Why one analyst thinks a Sanofi and Regeneron cholesterol drug will be yanked

first_img Expert view: New guidelines on cholesterol drugs are flawed About the Author Reprints @Pharmalot Earlier this week, Gbola Amusa of Chardan Capital Markets released an investor note in which he took a contrarian view. He wrote that the Street is underestimating the likelihood of a permanent injunction being issued and predicted that it is “reasonably likely” that Sanofi and Regeneron’s Praluent will be taken off the market later this year.As he views it, “when patent owners win (patent) infringement suits against products that are their sole competitors … such dynamics have historically led to a permanent injunction.” Since Praluent is the only current competitor to Amgen’s Repatha in this market, “precedents suggest that Praluent, too, will be removed from the US market.”Moreover, he believes Sanofi and Regeneron will not win a stay — or a halt in the proceedings — while the companies simultaneously appeal last week’s verdict. Most Wall Streeters, however, believe the drug makers will win a stay so that the appeals process can be allowed to play out. And the appeals process is likely to take a year or more, which means Praluent will, meanwhile, remain available. Please enter a valid email address. Leave this field empty if you’re human: Amusa, though, writes that most investors “could be disappointed.” With a decision on a stay for a permanent injunction expected by May or June, he believes it is “possible that Praluent could come off the shelves in the US soon after.” And his outlook is boosted thanks to flubs that Sanofi has committed in the past with filing patents, he added.As Amusa noted, the outcome of the hearing that began today will not be known for several weeks. But if he is proven to be correct, Amgen will be a big winner, especially since the company will soon face stiff competition for its key products from biosimilars. But another winner will be Amusa. Then again, he could also be one of the losers. Related: A court hearing gets under way today that one Wall Street analyst believes may significantly alter the fortunes of three big drug makers in the outsized market for cholesterol treatments.Amgen is seeking a permanent injunction to prevent Sanofi and Regeneron Pharmaceuticals from selling their injectable cholesterol drug called Praluent. The move comes after a federal court jury last week upheld two Amgen patents on its own Repatha medication in response to a patent infringement lawsuit filed in late 2014. Both drugs, known as PCSK9 inhibitors, debuted several months ago to considerable fanfare because they can lower cholesterol considerably. Sales for these drugs, however, have gotten off to a lower-than-expected start because insurers have pushed back on the cost — list prices for both injectables exceed $14,000 annually. Nonetheless, eagerly awaited cardiovascular outcomes data is expected to be released over the next year or so, and if the results are positive, the drugs could become important products for each of the companies.advertisement [email protected] Ed Silverman Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. PharmalotWhy one analyst thinks a Sanofi and Regeneron cholesterol drug will be yanked This explains why the patent dispute has been closely watched. Wall Street, however, is largely discounting the likelihood that Amgen will win a permanent injunction and looks for a settlement to be reached that will involve Amgen receiving a royalty payment on sales. Leerink analyst Geoffrey Porges, for instance, believes such an injunction is a “remote possibility.”But one analyst thinks otherwise.advertisement A cholesterol drug from Sanofi and Regeneron could be taken off the shelves in the coming months as the result of a patent fight with Amgen, an analyst says. Jacques Brinon/AP By Ed Silverman March 23, 2016 Reprints Newsletters Sign up for Weekend Reads Our top picks for great reads, delivered to your inbox each weekend. Tags AmgencholesterolRegeneron Pharmaceuticals Privacy Policylast_img read more

Watch: Episode 1: Could playing a dolphin in a video game help stroke patients recover?

first_imgI recently visited Krakauer and his colleagues at their lab in Baltimore to see what they were up to. Their research is the focus of the inaugural episode of “Science Happens,” a monthly video series in which I’ll be exploring labs and diving into the latest findings.If you’d like to suggest a lab where existing research is going on for a future episode of Science Happens, please drop me a note.advertisement Science Happens!Watch: Episode 1: Could playing a dolphin in a video game help stroke patients recover? By Carl Zimmer March 25, 2016 Reprints Episode 1: Could playing a dolphin in a video game help stroke patients recover?Volume 0%Press shift question mark to access a list of keyboard shortcutsKeyboard ShortcutsEnabledDisabledPlay/PauseSPACEIncrease Volume↑Decrease Volume↓Seek Forward→Seek Backward←Captions On/OffcFullscreen/Exit FullscreenfMute/UnmutemSeek %0-9 facebook twitter Email Linkhttps://www.statnews.com/2016/03/25/after-a-stroke-reteaching-the-brain-by-gaming/?jwsource=clCopied EmbedCopiedLive00:0004:5304:53  John Krakauer, a neuroscientist at Johns Hopkins University, has spent much of his career treating people with strokes. But he’s the first person to tell you that stroke rehab can be really lousy. Sometimes he likes to use the word medieval.It’s not that stroke rehab involves bleeding a patient or prescribing the eye of a newt. The problem is that current treatments don’t take into account the latest research on what happens to the brain during and after a stroke. Rehab is often focused on just a few survival skills, like holding a spoon to eat. Many stroke victims only make partial recoveries and are never the same again.Krakauer, the director of Johns Hopkins’s Center for the Study of Motor Learning and Brain Repair, wants to treat patients in a fundamentally different way: using video games to reteach the brain how to control the body.advertisementcenter_img This story originally published on Nov. 13, 2015. Tags brainneurologyrehabstroke In a new video series by STAT, Carl Zimmer takes viewers inside labs where promising research is taking place. In this episode, he visits Johns Hopkins University Medical Center where Dr. John Krakauer is treating stroke patients with a video game. Matthew Orr/STATlast_img read more

A serious stomach bug: Norovirus costs billions of dollars of lost productivity

first_imgQuick TakeA serious stomach bug: Norovirus costs billions of dollars of lost productivity By Ike Swetlitz April 26, 2016 Reprints Privacy Policy A norovirus particle. Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Read the most popular stories this hourBut keep in mind:When comparing the cost of norovirus to the cost of other diseases, it’s important to take into account how each of the numbers was derived, said Lee-Ann Jaykus, a professor at North Carolina State University and the scientific director of the NoroCORE project, a multi-institution collaboration in which two of this study’s authors are involved. There isn’t one correct way to interpret the numbers, she said.What’s next?More research is needed in order to develop and deploy a vaccine, Lee said. For example, norovirus is often underreported, making it difficult to know how to target interventions.The bottom line:There is still much work to be done, but this study indicates a norovirus vaccine could be a valuable public health investment. Perhaps best known for causing chaos on cruise ships and at Chipotle, the gastrointestinal disease norovirus may contribute to tens of billions of dollars of economic loss worldwide, a new study finds.Why it matters:There is currently no vaccine or treatment for norovirus, which sickens nearly 700 million people annually. Though some 200,000 people die of the virus annually, for the majority of sufferers it’s not that bad of an illness. However, researchers wanted to capture the lost productivity that days of sickness caused.The nitty gritty:Researchers used World Health Organization estimates and computer models to calculate the direct medical costs of norovirus, as well as economic losses that come from things like sick individuals taking time off from work. Those come out at about $4.2 billion and $60.3 billion, respectively, every year.advertisementcenter_img Please enter a valid email address. Charles D. Humphrey/CDC Leave this field empty if you’re human: “This suggests that there is a substantial need for a vaccine,” said Dr. Bruce Y. Lee, an associate professor of international health at the Johns Hopkins Bloomberg School of Public Health, and senior author on the study published Tuesday in PLoS ONE.Lee pointed out that since the costs associated with norovirus are so significant, a vaccine would not have to be perfect in order to make an impact.advertisement Tags norovirusviruseslast_img read more

At a hospice facility for children, a long goodbye is made a little less lonely

first_img Please enter a valid email address. For loved ones, scattering of ashes becomes a healing journey As the end nears, ‘death doulas’ ease the way EndnotesAt a hospice facility for children, a long goodbye is made a little less lonely Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. By Bob Tedeschi May 4, 2016 Reprints Inside the home, the boy labored and the rest of the family passed the time not knowing how to be.Berger can’t remember for sure when the grandfather reappeared, other than it was afternoon. It would have taken him multiple trips to carry that much food to the grills. Ribs, chops, chicken, pork, beef — in amounts far beyond what the family could possibly eat.The older man lit the fire and the family sprung to action, prepping food, clearing space, doing their jobs. The smell of barbecue filled the place, filled the boy’s room, as did the sounds of a family mobilized.When the meal was cooked the family delivered plates to every staff member and thanked them for their support through it all. People lingered late into the evening, chatting with staff, wrapping leftovers.Berger left, the grandfather left. Only a small few staffers remained.Those few were still there hours later when, in a darkened room with a moonlit view of two smoldering barbecue grills, a young boy took his leave. Armella Leung for STAT Leave this field empty if you’re human: When the boy with the big family lay dying, the place was quieter, pensive.It was a Saturday morning when Berger spotted the grandfather walking alone, as usual. Berger caught up to him, used the only thing he had.“I heard you’re good with a grill,” he said.The man said he wasn’t bad.We’ve got two big ones out back, he told him, nodding in the direction of the two dark hulks. Anyone can use them, he said.It wasn’t much of a conversation. The older man smiled quietly and walked on. Berger returned to work.center_img Related: This happens with younger people, hospice workers say. A disease can ravage their body, but their heart and other organs are often so strong that they’ll continue after most everything else has shut down, leaving survivors to an agonizingly long and lonely goodbye.This boy had had an aggressive form of cancer for months, and now he lay in what had become his de facto bedroom, in a facility that was largely given over to his enormous extended family because no one in the clan owned a home big enough to hold them all.Dr. Kevin Berger works at Ryan House. He’s been involved with pediatric palliative care for 19 years now, and has been at Ryan House for six years. He knew the look of someone who was lost.Berger asked someone on staff about the grandfather, looking for some way to reach him. The older man was a bit of a maestro at the barbecue grill, someone told Berger. He was known far and wide for it. It was a start, Berger thought.Berger believes Ryan House needs to comprehensively support families, and focus on interventions that help achieve their goals.As often as not, that means music, play, celebrations, sleepovers with friends in the community room.“These are meaningful, mindful interventions, as opposed to the traditional medical focus,” Berger said. “You can take a really sad time and make it into something where the family will remember an incredible event before the child’s death, and that’s the focus.” He was two generations removed from the boy in the bed, a weary young grandchild who had not fully awakened in days, and who in truth would not wake again.He would linger, silently, anywhere but the bedside — his oversize, calloused hands stuffed in the front pockets of his jeans. Then, unmoored, he would drift away, down the hall, outside, anywhere else.This all took place in Phoenix at a place called Ryan House, which is one of two freestanding inpatient pediatric hospice and respite facilities in the United States. The country might not need many facilities like this, but palliative care specialists say the country needs a lot more than two.advertisement Related: Privacy Policy Anyway. The man.He was the patriarch of this huge family that buzzed around the facility every day the boy was there. The staff knew the grandfather as a man of few words, and they also sensed how seriously he took his role as leader and protector. And now here he stood, powerless to help anyone, not least the boy who was struggling to die.advertisement Tags end of lifehospicepediatricslast_img read more

Democrats (mostly) hold their fire as Trump nominates Gottlieb for FDA

first_img Unlock this article — plus daily intelligence on Capitol Hill and the life sciences industry — by subscribing to STAT+. First 30 days free. GET STARTED WASHINGTON – Democrats mostly held their fire after President Trump on Friday nominated Dr. Scott Gottlieb as FDA commissioner.Gottlieb has close ties to, and has been widely endorsed by, the pharmaceutical industry. That’s been a red flag for Democrats in the past. But leaders of the left in Congress were largely quiet about the pick, perhaps because they know Trump had flirted with far more radical choices — including a libertarian who once proposed replacing agency scrutiny of new medications with a “Yelp for drugs.” What’s included? Tags drug developmentdrug pricingfinancepharmaceuticalspolicySTAT+White House What is it? GET STARTED Politics Democrats (mostly) hold their fire as Trump nominates Gottlieb for FDA By Sheila Kaplan March 11, 2017 Reprints Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Log In | Learn More STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Alex Hogan/STATlast_img read more

Mayo Clinic: Privately insured patients to get priority over Medicaid, Medicare patients

first_img About the Authors Reprints By Casey Ross and Andrew Joseph March 15, 2017 Reprints General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. Citing tighter profit margins, the chief executive of the Mayo Clinic recently told his employees that the prestigious health system will prioritize the care of privately insured patients over those on Medicare and Medicaid.That bold pronouncement by Dr. John Noseworthy — made in a speech to employees late last year — reflects the growing unease among hospital executives who are watching profits shrink due to steady increases in the number of government-insured patients. Medicaid, whose enrollment has increased dramatically under the Affordable Care Act, traditionally pays hospitals significantly less than commercial insurers.“We’re asking … if the patient has commercial insurance, or [if] they’re Medicaid or Medicare patients and they’re equal, that we prioritize the commercial insured patients enough so … we can be financially strong at the end of the year,” Noseworthy said.advertisement Health care leaders talk with Trump about the VA, Obamacare, and costs But those pressures — and Noseworthy’s statement — predate the current debate over the law. “There is this thought that hospitals treat whoever comes to their door, but this is a statement that lays out what happens,” said Christine Spencer, a health economist at the University of Baltimore. “It’s a surprise to hear it out loud like that, but hospitals, probably for decades, have engaged in these more subtle attempts to get privately insured patients over Medicaid or the uninsured.”In a statement, Mayo Clinic representatives said that about 50 percent of their care goes to patients on Medicare and Medicaid.“Balancing payer mix is complex and isn’t unique to Mayo Clinic. It affects much of the industry, but it’s often not talked about. That’s why we feel it is important to talk transparently about these complex issues with our staff. We will continue to discuss these complicated issues and work to find solutions that benefit our patients,” the statement said.As a top hospital system, Mayo stands to lose big on the spread between public and private insurance reimbursement from those sources, said Harold Miller, chief executive of the Center for Healthcare Quality and Payment Reform. Mayo told STAT that it lost $546 million in indigent care and in unpaid Medicaid portions in 2016 and $1.8 billion in unpaid Medicare portions.The health system’s market power gives it the ability to charge more for its services and command high payments from commercial insurers, a clout it can’t wield with the federal government. So, Miller said, in prioritizing those commercially insured patients, it is following the money.“It’s a very lucrative thing for them to do,” said Miller. While it makes sense from business perspective, he said, it doesn’t help to solve the underlying problem of America’s sky-high medical costs. “True leadership would be to figure out how to deliver high-quality services at the lowest cost possible,” Miller said. “If institutions are simply going to say, ‘I’m not going to serve patients unless I get paid more,’ that’s only contributing to the problem.” Privacy Policy @DrewQJoseph @caseymross [email protected] National Technology Correspondent Casey covers the use of artificial intelligence in medicine and its underlying questions of safety, fairness, and privacy. He is the co-author of the newsletter STAT Health Tech. Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. The Minneapolis Star Tribune reported his comments Wednesday.The policy is being implemented at an uncertain time for hospital finances, with many executives concerned that the GOP’s plan to replace Obamacare could cause people to lose both private and public insurance coverage, forcing hospitals to absorb more uncompensated care.advertisement Tags hospitalsinsuranceMedicaid HospitalsMayo Clinic: Privately insured patients to get priority over Medicaid, Medicare patients Please enter a valid email address. Related: The hunt for higher-paying patients plays out in all sorts of ways, experts said. A medical center may locate its satellite offices and target its advertising in wealthier suburbs. Hospitals might reduce emergency room services so they do not have to handle the chronic yet untreated issues — such as diabetes or high blood pressure — that regularly bring people without insurance to the hospital.Mayo’s policy would not apply to emergency care, and Noseworthy said that Mayo will continue to take all patients, regardless of their ability to pay. He said the change would affect only a small number of patients, and only in circumstances when government-insured individuals and those with private coverage are seeking care for similar medical problems at the same time.Mayo operates facilities in Minnesota, Arizona, and Florida, and has reported increases in unreimbursed costs related to Medicaid patients in recent years. The disparity in payments between commercial and government insurers has grown wider under the Affordable Care Act, which reduced Medicare reimbursements and instituted penalties for readmissions and poor quality. It also changed the mix of patients hospitals see by increasing the number of Medicaid patients. In Minnesota, the Medicaid rolls have expanded by 300,000 people.While having more paying patients is generally a good thing for hospitals, it can also exert financial pressure on hospitals that serve high numbers of Medicaid patients, because government reimbursements are lower and do not cover all billable costs. Experts said the gap between public and private reimbursement is ripe for public discussion. Mark Lennihan/AP [email protected] Casey Ross Leave this field empty if you’re human: Noseworthy said in his comments to employees that a recent 3.7 percent increase in Medicaid patients was a “tipping point” for Mayo. “If we don’t grow the commercially insured patients, we won’t have income at the end of the year to pay our staff, pay the pensions, and so on,” he said, “so we’re looking for a really mild or modest change of a couple percentage points to shift that balance.”Even if Noseworthy’s statement was a moment of honesty, Daniel Polsky, a health economist at the University of Pennsylvania, said it doesn’t mean he and Mayo should be chastised for their strategy.“I don’t think they should be shamed for saying it,” he said. “I think there should be some public discussion about whether elite systems such as Mayo should provide equal access to all payer types. I don’t know the answer to that, but it’s a reasonable debate.”Despite the increase in publicly insured patients, Mayo has still generated significant profits. Its income jumped to $612 million in 2013. Last year, however, income dipped to $475 million, translating to an operating margin of 4.3 percent. Andrew Josephlast_img read more

PhRMA report pins consumers’ pain from high drug prices on health plans

first_img Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. PhRMA report pins consumers’ pain from high drug prices on health plans GET STARTED What’s included? What is it? Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED By Dylan Scott March 29, 2017 Reprints WASHINGTON — A new analysis from the drug industry’s top lobbying group asserts that many Americans are feeling more of a pinch paying for their prescription drugs because customers are not fully benefitting from health insurers’ negotiations with drug makers.The new report, shared exclusively with STAT by the Pharmaceutical Research and Manufacturers of America, is the latest in an ongoing volley between drug makers, health insurers, and pharmacy-benefits managers. They are the major players, along with public officials, in the debate over drug affordability. Log In | Learn More STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Joe Raedle/Getty Images Politics Tags drug pricespharmaceuticalspolicySTAT+last_img read more

Critics say Trump birth control rule ignores science

first_img Related: It’s on the science that researchers are questioning the Trump administration. They say officials ignored some recent research and stretched other studies.“The interpretation is very selective in terms of the science that they use,” said Alina Salganicoff, director of women’s health policy at the nonpartisan Kaiser Family Foundation. “It’s always possible to find one study that validates your claim, but you have to look at the quality of the study and the totality of the research. You can make an argument that you don’t agree because of your religious or moral objections, but that is a different discussion.”advertisement APStock Does hormonal birth control put women at risk for depression? HealthCritics say Trump birth control rule ignores science Trending Now: “The vast majority of women use birth control at some point in their lives,” said Sonfield. “As a medical service, it’s far more universal than almost anything covered by insurance.”George Washington University public health professor Susan Wood, a former women’s health chief for the FDA, said there’s very clear clinical data that contraception prevents pregnancy. Why else would the FDA approve birth control pills?“They are just using this as a smoke screen,” Wood said of the administration. “They are picking out things that they like, and leaving out (studies) that support access to contraception.”The sexual revolutionThe Trump administration’s rule suggests there may be a link between birth control and promiscuity.It cites a study finding that between 1960 and 1990, “as contraceptive use increased, teen sexual activity outside of marriage likewise increased.” (The administration added a caveat that the study did not prove a cause-and-effect link.)Lawrence, the ob-gyn, said he thinks that’s a stretch.“There were a whole lot of other things going on in the ’60s,” he said, such as changing social mores about sex before marriage. Also, many people relied on condoms, diaphragms and spermicides.“The world of birth control in 2018 is about as similar to the world of birth control in 1960 as a Ralph Nader Chevy Corvair is to a space shuttle,” he said.— Ricardo Alonso-Zaldivar In a statement, Health and Human Services Department spokeswoman Caitlin Oakley responded to critics, saying: “The rules are focused on guaranteeing religious freedom and conscience protections for those Americans who have a religious or moral objection to providing certain services based on their sincerely held beliefs.”The administration also says some parts of the rules are meant to illustrate the sorts of concerns that religious objectors may have, and don’t necessarily reflect government policy.Here’s a look at examples from the Trump administration’s birth control rules that are raising questions:The morning-after pillEmergency contraception is birth control for use after unprotected sex, often called the “morning-after pill.”Referring to the morning-after pill as well as intrauterine devices or IUDs, the regulations state that the Food and Drug Administration “includes in the category of ‘contraceptives’ certain drugs and devices that may not only prevent conception (fertilization), but also may prevent implantation of an embryo.”Because of that, “many persons and organizations” believe emergency contraception methods cause “early abortion,” the regulations add.But Princeton researcher James Trussell said that while studies years ago suggested the morning-after pill might affect the lining of a woman’s uterus and interfere with the implantation of a fertilized egg, more recent studies have not found such an effect. “The preponderance of the evidence, and certainly the most recent evidence, is that there is no post-fertilization effect,” said Trussell.That’s not included in the administration’s rule.“The actual medical evidence is that it blocks ovulation,” or the release of an egg from the ovaries, explained Lawrence, the OB-GYN. “If you don’t ovulate, there is no egg to get fertilized. It’s not blocking implantation.”Effectiveness of birth controlThe Trump administration’s rule takes issue with the science behind the Obama-era decision to require most employers to cover birth control as preventive care.It suggests that some studies cited in a key 2011 report did not show a direct cause-and-effect link between increased birth control use by women and a decline in unintended pregnancy.But Adam Sonfield of the Guttmacher Institute said solid research does in fact exist. The organization does studies on reproductive health that are cited by opposing sides in the political debate.For example, Sonfield cited a Guttmacher report which found that women who used birth control consistently year-round accounted for only 5 percent of unintended pregnancies in 2008.center_img Comparing the Covid-19 vaccines developed by Pfizer, Moderna, and Johnson & Johnson By Associated Press Oct. 11, 2017 Reprints Related: Associated Press Sixty years after ‘The Pill,’ the war on contraception continues About the Author Reprints WASHINGTON — The Trump administration’s new birth control rule is raising questions among some doctors and researchers, who say it overlooks known benefits of contraception while selectively citing data that raise doubts about effectiveness and safety.“This rule is listing things that are not scientifically validated, and in some cases things that are wrong, to try to justify a decision that is not in the best interests of women and society,” said Dr. Hal Lawrence, CEO of the American Congress of Obstetricians and Gynecologists, a professional society representing women’s health specialists.Two recently issued rules — one addressing religious objections and the other, moral objections — allow more employers to opt out of covering birth control as a preventive benefit for women under the Obama health care law. Although the regulations ultimately address matters of individual conscience and religious teaching, they also dive into medical research and scholarly studies on birth control.advertisementlast_img read more

In new document, the Trump administration labors to defend its latest drug pricing proposal

first_img [email protected] In new document, the Trump administration labors to defend its latest drug pricing proposal @NicholasFlorko President Donald Trump Chip Somodevilla/Getty Images What is it? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Log In | Learn More WASHINGTON — The Trump administration is ready to defend its sweeping new drug pricing proposals. In a new Q&A, obtained by STAT, its authors parry several key criticisms — some already aired, some expected — from drug makers and other groups. The document, which is expected to be published later Tuesday according to a senior administration official, represents a quick response from the administration, which only unveiled the policy in an address on Thursday. About the Author Reprints Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTEDcenter_img Exclusive What’s included? GET STARTED Tags Congressdrug pricingpolicySTAT+White House Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. By Nicholas Florko Oct. 30, 2018 Reprints Nicholas Florko Washington Correspondent Nicholas Florko reports on the the intersection of politics and health policy. He is the author the newsletter “D.C. Diagnosis.”last_img read more

The CRISPR shocker: How genome-editing scientist He Jiankui rose from obscurity to stun the world

first_img By Sharon Begley and Andrew Joseph Dec. 17, 2018 Reprints Um, Doudna replied, you’ve dropped this shocking news on the world, right before our summit, and you’re not planning to mention it? He seemed surprised that she expected him to but agreed to have dinner with her and other members of the summit organizing committee that evening to talk it out. One of the world’s most celebrated biologists, Jennifer Doudna is not easily rattled. But she was struggling to process what she had just heard. Moments before, she met with the researcher whose bombshell had shaken the world of medicine like nothing since the birth of the first test tube baby 40 years earlier. As she walked up from the lobby of Hong Kong’s Le Méridien Cyberport hotel, the University of California, Berkeley, biochemist was shaking her head … as if that would jostle her thoughts into a place where everything made sense again.It was the last Monday in November, the day news broke that a little-known scientist in China named He Jiankui claimed he had created what instantly became known as the world’s first “CRISPR babies”: twin girls who came into existence as IVF embryos and whose genomes had been changed by the revolutionary DNA editor called CRISPR. It was something everyone in the burgeoning, multibillion-dollar field of genome editing knew would come one day, but which nevertheless shook even experts with its timing, its secrecy, and PR trappings that made the rollout of Beyonce’s “Lemonade” look amateurish.Doudna, who co-led a 2012 study showing that a weird bacterial immune system called CRISPR could edit DNA as niftily as Word edits documents, and hundreds of other experts were in Hong Kong for the International Human Genome Editing Summit. He Jiankui, who was scheduled to speak at the summit on Wednesday, had asked to meet privately with Doudna, one of the summit’s organizers. In his presentation, He had planned to talk about the ethics of embryo editing and his experiments on mouse, monkey, and human embryos, with nary a hint that two of those embryos were now living, breathing, baby girls whom He, in an astonishing YouTube birth announcement, called Nana and Lulu. Was that okay?, he asked Doudna as they sat in the lobby.advertisement Andrew Joseph Related: ‘CRISPR babies’ lab asked U.S. scientist for help to disable cholesterol gene in human embryos STAT+: General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. Jennifer Doudna speaks with the media during the Second International Summit on Human Genome Editing in Hong Kong. ISAAC LAWRENCE/AFP/Getty ImagesNobel dreams, second-rate scienceThe tensions between He and Doudna and other scientists came to a head that night in Hong Kong. But it was the culmination of acts marked by crowning ambition that others had seen in him for years.He, whose lab is at Southern (sometimes translated “South”) University of Science and Technology in the tech-booming city of Shenzhen, had sought to insert himself into the CRISPR elite. But they viewed his science as second-rate. He had hours-long discussions with a leading bioethicist who warned him against creating “CRISPR babies” — yet never revealed that the discussion was far more than academic. He confided in at least two U.S. scientists about his plan, but ignored their arguments that he was making a potentially disastrous mistake. He studied recommended ethical guidelines for embryo editing — but flouted them. He claimed he had been transparent about working toward pregnancies with CRISPR’d embryos — yet never breathed a word about those plans in his talks at science meetings and stalled for months before listing his experiment on an official Chinese registry of clinical trials.For a driven and fame-seeking scientist who had set his star on changing the world, heeding doubters and sticklers wasn’t part of the plan. Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Related: Claim of CRISPR’d baby girls stuns genome editing summit Forging CRISPR connectionsIn retrospect, He had been hiding in plain sight. Although he has been a shooting star in Chinese science for about five years, ever since he returned to the country of his birth after graduate and postdoctoral stints in the U.S., he is not an alumnus of any of the world’s leading CRISPR labs. He had written no important CRISPR papers before his shocking announcement. (He still hasn’t: The CRISPR babies experiment remains unpublished, and a study editing mouse, monkey, and human embryos without starting pregnancies has been rejected.) He was on no one’s radar screen.He tried to make up for it.In April 2016, he wrote to Feng Zhang of the Broad Institute of MIT and Harvard. After Doudna’s 2012 study showing that CRISPR can edit DNA in a test tube, Zhang and his colleagues got it to do so inside living cells, including human cells growing in a lab dish. That made him one of the world’s best-known CRISPR scientists. Alta Charo, bioethicist and legal scholar, University of Wisconsin UC Berkeley researcher, told of CRISPR’d baby study a year ago, warned scientist not to do it He identified himself as CEO of a Shenzhen-based DNA sequencing company called Direct Genomics and requested a tour of Zhang’s lab in Cambridge, Mass. The visit never happened. But He kept trying to make CRISPR connections.In late 2016, on a trip to the Bay Area, He emailed biologist Mark DeWitt of Berkeley’s Innovative Genomics Institute. Could DeWitt meet for coffee? They did, laying the groundwork for months of discussions.Then He aimed higher, contacting Doudna “out of the blue,” she said. He would be in the Bay Area in early 2017, he said; perhaps they could meet.The email landed at an opportune time. Doudna and Stanford bioethicist William Hurlbut had just received a grant of more than $215,000 from the Templeton Foundation to study “The Challenge and Opportunity of Gene Editing: a Project for Reflection, Deliberation, and Education.” To kick it off, they were holding a workshop at Berkeley for nearly 20 scientists, ethicists, and historians in January 2017. None, Doudna realized, were from outside the U.S.Maybe we should invite him, Doudna proposed to Hurlbut. They did. He came. On the workshop’s second day, in a session called “Evolution and Human Development,” He presented work on using CRISPR to edit mouse, monkey, and human embryos (without pregnancies). His talk did not leave much of an impression, “and I don’t think it was received very well,” Doudna said.He Jiankui speaks at the Second International Summit on Human Genome Editing in Hong Kong. ANTHONY WALLACE/AFP/Getty ImagesThat was partly because He was, in a sense, two years late. In 2015, scientists at Sun Yat-sen University in Guangzhou used CRISPR to edit the gene whose abnormality causes the often-fatal blood disease beta thalassemia. Their experiment, which also sent shock waves around the world, used nonviable IVF embryos. He, too, was using nonviable embryos. It didn’t seem like he was moving the science forward.Worse, another attendee recalled, scientists said He’s “science was sloppy and the application unnecessary.” One biologist challenged He on technical details of his work, especially how he analyzed the edited genomes for the unintended edits called off-target effects, a critical safety concern.Other scholars who attended were struck by what Harvard’s Sheila Jasanoff called He’s “great smoothness.” Although He did not explicitly discuss his ethical views, Jasanoff said, he “clearly did not have deep misgivings about plugging ahead with gene editing, and I sensed no exposure to the sorts of ethical debates our guys are routinely involved in.”To CRISPR’s leaders, “He wasn’t seen as a major player,” Doudna said. Having published no papers on CRISPR editing didn’t help; neither did presenting research that didn’t seem to move beyond what others had reported. Eros Dervishi for STAT Continuing in a cautionary vein, He concluded with the tragic case of Jesse Gelsinger, whose 1999 death in a gene therapy clinical trial set that field back years. That, He said, should be a warning to anyone hoping to turn CRISPR into a tool of medicine.As at Berkeley, the talk left scientists unimpressed. “It just didn’t stand out,” said Doudna, who co-organized the meeting.Viewing He as less than a heavy hitter in the genome-editing world, many skipped his talk. Computational biologist Max Haeussler of UC Santa Cruz, who shared a double room with He and did attend, is struck in retrospect at He’s discussing how dangerous editing human embryos is. “I found this remark already strange back then,” he said. “Everyone in the room knew that it’s out of the question to edit human embryos. Why mention that it’s dangerous?”In the room they shared, He and Haeussler talked shop, including how to detect off-target edits and how deeply He probed to find any. One red flag was that He was doing what’s called “short read sequencing,” meaning he sequenced short segments of DNA. That can miss big rearrangements along a chromosome. It’s similar to proofreading a document a sentence or two at a time. You’ll find garbles like, “Once time upon,” but not grossly out-of-place passages such as Cinderella losing her glass slipper before she goes to the prince’s ball. Later, in Hong Kong, He would say that large scrambles like that are not a big problem if there is no important gene nearby.He did not breathe a word of any plans to establish CRISPR pregnancies. That was about to change.Shocking secret begins to slip outTwo months after the Cold Spring Harbor meeting, Berkeley’s DeWitt had received a stunning email from He: The Chinese scientist planned to conduct a clinical trial creating the first genome-edited children, the soon-to-be “CRISPR babies.” He was enrolling patients, He told DeWitt, and had received ethics committee approval. Yet in apparent violation of Chinese regulations, He did not list the trial on China’s official trials registry until Nov. 8, 2018, which is thought to be the birthdate of Nana and Lulu.DeWitt didn’t know what to do with this information. Since He asked for confidentiality, “I let it be,” DeWitt said.He was back in Berkeley in January of this year, meeting DeWitt for dinner and reporting that his trial was on track. Since Nana and Lulu were born in the second week of November, He was about a month away from starting that pregnancy. DeWitt again tried to dissuade him, he said. William Hurlbut, Stanford bioethicist Nor did his academic pedigree. He, 34, grew up the son of rice farmers in an impoverished county in Hunan province, in southeastern China. According to Chinese media reports, he built a simple lab at home and in high school became obsessed with physics, earning an undergraduate degree in that discipline from the respected University of Science and Technology of China, in Hefei, in 2006. Flush with a scholarship to study in the U.S., he began pursuing a Ph.D. in physics and astronomy at Rice University. According to a 2010 article from the Rice news office, it was the only graduate school that accepted him.He was a star there, the news office said, specializing in mathematical modeling and computer simulations of biological systems. As president of the Rice Chinese Students and Scholars Association, he organized “a steady stream of events for a community of more than 400.” He made time for life outside the lab: “I love to play soccer,” he said then. “Oh, my God, Rice has six soccer fields! That’s awesome.”He earned his degree in less than four years (his thesis was on how CRISPR evolved), published three papers that the news office described as “of tremendous significance,” and in 2011 began a one-year postdoctoral fellowship at Stanford with bioengineer Stephen Quake. As he was finishing up at Rice, He expressed gratitude to his Ph.D. adviser, physicist Michael Deem, for encouraging him to apply his training broadly: “I did not restrict my work to conventional physics,” he said. “Instead, I applied the techniques and methods in physics to the study of biology and the economy.”The news office quoted Deem as calling He “a very high-impact student,” adding, “I am sure he will be highly successful in his career.” Eight years later, Deem was in He’s lab in China for work that would indeed make an impact, though perhaps not the kind he had in mind.China also had its eye on He. In April 2011, the city of Shenzhen, across the border from Hong Kong, launched its “Peacock Program” to attract scientists, setting them up in spanking-new labs and staking them to generous research budgets and salaries. He was a peacock. After Stanford, He became, at age 28, the youngest associate professor at the city’s Southern University.His focus: gene sequencing. Academia was too small a playground. In 2012, He founded Direct Genomics, which builds DNA-sequencing machines based on technology Quake developed. Its “GenoCare Analyzer,” for clinical diagnostics, reached the market last year, only the second Chinese-made sequencer in commercial use. “We’re a new generation of entrepreneurs,” He told a reporter in 2015. Government health officials, he said, “really hope our Chinese brand could be used in hospitals.” By 2017, Direct Genomics would raise 200 million yuan ($30 million) from investors and 40 million yuan in subsidies from the Peacock Program.‘I knew where he was heading’Although He failed to wow the high-powered attendees at the Berkeley workshop, he was bold about asking for advice. That winter He and DeWitt communicated several times, with He asking about the best way to analyze edited genomes for unintended, off-target alterations.But now something new entered the discussions. He told DeWitt he was planning to start pregnancies with CRISPR’d human embryos. DeWitt was aghast, he told STAT, and argued that there was no justification for such an experiment. The technology simply wasn’t ready to use on babies-to-be.The Berkeley workshop opened another door for He. He struck up a friendship with Hurlbut, and over the last two years had “several long conversations, like four or five hours long, about science and ethics,” Hurlbut said. He went to the Bay Area with some frequency, and made his goal clear.“I knew where he was heading,” Hurlbut said. “I tried to give him a sense of the practical and moral implications,” including ethical objections to research on human embryos. He pushed back; wasn’t it only a fringe group in the U.S. that adamantly opposes that?, he asked; if CRISPR can be used to prevent a dreaded genetic disorder in a baby who would otherwise inherit it, why should we hold a one-cell embryo in the same ethical regard as a suffering child?“My overall feeling,” Hurlbut said, “was that he’s a well-meaning person who wants his efforts to count for good.”In July 2017 He gave an updated version of his Berkeley talk, at a meeting on “Genome Engineering: The CRISPR-Cas Revolution” at Cold Spring Harbor Laboratory on New York’s Long Island. The data from his experiments in mouse, monkey, and human embryos included ways to improve CRISPR’s efficiency and measurements of its accuracy. He had injected CRISPR into the first human embryo, he said, on Nov. 10, 2016, doing two or three each month (though four that December). He reminded his audience of the many ways embryo editing could fail, including off-target edits and mosaicism (when only some of an organism’s cells are edited, creating a genetic patchwork with unknown implications for health). Related: He believed he would be hailed for his scientific first, especially in his homeland, as someone who did for China what the Sputnik engineers did for the old Soviet Union. In conversations with scientists and others, he brought up Dr. Robert Edwards, part of the team who created the world’s first test tube baby, won the 2010 Nobel prize for it, and brought joy to millions of otherwise infertile couples.No wonder He seemed stunned that Monday, as worldwide condemnation of his work grew and even the stars of the CRISPR firmament weren’t applauding him. Over the hastily arranged Cantonese buffet dinner at Le Méridien, Doudna and three other summit organizers peppered him with technical questions (How many embryos did you try to edit with CRISPR? How many succeeded? How did you decide which embryos to implant? What tests did you run to see if the editing worked as planned?) and challenged the ethics of the experiment (Why did you pick the gene CCR5, which is involved in HIV infection, to edit? Did the parents understand the risks to their potential child? How do you know?).After just over an hour, He had enough, participants told STAT. He pulled some cash out of his pocket, threw it on the table, and stormed off. Fearful of his safety, he left the Méridien and checked into another hotel. His dinner companions were left wondering if he would even show up for his scheduled talk at the summit on Wednesday. [email protected] He also spoke of Edwards, the test tube baby pioneer, with starry-eyed admiration. “He seemed to think that what he had done would vault him into the scientific pantheon, too,” Charo said. “He was just oblivious” to the fact that scientists and others around the world greeted his announcement with horror. But he also feared for his safety, showing his dinner companions what he called a threatening message on his phone, and worried that reporters would find him. Nervous and scared, He stormed off.The organizers were left wondering, would He show up for his summit talk two days later?The 500-plus people packing the standing-room-only auditorium at Hong Kong University on Wednesday wondered the same thing. Security men with earpieces crisscrossed the front of the room. Camera crews crowded the periphery. When Lovell-Badge introduced He, there were several anxious seconds before he emerged through a side door and took the stage. “He wants to help people,” Hurlbut recalled of those many conversations. “He has [two] children of his own and is sensitive to the meaning of human life. He wants it to be healthy and happy. He kept stressing to me the problems that people with HIV have in China.”In addition to what Hurlbut calls “a very earnest motivation to move the science forward,” something else was driving He. In more and more of his conversations, he was bringing up Edwards, the test tube baby doctor. People who spoke to He this year recall him as “grandiose,” convinced he was about to accomplish something for the history books.In describing his planned experiment to the ethics committee of the hospital that eventually approved it, He promised, “The project will stand out in the increasingly intense international competition of gene editing technologies,” according to a translation by Jing-Bao Nie, a bioethicist at New Zealand’s University of Otago. “This creative research will be more significant than the IVF technique which won the 2010 Nobel Prize, and bring about the dawn of the cure for numberless genetic diseases.”On the stage in Hong Kong: ‘Something didn’t look right’The organizers of the Hong Kong summit knew nothing of the preparations for the PR onslaught. As they assembled their list of speakers, He wasn’t on it.By springtime, He and others in his lab were presenting more of their work on embryo editing, again without mentioning pregnancies, to scientific conferences. At a genome-editing meeting in Suzhou organized by Cold Spring Harbor’s Asia unit, graduate student Feifei Cheng reported research using CRISPR to edit the heart-disease gene PCSK9 in human embryos. Again, it attracted little notice.At their last planning meeting, in October in Santa Monica, Calif., the organizers of the Hong Kong summit were finalizing their list of invited speakers. They were concerned that few were from Asia, said committee member Robin Lovell-Badge of London’s Francis Crick Institute. He’s name came up the first evening, over pre-dinner drinks. “The context was the rumor that had been going around for a few months that He had received local ethical committee approval” to start pregnancies with genome-edited embryos, Lovell-Badge said.center_img Sharon Begley Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. He took a crash course in bioethics. Then he created CRISPR babies “His demeanor was an odd combination of hubris and naivete,” she recalled in an interview. “He was very confident in his work, and totally not understanding what an explosion he had caused” — one that, some scientists feared, could derail hopes for using CRISPR to prevent some of the most devastating diseases lurking in the double helix.In the three weeks since the remarkable announcement about Nana and Lulu, STAT has pieced together the story of the years leading up to that fateful Monday. With details reported for the first time, it describes the many times He met with and spoke before some of the world’s leading genome-editing experts, the low opinion they had of his research, and the hints he dropped about his grandiose aspirations. It is based on interviews in Hong Kong and with experts on four continents, with scientists and others who have crossed paths with He, as well as on documents and published accounts. He did not reply to requests for an interview.advertisement Tags CRISPRgeneticsHIV/AIDSresearch Related: Please enter a valid email address. Related: Special ReportThe CRISPR shocker: How genome-editing scientist He Jiankui rose from obscurity to stun the world Exclusive analysis of biopharma, health policy, and the life sciences. [email protected] Privacy Policy China’s history with AIDS explains a puzzling aspect of the ‘CRISPR babies’ story “He was just oblivious.” @sxbegle He whipped through his 59 slides so quickly that it was only later, when they had a chance to scrutinize them, that scientists grasped how flawed the research was; He had introduced mutations into the girls with unknown consequences and that might not even protect against HIV.But even amid the rush of data, audience members were growing more and more appalled at what they saw as sloppiness. “I remember staring at the chromatogram,” said Kathy Niakan, a biologist at the Francis Crick Institute, referring to a graph showing the prevalence of different DNA sequences. “I remember not seeing what he was claiming had happened. Something didn’t look right.”After the presentation, Porteus and Lovell-Badge joined He on stage for an awkward Q&A. Porteus aimed to get specific answers to simple questions: How many embryos have you tried to modify? How accurate were the modifications? But He was cagey. Only after Porteus, who looked as if he wanted to be anywhere but there, circled back did He acknowledge a second, very early pregnancy.As audience members pressed He on the ethical and regulatory scrutiny of his trial, He never acknowledged that what he did might have been wrong. “For this particular case,” He said, “I feel proud.”The fallout: praise, then condemnationAt first, He got the plaudits he expected. In China, the initial reaction was laudatory, emphasizing that this scientific first belonged to the Middle Kingdom. The government-backed People’s Daily treated He’s experiment as a “milestone accomplishment China has achieved,” Otago’s Nie wrote.That attitude quickly flipped. The People’s Daily story was almost immediately pulled down. More than 100 Chinese scientists issued an open letter condemning He’s experiment. By Thursday, Chinese officials said they had halted the research and were investigating. Scientists called for independent verification of He’s (still unproved) claims.The reverberations quickly traveled more than 8,000 miles to the leafy campus of Rice in Houston. Deem, the AP had reported, was deeply involved in He’s experiment, including being present when parents gave consent to have their embryos CRISPR’d. Deem is also a co-author on a paper He tried to publish that described additional CRISPR experiments in early embryos, but without establishing a pregnancy.That Monday morning, Deem was accompanied into his students’ office area by Yousif Shamoo, Rice’s vice provost for research, according to someone with direct knowledge of the events. They told his graduate students and postdocs to turn over their files and research records as part of a university investigation into Deem’s role in the project.Rice University’s Michael Deem (left) and He Jeff Fitlow/Rice UniversityAlthough people at Rice and former students of Deem knew he had some research collaborations in China (Deem and He had co-authored at least three papers since 2016), his graduate students and fellows were shocked when they read about Deem’s involvement in He’s experiment. For one thing, in his lab at Rice Deem does not even do “wet biology,” meaning experiments with living cells or biochemicals, let alone clinical research. The school said it had no knowledge of the work.In a statement last week, attorneys for Deem said, “Michael does not do human research, and he did not do human research on this project.” They did not respond to questions about what AP reported.Scientists and others who have spoken to or emailed He, who has been on leave from his university since February, say he remains upbeat. He expresses confidence he will be vindicated both by the official investigations and by history.History’s verdict remains to be written. But no one believes CRISPR science has seen its last bombshell, and not only because of the second pregnancy He said is underway. Even those who condemn his experiment doubt it will be more than a speed bump on the road to editing of embryos to prevent severe inherited diseases. “We have to acknowledge there is interest in using [CRISPR] clinically,” Doudna said. To those calling for a moratorium or an outright ban on such research, she has one response: “It’s too late.” About the Authors Reprints Related: Leave this field empty if you’re human: The number of those in the know was growing. Also in January, He emailed Dr. Matthew Porteus of Stanford, whom he had met at the Berkeley workshop a year before and who is trying to develop CRISPR into a treatment for blood diseases such as sickle cell (in children and adults, not embryos). In contrast to embryo editing, which alters heritable DNA and is passed on to descendants, this kind of “somatic genome editing” is much less controversial: It alters only targeted cells, making changes that go no further than the patient.He asked if Porteus could meet the following month. One evening in February, Porteus ushered He into his Stanford office. The Chinese scientist began describing his experiments using CRISPR to edit monkey embryos, mentioning that he’d tried to start pregnancies but without success. No matter, He said: He had ethics-board approval for a clinical trial and was planning to move forward.Porteus was blindsided and angry, but also perplexed: he knew He was in frequent communication with his Stanford colleague Hurlbut, and was shocked that those conversations apparently had no effect. “I strongly rebuked him for even considering this,” Porteus recalled, adding that he told He to stop an experiment that could threaten the whole field of genome editing for disease treatment and prevention.“He was being reckless,” Porteus said. “He at least needed to speak to Chinese authorities in a formal way.”Porteus was especially concerned about the gene He intended to edit: CCR5. It makes a protein that acts as a portal by which HIV, the AIDS virus, enters cells. The medical rationale for trying to disable CCR5 was insufficient to edit it in an embryo, Porteus told He; to do so would threaten clinical use of CRISPR. Porteus also argued that He’s experiment didn’t come close to meeting the requirements for germline editing set out by a 2017 National Academies of Science report, such as rigorous assurance of safety and lengthy testing on lab animals. Later, He cited the report as a rationale for moving forward with the pregnancies.“He didn’t even acknowledge what he was doing might be wrong,” Porteus said. “I think he was looking for someone to tell him it was a great thing to do.” About Lulu and NanaVolume 90%Press shift question mark to access a list of keyboard shortcutsKeyboard ShortcutsEnabledDisabledPlay/PauseSPACEIncrease Volume↑Decrease Volume↓Seek Forward→Seek Backward←Captions On/OffcFullscreen/Exit FullscreenfMute/UnmutemSeek %0-9 facebook twitter Email Linkhttps://www.statnews.com/2018/12/17/crispr-shocker-genome-editing-scientist-he-jiankui/?jwsource=clCopied EmbedCopiedLive00:0004:4404:44  The Chinese scientist He Jiankui claims that two CRISPR-edited baby girls have been born. YouTube Porteus thought he had dissuaded He, and didn’t hear from him again until the eve of the Hong Kong summit. But if Nana and Lulu were born the second week of November, the pregnancy was just underway, or about to be, as He and Porteus sat talking.It would later emerge, initially from an Associated Press story and later from He’s summit talk, that he and his colleagues had enrolled eight couples in which the man is HIV-positive and the woman is not. After injecting a single sperm into an egg, the scientists injected their CRISPR molecules: a guide RNA that bloodhounded its way to CCR5 plus an enzyme that slashed the gene. In all, they injected 31 embryos, succeeding with 21. Analyses of cells when the embryos were 3 to 5 days old revealed a mishmash of edits. None replicated the CCR5 mutation known to protect against HIV. With the parents’ permission (He said they understood the genetic niceties), He implanted 11 of them anyway.He also spoke to Stanford’s Hurlbut that February, but did not tell him explicitly that he had started a pregnancy. Hurlbut nevertheless suspected that was He’s plan, and is puzzled about why He told others but not him.“I was advising him a very gentle sort of way because I wanted to keep the conversations going,” Hurlbut said. “But I told him, JK, you need to be careful. You have a bright future. You have a new baby. You could be humiliated by this” — where “this” meant an experiment that would bring the wrath of the scientific community, and possibly Chinese officials, down on him.Like Porteus, Hurlbut wondered about targeting CCR5. Because AIDS is preventable and treatable, many scholars argue, it does not meet the threshold of “serious, unmet medical need” that would justify embryo editing. Only diseases like Huntington’s, Tay-Sachs, cystic fibrosis, and perhaps familial Alzheimer’s clear that bar.He disagreed. He had been deeply moved by a visit he made to an “AIDS village” in China, with HIV-positive rates of 30 percent. In his country, he told Hurlbut, AIDS patients face enormous stigma and prejudice, struggling to find jobs, spouses, even housing. If someone with HIV wants to spare his or her child from the same misery, and if CRISPR’ing CCR5 can turn it into a form that blocks HIV infection, why is that any less justified than editing the Huntington’s gene? “My overall feeling was that he’s a well-meaning person who wants his efforts to count for good.” The ‘CRISPR babies’ experiment was more flawed than scientists first realized @DrewQJoseph When the committee began formal discussions the next day, Lovell-Badge raised the possibility of inviting He to speak in the session on embryo editing. “It was well-known that he had been conducting relevant research using mice, non-human primates and … [human] embryos in culture,” Lovell-Badge said.The committee discussed the quality of He’s research and his plans to start pregnancies. “We all thought this was far too premature,” Lovell-Badge said, but “it was felt that the summit was an opportunity to bring him into line”: that immersing He in discussions of ethics and safety for three days “would curtail any plans he had” for pregnancies.He accepted the invitation immediately. Lovell-Badge received He’s slides on Oct. 31. They expounded such ethical principles as “mercy for the needy,” genome editing “only for disease, not for vanity,” and “everyone deserves freedom from genetic disease.” There was nothing about pregnancies.The organizers, flying to Hong Kong from three continents, trickled into Le Méridien on Telegraph Bay on the weekend before the summit. By Saturday, news of the “CRISPR babies” was filtering out: A reporter at MIT’s Technology Review found the listing that He had entered in China’s clinical trials registry, explaining his plan to start pregnancies with CRISPR’d IVF embryos. The magazine’s story on He’s plans — but not the births — ran on the evening of Nov. 25 in the U.S., just after 8 a.m. Monday in Hong Kong. About two hours later, the Associated Press, which had interviewed and filmed He for months after being tipped off by He’s public relations adviser in April to the pregnancy, scrambled to run its story, including news of the births. He arrived at the hotel on Monday, after an hour-plus drive from Shenzhen with a colleague.He looks at a computer screen while working at a lab in Shenzhen in southern China’s Guandong province. Mark Schiefelbein/APAfter dashing across town for a 3:30 press conference, where they mostly begged off on commenting about He’s unverified claim, several of the organizers made the return trip to the hotel — and, for some of them, dinner with He in the hotel’s Nam Fong restaurant.“That,” said bioethicist and organizing committee member Alta Charo of the University of Wisconsin, “was an interesting dinner.”Over the Cantonese buffet, Charo fired questions at He, focusing on the bioethics of his experiment: how he recruited families, what he told them, why he chose CCR5. He opened his laptop and showed a spreadsheet, but it was in Chinese, leaving the scientists hardly better informed about key details. Asked why he had kept his experiment a secret, He said he had presented his work at science meetings. But he had never said he was going to establish pregnancies, and seemed not to understand why that was such a great leap beyond the embryo experiments he did talk about.last_img read more