Saturday, 30 November 2013

Of Mice And Men

There's been another article posted on the Internet by another research team presenting another breakthrough in research around treatments for ALS. This article, along with a host of others, presents a picture of incredible developments with fantastic possibilities, raising the hopes of thousands of terminally ill ALS patients, the hope for a treatment, a cure, a tomorrow and a future. The only problem is that so many of the research discoveries are not the hoped for miracle, they are not the hoped for cure.

Today's article is another in an ongoing bombardment of articles announcing a major advance in ALS treatments using a mouse model. In this particular study, researchers "succeeded for the first time in stopping the degeneration and loss of motor neurons and the progression of symptoms of the disease". This is indeed very good news for mice with ALS, however the key line in the article for those of us humans with this dreadful disease is "While this result was achieved with a model mouse, it is thought that a similar molecular mechanism underlies sporadic ALS in human patients..."

Notice that "it is thought" part. The truth is that researchers can cure lots of things in mice that they cannot cure in people. Diabetes springs to mind as one of the examples. They've been able to cure diabetes in mice for some time now and it is only recently that any sort of progress has been made in replicating the result in human studies. They have'd done it yet; there is still no cure for diabetes in humans. The harsh truth is that they just don't know how to make that leap from mice to men.

However, this article is not a lot of smoke and mirrors, nor is it a simple fund raising exercise. One of the greatest challenges in treating neurological disorders of any sort is having treatments which move across the "brain/blood barrier". We have a system in our brain that stops foreign genetic contaminants from transferring from the blood to the brain. This is a good thing as a lot of bad stuff moves through our bodies and we don't want that stuff in our brains. Watch the movie "The Fly" with Jeff Goldblum; you'll see why.

This particular study successfully introduced genetic material into the blood stream and that material crossed the barrier and impacted the genes that are thought to cause ALS in humans, and mice too. It showed that it is indeed possible to target genetic repair, even in cases of sporadic ALS, using a simple injection of genetic material. It paints the picture of a possible cure by doing something as simple as getting a shot at the doctor's office.

This research does not offer a cure, at least not in humans. What it does is show a pathway for delivery of genetic repairs, something that will benefit a great number of diseases. That's a good thing, for both mice and men.

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