Stem cells treat wasted muscles - Dogs with muscular dystrophy walk better after injections.

news@nature.com ^ | 15 November 2006 | Helen Pearson

[neverdem]

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Published online: 15 November 2006; | doi:10.1038/news061113-13

Stem cells treat wasted muscles

Dogs with muscular dystrophy walk better after injections.

Helen Pearson

Golden retriever dogs make a good animal model for muscular dystrophy Rossana Tonlorenzi & Maurilio Sampaolesi, San Raffaele Scientific Institute

An infusion of stem cells scraped from blood vessels has helped dogs with a form of muscular dystrophy to walk more normally, perhaps heralding a treatment for the human disease.

Muscular dystrophies are a group of widespread genetic disorders in which the muscles gradually break down. The most common form, called Duchenne muscular dystrophy, is caused by mutations in a gene called dystrophin and inevitably causes paralysis and death.

Giulio Cossu of the San Raffaele Scientific Institute in Milan, Italy, and his colleagues had previously shown that a type of stem cell called a mesoangioblast, which lives in the walls of blood vessels and can generate muscle cells, can help mice with a form of muscular dystrophy¹. So they set out to study the effect of the cells in an animal model that more closely mimics the human condition: golden retriever dogs with a mutation in their dystrophin gene.

The team extracted the blood-vessel stem cells from normal dogs, grew them to large numbers in the lab, and injected tens of millions of cells into sick dogs in five monthly injections.

Afflicted dogs usually develop troubles walking by 8 months old. But many of the treated dogs did much better; one was still walking well at 13 months of age. The results are published online in Nature².

Cossu says that the team has already identified the equivalent stem cells from humans and that clinical trials could start in a few patients by late 2007. But they will need to test more dogs first. "We're not rushing," Cossu says.

It's uncertain whether this type of treatment can repair heart muscle or the diaphragm, which also fail during muscular dystrophy. Furthermore, it is unclear whether the stem cells might lodge themselves elsewhere in the body and cause unwanted side effects.

Straight to source

Stem cells can travel through the blood, squirming their way out into muscles throughout the body. Rossana Tonlorenzi & Maurilio Sampaolesi, San Raffaele Scientific Institute

By examining the dogs' muscles, the team found that the stem cells had travelled through the blood, squirmed their way out of capillaries and into many muscles throughout the body. There, they had fused with existing muscle fibres, manufactured the missing dystrophin protein and rejuvenated the fibres.

Researchers have been testing other types of stem cell to treat muscular dystrophy, such as ones extracted directly from muscle or bone marrow. But these cell types have either revived only a few muscle fibres or needed to be injected directly into each muscle.

The real advantage of the mesoangioblast cells is that they can be injected into the bloodstream and swim to most muscles in the body, says neurologist Thomas Rando who studies muscular dystrophy at Stanford University, California.

Stem cells have been touted as the way to regenerate numerous failing tissues — but none have yet become tried-and-tested human treatments. Rando says that muscular dystrophy is a good disease to target, because the replacement cells only have to fuse with existing muscle fibres, rather than spawning brand new cells or tissues.

Tailor-made

In terms of treating human disease, cells donated from another person are not ideal because a patient has to take lifelong immunosuppressant drugs to avoid rejection.

Cossu's team looked at one possible solution to this problem: they extracted stem cells from sick dogs and used gene therapy to insert a working copy of the dystrophin gene into these cells. They then injected the dogs with their own, 'fixed' cells.

Dogs treated this way began to manufacture dystrophin, but they did not have improved symptoms. Cossu thinks the results might be improved by using a different version of the dystrophin gene (which is notoriously difficult to handle because it is so large - in humans it is more than 2.5 million base pairs long, and by far the largest gene in the human genome).

From all angles

Other methods to treat muscular dystrophy are nearing clinical trials. Some groups are trying to deliver a replacement dystrophin gene to all muscles using a virus. Others are working on drugs that encourage a cell to ignore the mutation in its gene and manufacture a correct form of dystrophin protein.

"I think we may be approaching a time when we'll see multiple approaches combined," says muscular-dystrophy researcher Jeffrey Chamberlain of the University of Washington, Seattle.

Visit our newsblog to read and post comments about this story.

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References

1. SampaolesiM., et al. Science, 301. 487 - 492 (2003). | Article | PubMed | ISI | ChemPort |
2. SampaolesiM., et al. Nature, published online doi:10/1038/nature05282 (2006).

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http://news.nature.com//news/2006/061113/061113-13.html

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[sageb1]

My niece died at the age of 10 from Werner Hoffman, a form of MD. I never knew dogs got it.

[AndyTheBear]

Sounds like a NON-embryonic source to me. An embryo (at the stage where stem cells are harvested) would not have blood vessels.

Such omissions are deliberate lies. The fact that this was the noncontroversial type of research that is far more succesful--and they left it out. Its just B.S.

[weegee]

Shoulda been in the headline (the real one, editor hiding a significant detail).

[Scotswife]

"Such omissions are deliberate lies. The fact that this was the noncontroversial type of research that is far more succesful--and they left it out. Its just B.S."

It's too soon after the Michael J. Fox hoopla. They don't feel like eating crow.

[neverdem]

Thanks for the link & history.

[soozla]

Equine vets are using stem cells from the animal's own fat to re-generate and repair tendon and ligament tissue on horses who have sustained these injuries. I'm looking into this procedure now for my horse!

[soozla]

VET-STEM

[Criminal Number 18F]

This recounts two attempted therapies, a succesful one with autologous stem cells, and one with poor success using genetically manipulated donor stem cells.

Yet the word autologous, and the word adult, do not appear in this article. This may account for the MSM playing this story as an embryonic stem cell case -- their "science" reporters may be so poorly educated as to not know the difference, and they have the template all ready. (Dim, but brilliantly coiffed....)

However, another MSM technique is also at play... and it may also have been used by the authors of this study! It is to avoid detailed discussion of the source of the stem cells, and to deliberately elide the difference between autologous and umbilical stem cells (which have been very successful) and embryonic stem cells (which, despite much research, are still at a stage wherein they are unsafe for human trials).

But yes, for anyone asking -- this is an adult cell, non-destructive therapy.

The article also contains an outright false statement: that stem cells have not been used therapeutically. According to science (and war) correspondent Michael Fumento, "for the last few years persons suffering from limited vision or outright blindness from corneal defects have had their vision restored through corneal stem cell transplants."

Fumento links to this site:

http://healthlink.mcw.edu/article/1031002392.html


And his own post (with more info about stem cell therapies) is here:

http://www.fumento.com/weblog/archives/2006/11/election_wont_h.html

Be sure to find his index of stem cell posts and articles. He has written prolifically on the subject.

d.o.l.

Criminal Number 18F

[Criminal Number 18F]

Good luck to you and your animal.

In addition to these animal therapies, there are over a thousand clinical trials underway of stem cell therapies at this moment.

Every stankin' one of them from adult stem cells of one kind or the other.

The reason that the press and the Dems promote ESCs is not because they want to do what ESCs can do (which is cause massive teratomas, so far). It's because they want abortion clinics to have a market for what is now waste that they must pay to dispose of.

While Planned Parenthood is a non-profit organization (although one that pays its leaders VERY well), its abortion clinics are often for-profit arrangements. IMHO this is all about giving them a new product line.

d.o.l.

Criminal Number 18F

[neverdem]

The article also contains an outright false statement: that stem cells have not been used therapeutically.

Good catch! I'm so used to seeing the term "stem cells" used haphazardly, I do it sometimes myself, that I just assumed that she was referring to embryonic stem cells.

"Stem cells have been touted as the way to regenerate numerous failing tissues — but none have yet become tried-and-tested human treatments."

Thanks for the links.

[hocndoc]

No, thank you! You keep us all informed on the Sci-tech headlines - I just give my opinions on my blog editorial comments website.

[AlexW]

"stem cells were from a "non - embryonic" line."

This is a great trick of the leftist MSM. All stem cell
research is lumped into embryonic stem cells.
If your against "embryonic" stem cell use, you must also be against ALL stem cells.

They do the same with global warming. If you don't believe that man is the reason for warming, then you don't believe there is ANY warming.

It makes my blood boil !

[hocndoc]

From the editorial on the research on Nature (emphasis is mine):

In one animal, the cells were released from a catheter into the aorta — the vessel leaving the heart that supplies the body with blood. This therefore allowed more widespread dissemination of the mesoangioblasts. The results of the stem-cell infusions were dramatic: this last animal displayed a marked improvement in its dystrophy and was walking well 5 months after the final injection; the other animals recovered to a lesser degree. In general, dogs receiving donor cells improved more than those receiving corrected autologous mesoangioblasts. Some muscles in the injected dogs had nearly normal levels of dystrophin, and even the muscles with only moderate levels of dystrophin showed significantly improved structure and function (Fig. 1).

At that LifeEthics posting I that I linked to earlier, there's the information that the first discovery on the benefit of (adult) stem cell therapy in Muscular Dystrophy came 1998when doctors discovered that a boy who had had a bone marrow stem cells transplant actually also had an unusually mild form of MD. It seems that his MD had been partially treated by the bone marrow transplant he'd received as a baby to cure his Severe Combined Immune Deficiency ("bubble boy" disease). Now, umbilical cord bone marrow transplants are done to treat the disease, but they have a huge risk compared to what we hope this (possibly autologous) specific stem cell infusion treatment would have.

(My grand daughter had an umbilical cord blood transplant in 2001, for a different kind of congenital immune dysfunction and bone marrow failure - her risk of rejection was very low when you add in the fact that she had no immune system to speak of to begin with and UCBlood has 1/4 the risk of graft vs. host that bone marrow stem cells carry. She is beautiful and healthy and 6 years old, now - we took her to the Spurs last night for the first time and she got to be on the jumbotron - of course, she, like her "G-dad" (granddad) was more interested in the Silver Dancers than I'd like.)

The search for "stem cells" plus Muscular Dystrophy on Google or Google News would have made the reporters much more informed on the history and benefits of non-embryonic stem cells in treating these diseases.

[Notwithstanding]

Click the image:


EMBRYONIC STEM CELL research = HUMAN CLONING!

[<1/1,000,000th%]

Adult stem cells enjoy another success.

[Tuscaloosa Goldfinch]

Thanks for the clarification -- I guess I just scanned that part (bad habit I have) .
So thankful your granddaughter is doing well!

[WhiteGuy]

Jerry's Dogs?

[fortunecookie]

Yes! Exactly. But then people might think...

[Dr. Scarpetta]

bump