
Scientists Designed a Drug for Just One Patient. Her Name Is Mila - Bostonian
https://www.nytimes.com/2019/10/09/health/mila-makovec-drug.html
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empath75
It seems better to me to think of this as a method for creating custom drugs
rather than to think of it as the creation of a single custom drug for one
patient.

Maybe the first one is extraordinarily expensive and risky, but what happens
after they do this for a dozen or a hundred or a thousand people?

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sambroner
This seems like great practice, despite being extraordinarily fraught with
difficult decisions. If we attempt a custom solution to hundreds or thousands
of these rapidly dying patients, we may learn a tremendous amount about each
disease we attempt to treat. If we don't make strides on the diseases, we will
at least learn to quickly iterate on drug manufacturing.

The quoted 7000 rare diseases won't be solved with traditional investment in
each one. It seems reasonable that we attempt alternative techniques and learn
from each attempt.

I worry about two things: That we will use custom drugs when traditional
techniques would be more effective, and that money that would otherwise be
effectively spent on traditional research is spent on the custom drug effort.

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deogeo
> Even in rapidly progressing, fatal illnesses, precipitating severe
> complications or death is not acceptable

Why not? If it was my life on the line, I'd take the risk.

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blacksmith_tb
Agreed, and in fact the FDA seemingly has already moved past that, they do
support compassionate use[1].

1: [https://www.cancer.org/treatment/treatments-and-side-
effects...](https://www.cancer.org/treatment/treatments-and-side-
effects/clinical-trials/compassionate-drug-use.html)

