Tag Archives: Clinical trial

New gene therapy for heart damage goes to clinical trial

English: Coronary circulation, with coronary a...
English: Coronary circulation, with coronary arteries labeled in red text and other landmarks in blue text. This vector graphics image was originally created with Adobe Illustrator, and modified with Inkscape. 32px|alt=W3C|link=http://validator.w3.org/? The source code of this SVG is valid. Category:Valid SVG (Photo credit: Wikipedia)

Researchers at Saint Louis University have had such promising results from a small (40 patients) earlier study using gene therapy for repairing heart failure damage that they’re now instituting a clinical trial.  The trial will use a genetically targeted regulatory protein replacement therapy to see how well it can repair damage in 200 patients who’ve either had a prior heart attack or have cardiomyopathy or advanced heart failure.

The therapy involves infusing a gene – a type of regulatory protein called SERCA-2a – down the coronary arteries that may help heart cells utilize calcium better, which is similar to an engine getting better gas mileage. This outpatient procedure is performed in a cardiac catheterization lab and aims to increase the level of SERCA-2a, which is reduced in the individual heart muscle cells in patients with heart failure and can thereby change the way those cells function.

Worldwide 52 sites will participate in the trial, 33 in the U.S.  To participate in the trial, please contact the Cardiology Research Office at Saint Louis University at 314-577-8876. Read the original article here.

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Mouse studies say nanoparticle-delivered drugs can be targeted

dick mouse
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Reports are all over the Internet about a recent study using nanoparticles as additions to vaccines that target lymph nodes. The study has found that, at least in mice, these nano-loaded treatments can boost antibody- and immune responses against lethal infections. The Duke University team that did the study says their loaded nanoparticles closely mimic the structure and actions of mast cells—those little guys that naturally help us fight infection. They say the fact that they can load the particles with different combinations of cytokines means they can steer the direction of the immune response.

Sounds very promising indeed. My first thought was, this is in mice. How well does mouse research translate into human treatments? First, I learned that the mouse has 99% of the same genes as we humans. Then, too, scientists already have a huge selection of sophisticated tools  for working with mice. Plus, the mouse’s tiny size makes it affordable for large studies.

Then I learned that lots of research studies conducted with mice have not translated at all well to humans. A global cross-discipline (academia, industry, clinical) group convened last year to discuss the whole mouse-as-model issue and came to some conclusions. The most significant of these, for our purposes here, seems to be that mice studies have been successfully translated mostly to validate drug targets and to determine safe and effective doses of combination treatments in humans. Read the entire (slightly windblown) mouse model conference report here.

Another example of successfully using nanoparticles as targeted carriers is in treating prostate cancer in mice. Tumors

Nanoparticle Characterization
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shrank significantly, and treated mice survived much longer than untreated, and longer, too, than even those treated with the same drug but not delivered with the targeted nano carrier. And in this study they aimed to have the targeted drugs bypass both healthy tissue and the immune system. It’s wonderful that such precision is possible.

But meanwhile, because I regularly research information about the very long time—often decades—it takes for asbestos exposure to show up as deadly disease in human beings, I continue to worry about the long-term effects of manufactured nanoparticles being injected into living creatures. I sincerely hope scientists are planning long-term followup studies of mice treated with nanoparticle-boosted drugs and vaccines. Before we head towards human clinical studies, let’s make sure the mice didn’t get saved to live another day and then die of complications from having nanomaterials delivered directly into their bodies.

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Gene therapy shows big promise for advanced heart failure

DNA vaccine and Gene therapy techniques are si...
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It’s only a small group of patients, but the results are dramatic. A new gene therapy for advanced heart failure looks really promising.

People can live a long time with heart failure if it’s kept under control. But if it gets to where they can’t keep the water from accumulating beyond safe levels in the body’s tissues, patients begin to feel like hell and experience more frequent cardiac events that put them in the hospital.

Researchers conducted a Phase II trial at Mount Sinai School of Medicine with a gene therapy developed there and found it stabilized or improved cardiac function in people with severe heart failure. The patients who were given a high dose of the therapy, called SERCA2a, benefited clinically (which means they felt better or lived longer) and had significantly fewer cardiovascular hospitalizations. The study appears online in the June 27 issue of the American Heart Association journal Circulation.

Simply put, the SERCA2a therapy consists of delivering an inactive virus that carries medication into cardiac cells. It then stimulates the heart cells to produce an enzyme that helps the heart pump more effectively in people with advanced heart failure.

Quality of life is often just as important as longevity. If you can feel okay and not have to go to the hospital every other week or month, it’s a lot easier to live your life more fully. Advanced heart failure is tough—it’s always exciting to see that science continues to find ways to use the tools of nature to help in relatively non-invasive ways.

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Stem-cell therapy for aging eye disease

A normal range of vision. Courtesy NIH Nationa...
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A normal range of vision.

It’s exciting to hear that researchers are thinking seriously about how stem-cell therapy might help older people. A company is now requesting permission from the FDA to start a clinical trial using it to try to help a common problem of aging—age-related macular degeneration (AMD).

The same view with age-related macular degener...
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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.

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Stem cells as proving ground for cancer studies

Stem cell diagram illustrates a human fetus st...
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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.

Scientists have discovered a gene that’s present in many forms of cancer, according to a report on Google news via AFP (an international online news source). In this recent study they’ve been able to use human stem cells as the testing ground to see how this new gene relates to cancer.

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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