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NIH Striving to Avoid False Hope in Chronic Fatigue

NIH Striving to Avoid False Hope in Chronic Fatigue

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BETHESDA, Md. — The National Institutes of Health is trying hard to bring real hope — not false hope — to patients with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), NIH director Francis Collins, MD, PhD, said during an exclusive interview with MedPage Today.

“Five years ago … there was this big excitement that there was a retrovirus that was turning up in people with CFS, and initially it didn’t appear to be in people who were unaffected,” Collins said Friday during an interview at the NIH main campus, at which a communications staff member was present. “It was a funny retrovirus that had only been seen in cultured cells in the lab, and maybe in mice, [and it was] called XMRV.”

“It was really exciting because it was a retrovirus, and we have drugs for retroviruses! It got published in Science to great excitement, but then almost immediately other researchers looking at very similar patients said, ‘We don’t see this’ or maybe ‘We see it in people who don’t have the disease,'” he continued. “We mounted a careful multi-site study funded by NIH with blinded samples to try to see [whether] this [held] up and sadly, it did not. That was such a blow to a community of suffering people who thought, ‘Finally, they’re on to something.'”

Collins said he has been “greatly moved and troubled” by the stories of CFS patients, “especially when many of those stories start with people who are highly active, and many of them fairly athletic, and some illness that sounds viral, like a really bad flu, hits them, but they don’t get better … Many of them are unable to resume normal activities and end up bedridden for months or years.”

In September, NIH awarded $7 million in grants to three clinical centers and a data coordination center to continue ME/CFS research. “Some people have said, ‘Your three centers are all doing the same things,'” he added. “Yeah, that’s intentional; we want to see immediately if something looks like it’s promising, is it promising really, or another false positive? We’ve had too many of those and we don’t want to make that mistake again.”

In addition, “I moved the program out of the place it was at NIH, which was at the Office of Research on Women’s Health,” he said. “It is true that women are affected more than men, but that was seen as not a particularly important place for an important disease to be.”

Collins asked Walter Koroshetz, MD, director of the National Institute of Neurological Disorders and Stroke (NINDS), to head up the NIH’s research effort, and NINDS senior investigator Avi Nath, MD, “to start a protocol in our clinical center to bring people with this disease for an intense 1 or 2 weeks of just looking at every possible cause. And that’s led to a big uptick in the amount of research that’s going on.”

Although the medical community has been criticized for not taking ME/CFS seriously, “it’s very hard for me to see how [that criticism is] fair when you hear stories of people who’ve gone rather suddenly from a full life to bedridden status — something dramatic happened there,” said Collins.

He added, however, that “there are problems [in that] CFS has become such a blurry diagnosis, that in there amongst hundreds of thousands or millions of people who carry that diagnosis is a whole heterogeneous group and there may be individuals … who have something else entirely or even people who are suffering from depression and are therefore feeling fatigue for that [reason]. I think that’s added to the difficulty that the medical care system has had coming to grips with this as a real disease that has a desperate need for new treatments.”

Thinking Big on Cancer Cures

Collins also expressed excitement over the additional funding — granted under the 21st Century Cures Act — that his agency will have for the “cancer moonshot” project. “We’re thrilled to have the opportunity to push this even faster,” he said. “It builds upon a foundation of cancer research that’s been going on for a long time … [It gives us] about $1.6 billion over 6-7 years to add to what was already available for cancer research.”

What is NIH doing with that money? “We convened 2 years ago a group of highly expert cancer research visionaries — from academia, the private sector, and advocacy [groups] — and said, ‘OK, guys, what do we need to do that we’re not already doing? Think big, think bold; don’t worry about risky projects if they can pay off,'” he explained. “They provided a blueprint of where we needed to go, a series of about 28 initiatives. Those have been our guiding documents to crank this up.”

Immunotherapy is one of the big areas the researchers are focusing on, he continued. “You can’t help but look at some of the amazing success stories of cancer immunotherapy of people who had widely metastatic disease and are now cured without going, ‘Wow, we are really onto something!’ — after all of these years of trying to figure out whether this could work. For leukemias that used to be refractory, and with lymphomas that failed chemotherapy, immunotherapy is looking really good.”

“The challenge is, how do we take those successes and expand the success rate to solid tumors where this just really hasn’t paid off yet — pancreatic cancer, colon cancer that’s already metastasized, breast cancer, prostate cancer?” Collins said. “But cancers are very clever and they hide their abnormal proteins, telling the immune system, ‘There’s nothing to see here.'”

“So we have to help the immune system recognize trouble, and that’s led to a host of these really dramatic new technologies like CAR-T cells, now approved for two different applications by FDA but still not yet for solid tumors; that’s the big frontier. But it ought to be possible to do that … and that’s where a lot of the moonshot money is going.”

Working with Commercial Partners

One way to speed up these therapies is partnership with industry, he said, “but they have the same frustrations about why it doesn’t work … So we need to have a better biological understanding of that process, basically biomarkers that will be predictive of whether a particular immunotherapy is going to be successful or not. We don’t have those.”

“After talking to industries that are most engaged over the course of more than a year, this has developed into a formal partnership: the Partnership for Accelerating Cancer Therapies, in which 11 companies all agreed to take part and contribute $5 million each … so they’re putting money on the table, and partnering with NIH with a very explicit set of goals,” Collins said. That partnership launched in October.

As great as some of the new therapies are, sometimes they stop working after 9-12 months, so researchers want to know “What’s the mechanism of resistance, and how can we avoid it?” said Collins. “Do we need to do the same thing we do with HIV or tuberculosis — hit them with two or three drugs at once instead of one at a time? Cancer may have that same propensity to develop resistance if you don’t have that full army at work, so combination therapy is very much of interest as well.”

As for the word “moonshot,” “It is helpful to have something that people can immediately identify — ‘Oh, that’s what you’re talking about!'” he said. “And if it sounds inspiring, exciting, promising, that’s even better. There were discussions about whether that’s the best label, but that’s what [former Vice President Joe Biden] wanted, and it’s in the legislation, so I guess we’re going to stick with it.”

Precision Medicine … for All of Us

Another term that gets a lot of discussion at NIH is “precision medicine.” Collins recalled that he wrote a book in 2010 called The Language of Life: DNA and the Revolution in Personalized Medicine. “That was the term I was using at that point.”

“Then there was a National Academies [report] that looked at the promise of all this,” he said. “They didn’t like ‘personalized medicine’ because they thought it sounded like every decision, every drug is going to be just for that one person, as opposed to recognizing that if you take a million people, maybe for 50,000 of them this is going to work and 30,000 need something else. So … they liked ‘precision’ better. That’s the idea of taking a one-size-fits-all approach, which most of medicine has been, and using all the data available to be more precise about what’s going to work for that particular person.”

“Ultimately, of course, one wants it to mean that all of us have a perfectly designed program for preventing illness that we can follow and it will keep us healthy, and if we’re still unlucky enough to get sick, there’s a precise intervention available that’s going to make us better,” Collins said. “And that is the goal — I will not step away from that goal, but we’re certainly not at a point where we can claim that’s the case.”

However, because the current initiative involves a nationwide program that the NIH is trying to get a million people to join, “I don’t think we want to call it the ‘Precision Medicine Initiative’ — we’ve got to have a name that’s more descriptive of what we’re trying to do, [which] is to learn as much as we can from as many willing partners as we can. [So the name is] ‘All of Us,’ and I think we’ve done a good job of branding it that way.”

“A secondary meaning [is] ‘all about us’ in terms of of our medical experiences, environmental exposures, diet, exercise, things we care about, and things we don’t care about,” he continued. “We want those million people to feel like they have embraced this program, that they want it to succeed … that they’re going to get a lot of information back about themselves, and that they’re excited to be part of this national adventure.”

Collins is hoping that the results of the initiative won’t take decades to implement in practice. “One thing that will help here is that at least half of the enrollees, as we currently imagine it, are going to be involved in health provider organizations that are both helping them engage in the research but are also their caregivers.”

“That setup will be particularly relevant as far as getting access to electronic health records, blood samples, their history of medications, and so on, because they have those systems in place,” he said. “But we want everybody in the United States to be able to take part if they want to, so there’s also a direct volunteer pathway for somebody who doesn’t happen to be in one of those health provider organizations, to call an 800 number, or go to the Web and sign up and also get involved.”

“We are very determined to make this a highly diverse million people, in terms of age, gender, geography, race, ethnicity, and socioeconomic status — we want this to be the kind of view of the nation that will also teach us things about health disparities.”

Part of that work involves encouraging minorities to participate, which has been an issue in the past, Collins said. “We’ve worked really hard on trying to understand the reasons why that has been the case, and how this project could be presented in a way that is different.”

“We’ve brought on board a chief engagement officer, Dara [Richardson-Heron, MD], an African-American woman, and she has been fantastically wise about how to build those relationships in a trusting way, and recognizing this doesn’t have an easy history and we need to be totally aware of that,” he said. Part of the outreach involves working with community health centers, whose patients are typically of lower socioeconomic status, to encourage those patients to participate.

To ensure that the All of Us project will launch properly, NIH has been beta testing it since last May, Collins noted. “We’ve enrolled over 10,000 people as test subjects to see whether the questionnaires, the access to electronic health records, the security systems, the blood samples — whether everything is moving the way it should as we bring online all these enrollment centers all over the country … and it’s looking really good.” As for a launch date, “We’re saying in the spring — maybe by next month we’ll be at the point of being able to settle on a [specific] date.”

Making sure the participants reap the benefits from the study — especially if it results in high-priced treatments — is an issue, he said. “We can’t, with this one study, solve [the problems of] our healthcare system.”

Collins noted that a New York Times article pointed out that Congress seems interested in supporting research studies but can’t agree on how to make sure people have access to healthcare. “We can’t solve that but we can certainly try to provide for people to have access to this study, which is going to give them information back about themselves.”


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