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7/67 Children Receiving Skysona Gene Therapy Develop Blood Cancer
- LarsDu88 2y agoA 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 agoIsn'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 agoYeah, like with everything in healthcare. Treatments are almost always harmful in some way, what counts is the benefit/risk ratio.
- LarsDu88 2y agoI 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 agoOn 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 agoWhich 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 agoSide 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 agoSounds 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 agoI guess a 10% risk of malpractice and wrongful death lawsuits is a pretty good justification for that pricetag.
- mlyle 2y agoThere'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 agoYeah, 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 agoNah, 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 agoYou 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...
- elil17 2y agoDoes this mean the therapy won’t be used anymore, or is Cerebral Adrenoleukodystrophy so bad that the FDA will let people roll the dice?
- kikokikokiko 2y agoIt'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.
- paulpauper 2y agoit's not free. it's $3 million. someone has to pay for it
- epicureanideal 2y agoAlthough hopefully if the government is paying for that it’s helping the underlying technology become cheaper over time.
- myprotegeai 2y agoPharma 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.
- fshbbdssbbgdd 2y agoIf 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.
- 2y ago
- uptownfunk 2y agoCurious why it ends up causing cancer..
- LarsDu88 2y agoLikely off target insertional mutagenesis
- oatmeal_croc 2y agoI 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?
- mlyle 2y agoWe 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).
- LarsDu88 2y agoAnother takeaway, particularly for the HN crowd is that gene therapy both works and still has some major kinks to iron out
- imtringued 2y agoIt is bad news for anyone wanting to use gene therapy for treating any non lethal disease.
- paulpauper 2y agoPeople dying of cancer as a consequence of treatment is typically bad news especially all within a few years of treatment.
- MarcoZavala 2y ago[dead]
- cies 2y ago"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.
- zxexz 2y agoI also prefer not to have to think too hard when reading a headline. "7/67" saved me from thinking "10.4%? What's N?".
- LarsDu88 2y agoYeah but then you get headlines like 10% of children get cancer. How many children? 10 children? A million children? When the sample sizes are small, it's better to just give the fraction IMO.
- quantumwoke 2y agoLot 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.
- docdeek 2y agoIn 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?
- gus_massa 2y agoIt's probably very similar to virus causing cancer that is well known https://en.wikipedia.org/wiki/Infectious_causes_of_cancer 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.
- LarsDu88 2y agoTo insert the gene, you use lentiviral transfection. The gene can wind up inserted in the wrong location of the genome, causing a mutation. Worst of all, natural selection can happen where landing in a region of the genome that promotes growth (i.e. cancer) will give those off target cells a selective advantage. Inside the petri dish, Darwinian natural selection means the most cancery cells are the fittest. There are ways of mitigating this. Like through selecting and screening monoclonal populations, but right now that process is painstaking and expensive. Alternatively (and concurrently), we could invest into gene insertion technologies with lower probability of off-target effects. Just one of the many reasons a treatment like this is expensive. You need to screen clones from every single patient because the treatment is unique to every single patient.
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- hackeraccount 2y agoWhat's worse Cerebral Adrenoleukodystrophy or the hematologic cancer? I mean if you just get cancer on top of the original disease that's obviously bad but if you're trading one problem for another that could potentially be a step in the right direction. I'm not an oncologist but I read The Emperor of All Maladies so I know a lot of blood cancers have high survival rates.