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Clinical trials are already well underway, and in other parts of the world, there have been over 400 successful transplants using the system. And presumably thousands of dollars won by bored medical couriers turning to their fellow passengers, patting their coolers, and saying, "Hey, betcha I can show you the freakiest thing you've ever seen ..."
Brian Kimball 18:49What I knew at the time was that that combination of therapy had only been approved about 15 months prior to my needing it. And so I felt fortunate to be in a setting where they had a lot of experience already at that point and using that combination, and that they were getting the outcomes that the trials had shown that they would.
Ken Shulman 22:23Unlike HIF, VEGF turned out to be a very attractive target. And drug makers have become more agile since Kaelin first discovered the oxygen-sensing mechanisms in VHL and HIF. A few years ago, a small Texas biotech company came out with a molecule that can inhibit HIF. That drug has shown great promise in phase two and phase three trials for kidney cancer and von Hippel Lindau disease. In addition to drugs that inhibit HIF, Kaelin's research has also led to drugs that stimulate HIF. Those drugs can help patients recover from heart attack or stroke, or counteract the effects of anemia. It's a very impressive yield for some very impressive research. But it's not over. Kaelin thinks that one day it may be possible to go even further up the chain to correct VHL disease at its source, at the VHL gene. Scientists have already treated some diseases at the genetic level by altering a person's DNA to prevent or treat that disease. But it's a lot trickier with cancer, in order for the genetic treatment to work, close to 100% of the tumor cells have to take up the corrected gene.
Ken Shulman 21:57Yet along with the problems, Armstrong and others also see opportunity. Because changes in the epigenome are by definition reversible, they offer an attractive target for therapies. The target is still a few years off, but we've already drawn much closer to it. A number of drugs, many developed by Dana-Farber researchers, are in clinical trials, primarily drugs for blood cancers, like leukemia and lymphoma. The drugs are mostly small molecules that are agile enough to operate within the cell nucleus, that tiny space where epigenetic mischief and magic happen. 153554b96e
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