Stem cell banks could pay medical dividends

Alan Boyle Science editor • Profile • E-mail

Someday, researchers will be able to order up living human cells afflicted with the genetic flaw they need to study. Gene-splicers will be able to correct the flawed code that causes diseases. And if you're struggling with one of those diseases yourself, your doctor just might be able to fix you, using semi-tailor-made cells ordered from a biobank.

The whole process would be almost as easy as drawing blood from a blood banks today.

Such are the breakthroughs that could spring from future efforts to create stockpiles of reprogrammed human cells.

"Banking cells sounds pretty boring and trivial," said cardiologist Timothy Kamp, co-director of the University of Wisconsin Stem Cell and Regenerative Medicine Center in Madison. "But it turns out that there is a lot of hard work that goes into that, to actually guarantee that you have high-quality, reproducible lots of cells."

Birth of a super-cell
The cells are known as induced pluripotent stem cells, or IPS cells. Last November, research groups in Japan and the United States announced that they had converted ordinary human skin cells into super-cells that appeared to be as versatile as embryonic stem cells, with the ability to transform themselves into virtually any tissue in the body.

For years, embryonic stem cells have been held up as the gold standard in regenerative medicine. By massaging such cells in just the right way, doctors could create new cardiac cells for faulty hearts, new neurons for broken spinal cords, new pancreatic cells for diabetics - the list goes on and on. But the cells have to be harvested from human embryos, which raises ethical as well as medical questions.

If IPS cells can be made safely, and if they truly do the same things that embryonic stem cells can do, researchers could theoretically take a skin sample from someone who had a particular genetic disease and manufacture an unlimited supply of cells that manifested that disease. That would open up a promising path to future cures.

From the lab to the clinic
The first step would be to study the living tissue in the lab, to find out how it responds to various therapies and genetic tweaking.

"I think we'll get things out of cells in a dish faster than putting cells in people," Kamp said.

He envisioned a repository for IPS cells that manifest genetic conditions ranging from sickle-cell anemia to a predisposition for bad drug reactions.

For example, Kamp is interested in the factors that contribute to a type of potentially fatal heart condition known as long-QT syndrome. Studying heart cells from a wide range of individuals with an inherited disposition for the syndrome could tell researchers which people are more vulnerable than others - or which ones might suffer an attack by taking a particular drug. (A decade ago, the allergy drug Seldane and some other antihistamines were banned from the U.S. market because of such a linkage.)

An IPS cell bank could be the source of heart cells for such studies, and many others besides. The result could be new drugs (or old drugs, perhaps like Seldane) that are suited to your genetic makeup.

Farther down the line, researchers could find ways to tweak the DNA in IPS cells to fix genetic flaws and create healthy tissue tailor-made for you. For example, UW-Madison dermatologist Joyce Teng is already looking into whether cells can be tweaked to fix rare genetic skin condition known as ichthyosis, Kamp said.

Cell transplantation has been applied to a wide range of maladies - including heart disease - but some diseases will be more suited to cell therapy than others.

"There are a number of fairly rare diseases that might end up being some of the first headline successes, because they're terrible diseases wtih pretty devastating shortening of life expectancy ... and also the genetics have been well-studied," he said. "The more complex diseases might take a little more work."

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