Innovation in clinical trials: WINTHER trial

Innovation in clinical trials: WINTHER trial



I was talking about the winter trial which is the first trial run by the run by the wind consortium and it is really a cutting-edge precision medicine trial it was the first ever trial that incorporated not just genomics but also transcriptomics in order to match patients with drugs so I think that the winter trial gave us extremely important information so when when the winter trial was created in about 2012 we had a genomics and really nothing beyond that in the clinic so our first question was is will transcriptomics help us increase the number of patients that can be matched transcriptomic is RNA expression so the genes are turned into RNA and it makes sense that the RNA would be important mediators of what actually happens in the tumor and so the first and maybe most important thing that the winter trial showed us that was that the RNA data was valuable so we do have other findings in addition to that the other finding was that it's not RNA data just from the tumor itself you have to compare the tumor to the normal tissue because the normal tissue has tremendous variability so if you look only at the tumor you're going to get a false impression of the RNA you look have to look at the difference between the tumor and the normal in order for that RNA data to be valuable if you don't do that the RNA data simply isn't going to be valuable so that is another important finding from the trial a third important finding from the trial was that matching patients to drugs is not a yes or no the degree of matching makes a difference so we developed a matching score which looked at the degree of matching and that means that if patients had a higher degree of matches so if they had a lot of alterations let's say they had ten alterations and we matched one of them that would be a 10% match but if they had one alteration and we matched that that's a hundred percent match and so one of the questions is does that make a difference and the answer is absolutely that makes a difference a 10% match resulted in less improvement than a hundred percent match so we now know that it's not just match yes or no it's the degree of matching and we need to optimize the degree of matching in every patient there were several things I think that stood out for me first of all I thought Jennifer dude knows lecture on CRISPR not only was extraordinarily clear and made a very difficult subject simple but it is apparent that the power of this technology that she helped discover may revolutionize several different diseases to be able to take a gene editing tool like CRISPR give one treatment and correct a mistake in patients cell cells and she gave the example of sickle cell anemia it almost boggles the mind but it's conceivable theoretically that this could cure sickle cell anemia with one treatment and do so permanently now of course we need to do the clinical trials and there may be side effects that we're not aware of but this is absolutely transformative for some of the diseases that exist today

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