Vinay Prasad explicitly believes Trumps right to try is possible with AMT-130 if uniQure is willing to sell their drug at their manufacturing costs. It is not.

As a general principle of political integrity if something like Right to Try has been mentioned on the order of 10.000x - 100,000x more than it’s actually been used, it is a program that’s more political shielding than real functional civil medical legislation. I think the term is “real fake news,” or something like that. The right to try program is kind of like the cities in documentaries on North Korea where there is stuff and there are people, but it’s not a real town and the captive unpaid actors are essentially slaves. In this case it’s more like a thin veneer of kindness wrapped around one of the most horrendous and grotesque American politicians that’s ever existed, but without the essential political weight behind it to actually enable anything remotely useful. The expanded access or compassionate use program remains the predominant way Americans currently access “new drugs” that aren’t available for use, and it will remain that way for some time to come.

This blog wrote about one used extensively under compassionate use. It goes by the name Treosulfan and the drug was eventually approved in 2025. It is used as a pre-operative bone marrow transplant chemotherapy that was developed out of Germany by Medac Gmbh and it is currently sold into the US by Medexus.

Treosulfan went through a painful and arduous journey into the US, enough such that somebody like me, who knows almost nothing about bone marrow transplants, knew that the drug was an obvious for choice use in certain patient demographics for BMT in AML (the elderly, kids, and babies), and knew so for about 2.5 years before it was actually approved. Survival and tolerability were higher among several published trials, but also the sheer difference in the list of side effects prevented by avoiding busulfan were well documented, no less than 7 years before its approval in the US. This is because we learned about the crucial differences in metabolism associated with fatal liver toxicity events from busulfan, but also differences in gonadal toxicity, and medical science had done so far earlier than the drug was ever made accessible. This was not theory that immediately presented with clinical data but it was a medical phenomenon that was understood and I do think if the average American knew about defibrotide and what it’s for, along with the history of treosulfan, that they would be extremely upset at the FDA and Medac for their handling of it.

Vinay in the latest podcast I know of immediately mentioned that it was possible for a drug like AMT-130 to be accessed by these programs and made a caveat that the program only fails due to the sponsors unwillingness to supply it. To which I say people from ages of 2 to 65+ years old in America lived and died without having possibility for a bone marrow transplant for leukemia because treosulfan was not available WHEN RIGHT TO TRY EXISTED ALONGSIDE PRE-ESTABLISHED COMPASSIONATE USE PROGRAMS THAT SUPPLIED TREOSULFAN. How on earth is someone getting AMT-130 for Huntington's when the program wasn’t supporting an IV drip of a medicine for leukemia???

The answer is they can’t, and I’ll chop my dick off and post it to live leaks if a single person in the US gets AMT-130 through Trumps right to try.