Orphan Drugs that is.
Before the 1980's, if you got a rare disease that was serious, you were out of luck. Pharmaceutical companies that run on profit bases simply could not expend the resources to develop medicines if there was no market of a minimum size to buy it. People used to die from Cystic Fibrosis in their teens- when I did Medicine, it was a death sentence.
But that changed because of intervention by legislation. In the USA, in 1983, an Orphan Drug Act got passed. This provided funding for development of these medicines by companies that had found something promising, but couldn't persue it. Cystic Fibrosis children can now live into their fifties. And it's getting even better for them with the latest protein assisting triple drug.
As great as that is, it might not affect you. But there is something that does.
The first disease we did in Pathology was something called Amyloidosis. There's a rare subtype of it that is inherited. And YAY, there is a nice Orphan Drug called Patisiran that works to help those few people. Patisiran is the first-in-class of a new kind of medicine, coded silencer RNA inside a lipid shell. It's fascinating how it works, but that's not the point.
That lipid shell technology?
It's in use in both the Pfizer and Moderna vaccines.
If the Orphan Drug Act had not been passed, literally billions of people wouldn't be getting their vaccine today. The spin off value of funding drug research and advancing the technology is enormous, permitting the development of new vaccines in record time, and salvaging not a few people this time, but a sizeable chunk of the world population. Patisiran's development can be traced all the way back to the 1990's, when the first work on silencing RNA was done in worms. The reason the mRNA vaccines exist is largely because the research has been going on for over 30 years, culminating in the capability to kick out a newly coded vaccine in a shell within weeks.
We're living through history, and this is analogous to a Moon Landing, except that it's going to become really commonplace going forward. Vaccine technology has been revolutionized, and we are going to see enormous strides.
Thanks to the Orphans.
Before the 1980's, if you got a rare disease that was serious, you were out of luck. Pharmaceutical companies that run on profit bases simply could not expend the resources to develop medicines if there was no market of a minimum size to buy it. People used to die from Cystic Fibrosis in their teens- when I did Medicine, it was a death sentence.
But that changed because of intervention by legislation. In the USA, in 1983, an Orphan Drug Act got passed. This provided funding for development of these medicines by companies that had found something promising, but couldn't persue it. Cystic Fibrosis children can now live into their fifties. And it's getting even better for them with the latest protein assisting triple drug.
As great as that is, it might not affect you. But there is something that does.
The first disease we did in Pathology was something called Amyloidosis. There's a rare subtype of it that is inherited. And YAY, there is a nice Orphan Drug called Patisiran that works to help those few people. Patisiran is the first-in-class of a new kind of medicine, coded silencer RNA inside a lipid shell. It's fascinating how it works, but that's not the point.
That lipid shell technology?
It's in use in both the Pfizer and Moderna vaccines.
If the Orphan Drug Act had not been passed, literally billions of people wouldn't be getting their vaccine today. The spin off value of funding drug research and advancing the technology is enormous, permitting the development of new vaccines in record time, and salvaging not a few people this time, but a sizeable chunk of the world population. Patisiran's development can be traced all the way back to the 1990's, when the first work on silencing RNA was done in worms. The reason the mRNA vaccines exist is largely because the research has been going on for over 30 years, culminating in the capability to kick out a newly coded vaccine in a shell within weeks.
We're living through history, and this is analogous to a Moon Landing, except that it's going to become really commonplace going forward. Vaccine technology has been revolutionized, and we are going to see enormous strides.
Thanks to the Orphans.
