COOL TOOL. See how the TALE protein (rainbow colored) recognizes the target DNA site and wraps around the double helix. When this TALE protein is fused to a nuclease (the scissors), creating a TALEN, the hybrid protein will clip the DNA at the target site. Credit: Jeffry D. Sander, Massachusetts General Hospital
If I made a spelling mistake in this blog, and you were my copy editor, you’d want to fix it quickly. You’d delete the wrong letter and insert the correct one. Well, DNA is a language too, with just four letters in its alphabet; and disease can occur with just one letter out of place if it’s in a vulnerable position (think sickle cell anemia or the premature aging disease, progeria). Wouldn’t it be great for tomorrow’s physicians to be able to do what the copy editor does? That is, if they could fix a genetic mutation quickly and efficiently, without messing up the rest of the text?
Can you believe the average length of time from target discovery to approval of a new drug currently averages about 14 years? That is WAY too long. Even more shocking is that the failure rate exceeds 95 percent, and the cost per successful drug surpasses $2 billion, after adjusting for all of the failures. The National Center for Advancing Translational Sciences was specifically established one year ago to apply innovative scientific approaches to the bottlenecks in the pipeline. An example of game-changing innovation is the NCATS collaboration with the Defense Advanced Research Projects Agency (DARPA) to develop a biochip for testing drug safety. Devices like this and other tissue chips may someday reduce the amount of animal and human clinical trials necessary to determine if a drug works. That could be a huge step toward making drug development faster and cheaper—which is better for all of us.
This week, I was excited to join some of the world’s top experts on technology and health at the 2012 mHealth Summit. It’s a booming field, with a recent Pew survey finding 11% of cell phone users and 19% of smart phone users now have at least one health app on their mobile devices.
Among the hot topics at this year’s Summit was the need for rigorous research to determine which of these apps actually serve to improve health—and which don’t! To learn more, check out this video featuring NIH-supported researcher Charlene Quinn.
Dr. Quinn’s work focuses on mHealth approaches aimed at managing diabetes, but her message is relevant to all of us who’d like to use our smart phones, iPads, and other mobile devices to improve our health.
Lung-on-a-chip. Source: Wyss Institute at Harvard University
Tissue engineering is turning into a very powerful tool to learn about biology. We haven’t quite figured out how to grow full sized replacement organs, but we’re able to cultivate miniature versions on a chip. These organs-on-a-chip are poised to revolutionize and fast-track drug discovery and development.
Already a new lung-on-a-chip, developed by NIH-funded investigators at the Wyss Institute in Boston, MA, is a game changer. This nifty little thumb-sized device offers a new way to model human diseases, and a cheaper and faster way to screen potential drugs.
Currently, molecules that are promising drug candidates are tested in test tubes or Petri dishes, then in animals, and then, if they’re successful, in a series of human clinical trials. It’s a long, costly process that, on average, takes about 14 years from discovery to clinic with a price tag of up to $2 billion.