GFP has the natural property of absorbing invisible ultraviolet light and producing green light – a discovery made in 1961 by Osamu Shimomura (who also shared the 2008 Nobel Award with Roger Tsien and Chalfie).

Chalfie’s “aha” moment, in 1989 at a department seminar, was a recognition that the light-producing properties of GFP could be harnessed as a sort of molecular flashlight.

It works like this: the gene for GFP is inserted alongside a gene, let’s say, that encodes a protein involved in the formation of the neural system. GFP “tags” the protein of interest, allowing it to be followed in real time inside living cells, with UV light as the trigger. It’s an incredibly powerful technique for revealing how and when genes are turned on, and how proteins move inside the cell.

In Chalfie’s case, he uses the tiny soil worm C. elegans as a model for human neuron development. C. elegans and the GFP technique have proven invaluable for studies in areas as diverse as neuroscience, organ development, and cancer.

Below is a nice 10-minute video from BBC News, telling the GFP story. It includes a few clips of live, green worms.

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For the technically inclined, video of Chalfie’s Nobel acceptance speech is here. The 2009 Nobel awards will be given in early October.

The more controversial and longer-term outcome, as suggested by the lead researcher, Dr. Karim Nayernia, is that stem cells might one day be used to generate sperm as a treatment for male infertility.

It’s worth pointing out that the same researcher accomplished a similar feat in 2006 using mouse embryonic stem cells to produce mouse sperm.  Only in this case, the experiment was taken one step farther – the sperm were injected into mouse eggs.  Though the mothers gave birth to live pups, they suffered from many abnormalities. All died within 5 months.

This example illustrates the technical and ethical challenges in moving reproductive technologies from the lab to the clinic. There really isn’t an easy path. It also should serve as a reminder that the United States needs to address the relative vacuum in efficacy, safety, and ethical oversight for the rapidly developing field of reproductive technology (a ~$10 billion/yr industry).

In the UK, reproductive technologies are closely regulated at the federal level by a governing body known as the Human Fertilisation and Embrology Authority (HFEA). The UK has carefully crafted rules on the research and clinical use of gametes (sperm/eggs), human cloning, genetic technologies as applied to human embryos or gametes, etc. In this case, any procreative use of sperm created from stem cells is prohibited.

In the United States there are few rules. The practice of medicine, by and large, is regulated at the state level. Our federal government leaders have been unwilling to call for a national discussion on implementing uniform rules for gamete donation, embryo creation, research, storage, etc. (a report from the 2004 Presidential Council on Bioethics notwithstanding). In fact, when Canada passed its Assisted Human Reproduction Act in 2004, it left the United States as one of the few developed nations without a national policy.

The result has been lax medical oversight, introduction of new technologies without sufficient studies on safety, and a piecemeal collection of rules.

The progress coming from the UK reminds us that we need to have a national discussion soon.

The piece concentrated on the DARPA-funded DEKA arm, developed by inventor Dean Kamen and his team of 40 engineers. Size and comfort were key issues in designing the limb. The final product is the size of an average person’s arm, weighs around nine pounds, and is buffered from the wearer’s body by small balloons that expand and deflate as pressure on the arm changes (the balloons inflate when the wearer picks up something heavy, and deflate when the arm is at rest).  Controlling the arm using their shoulders and pedals in a specially designed shoe, volunteers demonstrated their ability to pick up and drink from a soda bottle and eat a grape.

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The end of the segment touched on the future of prosthetic control, featuring Duke University engineer Jonathan Kuniholm. Kuniholm, who lost his forearm in Irag, demonstrated his ability to control a prosthetic hand using the nerves still intact in the remaining part of his arm. These nerves send out small electrical signals, which a processor in a prosthetic arm can be trained to interpret.

Similar work is being done here by Northwestern faculty member Todd Kuiken and his research team at the Rehabilitation Institute of Chicago. They are using an exciting new procedure called targeted reinnervation to reroute nerves that used to control a missing limb to different, intact muscle areas (rerouting nerves that used to control an amputee’s arm to his or her chest muscles, for example). These reinnervated muscles can then communicate with a prosthesis, again allowing the wearer to control their limb intuitively. Click here to read an SiS article on the Kuiken team’s work.

Essentially, this removes another block of the argument against stem cells – by removing the need for surgery, it cuts down on the risks involved in stem cell cures dramatically. Critical injuries that would require risky surgery to fix, even without the use of stem cells (examples include the addition of metal plates to hold together bone fractures) would now be nearly as simple to deal with as going in to the doctor for a vaccination. The nanoparticles used in the stem cells are already approved for use by the US for use in MRI scans, according to the article, so there can be no concerns about safety there. Hopefully, progress on to human trials will move swiftly and with success. (However, the scientific paper referenced by the BBC is as of yet unpublished. We shall see what unfolds with that in the future.)

It would seem the benefits of stem cells are only going up, as new breakthroughs in their research are cropping up all over the place, and the reasons not to use them are getting harder and harder to defend. Thus far, research has discovered ways to harvest stem cells from human skin, rendering the ethical embryo defense useless, and now we have discovered a way to easily make use of these stem cells in everyday medical use. All steam ahead in all areas, is what I say. Policy should be reflective of these breakthroughs, and hopefully Obama’s team can make a priority of it, after solving the economic crisis of course.

President Obama also issued a clear directive to all heads of federal agencies on the role that science and the scientific process must play in guiding his administration.  An excerpt:

“The public must be able to trust the science and scientific process informing public policy decisions.  Political officials should not suppress or alter scientific or technological findings and conclusions.  If scientific and technological information is developed and used by the Federal Government, it should ordinarily be made available to the public.  To the extent permitted by law, there should be transparency in the preparation, identification, and use of scientific and technological information in policymaking.  The selection of scientists and technology professionals for positions in the executive branch should be based on their scientific and technological knowledge, credentials, experience, and integrity.”

The eight-year winter is over.

The lifting of federal restrictions on stem cell research is being heralded in the scientific community.  Federal funds from agencies like the National Institutes of Health are one of the primary engines of basic research leading to medical and healthcare breakthroughs. Most experts expect the pace of stem cell research to increase significantly, though the promise of treatments or cures is still years away. The first human trial of a spinal cord injury therapy derived from embryonic stem cells was just approved by the FDA in January.

Not only will there be new funding available for stem cell research, but those researchers who relied on state or private dollars to fund their stem cell research programs during the ban will no longer have to run duplicate labs or facilities to separate their privately-funded research from that which was federally funded.

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The reasons behind the “ethical” side of the argument are straightforward: current embryonic stem cell technologies require the destruction of a human embryo to harvest the stem cells. Members of the “pro-life” movement equate a human embryo with a full person, thus such destruction is, in their eyes, essentially murder. Other opponents of stem cell research claim that manipulation of human embryos is dangerously close to human cloning, another highly controversial issue. Stem cell research for them would inevitably lead to a similar world to that found in the movie Gattaca (if you haven’t seen this film, it is absolutely worth a watch). On the other side of the argument are the scientists – stem cell research could save lives. Short, but none-the-less very strong.

The study reported on by BBC, found here, was able to manipulate the cells without the use of viruses – key to the side of research focused on moving around its opponents with regards to human embryos. Currently, it is possible to create stem cells from other adult cells using viruses to inject certain genes; however, such use or viruses makes the cells prone to becoming cancerous. It is this problem that makes such techniques more risky to use.

The study is so important because first, it manages to avoid the human embryo defendants, and make the technique considerably safer. While it is still in its early stages, this discovery could end the ethical debate about stem cells once and for all – and, more importantly, allow progress in an area with the potential to help many sick people. Frankly, if you ask me, I’d rather destroy the embryo and save a life than save the embryo and let someone suffer through life without treatment. But hey, that’s ethics for you – right for one man could be unthinkable for another.

What I find interesting (and a bit concerning) is that the company apparently never had to demonstrate to an independent regulatory body (e.g. the Center for Veterinary Medicine at the Food and Drug Administration) that its stem cell treatment actually works and is safe. It’s not that they skirted the law; it’s because the FDA is in an awkward place to regulate therapies derived from the animal itself.

Yes, the company cites many pleased pet owners and their own “proprietary data” as evidence for success. I have no real reason to doubt them. But given that many pet owners treat their companions as no less than small children, it would seem prudent to at least ask for proof that consumers are not being duped and their pets are not at risk.

Maintaining public confidence in the “trustworthiness” of medical science, regardless of animal or human, is especially importat at a time when the early data on human stem cell trials is starting to come out.

However, we must not forget that a huge portion of the money put toward research comes from government coffers. Research universities get much of their funding from government grants, which they in turn spend on teams of scientists that conduct studies and experiments in their respective fields. Today, with the recession at full tilt and new stimulus packages being put into place, what are the consequences of this structure to scientific progress?

According to a recent New York Times article, the new economic stimulus bill will relegate a large amount of capital through governmental divisions like the National Science Foundation and the National Institutes of Health. These organizations serve as the gateways from the government to the researchers, determining which projects to sponsor.

This calls attention to a few issues. First of all, since the government controls which foundations get funding, they largely determine what areas of science are prioritized more highly. This is a two-faced concept. Investments and research coming from the private sector are generally directed towards the areas that can generate the most profit, whereas the government would more actively promote more basic across-the-board research. This would be good, since a more diverse group of disciplines would be represented. However, federal control of the prioritization of ideas has the potential to neglect several important fields. For example, though it shouldn’t have been, embryonic stem cell research was a hotly contested issue, and in the end got only prohibitively restrictive federal funding. Government control allowed the misgivings of one man to get in the way of a potentially revolutionary area of new medical treatments.

Currently, many scientists think the issue is exactly the opposite: that the medical sphere is getting too much attention, and that more grants should be given out to researchers in other realms. The NIH, which is the bureau in charge of doling out health- and medicine-related grants, is receiving most of the money. Health is indeed an important area, but it is understandable that scientists in other fields are indignant at being marginalized.

Either way, though, the stimulus package means increased spending on science, which means more breakthroughs. The passage of the article that struck me the most said that since science had been largely pushed aside for the past six or so years, there are a glut of projects that haven’t been funded that can now receive public grant money. Over that time, the NIH alone has had 14,000 submissions that it deemed worthwhile, but lacked the capital to subsidize. With this newfound priority on science, researchers will finally have a chance to develop these projects. Expect advancements.

Cleverly, researchers spliced the gene in a specific part of the goat genome so that the goats would only produce the protein in their milk. It’s relatively straightforward to isolate the protein drug from other milk proteins and package it for market.

Given that the “bio-pharming” approach has been discussed for decades, it will be interesting to see if the promises of cheaper and higher quality drugs come true.

However, some animal rights groups have expressed concern about the use of animals for drug production. Other groups are concerned about monitoring these transgenic animals, so that they do not enter the food supply.

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