BETA What is precision and personalized medicine

You may hear the terms precision medicine and personalized medicine used to mean the same thing. And that's because they do. Both describe a new and more thoughtful approach to healthcare. Instead of relying on a one-size-fits-all treatment, doctors now look at what makes you unique, like your genes, your environment, and your lifestyle, to guide decisions about your care. The goal is simple – to find the treatment that's most likely to work best for you. This Health Tree University lesson discusses precision medicine and how it relates to myeloma. What is precision medicine? So precision medicine is a very hot topic across a variety of areas of medicine. And from my standpoint, there are two big components of precision medicine. One is something we very traditionally think about, and one is something that's a little more abstract but is equally as important, if not more important, at the moment. So precision medicine that we talk about now is finding a specific drug for a specific patient. Understanding that your myeloma may be different from someone else's myeloma, we need to find out what's special about your myeloma to give you specific targeted therapy. Venetoclax is a great example of this. If you have an 1114 translocation but someone else does not, giving you venetoclax is a precise way of attacking your individual myeloma. If you have other factors about your myeloma, if they're driven by other genetic markers, or if there's something else about your myeloma that we understand to target it from a genetic level, that's one type of precision medicine. And ultimately, our hope is to develop this much in the same way that we manage urinary tract infections. When people get urinary tract infections, you pee in a cup, they process it in a lab, They put little disks of all the antibiotics on the agar, and we know in a few days which antibiotic will fight your urinary tract infection, and we give you that antibiotic and you get better. Our hope is to develop precision medicine in much the same way from myeloma, that we can take a sample of blood or bone marrow, put it in a machinery, and it spits out an answer and says, for Jane Doe, for John Smith, this is exactly the right therapy to use based off of their genetics. That's the classical description of precision medicine. But to me, precision medicine has another component, and that's where we think of the individual factors that make up you as a person. So things like steroid dosage, prophylactic medicine, IV hydration, and supportive care. These things are crucial to providing optimal treatment for an individual patient. Steroids are all part of our therapies, but we may decide to give someone a lower dose because they have diabetes or have big side effects. We may take a drug like Revlimid and dose reduce it in an individual because their blood counts may be too low. These small things, which may not seem like much, are actually the cornerstone of what I envision as precision medicine in the current day, where we take all the information we have at our hand. And it may not be specifically genetically driven, but how to use the drugs and how to support you best through it is our way of precisely defining a treatment plan for an individual. Medicine is tailoring therapy to the individual patient and using the most information to best make that decision. And whether that's a combination of the standard tests that we have now plus newer assays potentially incorporating aspects of the immune system, that would be the optimal way, I think, to approach patient care. Precision medicine is really medications or trying to design medicines targeted to specific abnormalities traditionally thought of as genetic abnormalities within cancer cells. The classic example is actually not in myeloma, but in a type of leukemia, chronic myeloid leukemia, which is driven by a particular kinase. And inhibitors of that kinase are highly effective treatment for that type of leukemia. In myeloma, the best example of targeted or precision medicine is our medicines targeted for 11-14 translocated myeloma subtypes where Bcl2 inhibition seems to be pretty effective. Vanetoclax is the drug there that we would commonly use. How is personalized medicine related to precision medicine? Well personalized medicine is just that. You discuss with your physician. You know there's a physician-patient interaction and you adjust your medications based on a patient's needs, depending on the side effects they experience. Or sometimes adjust their medication to their lifestyle. Some people may need to travel a certain amount and an oral medication regimen might be preferred if it's possible and available over an IV or infusion medication approach. So personalized medicine, I mean there's overlap of course because understanding the biology of your patient's condition is what directs our clinical decision making about what's the most appropriate treatment choice. One of the things that we're beginning to understand and we're getting sort of more and more data as years progress and we make progress in our understanding of this disease are like how to adjust treatment approaches to patients that have high risk disease or based on MRD status or minimal residual disease status. So I think one of the best examples of this is in the maintenance setting where there's several randomized studies that have demonstrated to us that using two drug treatment approaches like lenalidomide and carfilzomib or lenalidomide and bortezomib in combination with patients with high risk cytogenetic features leads to better sort of remission duration times. In addition, individuals that are MRD positive or have some residual microscopic myeloma in their bone marrow after initial therapy benefit from the addition of daratumumab to lenalidomide maintenance. This has also been shown with recently reported phase three randomized data. So that's sort of an example of personalized medicine based on response. How does precision medicine work? At its sort of simplest, we obtain cytogenetic profiles and we can then say to someone, you have this feature, we can offer you this drug where you have these high risk cytogenetic features, you require additional maintenance in the maintenance setting. As we move forward, more sophisticated genetic testing that helps us understand through more complex computational models who should get treatment A versus treatment B versus treatment C, that's something that we would work toward and that would be I think truly professional personalized medicine, but I don't think we're there yet. More research needs to be done in that department. I spent most of my life studying genes and mutations in multiple myeloma and initially we were looking for precision medicine where we would tailor the therapy based on the genetics, but I don't think there's a future for that now and that's actually a good thing. The reason is because of the promise of immunotherapy in multiple myeloma, which isn't really targeting the mutations, but it's targeting what it means to be an antibody producing cell. So that's a cell that has BCMA on the surface would be one way, but there's other markers as well. There's so much promise in immunotherapy, which is to a large extent, a very important thing that doesn't care what the mutation is that caused the tumor, that I think that precision therapy is going to take a back burner for a while if not forever. If you found this lesson helpful, consider giving us a like and be sure to watch the other lessons in Health Tree University's Precision Medicine in Multiple Myeloma course. Our mission is to educate patients and their care partners and spread awareness about multiple myeloma. We'd like to thank our doctors, our sponsors, and of course our audience for making this video possible.