Chordoma Foundation Five Year Strategy

A lot of concerned effort, but also a lot of focus, uh, and doing things we've never done before as an organization operating in the field that we're operating for. That led us through, um, go the changing planning process over the last, uh, year and a half, and, um, many of you may have seen, uh, we launched our next five year contribution planning. That describes where we aim to be over the next multiple decades, but in particular what we need to do over the next five years, so I'm gonna kind of walk through that, uh, lay the, lay out kind of what the vision is, what we think is possible, and talk about some of the things that we are So first, I think just to summarize some of the things that we've heard, it is remarkable to see that, um, many of the approaches when much of the conventional wisdom that we talked about are all preferred. So, um, we heard about how radiation is playing a larger role, for example, in treating State of Cordona, but, um, this is what that actually looks like in practice. This is Tom Kettering's experience. Uh, over, uh, quite a number of decades, and you can see decade by decade, uh, how the mix evolved, um, such that now in the present, things are much more nuanced. Surgery is being used, uh, much more precisely when appropriate, combinations being used when appropriate, and increasingly, uh, radiotherapy is being used alone. So key advance in Applying these approaches in a more nuanced way. We look at skull base. Um, one of the things that become very clear is that achieving a, uh, a really good surgery, getting as much of the tumor out as possible is really important for long term out of many of us have probably heard. And several decades ago, that was not easy. Majority of patients had what's called subtotal resection, so not not all the tumor was able to be resected. But starting, um, not so long ago, um, right around the time that I was diagnosed about 20 years ago, there was some pretty significant surgical advances that started to enable the throat total resection rate to go up. So in a Increasing fraction of patients are able to remove the entire tumor. Now, maybe you see University of Pittsburgh, which is one of the leading small data centers. Now you see amazingly the majority of patients are able to receive gross oversection. So one of the things that this can tell us, we, we know that, um, uh, gross oversection is has been important, but Now we have long-term following patients been followed for years, um, after being able to continue aggressive sections. What does that actually look like? Well, now after following these patients for ten to fifteen years or more in some cases, so really significant difference in, uh, in regression free survival. So it doesn't necessarily mean overall survival, it's regression free survival, how long it takes for patients, uh, to present back after You can see really substantial difference between the growth oversection and the subtle oversection. And the fact that growth oversection rates are going up, that is really good news for many of us have probably heard about seven or eight years statistics that goes up on the internet. Many of us are probably older, exposed to older per second. This is reason to believe that that is going. Change dramatically as the data continues to mature. A really interesting finding, um, this is presented out research workshops that there's nuance between years low recession and gross lower recession, and essentially business thought that, well, we can get almost all of it out. That's so enough. I think the data is starting to show that. Actually, what really matters, uh, is to get the whole thing out at all possible. Uh, get the outcomes can be a lot better. So, uh, these are just a couple of many advances that are, uh, being brought there on sheep, and examples of how things are really changing for the better. And there are many of us in this room who are living proof that long term good outcomes are. But a couple that really come to mind and it turned out to be as inspirations. Johnny Nelson, right here, was diagnosed a couple years before I was. And I think many of us were diagnosed probably look to people who have come before, um, for inspiration, for maybe some indication of what maybe I could expect. So Johnny has always been that person for eight. Um, you know, uh, we were diagnosed with a similar age, we had similar operations, and now Johnny is more than twenty years, twenty-two or twenty-three years out, and uh, now has a spirit style of note. So, uh, hoping to continue following in Johnny's footsteps. Similarly, um, Mick, uh, Many of you have probably met Mid and his wife, Mid and White read previous conferences. They just recently celebrated their 15th wedding anniversary and, uh, continues to create. So, um, I think there's so many stories like that that are kind of recently common in our community, whereas, um, several decades ago that was just, uh, uh, less the norm that it That being said, um, choromas still do recur, and um, I think many of us in this room have experienced that. Myself included, um, about four years ago. I, uh, after being tumor-free for sixteen years, uh, a little spot on my MRI turned out to be a recurring choroma. And I was always under the impression that dealing with recurrences was a situation where there were not really any great conscience, and that the prognosis was about a recurrence early consciousness. I think that is changing as well, and I think I'm living proof of that. Probably in this crama living, living proof of that. Um, and I think this paper, um, from the Mayo Play illustrates That, um, there are for, uh, increasingly growing share of patients outcomes for long periods of time. So there's an AO series, uh, about half patients, uh, the recurrences were alive at five years. But, uh, importantly, those who are able to have surgery and radiation most recurrence. Now granted nine one was Uh, it's candidate for surgery and radiation may be not appropriate in all cases, but for those who weren't able to have surgery and radiation, median survival factory occurrence was ten years. Pretty incredible considering the numbers that many of us heard, uh, years ago that the average survival diagnosis was total seven years. So clearly there's a lot of progress being made, not only in the nation primary disease, but we So radiation surgery improving significantly both in the primary treatment setting as well as in the recurrent setting. But thankfully, there's also been substantial advances in drug dairy systemic therapies as well. There's a lot of room for improvement still, but there are some notable success stories. I think about Richard Lexon, who I believe is here. Benefited from so Richard, uh, doing great after, um, uh, dealing with, uh, uh, controlling portal. Not without a lot of analysis, but electric history and Payment applied, uh, to your profile and to understand what are the genetic payments of consumer. It doesn't happen in all cases, minority cases, but sometimes that leads to something that I call to consumer that can be exploited with the chromosome last growth is never the payment actually be prevented. Because one example of the growing, uh, possibility or potential of flying flights for supply. So all, what is all that built? That yes, it's bringing to bear the best possible features across all these different modalities, better outcomes as possible, not just like the life or improving quality of life, but only if we're able to access those, only if we're able to find security providers that not again in some parts of the country and rest of the world where, uh, they're just gonna One thing they will know those doctors are sometimes despite the knowledge, despite their expertise, like all three things in the same way, we get different specifications making a well-informed decision. And deciding what prior stay not attacking. And then finally, many of us, there's really significant logistical, financial, practical, emotional, social barriers and we have been union is available. So we see it as kind of And as an organization and as a community overcome these barriers more that Fed bail care, uh, you know, is is able to be accessed by everyone in this community. So to do that, um, I think it's all you are well aware to offer quite a number of resources and services coming forward over time as the That gap bidens these resources and search comes down. Uh, additionally, there's still some really important questions that we can't answer, but yesterday had In the day where we spent three or four hours talking about the unanswered question how we apply plants and answer. So a couple of those here on the screen and my primary element. So if you have an issue of primary, uh, You achieve that those percent is final sequences from negative margin, but they're really different sometimes, sacrificing the caution to have not a few to get those negative margin. Does that same principle apply in the currency? Is it worth going after every last cell, every last visible spec consumer at media expense of conscious? More that X found will make difference along the ground that we don't actually have the exact question in in the education. So more work that needs to be done, um, and in particular for the spine labor. So there's, uh, questions around, uh, that kind of are, uh, In some cases, but, um, or when the many should be done, the dynamic is one of the, uh, vector doses. And then the nature of the end of the fact that these sometimes capacity comes up really far getting problems. Sometimes you can actually cause more problems than consumer itself. So what is the kind of appropriate balance effect? Does it change loss balance that aggressively and does kind of play wax hole for more compounds? Uh, does that improve longer outcomes? Or, uh, my balance are issues better off if you have more Maybe in some cases there's a clear answer. So I would not want to read the project with the, uh, being some sort of information that in all cases, if not all are totally unknown. Very clear answers to companies, but in my business here is a very current. So to answer some of these questions, it requires large numbers of patients, okay, but really very new to each institution, only limited number of patients. So I just compliment that if it's for 200 publications over 12 decades, all federal, 100 patients over So there's some efforts that got underway on leadership of, uh, really, uh, strong collaborators we talked about last night, uh, to pull together studies possible to get to keep up to answer questions that only be answered when you pull down. years and years, uh, so we so much, uh, the ministry of her comes to data from all institutions in this enough better. It's just a matter of the and this is just the study for example sequence that is just answering the whole law question that we have three of And fourteen or fifteen questions that the question is what if we could actually just learn every patient? So much we learn from every patient. It always felt like there's a big opportunity that so much of our so many of our experiences actually don't capture and aren't contributing to, um, learning at this development. Thankfully now the last several years, the health, data platforms, there's these platforms now that are, catch the medical records from dozens or hundreds of medical centers across the country that has millions of these records in the Uh, and AI are making that easier to start online. Some challenges to leverage for Corea, and um, all of those have standardized billing codes for Cornell. So depending upon where the located, where you're treated, you have that might be totally different. And it's not actually trivial on the micro. figure out who are the growth patient position patient data so really kind of elementary things that we need to do as a community to leverage the sort of data resource that becoming a increasing priority growth so these two things together lead us to do some key priorities over the next five years to that enable patients to get better for that One, we've got it through access care by redoubling our investment in application services. There are not our first those now we are coming on board, and the key step in that direction is enhancing that evening, uh, the value of our patient service. Um, it's a lot more than we're gonna be doing is developing the additional content. And then side by side with that, a lot of done to enable traffic here movement. So we don't have to wait yet into the answers on those questions. Starting done on more real time, uh, the end of the learn mapping issue. So you wait doing that. as well as building a registry of our own surgery to avoid everyone very medical records that's going on. That being said, um, I think much of that applies to recording surgery and radiation, but sure, uh, there is a, uh, Growing role at this point always a year to play for demic therapies, and there's a lot of that at the moment the key is to better control the transfer. But it's not over time because more effective drugs started to work, using therapies. Earlier on the disease process, so perhaps this matter combination with certain Or can be an author term ideal actually in usually surgery that is compared to more surgery to a surgery less risky or more issues, or maybe negatively ambitiously cannot be the survey of any issue and enable issues to preserve their quality life not undergo a surgery. So thankfully, um, this lot of data that are happening right now in, uh, the growth, uh, potential therapy, drug therapy over time, the number of new tension drugs that are being developed has increased dramatically over the next day. What does that mean? There's a whole lot of tension drugs that be in high growing maps, and it's not just drugs that are improved that are kind of A lot of investment, a lot of new therapies that we have now higher. And not only is it just greater therapies, but there's entirely new modalities that are coming to the form. So we hear about this sort of chemotherapy. If you want basically over targeted therapy is Uh, strike it on TV caller. You have accounting like a whole zoom of, uh, of different modalities that almost may account. Some of them that are really talking about modalities that we have type that are getting a ton of depth, a ton of tension. But you think the antibody book trones are able to target and deliver highly on that. Um, you can see here the number of new clinical trials over to left this year as, uh, in hydrogen is gone just down left in the front. Uh, same with cell there is doctors lost on that line go down to lots of lobs market. Uh, a new emerging group is called radio line. That's kind of similar in my hydro front. Rather than bringing SEMO therapy to actually bring delivery radio therapy, rather than shooting at it, shooting at the score from the council delivering it to the same condition therapy. Um, you heard out the basis earlier that really high profile successes, uh, really high growth successes We need as a community really incumbent upon us to apply this breakfast to Roma and my way that Roma is better. And, um, I think fortunately, uh, we are now at a point where we are building on a foundation that is kind of cumulated layer by layer over the last few decades that enables us finally at this point apply cell therapies radio apply yet, uh, and by drug monitor gets New texture therapies that we talked about last night that impact relevant deals are year. All of those things require a huge amount of infrastructure, a huge amount of technology. Well, why are we at a turning point now? Why is this the moment that is so different previous moment? Because most of the essential enabled layers that are needed to bring those approaches to come up. They're built because what we've done over the last fifteen, twenty years, we don't need to spend in the fifteen or twenty years building that we've got the most right now. There's a couple things that still need to be done on top of that, but we're now building upon the infrastructure. So it's a totally different situation now that we're in three years ago, five years, we've been years ago. Although All in the team was like to say, um, we invested a lot in the community, a lot as an organization to build out some key enabling resources to enable scientists to understand the biology of the key to test drugs. We have been able to grow into grow our investment research plan to chain metrics migrated to research neighborhood. And it used to be that we were only valid resources to one research in kind of one key area understanding third market. Now, the time we actually build up research portfolios and dismantling those understanding files. We recently started investing in primary coverage as well as Many of you are aware, while for many years we've been reliant on technical researchers and companies supply their groups. Now, I think we actually have to be able to drive research on our own landing center, to be able to offer capabilities to companies and researchers a lot easier for them. And this has been over the last couple years, of course, enabling a lot of three things that are having to help that are coming in here come out toficer and read the format list currently. So our vision, and what is now eminently possible, I think you heard, uh, talk about it in particular. Our vision now is that. We go from having a very efficient, albeit no longer completely empty, but there's still pretty limited toolboxes of drug therapy set to having a largely growing toolbox of therapy that can be applied in a personalized manner, that can be used in combinations, and that can be scrubbed together if the tumor advances through Our work is everything we strong together to achieve long-term good outcomes. And, um, several kind of key colors, uh, where folks needed to realize that competitive. So we've got a huge initiative, we have something really big going forward, which is that my fellow and I really are aware of something that is Not always the case of a lot of cancer, and you've got to do more of the thing that is permanent vulnerability is not the normal issues. So far as Biddel's good can be a lot for, but Bin Del turn in a lot of the can when it comes to this target. It's just not the norm. It's an exceptional opportunity, and for a long time it's been Now we put no longer. Um, because of the damages like we heard about last night, um, because of UDRU and now because of the people that now visit laps, there is a incredible opportunity now finally for a primary drugs over the next five years, and that is much baseballs and capital with sufficient as resources So over the next five years, twenty thirty per sear goal at the end year, we saw us to have, uh, weighted a lot of next year, a lot of uncertainty was in the first crypto next hundred years in house and same time as I mentioned, there's this kind of growing pipeline of, uh, rug that we developed this next Cancer that are even available sooner, um, and couple with that. A lot that we learn about from data class, we think over the next five years as an opportunity, again, if we sufficient resources focus, that we can begin that we play that we're gonna get in of disease over with that to at least ten years. And finally, really, really grateful that, um, you may get more peaceful markets. Firmly is management time and effort how you get one thousand grounds. Realize any of the possibilities expect factory round recovery for this working varies have to make thousands of days and talk more about that as well as you can, uh, this twelve thousand hook you're going to make that file. So what do you see as the optimal ball is something? And for the hopefully screaming minority of issues that do have recurrences, there's better options for recurrence that keep growing lots of effect there. And visions came together to really prolong, uh, So that the hope, um, achieving this is going to require multiple requirement people participating in research Many of you, um, require us supporting one another to require inter access to care to require later to require all of together to operate. And now so many of you just in my experience, this is something that we do together, um, and Uh, I think that if we do that, if we focus on the priorities that identified this plan, we're gonna play resource level programs, we're gonna be in information that five years now that we are staying. That's gonna be a coming to the farm future that is more prior to. So speculative kind of obsolete right now, and I hope that, uh, that comes through, uh, So please let you hear to everyone already look at this on the website. There's an extent on the website. And if you are really interested in local business, the 40 plus page, for diesel version, but I just want to close by thanking all of you for helping at this point. Uh, this is really the result, uh. Layer after layer project after lay down and disappear as all we are standing on top of that round today, uh, the event is carrying. Thanks to all of you, uh, there's a breakdown, um, uh, twenty minutes. So we're gonna come back here and Thanks a lot for you.