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."

Comments

Dr. Stephen Hawking

[Anonymous Coward]

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

Big News?

[lobiusmoop]

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

Re:Big News?

[Daniel Dvorkin]

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:Big News?

[PopeRatzo]

You think it's the FDA that's been holding up stem cell research, and not the religious yahoos?

Re:Big News?

[Bruce Perens]

Well see, we have this massive bureaucracy in the USA called the "Food and Drug Administration

Were you aware that there are other countries? :-)

All of the folks arguing about health care don't make the connection that Hawking has lived so long in a country with socialized medicine.

Re:Wonder how this will cost

[Threni]

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.

Re:Wonder how this will cost

[Chris Burke]

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.

Existing Conditions?

[BJ_Covert_Action]

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?

Stem cells = Cancer

[sonnejw0]

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.

Re:Stem cells = Cancer

[sonnejw0]

Excuse me, but ignorant is something I am not. I am a Neurobiology researcher working specifically on neurodegenerative diseases. Your Google profile to which you have linked in your signature suggests you're unemployed, at least that's what I take "futurist", "gamer", and "slashdot.org commenter" to mean. Failed IT career?

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:What kind of stem cells?

[interkin3tic]

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:

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, 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.

Misleading Title

[DynaSoar]

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.