February 4, 2014

AMP press conference


JOHN BURKLOW: Good morning everyone. Welcome to today’s media briefing. I’m John Burklow and I’m the NIH Communications Director. Thank you for joining us. Just to give you a preview of the morning’s program, first we will hear from our speakers. To my left, Dr. Francis Collins, Dr. Mikael Dolsten and Shanelle Gabriel. Then I’ll open it up for questions from credentialed members of the media. And now, without further ado, I’ll like to turn it over to Dr. Collins, the Director of the National Institutes of Health.

FRANCIS COLLINS: Well, thank you, John. Good morning everyone. It is my great pleasure to announce today the Accelerating Medicines Partnership or AMP. AMP is a brand new partnership between NIH, 10 biopharmaceutical companies, the Foundation for the National Institutes of Health, FDA, several patient organizations and others. AMP is tackling a major challenge in drug development in an unprecedented scale.

As you know, AMP is a unit of current and we’re all AMP’ed up this morning [Laughter] about the promise of this new consortium. And, in fact, this morning we want to AMP-lify for you why we’re so excited. Now that you’ve recovered from those awful acronym acrobatics, let me take a moment to introduce you to representatives from the companies and our partners from the non-profit world. Part of the reason that we’re here is because of this unprecedented consortium and I want you to see who these folks are.

So I ask each of the many leaders to wave their hand as I call their names. And they will be available for one-on-one conversations after the formal part of the press conference. I’m going to do this by alphabetical order by last name so that no one will assume there is any preference here. So the companies and their representatives are: Dr. Lon Cardon, who is Senior Vice President, Alternative Discovery and Development at GlaxoSmithKline; Dr. Keith Demarest who is Vice President Global Health of Research, Cardiovascular Metabolic Area at Janssen, part of Johnson & Johnson.

Here at the table, who you’ll hear from in a minute, Dr. Mikael Dolsten, President Worldwide Research & Development at Pfizer. Next, Dr. Robert Filippone. I hope I have not abused the pronunciation of your name. Vice President for US Policy and Government Relations at Merck; Dr. Tim Harris, Sr. Vice President, Translational Medicine and Biochemistry at Biogen Idec; Dr. Jan Lundberg, Executive Vice President Science and Technology and President of Lilly Research Laboratories. I must say Jan spent many Fridays mornings on conference calls with me, Michael and Francis Cuss of Bristol-Myers planning this effort.

Dr. Gary Nabel, Sr. Vice President, Chief Scientific Officer and Deputy to the President for Global R&D for Sanofi; Dr. Satwant Narula, Vice President, Immunoscience and Fibrotic Diseases at Bristol-Myers Squibb; Dr. Lisa Olson, Vice President Immunology Research at AbbVie and Dr. Jonathan Zalevsky who, starting with a “Z” always get named last, which is really not fair, who is head of the Information Drug Discovery within Takeda’s Pharmaceutical Research Division.

Pretty impressive group of 10 pharmaceutical company leaders gathered here this morning to talk about this new consortium. In addition, we have non-profit organizations represented here, again in alphabetical order by last name. Dr. William Chin, the Executive Vice President of Scientific and Regulatory Affairs at PhRMA, the trade organization; Meryl Comer, President and CEO of the Geoffrey Beene Foundation Alzheimer’s Initiative; Steven Echard who is Executive Director of the Rheumatology Research Foundation; Leslie Miller Hanrahan, Vice President for Education and Research of the Lupus Foundation of America; Larry Hausner, Chief Executive Officer, American Diabetes Association; Harry Johns, President and CEO of the Alzheimer’s Association, and George Vradenberg, Chairman and Co-founder, UsAgainstAlzheimer’s.

We also have with us patients in the audience, one of whom you will hear from shortly, Shanelle Gabriel, who is affected with Lupus. We also have Cynthia Leoro, where is Cynthia, right here, who has type 2 diabetes and will also be available to speak with you about her experience with the disease and what AMP means to patients with diabetes.

Now, I’m not done. From NIH we have leaders of the institutes that have been particularly, deeply involved in this planning process, Dr. Robert Carter here is Deputy Director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases; Dr. Richard Hodes, the Director of the National Institute on Aging; Dr. Tom Insel, Director of the National Institute of Mental Health and Dr. Griffin Rodgers, Director of the National Institute of Diabetes and Digestive and Kidney Diseases.

From the Foundation for NIH, our major partner in the conduct of this project going forward, Dr. Maria Freire, who is President of FNIH, Dr. David Wholley, who has been our main collaborator at FNIH and a real champion of this effort. I also want to recognize our friends from BCG who have helped very much in the organization of getting us to this point and who are somewhere in the back of this room, Michael, Sarah and Chase.

And finally, although at the last minute she was not able to attend, I wanted to recognize Janet Woodcock from FDA, Director for the Center for Drug Evaluation and Research, who is also part of this enterprise as we go forward.

So that’s the cast of characters. What’s the play? Well, all of us are pooling our brains and our resources, about $230 million dollars over the next five years to transform the current model for identifying and validating the most promising drug targets. What does that mean in plain English? Well, it means we are going to try to increase the odds of picking the right targets to go after for the next generation of drug development. And not only that, we want to pick them at the very beginning of the development process and thus avoid wasting precious time and money chasing down duds.

Why do this now? Because the science is ripe. We’ve come a long way in recent years in our understanding of the causes of disease. As a result of technological advances and genomics, proteomics, imaging and more, researchers have increasingly been able to identify fundamental changes in genes, proteins and other molecules that predispose to diseases whose causes have vexed us for all of history.

In the last five years researchers conducting genome-wide association studies have identified more than 1,000 risk factors for common diseases. These are biological changes that ought to hold promise as drug targets. In fact, most of the known drug targets for common disease in these same studies. You might think of those as positive controls. Given that, surely there must be some additional therapeutic gems in this collection.

But here’s the challenge, only some of these biological clues will make good targets for effective therapeutics. And getting it wrong is costly, both in term of research and dollars and patients’ lives. Consider this. It takes an average of more than a billion dollars and 14 years to bring a discovery from the lab to the patients. From beginning to end of this pipeline, the current failure rate is about 99 percent. The most painful failures are those that happen in phase II and phase III clinical trials, where a drug that has already had years of work and millions of dollars invested in it, is found not to provide benefit for those with the disease.

All who care about therapeutics agree we need new strategies to avoid those failures. Patients and their loved ones simply can’t wait. So now it’s time to work together across the public and private sectors to harness the rich, new resources of biological knowledge and find better ways to develop greater numbers of effective therapeutics at a faster pace. That’s what AMP is all about. The public and private sectors came together because we realized collectively that this is a job too big for any single group. Although competition is often the name of the game in drug development, in this case the watchword is collaboration.

What’s more we will be putting all of our results out there for anyone to use. You heard me right. All data and analyses generated by AMP will be considered pre-competitive and will be made swiftly and freely available to the entire biomedical research community. Competition will come later after the initial discovery phase where we, the AMP team collectively, identify the most compelling targets. And then the full competitive power of the pharmaceutical industry will kick in to develop the actual therapeutic molecules.

AMP has been more than two years in the making, with hundreds of hours of intense discussions amongst creative minds from NIH, academia, industry and the non-profit world. And this is another, unique feature of this partnership. AMP partners have developed detailed, milestone driven research plans in close collaboration through a shared, integrated, governance structure. This is not a partnership in name only. We have rolled up our sleeves, left our affiliations at the door and put real skin in the game. The cost of this roughly fifty-fifty divided between NIH and the pharmaceutical industry.

Our focus is truly on the science and figuring out the best ways to find the right targets to go after. All who are involved in AMP would agree, you can’t change the world, or in this case drug development overnight. It’s just not possible. And to think so is actually counterproductive. So consequently, based on extensive polling of industry by our colleagues in BCG to identify the most compelling needs and opportunities, AMP will begin with three to five-year pilot projects in three disease areas: Alzheimer’s disease, type 2 diabetes, and the autoimmune disorders, rheumatoid arthritis and lupus. We hope and expect that these pilot projects will set the stage for broadening AMP to other diseases and conditions.

In these pilots researchers will use new technologies and patient data to distinguish which biological targets are most likely to respond to new therapies. They will also seek to characterize the most effective, molecular indicators of disease, which we call biomarkers. This information will then be used by those seeking to develop tests and treatments that are precisely aimed at those targets.

As an example of how this approach can lead to unprecedented advances in therapeutics, consider the recent identification of a drug target called PCSK-9. Individuals with reduced function of that gene turn out to have very low cholesterol levels and are dramatically protected against heart disease. But otherwise they are fine. This revelation has led to a furious race among several companies to be the first to gain approval of a drug that blocks PCSK-9. But we believe there are a lot of other PCSK-9s out there to be discovered. We just need to assemble the right team to do so.

You are looking at that team. AMP has the right stuff. This is not something that will just benefit big pharma. The entire biomedical research community, along with the entire patient advocacy community has a shared interest in compressing the timeline, reducing the cost and increasing the success rate of new, targeted therapies. We believe the stars are aligned and the opportunities ripe for all sectors of the research enterprise to join forces to resolve the critical issue of finding better targets so we can development better drugs at a more rapid pace.

I think the fact that 10 companies of this stature have signed on, have committed themselves to working with government, with academia, with non-profits and with patient groups, putting their own resources into it, really says it all about the potential of AMP. This landmark partnership will transform the way we develop medicines. And I think no one will be happier to hear that than patients and their loved ones for whom we really are dedicated in this effort.

So I now want to introduce my colleague and my friend, Mikael Dolsten of Pfizer. Mikael has been a stalwart champion of this partnership from the beginning. And I’ve asked him to say a few words about what AMP means to his company, Pfizer and to represent all the companies involved here today and also what it means to him personally as a physician scientist. Mikael.

MIKAEL DOLSTEN: Thank you very much, Francis. So, it’s a great moment for all of us to be here as a big team AMP-ing up our muscles. And thinking back almost 30 years ago, when I was a much younger physician-scientist with a passion to translate basic science in knowledge that could be impactful for human medicine, it was on one hand very inspiring but it was also frustrating because you were facing patients for which we had so few specific and targeted treatments, whether cancer patients, patients suffering from autoimmune diseases, metabolic diseases and new psychiatric diseases.

This frustration inspired and challenged me, I think similarly to many of my industry colleagues that you have here, to join the pharmaceutical industry to see if we could work within the industry but collaborate with academia and find a way to come up with treatment solutions. And over the last 25 years that I’ve spent in this industry, we’ve seen significant advances such as the number of targeted treatments initially for blood cancers and recently for lung cancers, biologics for autoimmune diseases, medicines for diabetes, cardiovascular and also some steps forward in many other areas.

However, for many of these complex, chronic diseases — too many patients progress and their health is declining. And for some of the diseases such as Alzheimer’s, breakthrough drugs have been elusive. However, today, I think we are reignited by the recent explosion in genomics, proteomics and high resolution imaging, which gives us an insight into first ideas and clues about many human diseases. And I wanted to say that it’s about 13 years ago, I think in February 2001, when Francis and a few other colleagues published first papers on the sequence of the human genome, which in a way was the first pillar to kind of a pre-AMP era. Without that we couldn’t have been here today.

But we are still navigating in a landscape where a lot of this richness in information is not in a consistent manner brought together. It’s fragmented and it’s not easily accessible to first move into solid drug discovery and drug development project as Francis alluded to. It’s almost like you are traveling in a landscape of biology but there are no clear signposts where to go. Your maps lack details. And what we would like to have is a precise navigation system, a GPS for human disease. And this GPS is urgently needed because we have pressing needs for patients suffering from many of these diseases. And we are wasting resources and time.

And we heard Francis mention some of the statistics. When we start the discovery process in an early phase, it’s usually one percent of them that may make it to a successful medicine. And when we move forward some of the most prioritized projects into late stage trials, where we started thousands of patients and we spend not millions but hundreds of millions of dollars and sometimes like in cardiovascular disease a billion dollars. It is still only 50 percent of the projects that really succeed.

So we can’t afford to continue like that because we want to serve all of the needs for patients and make real steps forward in the way we can treat and manage disease and do it in an efficient way for the entire healthcare system. So I think that challenge some two years ago made Francis, myself and many of our colleagues, to think what can we do differently? How can we think bigger, smarter? And how can we heal the innovation ecosystem, which in some way had too much cracks? And how can we nurture it to become a much more integrated innovation system. And AMP emerged as one potential solution and a solution that now has come to launch as the solution that we would like to embark on.

So it feels really exciting to be here today with you and announce this forward looking initiative. As Francis mentioned we decided to focus on three areas. Alzheimer’s disease, obviously a devastating, memory-robbing disease. And where we would like to, on the one hand make sure that we can validate and expand a collection of biomarkers that will have prognostic value. And those biomarkers will in some way, show in a numerical manner how a patient may improve and worsen and be integrated later on into drug development and allow you to treat early before the patient deteriorates and it’s difficult to reverse the disease.

It will be critical to have FDA colleagues involved here to get their guidance, how those biomarkers also can be regulatory endpoints. And I do hope at the next meeting we will have FDA presence in the room. We will also try to understand further the disease causing pathway in human’s brain and try to collect from a number of studies some integrated networks on what is driving the disease deterioration.

In the meantime, we would like to continue developing targets and understanding clues to diabetes and downstream complications. We have made a lot of progress in how we can manage glucose and lipids but the patients still progress, whether it’s the deteriorating pancreas or even worse downstream kidney complication causing millions of Americans to see an end of a journey in dialysis transplantation of fatal outcome. And we’re spending billions and billions of healthcare dollars inefficiently. We hope to create a portal of knowledge for diabetes patients that allows us to cherry pick targets that may help to preserve the pancreas, reduce insulin resistance or reduce the risk in cardiovascular events that is elevated in this population. And hopefully, I put a lot of hope to that, to also intervene with diabetic complications.

In the near term, we would like to address autoimmune diseases, rheumatoid arthritis. We have made great progress in RA but there are still many patients that do not respond to the biologics that for some have transformed their life. And we do believe here that if we drill down on the single cells of the immune systems and try to understand how they changed during disease, that we will be able to restore normal immune function and bring back life quality, reverse the disease process in RA and lupus. And for lupus, there is a very great need. We have over many decades only seen one drug with limited efficacy.

So I’m very excited to see we can pool our resources and put our best minds and talents to address this unfortunate situation. And the science is rich so I think we will see near term great advantages.

The AMP initiative is a key effort to bring a more productive ecosystem of innovation together. But it can’t stop here. We need to find additional ways to collaborate. And I wanted to give an example from my company, Pfizer, how we over the last couple of years have worked with a number of other pharmaceutical companies. We have developed drugs together with BMS to prevent occurrence of stroke; together with Merck to move forward new diabetes treatment; together with Lilly to explore new pain medication; and recently with GSK, combining drugs in oncology to see if we can create more long-lasting treatment outcome to what now is a good hope of months or a year to maybe in the future for decades of healthy living for patients suffering from many of this difficult diseases.

And let me end on a personal note. I look forward because of this initiative to be able to face patients, their families and caretakers, that we were able to overcome science barriers and business barriers because we wanted to accelerate the advancement of science to bring urgently needed treatment to patients. And I think AMP has the potential, as we discussed just yesterday, in the next five years to accelerate medical advances in a way that science will be easy to comprehend and our pipeline will look richer, and a patient can aspire to see that what we accomplished in five years that might have otherwise have taken 10 or more years.

So, I look forward to start this journey, to make sure that we build a very accurate GPS for human disease and be here to share with you, as we update our progress, and what hopefully will be then a GPS 2.0.


Thank you for your attention.


FRANCIS COLLINS: Thank you, Mikael. I hope you all felt the buzz here of AMPing your biological GPS. I’m now delighted to introduce our very special guest, Shanelle Gabriel who has dealt with the diagnosis of lupus for more than a decade. Shanelle is a singer, a poet, a lyricist and an advocate for lupus awareness. Born and raised in Brooklyn, New York, she has toured both nationally and internationally. She will provide a patient’s perspective and tell us what it is like to live with lupus and tell us what this partnership called AMP means to everyone seeking better treatment and cures. Shanelle.

SHANELLE GABRIEL: I’ve very overwhelmed. It’s a blessing to be able to speak in front of you all today. And I meant to bring a napkin up here because I’m trying not to cry. [Laughter] Don’t want to ruin my make-up. But this partnership means so much to me as a person living with lupus and with friends and family who are affected by Alzheimer’s, who have Alzheimer’s, type 2 diabetes and other rheumatoid illnesses.

So one of the common things I hear is, ‘But you look so healthy. You don’t look sick.” And I didn’t look sick in 2004 when I was diagnosed with lupus. It was my junior year of college. And while everyone was hanging out and partying, I noticed that I was just a lot more achy than I was before. I noticed that no matter how much sleep I got, I felt like I got none. And I kind of brushed it off as maybe I was just achy. I was a dancer in college. And maybe I’m just achy from working out and from all of that.

And slowly but surely I just started noticing that my fingers turned colors in the cold. And I went to college in upstate New York so it was like it was just cold. And I ignored all of these symptoms. It wasn’t until I lost my hair, and as a lady you know you don’t mess with your hair. And I went to a doctor then and said, “Forget all the other things. Let’s fix my hair first and then we will talk about the achiness and everything else.”

And he — prior to that — I’m sorry. I am skipping something. Prior to that, while I was in college I did see a doctor there. And she listened to my complaints and said, “Maybe you have allergies,” and gave me Sudafed and Benadryl. So I’m achy. Here I eventually know that I have lupus but my first diagnosis was allergies. And so, going back to New York, once again, I lost my hair. I realized I lost my hair. I went to a doctor and I was blessed to be at a hospital seeing primary care physician who was right next door to a rheumatologist who knew what lupus looked like.

So while it did take me a year and a half to be diagnosed, I was one of the lucky ones. Many people with lupus, and I interact with a lot of different people, it takes them years. They get diagnosed with everything from syphilis to just plain old crazy or you’re making these symptoms up. And since then I’ve been blessed to be able to speak about having lupus. And at first I didn’t want to because I didn’t want anyone to think any less of me because I was very much a hard worker. I was one of the super women that did everything and ran everything and was in charge of everything. And I had to give up dancing because it was just too much on my joints. And it just moved me into another path.

But this partnership, it means a lot because many of the medications I’ve been on were not for lupus. They were for other diseases. I was on medications that are pre-chemotherapy treatments. I’ve been on medications that are for people accepting liver donations, kidney donations or things of that nature. I’ve been on steroids which, as many of the doctors and people in here know and are shaking their head at me is a very toxic drug but is like the only drug that seems to work. There’s only been one drug that has targeted lupus since its existence.

And the need for therapies that target — a need for research, a need for early indication, early indicators of what these illnesses are, is very, very important. So it’s — so to see everyone in here teaming up together to try to find better ways to help people like me and people battling these illnesses, it means a lot. Being able to put aside any other agendas and be able to really try to think about the patients and the people that are affected means so much to me as a patient, as a person who has friends that have to prick their fingers and check their blood sugar, as someone who had a grandmother who passed away about a year ago who liked to ask the same question over and over, especially about the ex-boyfriend who is no longer in the picture, [Laughter], and there are so many other people that are dealing with and know and are affected by this.

And I hope that this initiative really will lead to many, many advances in these different therapies and this is not just the first and these illnesses are not just the first to be targeted. That these partnerships will happen for other illnesses out there so that we can continue to just knock them all out. And I had to include one AMP reference. I’m very, very AMP’ed up about this partnership. It’s a great opportunity and it’s a — I’m just hoping these leads to many new therapies that will mean we can all, myself, those living with type 2 diabetes, those living with Alzheimer’s can all, because we all look amazing on the outside — but we want to make sure that our insides look just as amazing as well.

Thank you so much.


JOHN BURKLOW: Thank you very much. You really brought it all home to why we are here today. So now we are opening it up for questions for credentialed members of the press. And please state your name and affiliation before your question. And, okay — oh, just to let you know Renate has a handheld mic.

VAL: [Initially without a mic] This is for Dr. Collins. I have sat here and listened to everything and I understand. But I’m dealing with a TV audience. So I guess what I’m asking is, why is this exciting? Give me just a one-line, two-line, why is this so exciting? Why should people be excited.

FRANCIS COLLINS: This is unprecedented partnership bringing the best and brightest scientists from the public and private sectors together to discover the next generation of drug targets that are going to transform our ability to treat Alzheimer’s disease, diabetes, rheumatoid arthritis and lupus. And that’s just getting started.

VAL: Thank you.

JOHN BURKLOW: Thank you, Val. Nell. Nell from NPR.

NELL GREENFIELD BOYCE: Nell Greenfield Boyce from NPR. So you’ve called this an unprecedented consortium. But doesn’t the Foundation for NIH have other public/private partnerships like the Biomarkers Consortium? So I’m just wondering what about this is different. What do you see is the key features of this that make it unique.

FRANCIS COLLINS: So, yes, FNIH is here in the room and very much have been involved in other kinds of industry consortia. You mentioned the Biomarkers Consortium. There are others on Alzheimer’s disease, ADNI, Alzheimer’s Disease Neuroimaging Initiative. But this has a different set of goals. The goal here is really to look at this wonderful deluge of information that’s coming out of the omics revolution in basic science and trying to sift through that very large dataset of opportunities and identify which of these new insights would point to the next drug target that a company might want to make a billion dollar bet on to bring it forward to market.

The Biomarker Consortium is looking, as it says, at biomarkers. In the Alzheimer's part of AMP there is a biomarkers emphasis but it is actually extending considerably beyond anything that has been previously contemplated bringing together biomarkers from various different sources and making sure that every Alzheimer's clinical trial gets every biomarker you can think of tested — so that you can begin to assess when you try a new therapeutic, what are the best indicators about whether it’s working or not.

But much of what AMP is doing is really more focused on identifying the molecule that undergird these diseases — and which of those might turn out to be the pathway, the node in that pathway that you believe if you developed a drug — that was an agonist or an antagonist, it would be helpful for people with that disease. To make the bets so they turn out right. To try to do something about the terribly disappointing failures that happen in phase II or III where you develop a drug, it turns out to be safe but it doesn’t work for that disease — apparently because it aimed at a target, which ultimately didn’t turn out to be the right one for treating that condition.

So much of this is very much in the front end of that long pipeline, but trying to be sure, if you want the pipeline to work, it better work at the beginning because otherwise you’ll get to the end and be very disappointed.

Mikael, maybe you should say, also, if you want to, add to that, why is this different and why is this not the same.

MIKAEL DOLSTEN: I think it is a more comprehensive approach, really coming together and prioritizing diseases where we think that science is evolving and patients’ needs are pressing. It’s a feeling of opportunities, and the challenges are the bottlenecks of really opening up translational, basic science to impact human medicine. It is the breadth of this many companies, NIH and academia, really going to the core of changing outcome of some very dramatic diseases.

And I think it is very timely. And I’ll ask other colleagues to chip in. But I have a considerable enthusiasm that this is unique, it is complementary and we should aspire high here.

JOHN BURKLOW: Thank you. Jocelyn.

JOCELYN (Science Magazine): I wondered if you could talk about the specifics of what the companies will bring to one of these projects versus what the academics will bring, like maybe the autoimmune diseases? Will companies contribute tissue samples? Or what are the two sides bringing to it?

FRANCIS COLLINS: So each one of these projects was designed over the course of about a year, with scientists from both sectors sitting down together to figure out, where is the scientific opportunity and what resources are necessary to make it happen. And each project is different in terms of the approach that it’s taking. You asked about the autoimmune diseases. The team that worked on this identified particularly the need to be able to understand what’s happening to the immune system and particularly what’s happening in relevant tissues. — so, for instance, the tissue that lines the joints in rheumatoid arthritis, called the synovium.

And not just to do this in bulk but actually to apply very, brand new and exciting technologies that allow you to look at single cells and to understand what’s happening in those single cells in the involved tissues — as a way of providing a much more precise amount of insight into the molecular basis of the disease — which in turn ought to point you toward potential ways of intervening by developing new drugs that target those pathways that are still poorly understood.

So that particular program, the companies and NIH are banding together, basically, to come up with the funds to support this kind of exploration in a very high tech way — about what happens in these diseases to the immune system in a single cell basis. And they will be working together, both to acquire the samples and to do the analysis to figure out what all this means.

The Alzheimer's effort particularly has two components. One is to try to make sure that all the clinical trials that are being conducted by NIH and several by industry also are, in fact, decorated with the complete suite of biomarkers that you would like to have included in order to assess whether that therapeutic intervention is working.

But the Alzheimer's proposal has another interesting piece to it, which is to do very careful systems biology analyses of the brain in Alzheimer's compared to normal. And there are such brain tissues available. They’ve never been explored in quite this way. That, again, we think should give us insights into pathways involved in Alzheimer's, getting beyond what we already know about amyloid and tau and a few other things into other spaces that may well be drugable.

The diabetes effort, a big part of it is to construct what is being called a knowledge portal. And the idea here is to take this wonderful proliferation of information about type 2 diabetes, which comes from genome wide association studies, now having identified about 80 areas in the genome that carry risk factors for this disease — but also a lot of other omics information about gene expression, about the epigenome and what happens to it in the pancreatic islet or in the muscle or in adipose or in the liver — and bring this all together in a fashion that you can make sense out of it.

Because all of us interested in this are excited but a little daunted and overwhelmed by the complexity of the data and the need to bring it into this knowledge portal together with patient information about phenotypes is something that no group can do alone. And this AMP enterprise can manage this in, I think, a very exciting way.

Those are just bits and pieces of what, in every instance, is a study design that goes on for 30 or 40 pages for each of these three projects that’s been very carefully designed. Mikael.

MIKAEL DOLSTEN: I also wanted to add, Francis alluded so well to the hardware that we’re bringing in and its complementary skills. Technology has access to patient cohorts where we have genotypes and phenotypes. So it is a really a rich collection that never has been brought together.

But also mention a little bit importance of the software to create these kind of diverse dream teams, which really are necessary rather than working more in isolated domains. And I think we want really to build a new collaborative culture, which is necessary if we are going to first come up with the right targets and find treatment solution as Shanelle Gabriel challenged us to do sooner rather than later. And I think this is a very important part of starting the journey with the idea of having joint teams across companies with NIH and academia. And that could really set the course for a different path in the future. And I think it is important for the patients.

We hope also, obviously, to create medicines that will have an impact on health and reduce healthcare spend overall by bringing patients back to full activity. And I do hope it will also increase the competitiveness of United States in the life science sector, in which many different countries and continents are starting to participate. And we have been a leader and I would love to see that we continue to be leader. And to be a leader you need to create the right culture. And that’s what we are doing today.

JOHN BURKLOW: Shanelle, if you would like to —

SHANELLE GABRIEL: I would say — I mean I think that for me, personally, lupus affects 1.5 million Americans every year. And that number is probably very dated and is not probably accurate because there are so many people who are misdiagnosed. And the idea of just being able to look for things, the idea of this collaboration where so many people are coming together to help me and to help you, my friend right there, and all the people that are out there that are affected by these diseases is just an amazing feat. And I can say that I’m impressed and I’m excited for you all. And I wish I knew science so I could be a part of it.

JOHN BURKLOW: Thanks, Shanelle.

__: You are part of it.

FRANCIS COLLINS: The most important part.

JOHN BURKLOW: Okay. Any other questions? If not — okay. Thank you for joining today’s briefing. As Dr. Collins said, we have a number of guests today and they will all be happy to stick around if you would like to do one-on-one interviews. And thanks again and have a good day.


This page last reviewed on November 3, 2017