Today of course is Presidents' Day -- a perfect time to talk about the new initiative that current President Barack Obama recently unveiled to boost research that leads to better treatments (maybe even cures!) for conditions like cancer and diabetes.
During his State of the Union address in January, President Obama briefly mentioned the Precision Medicine Initiative, a $215 million undertaking included in the country's federal budget for 2016. It's aimed at "pioneering a new model of patient-powered research that promises to accelerate biomedical discoveries and provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients."
Basically, the idea is to use all the medical and tech know-how we have to explore an individual's genetic makeup and figure out what treatments might best suit them -- and which could eventually lead to curing them and others with the same condition.
The capability to "deliver the right treatments at the right time, every time, to the right person" could mean an entirely new era of medicine, the President says. "We shouldn't just celebrate innovation, we must invest, nurture, encourage innovation. Make sure we're challenging it in ways that are most productive... that we're harnessing what's most special about America -- our ability to innovate."
Wow! Of course the notion of personalized medicine and genetic-mapping isn't entirely new; efforts have been underway globally for a long time. But what's new is the President's commitment to taking this to the next level.
Ten days after his address, President Obama expanded on the initiative and released more information about what's happening.
There's a comprehensive fact sheet with details on this initiative, along with an arsenal of information from White House blog posts, mainstream media coverage, and this insightful op-ed piece in the New England Journal of Medicine from National Health Institutes (NIH) Director Francis Collins.These are some highlights that caught our eye:
- $130 million to NIH for development of a voluntary national research cohort of a million or more volunteers to propel our understanding of health and disease and set the foundation for a new way of doing research through engaged participants and open, responsible data-sharing.
- $70 million to the National Cancer Institute (NCI), part of NIH, to scale up efforts to identify genomic drivers in cancer and apply that knowledge in the development of more effective approaches to cancer treatment.
- $10 million to FDA to acquire additional expertise and advance the development of high-quality, curated databases to support the regulatory structure needed to advance innovation in precision medicine and protect public health.
- $5 million to ONC to support the development of interoperability standards and requirements that address privacy and enable secure exchange of data across systems.
We also tuned into the Jan. 30 news conference at the White House, where the President spoke more on this. And while most of it was pretty general in terms of conditions and the research, we did hear President Obama mention diabetes twice -- once in general about curing D and other conditions, and another time on tracking vital stats like blood sugars with wearable technology. Very cool!
A Diabetes Advocate in D.C.
It's exciting to note that one of our Diabetes Community's advocates was right there in the audience, one of about two dozen patient advocates chosen to attend the news conference and also a networking meet-and-greet reception. Yes, our own Anna McCollister-Slipp, a longtime type 1, data entrepreneur, and FDA patient advocate was right there listening to the president speak! She got to hang with NIH Director Francis Collins (mentioned above), and here's what Anna tells us about the experience and what she thinks about this initiative:
I’m a pretty cynical Washingtonian. Not much impresses me or gets me excited anymore, but it’s not every day that you get invited to an event at the White House with the President. It was an incredible honor to be included. I don’t live all that far from the White House, so I drive or walk near there pretty frequently. The building becomes part of the backdrop of life in DC. But getting an invitation to attend a Presidential event -- especially one on a topic so important to me as this -- is a rare experience.
The speech was given from the East Room in the White House, and there was a reception prior to the speech in a large, marble foyer outside of the room. Just being in the building and walking through the halls, thinking about the decisions that have been weighed and made in those corridors is humbling. (I’m guessing there were about 100 people in attendance, along with media, camera crews, etc., but I’m not good at estimating crowds.)
Not only was it fun to be part of the event, but I was amidst some of our greatest minds in research and medicine, including top officials from FDA, HHS, NIH, the White House and ONC, as well as some of the most important and visionary patient advocates and leaders from around the country who have been instrumental in pushing the need for more precise approaches to understanding and treating disease. There was a real sense of enthusiasm and an odd sense of accomplishment that permeated the room, that as a community of patients, scientists, policymakers, researchers and physicians that we collectively have played our own little part in bringing us to a point where we are perched at the dawn of a new era of health and medicine.
I was among about 20-25 patient advocates invited to attend the event from around the country, representing an array of perspectives, conditions and expertise. It was encouraging to see the emphasis on including patients as a central part of the event and the process. It was a true reflection of this Administration’s commitment to recognizing the significant contributions that patients can make to driving research and policy changes most important to those who live with disease. I’ve seen that attitude reflected in the health IT policy advocacy that I’ve been able to do in the past few years, and it was very encouraging to see the patient focus in this event, as well. The fact that this President and his team decided to make that a central component of this announcement speaks volumes about how attitudes are changing about the role that patients can play in better understanding, treating and curing disease.
I don’t know how they compiled the list of those invited, but I presume I was included based on some of the advocacy that I’ve done as a frustrated-patient-turned-digital-health-entrepreneur over the past several years on digital health/devices/outcomes measures, CER, etc. In addition to my company (Galileo Analytics) and the volunteer advocacy that I do, I’ve recently signed on with the Scripps Translational Science Institute to help create a new program for involving patients in the design of clinical research in digital health and genomics. The need for precision medicine has been a driving force behind all of the advocacy work I’ve done relating to digital health, device innovation, outcomes measures/comparative effectiveness research and patient participation in research, so it was particularly exciting to see all of these elements come together in the form of a White House initiative to help focus the resources of the government to drive the next leap in medical understanding and treatment. It was a true honor to be included.
Anna with NIH Director Francis Collins
What it Means for PWDs
I’m not sure it’s clear at this point what this new initiative will mean for diabetes patients. There is a significant focus on genomics, but the White House has also said they want to include digital data and device data as part of the research efforts. This is something I have been emphasizing as important to those of us with diabetes, since we currently generate large volumes of data that ultimately goes to waste. Not only have we had a hard time getting access to our data for our own use, but we have no way of donating that data for use by researchers who might be able to find important patterns that could be used to develop new or better methods of treatment and care.
Most of the discussions around personalized medicine have focused on genetics. This is important. There are genetic underpinnings of diabetes, as well as the development of diabetes complications, and we are not immune to cancer and the many other tragic diseases for which genetic mutations have been identified as a cause. However, the interplay between the genome, exome, metabolites, epigenetics, etc. is incredibly complicated. I’ve long argued that it’s important to work with what we have in front of us, in structured, understandable, machine-readable data streams while we figure out the difficult science of the ‘-omics’. Let’s start with the ‘low-hanging fruit’ of medical device data, which is currently being wasted. We can get much further than we are today if we fully utilize the incredible data resources we already have, that each of us generates every day.
Another central element of the initiative is to recruit a million-person patient cohort, aimed at collecting a range of biological and device data streams. The objective is to integrate these data sources and organize the data in such a way that researchers can access and analyze connections, discover new patterns and hopefully find better ways to treat and cure disease. As a member of the #DOC, I’ve made it very clear that those of us in the diabetes patient community will be eager to participate, and I’m doing what I can to make sure that we do get included. They’re still in the initial phases of figuring out how to bring all of this together. As I learn more about how we can participate, I’ll be sure to let the 'Mine and larger D-community know and keep them involved in the process.
How Big is This?
This is kind of medicine’s version of the “We choose to go to the Moon” speech. We don’t yet understand how we will get there, but everybody in the room that day seemed to grasp the fact that we really are perched on the edge of a true revolution in health and medicine. This is the kind of effort that only government can do. The likelihood of precision treatments hitting the market is too far down the road at this point for industry to play a role. It takes the President’s unique ability to marshal the forces of government to make the leap that we need to make. But, as we have learned from the human space flight initiative of the 60s and the human genome project of the 90s, that investment yields incredible dividends.
In sum, this is going to be difficult. If it were easy, we would already be there by now. But you don’t need a Presidential Initiative to take on the easy stuff.
We were also thrilled to connect JDRF Chief Medical Officer Dr. Richard Insel, to hear what he and the JDRF think about this new initiative. Dr. Insel tells us he's excited to see the U.S. moving forward on this area of precision medicine, especially since other parts of the world like the UK have been pushing this for years.
But as of now, Insel echoes what Anna says about how the direct impact on diabetes research is still TBD; he says JDRF will certainly be talking with NIH about that.
Insel says that over the past decade, JDRF has already invested a "fair amount of money" to genetic risk factors and has identified 50+ genes associated with this condition. And now, that's used in screening babies and children for their type 1 risk, following them over time to examine the natural pathway of diabetes.
"This is an example of precision medicine, at least the beginnings, in understanding genome type and the genetic factors in type 1," he says.
But it's an especially critical moment now for precision medicine to come more into the picture, he says, "because we've come to understand that type 1 isn't a single disease -- it's more like a syndrome and it changes or varies so much depending on someone's age of diagnosis, how their beta cells may be functioning, and what envionrmental issues may be at play."
JDRF's ongoing work address this, with efforts like trying to classify the early stages of type 1, and the Feb.12 announcement that JnJ-owned Janssen Research & Development would be investing in "disease interception" to find ways to thwart type 1 using new diagnostic and pre-disease intervention tools.
Insel also said the JDRF is co-coordinating a public-private partnership in Europe led by Sanofi, addressing how genome-typing is used to study diseases and develop biomarkers in order to create faster, more responsive treatments for type 1. More on that should be rolled out formally in September, he said, and it's all a part of this changing tide in research related to precision medicine.
"This is all important, and we're very excited," Insel said. "We're building on genomic research that we've been developing, and all of this will help us better characterize type 1 diabetes."