useful ESCs are _not_ self-reproducing, and must be individually cloned from unfertilized eggs (traditionally, left over from IVF treatment).
but again, ESC are still essential for basic research.
And again, must they be human? And if ESCs have no viable treatment avenues, and we're aiming to move SC treatments into the practical realm, then it seems that we should be focusing on researching things that have more potential. More "potency" if you will.
Ethically, there are serious concerns for using ESCs, sure. But even pragmatically, ESCs just have so many barriers to overcome, that things like cord blood and IPSC seem to actually be able to go somewhere (such as this news article). If we know that there's in all likelyhood a dead-end at the end of the ESC road, wouldn't it be wiser to focus on IPSC? You seem reluctant to give up ESCs because of research -- fine. Why not continue with simian ESCs, or if you insist on human, existing stem cell lines? As you said, they're self-reproducing.
Please correct me if I'm wrong, but many of our seminal ESC researchers have already moved onto the more promising IPSC research. ESCs may have academic value, and you want to dedicate another 40 years of research to them. Given the power of IPSCs, and the significant scientific barriers to the usability of ESCs, combined with the serious ethical implications, I don't think that you have provided compelling evidence for why public funding is a reasonable thing to expect for ESC research.
Again you're only thinking in terms of treatments. ESC has more value in basic research.
But if it looks like a dead-end road, why spend our time messing with it?
Give ESC 40 years at least before you say they have no theraputic value.
I don't say that cloning has no therapeutic value. It's just that we've moved on. IPSC has proven to be so valuable so quickly, it's a veritable gold mine promising near-immediate returns. Given our extremely limited resources for scientific research, wouldn't you want to focus on things that have potential? Unless we seriously compromise human ethics, there will never be enough embryos to go around for everyone who would want ESC treatment. It's just not there.
The scientific community has moved on. Heck, even James Thomson -- the father of modern ESC research has moved on to IPSC. It's like you're asking for another 40 years to research some outdated and debunked theory because there may be value in it, when we'd rather all get on with something that's proven to be effective.
I've never had any frustration with people who feel it's unethical. I myself have questions about the morality of ESC. I think it should be funded
It never wasn't funded. It wasn't banned or unfunded under Bush, it wasn't banned or unfunded under Clinton. The only thing that those executive orders did was ban federal fund