@meggophone [email protected] Tags blood-brain barrierdrug deliverygene therapy Biologists have hijacked a virus to nix its harmful properties and harness its ability to enter the brain through the bloodstream. At least in mice, the virus can deliver genes to certain cells in the nervous system by bypassing the blood-brain barrier that often trips therapies up. Here’s what lead researchers Ben Deverman and Viviana Gradinaru of Caltech said about the findings, published in Nature Biotechnology. Medical first: Brain tumor breached with ultrasound By Megan Thielking Feb. 2, 2016 Reprints What’s the problem with current gene delivery into the brain?Usually gene delivery is done by invasive local injections. You take a needle, make a hole in the skull, and inject a few milliliters of a vesicle that delivers the gene. So you have a lot of copies of your delivery target locally at that injection site, but it dies down as the gene moves further from the injection site. If you want to deliver genes for research or therapy purposes, you want uniformity.What did you do to push for that uniformity?Our method is delivering it to the [network of blood vessels] since the vasculature touches nearly every cell in the brain. We took a virus that was known to cross the blood-brain barrier very efficiently and we evolved a variant of that. What makes these viruses attractive as gene delivery vehicles…is that we can strip them of all the viral genes.advertisement News Editor A method using viruses may be able to deliver therapeutic genes to the brain. Above, green fluorescent protein (GFP) is shown in the mouse cerebellum after the GFP gene is delivered using the method. Ben Deverman and the Gradinaru laboratory/Caltech Megan Thielking Lab ChatHijacking a virus to deliver genes to the brain Related: About the Author Reprints What are the limitations of that finding?Although these viruses have been used in research, they’re not very well characterized. We don’t understand the mechanism for how it crosses the blood-brain barrier. This method also takes very high doses of viruses, so we’re trying to make it more efficient.For more Lab Chats, subscribe to the free Morning Rounds newsletter.
He may get pulled into the debate over drug prices, too. At several hearings, lawmakers have criticized the FDA for failing to approve generic drugs quickly enough to bring the overall cost of prescription drugs down. In addition, some say the FDA should push brand-name drug manufacturers to hold down their prices, something that few people outside of Congress believe the agency currently has the statutory power to do.As US cases of the mosquito-borne Zika virus rise, Califf will also have to ensure the blood supply is safe, and push to speed testing and approval of potential vaccines against the disease. The National Institutes of Health and private companies are working to develop a Zika vaccine.And Califf will have to prove that he’s willing to push the FDA to take a harder line against the overuse of opioids — one of the issues that stalled his final approval by the Senate.“There’s a lot on his plate,” said Mary Woolley, president of Research!America, a DC-based group that pushes for public scientific research support. But Woolley, who has known Califf for many years, called him a great choice for the job. “I can’t say it enough,” Markey said on the Senate floor Tuesday. “Eighty percent of all people in the United States who die from heroin overdoses began on prescription painkillers, opioids that have been given to them by physicians.”“The nomination for Robert Califf to be the new head of the FDA gives us the opportunity to talk about this issue,” Markey added. “We have to now have an honest discussion about the role that agency is playing.”Manchin said he still believed that Califf is too close to the pharmaceutical industry to take appropriate action.“I believe the FDA must break its cozy relationship with the pharmaceutical industry,” said Manchin. Califf, he said, is “just not that person with the passion to change the culture in this important agency. We’ve let the sleeping giant go far too long.”Califf doesn’t seem to be planning a sluggish approach.In written responses to questions from Republican Senator Lamar Alexander of Tennessee, the chairman of the Senate Health, Education, Labor, and Pensions Committee, for his confirmation hearing in November, Califf sketched out his priorities for the job.Key among them is a plan to streamline clinical trials, to reduce their cost and give Americans quicker access to new medications while maintaining high safety standards. Califf said the agency can do so by adopting a two-tier system, using limited, focused trials for medications expected to make a big impact on a small population, and very large trials using electronic heath records and social media. Dr. Robert Califf will have to balance the competing pressures on the FDA for faster approvals and safe products. Win McNamee/Getty Images Califf wins Senate panel vote, but faces trouble on food labeling By Sheila Kaplan Feb. 24, 2016 Reprints PoliticsSenate confirms Robert Califf as new FDA chief, ending battle for top job Tags CongressFDApolicyRobert Califf “He is so well suited, by virtue of his long commitment to clinical research, to working across sectors public and private” to achieve the goals of speedier medical innovation and faster approvals of safe drugs and devices, Woolley said.It has been a long haul. Califf, a cardiologist, was vice chancellor of clinical and translational research at Duke University before taking the number two spot at the FDA in February 2015. The last FDA chief, Dr. Margaret Hamburg, stepped down in March of the same year.Since President Obama nominated Califf to replace Hamburg, he has won the support of many medical societies, patient advocacy groups, academics, and even the New England Medical Journal.But he has had to appease critics who contended that his past reliance on pharmaceutical industry money leaves him too close to the pharmaceutical companies to be an effective cop on the beat.His opponents, led by Democratic Senators Edward Markey of Massachusetts and Joe Manchin of West Virginia, vowed to spend as much as 30 hours talking on the Senate floor about Califf’s shortcomings after a procedural vote on Monday removed their ability to block his confirmation. But in reality, they and their Senate allies only managed to talk for a few hours on Tuesday as their colleagues moved on to other issues.Both Markey and Manchin voted against Califf on Wednesday, along with Republican Senator Kelly Ayotte of New Hampshire and Democratic Senator Richard Blumenthal of Connecticut.The holdouts said they believed that Califf would not push the FDA to do its part to resolve what they called an opioid epidemic, in which patients become hooked on prescription pain-killers, then move on to heroin. Related: Califf wins Senate panel vote, but faces trouble on food labeling Related: As the first permanent chief the FDA has had in nearly a year, Califf will face pressure from lawmakers and patient advocacy groups to speed the pace of medical innovation, even as the agency is under persistent pressure from critics who say that faster drug and device approvals will hurt the public.Califf will also have to manage the agency’s role in two major White House initiatives: the promotion of precision medicine and Vice President Joe Biden’s cancer “moonshot.”advertisement Califf has also promised to increase patient involvement in the drug approvals and communicate better with drug companies during the process. He has also vowed to work on attracting and retaining the best scientists.One of the biggest FDA problems Califf will inherit is the backlog of generic drugs awaiting approval.Far cheaper than brand-name drugs, generic medications are more in demand than ever. They make up 88 percent of all prescriptions in the United States but only 28 percent of the cost, according to the Generic Pharmaceutical Association. But the backlog of 4,300 drugs awaiting approval at the end of 2015 has drawn much criticism, especially from businesses who first filed their applications back in 2009.Califf will also have to get up to speed on food safety issues. Steven Grossman, deputy director of the Alliance for a Stronger FDA, a group devoted to increasing the agency’s funding, said that although Califf has been at the FDA since last year, his focus has been on medical products and tobacco.The FDA is still in in the midst of implementation of the Food Safety Modernization Act, passed in 2010, and Califf needs to be in the loop on the issue, Grossman said.“He will have to catch up quickly,” Grossman said. “This is going to be new territory for him.”Another fast-moving issue is the clash between the FDA and drug companies over off-label uses. Until recently, drug and device makers needed to prove that their products were safe and effective for treating an illness before they could be promoted for that purpose. But a court decision last summer started eroding that ban. Now, the FDA must negotiate a new standard.If confirmed, Califf said in November, he would work on revising the documents “in an effort to harmonize the goal of protecting the public health with first-amendment issues.”Califf will also have to continue pushing the White House Office of Management and Budget to approve the FDA’s proposed crackdown on e-cigarettes. The so-called “deeming rule” would permit the agency to regulate e-cigarettes and cigars. OMB has had the proposal since October and is still debating the issue with lawmakers and industry.Ellen Sigal, chairman of Friends of Cancer Research, a think tank, said she believes Califf’s past work for the pharmaceutical industry is a plus.“It’s an enormous asset that you actually understand how to do clinical trials, how to work with all sectors,” said Sigal, who got to know Califf while he was at Duke. “I think he’s thought a lot about what his immediate steps should be. It’s been difficult going through the confirmation process. I’m just sad it’s taken so long.” Related: WASHINGTON — The confirmation fight may have been tough, but the job could be even tougher.Now that the Senate has officially approved Dr. Robert Califf as the new commissioner of the Food and Drug Administration, he’ll inherit an agency that is being pushed to approve new medical treatments more quickly without sacrificing safety — and facing pressure to act on a host of public health issues, including drug prices and the Zika virus, over which his agency has little control.The Senate confirmed Califf Wednesday on a lopsided vote of 89 to 4, bringing a swift conclusion to a drawn-out battle with a handful of senators who charged that he was too close to the pharmaceutical industry and wouldn’t do enough to end opioid abuse.advertisement FDA official took name off papers Related: 2718760-Califf-Q-amp-as-Alexander (PDF)
2718760-Califf-Q-amp-as-Alexander (Text) Read Califf’s written answers to Alexander’s questions here Senate vote removes major hurdle in Robert Califf’s bid to lead FDA
By Helen Branswell Aug. 5, 2016 Reprints Tags abortionpolicySTAT-Harvard pollZika Virus Most Americans favor late-term abortion if Zika harms fetus, STAT-Harvard poll finds Miami residents pick up bug spray in the Wynwood neighborhood, where mosquitoes have spread Zika virus. Joe Raedle/Getty Images Senior Writer, Infectious Disease Helen covers issues broadly related to infectious diseases, including outbreaks, preparedness, research, and vaccine development. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED GET STARTED What is it? Americans’ strong aversion to late-term abortions drops precipitously if a developing fetus would likely be born with severe damage from the Zika virus, a new STAT-Harvard poll found.It showed that 59 percent of respondents thought women should have the right to end a pregnancy after 24 weeks of gestation if testing showed there was a serious possibility the fetus had microcephaly caused by the mother’s Zika infection. @HelenBranswell 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. Health 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’s included? Helen Branswell About the Author Reprints Log In | Learn More
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. @Pharmalot Ed Silverman What is it? Global spending on pharmaceuticals is forecast to reach $1.5 trillion by 2021, a whopping 32 percent increase from expected spending this year. Even so, the year-on-year rate of increase is expected to be less than it has been in the recent past, when new, high-priced medicines for treating hepatitis C and some cancers prompted a huge uptick in demand.Indeed, overall spending will rise nearly 1.5 times faster between 2017 and 2021 than between 2012 and 2016, growing by $367 billion compared with $148 billion. But spending is forecast to drop 9 percent in 2016 from the past two years, which represented nearly unprecedented spending growth, and reach anywhere from 4 percent to 7 percent, on a compounded annual basis, between now and 2021. GET STARTED Log In | Learn More Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED APStock What’s included? 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. Global spending on drugs to reach $1.5 trillion by 2021 By Ed Silverman Dec. 6, 2016 Reprints About the Author Reprints Pharmalot Tags drug pricingfinancepharmaceuticalsSTAT+ [email protected]
Pharmalot Say what? FDA warns a drug maker over a plant the UK regulator just blessed Ed Silverman File this under “Let’s agree to disagree.”Last June, the US Food and Drug Administration inspected a plant in India run by USV, which makes medicines and pharmaceutical ingredients, and found several manufacturing violations. These were detailed in a March 10 warning letter the agency sent to the company. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED What’s included? Tags FDApharmaceuticalsSTAT+ 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. GET STARTED Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Log In | Learn More [email protected] @Pharmalot By Ed Silverman March 24, 2017 Reprints A chemist works in a factory on the outskirts of Mumbai, India. Rafiq Maqbool/AP What is it? 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.
[email protected] Ed Silverman About the Author Reprints Tags addictionpharmaceuticalsSTAT+ Mallinckrodt has agreed to pay $35 million to settle charges of failing to report suspicious orders of controlled substances, the largest such deal involving a drug maker for such an infraction.The deal also includes what authorities are calling a “groundbreaking parallel agreement” with the Drug Enforcement Agency, which will involve collecting and analyzing data from customers in order to identify suspicious sales. Mallinckrodt pays $35 million fine for failing to report suspicious oxycodone orders What’s included? What is it? Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. 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. GET STARTED Log In | Learn More By Ed Silverman July 11, 2017 Reprints 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. Pharmalot APStock @Pharmalot
What is it? Three U.S. scientists win Nobel Prize for uncovering inner workings of the biological clock [email protected] Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. About the Author Reprints Sharon Begley By Sharon Begley Oct. 2, 2017 Reprints 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. What’s included? 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. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED The 2017 Nobel Prize in physiology or medicine was awarded on Monday morning to Jeffrey Hall, Michael Rosbash, and Michael Young for their discoveries of the molecular underpinnings of the circadian rhythms that help organisms adapt to our 24-hour days.The scientists “were able to peek inside our biological clock and elucidate its inner workings,” said Thomas Perlmann, secretary of the Nobel Committee for Physiology or Medicine, who announced the prize in Stockholm. “Their discoveries explain how plants, animals, and humans adapt their biological rhythm so that it is synchronized with the Earth’s revolutions.” Alex Hogan/STAT Tags research In the Lab @sxbegle Log In | Learn More
[email protected] What’s included? Pharmalittle: Migraine medication sales look promising; will the U.S. import drugs? General Assignment Reporter Liz focuses on cancer, biomedical engineering, and how patients feel the effects of Covid-19. 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. Pharmalot Log In | Learn More By Elizabeth Cooney Aug. 21, 2018 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. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED What is it? GET STARTED About the Author Reprints Elizabeth Cooney @cooney_liz Alex Hogan/STAT Good morning. It’s Elizabeth Cooney again, filling in for the vacationing Ed Silverman. Some food for thought to start the day. Comments, suggestions, tips welcome.A controversial Food and Drug Administration working group has begun discussing how to import drugs from other countries as a way to bring down prices for American patients, STAT says. “That work group has met and is outlining its plan of action,” Dan Best, Trump administration senior adviser on drug pricing, said on a phone call with reporters. “As more of that information becomes available, we’ll make it available to the market.” Tags drug developmentdrug pricingpharmaceuticalspharmalittleSTAT+