In this case, a team of experts from multiple disciplines “modeled the cardiovascular disease Barth syndrome, a rare X-linked cardiac disorder caused by mutation of a single gene called Tafazzin, or TAZ. The disorder, which is currently untreatable, primarily appears in boys, and is associated with a number of symptoms affecting heart and skeletal muscle function.”
The disease in this case results in very weak contractions of the heart muscle. The hope is that they may also eventually be able to model functioning tissue from patients with other diseases that produce other functional problems.
Why would they want to create functioning yet diseased human tissue outside a human being? The answer is that they can then experiment with and test all kinds of drugs and other treatments that they might not want to use directly on an actual living, breathing human being. In this scenario they were able to inject a genetic product that corrected the contractile problem right there in the lab.
While the article doesn’t say this, I’m thinking it could also mean in the long run fewer animals used for experimentation. And it could lead to shorter times before promising therapies can get to clinical trials.
Up til now stem cells have generally been injected or otherwise inserted into living tissue to get them to grow into specific types of organs or other tissue. Now scientists in the UK in collaboration with an Israeli research team have managed to grow human bone in the lab with stem cells from fat tissue. They’ve already successfully implanted a piece of lab-grown human bone into a rat’s leg, where it joined nicely with the creature’s existing bone.
The researchers use scans of the damaged bone to construct a gel-like scaffold that shows the stem cells how to grow into the shape of the needed replacement. Then the mold of stem cells is turned into actual bone in a special machine called a bioreactor that provides the conditions needed for this miracle to take place.
The bone grown from stem cells could theoretically be used to replace damaged or missing bone—for example in repairing a cleft palate. They mention using it to fix bones that have been crushed or otherwise mangled in accidents.
I suspect that once this process is perfected, far down the road, doctors may eventually be able to use it to construct replacement bone for arthritic hips and knees. Too bad it will be long after I and my arthritic relatives will be around to have any need for it.
If we needed any further proof of how far-reaching the effects of stem cell research can be on making medicine not only less invasive but also more efficient and effective, now comes another momentous discovery.
According to a BusinessWeek article, a couple of pharma companies have developed a way to use stem cells to develop “human” tissue (independent of a living, breathing person), and they’re using the tissue to test drugs for potentially dangerous side effects.
The cost to develop a new drug—which can in some cases exceed $4 billion—usually includes animal trials and then human trials. Researchers have found that stem-cell-generated tissue—they are regularly producing 7 billion heart cells a month from skin and blood stem cells (not embryonic)—mimics the reactions of actual human tissue. And that allows scientists to test drugs for bad effects long before human trials would normally be scheduled.
The happiest part of this report is that this isn’t just the promise of stem cells—this work is actually going on now. One of the pharma companies used the stem-cell tissue to re-test a drug they’d worked on earlier and discarded because of a bad side effect on test animals. They found the drug had exactly the same results on the stem-cell tissue as it had had on the animals. The company realized if it had had this capability back then, it could have stopped development much sooner and saved a bundle.
Consider the potential benefits of making full use of this capability:
How much faster might useful drugs get through the pipeline and out to the patients who desperately need them?
How much might the cost of new drugs come down with pharmaceutical companies saving millions of dollars in development costs?
How many animal lives might be spared because research can be done on this “artificial” tissue instead of on rabbits or mice or chimps?
I say again, with stem cell miracles around every corner, we’ve at last discovered heaven’s own way of healing. And what we do with that power now and in the future will be limited only by our own imaginations .
Looking at how bioscience news affects business, higher education, government – and you and me