So, in my most recent blog entry, I remarked upon the unlikelihood that gene-therapy will benefit Cystic Fibrosis sufferers anytime soon. Instead of building these gene-therapy-inspired castles in the sky, I suggest that the strategy we want to utilize here is the one which provides the greatest results per resources expended. That will not be attempted DNA-rewrites, and it will not be so-called “gene therapy”– at least not for a century or ten.
ON THE OTHER HAND… We do not want to go to the other extreme, and treat only the symptoms.
Granted, in some situations in life, all one can do is treat the symptoms. But symptom-alleviation is the shallowest care-approach of them all. It is merely hacking away at the branches of evil, when what we need to do is to rip the problem out by the roots. And if we can’t rip out the roots, then we must chop down the tree. And if we can’t chop down the tree, then we must lop off the main branches. In other words, the better our solution, the less tree will be left. It is only as a last– and partially defeated– resort that we merely shred-away the twigs and leaves of the symptoms.
When struggling against a complex problem, the best way to achieve victory is to thoroughly analyze the causes and conditions of the problem, with a view to discovering the problem’s weakest points so that those points may be attacked with overwhelming force.
The weakest points are those places where we are most likely to break through, and so it is here we must essay our greatest strengths. A thief does not break into a house through the walls. He pushes through a weak door, or disables a weak lock, or maybe even climbs-in through a window. Stealing Nature’s secret is the same… It is a question of strategic leverage.
We need to delve far enough into the deeper causes of a problem to have the fullest and most lasting impact we can. AND YET– we must not go so deep that we lose our way, or attempt something beyond the powers or control of our limited know-how.
When it comes to Cystic Fibrosis, most of the suffering and death comes from the impact of the disease on the lungs. The next most dangerous area is that of improper digestion, due to the greatly lessened ability of the body to deliver necessary digestive enzymes from the pancreas to the guts.
Fortunately, though it is certainly no ideal solution or day in the park for the sufferer, the digestion problem can be greatly alleviated by the taking of a retinue of enzyme-supplements.
As for the lungs…
The problem in CF always goes back to those damned malfunctioning Epithelial Cells (with their badly constructed membranes). In those suffering from CF, the Epithelial Cells (which normally produce a nice thin, workable mucus) instead produce an overly dehydrated mucus. This lack of hydration thickens the mucus, which tends to clog up the works. (The same thing is going on in the digestive tract, where the mucus of the Epithelial Cells there are clogging up the enzyme-delivery system).
If we can’t fix the underlying problem of the faulty cell-construction causing the mucus problem, then the next deepest level to attack –if we have the proper, impactful means– seems to me to be this dehydrated mucus.
I suggest that work be started immediately on developing a drug to rehydrate or re-liquify this mucus. For whatever reasons, it seems little work is being done along these lines.
Otherwise, the smart people fighting the war against Cystic Fibrosis have come up with some very clever methods for the mucus problem…
One of their oldest ideas is simply to vigorously pat the CF-sufferer’s back in different positions so that the mucus is coughed up.
Another approach is to infuse the lungs with dNase, a chemical which breaks-down much of the debris that gets caught-up in the overly thick mucus covering the Epithelial Cells. Once this debris is broken down, the hair-like (or perhaps better, “whip-like”) cilia of the Epithelial Cells can then move more freely, and can thus better carry-out their job of whipping unwanted germs and debris (caught in the mucus) up to where they can be coughed out or otherwise ejected from the pulmonary system.
Finally, lung transplants have proven to be a new lease on life for those suffering through the final stages of Cystic Fibrosis. Because the transplanted lungs do not contain the DNA for making the bad Epithelial Cells that CF-gene-containing DNA does, the lungs do NOT produce the symptoms of CF even after transplanted into a person with the disease. The recovery of the patient, for as long as the body accepts the lungs, is about as close to a miracle as anyone is likely to see in this world.
Unfortunately, as my source-books say, every transplant is eventually rejected. As seems so often the case in disease, the culprit is the body’s Immune System, which THINKS it’s doing us a favor when it rejects what it sees as a massive army of intruders in the body (the cells of the new lungs).
Perhaps one day we can learn to camouflage real or artificial lungs transplanted to the recipient so that the body’s Immune System does not see the new organ as a threat. Maybe, even in the near future, we can take samples from the patient’s body that his or her Immune System already recognizes and tolerates as “self,” and then coat the lungs, or a sack around the lungs, with this substance.
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Source 1: Understanding Cystic Fibrosis by Karen Hopkin
Source 2: Cystic Fibrosis: Everything You Need To Know by Wayne Kepron