A Company called BlueBirdBio was developing a gene therapy treatment for children with a rare neurological disease - Cerebral Adrenoleukodystrophy. It looks like the gene insertion went off target in many cells resulting in blood cancer, with one of the children dying as a result.
A single course of treatment of this cure costs $3 million, making this the second most expensive drug in the United States
There's all kinds of approved treatments that have a high risk of a bad outcome; if you are informed of those risks, you're not going to prevail in such a lawsuit.
Indeed, hoping to be compensated for an ordinary bad outcome through a malpractice lawsuit is one small reason why the US medical system is so screwed up: bad things can happen without it being anyone's fault, but we require to lay blame to get any recompense. Ideally, we'd insure patients against those bad outcomes.
You can if all the risks were not disclosed, especially if others come forward and it becomes a class action lawsuit. If a waiver is all it takes to shield oneself from malpractice lawsuit, then it would not be such a big problem.
Yeah, I didn't mean that the patients or their families should or would win the lawsuits, but the lawsuit potential still seems like a potential cost driver.
Nah, you usually have to sign all kinds of waivers to get access to this sort of thing.
It's customized, and very few treatments are sold for the amount of research and effort put into it, compared to some chemical formula that they can hand off to a factory to mix and stamp into millions of pills.
How do you compensate for death? How do you insure patients against death? Do you realize how cynic your comment is? Even though it is full of facts, these facts are pretty much beside the point. Ruining someones body isn't the same as a casual parking damage. I, for instance, have been a victim of deliberate malpractice as a child, which resulted in 100% blindness. NO money on this planet could actually compensate for what I have to cope with on a daily basis.
People can be harmed by medical procedures when someone does something wrong, but people can also be harmed during medical procedures without anyone doing anything wrong.
Ideally patients would be protected not only if someone made a mistake, but also if they were one of the unlucky people who got a bad outcome from a procedure that is normally beneficial.
> But if someone steals your car, the government won't put $30k in your account for a new one.
I understand your point and get that you didn't say this to argue with what I've said. I just want to emphasize that the situation here is different.
Here, though, we have a situation where people who are significantly harmed medically try and find malpractice even if none occurred, and juries are usually sympathetic. The consequence is medicine has become a much more defensive and adversarial system.
I'm sure that an effort to insure for morbidity from bad outcomes will cause fraud, but it might also save a lot of malpractice litigation costs and reduce the amount of resources spent on defensive medicine, too.
On the other hand, cerebral adrenoleukodystrophy would be expected to kill a significant fraction of those 67 and rob the rest of their cognitive capacity within a few years.
There are times where tolerating treatment-related mortality is the best alternative available.
Which is why these dangerous experimental treatments are used on patients with these classes of terrible diseases first. If it doesn't work or fails horribly it's not much different than what would happen naturally.
> Also the blood cancer has already killed one of the patients
This isn’t a fair interpretation of the death - the treatment of the drug-linked cancer bears a risk of multiple complications. One of these complications led to a patient’s death.
I must admit I'm not a very medically literate person - someone please break this down - is this good news or bad news? Were we expecting more people or lesser people to develop blood cancer?
We didn't expect the rate of oncogenesis from off-target gene insertion. It's bad news.
At the same time, this is a treatment for a terrible, terrible disease; so far the consequences seem smaller than what cerebral adrenoleukodystrophy would deal out on its own.
So it's bad news about something which is probably still a good therapy (but we sure would like a better one).
It's a death sentence, in the sense that you either die from it or you become a vegetable. If I'm not mistaken, the treatment cures the disease in 90% of the cases, so it's definitely worth rolling the dice. You literally have nothing to lose, and the payoff can be infinite. The Kelly criterion on doing or not doing the treatment is all in, all the time.
Pharma love when governments are on the hook for their outlandish prices. It's why there's a revolving door between public and private...to smooth deals like these.
If the government wasn’t allowed to hire from private, it would be starved of talent. That would be a great scheme to hobble any agency you don’t like.
In spite of this horrible outcome, can this tell researchers anything about what causes the cancer? As in, if they know what happened (the problem with the insertion of the gene) then does that tell us anything about what might cause a cancer, and with this knowledge help prevent it or treat it in others?
It's probably very similar to virus causing cancer that is well known https://en.wikipedia.org/wiki/Infectious_causes_of_cancer It may be helpful to understand why exactly this genetic treatment cused this cancer and fix it for future similar treatments, but not as generic improvement for all cancers.
Lot of negativity in the comments here. The disease is likely to kill you without treatment and the "cancer" is not solid organ cancer like most people are used to thinking of but mostly (5 out of 7 cases) myelodysplastic syndrome (MDS) which is different and curable in some cases, as this paper demonstrates. If it were me, I'd take my chances.
A Company called BlueBirdBio was developing a gene therapy treatment for children with a rare neurological disease - Cerebral Adrenoleukodystrophy. It looks like the gene insertion went off target in many cells resulting in blood cancer, with one of the children dying as a result.
A single course of treatment of this cure costs $3 million, making this the second most expensive drug in the United States
I guess a 10% risk of malpractice and wrongful death lawsuits is a pretty good justification for that pricetag.
There's all kinds of approved treatments that have a high risk of a bad outcome; if you are informed of those risks, you're not going to prevail in such a lawsuit.
Indeed, hoping to be compensated for an ordinary bad outcome through a malpractice lawsuit is one small reason why the US medical system is so screwed up: bad things can happen without it being anyone's fault, but we require to lay blame to get any recompense. Ideally, we'd insure patients against those bad outcomes.
You can if all the risks were not disclosed, especially if others come forward and it becomes a class action lawsuit. If a waiver is all it takes to shield oneself from malpractice lawsuit, then it would not be such a big problem.
> You can if all the risks were not disclosed, especially if others come forward and it becomes a class action lawsuit.
> > ...if you are informed of those risks...
Yeah, I didn't mean that the patients or their families should or would win the lawsuits, but the lawsuit potential still seems like a potential cost driver.
Nah, you usually have to sign all kinds of waivers to get access to this sort of thing.
It's customized, and very few treatments are sold for the amount of research and effort put into it, compared to some chemical formula that they can hand off to a factory to mix and stamp into millions of pills.
How do you compensate for death? How do you insure patients against death? Do you realize how cynic your comment is? Even though it is full of facts, these facts are pretty much beside the point. Ruining someones body isn't the same as a casual parking damage. I, for instance, have been a victim of deliberate malpractice as a child, which resulted in 100% blindness. NO money on this planet could actually compensate for what I have to cope with on a daily basis.
People can be harmed by medical procedures when someone does something wrong, but people can also be harmed during medical procedures without anyone doing anything wrong.
Ideally patients would be protected not only if someone made a mistake, but also if they were one of the unlucky people who got a bad outcome from a procedure that is normally beneficial.
His point is not about compensating for loss.
It's the fact that options laid out on the table with unknowns can be preferable to people than no options, or fewer options.
The same dichotomy exists for crime vs agreements:
- You'll be compensated if a party to an agreement breaks a clause or costs you money (say they fail to provide $30k of goods to your business)
- But if someone steals your car, the government won't put $30k in your account for a new one.
The same loss occurred. The second case was totally unwarranted, yet the individual outcome is worse.
Why? Because doing it any other way would make things worse. People would lie about stolen goods, conduct insurance fraud, etc.
Unfortunately in some situations, things "which are worse" are a different category and don't have an answer with government intervention.
> But if someone steals your car, the government won't put $30k in your account for a new one.
I understand your point and get that you didn't say this to argue with what I've said. I just want to emphasize that the situation here is different.
Here, though, we have a situation where people who are significantly harmed medically try and find malpractice even if none occurred, and juries are usually sympathetic. The consequence is medicine has become a much more defensive and adversarial system.
I'm sure that an effort to insure for morbidity from bad outcomes will cause fraud, but it might also save a lot of malpractice litigation costs and reduce the amount of resources spent on defensive medicine, too.
Even some biologics have cancer as a potential side effect. You get to sign all kinds of fun waivers before you get a prescription for them.
When the alternative is degenerative illness like psoriatic arthritis, sometimes you just have to roll the dice.
Sounds like they'd be (hopefully) working on better targeting of the gene insertion for the next iteration of this.
Without the off target gene insertion, it sounds like it'd be a winner.
Isn't that the Lorenzo's Oil disease?
So gente treatment for very serious and fatal disease causes very serious issues in 1/10 of patients?
As opposed to, you know, the effects of the original disease?
I think I would go with this treatment regardless
I should clarify... 7/67 SO FAR
Presumably the other children may manifest cancer down the line. Off target insertion was random
Also the blood cancer has already killed one of the patients
On the other hand, cerebral adrenoleukodystrophy would be expected to kill a significant fraction of those 67 and rob the rest of their cognitive capacity within a few years.
There are times where tolerating treatment-related mortality is the best alternative available.
Which is why these dangerous experimental treatments are used on patients with these classes of terrible diseases first. If it doesn't work or fails horribly it's not much different than what would happen naturally.
> Also the blood cancer has already killed one of the patients
This isn’t a fair interpretation of the death - the treatment of the drug-linked cancer bears a risk of multiple complications. One of these complications led to a patient’s death.
Side note, did not realize the director of mad max made a movie about this with Nick Nolte and Susan Sarandon in the 90s!
Yeah, like with everything in healthcare. Treatments are almost always harmful in some way, what counts is the benefit/risk ratio.
I must admit I'm not a very medically literate person - someone please break this down - is this good news or bad news? Were we expecting more people or lesser people to develop blood cancer?
We didn't expect the rate of oncogenesis from off-target gene insertion. It's bad news.
At the same time, this is a treatment for a terrible, terrible disease; so far the consequences seem smaller than what cerebral adrenoleukodystrophy would deal out on its own.
So it's bad news about something which is probably still a good therapy (but we sure would like a better one).
Another takeaway, particularly for the HN crowd is that gene therapy both works and still has some major kinks to iron out
People dying of cancer as a consequence of treatment is typically bad news especially all within a few years of treatment.
It is bad news for anyone wanting to use gene therapy for treating any non lethal disease.
Does this mean the therapy won’t be used anymore, or is Cerebral Adrenoleukodystrophy so bad that the FDA will let people roll the dice?
It's a death sentence, in the sense that you either die from it or you become a vegetable. If I'm not mistaken, the treatment cures the disease in 90% of the cases, so it's definitely worth rolling the dice. You literally have nothing to lose, and the payoff can be infinite. The Kelly criterion on doing or not doing the treatment is all in, all the time.
it's not free. it's $3 million. someone has to pay for it
Although hopefully if the government is paying for that it’s helping the underlying technology become cheaper over time.
Pharma love when governments are on the hook for their outlandish prices. It's why there's a revolving door between public and private...to smooth deals like these.
The prices are less outlandish in single payer healthcare systems as the government's monopsony can force prices down
You seem to be under the impression that the government works on your behalf to save you money.
It does. The Pharmaceutical Benefits Scheme here in Australia is a lever that the government pulls to negotiate lower drug prices
https://www.pc.gov.au/inquiries/completed/pharmaceutical-pri...
It also pays an additional $5B p.a. in drug subsidies.
If the government wasn’t allowed to hire from private, it would be starved of talent. That would be a great scheme to hobble any agency you don’t like.
It isn't as binary as you're making it. You can hire people who don't have conflicts of interest, or the perception of conflicts of interests.
In spite of this horrible outcome, can this tell researchers anything about what causes the cancer? As in, if they know what happened (the problem with the insertion of the gene) then does that tell us anything about what might cause a cancer, and with this knowledge help prevent it or treat it in others?
It's probably very similar to virus causing cancer that is well known https://en.wikipedia.org/wiki/Infectious_causes_of_cancer It may be helpful to understand why exactly this genetic treatment cused this cancer and fix it for future similar treatments, but not as generic improvement for all cancers.
Curious why it ends up causing cancer..
Likely off target insertional mutagenesis
Lot of negativity in the comments here. The disease is likely to kill you without treatment and the "cancer" is not solid organ cancer like most people are used to thinking of but mostly (5 out of 7 cases) myelodysplastic syndrome (MDS) which is different and curable in some cases, as this paper demonstrates. If it were me, I'd take my chances.
"7/67" this is why general public likes percentages. Or "one in X" type of ratios.
Next up: (e^-1)/3*3 children who got the COVID shot...
There's this saying in UX: don't make me think.
I also prefer not to have to think too hard when reading a headline. "7/67" saved me from thinking "10.4%? What's N?".
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