Cure Disease Moonshots: Transforming Disease Using Data, Technologies and Personalized Medicine Full

Mike Pellini MD, Chairman of the Board of Directors for Foundation Medicine, sits down with StartUp Health Co-founder and CEO, Unity Stoakes at the 2017 StartUp Health Festival to discuss about how analyzing tumors through genome sequencing is advancing the way physicians can treat cancer and potentially cure all diseases.

(Key takeaways from this episode of StartUp Health NOW can be found here.)

[00:04] Unity Stoakes: It’s absolutely been an extraordinary day. We’ve been talking about health moonshots all afternoon and all morning, as well as the Health Transformers achieving them. I thought we’d kick off the conversation, really just by, understanding your moonshot. What do you care about? What’s the moonshot? Not just of yourself, but also Foundation Medicine.

[00:25] Mike Pellini, MD: The first thing I want to do is thank you for the invitation to speak here. This is a remarkable conference and I greatly appreciate the fact that I got to follow Bill McDermott, Vice President Biden, and Craig Venter. [laughter]. So, that’s very kind. I think that was a Krein brothers joke, by the way. What you’ll hear from, I think, the story that you’ll hear from me and Foundation Medicine is one that’s really consistent with one that you heard from the Vice President.

[00:50] Mike: As you hear the Vice President describe what the moonshot looks like, in some regard you might think about it as some forward-looking statement that is going to get some legs in the coming years. Absolutely not. Versions of the moonshot are happening today in this country and when we first heard this idea of moonshot described about a year ago, I know I, and the vast majority of people of Foundation Medicine, felt like they were describing the story of something that we’d been working on for the past five or six years in Cambridge, Massachusetts.

[01:22] Mike: So what are we passionate about? What does our moonshot look like? Our moonshot is based on the idea that we have to understand every patient’s cancer at the level of the molecular blueprint. If we’re fixing a car back in the 1930’s, when that T-Model Ford roles in, it’s making a noise, and the mechanic on the way in, probably already has a pretty good idea of what the challenge is with that car. If he’s sitting at his gas station, his petrol station, back in the 1930’s. Cars today are so much more complicated that it’s probably just sheer luck if the mechanic has an idea of what that car sounds like when it’s rolling into the garage or into his station.

[02:06] Mike: That’s where cancer care is. We can’t just listen to, just speculate, based on a very small snapshot into a patient’s disease. That, okay, we understand it. We ran a single test. We look at a protein marker. We looked at a specific gene. Or we know this female has breast cancer and just because it’s this stage of breast cancer, we know how to treat her. No we don’t. We think we do. But we get it wrong far too often when it comes to cancer care. So, what we’re passionate about, is really using this technology that helps us understand the human genome at the level of the tumor’s genome.

[02:42] Mike: We want to be, we see ourselves, as the company that’s the best in the world at doing two things. Extracting information from a patient’s tumor and then giving that information context. So when it goes back to the physician, so when it goes back to the oncologist, and even, ultimately, to the patient, he or she understands exactly what to do with that information. Now in order to do that, just because we can do that from a technology standpoint today, there are so many barriers built into the system that stop us from doing that efficiently. That’s what we’re driving towards. And when I hear the vice president talk about where this has to go and what we have to do, I’m incredibly optimistic. Because It’s not about the invention of new technology. It’s about breaking down barriers and collaborating.

[03:31] Mike: And I disagree a little bit with Craig about, when someone asked him the question about, you know, are there any companies out there that you respect? Oh, yeah. There are so many companies doing amazing work at all levels of our ecosystem, and we do, in fact, have to find a way to make sure that these innovations are going to make it all the way to the patient. [03:47] Unity: So, you mentioned focusing on, not just the cancer moonshot, but also curing many diseases over the last several years. What’s different today? What’s changed over the last 5 or 6 years that’s really sets us up in a new position to really make more progress?

[04:07] Mike: I think there is two parts to the answer. One is what’s changed and one is what is changing. What is changed, and again I just keep referencing back to the vice president’s comments, because every time I hear him say this I think he’s spot on. This is no longer about the invention of new technology. Yes, we need new drugs. There’s no doubt about it. We need more drugs. But there are thousands. There are thousands of compounds in clinical development today that are targeted to the specific patient’s cancer. So, they are coming along, and they are going to continue to come along.

[04:41] Mike: So, what’s changed is that we have the technology available today to do this. What’s changing is, I think, just the social dynamics of the ecosystem. And understanding that, you know, I know a physician stood up earlier and said “I’m not really that excited about getting my genome sequenced.” Yeah that’s reasonable. I got mine sequenced, but it was kind of boring to be honest with you. I don’t know, I know I’m Italian, I didn’t get any surprises in my ethnicity. I didn’t find that I was predisposed to anything.

[05:12] Mike: But what we have to understand is that in the cancer world, getting the tumor sequenced is fundamentally different than me, Mike Pellini, getting my genome sequenced. That’s something cool that wasn’t helpful to me today, maybe it will be something valuable in the future. But when you can understand that tumor? And you can act upon that tumor and we, frankly, have the payor system that’s corrected in this country. We have access to drugs. Clinical trials are no longer residing in the top twenty or thirty or forty academic institutions in the country. They’re actually throughout the United States. So, if someone’s in a remote area, maybe they’re a hundred miles, or two hundred miles from San Francisco, California. They shouldn’t have to drive into U.C.S.F, or Stanford to get to a clinical trial. They should be able to go to the local oncologist and enroll in a clinical trial with cutting-edge care there.

[06:06] Mike: So that’s what’s changing.

[06:07] Unity: How soon do you think that’s going to happen?

[06:09] Mike: It’s changing as we speak. It’s, there are pharmaceutical companies, there are organizations like Foundation Medicine. There’s the moonshot that’s preaching this and making clinical trials a fundamental focus of what they do. But it’s not one organization. It’s, you know, Sanofi, is not going to be the one organization that changes everything. But Sanofi, and Roche Genentech, and Bristol-Myers Squibb, and all the pharmaceutical companies, and the Foundation Medicines of the world, and maybe even the LabCorp’s of the world, and, you know, tech organizations.

[06:39] Mike: That’s what has to come together in order to drive it, so there’s a holistic solution. What we should be able to do, and we will be able to do very soon, is, you get your tumor sequenced, and when that physician is sitting down with you in his or her office, you can pull up all the relevant information on the drugs that are available, and clinical trials that are not available half across the country, but they’re available in your own backyard.

[07:03] Mike: That’s what’s changing, and this is changing in the real time. So I’m incredibly optimistic that over the next two, three, four, five years, this landscape is going to overhauled yet again.

[07:14] Unity: So, one of the things that I think is so exciting today, is just, in the discussion at events like these, when you hear people like yourself, or the vice president or Dr. Venter,

[07:27] Mike: Those two names have never been in one sentence before.

[07:30] Unity: Well there’s this optimism now. That wasn’t around even a few years ago. You know, I think about the bold goal set recently, I think by the Chan Zuckerberg Initiative, to try to cure all disease in the next hundred years. How does this happen and more importantly, how do we speed that up from 100 years to less time than that?

[07:59] Mike: I wish I had the answer to that question, but,

[08:02] Unity: Is it collaboration?

[08:03] Mike: Well, that’s an aspect of it, so, let me think about it in a couple of different ways. 1, is that, there are many different types of people on this planet. If I over simplify like two types of CEO’s, right? There’s the CEO that’s the Craig Venter of the world, the Mark Zuckerberg of the world. These guys are thinking out ten, twenty, thirty, forty, fifty, and a hundred years. That’s not me. I know that’s not me. I certainly have a picture of what things might look like in ten or fifteen or twenty years. But I have a much clearer image of what things will, in fact, look like in five years. In six years. In seven years.

[08:40] Mike: So, I think we need a combination. We need the human longevities that are pushing far off into the frontier, right? They’re out there. They’re pioneering new land. Foundation Medicine was doing that five or six years ago. We were a pioneer and we continue to be. But we’re a pioneer that’s delivering this information to cancer patients today.

[09:01] Mike: And so, one way that I think about the answer to that question, is that we need folks in the trenches executing today. And that means we have to share information today. We have to know where every single company’s clinical trial is today. We have to know that when we see a cancer patient, and we see, you know, hundreds of specimens come into Foundation Medicine every single day of the week. And I always say, if you think you’re having a bad day? Walk in to our accessioning department. Sit down and spend an hour or two there opening up boxes and read that requisition form. Because we’re not getting the males with an early stage colorectal cancer or a likely curable prostate cancer or female with the very early-stage breast cancer. We’re getting patients that have really complex disease and we see hundreds every single day and, kinda, your heart sinks. And so what happens, not kind of, your heart sinks. My heart sinks when I see these patients.

[09:56] Mike: So, when we sequence that information, and we associate it, and contextualize that information with the clinical trials and the drugs that are available. Granted, it can benefit that physician and his or her patient on a one-off basis. Or, we can find a way to share that information. So, even if a physician and patient encounter here in San Francisco saw that patient, very complex patient, with maybe a bit of a unusual cancer, that that physician in Arkansas, that physician in Pennsylvania, that physician in Germany. When he or she is sitting down with the patient can also benefit from that.

[10:36] Mike: That’s very doable today and we can solve some of these issues in the next five years which will ultimately pave the way for that long-term vision. So, I’d like to break down these barriers today. We have to find a way to share data. It’s why our company, we are the largest contributor to the moonshot, to the big data initiative by moonshot. We have put out more information on pediatric patients with cancer. I’m told the data set that we released earlier last year is two to three times greater than all the publicly available information in the world for kids fighting cancer. I mean that’s insane. That one company in Cambridge, Massachusetts could put out information, information these sequence tumors on 1500 kids. And it doubles or triples the amount of publicly available information.

[11:26] Mike: Again, that’s what the vice president is driving it. It’s easy for organizations to do that. We all have to be thinking that way. And that’s how we’ll make real progress in this field.

[11:35] Unity: By the way, we are going to open it up to questions. so, we’d like to make this interactive. If anyone does have a question we’re going to have a couple of mic runners. Feel free to raise your hand and jump in. While you guys were thinking of questions, one of the big challenges, in addition to breaking down the data silos, and figuring out how to collaborate, really has to do with the business models. Things like cost and how reimbursement works with some of these new innovations. What are your thoughts there? Do you have any recommendations, or where things need to go to really fix things in a way that brings these types of technologies and innovations to more people?

[12:18] Mike: One area where the system is really broken down is that we’re in an era where innovation is happening so quickly. If you expect each company and every healthcare startup to find, to generate all the data and do everything that they have to do in order to ultimately get that payor to pay for what they do, it’s going to take us a long time to innovate it, if we’re each forced to do it on our own.

[12:44] Mike: If we’re taking, we take tests through the FDA. We take products through the FDA.We know the ground rules. We know what the FDA is going to need, we know what the timelines are, it’s very straightforward. The payor world is fundamentally different. The payor world, there are no standards that are established. If you know how you have to interact with one organization, that means you know how to interact with one organization. So it’s as if there are thousands of FDA’s. For each one of these organizations, you have work to do in order to convince them to pay for whatever your product is.

[13:17] Mike: Overstating it a little bit. But just a little bit. So, I think what we need to see out of the payors is is something that’s, they need to hear the collaboration message. Now fortunately, some of them are. Because, when there is an early stage opportunity for innovation. Something that could really impact health care. If that company could sit down with an innovation unit at a big payor. And convince them that if your approach is successful and if it can do what your organization, what you and your organization thinks it can do, and you work together with a that payor, or you work together with a set of payors that have a similar vision.

[13:59] Mike: We can actually generate the data in a much more consistent, a much more streamlined fashion. So, even if, I’ll make up the number, even if I have to invest $5000000 more in an effort to generate data, five million dollars more in a clinical study. Or maybe it’s 15 million dollars more. But I know on the back end of that there’s going to be a dataset that that payor, or better yet, that group of payors is going to make a decision upon. There’s a much greater likelihood that I, my shareholders, my VC’s, depended on who it is, are going to support that type of investment.

[14:35] Mike: So, the payors, they’re in a tough spot. There’s so much innovation happening that they’re not just saying, they’re not just putting their head in the sand. But, they’re getting bombarded every single day and I think we have to come up with a mechanism through which the relationship with payors? It’s not an adversarial one, it’s one where we’re all driven to the same end game, and we’re trying to drive the data that ultimately results in better care.

[15:04] Mike: So, you know, the payor challenge in this industry is enormous and it’s growing. But part of the solution rests in this notion of sharing and collaborating information and collaborating with information. It’s not, it’s really not all that difficult. But there has to be willing parties and I think we will see payors, at least forward thinking payors, start to engage companies and their earlier stages.

[15:33] Unity: It’s interesting. You have an interesting connection into StartUp Health. I thought it’d be useful to share that story. It’s going to lead into a couple of other questions.

[15:44] Mike: It’s a Krein brothers story. I asked Steven how long ago we met and I was thinking it was like a year-and-a-half ago but he reminded me that it was actually February of 2016. One of the reasons that I haven’t told the story, but one of the reasons I appreciate the chance to tell the story, is because I think it plays into the idea of being an entrepreneur and just doing things when you don’t necessarily know what they’re going to lead too.

[16:09] Mike: We have an extraordinary busy company. We work around the clock end we need that vision of, to improve cancer care, and that keeps us going. But I think it was probably December of 2015, maybe November, I received an invitation to speak at this conference down in Florida. I had no idea we’re Lake Nona was. I didn’t know anything about this meeting. But when the note came in, the request came in, I looked at it and I talked to Sue who heads up our communication who’s sitting over here, and it was impressive list of people and I said what do we know about this should I go do it?

[16:43] Mike: And we were debating, so it looks like a great group of people. I’m just going to go do it and we’re going to camp out for 3 days they are. That’s all I’m going to do, I’m not going to go down and give a talk and then return. And just come back to Cambridge. So I went down and on the very first night we are taking a tour of this facility and I didn’t know anyone there whatsoever, which was fine. But I was standing next to a person who also appeared to not know anyone there. So we were going through the tour and we started talking. And it turned out that it was Dr. David Shulkin, who is the undersecretary of the Veterans Affairs Administration.

[17:15] Mike: And we struck up a relationship and we spent about an hour, an hour and a half talking that night, and he’s doing some really cool things and that relationship has developed over the course of the past year. So, I thought, okay it’s already paid for itself. I learned something, we made a great connection here. The next night I was having a drink at a function and I think I sat down next to, I forget the exact connection, but we started, I’m sure it was over wine or beer, I was sitting down and talking to Steven. After a little while I said Steven Krein, I’ve been told that I have to get to know your brother. He’s a guy from Jefferson in Philadelphia. And so Steven, instead of saying, well, I’ll connect you guys via email afterwards and next time you’re in Philadelphia, see if you can meet him.

[17:54] Mike: Breaks out his cell phone, calls up Howard. And then the next thing I know he hands me his cell phone and Howard and I just started talking for about 20 minutes. We knew so many of the same people, there was so much overlap because I do a lot of work with Jefferson as well in the cancer center there. And it was just one chain of events after another. And the moral of the story is, you just never know. Right? We go down to Washington DC all the time now for these policy meetings and one thing that’s incredibly consistent about a policy meeting is that I have no idea what the outcome of that meeting or that day is going to be until after the fact.

[18:30] Mike: But you know what? More times than not something good comes from that day. Something good comes from that meeting. You know, I’ve had the chance to meet with the vice president a multiple times over the last year. Who knew that we would be so deeply involved in the moonshot and really one of the major data contributors to that effort. So when I look back on it, it’s just a couple of crazy brothers. But, that’s a good thing I say that in a very positive way. [18:56] Unity: There’s a crazy sister there too.

[18:58] Mike: I can’t confirm or deny that statement though.

[19:09] Unity: I’m sort of a brother, they dubbed me Ira Krein.

[19:04] Mike: But there’s something to this idea, if you just don’t know, and there’s a good group of people that gets together, good things can come of it. That’s certainly something I learned through this relationship. So thank you Steven.

[19:14] Unity: I think that is a great lesson. I’d love to open it up. Are there any questions in the audience? Yeah. Hey Liz, do you have a microphone?

[19:25] Audience Member: Hi, Jonathan Dariyanani, from Cognotion. Question for those of us who have friends and family with complex cancers. Would you encourage us to have them explore tumor sequencing at this stage if it’s not something that their oncologist is pushing? And how do you feel about the majority of the way oncologists treat this issue and how they interact with innovation at the provider level. Is there, is there the right attitude? Is there the right information? Or is this something where the consumer needs to be educated in, advocating as well.

[19:59] Mike: Yes, yes, yes, yes yes. I mean there’s this whole series of questions to unpack there. There’s clearly a bell curve, right? We know there’s a bell curve in the clinical community. Fortunately, what we’re seeing, we just announced the results from Q4. We continue to see volumes grow significantly, despite all the obstacles out there. So, the academic, virtually every major academic medical center in the United States is, if they have a patient with even a relatively complex, I’m kind of throwing in all the tumors, anything that’s not relatively straightforward, where there’s an eighty, ninety, ninety five percent cure rate, I’ll call that complex. They’re, almost all of them, are pushing the direction of, not just running a gene or five genes, or ten genes, but let’s explore the genome of the tumor.

[20:46] Mike: I think there’s important starting point. But we also see about 60% of our volume, each quarter, comes from the community. So it’s starting to reach out into the community. The frustration in the communities around the United States is, it’s a genuine one. It’s doctor saying, you know, I get this information back, I see there might be an opportunity for me to intervene with my patient, but I don’t know how to get that drug. I don’t know how to get that clinical trial. And that goes back to the idea where we have to share information, and break down these barriers. So, absolutely.

[21:19] Mike: I think 5 years from now, if a woman, just to go to a more straightforward example, if a woman is diagnosed with a very early-stage breast cancer and it’s ER and PR and HER2 positive, it’s a half centimeter tumor, the cure rate should be 95%, and I’m not convinced that even five years from now, she should be getting that tumor sequenced. But for that set of tumors,

[21:47] Unity: And is that because of cost?

[21:50] Mike: Well, because we know, because you know, you don’t want information just because you can do it.

[21:54] Unity: The false positives? Too much data?

[21:57] Mike:It’s data overload, data overload. But if we have something that works and there’s a 95% cure rate, maybe research takes it up a little bit further by then. I’m just not convinced that we have to be pushing sequence, pushing the sequencing of the tumor on an individual like that, on a patient example like that. I think that where the value is, is for all the others. Where the cure rate is not 95%. Where it might be 80%. Or it might be 20%. I think about individuals diagnosed with small bowel tumors. You know, tumors of the gallbladder.

[22:38] Mike:These patients, yes there is standard of care. But standard of care sucks. For so many patients. It’s horrible, right? And if your oncologist says, you know, here’s a win, 3 months, the data shows there’s three months of additional life. Well that can be very important at times. There’s a parallel path that I would encourage people to explore and I feel very strongly about that so might not be plan A. But in everything else, and so many things that we do in life, we have a plan B, a plan C, and a plan D. Don’t wait until plan A runs its entire course. The individual’s really in a tough condition. And then you say, my gosh, I’m going to go get the tumor sequenced.

[23:24] Mike: If you have a condition where you believe that there’s a high likelihood of recurrence get the information. Have your plan of attack, so if that patient’s tumor re-occurs, you have plan B. You have plan C. You have plan D. That’s how I would look at it. We have to turn this from a game of roulette into a game of chess. Far too many roulette wheels are being spawn with cancer patients. We need to focus on a game of chess. And make it strategic if we’re going to really continue to make a dent in this broad group of diseases.

[23:59] Unity: Time for one more question. While the mic’s getting there I want to ask a super quick question. There’s a lot of entrepreneurs in the room and throughout the festival. A lot of Health Transformers. How can they be helping an organization like yours?

[24:15] Mike: Focus on, as entrepreneurs, you focus on the challenges that can be overcome. The challenges in our space are not necessarily the ones that have to do with extracting that information, that genetic data from a patient’s tumor. It’s all about the delivery of health care. And so, anything that breaks down the barriers to allow a patient to get access to that drug, to help a pharmaceutical company connect with that patient, to help a payor see the light sooner, we have to overhaul the, we have to overhaul the health care, the payor system in this country. Think about the barriers that stand in the way of advancing technology. I love focusing on technology, But I’m not enamoured with technology. At least in terms of medical innovation. I think about, okay, if a technology is really exciting? One of the things that we need to focus on in order to open the door to get that technology to patient care, into the clinic, I should say. Those are incredibly meaningful. And if a company can do that, or better yet, if organizations, companies can do that, it changes the game. So we, it’s the delivery of healthcare. It’s absolutely the delivery of healthcare.

[25:29] Unity: Okay, we’ll take this one, and then you Ira.

[25:32] Audience Member: Second to the last question. Thanks.

[25:35] Unity: Can you say who you’re here with?

[25:37] Audience Member: Oh, Sorry. I’m Joshua Schiffman, I’m from Huntsman Cancer Institute, at the University of Utah. So, as a pediatric oncologist, I love, love, love that you’re so centered on pediatric oncology. A comment and then a question. I love the chess analogy as well. One thing though that I would caution against is, if you only sequence at diagnosis, obviously, there’s going to be evolution, clinical evolution, and so on. So, you may, if you only use the primary diagnostic sample, obviously, miss what you could target during relapse. So I think everyone should be targeted at diagnosis. And use that information at diagnosis, when it’s most relevant.

[26:13] Audience Member: My question is, how do you reconcile some of the reports that are coming out now, that different commercial sequencing groups have slightly different results. And how do the clinicians, and how should the patient use that information?

[26:30] Mike: Joshua, you know, you are spot on. What we know, what we believe today, what we know the data shows us, is that if you can fully assess the tissue sample itself, I think that’s our best estimate of truth. So then, the question comes in, and then even in that truth, the reason that we’re taking everything through the FDA? Is we know how darn hard it is to do what we do and it’s such an easy thing to screw up. You don’t plugged in a sequencer and say produced the results. It’s so easy to screw up. There are thousands of steps along the way, so we think new standards have to be established and we’re actually fans of the FDA getting a little bit more involved in this space. But then once the cancer is sequenced, right. There’s opportunities to use blood, to monitor disease. [27:19] Mike: And we think liquid biopsies, especially in the form of circulating tumor DNA is going to be a mainstay of cancer care in the future. Here’s the challenge. For every single cancer, the utility of that blood based sample could be different. What a pancreatic cancer sheds into the blood might be fundamentally, is fundamentally different from what a lung cancer might should into the blood. And until we get the data we just don’t know. And so we think there’s danger in saying, blood, blood, blood, blood, blood. It’s easier, get blood, and you can get the answer. We think we should say, Okay, tissue is the gold standard today, that we know. If you can’t get tissue, then blood is a nice second option, if it’s an organization that’s doing it well, and there are a few that are doing it well in the liquid biopsy, there are a few that are doing it well in the liquid biopsy side.

[28:09] Mike: So, there’s still so much for us to learn. But I agree with your comments a hundred, you know, I agree with them fully. We still, if there are disparities that pop up, and until we start working more closely with the FDA, until we set these standards higher, and higher, and higher, until we compare data in a large study, we’re just not going to know the answer. And so we need folks like you to help us tease it apart.

[28:30] Unity: So one last quick question.

[28:32] Mike: I’ll try to make my answers short.

[28:34] Unity: Ira Brind.

[28:39] Audience Member: We were talking about access. About payors. What percentage of these samples that are sent to you are covered by a third-party payor, and what percent are out-of-pocket.

[28:49] Mike: So, great question. I’m trying to think how do I answer it quickly. So I’ll try to be succinct here, and that is, about 30% of the tests that we receive are from patients who fall under Medicare. So Medicare beneficiaries. We’ve run over a hundred thousand specimens, we have not had a single test, really more than a, you know, we haven’t had very many tests reimbursed by Medicare. But that’s all changing right now. We finally see Medicare coming around. They’ve issued some coverage decisions for major indicate. They have one coverage indication for lung cancer, three more drafts were just issued. So, we see Medicare coming along.

[29:29] Mike: And we’re really pleased by that. Where the third party payors, we get, you know, we get roughly half of our tests paid for by third-party payors. But we also therefore have to work with patients on a very individual basis to make sure that if they can’t afford what we do, there’s no way that we’re going to withhold care. And so, we have a team of people, we have an outside nonprofit organization that works with us. And frankly, we bend over backwards for patients, because we will never, ever, ever turn down a patient. Because they can’t pay. So, we’re in the midst of this battle but we, I’m comfortable with where things are finally, because we’re seeing real progress on the payor font.

[30:12] Unity: Mike, I just want to thank you for everything that you all do, and for being here and sharing your insights today. It means a lot.

[30:18] Mike: I appreciate it. Okay, thanks everyone.

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