So, I just wanted to talk about our exploration of HHT. I’m going to call the drug HHT as a new treatment for leptomeningeal disease. Briefly, leptomeningeal disease is a rare complication of systemic cancer, like breast cancer, you know, goes to the liver, the lungs, or the bone, and sometimes it goes to the substance of the brain. Like if your brain’s a potato, it’s kind of the inside of the potato...
So, I just wanted to talk about our exploration of HHT. I’m going to call the drug HHT as a new treatment for leptomeningeal disease. Briefly, leptomeningeal disease is a rare complication of systemic cancer, like breast cancer, you know, goes to the liver, the lungs, or the bone, and sometimes it goes to the substance of the brain. Like if your brain’s a potato, it’s kind of the inside of the potato. Sometimes it goes to the covering, which is kind of the brown part. And the tumor cells can float around the spinal fluid in your spine and on your brain. People live about two or four months. So it’s a huge medical problem. It’s also true that there’s something about the brain and the spinal fluid. It’s a sanctuary site. So it’s like protective once the tumor cells are. So there’s a reason they like going there, which we don’t understand yet. And then there’s a reason they thrive. It’s a bit heartbreaking in clinic because often everybody’s cancer is fine everywhere else, but then they get a headache and this pops up, so it’s kind of heartbreaking. It’s like, I went through everything, my PET scans are normal, why is it up there now? Anyway, a barrier to discovering new treatments is growing tumor cells specifically from the CSF. So we call these patient-derived CSF CTCs, which doesn’t matter, but we’re the first group in the world to be able to grow these. We only did it in three patients with melanoma. And it doesn’t sound like a big deal, but nobody’s done it before. And once you have cells growing in a dish, you can then do the usual drug discovery things on them. So in this case, we’ve used a library of FDA-approved drugs because we want to get it in the clinic. We don’t care if we find some weird drug that isn’t approved in patients, and we’ve discovered that the cells were susceptible to this HHT, Homo-Harrington, which is a complex drug. So we’re just working out the details of this now. When we use the drug in mice, it cures like half the mice, which is good. But we really need to finish evaluating how useful it is and work out the exact mechanism, which is a little too much information for this discussion, I think. But you can tag the drug and see what it interacts with. And once you see what it interacts with, you can make sure that it’s just one target and not a whole bunch of ones. So it’s kind of exciting, but we recognized that we needed to have cells grow from the CSF so that we could make them accessible to industry and academia so that we could discover new treatments. And this is just one example of one target we found. So hopefully it’s going to help somebody. So thanks to everybody who does this. So this is done with Derek Duckett, who’s the head of drug discovery at Moffitt, which is a common theme in the work we do is we collaborate with a whole bunch of people. If you just want to get better treatments for people, present a trial here that shows something that works.
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