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.
The idea of the Canadian study is that a patient’s own stem cells are the most direct and effective way to repair damage and rebuild function in the heart. But because the stem cells from a damaged heart are not working up to normal capacity, scientists tested and found that adding extra copies of a gene that “stimulates blood vessel growth and improves tissue healing, known as endothelial nitric oxide synthase,” improves that function.
In other words, the gene stimulates the patient’s stem cells to reproduce more quickly and do their magic to help the heart heal itself. The Canadian trial is for post-heart-attack patients.The UK trial will be using a carrier virus to insert a gene into heart failure patients to help their hearts pump better.
Nothing but good news here – except that it will be two and three years before results are in. Stay tuned.
A researcher spent ten years and finally succeeded in getting human stem cells to grow into two distinct types of cells—auditory neurons and inner-ear hair cells. And now he’s used the appropriate neuron-type cells to re-connect the inner ear to the brain. In other words, to restore nearly 50% of hearing in gerbils (whose inner-ear hairs had remained undamaged).
It’s very niche research, but it demonstrates that restoring hearing is definitely possible to some degree. Plus other research shows it’s possible to restore hearing to mice born deaf and yet other gene therapy research showing you can restore function to hair, eye and heart cells and smell in mice.
So much of historical medical research has focused on devising invasive, even barbaric methods of arresting sickness. We refer to it always as “fighting” disease, killing cells, conjuring up images of swords, bullets and bombs. As we continue to plumb the magical powers of stem cell and gene therapies, it’s encouraging to think of the balance now slowly tipping more and more toward non-invasive ways of restoring, gently giving back, quality of life to those who suffer.
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.
stem cells after heart attacks. Healthy donors’ cells seem to work better than using the patient’s own compromised stem cells. Makes sense—if the heart is damaged, doesn’t seem right that cells from it would be okay. But of course it only looks obvious once we’ve actually learned it.
Fighting infection by controlling production of nitric oxide (NO). Silicate powder interacts with light to release NO and kill gram-negative bacteria without harming the host. Another useful partner for NO—and another good reason to get out and enjoy the sun.
Saving lives by preventing organ fibrosis (scarring). Peptide from collagen has prevented scarring in human skin samples and in lungs and skin of mice. It even reversed fibrosis that had already begun in mouse lungs. Wonder if they’ll ever be able to use it for COPD and emphysema victims.
Minimizing damage to healthy tissue by using nano-constructed cages for targeting and delivering drugs to specific cells. They’ve got the concept but need to work on the execution—e.g., controlling the porosity of the cage so the loaded drug doesn’t leak out before it reaches its target.
So much promise. My imagination goes wild with visions of a world with so much less suffering.
A small new study suggests that some kidney transplant patients who receive bioengineered stem cells from their donors may not need anti-rejection drugs long term. Five of eight patients who received the stem cells in addition to the organ were able to stop taking immunosuppressants after one year, according to Science Translational Medicine. If they can replicate this in a bigger study, it could mean reducing fewer drugs for transplant patients and being able to use more donor organs for transplants. There are typically around 47,000 people a year waiting for a kidney and that wait can currently take years.
Learn how stem cells work for transplants
Looking into the process of using them, NIH researchers have developed a way to monitor how stem cells function once transplanted. The method uses magnetic resonance imaging (MRI) and consists of two FDA-approved drugs that can attach to cells and a third that is detectable by MRI. The technique is being tested in brain tumor patients who receive transplants of engineered neural stem cells, according to Molecular Imaging. The technique will help doctors understand how many of the cells they transplant actually reach the target organ, and so help them regulate how they administer the cells, plus how to adjust doses and timing.
Wouldn’t it be great if we didn’t have to resort to selling organs—which would likely turn out to produce another crop of heart-wrenching episodes of Law & Order (the original)?
Parkinson’s is one of those diseases that must be studied in human neurons because animal models that don’t have the parkin gene never develop the disease so they can’t be used. And of course we can’t just cut into people’s brains for scientific purposes.
Researchers have been able to create stem cells by introducing genes into cells by using viruses. The cells then became stem cells. Unfortunately, viruses are known to mutate genes and that can easily trigger cancer in new cells.
Now a new technique uses plasmids to create stem cells—beating heart cells. Plasmids are elements of DNA that reproduce themselves inside cells and then gradually decay. Experimenters say the new technique is is affordable and efficient. They reported it “worked consistently for 11 different stem cell lines. In each of the 11 cell lines, each plate of cells had around 94.5 percent beating heart cells. It also worked for embryonic stem cells and adult blood stem cells.”
One day soon we’ll be beyond the arguments about where stem cells come from and can move on to discovering more of the healing secrets nature seems eager to unfold.
The same view with age-related macular degeneration
I saw first-hand how miserable it is to be blind when you get old. It crippled my ex’s grandmother for years. She couldn’t watch television, sew, read, or do anything to occupy herself in the last decade of her life. Then his mother went through the same thing.
Getting old is bad enough. If we are also robbed of our ability to navigate the world and are unable to enjoy so many formerly rewarding activities, it makes the struggle even more difficult.
Thank heavens the magic of stem cell therapies works for problems of the elderly, too. And that our researchers are interested in exploring ways to help people age more gracefully.
We already know about a host of diseases we can hope to battle more effectively using stem cells. Now I’ve just read about another inspired use of the seemingly limitless power of stem cells to help human beings battle disease.
The gene FOXM1, injected at higher-than-normal levels into stem cells from an adult human mouth, encouraged abnormal growth that mimicked the abnormal cell growth common with early cancer.
There is evidence that environmental and behavioral factors like UV ray exposure and smoking—the same stuff we’ve come to understand can result in cancer—can lead to increased levels of FOXM1.
I know this study doesn’t say this, but I’m very excited about the possibilities. How much faster may we be able to get to clinical trials for various treatments and drugs by using easily and readily available human stem cells as proving grounds instead of having to first experiment on animals and, later, pray that we’re getting it right with human beings.
Talk about a promising study…
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