Rare Cancers: Charting a Faster Route to Treatment
Welcome. To the forum, live streamed worldwide, from the leadership studio, at the Harvard th Chan School of Public Health I'm, Dean, Michelle Williams, the. Forum is a collaboration, between the Harvard Chan School and independent. News media each, program, features, a panel, of experts, addressing. Some of today's most pressing, public health issues the. Forum is one way the school advances, the frontiers, of Public Health and makes. Scientific, insights, accessible. To policymakers, and the public, I hope. You find this program engaging, and informative, thank. You for joining us. Welcome. My name is Natasha Luda and I'm the health policy editor, at The Economist, I'm also today's moderator, it's, great to be here on. My. Panel today starting. From my immediate right our Lory, glimmer who's, the president, and CEO of the Dana Farber Cancer Institute. Next, to her is Jill O'Donnell, Tommy who's, the CEO and, the director, of scientific affairs at the Cancer Research Institute. And on. The end is Greg, Simon who's the president, of the Biden cancer initiative and. The former executive director of, the White House counts, a moonshot task force and. Of course joining, us remotely hello, Rahzel is, Rahzel. Kozak, who's the senior deputy director. Of UCSD. Moores. Cancer Center, welcome everyone. So. This forum, is supported, by the economist, group and we're streaming live on, the website of the forum Facebook, and YouTube the, program will include a brief Q&A and you can, email questions, to us at the, forum at HS. PhD, harvard.edu. You. Can also participate in a live chat that's happening on the forum right now. So. Today's, event was. Inspired by Baroness Tessa Jowell, who passed away last year after battling brain cancer and. Tessa. Served. As a member, of parliament. Between. 1992. And 2015. And she, also initiated, and, managed. The winning bid for the London 2012, Olympics. And Paralympics and. At. Harvard, in 2016. She was the men demential. Senior. Leadership fellow, at. The chance school and she was also a board member at the Economist, group and. She. Was also had an extraordinary human, being and to, whom I cannot do justice to in a few short. Words but. Speaking. From a British perspective I can tell you that she was very widely and wired for, both her kindness and her determination, and, The. Economist's very much wanted to support this forum in honor of Tessa because, we. Knew this issue became, very important, for her, she. Spent the final months of her life, campaigning.
Through Improved cancer, treatment and global, cooperation to save lives and we're, delighted that her daughter, just, Mills is in the audience today. So. To, get just, briefly a sense, of how. Tessa championed. The need for this new approach to cancer, I'd like to for. Us to take a look at a speech she delivered, at the House of Lords and, here. She describes an effort called the eliminate cancer initiative or ECI, and she, also talks about GBM, which she refers to blastoma. Cancer which she had and this, clip is shown, with, the permission of parliament. So if we could cue the clip please that would be fabulous, no. One, nation can, solve. The problem, of GBM. On. Its own, it. Is an opportunity, that, Bhagat, belongs, to, the world. ECI. Claims. To do three, main, things. The. First link. Patients. And doctors across. The world through. A Trinity, a clinical. Trial. Network. Secondly. Speed. Up the, use of active. Tar trials, and. Thirdly. Build. A global. Database. To. Improve, research. And, patient. Care. Usually. Drug. Drug. Trials. Tested, tested only. One. Drug. At a time. They. Take years, and cost. A fortune, to deliver. They. Speed, up the process, and, save. A lot, of. Uncertain. Save a lot, of money. When. We, can see these. Approaches. To the delivery of cancer. Transformed. Speaking. To, Tessa's. Daughter yesterday, it. Struck me that at the heart of what at, I was concerned, with was how. To create, a universally. Excellent, care for rich. And poor rural. Or urban whatever, color or race. So. Let's turn to the forum I'm going, to just, introduce a couple of brief slides if. You could cue the first slide what. We're here to talk about is the treatment, of less common, and rare. Cancers. This this, slide is really just to show you it's, an illustrative, it's. An English. It's. A it's a. Diagram. Showing English, cancer. Treatments. And progress in there and really, we only need to take home message I want you to get, from this slide is that we've made lots of progress in, some cancers and there, are some cancers for which we have made very, little progress and. If. I, mean this slide here only shows pancreatic, cancer, but I think the point is is there are lots of cancers down at the bottom many many cancers, many, of which are rare and less common where, we really haven't made much. Progress at, all and the outcomes, are the same they were at. 20, years ago and. The. Challenge is really working. With small patient populations. And late diagnosis. The, second slide please again just for the broad. Context. Here is the monthly country. Cost of cancer drugs which. Has increased tremendously so. The question before us today is what. Can we do to make sure that, there are more effective, treatments, for rare cancers, and to make sure that they're accessible to. Patients who need, them, you. Can let go of the slides now thank you very much, so. With, that backdrop I'd, like to just start our. Conversation. With, Laurie actually, and I. Want you to give me a little bit about your perspective of, rare cancer research. So. Let. Me start by saying that, it's, an honor to be here. Tessa. Jowell was a remarkable, woman and I'm. So glad that we're that we're doing this. We. Have made a lot of progress in, cancer, over. The last decade and a half there.
Are Patients. Who. Had, end-stage. Metastatic. Melanoma. Which, is a skin cancer who. Are still. Alive today 10, years later because, of immunotherapy. There. Are patients, who, have had deadly, diseases, like, lung, cancer, who are still alive today because, of cancer genomics, which is targeted therapeutics. But as Natasha said. We. Are not there yet we are really at the end of, the beginning we're. At the next generation, of. Immunotherapies. And cancer. Genomics, and that's. Particularly, true for. The, intransigent. Cancer so as Natasha said we have done really well with, some cancers, breast cancer, prostate cancer Hodgkin's, lymphoma acute. Lymphocytic leukemia and, children done very well with those cancers, but there are some cancers that are completely. Intransigent. -. Therapeutics. And that includes glioblastoma, and. Pancreatic. Cancer, and ovarian cancer. And acute myelogenous leukemia for. Example, and then. There are the very rare cancers, when, I say very rare I mean less. Than 6 people per 100,000. People and interestingly. Enough we've, made quite a lot of progress on some of those cancers just in the last year in fact. The American Society of, cancer, oncologists. Chose. As their major advance, for 2018. The, treatment, of rare cancers. 5, very. Rare cancers, now, have therapies, that, they didn't have two, years ago that. Includes. Anaplastic. Thyroid cancer. Which. Now can be treated, it. Includes, a rare, tumor. Of your, joint called giant, cell tumor, it includes, a subset, of uterine, cancer called. Serous, uterine, cancer and mid. Gut neuroendocrine. Cancer and, finally a rare sarcoma, called desmoid, sarcoma, and these. Are diseases that are extremely, rare but now each, have, therapies. So, I do I am optimistic. But, we need to. Continue. To focus. Intensively. On those, cancers, that have proven to be difficult whether. They meet, the definition of very, rare cancers, or, if they simply. Argument. Sirs that we have been unsuccessful in, treating and, glioblastoma. Is, certainly. One of them and there are a number of new approaches, to glioblastoma I'm happy, to say actually that faculty. Members, researchers. At dana-farber just. Published, paper, in, nature. Showing. The first vaccine. Against, glioblastoma. It, does arouse, the immune system, there was no clinical, effect it is a very small early phase 1 clinical trial, but at least we have a. Vaccine. For. Glioblastoma. That. Does at least seem to activate, the immune system it's. Going to be the, the answer is going to be, combination. Therapy, we're, going to have to mix, chemotherapy. Radiation, therapy surgery. Immunotherapy. Cancer. Genomics, those are targeted, drugs that, identify. An attack, and disable, genetic, mutations, just. Like hiv/aids what, turned hiv/aids, from a lethal disease into, a manageable, disease so, that a 20 year old who. Becomes infected with HIV, has, virtually a normal lifespan it. Was the realization by basic, scientists, that you have to attack this very dangerous, virus in multiple, places at the same time and that. Is the future of. Cancer. Treatment. Is combining. Different, drugs together thank. You. So. And, we hear a lot about immunotherapy. At the moment so I wonder if you can tell me too because that's something I know the Cancer Research Institute. Focuses on tell me a little bit more about the promise and also the challenges, of immunotherapy please, yes, so I think immunotherapy. Has become something. That the average person out there has heard about and learned about and we've heard some remarkable. Results as Laurie has already mentioned at least in certain cancers and it's almost miraculous, when it does work and. These are intractable, cancers, you know metastatic. Melon. Eight years ago had no treatment at all and now, at, least 20, to 30 percent with one type of him you know that you know just giving one type of immunotherapy, that seems to be able to live and have durable responses, and I think this is what the immune system we now have proof.
Of Principle, that in the right patients, under the right circumstances, you. Can activate, your immune system harness, its power to. Deliver durable. Responses, so that's the. High point and this is happening in a variety of cancers and we have I think over the last eight years there's, been six, different checkpoint, blockades, that have been approved there's been two cars era peas that's been approved and oncolytic virus, has been approved by the FDA for treatment but again it's limited, those checkpoints are all to the same two targets pd-1 or PDL one but. I think this is showing the hope that we. Know, one can now question, whether your immune system can have a role in treating and controlling cancer, I think the challenge, is it's still only a minority of patients, that are getting immunotherapy. That have these miraculous, responses. And in some cancer types what are call kind of cold tumors, the. Immune system at least the immunotherapies. We have right now don't, seem to be working very well so with that we have the. Ability. That can we reverse. This can we get non-responders, to become responders, and I, totally, agree with what Laurie said I think, everyone in the medical community would agree it's going to come down to combinations. I think immunotherapy, has the potential to treat all cancers, but we have to learn how to use it appropriately and, there are a number of variety, of different types of immunotherapies. That's a big word that encompasses, a lot of different treatments, and I think we have to understand how to use them in combination in combination. With targeted therapy in combination. With what are the traditional, standards of care with chemotherapy and, radiation and I think the challenges, is how do we understand, what combination. Can, be used for which patient, and I think that's where it all comes down to research, it comes down to basic biological, research. Of understanding, the mechanisms, and pathways of the immune system and its interaction with cancer, and also, it weakens it requires, the, correlative science, that accompanies, early, phase and late phase clinical trials we need to understand, what is happening at, the tumor site when, patients get one, immunotherapy. And why they don't respond so I think to learn as much as you can from every patient in a clinical trial is one of the ways that isn't, essential, for our research, to give us the inform. Us what is the next priority, combination. And have a basic, scientific, and mechanistic, understanding of, why combining, two things would make sense for a specific patient and I think the future is probably in personalized, immunotherapy. Rahzel. You're doing some particularly, interesting, work you're heading, the the Center for personalized, cancer therapy at, UC, San Diego health and you're also co-chairing.
Dart, Which is the first federally. Funded immunotherapy. Trial. Devoted. To rare cancers. Can. You tell me a little bit about what these efforts are aiming to do I. Guess. Happy to do so and. It's. A pleasure to talk about this, so. The, Center for personalized, cancer therapy. The, main theme is that, each tumor is both complex, and. Different. And so. This is the extension, of rare cancers, if everybody, has. Their. Own portfolio. Of biologic. Changes, and what we try to do is to, look at each, patient. As an individual, understand. Them at the genomic level and, the immune level, with, powerful. New tools that we have now and then, to, give them combination. Therapy, but not just combinations. But customized. Combinations. That. Are suited, to the, biologic. Milieu, in every, patient, so, that's really what we're excited about and. I. Think that this is a, model. For. Rare tumors, but it also makes. Common. Tumors enter I, guess you could say rare tumors, if, you look at each, cancer. And each patient, as an individual. And now. The other, question is then how do you scale, that. Because. Rare tumors, it's really hard to do, clinical. Trials there's, a few hundred, err tumors, and the activation, energy, of each, of those trials, is significant. And that's why we started, dart, which. Is being done under slog, and partnership, with the National Cancer Institute and. Is. Really a National immunotherapy. Trial, giving. To checkpoint. Inhibitors. And. It. Is open at over. 850. Sites has, accrued, almost, 600. Patients, and in, one basket trial. We have numerous, cohorts. Almost 40 cohorts, of patients with. Different rare, tumors, so that we don't have to do a trial. In each disease, and, take the time to, activate, each of, those trials, but, we can do one, trial, open, it across, the country and accrue. In all, of those patients. Thank. You result that's absolutely fascinating um so. Gregor. I'm going to turn, to you and. One. Of the key challenges is getting cancer, treatment, on, to the market, faster. To help people with cancer and, what, are we doing right at the moment and where do we need to improve, thank. You and thank you for having me this is a really important discussion what. We're doing right is, that we have an FDA that's, done a record-breaking. Number of approvals, in the last few years over, the last ten, years really, but. Also designated. A lot of new drugs as breakthrough, drugs, and. As rapid. Approval, drugs because, where. You. See the results, in these trials very quickly if they're positive. And. So we are able to move things to the market with a relatively, few number of patients, per drug what. We're doing wrong is what we've always done wrong.
We, Haven't collaborated, we haven't shared data we, still don't have standards, in cancer, Lori. And I were at a meeting of the top several, cancer, centers in the country, and I, asked the question to the directors, how many of you would trust the pathology, report from. Of the other institutions. In the room and. One of the directors, says I don't trust them from my institution. And. That's because, we don't have standards so the Biden cancer initiative is working. With our advisory committee the FDA the National, Institute, for Standards and, Technology, to. Develop, assays to, measure, patient's, responses, to immunotherapy. Drugs because. Right now every company. Has its own yardstick. And. When the FDA compared. Them they were only 15, percent congruent. One. To the other this is unacceptable. Immunotherapy, gives. Patients autoimmune. Disease that's, intended to kill cancer not to give them diabetes, plus cancer, and. If you don't know if the patient has the proclivity to respond, or if, you don't know how well they're responding, then you're operating in the dark and we should be way past that the. Other thing is. Science. Is hard enough but. Human culture makes it that much harder when. We don't share results quickly, when we don't share failures, when. We have too few pediatric. Trials, and too many adult, trials, we. Are starving, the pediatric, community of the knowledge they need to, help children, because. Companies don't want a pediatric, drug if they don't have an adult indication. To pair with it and, we, have too many trials, with every company trying to get its own toy, because. They don't want to play together, this. Is unacceptable. And it's, bad for patients when. You have 1400. Or more trials, to, develop, drugs that are similar to drugs that are already on the market just. The control, arm is using thousands, of patients and causing. Them immense, cost. Lost. Time at work their caregivers, lost time we've. Got to do a better job of working together, because we are all in this together I had. Chronic, lymphocytic leukemia. 20. Years ago I might not be here even, ten years ago I might not be here but, because of people working together in CLL. I was successfully, treated two and a half years ago that's. The kind of story I wish we could have in, more cancers, and, it's not just about the science, it's about our culture. A. Little bit you. Know when I look, at, the. Future within. Let's say the next three to five years I would, like to see a time in which. A patient, comes, in the clinic is. Diagnosed. With cancer a biopsy. Is taken and. We. Can then predict. What. The response, of that patient, will be to. What drug, and that's. Because. We, are amassing, quantities. Of data sort of five different buckets, of data we. Have the. Pathology, which. Can now be digitized. Thanks, to machine learning we, have the, imaging. The radiology. What, that patient's cancer looks like that also can. Be digitized. By. Taking. Advantage of, machine learning artificial intelligence. We. Finally, now have genomics. So we can sequence at dana-farber we, offer every, patient, the opportunity, to have their tumor sequence, so we can figure out what the genetic mutation, is that they have that's, the third bucket the fourth bucket, is now, immuno. Profile, that that, Jill alluded, to where we can figure out what. Does the patient's immune system looks like, why does mrs. Jones respond, to an immunotherapy and, mrs. Smith doesn't when they have the same cancer well, there must be something different in their immune system we, have to figure out what that is and that's going to require a lot of immuno, sequencing. Which is done that's the fourth bucket and of course the fifth bucket is the, patient, record. And the outcome, we. Need to know which, patients, respond to what therapies and if we collect those five massive, buckets. Of data and. Analyze. Them, using. Machine learning we. Should within. The next I hope, three to five years be able to say to a patient here. Are the four, drugs, that you need to be on for your cancer there's no point putting you on this drug because you're not going to respond to what we know that or. We don't want to put you on this drug because you're going to have unexpected, unacceptable. Toxicity. From it you're gonna have type. 1 diabetes, or. Colitis. Severe colitis. Because. We don't patients, don't have time to waste right there's a limit to how many clinical, trials you can do when they take a long time so. You know my dream is that we will have the ability to have, a patient walk in and within. A few days say to him or her we, have your program we have your treatment, program we're, pretty sure you're going to respond to this combination, of drugs maybe.
Chemo. Plus. Immunotherapy. Plus, a targeted. Therapeutic, of cancer genomics it, could be anything but, we'll be able to do that for the patient saving, them precious time precious. Precious time, gosh. So many questions, arise, from all of that I guess one, question is, that great if I'm a patient, at the dana-farber Center, I'm going to have all that that work done and you hold all that data but. I mean more broadly when we think about you. Know any, individual. Cancer, patient, how, are they going to get access to that kind of analysis, now, in the UK we're. Going to be doing genetic analysis, for some some cancer, and that's great and there'll be a National Health Service, but maybe. We, could talk a little bit more broadly about how this. Information is actually going to end up serving, all patients. Not just your lucky ones, well. We're part of a consortium actually. Dana. Farber is with about 15 other cancer, centers and. That consortium shares. All, its genomic. Data right now eventually, when we have more immuno profile data will share the immuno profile data as well and that's. Very valuable because let's say you have, a. Rare, genetic mutation and, there. Is a drug, that is out, there that's, in clinical trials for example for, this rare, mutation, but Dana, Farber only, has four. Patients, who have that mutation but, Memorial sloan-kettering might have six patients and. You. Know MD, Anderson might have another four well, we can now go on to that site and say let's. Do it together let's do the trial together this this will give us enough patients, so that we can really understand, whether this drug is going to work. People who have this specific genetic mutation does. That no one else want to jump in on that topic. I mean, I think that just in in I think when it comes to immunology, we're, a little bit behind the curve in terms of like the genetic, analysis. Where you have genes and you if you and again I even with the genetic, analysis, I think it matching, a gene that you identified, a function, is still requiring, a lot of research so I mean I think we have that and I think the immune system is a little is is even more complicated than just a gene, so, there's, an awful lot of research that needs to be on me before we can understand. Even what, an immune profile really means so I think that, comes back to the fact of research across the whole bit you know from basic to.
Correlative, Science, on patient, samples, but I do think that it's. Important, for these early clinical trials, and this is as an as a heading, a not-for-profit I think it's a role a not-for-profit can, play in terms of bridging. The gap between academia, and industry finding, a way to do, multi center phase, one phase two trials not a multi center phase three trial but of multi centers you know the initial. Investigative. Thing that we're trying to get a signal and do, it very much like the basket trial that was referred to before but on a platform trial, we can have small cohorts, of ten or fifteen twenty patients where you can learn an awful lot from twenty patients when it comes from the immunological perspective. And I, think this. As you said there's a big. Initiation. Level you have to get over to start a trial so having these trials that, you can bring in a patient 20 patient cohort here 20 patient cohort here that's testing different combinations, you do deep science, on it and at, this point I think we want big signals we're not looking for little minor signals, where you want step changes, so within 20 patients you, can see if there's going to be it what may be a significant. Clinical response you match that to the correlative science, of understanding what's happening at the tumor microenvironment what's, happening to the immune system there and you can expand that cohort, if you don't get that signal close that cohort down but the trial stays open and you can try a different combination so, I think this is efficient, use of money it's efficient, use of time because, time is of the essence at least in the Fiat immunotherapy. The, field is changing so, rapidly there's. So much discovery, if you, take two or three years to get a 20 patient trial up and going which happens sometimes with single side investigator, initiated trials. The thing that you were asking, becomes obsolete by that time because the field has moved beyond you and now you've kind of wasted, that energy, in the time so I think doing, multicenter, bringing. People together to do it rapidly so, that we can get answers as quickly as possible because the science is moving fast and we want to make sure that we're not wasting patience, we're, not wasting money we're learning what we can and that we can help patients to, benefit. From this as quickly as possible. Couple. Of thoughts I'm. From a little town in Arkansas and, even in my little town you. Can find an investment, advisor but. If you want to find a cancer doctor you have to drive travel, an hour to Memphis. We, are this large country, and our. Health solutions, are not equitably, distributed, at all so. In response to our call for commitments. To, change, the way we do things the National minority health, Quality forum put, together a heat map of cancer. And cancer, disparities in, cancer outcomes in the, United States and guess, what people. Die from treatable, cancers, more in minority, communities poor communities, in the, south in areas, near mining, smelters, these. Are predictable. Problems, that have solutions. And we haven't dealt with those yet, we need to put you know the easy solutions. Out there first, one. Of which is everybody. Needs insurance because, the the main determinant, of who, survives, cancer, is who has insurance, number. One number, two we've, got to take care of our children so, at the BIDIN cancer initiative one of our board members from, a venture capital firm did a survey, of pediatric. Oncologist, about the best way to increase. Pediatric. Trials, and to. A person they said please no more public-private. Partnerships, they, haven't worked so. Our board member resigned from our board her, company, is seed funding a company, to. Do pediatric, trials. Apart. From adult trials, by, licensing. In assets, from pharma that they have a safety, profile, but, they've stopped working, on so. That we can go direct, to the problem, and even. Though we germinated, the idea we have no financial interest no governance, role it, was just the right thing to do our, board members company, was willing to do it and some. Of the best pediatric oncologists. Are working with her these, are solutions that that are real. But. You, know when, when. We know that we can't cure people, of curable.
Diseases Because, they don't come into the system we have to fix that first and as, we get to the more complicated, cancers. And more complicated, diseases we. Have to remember that clinical, trials shouldn't be a situation where you have to go to them we. Need to let people do trials where they are and that means that sinners, like Dana Farber and sloan-kettering which are starting to do this are helping. Put those protocols out to other places where people are treated in the community because, that keeps a lot of doctors, from, recommending patients. For trials if they think it's. Trial and error it's why they call it a trial and they. It would be incredibly, burdensome, and costly for them to commute, from. Somewhere, in Mississippi, to, New York or Boston. What. You said Greg it's really important. You. Know at a center like Dana Farber about. 20, to 25 percent of our patients are, eligible for clinical, trials and get put on clinical trials in. The. Community, it's, 3% of patients that have the opportunity to, be on clinical trials and that's not fair any patient. Whose cancer cannot be treated with currently, available therapeutics. Deserves, to, be offered a chance to be on a clinical trial and this is why we've. Established several. Satellites, throughout, New England and, we. Are, keeping. Patients, where they should be which is local, most, patients can be treated locally and they want to be in their home and I think that's right as long. As they're getting the same quality, of care that they would get if they came to, a tertiary, Cancer, Center it's. Not easy to do this I have to tell you it's not easy to do this because setting, up clinical trials is very complicated, it's, taking us time we have. Had some successes, but, it is absolutely, critical to. Eliminate. The disparities, in access, to, top-notch, cancer. Care. So, some missus yes, go ahead with all I'm sorry I, want to address that because I think our. Experience with dart is very pertinent to that and. With. The help. Of the co-operative, group swab and National Cancer Institute, as I mentioned, this, trial opened, quickly, at more, than 850.
Sites Across, the country and. When we started, the trial which is immunotherapy. For rare tumors, the big concern, was. That it would not accrue that was what everybody was worried about and actually, just the opposite, happened, it improved so, quickly that. We were overwhelmed. By the. Requests, to get on the child so, who are you, you would have addressed really, an unmet, need to. Have a clinical. Trial that, is a good, trial and attractive, to patients, in this case immunotherapy. Locally. Where, the patient, lives so. That they don't have to travel across the country I totally agree with you I think it's doable we've shown that we could do it with dart and this. Is a platform trial. So. Other. Trials, should be able to do the same thing for rare cancers. That's. Great some interesting, thoughts and all of that just, on the subject of insurance, and, the need for it can I just put a word in for universal, healthcare as well from my British perspective. Just. Make sure that's on the agenda. So. One of the things that, Tesla. Thought was very important, in rare cancers, was international, collaboration, and, maybe I could talk to you a little bit about this Greg, and increasing. The pool of patients, to work with how important. Is this how urgent, is this we've, seen that these, very adaptive. Widespread. Trials could be helpful we've talked about sharing of data let's talk a bit about international work, so, we, had a summit in Washington on, September, 21st, last year and we. Had 450. Local. Workshops, all. Over the United States in every state and in. Kenya Nigeria, and Canada, when. We got the photos, from the workshops. In Kenya and Nigeria a, group, of local. Women were doing a dance and. When you looked at the picture what you realized was they were demonstrating. Breast, self-examination. In. The dance with. Young girls and older women and. I was so blown away, that. That was that was what they had chosen to do on that day which is a wonderful, thing to do because breast cancer that. Is treatable here is still way too deadly, in in Africa in particular when. We were in the White House. Vice President Biden, executed. Over a dozen memoranda. Of understanding with, other countries for. The sharing, of proteomic, and genomic, data here. In the United States with the genomic data Commons and, the programs. At the NCI. Because. Cancer. Of course knows no borders and knows no parties. And. We, found that every country we talked to was more than willing to start sharing their data in fact, some of them more willing than some of our Cancer Centers so. That's it's a it's a constant, challenge at, least now people say they want to share 5, to 10 years ago that was not the answer I mean that's a really interesting point I mean sometimes you do find that individual, Institute's, are not gonna mention any names but that an individual, searches do want to hold on to their data they feel like they need to because, they have to publish and things like that are you saying you feel there's been something, of a kind of social shift or there's something, different happening I think it's now become in politic, to say you don't want to share and. Now the next step is okay how are we going to share and what's in it for me okay so how are we going to show them what is it infer that, so. We. Have we, have at the Biden cancer initiative a whole, working group on data sharing and the first thing is it's, going to be patient driven every.
Disruption, Of every industry in America is consumer. Driven patient. Driven Marriot. Didn't create Airbnb. Right. Taxi. Companies, didn't create uber, they, created, a situation where, consumers. Could demand a different kind of environment or different kind of service so, we, are accenting, the patient's, right of access to their medical records which is already the law we. Are creating, incentives for people to share their research data by, giving them credit when they publish and, by, reminding them that when you share your dataset, you're going to see a thousand, data sets that come back to you the, problem has always been I've, spent, 20 years building, up this database what if somebody else figures it out before I do I, understand. That but none of these arguments can compare, to the health of a patient that is delayed, because, of the system so. Basically what you're saying is if, the data is portable enough I get, my records and then I give them to whoever I, think, is going to help progressive council yes for instance here at the broad the. Count me in initiative. As. Well that. They count me an initiative is a great, example get. People that make sure they know they can get their records give them a place where they can share them so, we can learn from each other and good. Things will happen so that's people listening, to that should all who, you know may be who patients, should try and look up this counts count me an initiative absolutely, and you. Have a right, to access your records your. Record keeper may not know that so, you have to be adamant, about it but, just as you have the right to your financial, records, imagine. If your bank or investment, advisor said no no yeah I can't give you those you won't understand, them you, would have a fit you should have a fit about your health records well, count me in is, I think a really good beginning, and. It was it, was started, by Nick waggly, who's a breast oncologist at, dana-farber. He. Thought well let me I need more samples from patients with with breast cancer and so he did crowdsourcing. And was able to get about 50,000, samples, and that. Inspired, the creation of count me in which he is directing. For. Many cancers so patients, can as you, said they, can. Demand. Their records and they. Send in a sample of saliva or of mouth scrub. Or whatever so, that we can do the genomics, now that's, a good beginning but, the actual quality, of the electronic, medical records, they're not electronic, they're pdfs we have a ways to go to be, able to make those medical, records interpretable. And we. Just have the genomics, so you. Know we need, we need other buckets of data as we talked about earlier but. At. Least it's a way that patients, can say yeah I want to know what I have and I want to know what my genomics, are because I live in a rural area and I don't, have access to that that's. Right you know George Church said a really interesting thing as he often does. And. It's, it's a it's a great example of, thinking you know what would Google do Google turns everything upside down, but. So George shirt said you know we spend way too much time talking about the cost of genomic testing we, should pay people to, get genomic, testing, so, let's say you tested, a million, children at birth to. Find the one or two with the rare cancer, that you can deal with if you catch it quickly and you have a genetic defect that we have some, way of dealing with the. Money you save, from. Treating somebody early, in cancer, you, then can distribute, to all the other people who got tested, as their. Reward, for being tested, we, shouldn't be saying well we can't test people because it's too expensive and, we won't know what the benefits, are we know what the benefits, are we. Need to turn it around and say you know what if, you agree to get tested, if we end up saving the system money we're going to give you some money for your being willing, to do it in the last few minutes I want to get on to that really if, he subject, for. Any discussion, about cancer, therapies and that's on on drug costs, and I wonder if maybe some, of you could offer, a few thoughts on. Out-of-pocket. Costs, and also this. Proposal, as, well that. PPM's. Return. Money to patients. For.
Their Prescriptions, I wonder, if anyone wants to leap in and have. Some thoughts I made. A career of leaping. We. Talk way too much about price and not enough about patient, copay there. Should be zero out-of-pocket. Costs for cancer patients zero. 75. Percent of first treatments fail, somebody. Still has to pay for that neither. The pharmaceutical. Industry, nor the insurance, industry, is making their living off of that copay it is, designed, to discourage, you, of other drugs not, cancer, drugs it was designed, to discourage, overuse. Of, Viagra. Overuse, of lipitor, overuse, of chronic, disease drugs now, we have curative, drugs the. Patient, co-pays should be zero, and we, need to reform, the system so. That the pharmaceutical, companies, and the insurance companies. Can work out the, economics, between them and leave the patient out of it when. I was in Sweden and I was giving a talk and I said how much does a cancer, drug cost patients, in Sweden and they, said if it works it's zero. And. His financial toxicity. Real issue then, in the in the US yeah. Some. Cause of most bankruptcies, right, yeah right. So. I I've. Sort of been on both sides of the fence I'm. A basic, scientist, dominant, in academia, but. I also serve, on the board of directors of GlaxoSmithKline. Pharmaceuticals, and I was on the board of directors of bristol-myers Squibb, and. I. Guess, what I would say is there, are transformative. Drugs and there. Are me to drugs. Transformative. Drugs are those that completely, change, the outcome, for. A patient, like. Immunotherapies. Like. The, hepatitis, C drug, which. Saved. Money. It. Was expensive. $80,000. Now, it's $20,000, because the market forces come in and there's competition and the price goes down. But. When, you think of the millions Multi, multi millions of dollars that, that's saved in, terms, of hospitalization. The. Development, of liver cancer and patients, who had how about tired to see that. Drug is worth it okay. But. We can't you know we can't keep on raising the price of drugs so.
As More. And more immunotherapy. Addicts become available which, I hope they will and you're, combining them, with targeted. Therapeutics, you can't keep, on adding those together so we do need, to think, very very seriously. About, drug. Pricing and control and I said that I said, that very openly, look thank you for addressing that I really appreciate, that now. It's, time. To throw, it open to questions and. The. First one goes to Jess Mills who's in, front of us now so would, you like to make some comments Jeff, thank. You so much so. My. Name is I'm Jess MercyMe. Daughter. Of the indescribably. Magnificent, Tessa jail and I can't tell you what and I mean. It's been a incredibly. Special. And emotional, day for me being here with monks so many colleagues, and friends who she adored, and was loved by and returned. So. On the 24th. Of may 20. 2017. Without, any formal warning mum suffered. Two major seizures, and was subsequently diagnosed, with their grade four glioblastoma on, the left temporal lobe of her brain. As. I'm sure many people in this room know nothing can prepare patients, or families with for, a diagnosis, like glioblastoma. My, standard of care that offers no chance of long-term survival, or quality of life and only. 2% of patients making it onto a randomized controlled trial so we were cast off into the very very common, patient journey that. Really initially, was defined by its hopelessness. However. We. Were already a very lucky, end of, an awful spectrum. Spectrum. Because by virtue of the work mum had done in public health her whole life when. The news went up of her diagnosis, we were suddenly inundated, with, offers of help from, some of the leading minds. And your oncology, from around the world all desperate, to do whatever they could do to help and, in. The coming weeks that came that, that crushingly low-ceiling of options was, removed. And a, galaxy.
Of Other options were presented, to us her. Genomic data was sequenced, and then suddenly we were prescribed. Everything from, nutraceuticals. Up to off-label cancer, drugs to repurpose, medications. And. Just. This extraordinary, network of support, and advice and guidance that descended around us which honestly just transformed. The, whole experience, for us. But the thing mom kept saying was what. About the millions of patients that don't have this extraordinary, support, what, did they do. The. Only time I ever saw my mom cry throughout the whole of her illness was I read, it one of her Radio therapies appointments. That year that she was receiving through a National Health the National Health Hospital where, as you all know in in England patients, receive their standard. Of care completely, free of charge. And. She, came out of the appointment, and she was weeping and she was she, said to me I've just sat in that waiting. Room with her 15 other patients, like me and has, had the heartbreaking realization. That the fate of every single one of them has already been written by virtue of their privilege, or lack of it and how that's the goodness determine, them, to go beyond the crushing, limitations, at the standard of care here and in, her words this. Exemplified. The most despicable example. Of inequality. And it. Was really these experiences. That. Meant. In the last four months of her life she. Instigated, what. Was now. Become. Known as a test a gel brain cancer mission which has been brought together and, which. Which happened as a result of the speech she gave in the Lord that you've seen an extra oven and. I'm now leading. As. Part of the executive team on the tests of our brain cancer mission which is brought. Together the leading minds in government, department of health science. Research trials. Training. Patient. Advocacy, organizations. Data scientists. To come together to radically, rethink the. Way that we're approaching intractable. Intractable. Forms of cancer like glioblastoma. As, a way of trying to create an exemplar, across the board for, the way that things can be done for other rare cancers, and in a very short amount of time we have. Made some extraordinary progress which I feel, is indicative of, where the future should and could lie for other rare cancers, too so in three months time we're launching the first-ever, adaptive. Personalized, trial for brain. Cancer in the UK called the tesage our brain matrix, which. Is again, championing, the spirit of collaboration that, she, so passionately, believed in drawing. On 10, different centers of excellence within the UK to deliver the program the. Tesage our fellowship programme which is going to train nine, a year and the leading most, cutting-edge practices, around the world internationally. And. Also a completely, new optimized, model. Of standard of care for patients too, and so. At. This point I. It's. A huge moment of reflection and, it's a huge moment of inspiration to. Hear how. Aligned, we are in in, our visions for the future and, I just asked the question that if patients. Really are at the center and focus of, our. Visions, and the utmost, ambitions. For the future.
Let. Us all work with. Greater, collaboration let. Us never, ever as mum said put this in the too difficult box and, as. Mum said in the final words of her speech let's, all imagine a future where patients, live well with cancer and they don't just die from it thank. You, well, thank you so much Jess they. Were incredibly, heartfelt. Words. And having. Lost my own father to glioblastoma I, know that your, work. With. Tessa Giles charity, would, have been incredibly, valuable to, our family, at. The same time now. We've. Got some questions from. Online which I will take first, and. There's one which I thought was pretty interesting because, it will be a question that, a lot of people are facing right now because we've talked a lot about the future the near future and what we can do but, what about right now and so the question is this um and. The, name has been withheld I have a known mutation, sdh. B that causes inherited, cancers, with, only 250. Known, patients, with this disease could a complete, genome, of, our, chima tissue help, a researcher, find something to target, since we have currently. Half no cure so that's a seems to be a question, well. Absolutely, the, answer to that is yes and I just. Want to re-emphasize, what, Jill said we. Need to invest, in basic science. The. Discoveries. Are going, to take place in basic. Science, laboratories. Mmm. And we. Have inadequate. Government. Funding right now for, basic research. Pharmaceutical. Companies are great at developing the drugs but, we have to know what. Is there to target, and. Frankly. As. We. Said before we're at the tip of the iceberg, in, immunotherapy. We can only treat ten, different tumors and, only. 20% or, 30% of, those patients who have those tumors respond, so. What do we need to do we need to discover, more. Targets. And. Here. We have a lady who, knows what her target, gene is where the mutation is that. Gives us. The opportunity, to do, translational. Research on that genetic mutation, make sure it is functional. It really does account for the disease and then. To. Develop, test. It in preclinical, animal models. You. Know if they're going to help this patient what's, going to help this patient because. You. Need to know first of all whether the genetic mutation, is the one that's driving our disease and. You can you have to test that out in preclinical, animal models and then, you need to test it out in human, cells and once, you get to that point then. You can partner with the private sector because. Honestly the marriage, between. Basic. Research, in academia, and, the private, sector, is a marriage made in heaven for our patients, it's the fastest, way to get a new drug made but I mean if we're honest what, we don't know is the sort of immunological profile. On top of that so it could be that you could get your whole genome sequenced and you could still have you, know missing information it, seemed to be what you were saying is there's layers and layers of information right, there, and and and those layers of information are going to be gathered, in academic. Medical. Centers and, analyzed. In academic, medical centers and we're. Going to come up you, know already there are a dozen, at least, two dozen more, new targets, for immunotherapy. That we've. Discovered several new ones other cancer, centers are discovered, other, new targets, and, you. Know at dana-farber we, actually have a very strong medicinal. Chemistry, group, and so we can actually generate tool comp, against. That and test those compounds. In animals to see whether they. Have the required effect. We, can make an an animal. Model of that genetic mutation. Develop. A drug against, a genetic mutation testing. And the animal, and now. We're at a point where you, know we validated. It right, we've. Dearest, the, the, risk of developing. That drug, and, pharmaceutical. Companies would, be much more likely to say ok we.
Believe, All of these data that have been generated in, these academic, medical centers we trust it and we're. Going to try to make a drug that can go to humans let me just see are there any questions in the audience because I have quite a few online, I'm happy to yes lady down there please. Hi. My name is Kate Arline I work at Shepherd therapeutics, we are focused exclusively on, rare cancer and I, lead the efforts. To do a computational, analysis and the, big challenge that we see number one everything, that you brought up especially the disparities. In care the lack, of sequencing, those are big issues but from our standpoint what, we lack is information, and that's what's locked up in the institutions, and we're, at a point where computationally. We can use massive. Screening, massive processing to look for targets that are overlapped between patients, so I'm wondering what all of you are doing to make that information publicly accessible because, academic, labs traditionally, have done that work but, now smaller companies can do it now we can validate and process that ourselves and start looking at repurposing. In other ways of going after these targets so. Dana. Farber has spun out 30. Companies in, the last decade, most, of which are still alive and kicking, you, know science, has no. No boundaries if you're a scientist, you're gonna collaborate, with. The best person, you can to. Move your science, forward, and, I. Can. I can't speak for other cancer, centers but Dana Farber is very. Very open to. Sharing. Data. So. I'm. Maybe. I'm sorry, we're very tight on time Jesus thank you at the Cancer Research Institute we have funded a new, initiative that's called the CRI Atlas, and it's an open source a data source that, is. Based on the background of TCGA taking. Cancers, but putting in related, to those immunological. Components, and. Analysis of, all these cancers, and making, this open source for any, researcher. To come in we're hoping that this will become my thought of funding this which is a was a big deal for us to fund this was that this could become a repository that. The community, would able to put data in and share, it because we need and this is all curated, data because it's you know it's very easy to say we want to have a database and everybody can put it in the biggest problem is having the data put it in the right way have, it accessible and have it being able to be analyzed, and that's not an inconsequential thing. So I think it's, important, to have these repositories I, think I'm, hoping that from an immune perspective, this would be something that could be shared for the whole community an open, source for everyone Greg Corozal so even if you have anything to me all of the research, funded, through the cancer moonshot, 1.8. Billion dollars, per NCI has to be published in open access journals, as, a start and frankly, all cancer. Research should be. That's. Active genomic data Commons all, the genetic information from, the 10th the Cancer Genome Atlas is on, stories. There for anybody to look at ok, so we have a call for raw sequencing, data please from the front ok. Now it's. Our, job panelists. Should you choose to accept it to wrap up briefly. And. Give us a couple, of key takeaway, messages in the next two minutes so I will start - with with Rahzel actually because I feel very guilty that I have slightly ignored, you do you want to give us a few takeaways. I. Think, the takeaways, are that. Rare. Cancers, may be a model for, all cancers, and that we. Need to look at cancers, as individuals. And. We. Think of them as snowflakes, I'm from Canada so we're familiar with snowflakes, each snowflake is, complicated. Snowflake. Is unique, and we. Do genomics we'll review a mean code files that's what we've seen with cancer, each one, is complicated. Each one is unique and we need to treat them that way so, rare cancers, can be a model for everything. That we do, really, for all cancers. And that would be my takeaway well that's great I'd like us to rehabilitate. The snowflake. Suddenly. Would. You like to well. You. Don't need to have you don't have to be a PhD to know what the cancer research system, and care system should look like so. What I ended. Up telling my mother who was shocked at everything I was working on we're. Trying to create the cancer research care, and care system that most people think we already have, they. Think we share they think we put patients first they, think that everybody's working together and that there are standards and that their knowledge. Is disseminated. Among all doctors, in the country that's, not the case it should be the case we need a cancer, system that. Is worthy of the courage of people like Tessa gel and we, can get there but, it's going to require changing, some things up here as, well as in the lab. So. At the Cancer Research Institute, it's all about research and that's what I would say is the bottom line as, much as I say I think probably.
Less So, at the patient care level but at the research level there was a lot of collaboration that has gone on and it still goes on and I think that people, should realize that and it's going, to take research, from across from basic, laboratory correlative. Science, and clinical research and I think that's the only place the answers come from and I think funding for that needs. To be increased, Laurie. I. Am. Fed. Up with. Cancer. Researchers. And clinicians. Who. Don't put, the, patient, first and. Putting. The patient first to me means. Not. Being worried, about who's publishing, what when, but. Working together, sharing. Everything. We know because no. One, place and no one person can do this alone we, have to do it together you know. I I, I'm. Honored, to be the. CEO of an. Institution, like Dana Farber where, we deliver, both exceptional. Care and. Do. Extraordinary. Research. And. The. Thing I love best about dana-farber is that. Everybody. Buys into that mission, I. Wish. That were true of everybody. Who, did cancer, research because you, know we only go around once and. What. Is the, most important, thing we can accomplish. Why. Are we working so hard why why don't my oncologists. And our researchers, why are they there 18 hours a day, it is not for their own personal glory is because we want, to make. Cancer. Go. Away and I have. I like to say you know Dana Farber can disappear, when cancer disappears, but until then we. Need to keep on working and we must, work, together. Well. With that. I'm. A spring this panel. To a close and I'd like to apologize. To, all the people online, who sent in questions that, we weren't able to get to we, had some really, interesting incredible. Questions, one. For another time I think I'd. Like to encourage. Viewers. To tune in to the next forum one. Is on the spread of hate and racism on. February the 13th and, then. The. School, also has a new, center for global cancer prevention that, will have its inaugural, symposium on, February the 4th in time for World Cancer day and more. Information is available on, the forum site and, with that I'll, say thank you and good. Day to you all.