Senators urge FDA to approve Sarepta drug for Duchenne

first_imgThe missive is only the latest such attempt to sway the agency. The same lawmakers previously wrote the FDA to urge approval. And earlier this year, more than three dozen medical experts, some with ties to Sarepta, sent a letter in which they chastised FDA staff for pooh-poohing clinical evidence that the drug works. They argued that the totality of clinical trial data provides “substantial evidence” that the medicine is effective.But FDA medical staff has taken a dim view of the drug. At issue is whether eteplirsen can sufficiently produce higher levels of a protein called dystrophin. Without this protein, muscle fibers degenerate and voluntary movement becomes impossible. But FDA staffers questioned the validity of a 12-patient study, which was not run according to the usual standards. They also expressed doubts about whether the drug produced dystrophin and the viability of six-minute walking tests that trial participants underwent. In this instance, patient advocates and their many backers want the FDA to use what is called the accelerated approval process to endorse eteplirsen. This approach relies on a substitute outcome in a clinical trial to suggest a drug may have, but does not guarantee, a benefit.  The senators maintain that accelerated approval was designed for circumstances such as those posed by eteplirsen. Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. John Boal for the Boston Globe Related: Ed Silverman Privacy Policy Please enter a valid email address. @Pharmalot In their latest letter, the senators argued that the way the FDA framed the questions may have made it more difficult for some of the panel members to vote in favor of the drug. They then go on to note that the issues transcend this one drug, but can be applied to other situations where patients are clamoring for life-saving medicines and need a more flexible FDA.“Patients are crying out for the FDA to hear them,” they wrote. “They are engaged and knowledgeable and only want the agency to do what is already within their power.”Meanwhile, some Wall Street analysts believe the agency is unlikely to approve the Sarepta drug, although some also acknowledge that accelerated approval remains a possibility. The “FDA has gone to unprecedented lengths to give transparency into its evaluation process and actually seems to want to help the community as much as they are allowed to by their mandate,” wrote Cowen analyst Ritu Baral in a recent investor note.The story has been corrected to state that Senator Dan Coats is from Indiana, not Iowa.  Newsletters Sign up for Pharmalot Your daily update on the drug industry. FDA panel votes against Sarepta’s drug for Duchenne muscular dystrophy PharmalotSenators urge FDA to approve Sarepta drug for Duchenne Leave this field empty if you’re human: Nonetheless, the agency has also sent mixed signals. Janet Woodcock, who heads the FDA Center for Drug Evaluation and Research, took the unusual step of not only attending the advisory panel meeting last month, but also offering remarks. To some, she seemed to suggest that agency officials are considering more than just the scientific data that was criticized by the FDA’s own neurology division.The agency, she said, has “flexibility and that’s where we should take the views of the (patient) community into account … It’s possible to reach different conclusions based on the data presented today … Failing to approve a drug that actually works in devastating diseases — these consequences are extreme.”Her remarks were made before an overflow crowd that included hundreds of parents and their children, some of whom appeared in wheelchairs. In fact, only one of the dozens of people who spoke during the public session, argued against approval.The advisory panel, which consisted of scientists and doctors who are not FDA employees, voted seven to three, with three abstentions, against recommending approval. But asked whether accelerated approval should be granted, the vote was much closer vote: seven said no and six said yes. The agency is not obligated to accept panel recommendations, but usually does so. [email protected] Related: About the Author Reprints “For a disease like Duchenne muscular dystrophy and other diseases that are highly debilitating and almost certainly fatal, we hope you will employ these flexibilities and considerations for the maximum benefit of patients who have no other alternative,” wrote Ron Johnson, a Wisconsin Republican who chairs the Senate Committee on Homeland Security and Governmental Affairs, and Dan Coats, a Republican from Indiana, in their letter to FDA commissioner Dr. Robert Califf.advertisement As a crucial deadline nears for a closely watched regulatory decision, two Republican senators are urging the Food and Drug Administration to approve a controversial drug to treat Duchenne muscular dystrophy.In a letter sent last Friday, the senators expressed “disappointment” that an FDA advisory panel last month voted not to recommend eteplirsen to combat the disease, which is a rare and fatal genetic disorder that causes muscles to waste away. About 13,000 children, mostly boys, are afflicted and they typically die before turning 25 years old. The panel determined the drug was not effective.The FDA is scheduled to decide whether to approve the drug, which is made by Sarepta Therapeutics, by this coming Thursday. And its decision is being closely watched as a litmus test for the agency, which is grappling with increasingly assertive patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs.advertisement Why the FDA shouldn’t bow to parental pressure over Duchenne drug By Ed Silverman May 24, 2016 Reprints A hearing on findings from Sarepta’s clinical trial of a drug for treating Duchenne muscular dystrophy. Tags duchenne muscular dystrophyFDASarepta Therapeuticslast_img read more

A biostatistician’s death, and the unsung role of statistics in science

first_img Are science papers becoming too complex? Biostatistician Howard Barkan was also an accomplished jazz guitarist. Courtesy Jonathan Barkan It’s only fitting that I should write an obituary of sorts for Barkan, since we first crossed paths a little more than a decade ago while I was writing an obituary for the Lancet about someone else — Henrik Blum, a longtime professor at the University of California, Berkeley, who died in 2006. For years, I have been telling my journalism students at New York University that I had a little secret when it came to deconstructing scientific papers I wrote about: I kept a statistician in my back pocket. I told them they should do the same.My back pocket is a bit empty today. Last week, Howard Barkan, the biostatistician I kept close, died after suffering a massive heart attack while giving a lecture at the University of California, San Francisco. He was 70.And in reflecting on his legacy, I’ve been reminded of the valuable, and underappreciated, role of the numbers-minded sort in keeping science honest. As data sets grow ever larger and the statistics required to interpret them ever more complex, statisticians — working with scientists, granting agencies, and journalists — are undergirding our scientific findings with a sound mathematical foundation.advertisement Related: By Ivan Oransky July 29, 2016 Reprints The fact that in that case more people should have listened to the biostatistician and his colleagues is no surprise to any of us who’ve followed science closely.“Biostatistics is blooming,” Stanford Medicine magazine proclaimed in 2012, and it was right. Statisticians’ work becomes only more important in the era of Big Data. And yet statisticians “are like a cog in a bigger system,” as Ben Goldacre put it last year. “I would hope that statistics is becoming an integral part of science,” Goldacre told Statistics Views. “When you look at how statistical tools are routinely misused, it would be great if it was more thoroughly embedded.”That was the plan with a new project Barkan and I started discussing in March. We envisioned a blog and a numerical database, bundled together, the statistician embedded. Sadly, he will no longer be, but we will dedicate the project to him, in honor of the biostatisticians in all our back pockets. The WatchdogsA biostatistician’s death, and the unsung role of statistics in science It turned out that there was. I had recently begun writing a column called “Statistically Speaking” for a group of publications for health care professionals, and while I was getting great reader response, my readers also pointed out a few errors along the way. They were small — a few miscalculations or assumptions, that sort of thing, quickly corrected — but my editors and I wanted to make sure the copy was as clean as possible moving forward. Was there anyone I could consult for feedback and advice?And so Barkan became my statistical backstop, teaching me invaluable lessons with each phone call and email. That’s who he was, a generous and patient teacher who loved imparting wisdom and helping others improve their own work. “Those who needed help setting up a study or finding the correct statistical analyses could count on him to be ready with an answer and a smile,” said Jerome Minkoff, who worked with Barkan in the mid-1990s on studies of the effects of abuse on women’s likelihood of having mammograms and other preventive care.Barkan “always advised the students to jump right in and ‘play’ with the data and the analysis program to understand how it worked and where they had questions,” said Dr. Karen Sokal-Gutierrez, whose Berkeley students worked with him. Barkan was like the Zelig of statistics and epidemiology. (Another colleague, Melissa Farley, describes him as a Renaissance man; he mounted photography exhibits around the Bay Area and frequently played guitar in shows around the Chicago area when he lived there.) The last time I visited Barkan in Berkeley in March, we had a typically wide-ranging conversation, alighting on the lessons of the Tuskegee study and the FDA drug approvals process. He reminded me that he had been involved in studies that had sounded the alarm about a substance called hetastarch, used during heart surgery since it was first approved in the 1960s. Half a decade after Barkan and his colleagues began publishing their work, hetastarch was at the center of one of the largest scientific fraud scandals of recent years, featuring 94 retractions and earning a “black box” warning from the FDA in 2013. To keep science honest, study data must be shared I frequently end interviews by asking a question like, “Is there anything else I should have asked?” but Barkan flipped the script. Tell me about your work, he asked, in the sort of genuine way he showed interest in people, and I did. He wondered whether there was some way we could work together.advertisement Related: Tags statisticslast_img read more

Tightened rules for antibiotics for food livestock go into effect

first_img 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. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Senior Writer, Infectious Disease Helen covers issues broadly related to infectious diseases, including outbreaks, preparedness, research, and vaccine development. @HelenBranswell What’s included? Helen Branswell Log In | Learn More About the Author Reprints GET STARTED Tightened rules for antibiotics for food livestock go into effect By Helen Branswell Jan. 3, 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. What is it? A new rule that aims to safeguard essential antibiotics for humans by limiting their use in food animals is now fully in effect.Under the Food and Drug Administration policy, antibiotics that have been designated “medically important” — in other words, they’re needed to treat people — cannot legally be given to healthy animals to speed their growth. The policy, three years in the making, required producers of agricultural antibiotics to change labeling on the drugs to make clear they should not be used for so-called growth promotion. All manufacturers agreed to abide by the new rule. A new policy will keep certain antibiotics from being used to promote growth in food animals. Jamie McDonald/Getty Images Health Tags antibioticspharmaceuticalspolicylast_img read more

6 medical innovations that moved from the battlefield to mainstream medicine

first_imgHealth6 medical innovations that moved from the battlefield to mainstream medicine VA health system is a ‘living reflection’ of our history Tying offWar’s biggest killer has always been blood loss. That’s not a surprise, given the weapons of war over the ages, including swords, bayonets, bullets, grenades, and missiles. But it was a 16th-century Italian war that popularized a means to slow or stop the bleeding. In 1537, a French barber-surgeon named Ambroise Pare went to care for soldiers at the Siege of Turin. Horrified by the many bloody injuries he came across, Pare began fashioning ligatures, and tied them onto the soldiers near their wounds. He wasn’t the first to think of that — Romans and Arabs had also deployed the technique of tying a rope or belt onto a wounded limb — but it had fallen out of favor as doctors adopted other ways to stop bleeding, such as cauterizing wounds with boiling oil.That method, on a battlefield, was as inconvenient as it was (probably) painful. So Pare was instrumental in getting doctors to reconsider ligatures, or tourniquets, leading to the widespread use of them today — not just on the battlefield but in emergency rooms and at sites of accidents and natural disasters.advertisement Shedding lightSometimes medical discoveries take an extra-long route from the battlefield to the doctor’s office. In 1862, after the Civil War’s Battle of Shiloh, medical personnel noticed a glow in the wounds of soldiers from that fight. The mysterious light baffled the doctors even more when they noticed that soldiers whose wounds glowed had a better survival rate than those without illuminated injuries. That prompted many at the time to call the phenomenon “the angel’s glow,” indicating that celestial beings had healed the soldiers with heavenly light. It took almost 140 years, a microbiologist mom, and two teenagers to find a more earthly explanation.In 2001, after learning of the glowing wounds at a history exhibit, 17-year-old Bill Martin asked his mother, who studied bioluminescent bacteria, whether those could be responsible for the glowing wounds of the Battle of Shiloh. And like any good scientist, USDA microbiologist Phyllis Martin told her son to conduct an experiment to find out. So he did. The younger Martin and his friend Jonathan Curtis discovered that the glow came from Photorhabdus luminescens, a bacterium carried by nematodes — small worms which feed on insects. The boys concluded that as the soldiers crawled through mud, their wounds attracted insects, followed by the hungry nematodes. The nematodes’ bacteria not only break down the insect bodies for eating but also kill off competing microbes.It was the latter function that saved the soldiers. And since that discovery, medical scientists have begun looking into Photorhabdus luminescens as a way to treat antibiotic-resistant infections. Other researchers are using the glowing bacteria to develop a protease inhibitor to treat HIV and other diseases.Restoring flowOn the battlefield, blunt-force and impact injuries can stretch or crush veins and arteries. So, it’s not surprising that war and its attendant injuries have driven many of the advances in repairing blood vessels.Much of the progress came during the Korean War, which began in 1950. At that time, Army vascular surgeon Carl Hughes and his colleagues at Walter Reed Army Hospital set out to study the types of vascular injuries Korean War soldiers suffered and how they fared. Please enter a valid email address. Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Tags lists Red Cross personnel attend to wounded soldiers on a Russian battlefield during World War I. Wartime clinicians have often changed the way medicine is practiced more broadly. Hulton Archive/Getty Images By Leah Samuel Nov. 10, 2017 Reprintscenter_img Wartime medicine is an incredibly challenging setting for the doctors, nurses, and paramedics who practice it: Not only are the injuries frequently serious ones, but the tools at hand are often more limited than in a traditional hospital.Over the centuries, that has meant that battlefield medical personnel have had to innovate. Those wartime practices, in turn, often served to refine medical practice beyond the military.Here are six cases in which wartime clinicians changed the way medicine is practiced more broadly.advertisement Privacy Policy Leave this field empty if you’re human: Among the team’s discoveries was that while ligation — tying off or clipping injured vessels — stopped the bleeding immediately, it resulted in amputation far more often than simply taking the time to repair the artery or vein. This realization led to a dramatic drop in the number of wartime amputations from World War II to the Korean War.The discovery also helped popularize vascular repair surgery more broadly, by familiarizing surgeons with the techniques and with new tools such as the now-ubiquitous Potts clamp. Today those tools and techniques help treat everything from heart disease to varicose veins.Halting infectionWar also brought about the mass production of antibiotics, especially sulfanilamide and penicillin. World War II helped both of them find widespread respect, production, and use.In 1928, when Scottish bacteriologist Alexander Fleming noticed a weird mold had taken over his Petri dishes and eliminated the bacteria on them, his findings didn’t get much notice. But Fleming continued his research and kept talking up what he called “mold juice” (he didn’t come up with “penicillin” until later), eventually winning a Nobel Prize and attracting the attention of drug maker Pfizer. The company soon began mass-producing the drugs for distribution to medics during WWII, and ultimately, to doctors and hospitals across the country.In 1932, German biochemist Gerhard Johannes Paul Domagk discovered that the compound sulfanilamide could vanquish deadly strains of bacteria, like the streptococcus in his lab mice and in his first human test subject, his gravely ill young daughter. The wide distribution of so-called “sulfa drugs” began when World War II soldiers carried powdered sulfanilamide in their first-aid kits. By the end of the war, doctors were routinely using these antibiotics to treat streptococcus, meningitis, and other infections.Saving faceThough plastic surgery is often associated with cosmetic procedures, its origins were in reconstructive surgery. Today, reconstructive plastic surgery helps people with cosmetic concerns resulting from birth defects like cleft lips, physical assaults like acid attacks, and medical conditions like necrotizing fasciitis and other causes of disfigurement. And its origins trace back to 20-year-old Carleton Burgan.Hospitalized while serving in the Civil War, Burgan was taking mercury pills for pneumonia. They created a gangrenous ulcer on his tongue. Grangrene spread quickly from his mouth to his eye and led to the removal of his right cheekbone.Desperate, the young man offered up his face to Gurdon Buck, a New York surgeon. With a series of operations, Buck used dental and facial fixtures to fill in Burgan’s missing bone until the Army private’s face regained its shape. Buck also photographed the progress of Burgan’s facial regeneration. Buck went on to perform 32 more facial reconstructions for soldiers disfigured by bullets, bayonets, and musket balls, and he photographed many of those operations. Though primitive by today’s standards, Buck’s techniques planted the seeds of the sophisticated reconstructive surgery we have today. Related: Getting thereAs the Civil War got underway in the 1860s, transport for wounded soldiers consisted largely of a motley collection of vehicles operated by whomever happened to be available. And some of those people weren’t particularly suited for the job because they drank heavily, and/or fled with an empty wagon when the shooting started.Enter Jonathan Letterman, an Army doctor who developed an efficient and effective ambulance system that ultimately became a model for today’s local systems of emergency transport. At each battle, he set up caravans of 50 ambulances. Each vehicle carried supplies, including morphine and bandages, along with a driver, a stretcher, and two guys to carry it.Letterman built on his efforts as the war dragged on. He added a lockbox to the ambulances, under the driver’s seat, to prevent bandits from stealing drugs and other supplies. Spring suspensions made for a smoother ride over the uncertain and variable terrain between the battlefield and the hospital.His ideas led to better, faster recovery from war wounds. Now Letterman’s name graces an award for improving patient outcomes. Related: A century-old vaccine vial sheds light on one of medicine’s enduring mysteries last_img read more

Obamacare? That’s so last month. On Capitol Hill, drug prices are now the hot topic

first_img Obamacare? That’s so last month. On Capitol Hill, drug prices are now the hot topic Senior News Editor By Erin Mershon Nov. 29, 2017 Reprints Tags Congressdrug pricespolicyWhite House Politics 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. [email protected] What’s included? What is it? Erin Mershoncenter_img GET STARTED Log In | Learn More @eemershon About the Author 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. WASHINGTON — Drug prices finally have lawmakers’ attention.After years in which the debate over Obamacare has dominated all health policymaking discussion on Capitol Hill, U.S. lawmakers are increasingly turning their attention to the prices that everyday Americans pay for their prescription drugs. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Tasos Katopodis/Getty Imageslast_img read more

A big PBM wants drug makers to agree to rebate demands that would preserve its bottom line

first_img Mark Lennihan/AP Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED 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. [email protected] @Pharmalot GET STARTED Pharmalot What is it? About the Authors Reprints What’s included?center_img Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. By Ed Silverman and Ike Swetlitz Feb. 11, 2019 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. Ed Silverman A big PBM wants drug makers to agree to rebate demands that would preserve its bottom line Log In | Learn More As pressure mounts to overhaul opaque pharmaceutical pricing, OptumRx, one of the largest U.S. pharmacy benefit managers, recently demanded that drug makers make significant changes toward rebates, but in a way that would largely bolster its own bottom line.The move by the PBM — which is owned by UnitedHealth Group (UNH), the big health insurer — comes as the Trump administration and some drug makers seek to minimize, if not eliminate, the influence rebates have on drug prices. Pharmacy benefit managers collect rebates from drug companies in exchange for favorable placement on formularies, the lists of medicines that receive preferred coverage. Tags drug pricingMedicaidMedicarepharmaceuticalsSTAT+last_img read more

Walter Isaacson is writing a book about CRISPR. He’s got company

first_img CRISPR advances are coming fast. Here’s your guide [email protected] “My belief now is that the technology that will affect the first half of the 21st century most is biotech,” Isaacson said. “And so by telling the narrative story of the advances in biotechnology that began, at least the way I picked it, from the discovery of the structure of DNA to the present, it’s not just a book about policy and morals, but about the absolute breathtaking beauty of science.”Fans of Isaacson’s work should know it’s a little premature to think about preorders. His process usually boils down to two years of research and two years of writing, he said, and he started digging into CRISPR just six months ago.Closer on the horizon is the working-titled “Editing Mankind,” from three-time author Kevin Davies. Supported by a 2017 award from the Guggenheim Foundation, Davies set out to document CRISPR’s evolution from a bacterial curiosity to a ubiquitous scientific tool, explaining the many characters and biological nuances at play. The idea is to be “widely accessible but at the same time still convey the scientific details and drama that will interest people in the field,” he said.Davies’s book, sold to Pegasus, has a publication date set for April, which means he has a lot of writing to do this summer.“The deadline is looming fast, so I may have to pull a George R.R. Martin and ask for some extra time,” said Davies, executive editor of the recently debuted CRISPR Journal. “Though I don’t have his leverage, unfortunately, so we’ll how that turns out.” About the Author Reprints Related: The six letters that forever changed science will soon be the subject of thousands of words as a burst of new books on CRISPR genome editing make their way toward a retailer near you.One, from famed biographer Walter Isaacson, promises a sweeping narrative that goes from that double helix discovery to those CRISPR’d kids in China. Another will pick apart the potential of eradicating infectious diseases, while a third aims to make the underlying science understandable, and yet another asks whether any of this playing God business is ethically sound.It’s a crowded field, but one free of competitive acrimony. While the laboratories behind the genome-editing technology have fought it out in court, the corresponding book writers have mostly exchanged supportive emails and phone calls. And each believes the vast implications of CRISPR make room for a multitude of books.advertisement National Biotech Reporter Damian covers biotech, is a co-writer of The Readout newsletter, and a co-host of “The Readout LOUD” podcast. Leave this field empty if you’re human: Michael Specter is in a similar spot on the subject of deadlines. Over the past few years, the longtime New Yorker staff writer has gone from labs in eastern Massachusetts to villages in West Africa, gathering string on the undeniable benefits and risks of genetic engineering. His as-yet-untitled book will delve into how society should grapple with CRISPR’s unprecedented power. To him, the allure of, say, eradicating malaria with edited mosquitos or curing a rare disease with CRISPR is too strong for scientists ignore, but so too are the potential risks.“Down the road, the more mastery we have over life — the way we’ll be able to play with and eventually make the components of living beings — is going to be a fundamental issue in our world,” said Specter, who is an adjunct professor of bioengineering at Stanford University. “It can be great. It can be helpful. But it can be scary as hell. It can do terrible things. We need to have that conversation. And what I want with this book is to be part of that conversation.”First, he needs to deliver a manuscript to his editor at Crown Publishing Group by the end of the summer, “or else my child will suddenly inherit my house,” he said, presumably joking.Specter and his fellow CRISPR authors said they’ve had no trouble getting a line on the field’s pioneering scientists, including the Broad Institute’s Feng Zhang and the University of California’s Jennifer Doudna, who have been open and available despite the nagging fact that their respective institutions are engaged in a patent fight.For Baylis, the bioethicist, the challenge hasn’t been reaching sources but rather deciding when to stop writing. Her book, “Altered Inheritance: CRISPR and the Ethics of Human Genome Editing,” was all but finished when, in November, He Jiankui told the world that he had not only modified a pair of human embryos but implanted them into a woman who carried the twins to term. That forced Baylis into a monthslong “massive rewrite,” she said, and finishing the project meant ignoring incremental news that broke in the meantime.Unlike the other books in progress, Baylis’ effort is less to explain how we got here than to probe where we go next. Related: Isaacson’s approach is more like a survey of culinary history. The author, best known for his biographies of Albert Einstein and Steve Jobs, among others, said his fascination with CRISPR is a natural outgrowth of those earlier works. The first half of the 20th century was shaped by physics, Isaacson said, inspiring his book about Einstein. The second half was written by information technology and the ever-shrinking microchip, which is what led him to his 2011 biography, “Steve Jobs.”advertisement Please enter a valid email address. Privacy Policycenter_img Walter Isaacson, the famed biographer, is among a number of authors working on books about gene editing and CRISPR. Matt Winkelmeyer/Getty Images By Damian Garde July 2, 2019 Reprints Patent office reopens major CRISPR battle between Broad Institute and Univ. of California In the LabWalter Isaacson is writing a book about CRISPR. He’s got company “It’s like we’re all writing cookbooks, but we have different goals and agendas,” said Françoise Baylis, a Dalhousie University bioethics professor whose book will come out in September. “Some are concerned with different fruits and vegetables; some are looking at different ethnic cuisines; and some are offering advice on how to eat on $5 a day.” @damiangarde NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. Back in 2015, she was part of the international committee that gathered to set some rules on the ethical limits of genome editing. The result was a deceptively simple framework: Tinkering with human embryos can only be OK if the benefits outweigh the risks, and if there’s a broad societal consensus that doing so is worthwhile. The issue, according to Baylis, is that no one really knows what those things mean, and her book is meant to shed light on the great swaths of gray that get ignored in black-and-white ethical debates.“What I really wanted to do was write a publicly accessible book that would encourage people to think more broadly about the issues that I think are in play,” she said. “I’m trying to make space for a conversation that doesn’t not reduce everything to the science and that does not reduce everything to this narrow harm-benefit ratio. I’m trying to expand the conversation.”Her book, out on Harvard University Press in September, will begin what looks to be a metronomic publishing schedule for genome-editing nonfiction in the coming years. But Baylis, like Isaacson, isn’t worried about a flooded market or CRISPR-fatigued readers.“There’s about 50 books on Donald Trump that will come out, and gene editing is 100 times more interesting than Donald Trump,” Isaacson said. “So, you do the math.” Damian Garde Tags biotechnologyCRISPRlast_img read more

From a small town in North Carolina to big-city hospitals, how software infuses racism into U.S. health care

first_imgA STAT InvestigationFrom a small town in North Carolina to big-city hospitals, how software infuses racism into U.S. health care Casey Ross 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. About the Author Reprints What’s included? 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. Tags Artificial IntelligenceHealth DisparitiesHealth IThospitalsracism A railroad crossing in Ahoskie, N.C. Landon Bost for STAT Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED 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. [email protected] By Casey Ross Oct. 13, 2020 Reprints AHOSKIE, N.C. — The railroad tracks cut through Weyling White’s boyhood backyard like an invisible fence. He would play there on sweltering afternoons, stacking rocks along the rails under the watch of his grandfather, who established a firm rule: Weyling wasn’t to cross the right of way into the white part of town.The other side had nicer homes and parks, all the medical offices, and the town’s only hospital. As a consequence, White said, his family mostly got by without regular care, relying on home remedies and the healing hands of the Baptist church. “There were no health care resources whatsoever,” said White, 34. “You would see tons of worse health outcomes for people on those streets.” What is it? GET STARTED Log In | Learn More @caseymross last_img read more

NIH releases a plan to confront structural racism. Critics say it’s not enough

first_img Their plan to dismantle racism, NIH Director Francis Collins and his colleagues wrote, was just the beginning of what they recognize is a “monumental task.”advertisement By Usha Lee McFarling June 10, 2021 Reprints Hrabowski said he believed solving long-standing problems such as unequal research funding and low numbers of scientists of color would require far more funding than the report had pledged. He said universities also needed to work harder to increase the pool of scientists of color in a position to apply for NIH grants by supporting them from their undergraduate years through graduate school, postdoctoral positions, and their first faculty jobs.Omolola Eniola-Adefeso, a Black professor of chemical engineering at the University of Michigan, has been sharply critical of funding disparities at NIH. In February, she and a large group of fellow women biomedical engineers wrote an editorial called “Fund Black scientists,” which cited evidence that Black scientists must spend twice as much time on grants in order to be funded at the same rate as white scientists and are less likely to receive tenure in part because they don’t receive as many large NIH grants such as R01s. “NIH must change course,” the group wrote.Asked to review the new plan for STAT, Eniola-Adefeso said she found it disappointing. “There is nothing in that plan that is transformational,” she said. Even though the plan pledged to stop “siloing” diversity issues, Eniola-Adefeso said its focus on addressing funding disparities by increasing support for health equity research was itself siloing. “Health care disparities are important, but Black P.I.s are also interested in robotics, gene therapy, and CRISPR,” she said. “In some ways, that’s pigeonholing us. That’s saying you should tell a young Black girl who wants to study nanotechnology to study health disparities instead.”She said the NIH needed to repair the grant review process that has led to so little funding for Black scientists and, if they could not do that, simply fund more Black scientists, regardless of what topics they study. Since there are so few Black principal investigators, reaching funding parity would require a minuscule part — just 0.07% — of the NIH budget, she noted. And, as the NIH agreed in its plan, diverse perspectives and research teams enhance scientific productivity. But a plan to fund Black investigators, she and her co-authors noted in their editorial, would likely cause a “backlash from loud and privileged members of the majority.” These types of uproars, they noted, are what allows racism to persist.Eniolo-Adefeso said she was frustrated the plan had not adopted more of the suggestions outlined in her group’s editorial, such as prioritizing diverse research teams for funding and giving program officers the power to reevaluate grants of Black principal investigators who score highly but just below the threshold for funding, something she said the NIH does for research topics it prioritizes.“We gave them things we know will work because we are their reviewers and applicants,” she said. She was happy to see that the NIH-wide BRAIN initiative, which funds neuroscience research, will, for the first time, use diversity of grantees as part of the grant scoring criterion. “I hope that’s a pilot project that is expanded to the entire NIH,” she said.Bernard said the NIH was experimenting with other actions, not described in the plan, to close the funding gap, including increasing the diversity of grant review panels, removing the names of grantees from applications, and having all reviewers undergo implicit bias training. She said she and others would be looking closely to see whether the changes have an impact on funding rates. “We have a lot of work to do,” she acknowledged. National Science Correspondent Usha covers the toll of Covid-19 as well as people and trends behind biomedical advances in the western U.S. NIH Director Francis Collins and his colleagues wrote that the agency’s plan to confront structural racism was just the beginning of a “monumental task.” Graeme Jennings/AP 4Comments 4Comments Related: 4Comments JAMA editor-in-chief stepping down after backlash from podcast that questioned racism in medicine Saying structural racism is a chronic problem throughout biomedical research and within their own walls, leaders of the National Institutes of Health Thursday unveiled a plan intended to eliminate a big gap in grants awarded to white and minority scientists and boost funding for research on health disparities.The agency, the largest funder of biomedical research in the United States, said it would also expand a program to recruit, mentor, and retain researchers from underrepresented racial and ethnic groups, and appoint diversity and inclusion officers at each of its 27 institutes and centers.The report says NIH leaders failed to acknowledge numerous firsthand accounts of racism in the workplace and the organization has failed to attract, retain, and promote scientists from underrepresented racial and ethnic groups. Less than 2% of NIH senior investigators are Black.advertisement 4Comments Tags government agenciesHealth Disparitiesracism About the Author Reprints Related: When a cardiologist flagged the lack of diversity at premier medical journals, the silence was telling There was an error saving your display name. Please check and try again. Published in the journal Cell, the plan acknowledges that structural racism is a problem throughout society and says “biomedical science is far from free of its stain.” Not only have people of color experienced health inequities for centuries, the report notes, but scientists of color have been stymied in their careers by not getting adequate funding and other support from NIH. [email protected] Usha Lee McFarling Related: Advocates working to address inequities in science said they were encouraged by the NIH plan but said it is unlikely to bring about major changes, and they called on the agency to put much more money into the initiative. “There is no doubt there is a sincere desire on the part of NIH leadership to address this question, but is this enough to really move the needle?” asked Freeman Hrabowski, a Black mathematician and president of the University of Maryland, Baltimore County.Like many Americans, leaders of the NIH started grappling with the issue of racism in earnest after the murder of George Floyd last summer, at a time when those at the NIH — whose mission is to seek and apply knowledge to enhance the health of all people — were already reeling from the racial health inequities the coronavirus pandemic had laid bare.“I talk about this being a tipping point. There really has been a significant change in viewpoint nationwide and in the scientific community,” said Marie Bernard, the chief officer for scientific workforce diversity at the NIH and one of the leaders of the new effort, called UNITE. “This is an unparalleled opportunity to make change.”The plan’s release comes after Collins in March apologized that the agency had not done enough to address racism.The NIH doles out more than $40 billion in research dollars each year. The agency has been sharply criticized for entrenched disparities that see Black researchers funded at a rate barely half that of white researchers — 55%, a gap that has remained steady for the past decade.Graph showing the number of applicants for NIH R01 grants and funding rates by race. While the number of applicants from underrepresented minority scientists and their funding rates have increased in recent years, these grants are still funded at lower rates than white scientists. Collins et. al. CellThe new plan includes a number of programs, many already underway, to close numerous racial, ethnic, and gender gaps at the institute. In a major focus on the “significant underfunding” of research on minority health and disparities, NIH leaders plan to spend $60 million on projects aimed at reducing health disparities and another $30 million to study and address the impact of structural racism and discrimination on minority health. This new funding is meant to help address the funding gap: One study suggested that topic choice — focusing on community-based research as opposed to mechanistic science — explains 20% of the racial funding disparity.Many who have been on the front lines of work to increase diversity in science said that they were happy to see NIH addressing racism, but that NIH leaders needed to do far more work. “This report is a solid first step,” said Hrabowski, whose university’s Meyerhoff Scholars Program has produced the most Black undergraduates in the U.S. that go on to receive doctorates in the natural sciences and engineering. (Viral immunologist Kizzmekia Corbett, who led an NIH team that conducted groundbreaking research instrumental to Moderna’s coronavirus vaccine and is now at the Harvard T.H. Chan School of Public Health, is one famous graduate.)Hrabowski chaired a National Academies panel investigating how to improve diversity in STEM a decade ago, when just 2.1% of people with doctoral degrees in science were Black. Today, it is 2.3%. “We really have not made much progress,” he said. “The question we have to ask our society — and NIH — is what will it take to change the culture?” @ushamcfarling Manu Platt, a Black professor of biomedical engineering at the Georgia Institute of Technology, who has highlighted racism in science and academia in recent editorials and lectures, said he was excited, in some ways, about the plan. “I love that they are doing things. I like they are saying the word racism,” he said. But Platt said he wished NIH could work faster to increase funding to Black scientists to help keep them in tenured faculty positions. “It’s very difficult out there,” he said. “Funding Black investigators needs to be the linchpin.”Platt, who was recently asked to advise the National Institute of Biomedical Imaging and Bioengineering on diversity issues, said his experience serving on funding review panels showed him that systemic racism plays a clear role, even though race is not listed on grant applications. “They look at where people trained, who they trained with, and what institution they’re at. If you’re at an HBCU (historically Black college and university) or a smaller institution, you get penalized,” he said. “And structural racism may be playing a role in why people are not being hired at those larger institutions to begin with. It all feeds forward.”The NIH could easily prioritize funding for scientists from underrepresented groups, Platt said, as it does for early-career scientists to help them establish a funding track record and improve their chances of success and tenure. He said such a program would be a more direct route to increasing racial parity in funding than running programs to help improve the skills of minority scientists, such as grant-writing workshops.“I’d like to see more programs that don’t want to fix the investigators but want to fix the system,” he said.The NIH acknowledged in its plan that the 60 programs it previously had in place to address diversity had not done enough and that “widescale, systemic changes” that reached across the entire NIH were needed. NIH,” the plan states, “can no longer look the other way.” HealthNIH releases a plan to confront structural racism. Critics say it’s not enough Create a display name to comment This name will appear with your comment Acknowledging its ‘white patriarchy’ and racist past, the AMA pledges to dismantle causes of health inequities last_img read more

17-year-old arrested after deadly Alligator Alley crash

first_imgAdvertisementRecommended ArticlesBrie Larson Reportedly Replacing Robert Downey Jr. As The Face Of The MCURead more81 commentsGal Gadot Reportedly Being Recast As Wonder Woman For The FlashRead more29 comments AdvertisementDC Young Fly knocks out heckler (video) – Rolling OutRead more6 comments’Mortal Kombat’ Exceeded Expectations Says WarnerMedia ExecutiveRead more2 commentsDo You Remember Bob’s Big Boy?Read more1 commentsKISS Front Man Paul Stanley Reveals This Is The End Of KISS As A Touring Band, For RealRead more1 comments RELATEDTOPICS Amazon distribution center officially opens in Collier County June 16, 2021 COLLIER COUNTY, Fla. – A 17-year-old was arrested Wednesday after a deadly crash that killed a Venice teen on Alligator Alley in August. After months of investigation, Troopers arrested the teen who was driving with several others when he hit the back of a semi-truck and flipped the car killing one of his passengers. A16-year-old died in the semi-truck crash that shut down Alligator Alley in Collier County on August 4.The teen boy from Venice was in the car with four other teens; all were rushed to the hospital, according to Florida Highway Patrol. Only one of the teens was wearing a seatbelt. Collier school resource officers teach kids how to fish on first day of “Summerfest” June 16, 2021 Mosquitoes swarming Collier County, crews trap 25,000 in two nights June 17, 2021 Wounded Warriors organization accuses City of Naples of discrimination June 16, 2021 Advertisement Troopers confirmed the driver, a 17-year-old, hit a semi-trailer from behind in the northbound lanes, lost control, flipped the car, then hit a guardrail. Both northbound and southbound lanes shut down so emergency responders could treat the five teens. The semi-truck driver was not injured. The driver was arrested and charged with vehicular homicide, reckless driving, and driving without a license. Advertisement AdvertisementTags: collier countyI-75 Advertisementlast_img read more