Pharmalot, Pharmalittle: Valeant feels attacked, Alzheimer’s risk with prostate cancer drug

first_img By Ed Silverman Dec. 8, 2015 Reprints PharmalotPharmalot, Pharmalittle: Valeant feels attacked, Alzheimer’s risk with prostate cancer drug Alex Hogan/STAT About the Author Reprints Ed Silverman Tags ADHDAlzheimer’sValeant Pharmaceuticals Rise and shine, everyone, another busy day is on the way. And we are no exception as we take our regular sojourn to the STAT offices far from the Pharmalot campus. This calls for a few cups of stimulation, which we will have to procure from sources other than our trusty coffee kettle. While we forage, you may enjoy some of items of interest we have assembled below. Hope you conquer the world today and, of course, keep us in mind when you hear something fascinating …Valeant Pharmaceuticals is trying to find a buyer for Paragon Vision Services, a contact lens manufacturing unit, amid scrutiny from the US Federal Trade Commission, Reuters reports. The FTC is looking at whether purchasing Paragon gave Valeant monopoly control over the market for rigid contact lenses. Valeant purchased Paragon last May and acquired Bausch & Lomb in 2013.Meanwhile, Valeant Pharmaceuticals is chafing from the withering attacks on its business practices over the past several weeks, according to The Wall Street Journal. “We are being attacked by everyone. We have few friends to defend ourselves,” Theo Melas-Kyriazi, a Valeant director since 2005, tells the paper.advertisementcenter_img Men taking drugs to block testosterone to treat prostate cancer have nearly twice the risk of developing Alzheimer’s as men using other treatments, writes The Wall Street Journal. A study identified 16,888 patients with non-metastatic prostate cancer between 1994 and 2013. Nearly 2,400 were treated with anti-androgen drugs and had an 88 percent higher risk of an Alzheimer’s diagnosis than those who did not take the drugs.The FDA approved a chewable tablet containing extended-release methylphenidate, which is the key ingredient in Ritalin, for treating ADHD in children six years and older, MedPage Today informs us.advertisement @Pharmalot AstraZeneca plans to use a mobile app to gather information about side effects in three clinical trials studying a treatment for ovarian cancer, Medical Marketing & Media says.TherapeuticsMD stock jumped after the drug maker said a trial of a hormonal treatment to reduce vaginal pain during intercourse was successful, Bloomberg News writes.Drug makers asked the FDA to further explain the intended use of quality metrics data to be collected after the agency issued draft guidance this summer, Regulatory Focus reports.Roche signed a development deal that could be worth more than $500 million with SQZ Biotech, which developed a chip for helping the body’s immune system to fight disease, The Boston Business Journal tells us.Hetero became the first company in India to receive approval to market a generic version of Gilead Sciences’s Harvoni hepatitis C treatment under a non-exclusive licensing agreement, The Economic Times writes.A meta-analysis that compared the safety and efficacy of once-weekly GLP-1RAs treatments for type 2 diabetes failed to find a clear winner, MedPage Today says. [email protected] Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry.last_img read more

Express Scripts sued by compounding pharmacies for alleged antitrust practices

first_imgCompounding involves making a personalized medicine for a patient. But in 2014, the benefits manager began blocking coverage for ingredients used to make ointments, creams, and powders that are found in topical treatments. The company said the average cost for each prescription jumped to $1,100 from $90, and maintained that less costly medicines are readily available.“We have no comment on the litigation, but we will vigorously defend ourselves,” an Express Scripts spokesman wrote us. “We also remain committed to help our clients save money by controlling compound spend and providing access to cost-effective alternatives.”The move to cut back on covered ingredients has riled compounding pharmacies, which have been under a microscope ever since a 2012 outbreak of fungal meningitis was traced to the New England Compounding Center. The episode led to 64 deaths around the country and was described by federal health officials as the worst public health crisis in the United States in decades.In response, the Food and Drug Administration has cracked down on compounding pharmacies by increasing the number of inspections, and, in rare cases, taking legal action to halt allegedly unsafe practices. The justifiable emphasis on safety has forced many compounding pharmacies to enhance operations, but the move by Express Scripts to limit coverage has further pressured their bottom lines.The compounding pharmacies are striking back. In November 2014, three others filed a lawsuit claiming Express Scripts illegally blocked legitimate prescriptions and unfairly forced patients to seek more expensive medicines or simply not seek treatment. The pharmacies maintained the benefits manager violated federal law because it lacks authority to essentially alter terms of health plans.In the latest lawsuit, the benefits manager is accused of increasing rejection rates for claims submitted for compounded medicines; sending “misleading” letters to patients to say the compounded medicines they were prescribed lacked FDA approval and were unsafe; instructing doctors not to write prescriptions for compounded medicines; and creating obstacles for compounding pharmacies to receive reimbursement, among other things.For the 12-month period beginning on Oct. 1, 2014, through Sept. 30, 2015, the economic impact of the moves made by Express Scripts and other pharmacy benefit managers exceeded $100 million, according to the lawsuit. And the compounding pharmacies allege that about half of that is attributed to the actions taken by Express Scripts.The plaintiffs are seeking an injunction against Express Scripts and damages to be determined. Related: “It’s really a threat to compound pharmacy existence as a whole,” Steven Bloch, an attorney for the compounding pharmacies, told us. The lawsuit estimates that the pharmacies lost tens of millions of dollars in business.As a pharmacy benefits manager, Express Scripts negotiates contracts for prescription drug coverage on behalf of corporations, government agencies, and unions, among others. In this role, the company helps determine pricing and access through formularies, which are lists of preferred medicines.advertisement Compounding pharmacies are suing Express Scripts for antitrust and trying to force them out of business. Jeff Roberson/AP Express Scripts has taken “a series of unreasonable restrictions and rules that would make it impossible for [the compounding pharmacies] to fill prescriptions” for patients “and obtain reimbursements that would cover their costs,” according to the lawsuit, which was filed in federal court in St. Louis. The company and the other benefits managers “employed tactics designed to ensure that the compounding pharmacy industry …  cannot survive.”advertisement FDA sues to stop a wayward drug compounder By Ed Silverman Jan. 20, 2016 Reprints For the second time since Express Scripts began blocking coverage of hundreds of ingredients used to make compounded medicines, several compounding pharmacies have filed a lawsuit accusing the pharmacy benefits manager of using illegal tactics.In the latest instance, a half-dozen compounding pharmacies have charged Express Scripts with violating antitrust laws and is attempting to force them out of business. And they allege that other pharmacy benefits managers are conspiring to do the same thing, although none are named as defendants. PharmalotExpress Scripts sued by compounding pharmacies for alleged antitrust practices [email protected] Ed Silverman Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. @Pharmalot About the Author Reprints Tags Express Scriptslawsuitslast_img read more

Climate change is speeding the spread of Lyme disease

first_img Nymphs questing through the forest. The phrase conjures up images of a scene from “Game of Thrones.” But encountering a real nymph on its quest offers a potentially harmful brush with climate change.Immature deer ticks are called questing nymphs. They now inhabit a wide swath of North American forests, but they didn’t always. During early summer, their quest is for blood. The season now starts earlier and lasts longer than it did in the past, which is good for the ticks. But it’s bad for humans, because these ticks carry the bacteria, viruses, and parasites that cause Lyme disease, anaplasmosis, deer tick encephalitis, and babesiosis.I have collected thousands of nymphs as part of my dissertation research on the invasion of Lyme disease across North America. I’ve witnessed along the way that where these ticks thrive has been heavily influenced by humans.advertisement Tags climate changeenvironmental healthLyme disease By Katharine Walter July 1, 2016 Reprints A deer tick resting on a blade of grass. CDC Deer tick invasionEncounters with ticks didn’t always cast a dark shadow over North American summers. Cases of Lyme disease first appeared in 1976 in the woodsy suburb of Lyme, Connecticut. At that time, deer ticks were found only in a hotbed encircling Long Island Sound, along with a small area in Wisconsin.Since the 1970s, deer ticks have rapidly extended their reach north, west, and south. The most recent map shows that deer ticks now roam throughout the eastern coastal states, from Maine to Florida, and across the Midwest. They are now established in 45 percent of US counties. That means the deer tick has more than doubled its reach in the 20 years since the previous map was published.advertisement The spread of Lyme disease has closely followed the spread of the forest nymphs. Lyme disease is now the most common disease transmitted by a vector — a mosquito, tick, or other bug — in United States. More than 30,000 cases are reported each year, and the Centers for Disease Control and Prevention estimates that 10 times as many Americans develop the disease.Regions where ticks that carry Lyme are established (red) or reported (blue). Alex Hogan/STATIn part, ticks are following the spread of one of their favorite sources of blood: deer. As deer populations exploded over the last sixty years, thanks to strict hunting laws and the largely predator-free and deer-friendly landscapes in New England and the Midwest, deer ticks followed. However, the steady crawl of ticks north into Canada can’t be explained by deer alone.Ticks spend the majority of their lives on the forest floor. They are vulnerable to changing local climates and death by freezing, drowning, or desiccation. Warmer winters and longer summers let more ticks survive and thrive further north each year. Warmer temperatures quicken the tick life cycle, too. Tick eggs hatch sooner and ticks spend more time questing for blood, and so are increasingly likely to feast on a human and pass on a disease-causing pathogen. Because more ticks survive and mature more quickly, diseases can be transmitted faster.Species that thrive under climate changeThe barriers we have created — the heated, cooled, and (somewhat) bug-free spaces we inhabit — give us an artificial sense of immunity to the disturbances shaking our fragile ecosystems. Nymphs don’t respect the barriers of urbanization and wealth that protect many Americans from vector-borne diseases. Window screens, socks, and our skin don’t stop the invasion of nymphs, reminding us of our vulnerability to ecological changes brought about by climate change, habitat fragmentation, and deforestation.As we worry about the ability of some species to run from climate change and escape extinction, ticks, mosquitoes, kissing bugs, and the parasites they carry may thrive under climate change. Where will these crawling and flying disease carriers move? And who will be at risk for what were once called tropical diseases? First OpinionClimate change is speeding the spread of Lyme disease [email protected] Related: About the Author Reprints The consequences of climate change will vary dramatically across the globe and are difficult to predict. The yellow fever mosquito (which also carries dengue, Zika, and chikungunya viruses), for instance, is predicted to spread rapidly in some areas, including eastern North America and large parts of southeast Asia, and become less common in others areas, like much of Australia.A changing climate will affect mosquito-borne diseases in subtler ways, too. In a warmer climate, the dengue virus matures more quickly (up to a certain temperature). That means an infected mosquito can more swiftly spread the virus.The consequences of climate change will be felt most profoundly by people living in or near areas where diseases carried by mosquitoes and other vectors are already common, and where poverty makes it difficult to stamp out these diseases.A forest nymph brushing against a hiker doesn’t begin to drink blood immediately. She crawls across the skin, searching for a comfortable dinner spot. She grips her prey with spindly legs and uses knife-like mouthparts to slice into human skin. She secretes cement around the wound, binding herself to her host, and then begins to imbibe. Once attached, this offspring of a changing climate can’t be simply brushed off.Katharine Walter is a graduate student in the Department of Epidemiology of Microbial Diseases at Yale University. Katharine Walter Persistent Lyme disease symptoms aren’t helped by long-term antibiotics last_img read more

European regulator recommends suspending numerous drugs over clinical trial problems

first_img Semler conducted “flawed” studies on generic drugs, including some sold by Novartis, the EMA says. Georgios Kefalas/Keystone/AP By Ed Silverman July 22, 2016 Reprints PharmalotEuropean regulator recommends suspending numerous drugs over clinical trial problems Privacy Policy The European Medicines Agency on Friday recommended suspending the sale of dozens of generic medicines — many of which are sold by Novartis and Teva Pharmaceuticals — over concerns about “flawed” studies that were conducted by an Indian clinical research organization.The move comes three months after the US Food and Drug Administration alerted an untold number of drug makers of problems at the Semler Research Center, which is located in Bangalore. An inspection last fall found “significant instances of misconduct and violations of federal regulations, including the substitution and manipulation of study subject samples.”Two weeks earlier, the World Health Organization had issued a notice to Semler for the same reasons. As we wrote previously, the global health agency had examined company computer servers and found a spreadsheet file containing detailed instructions for manipulating drug samples that were used in clinical trials for its clients. The WHO inspections were conducted early and late last year.advertisement Tags clinical trialsEMAFDA @Pharmalot Leave this field empty if you’re human: As we indicated in an earlier post, this is the second time in the past year that the WHO issued a so-called notice of concern to an Indian CRO. In July 2015, the global health agency cited problems at Quest Life Sciences involving a clinical study of different AIDS drugs and recommended rejecting the study, according to the notice.Meanwhile, regulators in each EU country will have to determine the extent to which specific medicines that were tested by Semler are critically needed before proceeding with suspensions. The EMA noted there is no evidence, to date, that any patients were harmed or that any of the drugs were not effective. FDA and WHO warn about clinical trials run by an Indian company These moves are yet another knock on the Indian pharmaceutical industry, which has been under a microscope in recent years over a raft of quality-control concerns. Numerous drug makers and ingredients suppliers have been issued warnings for various manufacturing gaffes and, in some cases, the FDA has banned shipments into the United States.Last year, for instance, the European Union suspended hundreds of drugs that were authorized for use based on what European regulators called “flawed” studies conducted by GVK Biosciences, a clinical research organization based in Hyderabad. The episode triggered protests from drug makers and caused a brief diplomatic row between the Indian government and the EU.The implications for the generic drug makers affected may be substantial.The EMA will recommend lifting suspensions if drug makers provide alternative data demonstrating bioequivalence. The FDA is requiring the same thing. And so, these companies must decide whether it will be worth the expense of conducting new studies for pending or approved applications. They will also have to review drugs in their pipelines where studies were run by Semler and find a new CRO, which could result in delayed filings.center_img Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. “The findings from (the) FDA and WHO inspections call into question the quality management system in place at Semler, and, thus, on the reliability of the data of all bioequivalence studies, including those used to support marketing authorization applications in the European Union,” the EMA wrote in a statement explaining its decision. Bioequivalence studies are usually the basis for approving generic drugs.Semler could not be reached for comment.advertisement [email protected] Ed Silverman About the Author Reprints Newsletters Sign up for Pharmalot Your daily update on the drug industry. Please enter a valid email address. Related:last_img read more

Trump opioid panel will recommend nationwide drug courts, tightened requirements for prescribers

first_img Lev Facher GET STARTED @levfacher Tags addictionDonald Trumpopioidspolicy WASHINGTON — President Trump’s commission on combating the opioid epidemic plans to encourage the federal government to establish drug courts in every federal judicial district, adjust reimbursement rates for addiction treatment, and streamline federal funding used by state and local governments to implement drug treatment and prevention programs, according to a draft of the panel’s final report.Those steps are among the 53 recommendations laid out in the draft, a copy of which was obtained by STAT. The final report is set to be released on Wednesday. Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Log In | Learn More What’s included? By Lev Facher Oct. 30, 2017 Reprints What is it?center_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. [email protected] About the Author Reprints Trump opioid panel will recommend nationwide drug courts, tightened requirements for prescribers Washington Correspondent Lev Facher covers the politics of health and life sciences. President Trump with New Jersey Gov. Chris Christie, the chairman of the president’s commission on combatting the opioid crisis. Pablo Martinez Monsivais/AP Exclusive 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.last_img read more

With brain implants, scientists hope to translate paralyzed patients’ thoughts into speech

first_img The technology needn’t give these patients the ability to deliver a Shakespeare soliloquy. More and more experts therefore think a system that decodes whether a person is silently saying yes or no or hungry or pain or water is now within reach, thanks to parallel advances in neuroscience, engineering, and machine learning.“We think we’re getting enough of an understanding of the brain signals that encode silent speech that we could soon make something practical,” said Brian Pasley of the University of California, Berkeley. “Even something modest could be meaningful to patients. I’m convinced it’s possible.”advertisement By Sharon Begley Nov. 15, 2018 Reprints Eros Dervishi for STAT Related: Speech BCI faces even higher hurdles. Decoding the intention to articulate a word involves reading more brain signals than movement, and it hasn’t been clear precisely which areas of the brain are involved. The main challenge is that language is encoded in an extensive brain network, and current recording techniques can’t monitor the whole brain with high enough spatial and temporal resolution, said Stephanie Martin of the University of Geneva, who last year won an award for her progress toward a speech BCI.The brain is also very noisy, and the electrical activity that encodes speech tends to get drowned out by other signals. “That makes it hard to extract the speech patterns with a high accuracy,” she said.Current assistive technologies for people whose paralysis, ALS, or other condition leaves them unable to speak “are not very natural and intuitive,” said Martin, who is part of a European consortium on decoding speech from brain activity. Patients gaze at a screen displaying letters, scalp electrodes sense brain waves that encode eye movement and position, and the chosen letters spell words that a speech synthesizer says aloud. The late cosmologist Stephen Hawking, who had ALS, used a system like this. But scientists think they can do better by “directly exploiting neural correlates of speech,” Martin said.Computational neuroscientist Frank Guenther of Boston University developed the first speech BCI way back in 2007. It used electrodes implanted in the brain of a man with locked-in syndrome to eavesdrop on the motor cortex’s plans to speak. They picked up signals corresponding to moving the tongue, lips, larynx, jaw, and cheeks in a way that would produce particular phonemes (though the study got only as far as vowels).The project ended after Guenther’s collaborator, neurologist Phil Kennedy, ran afoul of federal health regulators and was barred from implanting electrodes in any more patients. It didn’t help that Kennedy, frustrated with the field’s slow progress, had his own brain implanted with electrodes, a power coil, and transceiver by a neurosurgeon in Belize in 2014, and initially seemed to have suffered brain damage.Undeterred by such reputational setbacks, other neuroscientists are teaming up with electrical engineers to develop a system of implants, decoders, and speech synthesizers that would read a patient’s intended words, as encoded in brain signals, and turn them into audible speech. One aspect of speech BCI that could one day make their use widespread, Guenther said: The hardware is much less expensive than robot arms, which can cost hundreds of thousands of dollars. The whole process was like translating the operating manual for a Ferrari from Italian to English to Japanese and back to Italian: The final version often sounds nothing like the original. And that’s what previous research on brain-computer interfaces for speech had gotten: a string of mostly unintelligible sounds. “Before this, you couldn’t reconstruct the sounds of speech from electrical data very well,” Mesgarani said.The test for his brain-computer interface was whether the tinny sounds coming from the vocoder bore any resemblance to the sounds of the stories and the digits the participants had heard. They did: Intelligibility reached 75 percent, compared to slightly more than half that for earlier speech BCIs, the scientists report in a paper posted on the bioRxiv preprint site; it has not been peer-reviewed but the authors have submitted it to a journal.Averaging all of someone’s neural responses to a particular speech utterance (repeated many times) improved the accuracy of the reconstructed, synthesized speech, as did taking readings from more electrodes of the 128 in the array.The next step is to test the deep neural network on brain signals evoked by imagining speaking, Mesgarani said. “Previous studies have shown it’s possible” to detect the signals that encode such unspoken speech, he said; the bottleneck has been in the acoustic processing and verbal synthesizer.By improving the back end of a potential speech BCI, he said, “we have a good framework for producing accurate and intelligible reconstructed speech from brain activity,” something he calls “a step toward the next generation of human-computer interaction systems … for patients suffering from paralysis and locked-in syndromes.”What begins as technology for people with disabilities could spread to everyone else — or maybe go in reverse. At a 2017 neurotech conference at the Massachusetts Institute of Technology, Facebook’s Mark Chevillet described the company’s “thought to typing” BCI research as being guided by one question: “What if you could type directly from your brain?”The goal of the project, which he directs, is “to develop a silent speech interface that will let you produce text five times faster than typing, or 100 words per minute.” The company is studying whether high-quality neural signals detected noninvasively (not even the most ardent Facebook-ers would likely agree to brain surgery) can be accurately decoded into phonemes. If so, the next step is to feed the signals into a database that pairs phoneme sequences with words, then use language-specific probability data to predict which word the signals most likely mean (much like auto-fill in Gmail).“This is not science fiction,” Chevillet told the conference. The brain surgeon began as he always does, making an incision in the scalp and gently spreading it apart to expose the skull. He then drilled a 3-inch circular opening through the bone, down to the thick, tough covering called the dura. He sliced through that, and there in the little porthole he’d made was the glistening, blood-flecked, pewter-colored brain, ready for him to approach the way spies do a foreign embassy: He bugged it.Dr. Ashesh Mehta, a neurosurgeon at the Feinstein Institute for Medical Research on Long Island, was operating on his epilepsy patient to determine the source of seizures. But the patient agreed to something more: to be part of an audacious experiment whose ultimate goal is to translate thoughts into speech.While he was in there, Mehta carefully placed a flat array of microelectrodes on the left side of the brain’s surface, over areas involved in both listening to and formulating speech. By eavesdropping on the electrical impulses that crackle through the gray matter when a person hears in the “mind’s ear” what words he intends to articulate (often so quickly it’s barely conscious), then transmitting those signals wirelessly to a computer that decodes them, the electrodes and the rest of the system hold the promise of being the first “brain-computer interface” to go beyond movement and sensation.advertisement Related: Paralyzed man feels touch through mind-controlled robot hand With rehab and a jolt to the spinal cord, paralyzed patients take steps again Leave this field empty if you’re human: Guenther’s 2007 system, he said, “was ancient by today’s standards. I don’t think the problems [that have held back speech BCI] are unsolvable.”Neither does electrical engineer Nima Mesgarani of Columbia University, who is leading a project to reconstruct speech from the signals picked up by electrodes like those Mehta implanted.The reason such a device has a prayer of working is that the human brain doesn’t make hard-and-fast distinctions between fantasy and reality. When the brain imagines something, the neuronal activity is extremely similar, in location and pattern, to when it does something. The mental image of a pumpkin pie produces activity in the visual cortex very much like that while seeing one; imagining taking a jump shot evokes neuronal activity like actually executing one.So it is with “covert” or silent speech: Rehearsing what you’re going to say without moving the lips or tongue “creates the same patterns of brain activity as actually speaking,” Mesgarani said.So does mentally listening to your silent speech. “Think of it as the mind’s ear,” said Berkeley’s Pasley. Say the word giraffe. Then say it silently. Inside your brain, the second syllable should sound louder than the first, and probably rises in pitch. Those and other qualities make up the word’s spectrogram, Pasley explained. STAT+: If all goes well, it will conquer the field’s Everest: developing a brain-computer interface that could enable people with a spinal cord injury, locked-in syndrome, ALS, or other paralyzing condition to talk again. Crucially, brain activity corresponding to the mind’s ear occurs in the auditory cortex, which also hears sounds from the outside world: The overlap, Pasley and his colleagues report in a paper in next month’s Cerebral Cortex, “is substantial.”That allows eavesdropping devices to reconstruct silent speech, if far from perfectly. In a study Martin conducted with Pasley when she was at Berkeley, participants who had electrodes placed in their brains were asked to think about saying aloud a series of words such as cowboys, swimming, python, and telephone. Unfortunately, the accuracy of the software’s interpretation of the resulting brain signals for word pairs such as spoon and battlefield was only slightly better than flipping a coin. That was a big improvement, though, on an earlier system that scored under 40 percent in figuring out what vowel or consonant, not even a whole word, was encoded by brain activity during covert speech.The Berkeley results were good enough for a proof of concept, but not much more. “The reconstructed speech [from that and similar studies] hasn’t been intelligible at all,” Mesgarani said. “We’re trying to overcome the intelligibility barrier.”The best way to do that, he said, is with machine learning, or training software to interpret the brain activity corresponding to covert speech, learn from its mistakes, and get progressively better.To test his ideas, Mesgarani teamed up with Mehta, who recruited five epilepsy patients for the study. During their surgeries, he placed a grid of electrodes (the flat array is called electrocorticography) on the surface of two regions of the auditory cortex: over Heschl’s gyrus and the superior temporal gyrus. The latter contains Wernicke’s area, which figures out what words to use. Both gyruses process features of speech, including volume, intonation, frequency, and, crucially, phonemes — the smallest units of sound, such as “sh,” that comprise a spoken language.The volunteers then listened to people saying digits (“one, two, three …”) and reading stories for 30 minutes. Acoustic processing software extracted the neural activity evoked by listening to speech, essentially a sequence of complex electrical signals. A “deep neural network” that Mesgarani and his team developed to, basically, infer the language sounds that correspond to the neural activity then analyzed that activity. Those inferences were translated back into electrical signals. Those were sent to a vocoder, a synthesizer that produces sounds from features of electrical signals such as frequency and other auditory elements. Senior Writer, Science and Discovery (1956-2021) Sharon covered science and discovery.center_img Further in the future, Facebook and others envision similar technology facilitating consumer products that translate thoughts into text messages and emails. No typing or Siri necessary.The first brain-computer interfaces (BCI) read electrical signals in the motor cortex corresponding to the intention to move, and use software to translate the signals into instructions to operate a computer cursor or robotic arm. In 2016, scientists at the University of Pittsburgh went a step further, adding sensors to a mind-controlled robotic arm so it produced sensations of touch.For all the enthusiastic media coverage they get, brain-computer interfaces are neither routine nor even widely available more than a decade after the first prototypes. Many of the projects floundered after initial excitement. Most such systems require clunky cables as well as large boxes packed with signal analyzers and other electronics, said Pitt’s Jennifer Collinger, who helped develop the tactile robotic arm. She and her colleagues recently got an $8 million grant from the National Institutes of Health to give it to additional patients at Pittsburgh and keep improving the device.In addition, today’s brain electrodes last only a few years, meaning people would need repeated brain surgery, and current BCI systems, while OK in a lab, aren’t reliable enough for real-world use, Collinger said. Sharon Begley Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. In the LabWith brain implants, scientists hope to translate paralyzed patients’ thoughts into speech Please enter a valid email address. Tags neurologyneuroscienceresearch Comparing the Covid-19 vaccines developed by Pfizer, Moderna, and Johnson & Johnson About the Author Reprints Privacy Policy @sxbegle Exclusive analysis of biopharma, health policy, and the life sciences. [email protected] Trending Now:last_img read more

There’s a massive new data set that aims to help artificial intelligence work better for biotech — and it’s free

first_img 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? Kate Sheridan Biotech Apstock About the Author Reprints General Assignment Reporter Kate covers biotech startups and the venture capital firms that back them. [email protected] There’s a massive new data set that aims to help artificial intelligence work better for biotech — and it’s free center_img Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. First 30 days free. GET STARTED By Kate Sheridan May 6, 2019 Reprints Artificial intelligence is the hot new thing in drug discovery and development. AstraZeneca, Pfizer, and Merck have all relied on machine learning to advance their research. Bristol-Myers Squibb and Boston-based Concerto HealthAI established a new partnership in March. And Relay Therapeutics raised an astonishing $400 million series C round to support its efforts to use AI techniques to understand the way proteins bend and twist and create new drugs.But if artificial intelligence programs are actually going to make an impact, they’re going to need a lot of data — and high quality data is hard to come by. Currently available data sets aren’t ideal for machine learning, and relying on that data might even set certain algorithms down the wrong path. 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. @sheridan_kate What is it? Tags biotechnologydrug developmentmedical technologyresearchlast_img read more

The latest failure in Alzheimer’s casts doubt on Biogen’s ostensible success

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. National Biotech Reporter Damian covers biotech, is a co-writer of The Readout newsletter, and a co-host of “The Readout LOUD” podcast. @damiangarde Tags biotechnologyBostonSTAT+ The latest failure in Alzheimer’s casts doubt on Biogen’s ostensible success The prevailing theory of how to treat Alzheimer’s disease endured its 1,001st cut on Thursday, as results from a lengthy clinical trial showed that reducing toxic plaques in the brain had no effect on slowing cognitive decline.While the disappointing result is only the latest in a metronomic series of failures, it could have implications for the drug industry’s only ostensible success: a plaque-targeting treatment from Biogen soon to undergo Food and Drug Administration review. Damian Garde Log In | Learn More GET STARTED What’s included?center_img About the Author Reprints What is it? Biotech [email protected] By Damian Garde April 2, 2020 Reprints Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. In the Alzheimer’s affected brain, abnormal levels of the beta-amyloid protein clump together to form plaques (seen in brown) that collect between neurons and disrupt cell function. National Institute on Aging, NIHlast_img read more

Pharma is only ‘inching forward’ in boosting access to medicines in low-income countries

first_img Ed Silverman [email protected] Pharma is only ‘inching forward’ in boosting access to medicines in low-income countries Unlock this article by subscribing to STAT+ and enjoy your first 30 days free! GET STARTED Pharmalot Columnist, Senior Writer Ed covers the pharmaceutical industry. Adobe Tags drug pricingglobal healthSTAT+ By Ed Silverman Jan. 27, 2021 Reprints 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.center_img What’s included? What is it? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. Like the proverbial tortoise, a new analysis finds that while the pharmaceutical industry is taking steps to provide access to its medicines, it is doing so at a slower pace than is needed for lower-income countries.The most notable sign of progress is in the R&D planning stages, as more drug makers are thinking ahead about access issues. Specifically, eight companies are developing approaches for systematically ensuring all of their R&D projects are positioned to increase access in low-income countries after product launches. Log In | Learn More Pharmalot @Pharmalot GET STARTEDlast_img read more

The parents hoped an existing drug might keep their kids from having seizures. Then they saw the price

first_img What happens next, the company says, depends on the results of the trial.“In terms of access to our medicine, at this point there are no efficacy or safety data for glycerol phenylbutyrate for people living with SLC6A1 or STXBP1. That is the purpose of research such as the [investigator-initiated trial], to begin the process of determining if potential efficacy outweighs potential risk in this unique patient population,” the company said.Even if Ravicti works in Maxwell, Freed is worried that it will be difficult to convince her insurance company to pay for it.“It’s more expensive than a gene therapy, because you seriously could be on this drug for the rest of your life,” she said.Son Rigby appreciates that Horizon is collaborating with the clinical trial and is staying positive, though she lives with a difficult reality: In 2019, she found out that Juno would not qualify for the trial because she doesn’t have seizures.Investigators have decided that all trial participants must be experiencing active seizures, because decreasing seizures is one of the clearest signals they might detect to prove that the drug is working.Son Rigby supports the decision, which will improve the chances that the trial succeeds. To her, it just means that she must do all she can to move the trial full speed ahead, to find out if the drug works, and if so, to convince both Horizon and their insurers that Juno and other children should receive Ravicti.“This is the first thing we’ve been able to get out of the gate,” Son Rigby said. “So let’s push it as hard as we can.” About the Author Reprints Trending Now: By Erika Check Hayden June 3, 2021 Reprints Related: Related: Tags drug pricingneurologypatientspediatricsrare disease Freed connected with Son Rigby, and the two decided to collaborate. Grinspan and Scott Demarest of Children’s Hospital Colorado will test whether Ravicti is safe and well-tolerated in 10 children with STXBP1 mutations and 10 with SLC6A1 mutations. They will also look for improvements in symptoms such as seizures, brain activity, movement disorders, sleep, and quality of life to help inform future studies. The trial is expected to report results in the fall of 2022.The drug seems to work similarly in both Juno’s and Maxwell’s diseases. In both, genetic mutations cause proteins to form incorrectly. This prevents neurons from communicating with each other.In the lab tests, Ravicti seems to fix these defects by acting as a “molecular chaperone,” a type of molecule that stabilizes healthy proteins.“This could be a home run,” Freed said.Maxwell Freed plays at home, while his mom reads to his twin sister, Riley. Kim Leeson for STATThe lab data have already convinced some patients to try the drug related to Ravicti, sodium phenylbutyrate, sold as Buphenyl in the United States.In Belgium, for instance, Leyla Vardar’s son, who carries an SLC6A1 mutation, began taking sodium phenylbutyrate this year. At first, he experienced severe headaches and other side effects, so his doctor reduced his dose. Now, after three months on the drug, Vardar said she has seen “amazing” physical and intellectual improvements in her son, Axel — though one child’s experience isn’t hard evidence that the drug works.Axel’s teachers say he is more engaged in school, his reading has improved, and he interacts more with his parents and the world around him. He’s having fewer physical symptoms, such as seizures and vomiting, and he can jump higher in his soccer games, Vardar said.Axel feels better about himself, and her family is more stable, Vardar said: “This is the first time that we see him building and not losing what he’s doing.”Vardar’s family is paying the full cost of the drug, about 795 Euros a month, or roughly $12,000 per year. But that’s not possible in the United States, where one company sells both Ravicti and Buphenyl. There are as many as 7,000 rare diseases, each found in fewer than 200,000 U.S. patients, but altogether seen in as many as 25-30 million Americans. Many of these diseases are too rare to interest drug companies. So Freed and a growing number of other parents are raising money for clinical trials — as much as $4 million to $7 million for research on cures, such as gene therapies.They’re also funding drug repurposing studies, which aim to find existing drugs to stabilize their children during the long hunt for cures. These trials are usually much cheaper and faster than developing new drugs. Parent foundations are paying $320,000 for the Ravicti trial, which has been in the making for three years. In contrast, developing a new drug can cost billions of dollars over a decade or more. Freed calls the trial — for which the drug’s maker is providing Ravicti for free — a “steal of a deal.”But a trial might not even be necessary if Ravicti — which was approved in 2013 to treat rare urea cycle disorders that disrupt the body’s waste removal system — were less costly. The trial is just the first of many steps needed to ensure that patients can access the drug. Even if the drug appears to help some patients, for instance, insurers might not pay for it based on the results of a small trial. Further studies may be needed, and the company that sells Ravicti might need to agree to apply for FDA approval for STXBP1 and SLC6A1 patients.“The problem with these rare disease drugs is that the prices that are being asked by the drug manufacturers are not based on any reality at all,” said Stacie Dusetzina, associate professor of health policy at Vanderbilt University.“It’s basically pricing as high as the market will bear, and in the rare disease context, that number is very rapidly growing,” Dusetzina said.The motivation for the trial came from a mother named Charlene Son Rigby. In 2016, Son Rigby learned that her daughter, Juno, carried a mutation in the STXBP1 gene. Little was known about the incurable condition, which affects at least 1,000 people worldwide and causes epilepsy, autism, and intellectual and physical disabilities.Juno, now 7, has global developmental delay. She didn’t walk until she was almost 5, and speaks just a few words, including “balloon” and “ball.” Amber Freed looks through photo prints of her son, Maxwell, in her home office in Frisco, Texas. She and other parents are funding a clinical trial to investigate whether a pricey drug can help their children with a rare form of epilepsy. Freed mails the prints out to supporters and those she networks with in her research. Kim Leeson for STAT ‘It’s not a cure’: A gene therapy is opening a new chapter for children, but challenges endure Privacy Policy Comparing the Covid-19 vaccines developed by Pfizer, Moderna, and Johnson & Johnson HealthThe parents hoped an existing drug might keep their kids from having seizures. Then they saw the price When Amber Freed heard in 2020 that a drug might help save her young child from a rare, progressive form of epilepsy, she was ecstatic. The news came just in time. Many children with the genetic mutation that causes her son’s illness begin experiencing debilitating seizures as 4-year-olds. Maxwell was 3.Even better, the drug, Ravicti, was already approved by the Food and Drug Administration, so a doctor might be able to prescribe it right away. But there was a catch: At around $740,000 per year, Ravicti is one of the most expensive drugs in the world.It’s a common story for rare disease drugs, even older “repurposed” ones like Ravicti that aren’t gene therapies or other complex technologies. But what Freed and other parents did next is less common: They are funding a clinical trial to investigate whether Ravicti can help their children, hoping to generate enough data to convince insurers that they should pay for the treatment. One patient has already completed the trial, which aims to enroll 19 more children with rare diseases caused by mutations in two genes, STXBP1 and SLC6A1.advertisement “This is a safe, known drug,” said Freed, who hopes her son will soon enroll in the trial. “A small molecule should not be this hard to get.”advertisement The trial both highlights the increasingly popular strategy of repurposing existing drugs for rare diseases, and exposes growing tensions over the prices for these treatments. Ultra-rare but not forgotten: New drug development paradigms to treat the rarest of diseases Erika Check Hayden Erika Check Hayden is director of the Science Communication Program at the University of California, Santa Cruz. Related: Newsletters Sign up for Daily Recap A roundup of STAT’s top stories of the day. Leave this field empty if you’re human: In 2017, Son Rigby formed the STXBP1 Foundation to spur research into potential gene therapies and other treatments. She soon got promising news. Scientists led by Jacqueline Burré at Weill Cornell Medicine in New York had tested a molecule called 4-phenylbutyrate in brain cells and in C. elegans worms with STXBP1 mutations. The molecule improved some of the damage caused by the genetic mutations, Burré’s team reported in 2018.Weill Cornell pediatric neurologist Zach Grinspan was eager to try the molecule in his patients. It’s sold in two forms, sodium phenylbutyrate and glycerol phenylbutyrate, or Ravicti.Grinspan wanted to use the better-tasting Ravicti in his patients. Since it is already approved, Grinspan could have written his patients an “off-label” prescription for Ravicti, allowing them to try it even though it wasn’t approved to treat their disease.But he realized that insurers would likely balk at the drug’s price. So he began organizing a clinical trial of Ravicti in patients with STXBP1 disorders.At the same time, Freed was learning that her son, Maxwell, also had a rare, incurable childhood epilepsy. Maxwell’s disease results from errors in the gene SLC6A1. When Maxwell received his diagnosis, Freed quit her job as an equity analyst and formed a foundation, SLC6A1 Connect. The foundation funded research on a gene therapy, which may enter clinical trials as soon as this year.In 2020, a researcher funded by SLC6A1 Connect brought hopeful news. Jing-Qiong (Katty) Kang, a Vanderbilt University neurologist, found that Ravicti could help correct some of the defects seen in cells and mice carrying Maxwell’s specific mutation. @Erika_Check Horizon Therapeutics bought both drugs from another company in 2015. Horizon then tripled Ravicti’s price, and has raised Buphenyl’s price from $40,000-$130,000 per year to $243,000.Asked about the price increases, the company told STAT, “Horizon evaluates price adjustments based on several factors including the costs to invest in research to improve our current therapies and develop new rare disease medicines.” The company added in a statement that it has expanded the ages of patients approved to receive Ravicti, provides free genetic testing to patients with urea cycle disorders, and provides financial assistance for patients who need it.It’s not the first time that Horizon has raised a drug’s price by a large margin. In 2013, for instance, it bought the rights to sell the pain drug Vimovo, then increased the drug’s price by 597% in 2014.Indeed, some economists question whether patients’ families should have to pay for the Ravicti trial in STXBP1 and SLC6A1 patients.Ravicti has already generated $1.15 billion in sales for Horizon. At this rate, it could well earn the company enough money to develop its next new drug, said William Padula, a health economist at the University of Southern California.“Expecting the public at this point to help fund their R&D for them, independent of the money they’re making in revenue off of Ravicti, doesn’t seem fair, given the price,” he said.Horizon emphasized in a statement that the current trial “is designed to assess tolerability, safety and pharmacokinetics (the absorption, distribution, metabolism, and excretion) of glycerol phenylbutyrate [Ravicti] for those with STXBP1 or SLC6A1, not efficacy.” Please enter a valid email address. Biogen to expand access to its ALS drug, but move may come too late for some patients last_img read more