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Buck Goldstein

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Beyond Science Fiction

Posted: 07/01/08 03:32 PM ET

As you head for the beach with a canvas bag full of science fiction, the activities of one of the world's great inventors will top anything you or your favorite author can possibly conjure up. This year's winner of the $500,000 Lemelson-MIT award for invention, Dr. Joe DeSimone, (full disclosure -- the author is a shareholder and advisor to Liquidia, a company founded by DeSimone) has been designing nano-particles in his lab at the University of North Carolina that are 100 times smaller than a red blood cell and as smart as a microprocessor. He has discovered that size and shape really do matter and when these properties can be precision-controlled the sky is the limit. Lately DeSimone has been concentrating on nano-medicine. He's been working on some remarkable projects.

-- By altering the size, shape and modulus of particles substantially smaller than a human cell DeSimone can direct them to a particular cell or group of cells within a particular organ in the body. He can also load the particles with a particular drug therapy by constructing nano-sized compartments within a tiny "ice tray" and filling the compartments with the desired drug or therapy. At MIT on Thursday night, DeSimone showed YouTube-quality videos of his particles approaching and entering targeted cells and then dropping their cargo virtually on demand. This performance created audible gasps from a group of distinguished scientists of all ages that had assembled for the ceremony.

The first frontier for this breakthrough may be oncology where such targeted drug delivery is obvious. If you can kill the "bad cells" without touching the good ones entirely new ways of thinking about cancer therapies are on the table. The work is still in its infancy but good results have already been obtained in prostate and ovarian cancer cells.

-- DeSimone is using these same principles to produce particles that mimic red blood cells. Not only do these particles look and act like red blood but they are "smart" as well. What this means is they can be designed to circulate in targeted parts of the body and not others (their shape and modulus confine them to specified parts of the circulatory system). They can also be designed to circulate for a specified period of time and then be excreted (this is effectuated by the particles changing in shape and flexibility over time). A therapy that can be infused into a particle that will circulate like blood in the body for a given period of time and then be excreted would have profound implications for the delivery of all kinds of drug therapies. Intra-venous transfusions might be replaced by drug infused particle injections. Vaccines that require multiple doses to be effective may be delivered via a smart particle that can achieve the same result. Again, full implementation of the technology has not been achieved but the platform has been created.

-- Even further out, at least from the perspective of a non-scientist like me, DeSimone and his colleagues are working to deliver a therapy called "silencing RNA" or siRNA. The theory behind this approach, which many believe will be foundational for the future of drug therapy, is that certain diseases can be cured even before they manifest themselves by identifying the proteins that cause them and then preventing those proteins from every firing. The trick is achieving "knock down" by altering the functioning of targeted proteins within a designated cell -- no trivial task. Because DeSimone can produce smart but tiny particles and they can be infused with a cargo that may achieve "knock down" one of the big barriers to unlocking the promise of siRNA may be addressed with revolutionary consequences for the way we think about treating disease.

Of course, there are huge hurdles associated with each example, not the least of which being how to produce huge quantities of custom designed particles all with exactly the same characteristics. But even what has been achieved so far provides a sense of what's possible and will provide the basis for scores of other ideas some of them conceived on the Fourth of July in between sessions with a great science fiction novel.


 

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