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Comment by jessriedel | Hacker News Reader

> The small study, which has been funded by ex-Facebook president Sean Parker, will augment T cells – potentially a way to treat cancer – but will be used to see whether Crispr is safe in humans, rather than being used as an actual treatment.

This is misleading, right? Yes, it's true that Stage I trials are designed (and in particular, statistically powered) to test safety rather than effectiveness. But that doesn't mean they are making gene edits with no therapeutic purpose. That would be highly unethical. The gene edits are definitely intended to be an "actual treatment". It's just that the number of subjects is so low there isn't any hope of measuring the effect.

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Typically in Stage 1 trials, they test safety effectiveness by giving doses of varying size, often times below the level of clinical effectiveness. They also give these to much smaller populations, usually fewer than 25 people. The main goal of these trials is to figure out "safety and dosage" -- so they measure side effects at each dosage and try to discern interactions with the body.

They'll be especially careful with the CRISPR drugs due to the disasters that Gene Therapy trials have previously undergone. Most notably, TGN1412 was given at a dosage about 500x smaller than the safe dosage in animals but caused almost immediate cytokine storms that nearly killed all 6 participants in the trial.

> they test safety effectiveness by giving doses of varying size, often times below the level of clinical effectiveness.

OK, good point. But even then there needs to be a reasonable belief that either (1) the likelihood of clinical effectiveness is nonzero, and is still worth trying compared to the smaller chance of bad effects, or (2) the same patient will be able to receive a clinically effective dose in the future based on the safety information gained testing the smaller, clinically ineffective dose on them.

I believe you're right. For a Phase 1, you would need to try the same treatment that you want to use for therapeutic purposes, not a "sham" treatment

I haven't run the numbers, but I guess if more than, say, 17 patients go into full remission, then the results will be significant.

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