Monday, July 3, 2017

First Speak No Ignorance - Turning a Dying Baby Into A Political Football

Like just about anyone I've read on the topic, in the past half hour, I know next to if not absolutely nothing about nucleoside bypass therapy, the American doctor who wants to try it on a dying baby in Britain, the court case his parents brought to the European Court of Human Rights to be allowed to take their critically ill baby out of the hospital or anything else about it.  I don't know who is right except to say that Donald Trump getting involved is a really bad idea because if we don't know anything, he is certain to, somehow, have achieved knowing even less.  His tweet on the topic is sheer politics, having nothing to do with the baby, his parents or any fact in the case, it is sheer political showmanship which is about as real as his fake TV show which has about as much heart in it as a page of the Neilsen ratings or one of Rasmussen's phony polls

I won't have anymore to say about it except that I quickly scanned this paper, Emerging therapies for mitochondrial disorders, from last June.  Especially this section:

A recent systematic review identified over 1300 reports using a variety of approaches expected to bypass or enhance components of mitochondrial function. However, the vast majority of these reports are open-labelled case series with less than five subjects. Although ∼30 randomized trials have been carried out to date, no treatment has shown a clear cut benefit on a clinically meaningful end-point (for reviews see Pfeffer et al., 2012; Kerr, 2013). It is therefore likely that components of the traditional ‘mitochondrial cocktail’ do not have a major therapeutic impact on most mitochondrial diseases. There is therefore a clear need for the field to ‘think outside the box’ when developing new treatments, harnessing the massive increase in our understanding of mitochondrial disease pathogenesis. After preclinical evaluation in cellular and animal models, new treatments showing promise should be studied in patients using a rigorous approach (Pfeffer et al., 2013). This review focuses on these new developments, with a particular emphasis on mtDNA diseases, which were previously thought to be intractable. Here we critically appraise each approach, and highlight areas where there is likely to be traction in the future. This is timely, because both small and large pharmaceutical companies are starting to see the potential market in developing treatments for these so-far untreatable disorders.

Somehow, that last sentence, especially "both small and large pharmaceutical companies are starting to see the potential market in developing treatments for these so-far untreatable disorders." gives me serious pause about anyone thinking they know subjecting a critically ill baby to one is a good thing or, for that matter, a bad thing.  Call me skeptical but I don't think the people opining on either side know what the're talking about.  Certainly not as much as the various legal officials who have at least had evidence presented to them.  I have no idea what Pope Francis might have seen but, for him, at least, the dying baby isn't a political football.

So, that's a long way to say I don't have any response to your challenge to defend the Pope's position on this because I don't know who's right, though I certainly can see how the child is being used by Trump and others.  While you're rightly slamming Trump for doing that, don't do it, yourself.

1 comment:

  1. I'm guessing this story was on FoxNews this morning; how else would Trump know about it?

    I'm not a fan of experimental medicine. I've seen the results "up close and personal," as TV news used to say. It tends to advance scientific knowledge at the suffering of the individual for whom the experiment fails, I knew a cancer patient who took an experimental drug when all else had failed. It failed, too, and only made her final days fewer and added some misery to them.

    Which is not the basis for excoriating experimentation; we wouldn't have open heart surgeries without it. But is this a situation where the patient will benefit, or merely benefit our knowledge by failure? Back when "artificial hearts" were the brave new experiment (remember those?), my parents attended a lecture by a doctor who pointed out medical experiments on people were what we condemned the Nazis for; he drew a straight line to the artificial heart (which was never going to keep anyone alive long, and we never going to shrink to the size of a real human heart).

    There are many considerations. My sympathies are with the Pope, not with Trump.