Human life expectancy has increased from 48 to 79 in the last 100 years, thanks to us mapping the molecular basis of about 4000 diseases. So why have we only found treatments for about 250 of them?
Francis Collins – an American physician-geneticist noted for his discoveries of disease genes and his leadership of the Human Genome Project – is the director of the National Institutes of Health in Bethesda, Maryland. He wants to re-evaluate drug research to be developed with engineering principles rather than our existing FDA methodology, and believes he has an answer that can unlock the power of linking research and technology to help bring relief to thousands of people suffering throughout the world.
Traditional drug research begins by focusing on generating designer drugs – mixtures of hydrogen, carbon, oxygen, nitrogen, and a few other atoms all cobbled together – in vast quantities, thousands of unique compounds, and taking them from 10,000 drugs discovered, to 250 compounds that show promise, to just 5 or so that make it clinical trials, to the 1 drug that could potentially treat Alzheimer’s or Autism. And this process can easily cost over a billion dollars and 14 years – each!
Collins points to genome sequencing, which has dropped by several orders of magnitude from a hundred million dollars per person down to about $10,000 per sequence, which allows teams to very efficiently study diseases like Hutchinson-Gilford progeria by experimenting with those 10,000 compounds using a computer simulation instead of only getting to try out the few drugs that make it to clinical trials. This drops the timeline from 14 years to get to clinical trials, down to just 4, bringing drugs to afflicted patients so much more quickly that it is revolutionary.
Collins offers an alternative – force Pharma companies to “open the vaults” on their old, failed drug compounds and let them be tried out systematically on the thousands of diseases by using technology and genome sequencing, harnessing the brute force of computing power, as well as morphing cells into the relevant type of organ, allowing simple skin cells to be researched as if they were lung cells.Francis Collins speaks enthusiastically at his latest TED talk about his research at the NIH and pleads for more companies and colleges working together, as well as more funding and talent delivered into the government’s coffers to get to work tackling these diseases. He believes that with the best and brightest dedicating themselves to innovative methods of research rather than the old-fashioned pharmaceutical research methods we have now, we can revolutionize the very nature of medical and pharmaceutical research itself. As he states in his closing message:
“This is research that’s high-risk, sometimes high-cost. The payoff is enormous, both in terms of health and in terms of economic growth.”