Month: June 2021

first_img Tags anxietymental healthpost-traumatic stress By Leah Samuel March 23, 2016 Reprints And psychologists are now using these kinds of events to better understand the phenomenon of resilience — how it’s made, what it looks like, who has it, and how to create it where it doesn’t yet exist.All that will come later, though. In the immediate aftermath of Tuesday’s attacks, the Belgian Institute for Psychotraumatology started mobilizing a team of emergency responders to provide mental health services for citizens who feel affected by the attacks.According to Naomi Vandamme, a clinical psychologist with the organization, her team is particularly focused on those who are most vulnerable to post-traumatic stress disorder.“PTSD rarely comes alone,” Vandamme said. “People who get it will often have some other disorder, like depression or anxiety. And those who had already been traumatized before the event will experience more post-traumatic stress.”One of the first signs of post-traumatic stress that Belgium can expect to see is that some people will avoid certain areas, Vandamme said.“People may think, ‘I’m not going to take the metro,’ or ‘I won’t go into the city,’ or ‘I’m not going to take my vacation because it might not be safe,’” she said, adding that those behaving so fearfully will need supportive care. But it is what happens after those feelings are manifested that most intrigues mental health experts. Researchers now want to go beyond the trauma to find out how some people recover quickly or limit the emotional and mental damage. Related: A Brussels hospital is tending to terrorism victims days after tending to terrorist suspect People react outside Brussels airport after explosions on Tuesday. Geert Vanden Wijngaert/AP Tragic events — from the 9/11 attacks to the Boston Marathon bombing to the recent shootings in San Bernardino, Calif., and Paris — have all brought attention to their psychological impact. Research has shown, for example, that after terrorist attacks, later disasters and media coverage can increase post-traumatic stress.advertisement Related: Recovering from a traumatic event like the airport and subway attacks in Brussels this week starts long before the final tally of casualties and damage. For psychology professor Elke Van Hoof, it started in her classroom on Tuesday morning at Vrije University in Brussels.As her students’ phones started pinging with news of the terror, “we stayed in the classroom together and talked,” Van Hoof told STAT, just hours after the attack.Some students cried, others got angry. “It’s okay to feel angry and to feel despair,” Van Hoof said.advertisement HealthBrussels attacks: A tragic opportunity to study mental health response to terror How Paris hospitals dealt with the terror attacks Vandamme and her colleagues will also be researching the fallout from the deadly bombings in Brussels — yet another tragic data point in the growing literature about how people do or don’t persevere through the chaos.“It’s a little too soon to know what we will study, but we have a research team in place,” Vandamme said. “I would be very interested in comparing the group that will have a lot of trauma-related disorders to those that don’t. What I would love to learn about are the people who will not have a problem.“We do know that people who have a safe relationship with a supportive figure in their lives, and people with a stronger sense of identity, have better coping skills,” Vandamme added. “But we want to learn how, so we can bring services that can bring people closure on these events.”last_img read more

first_img About the Author Reprints @kweintraub Karen Weintraub is an independenthealth/sciencejournalist, journalism teacher, and bookauthor. HealthHeather Greenlee: Promoting veggies, and acupuncture, to help cope with cancer By Karen Weintraub April 8, 2016 Reprints For generations, people with cancer were treated solely for their tumor — not for the pain and other symptoms that came with it. So patients quietly turned to alternative and complementary therapies, outside the halls of medicine.Now, researchers like Heather Greenlee, an assistant professor of epidemiology at Columbia University’s Mailman School of Public Health, are trying to bring those two approaches into harmony.Greenlee, who focuses on breast cancer, talked with STAT about which alternative approaches are worthwhile, how to choose treatments wisely, and what it’s like to advocate for alternative medicine in a conventional world.advertisement Karen Weintraub Tags acupuncturealternative medicinecancer Heather Greenlee, assistant professor of epidemiology at Columbia University’s Mailman School of Public Health. Molly Ferguson for STAT Are there things cancer patients can do that might reduce their risk of a recurrence?We’re testing a plant-based diet — increasing fruits and vegetables and decreasing fat, red and processed meats, sugars, processed, refined foods. One of our chefs says she’s teaching people to cook like their grandmothers.Do you eat this way yourself?I try! It’s better for cardiovascular disease and diabetes prevention. Also, I just feel better.What inspired you to go into this area of research?My stepfather was diagnosed with a brain tumor when I was pretty young. I quickly saw where the medical system failed him. Seeing how our whole family really suffered through that, I thought there was a lot of room for improvement.You have a PhD in epidemiology as well as a degree in naturopathic medicine. Do you feel like you get less respect than you would if you had an MD?Do conventional medical schools tend to want people to have the degrees they offer? Yes. [But] I work at one of the top public health schools, so I’m able to do the work I want to do.Are you concerned that patients will use alternative treatments instead of conventional ones?There are some pretty wacky things that people do. A lot of patients use integrative therapies out of fear, out of hope, or out of a belief system. But medical decisions need to be made based on facts.But this is challenging for anyone to figure out, regardless of education, no?Steve Jobs [the founder of Apple who died in 2011 after initially choosing to treat his rare cancer with alternative therapies rather than surgery] had access to every single resource in the world, and he still was not making evidence-based decisions about his oncology care.Does this idea that we could be doing something to prevent cancer lead to a blame mentality? That patients who haven’t taken and done everything possible feel like they deserve their illness?We don’t want people to be blaming themselves for not meditating enough. We want them to say, “What are the choices I have in front of me?” There are no guarantees with any of these. There are plenty of people who have pristine diets who still die of cancer.You’re working in a high-stress job in New York City. You must be living under a lot of pressure yourself, no?I need to do more yoga.Heather Greenlee is an assistant professor at Columbia University’s Mailman School of Public Health and a past president of the Society for Integrative Oncology. This interview has been edited and condensed. What are the most effective complementary therapies?There’s a lot of strong evidence on the use of mind-body therapies for things like stress management, anxiety, depression. And there’s a growing body of evidence on acupuncture for pain management.How do you personally hope to change integrative medicine?Historically, integrative therapies have been only available to people who were wealthy. If these are really beneficial therapies that have low toxicity, we want to get them to everybody.advertisementlast_img read more

first_img By Sharon Begley May 16, 2016 Reprints About the Author Reprints Bertrand Guay/AFP/Getty Images Does exercise keep the brain young? [email protected] Please enter a valid email address. Gut Check is a periodic look at health claims made by studies, newsmakers, or conventional wisdom. We ask: Should you believe this?The claim: Exercise reduces the risk of 13 kinds of cancer, researchers reported on Monday.Tell me more:The research, in JAMA Internal Medicine, pooled data from 12 earlier studies, the oldest of which began in 1982, of 1.44 million people. That made it the largest-ever analysis of exercise and cancer. Volunteers were asked how much physical activity they’d engaged in over the past year. Half exercised less than about 150 minutes per week, half exercised more. Their health was tracked for seven to 28 years.Compared to the bottom 10 percent of exercisers (measured by minutes of leisure activities, such as walking, running, and swimming each week), the top 10 percent had 42 percent less esophageal cancer, 27 percent less liver cancer, 26 percent less lung cancer (but only for current and ex-smokers), 21 percent less endometrial cancer, 16 percent less colon cancer, and 10 percent less breast cancer. Overall, they had 7 percent less cancer. (They also had a 27 percent higher risk of malignant melanoma, but only in places with higher levels of UV, probably because exercising exposed them to cancer-causing sunlight.) The researchers found no association between exercising and 13 other cancers, including pancreatic, ovarian, and brain.advertisement Related: Tags American Cancer Societycancerexercise Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. Sharon Begley @sxbegle ‘The Biggest Loser’ shows the complexity of keeping weight off Privacy Policy One of those “other factors” is weight. Like smoking, obesity is associated with less exercise and more cancer. Finding that more sedentary people have more cancer could be a roundabout way of seeing that obesity raises the risk of some cancers. When the scientists used standard statistical techniques to factor out body mass index, most of the reductions in cancer risk held up, though the benefits of exercise shrank. Unfortunately, a 2016 paper has cast doubt on whether statistical techniques to control for such confounding factors work as advertised.Even more worrisome are the confounders you don’t even think to control for. The study did not include genetics, which might influence both exercise and cancer risk, said statistician Rebecca Goldin, a professor of mathematics at George Mason University who was not involved in the research. “Other health factors might influence both, too. Imagine you have something simmering before cancer has reared its ugly head, which might cause you to both not exercise and develop cancer.” That would show up as the association the study found, undermining the idea that exercise helps prevent cancer. Gut CheckDoes exercise prevent cancer? Related: Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. “This study highlights that physical activity has benefits for cancer prevention,” said Alpa Patel of the American Cancer Society, who led the study with Steven Moore of the National Cancer Institute.In the United States, men have a 42 percent chance of developing cancer; women, a 38 percent chance. A 7 percent reduction lowers those risks to 39 percent and 35 percent, respectively. Patel called that a “significant” drop, adding that “the more interesting finding is the vast number of cancer sites associated with inactivity.”advertisement Leave this field empty if you’re human: “If people have precancerous illness, there might be latent effects that cause them both to have poor health and not to exercise much,” coauthor Moore agreed.Another concern is that the study compared cancer incidence among the bottom 10 percent of exercisers to the top 10 percent. Those extremes could well differ on many more attributes than just exercise. Someone who works out more than 90 percent of the population might have other habits that avert cancer, including eating a healthy diet and undergoing regular cancer screening (especially for cervical or colon cancer) to detect abnormalities before they become tumors. Exercise enthusiasts and couch potatoes could also differ in socioeconomic characteristics that affect risk of cancer. The study authors tried to control for these, too.The verdict:We all hope that healthy living will keep cancer away, but the evidence that physical activity does that is far from definitive. And even if this study is correct, the benefit is small. Hundreds of studies have examined whether exercise is linked to a lower chance of developing cancer. They have been inconclusive for virtually every kind except colon, breast, and endometrium. And even for those three, the studies did not prove causation. Neither did the new one.Really?An observational study like this — ask volunteers how much they exercised, then see who develops cancer — can’t show that exercise caused a lower risk of cancer. It can only show that the two go together. Proving causality requires assigning some volunteers to exercise, others to be sedentary, and then comparing their cancer incidence. Only that type of study can make sure some third factor is not at work.Smokers, for instance, are less physically active (it’s hard to use a treadmill when you’re hacking or gasping for breath) and more likely to develop lung and other cancers. The researchers acknowledge that they “cannot fully exclude the possibility that diet, smoking, and other factors may affect the results.”last_img read more

first_imgBusinessFruity with a hint of double helix: A startup claims to tailor wine to your DNA Medical geneticist Dr. Jim Evans isn’t impressed. A new wine delivery service called Vinome is promising to deliver “the ultimate personalized wine experience” — customized to your DNA.There isn’t much (or, really, any) science to back it up. But it’s got a very big name in its corner.Vinome just inked a deal with a startup called Helix, which in turn is backed by the world’s biggest DNA sequencing company, the powerhouse known as Illumina. For the past 15 years, Illumina has been selling machines that can quickly decode the human genome. Medical researchers around the world use them. But the company wants to conquer the consumer market, too. That’s why it spent $100 million to launch Helix, which teams up with app developers who can find creative ways to use a customer’s genetic data.advertisement NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. Privacy Policy Where do such recommendations come from?Vinome surveys customers about their taste preferences and looks at their genetic variants in genes such as TAS2R38, a taste receptor. A small body of research suggests variations in markers on that gene can affect whether people can taste a bitter chemical found in certain vegetables — and as a result, whether they like Brussels sprouts.Cofounder and lead scientist Sara Riordan acknowledges that her team found “virtually nothing” in the scientific literature that linked DNA variations with affinities for certain wines. Even the general literature on the genetics of taste and smell is “pretty small,” she said.So Vinome decided to gather its own evidence. The team analyzed about 40 genetic variants across about 500 people. Then they had participants taste and rate a dozen wines and fill out a survey about their taste preferences.The taste test was not designed with a control group — which is generally considered essential for reliable scientific evidence.But all of it was enough to win over Helix. (Helix “understands that there are no genetic studies that show genetic polymorphisms related to individual wines,” Lu said. He also said Helix takes “the underlying scientific content in each of our products very seriously” and noted existing literature on taste genetics as well as the statistically significant associations found in Vinome’s taste test.)Vinome has gone ahead and filed for patents and plans to try to publish its findings in a scientific journal, Andrews said. He’s not deterred by critics who point out that the science behind his venture is flimsy at best.“I hear the skepticism,” Andrews said, “but those people just aren’t wine fans.” Related: “It’s just completely silly. Their motto of ‘A little science and a lot of fun’ would be more accurately put as ‘No science and a lot of fun,’” said Evans, who’s a professor and researcher at the University of North Carolina.“I’d put this in the same category as DNA matching to find your soulmate,” he said. “We just simply don’t know enough about the genetics of taste to do this on any accurate basis.”Vinome is just one in a growing wave of targeted consumer genetic tests, which promise to deliver insights about nutrition, weight, and athletic training based on analysis of cheek cells, blood, or saliva. The tests could greatly expand the market for DNA analysis, to the benefit of companies like Illumina, which lately has been struggling to meet sales forecasts. By Rebecca Robbins Oct. 27, 2016 Reprints Women are fast catching up to men in alcohol consumption — and abuse center_img The company soft-launched online in May and says it’s already shipped about 300 bottles of California wines to dozens of customers. (The minimum purchase is three bottles —  plus genetic testing —  for $199.)  The official launch will come this holiday season.Next year, Vinome will roll out an app in partnership with Helix. In a statement provided to STAT, Dr. James Lu, Helix’s senior vice president of applied genomics, said his company “is excited to partner with Vinome because of their novel, fun and modern approach to building a wine recommendation algorithm.” Lu also compared Vinome to Netflix.A promotional video on Vinome’s website features four people enjoying a picnic — with wine, of course — under a beautiful canopy of trees. “Most things are best when the guessing stops,” the narrator intones — just before a woman with light hair is matched with “aromatic, floral, bright whites” and a man with dark hair is matched with “powerful, textured, bold reds.” Such as selling them wine.For about $65 per bottle, Vinome promises to pick out “great wines that are perfectly paired to you” based on an analysis of 10 genetic variants in your DNA, collected via saliva samples. The company — which is based, of course, in Northern California’s wine country — even incorporated the distinctive double helix of DNA into its logo of a corkscrew.advertisement Leave this field empty if you’re human: But public health experts see reason for concern. There’s rarely a straight line between a genetic variant and a complex trait like fitness, weight — or affinity for a good merlot. A complex interplay of genes and environmental factors shape our lives.“Elevating genes to this kind of status for these kind of complex traits can be inappropriately distracting” from the important social and environmental causes of problems like obesity, said Tim Caulfield, a health policy professor at the University of Alberta who studies consumer genetic testing.(On top of that, he said he saw a certain irony in adding a “deterministic vibe” to wine drinking. “It seems like the last place you’d want it,” he said. “I hope they don’t do that with beer.”)‘Arguably defensible’ scienceVinome acknowledges the skepticism, but CEO Ronnie Andrews says he’s sure they’re on to something.His team includes scientists with backgrounds in molecular diagnostics and cancer genomics.“Listen, we wouldn’t risk our reputations to launch something in this space … if we didn’t feel like we had something that was arguably defensible in terms of the science, but more importantly would present an incredible experience and a fun experience,” said Andrews, who has formerly held executive roles at General Electric Healthcare and Roche. Please enter a valid email address. Related: Episode 4: A shoppers guide to the genome sequencing market Jeff Pachoud/AFP/Getty Images Tags DNAgenetic testingnutritionlast_img read more

first_img Aedes Aegypti mosquito larvae swim in a container displayed at the Florida Mosquito Control District Office, in Marathon, Fla. Wilfredo Lee/AP A proposal to release genetically altered mosquitoes into the United States for the first time hit a possible stumbling block on Tuesday, with voters in the Florida Keys expressing ambivalence about a field trial there.Residents split on the possibility of a trial, with one ballot initiative open only to residents of Key Haven, where the trial would take place, failing, and another initiative open to all residents of surrounding Monroe County being approved.The results could make it more difficult to move ahead with the release of the mosquitoes in Key Haven.advertisement General Assignment Reporter Andrew covers a range of topics, from addiction to public health to genetics. @DrewQJoseph The mosquitoes would have their DNA edited in a way designed to stave off diseases like Zika and dengue, but opponents of the field trial have raised concerns about the impact on the environment and public health.The results of the referendums were nonbinding. The decision now falls to the five board members of the Florida Keys Mosquito Control District, who have the final say on the trial. Most have said they will follow the public vote, so it’s not clear how they will respond to the split.advertisement [email protected] By Andrew Joseph Nov. 8, 2016 Reprintscenter_img PoliticsFlorida Keys voters split on genetically modified mosquito trial Dom Smith/STATIn the Key Haven referendum, almost two-thirds of voters opposed the trial. In the countywide referendum, about 58 percent of voters backed it.The trial, if approved, would authorize the release of Aedes aegypti mosquitoes genetically altered by the British biotech company Oxitec. The genetic modification is aimed to ensure that any offspring produced by the Oxitec mosquitoes and the wild mosquitoes will die before they can mature and breed, cutting the population overall.In field trials in Brazil, Panama, and elsewhere, mosquito populations declined by up to 90 percent following the introduction of the Oxitec mosquitoes. The mosquitoes also helped cut dengue virus transmission in trials, the company says.In Florida, opponents of a trial have argued that they don’t want the area to be used as the site of an experiment and have said they do not believe studies that show the modified mosquitoes are safe for the environment.This summer, however, the Food and Drug Administration gave its nod of approval to the trial, finding that it would be safe for both people and the environment.Dom Smith/STATIf the trial proceeds, the mosquitoes would be released in Key Haven in an experimental zone separated from a control zone by a buffer area. (The mosquitoes only fly a few hundred yards in their lives.) Experts would then compare the mosquito populations in the experimental and control zones to see if the trial did in fact reduce the insects’ numbers.Aedes aegypti mosquitoes have spread dengue in the Florida Keys in the past, and are responsible for the ongoing Zika outbreak in Miami-Dade County. They also transmit chikungunya and yellow fever. About the Author Reprints Andrew Joseph Tags genetically modified organismsmosquitoespolicyZika Viruslast_img read more

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. Log In | Learn More What’s included? Barbara Schaal, president of the American Association for the Advancement of Science. Alissa Ambrose/STAT By Stephanie Simon Feb. 15, 2017 Reprints The big research initiatives launched by President Barack Obama should have enough momentum to keep rolling in the coming years, even if President Donald Trump doesn’t prioritize them, leading advocates for science said Wednesday.Obama, a self-described “science geek,” announced the BRAIN Initiative, which aims to dive down to the cellular level to understand the workings of the human brain, in 2013. The Precision Medicine Initiative came two years later; it seeks to collect detailed health data from at least a million volunteers to fuel research on a variety of diseases. Both projects are housed in the National Institutes of Health. In the Lab Obama’s ‘big science’ initiatives will keep rolling under Trump, AAAS leaders predict 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 What is it? GET STARTED Tags mental healthpolicyresearchWhite Houselast_img read more

first_imgPharmalot 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. APStock Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. @Pharmalot Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED Log In | Learn More Tags policySTAT+ What is it? Once again, the European Medicines Agency is taking action against a clinical research organization in India over faulty study data.In the latest episode, the agency recommends suspending more than 300 drugs that are for sale or are being reviewed for use across the continent based on “misrepresentation of study data” conducted by Micro Therapeutic Research Labs, according to this statement.center_img About the Author Reprints By Ed Silverman March 27, 2017 Reprints European regulators recommend suspending hundreds of drugs over troubled studies [email protected] 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. Ed Silverman What’s included? GET STARTEDlast_img read more

first_imgPharma [email protected] Sage Therapeutics, a biotech developing an experimental treatment for postpartum depression, this week trumpeted strong results from a clinical trial, which cheered investors and pushed up its stock price. But outside the lab, the company’s aggressive efforts to raise awareness of postpartum depression have proved divisive.The campaign’s message: “When it comes to postpartum depression, silence sucks.” It features close-up photos of distressed, tearful women who can’t speak — because they have pacifiers stuck in their mouths. What’s included? News Editor About the Author Reprints What is it? The campaign features women with pacifiers in their mouths and the slogan “Silence Sucks.” Sage Therapeutics By Megan Thielking June 14, 2017 Reprints GET STARTED 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. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Megan Thielking 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. A campaign to raise awareness of postpartum depression hits a nerve @meggophonelast_img read more

Who will pay for CRISPR?

first_imgFirst OpinionWho will pay for CRISPR? CRISPR will allow us to alter our existing genes. But it often relies on using viruses to shuttle the molecular gene-editing systems into our cells, and can be as expensive as other gene therapies.Editas Medicine plans to use CRISPR-Cas9 to treat various diseases, including Leber congenital amaurosis. “Enthusiasm is great for interventions in the eye,” New York University bioethicist Arthur Caplan told me. “They permit trying one eye at a time and it is easy to tell if anything positive happens. Safety is much easier to ensure.” But in its annual report, Editas noted “significant uncertainty” on whether payers would cover the treatment. In fact, a handful of insurance companies (VantageBlue from Blue Cross Blue Shield of Rhode Island, Select Health, and VIVA Health) have issued policy documents that exclude gene therapy from coverage, a move that experts say establishes policy against paying for CRISPR-based therapeutics.The Institute for Clinical and Economic Review released a report in March stating there are 12 to 14 gene therapy candidates that are expected to be among the first for commercial approval. “With payer budgets already stretched, and reining in the costs high on the agenda, both public and private payers will likely balk at the cost of some of these gene-based treatments,” the American Journal of Managed Care wrote in a reflection on the report. “Europe has the lead in approved gene therapies, and the first such drug to be approved had a launch price of $1.4 million. Can the US health care system absorb the cumulative impact of such prices, considering that 10 percent of the population has a rare condition linked to a genetic defect?”Five major gene therapy companies went public last year, suggesting that investors are ready to bet on the commercial prospects. Editas signed a deal with Juno Therapeutics that could be worth up to $737 million. The companies would combine CRISPR with other tactics to trick the immune system’s T cells to fight cancer. Those tactics could include disabling genes in T cells that prevent cancer cells from shutting down a T cell response, and adding bits of genetic code to engineer new receptors into T cells to let them attach to abnormal proteins in cancer cells called neoantigens.Gene and cell therapies that run into the six figures and beyond are poised to heighten the cost of cancer treatments, which not everyone may be able to afford. In fact, oncologist Dr. Siddhartha Mukherjee, author of the bestselling “Emperor of All Maladies,” gave a speech this month at the annual American Society of Clinical Oncology meeting that warned about dividing the world “into the rich who can afford personalized cancer treatment and the poor who cannot.” A few years ago, Dutch drug company uniQure set up a plant in Lexington, Mass., to make a gene therapy called Glybera, at the time the most expensive drug in the world. It used viruses to slip copies of a gene into human cells to restore an enzyme needed to break down fats. The cost? $1.4 million per patient. The company eventually abandoned its bid to bring Glybera to the U.S. and, after having sold it just once in Germany, recently withdrew it from European markets, rendering it a commercial failure.Spark Therapeutics of Philadelphia is vying to bring the first gene therapy to market in the U.S. to treat a rare genetic eye disease called Leber congenital amaurosis 2. Analysts said it could cost a half-million dollars per eye. Like Glybera, Spark’s treatment is a form of traditional gene therapy, which makes use of viruses to get bits of restorative code into our cells.advertisement The ruckus over the CRISPR gene-editing system hides a dark reality: its high cost may make it unaffordable and questions remain whether most insurance companies will pay for it.As CRISPR begins to move forward in clinical trials, there are some signals about how it may — or may not — be received commercially. Other types of gene therapies carry a price tag that is likely to induce sticker shock. If adopted, these therapies will add striking new cost burdens to our health care system.“The cost isn’t coming down,” said Mark Trusheim, director of the Massachusetts Institute of Technology’s NEW Drug Development Paradigms, a think tank working on the problem of how we will pay for expensive new drugs. “Companies will say, ‘We are developing these medicines, just pay us’; insurers will say, ‘We can’t afford it.’”advertisement NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. Orkin and Reilly also like the idea of using U.S. government funds from the Orphan Drug Act, established in 1983, to pay gene therapy companies for their commercial products. Another idea for keeping gene therapy, including CRISPR-based therapies, affordable is that investors could ask insurance companies to buy in bulk. MIT’s Ernst Berndt, inspired in part by volume purchases of vaccines in Africa, has proposed “advanced market commitments” through which insurance groups commit to buying a bunch of expensive drugs. That model that could be applied to gene therapies, but the insurers may not go for it without a bit more give.In 2009, the Biologics Price Competition and Innovation Act created a pathway for approving generic biologics, also known as biosimilars. It may apply to CRISPR-based biosimilars, but generic gene-editing — and thus competition to drive down prices — is unlikely to appear for decades. Cathryn Donaldson, a spokesperson for American’s Health Insurance Plans, noted that a lack of generic forms of CRISPR means “drug makers may charge whatever they want for their branded medication. “In 1968, Garrett Hardin argued in his now-classic essay, “The Tragedy of the Commons,” that a shared-resource system will tend to be depleted by self-interested individuals. He also argued against exponential growth — to which we could add today the growth of biotech valuation.Health care is a limited shared resource, and expensive new technologies could add pressures resulting in unequal access, especially to cancer therapies. Given the aggressive drive for money, and without new approaches in thinking, we are headed for disaster. One of two things will happen: either we will embrace a national health care system with broad access but that severely limits expensive new drugs, gene therapies, and CRISPR-based biologics; or these treatments will be available to only the wealthiest among us who can pay for them, a dystopian vision which is perverse but perhaps more realistic considering the pressures for a return on investment.Writer Jim Kozubek is the author of “Modern Prometheus: Editing the Human Genome with Crispr-Cas9,” published by the Cambridge University Press. Privacy Policy [email protected] APStock Please enter a valid email address. Leave this field empty if you’re human: Tania Bubela, a law and policy expert, and Chris McCabe, a health economist, both at the University of Alberta, will be holding a workshop in late June in Banff, Canada, to explore how to enable access to high-priced technologies. According to Bubela, gene-editing systems such as CRISPR-Cas9 promise to heighten the tension around health care policy. One idea for easing the tension is for regulators to permit drug makers to get reimbursed from insurers before their gene therapy gets FDA approval, while requiring drug makers to collect more data before charging full price — a kind of price control.“Companies will charge whatever the market will bear,” Bubela told me. “I’m not even sure that many of these gene therapies will work, and not all medicine is worth the price.” But if these technologies become broadly used, especially in altering T cells for cancer, payers won’t meet the demands of steep prices, and Bubela predicts that “the system implodes under its own weight.”I believe that part of the problem lies in financial dealings. The Broad Institute, for instance, holds patents to gene editing tools such as CRISPR-Cas9 and CRISPR-Cpf1 and has issued exclusive licenses to Editas to use these tools for medical purposes. It could issue more-affordable CRISPR licenses one gene at a time, say directly to Juno Therapeutics, which now accesses them through its multimillion dollar deals with Editas. But that would cut Editas investors out of the loop. Such deals tend to inflate drug prices, since venture and public investors in Editas demand a cut on each CRISPR application. As investors engage in layers of transactional deals along the top of the food chain, the costs of gene therapies go up while the financiers may shift blame for a lack of patient coverage to insurance companies.Dr. Stuart Orkin, a pediatric oncologist at Boston Children’s Center, and Dr. Philip Reilly, a partner at Third Rock Ventures, an Editas funder, coauthored a paper in Science magazine saying that sticker shock shouldn’t halt commercialization. It can cost $300,000 a year to treat a single hemophilia patient with existing standard treatments and $25,000 to treat a single sickle-cell patient. Given costs like those, one-time gene therapy treatments running into the six figures may be comparatively affordable if an insurer makes payments to a drug-maker over a decade that are tied to the drug’s continued performance. In fact, the idea of spreading payments over years as annuities originated with corporate-friendly FDA commissioner Scott Gottlieb in a 2014 paper he co-authored for the American Enterprise Institute.Other performance-based models are being tested. GlaxoSmithKline, for example, is trying to bring a $665,000 gene therapy to the U.S. to treat an immune system disorder. The company said it will tie the cost of the drug to its performance in patients — with a money-back guarantee. “The reality is it’s very tough, and it doesn’t come easy,” said Jonathan Appleby, a chief scientific officer for the company’s rare disease unit. By Jim Kozubek June 26, 2017 Reprints Related: Broad Institute prevails in heated dispute over CRISPR patents About the Author Reprints Do CRISPR enthusiasts have their head in the sand about the safety of gene editing? Jim Kozubek Tags cancerCRISPRpharmaceuticals Related:last_img read more

first_img Pete Frates, who was taken to Massachusetts General Hospital last week, has been released from the hospital. Steven Senne/AP BOSTON — A man who inspired the ice bucket challenge to raise millions of dollars for Lou Gehrig’s disease research has been released from the hospital.Pete Frates’ family posted on their Facebook page Wednesday evening that Frates is home.Frates posted a picture on Twitter of him surrounded by his family. The post says: “good to b home.”advertisement Pete Frates was diagnosed with amyotrophic lateral sclerosis in 2012. The disease weakens muscles and impairs physical functioning. There is no known cure.advertisement HealthMan who inspired ice bucket challenge is home from hospital The ALS Ice Bucket Challenge raised more than $220 million when it took off worldwide on social media in 2014. By Associated Press July 13, 2017 Reprints The ice bucket challenge, made famous by ALS patient Pete Frates, raised millions. Here’s how the money was used Related: Frates was taken to Massachusetts General Hospital last week. His brother recently told a Boston sports radio show he had his “fingers crossed” that Frates would be released this week. About the Author Reprints Associated Presslast_img read more

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