Today we know the molecular cause of 4,000 diseases, but treatments are available for only 250 of them. So what’s taking so long? Geneticist and physician Francis Collins explains why systematic drug discovery is imperative, even for rare and complex diseases, and offers a few solutions — like teaching old drugs new tricks.
Because if you look at this particular slide of u.s. life expectancy, but look what happened in the course of that century. average life expectancy of a child born today, age 79, turns out it’s about 4,000, which is pretty amazing, it’s exciting to see that in terms of what we’ve learned, to take this fundamental information that we’re learning a bridge that would look maybe
Something like this, in reality, trying to go from fundamental knowledge and it’s those shapes that determine whether, in fact, so look at this picture here — a lot of shapes dancing around for you. now what you need to do, if you’re trying to develop that will ultimately provide benefit and will be safe. and when you look at what happens to that pipeline, ultimately,
Maybe you can run a clinical trial with four or five of these, so we have to look at this pipeline the way an engineer would, and that’s the main theme of what i want to say to you this morning. is the successful approval of a drug for cystic fibrosis. cystic fibrosis had its molecular cause discovered in 1989 discovering what the mutation was in a particular gene where
We have, for the first time, the approval by the fda of a drug that precisely targets the defect in cystic fibrosis the bad news is, this drug doesn’t actually treat all cases of cystic fibrosis, and it won’t work for danny, and we’re still waiting but it took 23 years to get this far. that’s too long. well, one way to go faster is to take advantage of technology, and to
Be able to then read out the letters in that dna code, that are our instruction book and the instruction book for all living things, which used to be in the hundreds of millions of dollars, where it is less than 10,000 dollars today to have your genome sequenced, or mine, and we’re headed for the $1,000 genome fairly soon. how does that play out in terms of application to
A disease? and it is the most dramatic form of premature aging. only about one in every four million kids has this disease, let me show you a video of what that does to the cell. the normal cell, if you looked at it under the microscope, would have a nucleus sitting in the middle of the cell, which is nice and round and smooth in its boundaries well again, by knowing
Something about the molecular pathways, one of those many, many compounds that might have been useful well that was exciting, but would it actually work in a real human being? from the time the gene was discovered to the start of a clinical trial, that they are in fact a remarkable group of young people i’m going to invite one of them, sam berns from boston, sam is 15
Years old. his parents, scott berns and leslie gordon, both physicians, are here with us this morning as well. what it’s like being affected with this condition called progeria? but when there is something that i really do want to do that progeria gets in the way of, like marching band and that just shows that progeria isn’t in control of my life. here in the auditorium
And others listening to this? what would you say to them both about research on progeria and that just shows the drive that researchers can have and hopefully progeria can be cured in the near future, he is, by the way, a straight-a+ student in the ninth grade about that particular story, and then try to generalize all over the place for these diseases, as sam says,
It’s such a rare disease, it would be hard for a company to justify spending hundreds of millions of dollars to generate a drug. but it has exactly the right properties, the right shape, wouldn’t it be great if we could do that more systematically? could we, in fact, encourage all the companies that are out there of being effective for the treatments they were tried for?
Now we’re learning about all these new molecular pathways — or whatever word you want to use, for new applications, we have many discussions now between nih and companies and you could expect quite a lot to come from this. there are quite a number of success stories one can point to and you can see from the table there are others as well. so how do we actually make that
A more generalizable effort? national center for advancing translational sciences. it just started last december, and this is one of its goals. how do we know, for instance, whether drugs are safe before we give them to people? we test them on animals. or a heart cell or a kidney cell or a brain cell for any of us. for whether a drug is going to work and whether it’s
Going to be safe? this is something created by the wyss institute in boston, and what they have done here, if we can run the little video, turn them into the kinds of cells that are present in the lung, that allow you to see what happens when you add a compound. all the places where you want to see whether a drug and ultimately, because you can do this for the individual,
Will be you on a chip, what we’re trying to say here is the individualizing of the process of developing drugs there has never been a time where there was more excitement what do we need to capitalize on this? first of all, we need resources. this is research that’s high-risk, sometimes high-cost. and in terms of economic growth. we need to support that. between academia
And government and the private sector and patient organizations, just like the one i’ve been describing here, in terms of the way in which we could go after repurposing new compounds. and third, and maybe most important, we need talent. from many different disciplines to come and join this effort — this is the 21st-century biology that you’ve been waiting for, i think
It’ll be the goal of the poets and the muppets it matters for now. it matters as soon as possible.
Transcribed from video
Francis Collins: We need better drugs — now By TED