3 ms·

I don’t think the author of the linked article understands either. Why is it obvious that myostatin inhibitors would ameliorate muscular dystrophy in mice but n

I don’t think the author of the linked article understands either. Why is it obvious that myostatin inhibitors would ameliorate muscular dystrophy in mice but not humans? Nothing in this essay answers that question. Some people with certain types of muscular dystrophy spend years or even decades at near-normal muscle strength, so if you could return them to their previous muscle mass somehow, presumably it would do some good.

klevertree 5y ago
Sorry, didn't mean that it was obvious scientifically. I meant obvious in terms of pharmaceutical progress. They built a mouse model to mimic muscular dystrophy and cured the mouse model of muscular dystrophy, which is why they felt confident spending their millions on human muscular dystrophy. Unfortunately, it was not a good model, in the same way that most most mouse models are not good models.
klipt 5y ago
> Why is it obvious that myostatin inhibitors would ameliorate muscular dystrophy in mice but not humans? It's not obvious, that's why we have clinical trials. What is obvious is that if your research process tests everything in mice first, and only tests in humans what already works in mice, you will find many treatments that work in mice but not humans. (Any treatment with the opposite character - works in humans but not mice - gets discarded by your process and never tested in humans...)
- rory 5y ago
  > Any treatment with the opposite character - works in humans but not mice - gets discarded by your process and never tested in humans Which actually seems suboptimal in a case like this. We know the risks and side effects of steroids in the human body pretty well at this point, and they're not not too bad. If I had muscular dystrophy I'd probably be willing to give them a shot, even if they didn't work in mice.
- BadCookie 5y ago
  Right. I completely agree that human clinical trials were required, but the essay claims that the results of the trial were “predictable” and “might seem pretty obvious.” There’s a saying that it’s a great time to be a mouse with muscular dystrophy. It does seem like using mice as a model hasn’t worked very well, but I am not sure what the alternative might be (chimps?) or why researchers continue to use mice anyway. Edited to add: It would probably be emotionally/ethically more difficult to use chimps … they do some pretty awful things to the mice, making them run until they collapse repeatedly and then killing them at the end (for example).

klevertree 5y ago

Sorry, didn't mean that it was obvious scientifically. I meant obvious in terms of pharmaceutical progress. They built a mouse model to mimic muscular dystrophy and cured the mouse model of muscular dystrophy, which is why they felt confident spending their millions on human muscular dystrophy. Unfortunately, it was not a good model, in the same way that most most mouse models are not good models.

klipt 5y ago

> Why is it obvious that myostatin inhibitors would ameliorate muscular dystrophy in mice but not humans? It's not obvious, that's why we have clinical trials. What is obvious is that if your research process tests everything in mice first, and only tests in humans what already works in mice, you will find many treatments that work in mice but not humans. (Any treatment with the opposite character - works in humans but not mice - gets discarded by your process and never tested in humans...)

rory 5y ago
> Any treatment with the opposite character - works in humans but not mice - gets discarded by your process and never tested in humans Which actually seems suboptimal in a case like this. We know the risks and side effects of steroids in the human body pretty well at this point, and they're not not too bad. If I had muscular dystrophy I'd probably be willing to give them a shot, even if they didn't work in mice.
BadCookie 5y ago
Right. I completely agree that human clinical trials were required, but the essay claims that the results of the trial were “predictable” and “might seem pretty obvious.” There’s a saying that it’s a great time to be a mouse with muscular dystrophy. It does seem like using mice as a model hasn’t worked very well, but I am not sure what the alternative might be (chimps?) or why researchers continue to use mice anyway. Edited to add: It would probably be emotionally/ethically more difficult to use chimps … they do some pretty awful things to the mice, making them run until they collapse repeatedly and then killing them at the end (for example).