FDA OKs First Human Trial of Neural Stem Cell Therapy 149
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."
Re:Wonder how this will cost (Score:4, Informative)
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.
Re:What kind of stem cells? (Score:1, Informative)
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.
Stem cells = Cancer (Score:4, Informative)
But those are the problems this research will address. I'll be eager to see the results in two years.
Re:Big News? (Score:4, Informative)
Well see, we have this massive bureaucracy in the USA called the "Food and Drug Administration", whose job it is to kill people by impeding medical research.
The job of the FDA with regards to medical research is to ensure that what's called "medical research" is actually both "medical" and "research" by reasonable definitions of those words. Do you really not understand why this is necessary?
Re:What kind of stem cells? (Score:4, Informative)
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:
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:
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.
Re:Stem cells = Cancer (Score:3, Informative)
At any rate, stem cells have the potential to endlessly reproduce based on the presence of growth factors. Many forms of cancers and precancerous states are characterized by rapid and uncontrolled expression of growth factors. If a cancer patient with one of these common root causes is introduced to these stem cells, they suddenly will produce a massive tumour at the injection site. Which is exactly the concerns expressed by the research group that is doing this phase 1 safety trial, hence the need for a safety trial.
My research involves delivery methods for introducing growth factors that activate innate progenitor cells to replace cells damaged or lost in neurodegenerative diseases. There is a reason why there is only a miniscule fraction of progenitor and stem cells in the adult human body: because if there are a lot, there is a huge chance that one of these cells will either be mutated by environmental radiation, or a mutation of a nearby cell that causes it to dysregulate expression of hormone signals. It is an evolutionary adaptation to improve fitness.
My homepage is a list of concerns about current political and social trends. It obviously does not reach deep into every issue. I don't have the time to do that. You obviously think you're far superior to everyone else in the world, though, so why bother with me and my 'little' views. It seems that my opinions have so rattled you emotionally that you cannot possibly deal with it without being 'snarky' on the internet. Perhaps you could better spend your time by earning an income, or acquiring amicable social skills.
Re:Wonder how this will cost (Score:2, Informative)
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.
Re:Wonder how this will cost (Score:3, Informative)
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.
Misleading Title (Score:5, Informative)
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.
Re:What kind of stem cells? (Score:3, Informative)
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