Tag: medicine

  • A Popular Decongestant Doesn’t Work. The FDA Is Finally Doing Something About It

    A Popular Decongestant Doesn’t Work. The FDA Is Finally Doing Something About It

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    In a long-sought move, the US Food and Drug Administration on Thursday formally began the process of abandoning oral doses of a common over-the-counter decongestant that the agency concluded last year is not effective at relieving stuffy noses.

    Specifically, the FDA issued a proposed order to remove oral phenylephrine from the list of drugs that drugmakers can include in over-the-counter products—also known as the OTC monograph. Once removed, drugmakers will no longer be able to include phenylephrine in products for the temporary relief of nasal congestion.

    “It is the FDA’s role to ensure that drugs are safe and effective,” Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “Based on our review of available data and consistent with the advice of the advisory committee, we are taking this next step in the process to propose removing oral phenylephrine because it is not effective as a nasal decongestant.”

    For now, the order is just a proposal. The FDA will open up a public comment period, and if no comments can sway the FDA’s previous conclusion that the drug is useless, the agency will make the order final. Drugmakers will get a grace period to reformulate their products.

    Reviewed Reviews

    The slow-moving abandonment of phenylephrine is years in the making. The decongestant was originally approved by the FDA in 1976, but it came to prominence after the “Combat Methamphetamine Epidemic Act of 2005” came into effect, and pseudoephedrine—the main component of Sudafed—moved behind the pharmacy counter to keep it from being used to make methamphetamine. With pseudoephedrine out of easy reach at drugstores, phenylephrine became the leading over-the-counter decongestant. And researchers had questions.

    In 2007, an FDA panel reevaluated the drug, which supposedly works by shrinking blood vessels in the nasal passage, opening up the airway. While the panel upheld the drug’s approval, it concluded that more studies were needed for a full assessment. After that, three large, carefully designed studies were conducted—two by Merck for the treatment of seasonal allergies and one by Johnson & Johnson for the treatment of the common cold. All three found no significant difference between phenylephrine and a placebo.

    Last year, the FDA reevaluated the drug again, taking into consideration the new studies and taking a deeper look at the 14 studies from the 1950s to 1970s that earned phenylephrine its initial approval. The FDA noted that those 14 studies assessed congestion using a dubious measure of nasal airway resistance that has since been abandoned. But even with the shoddy measurement, the studies provided mixed efficacy results. And the overall finding of efficacy hinged on only two of the studies, which were conducted at the same lab.

    Too Good to Be Real

    No other lab was ever able to replicate the positive results from those two studies. And when FDA scientists carefully looked through the data, they found evidence that some of the numbers could have been fudged and that the results were “too good to be real.”

    As a final nail in phenylephrine’s coffin, modern studies suggest that when phenylephrine is taken orally, it’s highly metabolized in the gut, leaving less than 1 percent of the consumed dose as active in the body. The finding explains why oral doses don’t cause the constriction of blood vessels throughout the body that could lead to an uptick in blood pressure—a side effect sometimes seen with pseudoephedrine. While researchers initially thought the lack of blood pressure increases was a positive finding, in retrospect, it was a hint that the drug wasn’t working.

    With that, a panel of advisers for the FDA voted unanimously, 16 to 0, that oral doses of phenylephrine are not effective at treating a stuffy nose. Afterward, CVS announced that it would remove products that had phenylephrine as the sole active ingredient.

    Despite the seemingly damning evidence, the industry group representing makers of phenylephrine-containing products—the Consumer Healthcare Products Association (CHPA)—still disputed the FDA’s move.

    “CHPA is disappointed in FDA’s proposal to reverse its long-established view of oral PE [phenylephrine],” CHPA CEO Scott Melville said in a statement Thursday. The CHPA maintains its position on the drug’s efficacy. “As science and methods advance, new data should be considered in the context of the full weight of available evidence, not as a complete replacement of the previous body of evidence—especially when considering an ingredient as safely and widely used as PE. CHPA will review the Proposed Order and submit comments accordingly,” Melville said.

    This story originally appeared on Ars Technica.

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  • Conspiracy theorists are turning their attention back to HPV vaccines

    Conspiracy theorists are turning their attention back to HPV vaccines

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    We are living in a vaccine-hesitant moment, with conspiracy theories thriving on social media. We need to push back, says Simon Williams

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  • Donald Trump Vows to Let Robert F. Kennedy Jr. ‘Go Wild on Health’ If Elected

    Donald Trump Vows to Let Robert F. Kennedy Jr. ‘Go Wild on Health’ If Elected

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    At his rally on Sunday at New York City’s Madison Square Garden, former president and Republican presidential nominee Donald Trump said that if elected he would allow wellness conspiracist and anti-vaccine activist Robert F. Kennedy Jr. to “go wild on health.” Kennedy, a former Democrat and scion of the famous political family, initially ran as an independent third-party and potential spoiler candidate, and has spent the better part of two decades spreading conspiracy theories that would likely inform the policies of a Trump administration.

    In August, Kennedy suspended his presidential campaign and threw his weight behind Trump. (Both the Trump and Kennedy campaigns received support from billionaire donor Timothy Mellon.) There were early indications that he might have a place in a possible Trump administration, particularly in some areas focused on health. Kennedy himself even created a spinoff of Trump’s MAGA slogan with his own Make America Healthy Again, or MAHA. But Trump’s speech seems to indicate that Kennedy would indeed have a place in the cabinet, perhaps running Health and Human Services (HHS).

    Kennedy has since hit the campaign trail stumping for Trump alongside another former Democrat and conspiracy theorist, Tulsi Gabbard.

    Kennedy has spent years spreading health mis- and disinformation, particularly about vaccines. In 2014, Kennedy joined Children’s Health Defense (CHD) as a member of its board. CHD pushes debunked conspiracy theories linking conditions like autism with vaccines and other environmental factors. In 2021, Meta banned Kennedy’s Instagram account for spreading disinformation about the Covid-19 vaccine, and he was named by the Center for Countering Digital Hate (CCDH) as one of 12 people responsible for 65 percent of vaccine disinformation across Instagram, Facebook, and Twitter. Thanks to the Covid-19 pandemic, Kennedy’s own profile, as well as that of CHD, began to rise. CHD raised more money in 2021 than it ever had before.

    Meta reinstated Kennedy’s Instagram account last year when he announced his run for the presidency, and it remains up, despite the fact that he is no longer running for office. CHD remains banned from Meta’s platforms. More recently, Kennedy has echoed unfounded conspiracies that could undermine faith in the integrity of the 2024 elections.

    During his presidential campaign, Kennedy tried to distance himself from the anti-vax movement. Still, he continued to spread disinformation, like falsely saying that the Biden administration had violated the Nuremburg Code by mandating vaccines. And his vision for making America healthy again is drastic. Last Friday, he posted on X to warn the Food and Drug Administration (FDA) that its “aggressive suppression of psychedelics, peptides, stem cells, raw milk, hyperbaric therapies, chelating compounds, ivermectin, [and] hydroxychloroquine” was about to end.

    The Department of Health and Human Services oversees 13 agencies, including the FDA and the National Institutes of Health (NIH). In an interview with NBC News while he was still running for president, Kennedy said he would gut those agencies, which he has said are now captured by corporations. He would also impose more testing on already existing vaccines, which health experts told NBC would result in many children being unable to get vaccinated. (Trump, for his part, has claimed he would withhold funding from schools that require vaccination.) Kennedy’s plan would also include dismissing scientists at the NIH who study infectious diseases, focusing instead on the environmental factors and vaccines that he believes cause illnesses.

    During his campaign, he held a health policy roundtable with doctors that pushed fake Covid-19 treatments.

    Trump campaign spokesperson Steven Cheung told WIRED that “President Trump announced a Trump-Vance transition leadership group to initiate the process of preparing for what comes after the election. But formal discussions of who will serve in a second Trump Administration is [sic] premature.”

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  • A Neuralink Rival Says Its Eye Implant Restored Vision in Blind People

    A Neuralink Rival Says Its Eye Implant Restored Vision in Blind People

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    One of these, called the Argus II, was approved for commercial use in Europe in 2011 and in the US in 2013. That implant involved larger electrodes that were placed on top of the retina. Its manufacturer, Second Sight, stopped producing the device in 2020 due to financial difficulties. Neuralink and some others, meanwhile, are aiming to bypass the eye completely and stimulate the brain’s visual cortex instead.

    Hodak says the Prima differs from other retinal implants in its ability to provide “form vision,” or the perception of shapes, patterns, and other visual elements of objects. What users see isn’t “normal” vision though. For one, they don’t see in color. Rather, they see a processed image with a yellowish tint.

    The trial enrolled people with geographic atrophy, an advanced form of age-related macular degeneration, or AMD, that causes gradual loss of central vision. People with the condition still have peripheral vision but have blind spots in their central vision, making it difficult to read, recognize faces, or see in low light.

    In AMD, specialized cells called photoreceptors are damaged over time. Located at the back of the retina, photoreceptors convert light into signals that are sent to the brain. “The photoreceptors are lost but the retina is preserved to a large extent. In our approach, the implant takes the place of the photoreceptors,” says Daniel Palanker, a professor of ophthalmology at Stanford University, who invented the Prima implant.

    Green Pixels

    The Prima implant is a honeycomb pattern of 378 independently controlled pixels that convert infrared light into electrical signals. It measures 2 mm x 2 mm.

    Image Courtesy of Science Corp

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  • The Maker of Ozempic Is Trying to Block Compounded Versions of Its Blockbuster Drug

    The Maker of Ozempic Is Trying to Block Compounded Versions of Its Blockbuster Drug

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    Drugmaker Novo Nordisk is taking action to curb the massively popular compounded semaglutide industry, which provides copies of its blockbuster weight-loss drugs Ozempic and Wegovy to patients—often for much lower prices.

    The Danish pharmaceutical company is lobbying the US Food and Drug Administration to add semaglutide to the agency’s Demonstrable Difficulties for Compounding (DDC) lists, which would block compounding pharmacies from producing dupes of the drug. In a filing posted by the agency on Tuesday, lawyers for Novo Nordisk reason that semaglutide belongs on these lists “due to the complexities associated with their formulations,” among other reasons.

    “These drugs are inherently complex to compound safely, and the risks they pose to patient safety far outweigh any benefits. Novo Nordisk’s aim with this nomination is to ensure that patients receive only FDA-approved, safe, and effective semaglutide product,” says Novo Nordisk director of media relations Jamie Bennett.

    FDA press officer Amanda Hils told WIRED via email that the agency “is reviewing the petition and will respond directly to the petitioner.”

    If granted, the designation would have seismic implications for the compounding industry—and for the likely millions of people currently taking compounded GLP-1 drugs.

    Injectable GLP-1 drugs including semaglutide and tirzepatide have been in shortage since 2022 because of their huge popularity. In the US, when the FDA declares that a drug is in shortage, certain licensed pharmacies are permitted to make “compounded” versions of the medication, which are mixed in-house and are supposed to contain the same active ingredients as the original drug.

    Telehealth providers have capitalized on the GLP-1 drug shortage, offering patients compounded versions via quick virtual appointments. The practice has created tension with the pharmaceutical companies that make the brand-name drugs, since the compounded versions are sold at much lower prices. Ozempic and Wegovy can cost around $1,000 a month without insurance, while compounded semaglutide is advertised for as low as $100 a month online.

    Unlike generic medications, which are manufactured after drug patents expire, compounded medications are not subject to FDA approval before hitting the market. This means that the FDA cannot vouch for the safety, effectiveness, or quality of compounded drugs before they’re sold to patients. The FDA has received multiple reports of adverse side effects, including hospitalization, related to possible dosing errors associated with compounded semaglutide products.

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  • Some fish regrow injured fins and we’re closer to understanding how

    Some fish regrow injured fins and we’re closer to understanding how

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    The African killifish

    The African turquoise killifish (Nothobranchius furzeri) can regrow its fins

    Jill Toyoshiba

    Researchers have uncovered new details about how some fish can regenerate their tail after an injury, which could inch us closer to employing regenerative medicine in people.

    “Humans have all the components to regenerate, but we don’t do it,” says Augusto Ortega Granillo at the Stowers Institute for Medical Research in Missouri. “There’s something missing, and we believe that the missing part, it’s a regulation part.”

    One mystery about animal regeneration is how…

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  • The Gut Might Hold the Key to Treating Long Covid in Kids

    The Gut Might Hold the Key to Treating Long Covid in Kids

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    Yonker and her colleagues will administer larazotide to 32 patients between the ages of 7 and 21, who will take the drug for eight weeks; a further 16 patients will receive a placebo. To qualify for the trial, patients must have a detectable presence of the Covid-19 spike protein in their blood. The intention is to see whether reducing intestinal permeability can make a noticeable difference to the young patients’ symptoms and quality of life.

    Running such a trial has not been straightforward. “It began last year, but we had to put it on pause for a few months because of staffing and drug supply issues,” says Yonker. “One of the requirements is that we need to ensure that the spike protein is present in the blood, which can involve multiple blood draws from these children, which takes time. Because of this I expect it will take one to two more years to recruit all the patients we need, but I would love to move faster.”

    The results, when they emerge, will help researchers determine whether a leaky gut is likely to be a major cause of disease in at least a subset of patients, and whether larazotide should be trialed more widely as a possible treatment.

    There could be other gut-related involvement: Brodin believes that in some children, the virus lingers in the gut rather than being fully excreted, enabling it to actively damage the intestinal wall and contributing to gastrointestinal problems. He suspects that this viral persistence can then induce an autoimmune reaction in the bloodstream, causing further symptoms.

    Yonker’s trial is also encouraging other pediatric long Covid researchers to initiate their own trials, exploring other theories behind the condition. Danilo Buonsenso, a pediatrician at the Gemelli University Hospital in Rome who conducted the first study examining whether children were developing long Covid, described the study as fascinating. He is now trying to get funding for an ambitious trial testing multiple treatments.

    Buonsenso’s work includes studies suggesting that blood clots, as well as inflammation within the lining of the blood vessels known as the endothelium, may play a role in driving certain symptoms. Separately, he led a study that found some children with long Covid also struggle with a condition called postural tachycardia syndrome (POTS) that affects many adults with long Covid, too.

    “In my opinion, there will not be a single drug that will solve long Covid, as multiple things are being documented,” he says. “We need to investigate the role of low-dose anticoagulants with known effects on reducing endothelial inflammation. But we also need to look at specific drugs for POTS and specific medications for the neurocognitive symptoms like chronic pain and headache.”

    Meanwhile, Yonker’s focus on the gut represents a crucial first step. Given the desperation of children suffering from the condition, and their parents, she is hopeful that if her trial proves successful, it will provide an evidence-based treatment option specifically for children. “I think it’s extremely important to advocate for timely treatment advances in children suffering from long Covid rather than wait for trickle-down guidance, based on what we see from trials in adults,” she says.

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  • RSV Can Be a Killer. New Tools Are Identifying the Most At-Risk Kids

    RSV Can Be a Killer. New Tools Are Identifying the Most At-Risk Kids

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    After 25 years as a pediatric infectious diseases specialist, Asunción Mejías is too familiar with the deadly unpredictability of respiratory syncytial virus (RSV), an infection that hospitalizes up to 80,000 children under the age of 5 every year in the US.

    “It’s a disease which can change very quickly,” says Mejías, who works at St. Jude Children’s Research Hospital in Memphis, Tennessee. “I’ve always told my colleagues that for every two children that are admitted, one can go to the ICU in the next three hours and the other one may go home the next day. It’s totally unpredictable.”

    RSV infections are very common, to the point that nearly every child will have one before they turn 2 years old. Most children experience symptoms similar to a cold, like coughing and sneezing, but some can develop severe lung disease: RSV is responsible for more than 100,000 infant deaths globally every year, nearly half of which are in babies under 6 months of age.

    The problem is, aside from a few known risk factors such as premature birth and preexisting lung conditions, it’s hard to tell which children will be worst affected. “Eighty percent of children that end up in the hospital with RSV seem totally healthy,” Mejías says. “They were born full term, and don’t have any risk factors for severe disease.”

    So around the world, different research groups are attempting to train machine learning algorithms or develop statistical models that can indicate which children are most vulnerable to RSV. Based on vast databases of electronic health records, these tools aim to identify groups of risk factors that can help predict which children are more likely to be hospitalized with an infection. Health care providers can then use this information to prioritize the most at-risk children for vaccines and other preventative measures.

    Earlier this year, respiratory epidemiologist Tina Hartert and her colleagues at Vanderbilt University developed one such tool using a statistical model to identify a set of 19 risk factors for RSV, after training it on data from more than 400,000 infants on the Tennessee Medicaid program. “It allows an individual infant’s risk to be calculated at birth,” says Hartert.

    Some of the variables used in the tool are unsurprising. Prenatal smoking, for example, is known to impair lung development in the unborn fetus, making a baby more vulnerable to viral pneumonia, while babies with a low birth weight already lack the strength to breathe normally. However, in many cases, Hartert says it is a combination of different risk factors that converge to make a child vulnerable. “Assessing just individual factors misses lots of at-risk infants,” she says.

    In 2023, regulators in the US approved a vaccine called Abrysvo that is designed to be given to mothers during weeks 32 to 36 of pregnancy, with the aim of ensuring that babies are born with protective antibodies against RSV. They also approved a drug called Beyfortus, a laboratory-made protein called a monoclonal antibody, which can be administered through a single injection to provide protection ahead of the winter RSV season.

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  • Scientists Crack a 50-Year Mystery to Discover a New Set of Blood Groups

    Scientists Crack a 50-Year Mystery to Discover a New Set of Blood Groups

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    There were many challenges in the process of confirming the role of the MAL gene, including a study by rival researchers that suggested a completely different gene could be responsible. “We suddenly thought, ‘Oh no, maybe all this work we’ve been doing has been wasted,’” recalls Tilley. “That was a real low point.” Thornton chimes in: “But we were convinced we were right.”

    In the end, the other study turned out to be wrong, and one of its authors later joined forces with Tilley, Thornton, and their colleagues. Together, the group was subsequently able to prove the significance of the MAL gene in some key experiments. First, following painstaking efforts to find antibodies that would react with it, they established that the crucial AnWj antigen (encoded by the MAL gene) was indeed present on the surface of most people’s red blood cells. Then, they took AnWj-negative blood cells, lacking said antigen, and inserted a complete MAL gene into those cells. This had the hoped-for effect of generating the antigen on the cell surface, turning the cells AnWj-positive. That was definitive proof that the researchers had found the gene responsible for this rare red blood cell variation.

    Now that they know the gene in question, it should make it much easier to find AnWj-negative people who could become blood donors so that, if people affected by this blood group ever need a transfusion, they can have one safely.

    “What they did was really clever,” says Sara Trompeter, a consultant hematologist and pediatric hematologist at University College Hospitals London. Trompeter also works for NHS Blood and Transplant but was not involved in the AnWj study. “They presented it at a conference, some of their early work. It was like watching one of those detective shows where they’re just picking up on tiny clues and testing hypotheses—things that other people might have ignored.”

    Mark Vickers, a hematologist at the University of Aberdeen, who also was not involved in the study, agrees that the results are robust. “They’ve really gone to town and done some very nice work,” he says. “As far as this blood group is concerned, this is going to be the unequivocal landmark paper.”

    There are few indications as to what factors might influence someone to have genes that make their blood AnWj-negative. One family of AnWj-negative individuals in the paper was Arab-Israeli, but the authors stress that there is no clear link to ethnicity at this stage. The vast majority of people who are AnWj-negative are not genetically predisposed to it. Rather, they have such blood because of a hematological disorder or because they have one of the cancers that can affect their MAL gene. “It’s not truly negative. It’s just suppressed,” says Thornton, referring to those cases.

    There are questions remaining though. Babies don’t actually develop the AnWj antigen on their red blood cells until they’re seven days old. The mechanisms as to why that is remain murky. Vickers suggests it could be something to do with the variety of changes that happen in a fetus’s blood around the time of birth—for example, when its dependence on nutrition and oxygen from its mother’s blood ends.

    Tilley, Thornton, and colleagues were also responsible for discovering the genetic basis for the 44th blood group system, called Er, in 2022, as well as the MAM blood group system in 2020, among others. During the past decade or so, blood researchers around the world have described roughly one new blood group system every year, on average. “We’ve got some more in the pipeline,” teases Thornton.

    There are still a handful of enigmatic blood samples—blood that reacts to other people’s blood in unexpected ways—out there, tucked away in lab storages. Scientists—mindful of the patients whose lives are affected by this, who will struggle to find matching blood donors, or who, in some cases, may suffer devastating complications during pregnancy—regularly pore over those samples, hoping to explain them one day.

    At least one more mystery has been solved. Describing how she feels upon seeing her and her colleagues’ paper published at last, and reflecting on nearly 20 years of work, Tilley just says: “It’s a huge relief.”

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  • The Uncertain Path Forward for Psychedelic Medicine

    The Uncertain Path Forward for Psychedelic Medicine

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    But psychedelic compounds are tricky to test in this way because their psychedelic effects are so recognizable to those who take them. In the Lykos trials, around 90 percent of the participants were able to correctly guess whether they received MDMA or a placebo, effectively “unblinding” the study.

    If participants knew they received MDMA, they could have been more receptive to the psychotherapy and felt more positive about the trial experience. And if they knew they hadn’t, they might have been predisposed to think that the psychotherapy they received was less effective. Both scenarios could have influenced how they reported their PTSD symptoms after the MDMA sessions.

    “Once you have an unblinded trial, you potentially have all kinds of questions about efficacy,” says David Rind, chief medical officer of the Boston-based nonprofit Institute for Clinical and Economic Review, which published a report in May raising concerns about the validity of the Lykos trial data.

    Blinded trials with a placebo group are often considered the gold standard in medical research, but Rind says there are other ways to ensure reliable results. For instance, instead of giving participants in the control arm an inert placebo, Lykos could administer a safe but active drug that is known to produce some physiological effects. This would at least leave patients in doubt about what they received, Rind says.

    Another issue Lykos will have to address is the therapy part of its treatment. The company says its treatment manual allows for a “personalized experience,” but FDA advisers had concerns about the variability of psychotherapy offered in the trials. Rind says because Lykos was testing its own psychotherapy protocol in both the drug and placebo groups, rather than an established trauma-focused therapy, it’s hard to know how effective the therapy component was.

    One way to address this would be to study an established trauma therapy in combination with MDMA, or test different psychotherapy approaches head-to-head.

    Sandhya Prashad, president of the American Society of Ketamine Physicians, Psychotherapists, and Practitioners, says the therapy component likely complicated Lykos case to the FDA. “I don’t think the FDA knew what to do with that,” she says.

    She thinks the psychedelics field can learn from the 2019 approval of Spravato. Johnson & Johnson asked the FDA to approve just the drug, rather than the drug alongside psychotherapy.

    Because Spravato can induce disassociation and hallucinogenic effects, the FDA has special requirements around how it is prescribed. It must be administered in a certified medical office where a health care provider can monitor the patient. A patient doesn’t get therapy during the session.

    However, Prashad says she understands why Lykos was seeking approval for MDMA combined with psychotherapy. Compare Spravato to generic ketamine, which was approved as an anesthetic in 1970 and is often given off-label as a depression treatment. Giving ketamine off-label doesn’t come with the same FDA requirements. It’s unregulated as a depression treatment, says Prashad. “You see a lot of subpar quality of care. I think Lykos was trying to prevent that and roll this out in a responsible way.”

    Other companies are pursuing psychedelics solely as a drug rather than combining it with psychotherapy. Biotech company Compass Pathways is testing psilocybin, the active compound in magic mushrooms, in Phase 3 trials for treatment-resistant depression. A licensed medical professional prepares participants for the psilocybin session, observes and is present with them during their session, and provides follow-up support after. Sessions can last six to eight hours. The company notes that this type of psychological support is not psychotherapy.

    Similarly, Beckley Psytech is studying a derivative of DMT given intranasally, as well as an IV version of psilocybin, as potential treatments for depression. Both are designed to have short-acting effects, with the peak experience lasting just 10 to 15 minutes. In the Lykos trials, MDMA sessions lasted eight hours.

    “What we offer during the therapeutic session is support, there’s no psychotherapy,” says Rob Conley, Beckley Psytech’s chief scientific and medical officer. “From a safety standpoint, we think short is good.”

    Whether Lykos will stick with its plans to pursue MDMA-assisted therapy, or pursue approval or just MDMA, remains to be seen. Either way, the company said it remains “deeply dedicated” to bringing MDMA to those suffering from PTSD.

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