Mark Levin, co-founder of Millennium Pharmaceuticals, explains personalized medicine as delivering the right drug to the right patient at the right time. He points to Gleevec for leukemia and BRAF therapies for melanoma as proof it works, and identifies a critical gap: venture capital has moved downstream, leaving early-stage academic discoveries starved for funding. China is advancing faster in this space, threatening U.S. leadership.
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So, Mark Levin, Third Rock Ventures, welcome. Thanks for joining the Journal of Commercial Biotechnology for this discussion.
Thanks, Ralph. It's great to be here.
And that was a great speech. You certainly have a lot of enthusiasm for personalized medicine still. Now, you were involved with the Personalized Medicine Coalition at a very early stage, were you not?
Yeah, we at Millennium Pharmaceuticals, which was a company that we founded around personalized medicine, we actually founded it in our office probably 10 years or so ago. Brian Munro and I were sitting around talking about how we really needed to make a difference, not just in technology, but in Washington. And so we funded the coalition and it's just gone extraordinary places since then.
And has the definition of what is personalized medicine changed much over those years? You know, the science has moved forward a lot.
Yeah, I don't, Ralph, I don't think the definition has changed a lot. It was always the right drug for the right person at the right time. And, you know, if we go back and we look at therapies 10, 20, 30 years ago, if you get a drug for heart disease, for instance, it might be cholesterol because you do the phenotypic measurement of cholesterol, but that's not necessarily the cause of your heart disease. Or if you have lung cancer, you might get chemotherapy for lung cancer, at least in the past, but that was really killing rapidly dividing cells. But today we don't have phenotypic— we have phenotypic information, but now we have genotypic information. We can look at the heart problem, we can look at the cancer, we can look at the genes, the combination of mutations that actually cause these diseases, and then and develop drugs and diagnostics around that.
Can you give a couple of examples of success stories that we've had in the last 10 years or so?
Oh, there's just been some extraordinary breakthroughs just over, actually over the last couple years. One example actually is Gleevec, which has been an incredible drug for CML. That biology was done 30 years ago, and yet it took 25 or 30 years to bring that product to the market because of the long understanding of the biology, the chemistry, and the whole process to get it approved. Today, there's been a couple drugs recently that have been approved, but about 3 years from discovering the mutation to actually bringing the product to market. There's a brilliant product in melanoma today that actually goes after a mutation called BRAF. The protein is BRAF and there's a mutation in BRAF. And people have identified this mutation and now we're treating metastatic melanoma patients that had no alternative before.
And what are you getting excited about at the moment? I know you're doing a lot of investments. What are you looking What are you excited about?
Well, we're very excited certainly about the cancer area. We founded a company called Foundation Medicine where we're actually working with cancer patients and their doctor. Cancer patient comes into the office, a biopsy is taken, it's sent to the company, and we actually sequence all of their cancer genes. We find out what the mutations are in those genes, and then we search all the literature, drugs on the market, drugs in the clinic that go after that mutation. That information goes back to the doctor, and then the doctor then recommends that particular drug drug. That's just incredible. Another very exciting area for us is in the heart disease area. For instance, hypertrophic cardiomyopathy, a rare genetic disorder in many ways amongst lots of heart diseases. We're actually now beginning to look at the mutations in these heart disease patients and developing drugs specifically for the mutations that they have in their sarcomeres. The science and biology is exploding now as this next-gen sequencing efforts have really brought a lot of things forward.
So the sequencing, the science, all of this is going incredibly well, but yet some of your colleagues in the VC world are reluctant to invest in personalized medicine companies. What is going on there?
Well, that's a great question, and actually we're all worried about that. I think as a country we should be worried about— over the last decade, the pharmaceutical industry has moved very much downstream based on the challenges that they've had over the last 10 years. Biotech has not been as aggressive potentially in some of the early-stage efforts, and as pharma moved downstream and the public markets were not as strong, the venture capitalists in many cases moved downstream. They were not willing to take the risks in the early-stage areas. So what's happened now is there's this huge hole where many people are no longer able to develop their breakthroughs because the funding is not there.
What stage of research is that happening?
Well, it's happening after an academic makes a basic discovery in the lab, whether it's in cancer or rheumatoid arthritis, maybe associated with a personalized medicine biomarker. Many of those are lying fallow now in academia. Because there's not the dollars in the venture capital community to really bring those forward.
So it's a problem of investment at translational research stage?
Well, it kind of goes from the fact that pharma has moved downstream, the IPO markets have dried up a bit, we're seeing real challenges on the reimbursement side, some challenges on the regulatory side in many ways, and so as we talk to our VC colleagues and they think about developing drugs or diagnostics or devices, they bring these issues up Immediately. And what they're saying is we need to see these solved. We need people to work together in order to really form a strong team to really bring these products forward as opposed to everybody focused on their own efforts. Okay.
So when you're saying that people really need to work together, who are the key stakeholders and who's not working with who?
Well, I think the key stakeholders are certainly the government, regulatory aspects of that, the reimbursement aspects of that. I think the pharma and biotech need to step up and be much more thoughtful about how they're going to work work with a lot of these government institutions. Academia is a big part of that. The NIH funding is flattened out now for us. That's going to be a huge challenge for these breakthroughs. So, you know, it doesn't really fall on one group's shoulders in any way, but what's most important is that we all work together trying to maximize our own effort. Because when everybody does that, then you end up with extraordinary high cost and you end up with a low-quality product. We've got to get everybody at the same table to really make a difference.
How do we do that?
Well, I think PMC is making a major effort there. I know PCAST, the President's Council, is working very hard on that. There's lots of different groups on it. I guess to be honest with you, I'm not terribly happy with lots of the results. I hear people coming out of these meetings, you know, really complaining about people not working together, the politics of the situation. There's lots of challenges. But if we're going to make a difference for patients, people have to set that aside and really focus on how we solve all these problems together. As opposed to independently.
Is this a global problem, or are other countries doing this a little bit better?
Well, no, it's a global problem. The US is clearly the leader in personalized medicine, and the majority of the research is done here. But I think it's an excellent point. There's lots of countries, certainly China and other countries around the world, that are passing us in many areas. We better watch out here. They're being very aggressive in this space, and so we, we need to make sure that we retain leadership here, not only for the patient but certainly for our economy. And being a world leader in healthcare and medicine. It's really important to who we are as Americans.
