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Brainstorm Health Daily: April 4, 2017

April 4, 2017, 6:43 PM UTC

Few words in the English language conjure up as much angst as the word “biopsy.” The term evokes not only the fear of a sudden life-changing diagnosis (yes, there’s that), but also the image of a knife: A biopsy, after all, often involves cutting into a person.

But what if it didn’t? What if instead of having to extract tissue from your body to diagnose a disease, a doctor could find out more precise and more comprehensive information—and get it much faster—by taking a small sample of blood? That is the thinking behind the elusive “liquid biopsy”—a concept whose theoretical simplicity has long been sneered at, scoffed at, and had sand kicked in its face by the rude complexity of the challenge.

Sure, physicians already have plenty of blood tests to aid in the diagnosis and prognosis of various cancers, as they do with gobs of other diseases. But for the most part, the proteins measured today (PSA, CA-125, HER2, for examples), often don’t provide enough of the answers—or specific enough answers—needed to make an informed treatment decision.

So, for the past two decades or so, researchers have tried to see if they can get better information from blood-borne snippets of DNA. Could telltale mutations, patterns, or other such “fingerprints” in the blood tell us not only whether or not a person has cancer, but also the exact subtype, where it is, how far it has progressed, the prognosis, and whether or not the disease is likely to respond to certain drugs?

At last, it seems, we may be getting tantalizingly close to those goals. This week, at the humongous (20,000-plus attendee) annual meeting of the American Association for Cancer Research, there were no less than 130 presentations, “poster” sessions, and panels on liquid biopsy studies—with research groups revealing their progress in efforts to detect, amplify, decode, and (most importantly) identify meaningful information from blood-circulating tumor cells (CTCs), other fragments of DNA (cfDNA) and tumor DNA (ctDNA) floating outside the confines of cells, exosomes (tiny sacs that are, somewhat mysteriously, released from cells), and microRNA.

Much of the progress, it’s worth noting, has been made possible by a remarkable collaboration begun in 1991, christened the International Liquid Biopsy Initiative Team, which has helped propel the science forward, thanks to a number of technological breakthroughs. And that team, now led by Luis A. Diaz, at New York’s Memorial Sloan Kettering Cancer Center, got some justifiable praise at the AACR’s opening plenary session on Sunday.

“These people stuck together as a team for over 25 years,” Pat LoRusso, a medical oncologist at the Yale Cancer Center, told me after presenting the AACR’s “Team Science” Award to the group. “[Guided by legendary Johns Hopkins researcher Bert Vogelstein], they first identified that you could find somatic mutations in stool.” (Though, I should point out, it wasn’t until decades later, in 2014, that the FDA approved the first noninvasive DNA-based screening test for colorectal cancer.) Then DNA signatures were revealed in the urine sediment (cells and debris from the urinary tract) of patients with bladder cancer.

“And they kept saying, ‘Let’s go one step beyond that,’” says LoRusso.

Such is the poetry of progress: Let’s go one step beyond that.

Amen. Sy has the day’s news below.

Clifton Leaf, Editor in Chief, FORTUNE


Digital health funding has had a record-breaking first quarter. Digital health venture funding is off to a historic start in 2017, according to a new report from StartUp Health. The group, which describes its mission as "organizing and investing in a global army of thousands of entrepreneurs passionate about reinventing the future of health," finds there were $2.5 billion of deals in the first quarter of 2017, including a blockbuster $914 million Series B round for GRAIL - one of the companies chasing the exciting prospect of a true "liquid biopsy" (which Cliff discusses extensively in his essay today) in which cancer can be detected via a simple blood test, with an assist from high-intensity genomic sequencing. A few other notable trends? Seed funding rounds in digital health are getting smaller while Series As are ballooning, and although the number of deals in Q1 2017 hit a six-year low, the amount of money being invested per deal has been increasing, with $500 million to $900 million transactions becoming more commonplace. Some of the biggest funding of 2017 to date (besides the GRAIL mega-round): $115 million raised by population health firm Alignment Healthcare, $100 million by PatientsLikeMe, and $90 million by Nuna, which is attempting to create a unified Medicaid dataset that can be more effectively analyzed. Check out the full report here.

Will Zocdoc be able to hit its ambitious financial targets this year? The doctor database platform Zocdoc may be significantly behind when it comes to reaching ambitious financial targets laid out over the last several years, according to The Information. The publication says it was able to get its hands on internal Zocdoc documents and that the figures show it would be extremely difficult to achieve the aggressive revenue growth the company, which raised $130 million at a $1.8 billion valuation, has boasted, especially since a number of businesses have yet to launch. Zocdoc said it wouldn't comment on financial figures and that its growth was still robust. The digital health firm has gone from signing up just doctors, who pay to list on the service, to striking deals with hospitals as a growth strategy. (The Information)


Paratek rockets up on antibiotic data. Shares of biotech Paratek Pharmaceuticals soared more than 30% in early Tuesday trading as the firm heralded late-stage clinical trial results for an experimental antibiotic in treating patients with hospital-acquired pneumonia. The treatment, omadacycline, was shown to be about as effective as a different antibiotic called moxifloxacin three to five days after the first dose, and actual elicited a stronger response than that treatment five to 10 days after treatment ended. Paratek has been on a bit of a roll recently; just last week, an acne drug that it's developing with partner Allergan showed promise when compared to placebo. (Boston Business Journal)

A hit and a miss for Bristol-Myers' cancer immunotherapy Opdivo. Bristol-Myers Squibb got one piece of good news and one not-so-great tidbit for its new flagship immunotherapy treatment Opdivo during an ongoing cancer conference. First, the good news: a combination of Opdivo with an older BMS immunotherapy called Yervoy helped severe melanoma patients live longer. In fact, 64% of patients on the combo treatment were alive two years after treatment began, compared with 59% of trial participants treated with Opdivo alone. Now for the less-stellar part: Opdivo didn't fare so well in a late-stage study in brain cancer, in which it failed to best another drug called Avastin. Still, going forward, Bristol-Myers and other drug makers will continue attempting combinations of their new, so-called "checkpoint inhibitor" drugs with other immunotherapies in an effort to make them more effective for a wider population of patients - and, not to mention, to bring in higher sales since the two-drug cocktails would be more expensive. (Reuters)

Mallinckrodt settles opioid probe with $35 million. Pharma giant Mallinckrodt will pay the federal government $35 million to settle a probe centering on the firm's distribution practices for both generic and brand name opioid painkillers. The deal, which doesn't come with an admission of guilt, was struck with the Drug Enforcement Agency and a pair of U.S. attorneys. Drug makers that sell opioids have been under the gun in the wake of the national opioid epidemic, which some lawmakers and critics have said the biopharma industry is in part responsible for thanks to aggressive marketing practices. (Salt Lake Tribune)


Kansas legislators narrowly fail to overturn governor's veto of Medicaid expansion. The deeply Republican Kansas legislature fell just three votes shy of a needed supermajority in a bid to overturn Governor Sam Brownback's veto of a bill that would have significantly expanded the state's Medicaid program for the poor under Obamacare. Hospitals, particularly rural providers, and patient advocates had lobbied hard for the Medicaid expansion amid uncertainty whether or not the program will continue to exist the Trump administration and Congress attempt to dismantle the health law. Had the bill passed, it was expected to provide government health benefits to about 150,000 low-income Kansas residents. (New York Times)

Biomedical research cuts face stiff opposition in Congress. President Trump's proposal to cut biomedical research funding by some 18% is drawing some bipartisan criticism in Congress. "I’m extremely concerned about the potential impact of the 18% cut," said Oklahoma Congressman Tom Cole, the Republican who chairs a powerful House Appropriations subcommittee in charge of National Institutes of Health (NIH) funding. "This committee and certainly me, personally, will be very hesitant." The Trump administration has proposed a $5.8 billion cut to the NIH, as well as $1.2 billion in funding cuts this very year that would stem mostly from research grants. Former Vice President Joe Biden slammed the proposal on Monday in a speech during an ongoing cancer conference in Washington, D.C. - a cause near and dear to Biden's heart, especially since the death of his son Beau Biden from brain cancer. "This is not the time to undercut progress, for God's sake," said Biden in his speech. "This is the time to double down."

Trumpcare may not be dead. The word on Capitol Hill is that President Donald Trump and Congressional conservatives are trying to figure out a way of taking a second stab at Trumpcare following the legislative crash-and-burn of the American Health Care Act (AHCA). Provisions being discussed with the hardline House Freedom Caucus would push the Obamacare dismantling drive even further to the right, allowing states to opt out of a pair of measures that keep premiums in check for sicker, older, and poorer Americans: "community rating" that doesn't allow insurers to charge you more based on your medical neediness and "essential health benefits" that force insurance companies to cover certain services such as prescription drug coverage and maternity care. I'll have more on what making these Obamacare provisions optional might do the individual insurance market later today. (Fortune)


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Produced by Sy Mukherjee

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