Science Friday: Stem Cells

In short, for these embryos, different rules apply depending on when they die.

To be sure, this rule applies only to federal funding. If you have private funding, you can still do pretty much anything you want with ESCs short of growing an entire human clone. So again, another contradiction – if “the fact that a living being is going to die does not justify experimenting on it or exploiting it as a natural resource”, shouldn’t that apply no matter whose money you’re using?

Truth be told, “only” 28% of biomedical research funds in this country are provided by the government. The remaining 72%, or $72B each year, comes from private industry and nonprofits. But there’s more at stake than just the money. It’s also an issue of public policy, of shaping the national discourse. If our President dismisses ESC research as being unethical, then this research will be discouraged and we will be denied the possibility of the huge benefits that can come of it.

In June, this question very suddenly became more than academic for me. In June, my wife and I learned that our son, now 19 months old, will never walk. He has a genetic neuromuscular disorder called Spinal Muscular Atrophy (SMA), type 2. The very short version is that his motor neurons don’t work right; furthermore, they are dying – as he gets older, his condition will worsen. He has limited use of his legs, which aren’t strong enough to support him. He’s never even crawled, but we are hopeful that physical therapy may eventually make this possible, though it seems unlikely. Walking is pretty much right out.

Unless, of course, a medical miracle occurs. Which, actually, is starting to look possible these days. In September, Italian researchers reported on an experiment wherein they afflicted mice with SMA (by disabling the same gene that causes it in humans) and then injected these mice with specially prepared ESCs. The hoped-for result was somewhat disappointing: only a very small number of the injected ESCs grew into neurons in more or less the right place. But this experiment had its Eureka moment: the presence of the stem cells somehow encouraged the damaged neurons to perform better, recovering about 50% of their lost capabilities.

And last week, University of Wisconsin researchers announced that they can now derive seemingly full-functioning stem cells from a patient’s skin cells. They have used this technique to make liver cells from skin cells, as well as grow SMA-afflicted neurons in a lab out of an SMA patient’s skin cells. This has huge implications for drug-testing experiments, as well as observing in great detail the progression of all sorts of genetic disorders on a cellular level.

Now, it might seem to you that, even if Obama hadn’t announced that he would probably overturn the stem cell policy on day one, that this new technique obviates the need for ESCs. And you’d be right. If these derived stem cells live up to the promise that they hold (and more research needs to be done to confirm this), they will be a non-controversial replacement for embryonic stem cells. Progress will be able to go forward once again on research that could affect so many lives.

But how many years did this policy hold us back for? How many diseases cured in the next 8 years will we look at and say, “Those could have been cured when Bush was still in office… if only…”

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Of course, I can’t write a public article about this without going into PBS-style fundraising mode. If you want to find out more about SMA, or donate to research to find a cure, I highly encourage you to investigate the Families of SMA.