I don’t at all see why this is relevant to LessWrong, but for the sake of doing things properly here’s a link to the actual paper.
Note in particular that:
We chose a single-arm study design. First, HGPS, although
genetically uniform, has wide range of clinical severity at any given
age. There is no validated disease severity score, and age matching
would not be an effective way to pair cases vs. controls. Additionally,
our pretrial clinical data on rate of weight gain showed
that, although interpatient rates of weight gain vary widely, intrapatient
rates are extremely consistent after age 2 y (13). Thus, each
child served as his or her own control for the primary outcome
measure, and this consistency lent itself well to a single-arm design.
Finally, given the 100% mortality rate in HGPS, we designed
a trial that would allow all patients to receive potential therapy.
So the results could just be due to the placebo effect. Not that there’s anything wrong with using this format for an exploratory trial, just that it’s only a small amount of evidence. Especially given that they only had 25 participants (a quarter of the worldwide population of sufferers!).
Bonus: the drug may help slow aging in healthy humans as well.
I don’t at all see why this is relevant to LessWrong, but for the sake of doing things properly here’s a link to the actual paper.
Note in particular that:
So the results could just be due to the placebo effect. Not that there’s anything wrong with using this format for an exploratory trial, just that it’s only a small amount of evidence. Especially given that they only had 25 participants (a quarter of the worldwide population of sufferers!).
Where did you get that idea from?