4 ms·
Scientists Designed a Drug for Just One Patient. Her Name Is Mila
- empath75 7y agoIt 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?
- sambroner 7y agoThis 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.
- deogeo 7y ago> 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.
- blacksmith_tb 7y agoAgreed, 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/clinical-trials/compassionate-drug-use.html https://www.cancer.org/treatment/treatments-and-side-effects...