The Risks of Drugs Trials

This is tragic:

The death of 11 babies born to women who were given sildenafil during a drug trial has led to the termination of the experiment – and an anxious wait for other mothers involved. Sildenafil is sold by Pfizer as Viagra, but the pills used in the study were not ones produced by the pharmaceutical giant.

The trial was designed to test whether the medication could help boost babies’ growth in the womb.

The research was carried out at 10 hospitals across the Netherlands and involved women whose placentas had been underperforming.

Viagra, which dilates the blood vessels, is used for erectile dysfunction in menand is prescribed for people with high blood pressure. The hope, backed up by experimental research on rats, had been that the drug would encourage a better flow of blood through the placenta, promoting the growth of the child.

The women taking part in the trial all had unborn babies whose growth had been severely limited in the womb. The children’s prognosis, given a lack of available therapy, was regarded as poor as a result.

The trial was terminated last week when an independent committee overseeing the research discovered that more babies than expected were being born with lung problems.

The problem with any new medicine or treatment is, no matter how much research you do on rats, monkeys, and in simulations eventually you have to try it out on humans. Until then, you’re never sure what the results will be and sometimes, as in this case, they’re not good.

Back in 2006 a human drugs trial went catastrophically wrong and turned into a full-scale medical emergency, leaving the victims disfigured for life:

On a hospital ward, patients were writhing and screaming in agony. Their organs were failing, their heads swollen and many were projectile vomiting as their immune systems began to completely shut down. It looked like a scene from a horror film, yet this was the appalling reality for six young men who had been in perfect health until they signed up to take part in a drugs trial a decade ago.

It was, they believed, a chance to make some easy money and do their bit for medical science at the same time.

But what should have been a routine trial in a private clinic at Northwick Park Hospital, North-West London, soon spiralled into one of the most infamous medical emergencies, and became known as the ‘Elephant Man’ drug trial because of its shocking side-effects. Now a BBC documentary is to revisit the dramatic events that resulted in the young men fighting for their lives.

Human drugs trials take place every day, and horror stories like the ones above are rare indeed. But until someone can come up with a robot which can exactly mimic the human body’s reaction to any drug, they’ll remain the price to pay for modern medicines.

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12 thoughts on “The Risks of Drugs Trials

  1. Maybe this is what we should do with the ‘Beatles’ ISIS crowd.
    Or offer free passports to anyone willing to try out whatever medical shit we want on them.
    Actually many of the world’s poorest people would do this for a living.
    I would be utterly unshocked to discover that China’s prison population are heavily involved in drugs trialling without even knowing it.
    In the enlightened west we treasure the sanctity of human life. Officially anyway. Elsewhere – not so much.

  2. These are babies that would have died any way. The lung problems suggest that there were premature delivery.

    I sudpect this was v. Complicated and had little impact on the births.

  3. Tim, I’m guessing you know Peter Risdon (who previously blogged as “Freeborn John”, was a regular at Timmy W’s and I believe some other places you frequent) died of cancer a little while ago. It seems Darius Guppy was still chasing him through the courts as he was in the final stages of his illness which must have been an extra bugger.

    His site lasted a few months after his death but the domain’s expired now. A shame because he’d written a couple of very interesting entries about living with cancer – not so much the emotional stuff, though he did talk about maintaining the right mindset and attitude, but lots about the practicalities of it all, methods of adapting lifestyle, the scientific evidence, how to go about chasing the relevant doctors and so on.

    It was really good content – practical, clear-headed, realistic, honest, but above all, it looked like it had the potential to be very useful if the worst comes to the worst.

    He did sketch out a couple of extra posts that he intended to write, including one on clinical trials – what to expect, how to get on one. He wasn’t expecting any miracles and knew the risks but at his stage he knew a trial would be his only shot of recovery.

    Very sadly he never wrote it, and I feel pretty selfish for my personal disappointment at that.

  4. Tim, I’m guessing you know Peter Risdon (who previously blogged as “Freeborn John”, was a regular at Timmy W’s and I believe some other places you frequent) died of cancer a little while ago.

    Ah no, I didn’t know he died. I know he was the person responsible for breaking the NHS monopoly on the supply of spectacles, and for that he deserves a statue on the fourth plinth at Trafalgar Square.

  5. That Hansard transcript is like a bunch of doddering old scientists sagely warning that humans cannot travel at more than 30mph because they’ll be crushed by the pressures.

    “The Committee cannot accept that after the prescription has been issued optical appliances take on the status of mere consumer durables ”

    Perhaps the Committee would like to throw itself in the Thames?

  6. Well, the most important numbers are missing here:
    Number of live births in each treatment group
    Number of live _and otherwise healthy_ births in each treatment group.

    We can’t even interpret the seemingly impressive difference in lung-related mortality between the groups without those!

    All the newspaper reports seem to be word-for-word identical, so I assume no paper has a science journalist capable of realising the importance of this question and asking it.

  7. All the newspaper reports seem to be word-for-word identical, so I assume no paper has a science journalist capable of realising the importance of this question and asking it.

    I think that’s a pretty solid assumption, yes.

  8. @BiG

    Hope you’re recovering okay! Best wishes.

    One of the curious things about this, so far as I could make out from the media coverage, is that that there were a couple of earlier trials (presumably smaller ones) where this adverse outcome didn’t occur, so it took them by surprise. Presumably they’ll be looking closely to see if anything differed between the ways the trials were conducted.

    In general, once something goes as crappily wrong as this trial has, is the whole idea almost invariably condemned to the dustbin? Or so long as there were strong indications that the drug was actually doing what it was supposed to do in most cases, do researchers think about ways of moving forward that might keep the beneficial effects but reduce the side-effects, for example changing the dose or using a similar but not identical chemical?

  9. Thanks for the wishes MBE. I am feeling normal (except you never feel really normal after a two-week hospital stay). What I have will never truly recover, but equally it shouldn’t threaten me, provided we can keep it from deteriorating. I could be good for decades (in the words of the consultant), or in trouble next month. Keep your fingers crossed for me.

    Three things kill drugs that get high profile devastating adverse effects. The adverse publicity puts potential volunteers off, and the reluctance of ethics committees to allow further trials. Thirdly, sponsors lose interest, because there are always other molecules to look at and you can’t get insurance any more for further trials. Interestingly however, the infamous TeGenero product is back in clinical trials. In Russia.

    You’re quite right that sildenafil has been tried in this indication before.

    It is hard to comment on the results of this trial without knowing the numbers I mentioned above, other than to say, if the drug does increase live births and the trade-off is more sick births, it is still incredibly difficult to assess the benefit-risk ratio. Usually we treat patients that are competent and capable of consent and understanding risk. Fetuses aren’t, and they are the real patients here. The mothers are likely to take a view of the benefit-risk ratio that is highly influenced by circumstances. A childless woman in her 40s on her 6th IVF cycle will think very differently to a 24-year-old already on her fourth child and with plenty of time to try again.

    The question should be “are the sick/dying babies babies who wouldn’t have been born without treatment anyway”, but the whole baby thing throws a big spanner in the works. Even if the answer is positive, I just can’t see it getting past any regulator, so the drug will never get licensed in this indication.

    Far more interesting is the fact that the drug is widely available, and will remain so, in certain other indications. I can all but guarantee there will be off-label use of this drug in this indication by the desperate and there is no way to stop it. It wouldn’t surprise me if the drug’s OTC status (where it has it) is reviewed, but I would stake a lot of money that there will never again be a clinical trial.

  10. What I have will never truly recover, but equally it shouldn’t threaten me, provided we can keep it from deteriorating. I could be good for decades (in the words of the consultant), or in trouble next month.

    German citizenship?

    Joking aside, best of luck fella. I hope you can keep the little bastard down, whatever it is.

  11. @BiG

    Cheers for the informative answer. Hadn’t thought about the implications for getting the trial insured.

    Will keep fingers crossed for you – I also have fairly serious recurring health issues, albeit with some very decent quiescent periods in between. I’m lucky to have been born this side of the “golden age” of drug development, else I’d be long dead. But in my experience, the uncertainty that comes with it – could be in serious trouble next week, but might not flare up for a couple of years – has actually proven far more impactful than the spells of ill health themselves. The symptoms may have been grisly, I don’t like quacks, the novelty value of finding yourself laying in a hospital ward wears off pretty damn quickly – but (and this is survivorship bias speaking here) I’ve always made it through to the other side okay, even if it’s sometimes taken 6 months or a year to get well enough to go back to work. Patience is a learnable virtue and you can adjust your expectations as to how long it takes to get back firing on all cylinders, or even what “on all cylinders” is going to mean in future.

    On the other hand, the uncertainty never really goes away regardless of how good you’re feeling, and it can compel you to bend the whole grand arc of your life to fit around it. Ideas of “financial security” or “career plans” or more general “life goals” can all face a stern re-examination, depending on the probability and severity of possible future health events. In my case I’d describe the likely severity as “manageable” (some people still do die and even the drugs I take have potentially fatal side-effects from long-term use, but I’d be extremely unlucky) and the probability of recurrence over any 5-year period as “very high” (with an average time of 18 months – 2 years between flare-ups, but complete uncertainty as to when, and therefore no guarantees if I scheduled a particularly busy year for myself, that those plans would not be derailed). It sounds like the severity in your case might be rather worse but the probabilities, I hope, rather more favourable.

    It’s true we could all be hit by a bus tomorrow, but we don’t live in paralytic fear of scheduling anything in our diaries for the week after, just in case we’re in post-charabanc-prang recovery. We just assume it’s not going to happen to us, and take reasonable steps to prevent it (“Look both ways, don’t move your feet // Till you look both ways, when you cross the street”). But when we’re faced with events beyond a certain threshold of probability, or which feel more “personal” to us (e.g. because we have now directly experienced our first bout), the nagging doubts start seeping it pretty quickly.

    Best of luck for the recovery. Hopefully you’ve got a lot of support from family/Mrs BiG.

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