Month: <span>June 2021</span>

Donald Trump says Medicare should negotiate drug prices

first_img About the Author Reprints Companies generally can set the prices for approved drugs because the US government doesn’t regulate medicine prices, as other countries do. The powerful pharmaceutical lobby has repeatedly fended off such proposals that would cut into profits.Trump also boasted about his high poll numbers nationwide and in the early voting states of Iowa and New Hampshire. He slammed President Barack Obama and bashed Republican rivals, specifically Jeb Bush and Ted Cruz. By Associated Press Jan. 26, 2016 Reprints Trump says Medicare could save $300 billion if it negotiated drug prices. John Minchillo/AP Related: Washington has big hopes, but little power, to negotiate drug prices center_img PoliticsDonald Trump says Medicare should negotiate drug prices Tags Donald Trumpdrug pricespolicyPresidential campaign Associated Press Republican front-runner Donald Trump says he could save Medicare billions of dollars by getting the massive federal agency to negotiate prices with the major pharmaceutical companies.Trump told an enthusiastic crowd of about 1,000 people packed into a high school gymnasium Monday night in Farmington, N.H., that Medicare could “save $300 billion” a year by getting discounts as the biggest buyer of prescription drugs.Said Trump: “We don’t do it. Why? Because of the drug companies.”advertisementlast_img read more

Pharmalot, Pharmalittle: More Robert Califf opposition, J&J settling thousands of lawsuits

first_img Rise and shine. Another hectic day is on the way. Of course, busy is good, as they say, so no need to kvetch, yes? So time to dig in to the usual routine of deadlines and phone calls and whatnot. As always, we are helped along by a few cups of stimulation, which are especially comforting since a new study says they do not make our heart race any faster. So feel free to join us. Meanwhile, you have a splendid day and let us know if you hear anything interesting …US Senator Joe Manchin (D-W.Va.) plans to filibuster the nomination of Dr. Robert Califf as Food and Drug Administration commissioner, making him the fourth senator to raise objections. “The FDA and Commissioner’s number one priority should be public health and it is inappropriate for the FDA Commissioner to have had such close financial ties with the pharmaceutical industry,” he said in a statement.Artisan Partners, a major shareholder in Johnson & Johnson, urged several activist investors to pressure the company to consider separating its three product divisions, Reuters reports. Artisan, which manages nearly $100 billion, also suggested the health care giant should look at replacing board members and review standards for executive pay and financial reporting.advertisement [email protected] PharmalotPharmalot, Pharmalittle: More Robert Califf opposition, J&J settling thousands of lawsuits @Pharmalot Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Tags FDAJohnson & JohnsonRobert Califf Alex Hogan/STATcenter_img Chinese regulators halted drug imports from four drug makers for violating good manufacturing practices, and India’s Aurobindo Pharma was sanctioned for delaying an inspection, Regulatory Focus says.Despite speculation, Biogen has not specifically ruled out raising prices again this year for its multiple sclerosis drugs, TheStreet tells us.Novartis said US government and commercial insurers are taking longer to cover new medicines than in Europe, which contributed to weak sales of its new Entresto heart failure treatment, Reuters reports.The FDA agreed to review a Merck drug that may prevent the recurrence of the Clostridium difficile superbug, The Wall Street Journal writes.Vertex Pharmaceuticals hasn’t yet provided guidance on its newest cystic fibrosis drug, and The Boston Business Journal provides some reasons. Ed Silverman By Ed Silverman Jan. 28, 2016 Reprints About the Author Reprints Roche CEO Severin Schwan believes drug makers should expect tough scrutiny on pricing ahead of the US presidential election and focus on more innovative products to avoid further pressure, The Financial Times writes. “If you have truly differentiated medicines … then I do believe that societies will continue to reward this innovation and this is particularly true in the US,” he said.Johnson & Johnson is moving to settle thousands of lawsuits filed by women who blamed the company’s vaginal-mesh inserts for their injuries, according to Bloomberg News. The health care giant agreed to pay more than $120 million to resolve between 2,000 to 3,000 lawsuits that alleged women suffered organ damage and constant pain by mesh devices that that were surgically inserted but eroded in their bodies.advertisementlast_img read more

Could this widely used calculator be needlessly driving people to take statins?

first_img Related: The new research strikes at the heart of the risk calculator by saying its core algorithm errs on the high side. It is the largest study to find a severe overestimate of risk, which gives it credibility. And unlike some predecessors, the Kaiser study cannot be criticized for “missing” some strokes and heart attacks; it has complete records for everyone. “We had full follow-up on all of our patients,” said senior author Dr. Alan Go.Another point in its favor is that it jibes with recent research. A 2015 study called MESA, of about 7,000 people, discovered that the risk calculator overestimated the odds of stroke or heart attack by 86 percent in men and 67 percent in women. Of people predicted to have a risk of 7.5 percent to 10 percent, only 3 percent of men or 5 percent of women actually had heart attacks or strokes. Gut Check is a periodic look at health claims made by studies, newsmakers, or conventional wisdom. We ask: Should you believe this?The Claim: The most influential calculator of the risk of having a heart attack or stroke overestimates the likelihood of those events by 500 percent or more. More than 7,000 people access the online tool every day, according to the American College of Cardiology. And physicians use it routinely in their offices to identify people who should be prescribed cholesterol-lowering statins. That suggests many people have been needlessly prescribed the drugs.Tell Me More:The risk tool, released by the American Heart Association and the cardiology group in 2013, combines age, sex, smoking status, cholesterol level, blood pressure, and other factors to calculate a person’s risk of having a heart attack, stroke, or fatal coronary disease in the next 10 years. The idea is to guide therapy more precisely than cholesterol levels alone do; most people with a risk of 7.5 percent or more are advised to take a statin, while at 5 percent they’re told they and their doctors should consider doing so.To see how well the predictions matched reality, researchers at Kaiser Permanente, the large health care provider in California, followed 307,591 nondiabetics aged 40 to 75, none diagnosed with atherosclerosis or prescribed statins. That matched the population the calculator is aimed at. Starting in 2008, the researchers compared the calculated risks to the actual incidence of heart attack, stroke, or death from blocked arteries.advertisement Philippe Huguen/AFP/Getty Images Another likely flaw is that the calculator doesn’t take into account diet and exercise. “If people have a healthier lifestyle, the same ‘inputs’ might add up to less risk” than the calculator shows, said Kaiser cardiologist Dr. Jamal Rana, the study’s lead author. That is, being this age with this cholesterol level and this blood pressure adds up to a lower risk because you’re exercising, avoiding trans fats, and loading up on kale.The Verdict:The new study is not definitive, but more and more evidence says Americans are being told they have a much greater chance of having a stroke or heart attack than they actually do, with millions unnecessarily being prescribed statins. Heart attack patients are getting younger — and more obese Sharon Begley By Sharon Begley May 6, 2016 Reprints TALIA BRONSHTEIN/STATSource: Accuracy of the Atherosclerotic Cardiovascular Risk Equation in a Large Contemporary, Multiethnic Population Watch: Episode 1: Could playing a dolphin in a video game help stroke patients recover? TALIA BRONSHTEIN/STATSource: Accuracy of the Atherosclerotic Cardiovascular Risk Equation in a Large Contemporary, Multiethnic Population Really?The risk calculator was criticized as soon as it was released in 2013, and the skeptics said the new study confirms their suspicions. If physicians use the risk predictions to identify people who should be on a statin, as the cardiology groups recommend, then at least some of those prescribed the powerful drugs “won’t benefit from them,” said epidemiologist Nancy Cook of Brigham and Women’s Hospital in Boston, who was not involved in the new study. Please enter a valid email address. Leave this field empty if you’re human: The heart groups criticized MESA for including patients taking statins, arguing that the drugs were the reason that the incidence of heart attacks and strokes was below what the calculator spat out. The Kaiser study excluded people on statins, so that criticism doesn’t apply.But a related one might. Excluding people who started taking statins during the study “substantially skewed their [final] population to one with the lowest risk of having a heart attack or stroke,” said Dr. Donald Lloyd-Jones of Northwestern University Feinberg School of Medicine, a member of the task force that developed the risk calculator. “Kaiser is renowned for identifying high-risk people and getting them on therapy.” He acknowledged, however, that the calculator is meant for people not already being treated with statins.The new study reported data for only five years, while the calculator’s risks are for 10. But the chance of a heart attack or stroke rose by about the same amount annually, a linear trend that after 10 years would still have left the number of cardiovascular events well below what the calculator predicts.It is not clear why the algorithm overestimates risks. One possibility is that the patients it is based on participated in 1990s-era research and might not be representative of today’s population. For one thing, they were older and whiter.A more interesting explanation is that the inputs for the algorithm — blood pressure, cholesterol, age, and the others — have different consequences in 2016 than they did in the 1990s.For instance, said Kaiser’s Go, blood pressure drugs (which more people are taking than in the 1990s) “might have beneficial effects apart from their effects on hypertension, so a blood pressure of 150/80 might give you a lower risk today than it did in the 1990s.” Yet the calculator assumes that 150/80, and hypertension generally, imparts the same risk of heart attack and stroke as it did decades ago.center_img About the Author Reprints Actual vs. Predicted Rates of Stroke, Heart Attacks, Deaths from Heart Disease in Non-diabetic Women Subgroup (N=189,511)Cumulative 5-year risk observedCumulative 5-year risk expected0%2%4%6%8%10%>=5%3.75% – < 5%2.5% - < 3.75%< 2.5%Cumulative 5-year risk5-year predicted risk group5-year predicted risk groupCumulative 5-year risk observedCumulative 5-year risk expected>=5%0.0150.0843.75% – < 5%0.0070.0432.5% - < 3.75%0.0050.031< 2.5%0.0020.009Cumulative 5-year risk expected Newsletters Sign up for Daily Recap A roundup of STAT's top stories of the day. [email protected] Privacy Policy Actual vs. Predicted Rates of Stroke, Heart Attacks, Deaths from Heart Disease in Non-diabetic Men Subgroup (N=118,080)Cumulative 5-year risk observedCumulative 5-year risk expected0%2%4%6%8%10%>=5%3.75% – < 5%2.5% - < 3.75%< 2.5%Cumulative 5-year risk5-year predicted risk group5-year predicted risk groupCumulative 5-year risk observedCumulative 5-year risk expected>=5%0.0210.0893.75% – < 5%0.0110.0442.5% - < 3.75%0.0080.031< 2.5%0.0040.014Cumulative 5-year risk expected Related: @sxbegle Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery. By 2013 there had been 2,061 such events; according to the risk calculator, there should have been 10,151. Among the nondiabetic patients whose calculated risk was 2.5 percent to 5 percent, the actual chance of suffering a cardiovascular event was roughly one-fourth that amount, the researchers reported in the current Journal of the American College of Cardiology. In the highest-risk group, with a predicted risk above 5 percent, actual risk was only 1.85 percent. Actual risks were closer to predictions for people with diabetes, whom the researchers analyzed separately.The overestimates occurred for both sexes and all races and ethnicities.advertisement Gut CheckCould this widely used calculator be needlessly driving people to take statins? Tags heart diseasestatinsstrokelast_img read more

Medical devices get to market faster in Europe — but are tied to more safety issues

first_img Related: Landmark effort to speed drug approvals nears critical phase in Congress Currently, companies in the United States that want to sell high-risk devices, such as implants designed to support and sustain life, must first demonstrate that their products are reasonably safe and effective — usually through clinical trials. Related: Republicans seize on reports critiquing FDA to push for agency reforms By Sheila Kaplan June 28, 2016 Reprints WASHINGTON — Medical device companies have argued that streamlining the regulatory process at the Food and Drug Administration could help get their devices onto market more quickly and ultimately help save lives.But a new study comparing US and European approaches to medical devices suggests that doing so could also carry significant risks for patient safety.The two-continent study — led by Harvard Medical School researchers — comes as Congress is considering legislation that would make it easier to get drugs and medical devices through the regulatory process.advertisement Kesselheim said the researchers did not expect to find such striking results.The medical device industry has argued that the regulatory process can be streamlined without sacrificing patient safety.One of the industry’s major trade groups, the Advanced Medical Technology Association, said it was reviewing the BMJ study but cautioned that it “touches on a complex issue” that may make it difficult to compare the US and European systems.“It is important to note that the EU has recently taken steps to significantly strengthen its oversight of medical devices,” said Ralph Ives, the group’s executive vice president for global strategy and analysis.Kesselheim said he believes speeding up the approval process for devices in the United States would be problematic.It “moves our regulatory system closer to the European system, at the same time that the people in Europe are trying to come up with proposals to move their system closer to ours,” he said.A bill that would accelerate the approval of drugs and medical devices, known as the 21st Century Cures Act, passed the House late last year. The Senate could consider its own version of the legislation in the coming weeks. In the European Union, by contrast, medical devices can be sold as long as they perform “as intended” and “are likely to be safe.” They generally get to market faster than in the US.advertisement HealthMedical devices get to market faster in Europe — but are tied to more safety issues In the new study, published Tuesday in the BMJ, researchers found that medical devices that were first approved in the European Union were associated with a greater rate of safety issues than devices first approved by FDA.Researchers examined 309 devices, 206 of which were approved by the FDA and the EU. Sixty-three percent were approved first in the EU.The researchers found that the devices approved first by the EU were three times more likely to require safety alerts and recalls.The study was led by Dr. Aaron S. Kesselheim, along with Thomas J. Hwang and Dr. Jessica M. Franklin, all of Harvard, as well as Elisaveta Sokolov of King’s College in London.“There are well-known differences between the systems for device authorization in the US and the EU,” said Kesselheim. “I think there is a concern that the EU is trading speed for safety, and I think that’s what is borne out in the study.” The process for approving medical devices like pacemakers differs between the US and the European Union. APStock Tags FDAmedical devicespolicyregulationslast_img read more

Allergy treatments enter a new era — with a focus on stopping reactions before they start

first_img NewslettersSign up for The Readout Your daily guide to what’s happening in biotech. Sean Parker, the internet mogul of Napster fame, donated $24 million to set up an allergy research center at Stanford University. Nestlé this month invested $145 million in a startup aimed at tackling peanut allergy. And the Broad Institute of Cambridge, Mass., recently launched a new initiative to unravel the basic biology of food allergy.The potential market is huge: It’s estimated that 50 million Americans have allergies. As many as 10 percent of children suffer from hay fever, nearly 12 percent have skin allergies, and 5 percent have food allergies, most commonly peanuts, dairy, and shellfish, according to the 2014 National Health Interview Survey.advertisement WATCH: How EpiPens — and epinephrine — stop a serious allergic reaction Why poison ivy is so itchy — and how scientists are working to quell the rash BusinessAllergy treatments enter a new era — with a focus on stopping reactions before they start “I foresee that a lot of allergy therapies will become more and more specific and targeted, and more customizable to the individual patient,” said Andrew Long, the lead investigational drug pharmacist at Stanford’s Sean N. Parker Center for Allergy & Asthma Research. Answering ‘home brews’ with scienceThe experimental treatment that may be closest to market is also one of the simplest. Bay Area startup Aimmune, backed in part by Nestlé with that $145 million investment, is creating a methodical peanut desensitization pill that slowly weans patients away from allergy.The company has identified the peanut proteins that trigger allergic reaction and is filling little capsules with the stuff. Patients start out by taking half a milligram of peanut protein, and gradually work their way up — over the course of about six months — to the equivalent of eating a single peanut.“The people who need it the most are the people who have the most profound and potentially life-threatening reaction,” said Dr. Daniel Adelman, chief medical officer of Aimmune.The capsules are science’s answer to previous efforts from allergists, who would concoct “home brews” of peanut protein to desensitize patients under the table. Aimmune’s peanut powder is in Phase 3 trials — and, despite its relative simplicity, is still considered something of a trailblazer in allergy science.Tackling multiple allergies at onceResearchers at Stanford University are building on the concept of desensitization.One of the drugs Long is excited about: omalizumab, a biologic drug made by Genentech under the trade name Xolair.To understand how it works, you have to back up and look at why you get all itchy and wheezy when you encounter an allergen. Such responses make sense from an evolutionary standpoint: They initially came into being to protect our bodies against toxins, like snake venom, or to ward away parasites. But they can be deadly in the modern era. Please enter a valid email address. APStock Two types of immune cells — called mast cells and basophils — are the biggest culprits behind allergic reactions. When a person encounters an allergen, let’s say peanut protein, a type of antibody called immunoglobulin E, or IgE, gets activated. This stimulates the mast cells and basophils to release a storm of chemicals that provoke an allergic response to help exorcise peanut protein from the body.Genentech’s drug is an engineered antibody that binds to human IgE, blocking it from triggering that chemical storm.It was approved by the FDA in 2003 to treat a form of asthma often triggered by allergens, but Stanford researchers are now testing it on patients with food allergies — and finding that it might help speed along classic allergy desensitization therapy, in which patients are slowly introduced to escalating quantities of the allergen, via allergy shots or those “home brew” concoctions.“Right now, the process of immunotherapy is painstakingly long — it takes not just weeks or months, but years, to treat a single allergy this way,” said Dr. Toshi Kawakami, a researcher at the La Jolla Institute for Allergy and Immunology.This timeline becomes untenable for patients with multiple allergies — and about 70 percent of people who have an allergy to one type of food will also be allergic to another, Long said.So Stanford has paired Xolair with desensitization therapy to treat up to five different food allergens at any given time. Instead of taking years to gird a patient’s body against peanuts, they’re able to help her control her reactions to, say, hazelnuts, fish, dairy, and wheat as well. Privacy Policy Biotech Correspondent Meghana covers biotech and contributes to The Readout newsletter. By Meghana Keshavan Nov. 29, 2016 Reprints About the Author Reprints Tags biotechnologygeneticsresearch Related: @megkesh Allergy treatments haven’t advanced much in decades, even as hundreds of millions around the world suffer from wheezing, itches, and rashes — and in severe cases, risk death — from exposure to allergens ranging from eggs to pollen to dog dander.But hope may be on the way.Scientists who study the immune system are beginning to understand the root cause of allergies — and are starting to work on next-generation therapies that could stop allergies in their tracks, rather than simply treating symptoms. Private investors and corporations are pouring money into the field.advertisement Looking at IgE from a different angle, Harvard statistical geneticist Liming Liang has been hunting for new drug targets that might modulate how, exactly, the antibody is expressed. In a paper published last year in Nature, Liang’s team found 30 genes that are involved in kicking off the allergic response.“We think we’ll be able to identify a potential drug target here for allergy reaction — but, of course, it’ll take quite a long time to turn that into a medicine used by patients,” Liang said.Testing a DNA vaccine for allergensAnother compelling approach is being taken by Japan’s Astellas Pharma, which is developing a DNA vaccine meant to protect the body from cedar pollen. (Hay fever has been called a “national affliction” in Japan, affecting a quarter of the population.)The underlying research, from Immunomic Therapeutics and Johns Hopkins University, involves attaching a fragment of DNA from, say, cedar pollen to a template vaccine that can embed itself inside the cell. Once there, it revs up an aggressive immune response and imprints an “immunological memory,” which means the immune system will respond even more quickly to future exposures to the allergen.The idea is to create resistance to an allergen without ever having to expose the patient to that substance. If it works, it’d be fairly easy to swap out the cedar pollen DNA for a different allergen — for instance, the genetic material that codes for peanut or cat antigens — and tack it onto this vaccine template.“The beauty of this is that, unlike food desensitization or skin patches, patients aren’t exposed to the circulating antigen,” Long said. “That hypothetically decreases the risk for adverse events.”Overcoming an industry’s fearsFor all the research on allergies, none of the drugs in the pipeline is a sure thing.For a cautionary tale, look no further than Circassia Pharmaceuticals, which has been working on an experimental immunotherapy drug to forestall cat allergies. Interest in Circassia had been sky-high — the small biotech’s market value had shot up to $1 billion earlier this year on speculation that its drug would prove effective.But in a Phase 3 study, it turned out that the placebo effect was as effective as the drug itself. The company’s stock plummeted on that news this past June. Related: Meghana Keshavan Leave this field empty if you’re human: It’s not just that solving allergies is hard. Current medications — such as Benadryl, Claritin, and epinephrine (best known for powering the EpiPen) — do a decent job controlling symptoms in most patients, so there’s little incentive to innovate.“There’s a dearth of new allergy products because antihistamines work so well,” said Dr. Todd Brady, CEO of Aldeyra Therapeutics, a startup in Lexington, Mass., that’s developing a drug to reduce eye irritation from allergies.“They’re generic, they’re cheap, they’re safe, and easy to use — but unfortunately, a lot of patients suffer because of that phenomenon, because not everyone responds to antihistamines,” Brady said.Another reason for the hesitancy: liability concerns. It’s a risky proposition to give patients the foods they’re deathly allergic to — inducing anaphylactic reactions simply to test drugs in clinical trials.“At the end of the day, it’s a fairly 19th-century approach,” said Dr. Wayne Shreffler, a researcher at Massachusetts General Hospital who is working on the Broad Institute’s Food Allergy Science Initiative. “There’s really no doubt that there have been cold feet in the industry about this issue.”But investigators around the world have shown they can carry out these “food challenge” trials safely — which could help hasten the development of new drugs.“In school, we didn’t learn anything about allergy treatment, except antihistamines and epinephrine,” Long said. “But now, we’re seeing this whole new spectrum of treatments. It’ll be interesting to see how this translates into making it into a pharmacy, and into the hands of patients.”Correction: A previous version of the story misstated some aspects of Aimunne’s work on peanut allergies. Related: [email protected] 5 reasons why no one has built a better EpiPen last_img read more

Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease

first_img Biotech Correspondent Meghana covers biotech and contributes to The Readout newsletter. Privacy Policy In the LabSaving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease Newsletters Sign up for Morning Rounds Your daily dose of news in health and medicine. Things had taken a turn for the worse for Mila by November, though. She’d been experiencing seizures for months, but suddenly she was having around 30 every day. Her legs were giving out while she walked; she would choke as she ate food that had already been pureed. Things Mila found funny no longer made her laugh.So Mila’s family began the clinical trial in earnest — despite the fact that milasen had barely been evaluated in animals, and its efficacy was largely unknown. But as long as the drug didn’t cause Mila excruciating pain every day, the answer was simple.“There was no other option,” Vitarello said.Mila began taking milasen in January this year. It’s administered through an IV in her spine, and is meant to be given once every three months. Yu began dosing her cautiously, escalating the amount she was given as it became apparent she could tolerate the treatment.And it seems to be helping.“I’ve seen quite a lot of improvements,” Vitarello said. “To other people they may be small, but to me, they’re huge.”Courtesy Julia VitarelloWhereas Mila once had 30 seizures per day, lasting about two minutes each, she now seizes about five to 12 times a day, and only for a couple of seconds.“The rule with Batten is that the seizures are supposed to get monotonically worse and worse,” Yu said. “Instead, they’re becoming less intense.”She doesn’t slump, but sits upright. Her arms and legs aren’t as spastic. And, most importantly, Mila is more alert. She’s listening carefully when she’s spoken to, and laughs when things are funny. She’s aware of the world around her.“She’s just here, now. She’s present,” Vitarello said.It’s still early, and Yu cautions that his team will understand more about Mila’s progress in the coming months. It’s tough, on a molecular level, to measure how the drug works — the cells most affected by the lethal protein buildup are in the brain.Because Mila’s specific mutation is so rare, it’s unlikely milasen can be used on its own to treat other children with Batten. There’s a chance, however, it may help others who carry the same retrotransposon somewhere in their genome, so Yu’s team examined another 500 full genomes, just to see if a similar retrotransposon would pop up anywhere. So far, they’ve been unable to find one.Still, Yu’s team, and Vitarello, hope that drug development might be similarly fast-tracked for other diseases, and other children.“Right now, we’re bushwhacking through the trees, because there is no clear path,” Vitarello said. “But Mila’s story, I hope, is breaking some ice — so that something similar could happen for other children with a lot of different diseases. There’s no reason it can’t — one step at a time.” Tags geneticspatientsrare disease Researchers behind Biogen’s breakthrough drug win big at ‘Oscars of science’ [email protected] Exclusive analysis of biopharma, health policy, and the life sciences. Comparing the Covid-19 vaccines developed by Pfizer, Moderna, and Johnson & Johnson Related: By Meghana Keshavan Oct. 22, 2018 Reprints LISTEN: Pharma at the ballot box, the latest Alzheimer’s argument, and truly personalized medicine STAT+: Tamar Grossman, director of translational medicine at Ionis Pharmaceuticals, is less hopeful. Her company developed Spinraza, and she said during a public conversation at last week’s American Society of Human Genetics conference in San Diego that the breakneck pace at which milasen was developed will be hard to replicate.“The uniqueness of this program is N of 1: It’s one patient, one disease — a unique situation,” Grossman said.As soon as you have more than one patient, she said, the FDA requires a placebo-controlled trial, and a natural history trial — tracking patients who might have a predisposition for developing the disease, she said.“I’m sorry to disappoint,” she said. “But it can’t happen next week.”There’s also the matter of cost. Although Yu declined to say how much Mila’s treatment cost, funding came from the Batten foundation, Boston Children’s, and Yu’s own research funds.Vitarello acknowledged the “stars did align for Mila’s treatment.” A sick girl’s proactive family found a physician who had the ingenuity — and funding — to take a deep dive into her condition. A groundbreaking new drug, Spinraza, had just months prior been approved — and served as inspiration, and a template, for Yu’s team to follow.“It’s amazing, and inspirational, but it has a context,” said Jannine DeMars Cody, founder and president of the Chromosome 18 Registry & Research Society and a professor at University of Texas Health Science Center at San Antonio. “It’s 30 years of parents and scientists working to create the context so something like this could happen so quickly.”An earlier version of this story incorrectly described the amount of money Vitarello raised. It also incorrectly described the precise target of milasen.center_img “In a way, this is more like a bone marrow transplant or a surgery than a pill,” said Dr. Timothy Yu, the Boston Children’s neurologist who was largely responsible for bringing Mila’s therapy to fruition.But now, nine months into her unique treatment, Mila’s parents — and physicians — are hopeful.“Batten is really cruel, and she lost a lot before she began therapy,” Vitarello said. “But to me it seems her disease has absolutely stopped.”Mila was always an extremely advanced child. Growing up in Colorado, she was skiing by 2 ½, loved biking and hiking, and was a complete chatterbox.But at age 3, Vitarello began noticing subtle things about her daughter that concerned her. At first, Mila’s foot started turning inward. Then at age 4, people would remark that Mila — who had always been so coordinated — was a clumsy little girl. By 5, however, things were becoming more obvious. One guess was autism, but Vitarello knew that wasn’t quite right, given how rapidly her talkative, adventurous daughter had regressed. When Mila’s blindness became apparent, the search for an answer kicked into high gear.After shuffling from neurologist to neurologist, Mila received clinical genetic testing, which revealed she carried one mutation that leads to Batten. And she showed all the hallmark symptoms of the disease. In late 2016, the diagnosis was made.“I felt an incredible sense of joy and relief, which might sound strange, but it’s the truth,” Vitarello said. “This confirmed that I wasn’t dreaming, that I wasn’t crazy — at least I knew what this was.”It was also horrible.Batten disease has a very specific trajectory, and it’s always devastating. It’s part of a family of diseases called lysosomal storage disorders — in which a genetic defect leads to a deficiency of an important metabolic enzyme. This, in turn, leads to a toxic buildup of proteins and lipids in the brain — and those affected end up with degenerative disease.There are more than a dozen different variants of Batten disease, caused by miscoding in slightly different genes, but as Vitarello describes it, the illness combines symptoms of Parkinson’s disease, dementia, and epilepsy, with blindness added in. There’s some work being done in therapeutics for Batten — the Food and Drug Administration last year approved an enzyme replacement drug, Brineura, to treat one form of the disease. But there’s nothing available for Mila’s gene variant, which is called CLN7. As 6-year-old Mila’s favorite movie, “Frozen,” played, her father held a newspaper up in front of her face. She was rapt — her favorite song was on — but she didn’t even flinch.That’s the moment Mila’s parents realized that she had gone completely blind. And they finally knew, now, after months of concern and sinking suspicion, that something was dangerously wrong with their bright, engaging daughter.Mila has Batten disease, an ultra-rare, neurodegenerative genetic disorder. For every child diagnosed, the prognosis has been tragic: Batten is always fatal.advertisement Leave this field empty if you’re human: “When I was first learning about Batten, I saw MRIs with empty brains. Children lifted with mechanical lifts out of their beds, who were previously laughing and playing and going to school,” Vitrarello said.Her way of coping with her daughter’s devastating prognosis was research, and activism. She launched her own philanthropy — Mila’s Miracle Foundation to Stop Batten — and used her skills as an advertising and marketing professional to raise awareness and funds for further study. With the help of crowdfunding site GoFundMe and other sources, Vitarello raised nearly than $3 million — with aims, initially, to fund a gene therapy for her daughter’s variant of the disease.She also reached out to anyone who might be able to help. That, ultimately, led her to the doctor who would change her daughter’s odds for survival: Yu, a neurologist who researched pediatric genetics at Boston Children’s Hospital.Mila’s parents knew that she carried one copy of the CLN7 gene from her father. But they wanted a full molecular diagnosis: They still didn’t know what DNA her mother had contributed — and Batten, as a recessive illness, would only manifest if a person carried two copies of the diseased gene. If they were going to go down the gene therapy route, they needed a complete understanding of Mila’s disease.So Vitarello put out a plea on Facebook, back in January 2017, saying that she needed someone to pore through Mila’s entire genome in search of the missing variant, and to do it quickly. Her post made its way to a Facebook group of physician mothers — and was forwarded along until it found its way to Yu.Yu got in touch with Vitarello, and offered to do whole-genome sequencing for Mila at Boston Children’s. A month later, Yu had found the other copy of the Batten gene.He also had an idea.The mutation Mila had inherited from Vitarello was intriguing — it’s a called a retrotransposon, which in this case was a 2,000-letter stretch of code that moved from one portion of the genome into her CLN7 gene and inserted itself there, altering it. As a result, the protein that this gene was supposed to help manufacture was shortened and ineffective. But the necessary DNA coding was still there — unchanged — to make the right enzymes in Mila’s body. It just was being overshadowed by the retrotransposon, an interloper. Drug development typically takes several years before a new therapy can even make it into clinical trials. Mila, however, made it from diagnosis to bespoke therapy in just over a year. Her case serves as a proof-of-concept in efforts to rapidly develop and deliver precision medicine — as tailored to a single patient. The drug was designed specifically for Mila’s unique mutation, and it’s not clear whether many more children carry it as well, or will benefit from the therapy.advertisement Trending Now: About the Author Reprints Please enter a valid email address. Dr. Timothy Yu (left) with Mila and her mother, Julia Vitarello. Katherine C. Cohen/Boston Children’s Hospital But Mila’s parents, Julia Vitarello and Alek Makovec, now have a glimmer of real hope. Thanks to a remarkable effort on the part of scientists, regulators, and Mila’s family, an experimental therapy — developed in record time — could very well be halting her disease in its tracks. Around this time, a new drug called Spinraza had just been approved by the FDA. It treats a genetic illness called spinal muscular atrophy, and has dramatically improved the lives of children with that debilitating condition. It works by binding to faulty RNA that’s produced by the mutant DNA — allowing the correct portion of RNA to manufacture a functional enzyme. The drug, called an antisense oligonucleotide, worked in the exact way that might have been able to help Mila — if Yu was able to engineer a drug that could bind to her own unique mutation.And that’s exactly what he suggested to Mila’s parents.Yu began looking for a way to synthesize his own oligonucleotide drug. They studied just about every paper linked to Spinraza’s development, and carefully mapped out the intricacies of why Mila’s genes were behaving as they were. They worked closely with FDA regulators. They made a lot of phone calls. They talked to anyone who would listen.“This was an aggressive timeline — perhaps the most aggressive attempt at trying to bring a drug into a human that we’ve seen,” Yu said.The drug was designed by August that year, and scientists began testing it in Mila’s cultured cells. From there, Yu began rallying dozens of scientists who might be able to help with manufacturing, toxicology, and advanced testing. This included members of Boston Children’s experimental therapeutics unit, and others in neurology, genetics, and anesthesia experienced in administering Spinraza.Yu’s team sped through regulatory hurdles, under the FDA’s compassionate use pathway, and a single patient clinical trial was greenlighted by the winter. A contract manufacturer created doses of the drug to administer to Mila. It was named, fittingly, “milasen.” Meghana Keshavan Related: @megkesh last_img read more

We want your pick for the best health and science books to read this summer

first_img [email protected] By STAT staff May 28, 2019 Reprints About the Author Reprints Don’t MissWe want your pick for the best health and science books to read this summer Summer is around the corner. And STAT wants to know which books with health, medicine, or science themes you’d recommend people toss in their vacation bag.Fill out the form below, and you may see your pick in our upcoming list. To get a little inspiration, check out our past summer book lists from 2018, 2017, and 2016.The submission period has ended. STAT staff CHARLY TRIBALLEAU/AFP/Getty Imageslast_img read more

Pharmalittle: Pelosi claims trade deal shows progress on drug issues; Novartis suffers another trial setback

first_img @Pharmalot About the Author Reprints Pharmalot [email protected] Ed Silverman Tags Congressdrug pricingpharmalittleSTAT+ 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. Pharmalittle: Pelosi claims trade deal shows progress on drug issues; Novartis suffers another trial setback Alex Hogan/STAT Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTEDcenter_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. GET STARTED Log In | Learn More What is it? Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. By Ed Silverman Nov. 1, 2019 Reprints What’s included? And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is rather modest. We hope to spend time with one or two short people, enjoy a musical event, and check in on some Pharmalot ancestors. And what about you? This is a fine time to spend time outdoors and enjoy the seasonal changes. You could bolster the economy by purchasing a few sweaters, plan a holiday getaway, or perhaps make time for someone special. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you soon …House Speaker Nancy Pelosi says progress is being made every day toward approving the trade agreement President Trump worked out with Canada and Mexico, Reuters writes. Democrats say they are working closely with the U.S. Trade Representative to get their concerns addressed. They have pressed for measures to ensure good enforcement of labor and climate provisions of the deal, as well as changes in provisions dealing with pharmaceuticals.last_img read more

New data show Bluebird, Bristol CAR-T drug could be pioneering myeloma therapy. Competitors may be close behind

first_imgBiotech New data show Bluebird, Bristol CAR-T drug could be pioneering myeloma therapy. Competitors may be close behind 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. 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. Damian Garde Senior Writer, Biotech Adam is STAT’s national biotech columnist, reporting on the intersection of biotech and Wall Street. He’s also a co-host of “The Readout LOUD” podcast. ORLANDO, Fla. — On the eve of a major blood disease research meeting here, Bluebird Bio and Bristol-Myers Squibb have released results from a pivotal clinical trial of their CAR-T therapy targeting multiple myeloma.The data for the CAR-T treatment called ide-cell (formerly BB2121) have been highly anticipated because they could lead to the first regulatory approval for a personalized cell therapy in this type of lethal blood cancer. The drug achieved all of the study’s efficacy and safety goals. At the same time, the results raise some questions about the treatment’s commercial potential and may leave the door open for competing multiple myeloma therapies to surpass it relatively soon.  What’s included? Log In | Learn More [email protected] Ruby Wallau for STAT @damiangarde center_img Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Adam Feuerstein What is it? About the Authors Reprints [email protected] By Adam Feuerstein and Damian Garde Dec. 6, 2019 Reprints National Biotech Reporter Damian covers biotech, is a co-writer of The Readout newsletter, and a co-host of “The Readout LOUD” podcast. @adamfeuerstein GET STARTED Tags ASHbiotechnologyBostonCAR-Tdrug developmentSTAT+last_img read more

Endocrinologists urge federal lawmakers to act to lower insulin costs

first_img Amid ongoing concerns over the cost of insulin, a leading physicians group is calling for a raft of measures that would increase affordability for the life-saving diabetes treatment, a move that comes as the incoming Biden administration readies its agenda for addressing prescription drug prices.At the top of its list, the Endocrine Society would like to see the federal government negotiate pricing with drug companies, as well as place limits on future wholesale price hikes that are tied to the overall inflation rate and restrict the out-of-pocket costs borne by people with diabetes. What is it? @Pharmalot Unlock this article — plus daily coverage and analysis of the pharma industry — by subscribing to STAT+. First 30 days free. GET STARTED GET STARTED Endocrinologists urge federal lawmakers to act to lower insulin costs What’s included? Ed Silverman About the Author Reprints KEREM YUCEL/AFP via Getty Imagescenter_img 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. By Ed Silverman Jan. 12, 2021 Reprints Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Pharmalot 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] Tags diabetesdrug pricingSTAT+last_img read more