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A Company called BlueBirdBio was developing a gene therapy treatment for children with a rare neurological disease - Cerebral Adrenoleukodystrophy. It looks lik

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

raverbashing 2y ago
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
- littlestymaar 2y ago
  Yeah, like with everything in healthcare. Treatments are almost always harmful in some way, what counts is the benefit/risk ratio.
- LarsDu88 2y ago
  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
  - mlyle 2y ago
    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.
    - Sabinus 2y ago
      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.
  - ejstronge 2y ago
    > 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.
- LarsDu88 2y ago
  Side note, did not realize the director of mad max made a movie about this with Nick Nolte and Susan Sarandon in the 90s!
justinclift 2y ago
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.
kragen 2y ago
I guess a 10% risk of malpractice and wrongful death lawsuits is a pretty good justification for that pricetag.
- mlyle 2y ago
  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.
  - kragen 2y ago
    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.
    - zdragnar 2y ago
      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.
  - paulpauper 2y ago
    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.
    - mlyle 2y ago
      > 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...

raverbashing 2y ago

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

littlestymaar 2y ago
Yeah, like with everything in healthcare. Treatments are almost always harmful in some way, what counts is the benefit/risk ratio.
LarsDu88 2y ago
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
- mlyle 2y ago
  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.
  - Sabinus 2y ago
    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.
- ejstronge 2y ago
  > 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.
LarsDu88 2y ago
Side note, did not realize the director of mad max made a movie about this with Nick Nolte and Susan Sarandon in the 90s!

justinclift 2y ago

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.

kragen 2y ago

I guess a 10% risk of malpractice and wrongful death lawsuits is a pretty good justification for that pricetag.

mlyle 2y ago
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.
- kragen 2y ago
  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.
  - zdragnar 2y ago
    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.
- paulpauper 2y ago
  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.
  - mlyle 2y ago
    > 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...