In 2005, the reclassification of pseudoephedrine to behind-the-counter status led to widespread use of oral phenylephrine in OTC decongestants, despite evidence of its ineffectiveness. Randy Hatton, a clinical professor in the College of Pharmacy at the University of Florida, and his colleague worked to bring this issue to the FDA's attention, revealing loopholes in the regulatory process for older OTC drugs. Hatton writes in an opinion piece for Scientific American: Before the FDA required that drugs had to be proven effective, it determined whether OTC drugs were effective through expert panels that reviewed existing data. These OTC monographs establish what older OTC ingredients can be marketed without FDA approval. The oral decongestant monograph panel reviewed a few published studies and multiple unpublished studies for phenylephrine. Of the unpublished studies, only four studies showed oral phenylephrine was effective, while seven showed it was no better than placebo. We requested copies of all evidence used by the nasal decongestant review panel via a Freedom of Information Act request and performed a systematic review and meta-analysis ourselves. [...]

The FDA has multiple regulatory processes for different types of medicinal compounds. People are perhaps most familiar with the New Drug Application process, which leads to clinical trials for prescription drug approvals. However, many OTC or nonprescription drugs are regulated differently. In fact, a law passed in 1951, the Durham-Humphrey Amendment to the 1938 Food, Drug, and Cosmetic Act, created the categories of prescription and nonprescription drugs. In 1962, the act was amended again so that drugs had to be shown to be effective, hence the requirement for well-done clinical trials. But what about the drugs that were approved before 1962? This is the loophole that some OTC drugs fall through. For prescription drugs, FDA tried to address pre-1962 approvals through a review of over 3,000 prescription drugs. Most of those drugs have now been reviewed and addressed, but there are still unapproved prescription drugs on the market today, such as an extended-release form of oral nitroglycerin. For nonprescription drugs, FDA established the OTC monograph process 10 years after the 1962 amendment to the Food, Drug, and Cosmetic Act, which required products not proven effective to be reconsidered. FDA formed advisory panels grouping hundreds of ingredients into 26 categories based on the products' uses. After gathering all available information, both published and unpublished, from manufacturers, the advisory panels issued final reports to FDA about whether these ingredients were GRASE (generally recognized as safe and effective), not GRASE, or inconclusive. GRASE ingredients can be used in nonprescription drugs without FDA approval if the use matches the monograph. "The oral phenylephrine example shows that FDA needs more funding to look at these old drugs," concludes Hatton. "We need public funds to support independent researchers who want to examine these products objectively. The government should be able to spend millions to save consumers billions on ineffective products. Companies that market these products have no incentive to prove they don't work. Nonprescription drugs must be effective -- not just safe."

An anonymous reader quotes a report from the Los Angeles Times: It was the late 1980s when Gary Mund felt his pinky tremble. At first it seemed like a random occurrence, but pretty quickly he realized something was seriously wrong. Within two years, Mund -- a crew worker with the Eastern Municipal Water District in Riverside County -- was diagnosed with Parkinson's disease. The illness would eventually consume much of his life, clouding his speech, zapping most of his motor skills and taking away his ability to work and drive. "It sucks," said Mund, 69. He speaks tersely, because every word is a hard-won battle. "I was told the herbicide wouldn't hurt you."

The herbicide is paraquat, an extremely powerful weed killer that Mund sprayed on vegetation as part of his job from about 1980 to 1985. Mund contends the product is responsible for his disease, but the manufacturer denies there is a causal link between the chemical and Parkinson's. Paraquat is manufactured by Syngenta, a Swiss-based company owned by the Chinese government. The chemical is banned in at least 58 countries -- including China and Switzerland -- due to its toxicity, yet it continues to be a popular herbicide in California and other parts of the United States. But research suggests the chemical may cross the blood-brain barrier in a manner that triggers Parkinson's disease, a progressive, neurodegenerative disorder that affects movement. Now, Mund is among thousands of workers suing Syngenta seeking damages and hoping to see the chemical banned.

Since 2017, more than 3,600 lawsuits have been filed in state and federal courts seeking damages from exposure to paraquat products, according to Syngenta's 2022 financial report (PDF). [...] Paraquat is 28 times more toxic than another controversial herbicide, Roundup, according to a report from the Pesticide Action Network. (Roundup has been banned in several parts of California, including a 2019 moratorium by the Los Angeles County Board of Supervisors forbidding its use by county departments.) Paraquat also has other known health effects. It is listed as "highly toxic" on the U.S. Environmental Protection Agency's website, which says that "one small sip can be fatal and there is no antidote." The EPA is currently reviewing paraquat's approval status. However, both the EPA and Syngenta cited a 2020 U.S. government Agricultural Health Study that found there is no clear link between paraquat exposure and Parkinson's disease. A 2021 review of reviews similarly found that there is no causal relationship.

An anonymous reader quotes a report from The Register: It will take 283 years for female representation in IT to make up an equal share of the tech workforce in the UK, according to a report from the British Computer Society, the chartered institute for IT (BCS). BCS has calculated that based on trends from 2005 to 2022, it would take nearly three centuries for the representation of women in the IT workforce -- currently 20 percent -- to reach the average representation across the whole UK workforce, currently at 48 percent. BCS's annual Diversity Report also found that progress towards the gender norm was stalling in IT jobs. Between 2018 and 2021, the proportion of women tech workers rose from 16 percent to 20 percent. But there was no change in 2022, according to BCS analysis of data from the Office for National Statistics.

Julia Adamson, BCS managing director for education and public benefit, said in a statement: "More women and girls need the opportunity to take up great careers in a tech industry that's shaping the world. A massive pool of talent and creativity is being overlooked when it could benefit employers and the economy. There has to be a radical rethink of how we get more women and girls into tech careers, and a more inclusive tech culture is ethically and morally the right thing to do. Having greater diversity means that what is produced is more relevant to, and representative of, society at large. This is crucial when it comes to, for instance, the use of AI in medicine or finance. The fact that 94 percent of girls and 79 percent of boys drop computing at age 14 is a huge alarm bell we must not ignore; the subject should have a broader digital curriculum that is relevant to all young people."

Bill McGuire is professor emeritus of climate hazards at University College London. He also writes on CNN that it's "essential" to laugh in the face of the climate crisis: If you don't laugh, you will cry, and that marks the beginning of a very slippery slope. As civilization faces a threat that dwarfs that of every war ever fought combined, and the outcome of the latest climate COP offers little hope, it's something we need — not only to remember — but to actively adopt as a weapon in our armoury to fight for a better future for our children and their children. They say that laughter is the best medicine, but weaponised comedy has the potential to do more than just make us feel good. Not only can it help inform and educate about global heating and the climate breakdown it is driving, but also to encourage and bolster action...

This is why ventures like "Climate Science Translated," which I took part in earlier this year, are so important. The British-based project — brainchild of ethical insurer Nick Oldridge and the climate communications outfit Utopia Bureau — teams climate scientists up with comedians, who 'translate' the science into bite-sized, funny and pretty irreverent chunks that can be understood, digested and appreciated by anyone.
You can see four of the videos on their web site. "Climate science is complicated," each video begins. "So we're translating it into human."

For example, last month Dr. Friederike Otto, senior lecturer on climate science at London's Imperial College, created a new video with comedian Nish Kumar: Dr. Otto: Human-caused climate change is fundamentally changing the fabric of the weather as we know it. It's leading to events which we've simply never seen before.

Comedian Kumar: Translation: Weather used to be clouds. Now we've made it into a sort of Rottweiler on steroids that wants to chew everyone's head off.

Dr. Otto: The continuing increase in global average temperature is already causing higher probabilities of extreme rainfall and flash flooding, as well more intense storms, prolonged droughts, record-breaking heatwaves, and wildfires.

Kumar: Very soon climate scientists are just going to ditch their graphs and point out the window with an expression that says, "I fucking told you!"

Dr. Otto: This is not a problem just for our children and grandchildren. This is an immediate threat to all our lives.

Kumar: I don't know if you're familiar with the film The Terminator, but if someone came from the future to warn us of this threat, they'd have travelled from next Wednesday.
And three weeks ago a follow-up video came from earth systems science professor Mark Maslin from London's University College, teaming up with comedian Jo Brand: Professor Maslin: We are heading for unknown territory if we trigger tipping points — irreversible threshholds which shift our entire ecosystem into a different state.

Comedian Brand: If you liked climate crisis, you're going to love climate complete fucking collapse...

Professor Maslin: The irony is solar and wind power are now over 10 times cheaper than oil and gas. We can still prevent much of the damage, and end up in a better place for everyone.

Brand: With wind and sun power, we save money, and don't die. It's a pretty strong selling point.

Professor Maslin: Most people actually are in favor of urgent action. The reason governments are not transitioning fast enough is because the fossil fuel industry has a grip on many politicians. In fact, governments subsidize them with our taxpayer money — over $1 trillion a year, according to the IMF.

Brand: We are paying a bunch of rich dudes one trillion dollars a year to fuck up our future. I'd do it for that money. When can I start?
Each video ends with the words "All Hands On Deck Now", urging action by voting, contacting your representative, joining a local group, and protesting.

Climate hazard professor Bill McGuire writes on CNN that he hopes to see a growing movement: As Kiri Pritchard-McLean pointedly observes: "If comedians are helping scientists out, you know things aren't going well...." There is even a "Sustainable Stand-up" course aimed at teaching comedy beginners about how climate and social issues can be addressed in their shows, and which has run in 11 countries.

The Washington Post reports that America's largest pharmacy chains have "handed over Americans' prescription records to police and government investigators without a warrant, a congressional investigation found, raising concerns about threats to medical privacy." Though some of the chains require their lawyers to review law enforcement requests, three of the largest — CVS Health, Kroger and Rite Aid, with a combined 60,000 locations nationwide — said they allow pharmacy staff members to hand over customers' medical records in the store... Pharmacies' records hold some of the most intimate details of their customers' personal lives, including years-old medical conditions and the prescriptions they take for mental health and birth control. Because the chains often share records across all locations, a pharmacy in one state can access a person's medical history from states with more-restrictive laws. Carly Zubrzycki, an associate professor at the University of Connecticut law school, wrote last year that this could link a person's out-of-state medical care via a "digital trail" back to their home state...

In briefings, officials with eight American pharmacy giants — Walgreens Boots Alliance, CVS, Walmart, Rite Aid, Kroger, Cigna, Optum Rx and Amazon Pharmacy — told congressional investigators that they required only a subpoena, not a warrant, to share the records.

A subpoena can be issued by a government agency and, unlike a court order or warrant, does not require a judge's approval. To obtain a warrant, law enforcement must convince a judge that the information is vital to investigate a crime. Officials with CVS, Kroger and Rite Aid said they instruct their pharmacy staff members to process law enforcement requests on the spot, saying the staff members face "extreme pressure to immediately respond," the lawmakers' letter said. The eight pharmacy giants told congressional investigators that they collectively received tens of thousands of legal demands every year, and that most were in connection with civil lawsuits. It's unclear how many were related to law enforcement demands, or how many requests were fulfilled.

Only one of the companies, Amazon, said it notified customers when law enforcement demanded its pharmacy records unless there was a legal prohibition, such as a "gag order," preventing it from doing so, the lawmakers said...

Most investigative requests come with a directive requiring the company to keep them confidential, a CVS spokeswoman said; for those that don't, the company considers "on a case-by-case basis whether it's appropriate to notify the individual."
The article points out that Americans "can request the companies tell them if they've ever disclosed their data...but very few people do.

"CVS, which has more than 40,000 pharmacists and 10,000 stores in the United States, said it received a 'single-digit number' of such consumer requests last year, the letter states."

An anonymous reader shared this report from CNN: The quest for new antibiotics is going back to the Stone Age. The urgency to identify possible candidates has never been greater as the global population faces nearly 5 million deaths every year that are associated with microbial resistance, according to the World Health Organization. A research team led by bioengineering pioneer César de la Fuente is using artificial intelligence-based computational methods to mine genetic information from extinct human relatives such as Neanderthals and long-gone ice age creatures such as the woolly mammoth and giant sloth. The scientists say some of these small protein, or peptide, molecules they have identified have bacteria-fighting powers that may inspire new drugs to fight infections in humans. The innovative work also opens up a completely new way to think about drug discovery.

"It has enabled us to uncover new sequences, new types of molecules that we have not previously found in living organisms, expanding the way we think about molecular diversity," said de la Fuente, Presidential Assistant Professor at the University of Pennsylvania, where he heads the machine biology group. "Bacteria from today have never faced those molecules so they may give us a better opportunity at targeting the pathogens that are problematic today...." The idea to look at extinct molecules came up during a lab brainstorm when the blockbuster movie "Jurassic Park" was mentioned. "The notion (in the film) was to bring back entire organisms, and obviously, they had a lot of issues," De la Fuente said. His team started thinking about a more feasible idea: "Why not bring back molecules from the past?" Advances in the recovery of ancient DNA from fossils mean that detailed libraries of genetic information about extinct human relatives and long-lost animals are now publicly available...

In research expected to publish next year, de la Fuente and his colleagues have developed a new deep-learning model to explore what he describes as the "extinctome" — the protein sequences of 208 extinct organisms for which detailed genetic information is available. The team found more than 11,000 previously unknown potential antimicrobial peptides unique to extinct organisms and synthesized promising candidates... He said that the peptides they discovered displayed "excellent anti-infective activity" in mice.

"Molecular de-extinction offers a unique opportunity to combat antibiotic resistance by resurrecting and tapping into the power of molecules from the past," he said.

Scientists have pinpointed the cause of nausea and vomiting in pregnancy, "finding that the severity of illness is influenced by how much of a hormone called GDF15 the growing fetus makes," reports Science Magazine. The findings have been published in the journal Nature. From the report: GDF15 is present in the blood of nonpregnant people and is known to be associated with nausea; it's also been tested as a weight loss aid because of its tendency to suppress appetite. Levels of the hormone rise sharply in early pregnancy and increase throughout gestation. Pregnant people with higher GDF15 concentrations have been documented as having a higher risk of vomiting and nausea. Some researchers suggest the hormone-caused aversion to some smells and tastes might encourage an expectant parent to avoid foods potentially dangerous to the fetus.

To find out more about GDF15 changes during pregnancy, University of Cambridge physician-scientist Stephen O'Rahilly and colleagues studied half a dozen pregnant people who were known from previous genetic screening to produce a slightly different version of the GDF15 protein from their fetuses. Researchers could take advantage of that difference to trace whether GDF15 in the parent's blood originated in the parental or fetal genome: Almost all of it came from the fetus, O'Rahilly says. The team also took a closer look at the link between GDF15 levels and pregnancy sickness. Consistent with previous research, questionnaires from more than 300 participants showed that people who reported vomiting and nausea had significantly higher levels of circulating GDF15 on average than people without these symptoms. The researchers also found elevated levels of GDF15 in an analysis of more than 50 women hospitalized with hyperemesis gravidarum.

Still, hormone levels alone couldn't explain the difference in sickness severity. "There was a big overlap" in GDF15 levels between the groups, O'Rahilly says. He and co-author Marlena Fejzo, a researcher at the University of Southern California, suspected that people's sensitivity to GDF15 might also play a role. (Fejzo began to study the condition after her own pregnancy loss following hyperemesis gravidarum.) To test the idea, the researchers studied 10 nonpregnant people with a rare genetic variant known to carry a heightened risk of hyperemesis gravidarum. These people had reduced GDF15 levels in their blood, hinting that naturally low levels of the hormone might predispose someone to sickness during pregnancy. The researchers found the opposite when they asked 20 pregnant people with beta thalassemia, a blood disorder associated with high GDF15 levels, about their pregnancy symptoms: Just 5% of this group reported nausea or vomiting. O'Rahilly's lab found a similar pattern in animal experiments.

An anonymous reader shares a report: This year has been marked by many terrifying things, but perhaps the most surprising of the 2023 horrors was ... eye drops. The seemingly innocuous teeny squeeze bottle made for alarming headlines numerous times during our current revolution around the sun, with lengthy lists of recalls, startling factory inspections, and ghastly reports of people developing near-untreatable bacterial infections, losing their eyes and vision, and dying.

Recapping this unexpected threat to health, the Food and Drug Administration on Tuesday released an advisory titled "What You Should Know about Eye Drops" in hopes of keeping the dangers of this year from leaking into the next. Among the notable points from the regulator was this stark pronouncement: No one should ever use any homeopathic ophthalmic products, and every single such product should be pulled off the market. The point is unexpected, given that none of the high-profile infections and recalls this year involved homeopathic products. But, it should be welcomed by any advocates of evidence-based medicine.

A new study from the University of Warwick found that artificial intelligence can analyze X-rays and diagnose medical issues better than doctors. The BBC reports: Software was trained using chest X-rays from more than 1.5m patients, and scanned for 37 possible conditions. It was just as accurate or more accurate than doctors' analysis at the time the image was taken for 35 out of 37 conditions, the University of Warwick said. The AI could reduce doctors' workload and delays in diagnosis, and offer radiologists the "ultimate second opinion," researchers added. The software understood that some abnormalities for which it scanned were more serious than others, and could flag the most urgent to medics, the university said.

To check the results were accurate, more than 1,400 X-rays analysed by the software were cross-examined by senior radiologists. They then compared the diagnoses made by the AI with those made by radiologists at the time. The software, called X-Raydar, removed human error and bias, said lead author, Dr Giovanni Montana, Professor of Data Science at Warwick University. "If a patient is referred for an X-ray with a heart problem, doctors will inevitably focus on the heart over the lungs," he said. "This is totally understandable but runs the risk of undetected problems in other areas".

An anonymous reader quotes a report from STAT: The Food and Drug Administration on Friday approved the world's first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations -- ushering in a new era of genetic medicines for inherited diseases.

In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientific name is exa-cel, is described as a potential cure because the genetic fix enabled by CRISPR is designed to last a lifetime, although confirmation will require years of follow-up. The FDA decision comes three weeks after regulators in the U.K. were the first to clear the drug. Approval in the European Union is expected next year. The FDA is also expected to rule on exa-cel as a treatment for beta thalassemia, another inherited blood disorder, by March 30.

The FDA on Friday also approved another sickle cell treatment, a gene therapy from Bluebird Bio called Lyfgenia. Patients will now have the option of two cutting-edge therapies that provide potentially curative benefits. Scientists Emmanuelle Charpentier and Jennifer Doudna published their first CRISPR paper just over a decade ago. In 2020, the research won the pair a Nobel Prize. Reflecting on the approval of Casgevy, Charpentier told STAT via email that she was "excited and pleased" for what it means for patients and their families.

The Biden administration is threatening to cancel the patents of some costly medications to allow rivals to make their own more affordable versions. The Associated Press reports: Under a plan announced Thursday, the government would consider overriding the patent for high-priced drugs that have been developed with the help of taxpayer money and letting competitors make them in hopes of driving down the cost. In a 15-second video released to YouTube on Wednesday night, President Joe Biden promised the move would lower prices. "Today, we're taking a very important step toward ending price gouging so you don't have to pay more for the medicine you need," he said.

White House officials would not name drugs that might potentially be targeted. The government would consider seizing a patent if a drug is only available to a "narrow set of consumers," according to the proposal that will be open to public comment for 60 days. Drugmakers are almost certain to challenge the plan in court if it is enacted. [...] The White House also intends to focus more closely on private equity firms that purchase hospitals and health systems, then often whittle them down and sell quickly for a profit. The departments of Justice and Health and Human Services, and the Federal Trade Commission will work to share more data about health system ownership.

While only a minority of drugs on the market relied so heavily on taxpayer dollars, the threat of a government "march-in" on patents will make many pharmaceutical companies think twice, said Jing Luo, a professor of medicine at University of Pittsburgh. "If I was a drug company that was trying to license a product that had benefited heavily from taxpayer money, I'd be very careful about how to price that product," Luo said. "I wouldn't want anyone to take my product away from me."

Researchers at Tohoku University in Japan found that wasabi improves both short- and long-term memory. CBS News reports: Rui Nouchi, the study's lead researcher and an associate professor at the school's Institute of Development, Aging and Cancer, told CBS News the results, while based on a limited sample of subjects without preexisting health conditions, exceeded their expectations. "We knew from earlier animal studies that wasabi conferred health benefits," he said in an interview from his office in northeast Japan. "But what really surprised us was the dramatic change. The improvement was really substantial."

The main active component of Japanese wasabi is a biochemical called 6-MSITC, a known antioxidant and anti-inflammatory known to exist in only trace amounts elsewhere throughout the plant kingdom, Nouchi said. The double-blind, randomized study involved 72 healthy subjects, aged 60 to 80. Half of them took 100 milligrams of wasabi extract at bedtime, with the rest receiving a placebo. After three months, the treated group registered "significant" boosts in two aspects of cognition, working (short-term) memory, and the longer-lasting episodic memory, based on standardized assessments for language skills, concentration and ability to carry out simple tasks. No improvement was seen in other areas of cognition, such as inhibitory control (the ability to stay focused), executive function or processing speed.

Subjects who received the wasabi treatment saw their episodic memory scores jump an average of 18%, Nouchi said, and scored on average 14% higher than the placebo group overall. The researchers theorized that 6-MSITC reduces inflammation and oxidant levels in the hippocampus, the area of the brain responsible for memory function, and boosts neural plasticity. Compared with the control group, the study said, subjects dosed with wasabi "showed improved verbal episodic memory performance as well as better performance in associating faces and names, which is often the major memory-related problem in older adults." But here's the rub: most of the "wasabi" you order at sushi restaurants is made of ordinary white horseradish, dyed green. "Genuine wasabi must be consumed fresh, with the stubbly rhizome, or stem of the plant, grated tableside just before eating," notes the report. "On the plus side, just a small dab offers the same benefits as the capsule supplements used in the Tohoku study, or 0.8 milligrams of 6-MSITC."

The study has been published in the journal Nutrients.

schwit1 shares a report from the New York Post: A landmark study out of the University of Adelaide and University of Essex has found that living in a private rental property accelerates the biological aging process by more than two weeks every year. The research found renting had worse effects on biological age than being unemployed (adding 1.4 weeks per year), obesity (adding 1 week per year), or being a former smoker (adding about 1.1 weeks). University of Adelaide Professor of Housing Research Emma Baker said private renting added "about two-and-a-half weeks of aging" per year to a person's biological clock, compared to those who own their homes.

"In fact, private rental is the really interesting thing here, because social renters, for some reason, don't seem to have that effect," Professor Baker told the ABC News Daily podcast. She said the security of social renting -- aka public housing -- and homeownership has compared to people living with an end-of-lease date on their calendars. "When you look at big studies of the Australian population, you see that the average rental lease is between six and 12 months," she said. "So even if you have your lease extended, you still are living in that slight state of kind of unknowingness, really not quite secure if your lease is actually going to be extended or not." "We think that that is one of the things that's contributing to loss of years, effectively."

Traumatic memories had their own neural mechanism, brain scans showed, which may help explain their vivid and intrusive nature. From a report: At the root of post-traumatic stress disorder, or PTSD, is a memory that cannot be controlled. It may intrude on everyday activity, thrusting a person into the middle of a horrifying event, or surface as night terrors or flashbacks. Decades of treatment of military veterans and sexual assault survivors have left little doubt that traumatic memories function differently from other memories. A group of researchers at Yale University and the Icahn School of Medicine at Mount Sinai set out to find empirical evidence of those differences.

The team conducted brain scans of 28 people with PTSD while they listened to recorded narrations of their own memories. Some of the recorded memories were neutral, some were simply "sad," and some were traumatic. The brain scans found clear differences, the researchers reported in a paper published on Thursday in the journal Nature Neuroscience. The people listening to the sad memories, which often involved the death of a family member, showed consistently high engagement of the hippocampus, part of the brain that organizes and contextualizes memories. When the same people listened to their traumatic memories -- of sexual assaults, fires, school shootings and terrorist attacks -- the hippocampus was not involved.

[...] Indeed, the authors conclude in the paper, "traumatic memories are not experienced as memories as such," but as "fragments of prior events, subjugating the present moment." The traumatic memories appeared to engage a different area of the brain -- the posterior cingulate cortex, or P.C.C., which is usually involved in internally directed thought, like introspection or daydreaming. The more severe the person's PTSD symptoms were, the more activity appeared in the P.C.C. What is striking about this finding is that the P.C.C. is not known as a memory region, but one that is engaged with "processing of internal experience," Dr. Schiller said.

The U.S. Food and Drug Administration is reviewing a cutting-edge therapy called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment. "If approved, exa-cel, made by Boston-based Vertex Pharmaceuticals and the Swiss company CRISPR Therapeutics, would be the first FDA-approved treatment that uses genetic modification called CRISPR," reports CNN. From the report: CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop. [...] The new exa-cel treatment under FDA consideration can use the patient's own stem cells. Doctors would alter them with CRISPR to fix the genetic problems that cause sickle cell, and then the altered stem cells are given back to the patient in a one-time infusion.

In company studies, the treatment was considered safe, and it had a "highly positive benefit-risk for patients with severe sickle cell disease," Dr. Stephanie Krogmeier, vice president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel. Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshapen red blood cells block normal circulation and can cause moderate to severe pain. It's the top reason patients with sickle cell go to the emergency room or are hospitalized. Before the treatment, patients experienced about four of these painful crises a year, resulting in about two weeks in the hospital.

The FDA sought the independent panel's advice, in part, because this would be the first time the FDA would approve a treatment that uses CRISPR technology, but Dr. Fyodor Urnov, a professor in the Department of Molecular and Cell Biology at the University of California, Berkeley, reminded the committee CRISPR has been around for 30 years and, in that time, scientists have learned a lot about how to use it safely. "The technology is, in fact, ready for primetime," Urnov said. With this kind of genetic editing, scientists could inadvertently make a change to a patient's DNA that is off-target, and the therapy could harm the patient. [...] The FDA is expected to make an approval decision by December 8.

"To celebrate my life, I've arranged to buy up others' medical debt and then destroy the debt," reads a posthumous tweet posted Tuesday after the death of 38-year-old Casey McIntyre.

The Washington Post explains... McIntyre, who served as publisher at Razorbill, an imprint of Penguin Random House, was diagnosed in 2019 and proceeded through treatment without taking on debt, [husband Andrew Rose] Gregory told The Washington Post. But many fellow cancer patients she met were in more precarious financial positions, Gregory added. "We were both so keenly aware that Casey had great health insurance through Penguin Random House," said Gregory, 41. "Casey had no medical debt...."

About nine months before McIntyre died, her husband came across a video online about members of a North Carolina church who purchased nearly $3.3 million of local residents' medical debt for $15,048 in a "debt jubilee," a historical reference to ancient stories about personal debts being canceled at regular intervals. The couple chose to make monthly donations to RIP Medical Debt, the same organization that partnered with the North Carolina churchgoers. The nonprofit organization aims to abolish medical debt "at pennies on the dollar," according to its website. For every $100 donated, the company relieves $10,000 of medical debt. As of Saturday, nearly $200,000 had been donated to RIP Medical Debt in McIntyre's memory, which would wipe out approximately $20 million of unpaid medical bills. [By Sunday afternoon it had risen to over $334,000...]

Allison Sesso, president and chief executive of RIP Medical Debt, said her organization found out about McIntyre's case after McIntyre's posthumous social media post went viral. Sesso said the pace of donations was record-setting for her charity. "What an incredible gesture to the world as you're exiting," Sesso told The Post. "This final act of generosity is blowing up. The amount that they're raising and the rate at which this has gone is not something that we're used to."
McIntyre's post on X has now received 65,400 likes — and 3,086 reposts.

According to a new study published in JAMA Internal Medicine, women in the U.S. are now projected to live about six years longer than U.S. men. TIME reports: [T]he 2021 data represent the largest gender-based life expectancy gap in the U.S. since 1996. The gulf began to widen before the COVID-19 pandemic, the authors note, but the trend accelerated from 2019 to 2021. Deaths from COVID-19 and unintentional injuries, a category that includes accidental drug overdoses, were the largest contributors to the widening of the gap, but differential rates of homicide, heart disease, and suicide deaths also played a role, according to the report. It's well-established that men die of these causes more frequently than women, and in recent years, they have been some of the most common causes of death overall. Heart disease, COVID-19, and unintentional injuries accounted for three of the top five in 2021.

The gender gap would have been even wider, the authors note, but for factors including increases in maternal mortality and decreases in cancer deaths among men. Overall, the data underscore the continued importance of limiting COVID-19's spread, and of finding better ways to improve national mental health and prevent drug overdoses and suicides -- fatalities sometimes labeled by experts as "deaths of despair."

Doctors have long relied on a few key patient characteristics to assess risk of a heart attack or stroke, using a calculus that considers blood pressure, cholesterol, smoking and diabetes status, as well as demographics: age, sex and race. Now, the American Heart Association is taking race out of the equation. From a report: The overhaul of the widely used cardiac-risk algorithm is an acknowledgment that, unlike sex or age, race identification in and of itself is not a biological risk factor. The scientists who modified the algorithm decided from the start that race itself did not belong in clinical tools used to guide medical decision making, even though race might serve as a proxy for certain social circumstances, genetic predispositions or environmental exposures that raise the risk of cardiovascular disease.

The revision comes amid rising concern about health equity and racial bias within the U.S. health care system, and is part of a broader trend toward removing race from a variety of clinical algorithms. "We should not be using race to inform whether someone gets a treatment or doesn't get a treatment," said Dr. Sadiya Khan, a preventive cardiologist at Northwestern University Feinberg School of Medicine, who chaired the statement writing committee for the American Heart Association, or A.H.A. The statement was published on Friday [PDF] in the association's journal, Circulation. An online calculator using the new algorithm, called PREVENT, is still in development.

Britain's drugs regulator has approved a groundbreaking treatment for two painful and debilitating lifelong blood disorders, which works by "editing" the gene that causes them. From a report: The Medicines and Healthcare products Regulatory Agency (MHRA) has given the green light for Casgevy to be used to treat sickle cell disease and beta thalassemia. It is the first medicine licensed anywhere that works by deploying gene editing that uses the "genetic scissors," known as CRISPR, for which its inventors won the Nobel prize for chemistry.

Casgevy's developers hope the pioneering treatment could banish the pain, infections and anaemia sickle cell disease brings and the severe anaemia experienced by those with beta thalassemia. About 15,000 people in the UK, almost all of African or Caribbean heritage, have sickle cell disease. About 1,000 -- mainly of Mediterranean, south Asian, south-east Asian and Middle Eastern background -- have beta thalassemia and need regular blood transfusions to treat their anaemia. Experts in the illnesses hope Casgevy may be a cure, making it no longer necessary for people with the conditions to have a bone marrow transplant. Until now this has been the only treatment available, even though the body can reject the donor marrow. The Sickle Cell Society welcomed the MHRA's decision as a "historic moment for the sickle cell community" which "offers [them] newfound hope and optimism."

An anonymous reader quotes a report from NBC News: A prolonged decline in male fertility in the form of sperm concentrations appears to be connected to the use of pesticides, according to a study published Wednesday. Researchers compiled, rated and reviewed the results of 25 studies of certain pesticides and male fertility and found that men who had been exposed to certain classes of pesticides had significantly lower sperm concentrations. The study, published Wednesday in Environmental Health Perspectives, included data from more than 1,700 men and spanned several decades. "No matter how we looked at the analysis and results, we saw a persistent association between increasing levels of insecticide and decreases in sperm concentration," said study author Melissa Perry, who is an environmental epidemiologist and the dean of the College of Public Health at George Mason University. "I would hope this study would get the attention of regulators seeking to make decisions to keep the public safe from inadvertent, unplanned impacts of insecticides." [...]

Scientists have long suspected changes to the environment could be contributing, and they've been probing the role of pesticides for decades in studies of animals and in human epidemiology research. The new analysis focuses on two groups of chemicals -- organophosphates and some carbamates -- that are commonly used in insecticides. The researchers looked at data collected from groups of people with exposures to pesticides and others who were not. Most, but not all, of the research centered on exposures in the workplace. The researchers controlled for outside factors that could contribute to lower sperm counts like smoking and age. Perry said researchers aren't sure how pesticides are affecting sperm concentrations and more research will be needed.

It's likely that pesticides are one of many environmental factors that could be contributing to a decline in sperm concentrations. The trend of sperm concentration declines has been widely observed in studies around the world, but it's a complicated topic and some scientists still have reservations. Sperm are notoriously difficult to count and the technology to do so has changed over the years. There are many confounding factors that can affect male fertility, including age, obesity and opioid use, to name a few. Sperm concentrations are one important data point to consider, but other factors -- like how sperm are shaped and how they swim -- are also critical to male fertility. Perry said she hopes agencies like the Environmental Protection Agency begin to factor the impact of chemicals and pesticides on reproductive health in their assessments. "Given the body of evidence and these consistent findings, it's time to proactively reduce these insecticide exposures for men wanting to have families," Perry said.