Dinakar Singh
👤 SpeakerAppearances Over Time
Podcast Appearances
And that really was a pretty treating thing from developing a drug as well.
And so again, not to get too much in the weeds, but right into these things.
And it really seemed like there was at least something to be tried here.
As it happened, there was interesting coincidence, which is that NIH at that time, for the first time ever, the world is very different today, but rare disease was not something people focused on back then.
No one cared.
There had been one rare disease drug really ever, and companies were focused on big things at that time, statin, diabetes drugs, oncology.
No one wanted to work and look at rare disease.
NIH was also funding all these big diseases, but they actually came up with a novel program at that time.
to have the first ever grants awarded for translational research.
And so they actually picked SMA because of this cool, nifty new disease and said, we think this is the best target.
We'd like to have a small program where we can actually go and provide some grants to work not just on basic science, but work on actual drug discovery.
Again, today, this seems completely obvious.
Back then, that was a really novel thing.
Unfortunately, there were a bunch of changes.
The head of NIH stepped down and the head of NINDS, the Neurological Institute, stepped down.
And so you had acting directors in both places.
And I joke to people that when you have acting directors, that means there is no action on stuff.
And so nothing was really happening.
So there's pitiful grant money from NIH for this disease, even though other diseases of similar size and a lot less potential probability success were getting tons of funding.
Because, I mean, there is just a latency bias in things.