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While I agree with your first point, I think you're off base on the second. If, as the OP suggests, it would be possible to develop a toolkit such that the con

by luma 7y ago

While I agree with your first point, I think you're off base on the second. If, as the OP suggests, it would be possible to develop a toolkit such that the concerns about time/resources you raise in your first point are moot, then I think it's worth chasing that down. The result isn't saving 3 people but rather making it possible (profitable?) to do the same for anyone with these sorts genetic transcription errors. While you're correct that an individual treatment using this approach can never be tested against thousands of patients to determine safety (as it might only be needed by 3 people), it's either try it or watch thousands of people die.

carlmr 7y ago
>f, as the OP suggests, it would be possible to develop a toolkit such that the concerns about time/resources you raise in your first point are moot, then I think it's worth chasing that down. I do agree that this is something worth following. If it's really provable that this platform solution is universally safe, that's amazing. But that doesn't mean the system is broken. Just that until now, this was probably not the proverbial low-hanging fruit of medicine, even with a million people there are diseases affecting billions. It may still be worth more investing in those disease that affect billions. >it's either try it or watch thousands of people die. How many of these genetic diseases are deadly? A lot of these mutations may be bad but not deadly. If now the treatment is universally approved and kills some people, think about who's going to take the responsibility for it?
- luma 7y ago
  > If now the treatment is universally approved and kills some people, think about who's going to take the responsibility for it? Same as any other medical intervention. A substantial number of people die as the result of routine surgery for problems that themselves weren't life-threatening. Medicine, like engineering, involves tradeoffs and risk assessment, and there is not likely to be a perfect outcome. All we can do is work on the "best" outcome for the most people.
mbreese 7y ago
> The result isn't saving 3 people but rather making it possible (profitable?) to do the same for anyone with these sorts genetic transcription errors. The amount of work that goes into making sure that any single treatment isn't going to have unknown side effects is non-trivial. Just because there is a common toolkit doesn't make this safety testing go away. It automates the design of the safety tests somewhat, but doesn't make them any less important. This means that for every... single... anti-sense oligo that's designed, there needs to be significant testing done to make sure that it does what is expected and doesn't have any adverse side effects. In many cases, it's the first part -- the design of the treatment -- that is rate limiting. The benefit of something like these N-of-1 ASO trials is that you're streamlining the first step in the process. But that doesn't mean you can just skip the other steps. And yes, these steps are expensive.

by luma 7y ago

carlmr 7y ago

>f, as the OP suggests, it would be possible to develop a toolkit such that the concerns about time/resources you raise in your first point are moot, then I think it's worth chasing that down. I do agree that this is something worth following. If it's really provable that this platform solution is universally safe, that's amazing. But that doesn't mean the system is broken. Just that until now, this was probably not the proverbial low-hanging fruit of medicine, even with a million people there are diseases affecting billions. It may still be worth more investing in those disease that affect billions. >it's either try it or watch thousands of people die. How many of these genetic diseases are deadly? A lot of these mutations may be bad but not deadly. If now the treatment is universally approved and kills some people, think about who's going to take the responsibility for it?

luma 7y ago
> If now the treatment is universally approved and kills some people, think about who's going to take the responsibility for it? Same as any other medical intervention. A substantial number of people die as the result of routine surgery for problems that themselves weren't life-threatening. Medicine, like engineering, involves tradeoffs and risk assessment, and there is not likely to be a perfect outcome. All we can do is work on the "best" outcome for the most people.

mbreese 7y ago

> The result isn't saving 3 people but rather making it possible (profitable?) to do the same for anyone with these sorts genetic transcription errors. The amount of work that goes into making sure that any single treatment isn't going to have unknown side effects is non-trivial. Just because there is a common toolkit doesn't make this safety testing go away. It automates the design of the safety tests somewhat, but doesn't make them any less important. This means that for every... single... anti-sense oligo that's designed, there needs to be significant testing done to make sure that it does what is expected and doesn't have any adverse side effects. In many cases, it's the first part -- the design of the treatment -- that is rate limiting. The benefit of something like these N-of-1 ASO trials is that you're streamlining the first step in the process. But that doesn't mean you can just skip the other steps. And yes, these steps are expensive.