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STAT+: Influential Democratic think tank pushes plan for government to lower health care costs

WASHINGTON — An influential progressive think tank is proposing that Democrats assert more federal government control over hospitals and private insurers to make health care more affordable.

Democrats have set out to gather ideas for overhauling the health care system in preparation for potentially regaining control of Congress and the presidency. Democrats see themselves in a “blue sky moment” following Republicans’ big cuts to health care funding, and they want to give themselves plenty of time to devise a fresh approach so they’re ready to strike if and when they get the chance.

The latest blueprint comes from the Center for American Progress, first reported by The Bulwark.

Continue to STAT+ to read the full story…

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Neuroblastoma Tumor Growth in Mice Suppressed by Blocking Enzyme to Inhibit mTOR Signaling

Neuroblastoma is the most common tumor among children under a year of age, and while in its gentlest form neuroblastoma can regress on its own, it can also take an aggressive form, with high-risk neuroblastoma carrying a five-year survival rate of about 40%.

Researchers at The Hebrew University of Jerusalem have now discovered a mechanistic explanation for how neuroblastoma sustains itself and identified a potential approach to severing that mechanism, by inhibiting nitric oxide (NO) production to suppress mTOR signaling. The collective results from work in human neuroblastoma cells and experiments in a mouse xenograft model showed that inhibiting the enzyme neuronal nitric oxide synthase (nNOS) to inhibit NO production suppressed mTOR signaling and slowed tumor growth.

Professor Haitham Amal, PhD, head of The Laboratory of Neuromics, Cell Signaling, and Translational Medicine, is senior and co-corresponding author of the team’s published paper in Brain Medicine, titled “Targeting nNOS suppresses AKT–TSC–mTOR signaling and inhibits neuroblastoma growth.” In their paper the team concluded “Inhibition of nNOS suppresses mTOR signaling, reduces cellular malignancy, and attenuates tumor growth in vivo, identifying the nNOS-mTOR axis as a promising therapeutic target in neuroblastoma.”

Neuroblastoma accounts for roughly 28% of all cancers diagnosed in infants across Europe and the United States. “Neuroblastoma (NB) refers to a spectrum of neuroblastic tumors that originate from the neural crest cells during fetal development,” the authors wrote. “Neuroblastoma is predominantly a pediatric malignancy, with approximately 97% of cases occurring in children.”

NBs can range from spontaneous regression to maturation to an aggressive, deadly metastatic disease. And as the investigators noted, “Despite major advances in multimodal therapy, high-risk neuroblastoma remains associated with poor prognosis, frequent relapse, and therapy resistance, underscoring the need for a better understanding of the signaling pathways that regulate tumor cell survival, differentiation, and metabolic adaptation.”

Nitric oxide (NO) is an essential regulator of carcinogenesis in various tumors, including NB, the authors pointed out. “Nitric oxide (NO) is a ubiquitous free radical signaling molecule produced in multiple organs and tissues), such as those of the central and peripheral nervous systems.” But at elevated concentrations NO becomes reactive, generating nitrogen species that chemically modify proteins through a process called S-nitrosylation. That modification has been implicated in every stage of cancer progression.

The relationship between nitric oxide and tumors is not simple. Very high concentrations can damage DNA and trigger apoptosis. Lower, sustained levels appear to do the opposite, promoting survival and metastasis. Amal and colleagues had previously demonstrated that nitric oxide drives glioblastoma progression. The question that remained was whether the same enzyme, neuronal nitric oxide synthase, was performing a similar service for neuroblastoma, and if so, through which downstream pathway. The answer turned out to be mTOR.

The team attacked nNOS from two directions. They treated human SH-SY5Y neuroblastoma cells with BA-101, a selective pharmacological inhibitor, at 100 μM for 24 hours. Separately, they silenced the nNOS gene with small interfering RNA. The reasoning was that if a drug and a genetic tool produce the same result, you are looking at biology, not pharmacological noise.

The experiments produced the same result. BA-101 reduced NADPH-diaphorase activity, the standard readout of NOS function, by 35-40%. Genetic silencing cut it by 45-50%. Nitrite levels, a stable proxy for nitric oxide production, fell 65-70% with BA-101 and 55-60% with siRNA. Colony formation, the most direct measure of proliferative capacity, dropped significantly after both BA-101 treatment (p < 0.001) and nNOS silencing (p < 0.01). The cells were losing their ability to multiply.

What followed downstream was systematic. Protein tyrosine nitration, measured by 3-nitrotyrosine immunoreactivity, fell sharply after BA-101 treatment (p < 0.01) and nNOS silencing (p < 0.001). The chemical signature of nitrosative stress was fading.

The results then confirmed that AKT phosphorylation decreased (p < 0.01 with BA-101; p < 0.05 with siRNA), while total AKT remained unchanged. Phosphorylation of mTOR itself declined under both conditions (p < 0.01 each). The downstream mTORC1 substrate ribosomal protein S6 followed (p < 0.05 with BA-101; p < 0.01 with siRNA).

And here, the most telling detail, that TSC2, a master negative regulator of mTOR signaling, rose significantly under both treatments (p < 0.05). Removing the nitric oxide signal had allowed the cell’s own braking system to re-engage. In summary, the authors noted, “Pharmacological inhibition of nNOS with BA-101 (100 μM, 24 h) or genetic silencing of nNOS with siRNA caused upregulation of the key negative regulator TSC2 and decreased phosphorylation of AKT, mTOR, and RPS6, indicating suppression of mTOR pathway activity.”

Synaptophysin, a neuroendocrine tumor marker used to gauge the malignant identity of neuroblastoma cells, decreased significantly with BA-101 (p < 0.01) and nNOS knockdown (p < 0.05). The tumor cells were not merely growing more slowly. They were becoming, at a molecular level, less recognizably cancerous. In summary, the investigators noted, “Our results show that inhibition of NO production in the human NB cell line (SH-SY5Y cells), either by pharmacological intervention using the selective nNOS inhibitor BA-101 (41) or by genetic ablation using the specific siRNA, successfully suppressed NB malignancy.”

Schematic model illustrating the NO-mTOR signaling axis in neuroblastoma. Under basal/pathological conditions (left panel), and nNOS inhibition (right panel). [Haitham Amal]
Schematic model illustrating the NO-mTOR signaling axis in neuroblastoma. Under basal/pathological conditions (left panel), and nNOS inhibition (right panel). [Haitham Amal]

But if blocking nitric oxide suppresses mTOR signaling, then flooding the cell with nitric oxide should amplify it. The researchers tested this by exposing SH-SY5Y cells to SNAP, a nitric oxide donor, at 200 μM for 24 hours. This converse experiment produced the converse result. 3-nitrotyrosine rose (p < 0.05), and TSC2 fell (p < 0.01). Phosphorylation of AKT, mTOR, and RPS6 all increased (p < 0.05 for each).

The team then tested their findings in a xenograft mouse model of neuroblastoma, treated with BA-101. “Importantly, to extend these findings to an in vivo context, we further assessed the impact of pharmacological nNOS inhibition on tumor growth in a xenograft NB model,” they stated. The investigators found that while tumors in control animals grew to approximately 1.5 cm in their largest dimension, the treated tumors did not. Final tumor volume and weight were dramatically reduced in the BA-101 group. ‘Quantitative analysis revealed a dramatic decrease in the final tumor volume and weight in the BA-101-treated group (p < 0.001) compared with controls,” they noted.

Body weight did not differ significantly between groups, suggesting that the compound was tolerated without gross systemic toxicity. In summary, the authors wrote, “Our finding demonstrate that the pro-tumorigenic effects of nNOS in SH-SY5Y involve activation of themTOR signaling pathway.” Importantly, both genetic inhibition of nNOS using siRNA and pharmacological inhibition with BA-101 effectively suppressed mTOR pathway activation and reduced malignant properties of NB cells, highlighting the therapeutic relevance of targeting nNOS signaling.  “These findings indicate that pharmacological inhibition of nNOS effectively suppresses xenograft tumor progression, highlighting the critical role of nNOS-derived NO in promoting neuroblastoma growth in vivo.”

“The magnitude of the in vivo suppression caught our attention,” said Amal, the study’s corresponding author, who holds appointments at the Institute for Drug Research, School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, and the Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center at Boston Children’s Hospital, Harvard Medical School. “We had demonstrated the role of nitric oxide in glioblastoma previously, but the consistency of the neuroblastoma results across every assay, from protein phosphorylation to colony formation to xenograft growth, points to nNOS as something more than a contributor. It appears to be a central driver of the signaling that sustains this tumor.”

Added first author Shashank Kumar Ojha, PhD, first author of the study and a researcher at the Institute for Drug Research, The Hebrew University of Jerusalem, added, “What convinced me was the concordance between the pharmacological and genetic approaches. When BA-101 and siRNA independently produce the same pattern of effects across NADPH-diaphorase activity, nitrosative stress markers, mTOR pathway phosphorylation, and clonogenic growth, you can be confident the biology is real. That reproducibility is what gives you a therapeutic hypothesis worth testing further.”

The authors acknowledged limitations to their study. The in vitro work relied on a single cell line, SH-SY5Y, which cannot capture the full genetic heterogeneity of neuroblastoma or the complexity of the tumor microenvironment. The chemical identity of BA-101 is currently undisclosed pending patent issuance, which means independent replication by other laboratories must wait. Whether nitrosative stress directly underlies its functional impairment, or whether an intermediary mechanism is involved, remains an open question that the authors explicitly flag for future investigation. “Future studies using patient-derived cells, organoids, or genetically engineered mouse models will be important to further validate and extend these observations,” they stated. Nevertheless, the authors suggest, the limitations do not diminish the central discovery of a druggable nNOS–mTOR axis.

mTOR inhibitors such as rapalogs and catalytic mTOR inhibitors have shown limited efficacy as monotherapies in neuroblastoma, undermined by feedback activation and resistance mechanisms. The present study suggests the potential for a different attack strategy. Rather than targeting mTOR at the lock, intervene upstream at the hand that turns the key. By reducing nitric oxide-dependent mTOR activation, nNOS inhibition may sidestep the compensatory pathways that have frustrated direct mTOR blockade. “Collectively, these results identify the nNOS-mTOR axis as a key driver of neuroblastoma progression and suggest that nNOS inhibition represents a promising strategy for NB treatment,” they concluded.

The post Neuroblastoma Tumor Growth in Mice Suppressed by Blocking Enzyme to Inhibit mTOR Signaling appeared first on GEN – Genetic Engineering and Biotechnology News.

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Smart MRI Agents Combine Cancer Imaging and Therapy in Single Platform

Researchers at NYU Abu Dhabi have developed manganese-based molecules that combine cancer detection and treatment within a single system, allowing for simultaneous imaging and therapy using magnetic resonance imaging (MRI). The research, published in the Journal of the American Chemical Society, details the development of metal–organic structures that remain stable in healthy tissue but become active within the tumor microenvironment, where they both enhance MRI contrast and induce cancer cell death.

“Our goal was to create materials that allow doctors to see cancer clearly and treat it at the same time,” said lead author Farah Benyettou, PhD, a research scientist at NYU Abu Dhabi. “The ability to image and target brain tumors with high precision is particularly exciting.”

The molecules the researchers developed are composed of manganese ions coordinated with organic frameworks arranged into interlocked topologies. Unlike conventional drugs, which are small and relatively simple, these molecules have interlocked structures that resemble knots and rings. This design allows them to behave differently inside the body, improving both imaging and therapeutic performance.

“Manganese (Mn)-based metal–organic architectures offer a unique avenue for integrating magnetic resonance imaging (MRI) and cancer therapy within a single molecular platform,” the researchers wrote. The geometrical complexity and electropositive, pH-labile coordination framework allow the molecules to remain intact in normal tissue but disassemble when exposed to the acidic tumor microenvironment.

This pH-responsive behavior is the key to their dual function. In healthy tissue, the molecules maintain stability and limit off-target effects. Once inside tumors, where acidity is elevated, they release Mn2+ ions. These ions enhance T1-weighted MRI signals, making tumors more visible, while also triggering biological pathways that lead to cancer cell death. The researchers wrote that this process culminates in “lysosomal acidification, pH-triggered disassembly, Mn2+ release, ROS accumulation, and caspase-dependent apoptosis,” marrying their imaging capability directly to therapeutic action.

The novel molecule design builds on prior prior research of manganese-based imaging agents and topological chemistry. The researchers noted that conventional gadolinium-based contrast agents have safety limitations, including toxicity and accumulation in tissues, while earlier manganese agents lacked stability and tumor targeting. “These drawbacks underscore the need for next-generation Mn platforms with enhanced stability and tumor specificity,” the researchers wrote. In previous studies, the NYU Abu Dhabi researchers had demonstrated that metal-templated trefoil knots could induce apoptosis in drug-resistant cancer cells, a finding that spurred their efforts to integrate therapeutic activity with an imaging agent.

To evaluate the new molecules, the team conducted both in vitro and in vivo experiments, focused on glioblastoma. In cell studies, Mn-TK and Mn-BR showed selective toxicity toward cancer cells while sparing normal cells. In animal models, the molecules accumulated in tumors, produced strong MRI contrast, and inhibited tumor growth.

An important finding of the study was data that showed both Mn-TK and Mn-BR were able to cross the blood–brain barrier and accumulate in glioblastoma tumors. This has traditionally been a major limitation of MRI contrast agents, which often fail to image tumors in the brain.

The implications for clinical care include the potential to replace separate diagnostic and therapeutic steps with a single intervention. By combining imaging and treatment, the molecules could provide earlier detection, more accurate tumor delineation, and targeted therapy with reduced side effects. The manganese-based design may also offer a safer alternative to gadolinium, which could produce long-term retention and toxicity.

“This work introduces a generalizable strategy for designing manganese-based theranostic agents by integrating topological coordination chemistry with tunable lipophilicity and electrostatics,” the researchers noted, adding that this method could be used to develop additional agents tailored to different cancers or imaging needs.

Next steps for the team include further evaluation of safety, optimization of molecular design, and studies to support clinical translation. The researchers identify Mn-TK and Mn-BR as leading candidates due to their combination of tumor targeting, imaging performance, and therapeutic activity. Continued work will likely focus on refining these properties and assessing their performance in additional disease models.

The post Smart MRI Agents Combine Cancer Imaging and Therapy in Single Platform appeared first on Inside Precision Medicine.

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23andMe Reports Genetic Predictors of Response to GLP-1 Drugs for Obesity

On any given morning, skyrocketing numbers of people reach for a small injection pen (and soon a pill) that, just a few years ago, was barely available outside of diabetes clinics. Drugs like semaglutide and tirzepatide have become cultural phenomena, reshaping not only medicine but also public discourse and the advertising industry around weight, metabolism, and obesity. Today, it is impossible to open a magazine, turn on the TV or radio, or walk down the grocery aisle without encountering some form of advertisement for these GLP-1 receptor agonists (GLP-1RAs). 

Almost any individual in the United States can obtain a subscription to a GLP-1RA without having to visit a doctor’s office. Just visit Hims/Hers, Ro, or Noom and answer a few questions about weight, height, goals, and concerns to get a prescription. (One such site claims it is taking weight and height data and “combining with clinical data,” whatever that means, before presenting a plan and steps for ordering a prescription.) 

But there are some major problems, one being that these drugs don’t work uniformly. Some patients respond to GLP-1RAs almost immediately, reporting diminished cravings within days. Others see little change. Side effects, too, can vary dramatically, from mild discomfort to debilitating nausea and vomiting. The spread of outcomes is wide and not fully understood. 

Before blindly beginning to take a drug that, on the one hand, has seemingly miraculous effects and, on the other hand, might cause serious side effects like pancreatitis, gallbladder disease, and kidney failure, wouldn’t prescribers and prescription seekers want to know this information? 

A study published in Nature by the 23andMe Research Institute—the new nonprofit entity founded by the company’s co-founder, Anne Wojcicki, for $305 million to replace the bankrupt biotechnology company—suggests the answer may be found, at least in part, in something far more fundamental than diet or willpower: our genes. 

In speaking with Inside Precision Medicine for the first time since the company filed for bankruptcy and was resold to the nonprofit public benefit corporation, Adam Auton, PhD, vice president of Human Genetics at the 23andMe Research Institute, said, “The ‘GLP-1s’ have completely transformed weight loss management. A huge fraction of the population is benefiting. It’s a very natural question: Are people’s experiences on GLP-1s modulated by genetics?”  

The short answer is, yes. Auton and 23andMe Research Institute scientists have provided genetic evidence that variation in drug target genes contributes to variability in response among individuals, laying the groundwork for consumer-based precision medicine approaches to obesity treatment and beyond. 

Crowd-sourcing GLP-1 genetics 

To better understand why responses to GLP-1 receptor agonists vary so widely, the 23andMe Research Institute team leveraged its uniquely large and engaged research cohort. Over the past decade, the company has assembled genetic data from more than 15 million participants who consented to research, enabling analyses that would be difficult in traditional clinical trials. Immediately following the company’s filing for bankruptcy in March 2025, 23andMe reported that over 1.9 million users requested for their data to be deleted. Auton told Inside Precision Medicine that the current number of consented customers is around 11 million. 

Building on this resource, Auton and colleagues launched a targeted survey asking participants detailed questions about their GLP-1 drug use, including medication type, duration, dosage, weight loss, and side effects. More than 27,885 customers responded, providing a rich, real-world dataset. “That’s the power of having a large, engaged cohort,” said Auton. “You can ask a question and very rapidly get meaningful data back.”

Using these data, Auton and colleagues conducted a genome-wide association study (GWAS), scanning millions of genetic variants to identify those associated with treatment outcomes. “You’re starting with the entire genome,” Auton explained. “You’re testing every variant for correlation with the trait of interest. And when you see a signal, it tends to be overwhelming.”  

The team focused on two primary traits: weight loss and the presence of side effects. The strongest association emerged in GLP1R, the gene encoding the GLP-1 receptor—the direct target of these drugs. A missense variant, rs10305420, was linked to significantly greater weight loss, with each copy associated with an additional 0.76 kilograms lost.  

“It made very clear biological sense,” Auton said. “This is the receptor that the drug is acting on.” The missense variant may affect how much receptor is expressed on the cell surface, meaning individuals with more receptors could experience a stronger response to the same dose. 

A second key finding involved a substitution in GIPR (rs1800437; p.Glu354Gln), which encodes the receptor for glucose-dependent insulinotropic polypeptide and is targeted by dual agonists such as tirzepatide. Unlike the GLP1R result, this association was not related to weight loss but to drug tolerability. Carriers of the variant were more likely to report nausea and vomiting—but only when taking medications that act on the GIP receptor. No such effect was observed among users of semaglutide, which does not target GIPR.  

“It was very, very clean,” Auton said. “We saw this effect specifically in people taking the medications that actually target that receptor.”  

Together, these findings underscore a central principle of pharmacogenetics: genetic variation can shape not only whether a drug works, but also how it is experienced, often in highly drug-specific ways. 

Who is represented 

One of the study’s more unconventional aspects is its reliance on self-reported data, a method sometimes viewed with skepticism in clinical research given the limits of memory and potential inaccuracies in reporting weight loss or medication use. Anticipating this concern, scientists at the 23andMe Research Institute validated their findings using a subset of participants who also shared electronic health records (EHRs), enabling direct comparison between self-reported and clinically recorded data.

The results were reassuring: survey-reported weight loss closely tracked with medical records, and medication histories aligned well across both sources. Although participants tended to slightly overestimate weight loss, they also reported longer treatment durations, effects that largely offset each other. Importantly, the genetic associations remained robust under independent scrutiny, with replication in the All of Us Research Program, a large, federally funded dataset based on clinical records rather than self-report. 

While weight loss is the headline feature of GLP-1RAs, side effects often determine whether patients persist with treatment. Nausea, vomiting, and gastrointestinal discomfort are among the most common reasons for discontinuation, yet they are frequently underreported in traditional clinical datasets. EHRs may document when a medication is stopped but rarely capture why. Self-reported data addresses this gap by directly capturing patient experience. 

“We were able to ask people directly about their experiences,” Auton said. “That’s something that’s often missing from clinical datasets.” By linking these experiences to genetic variation, the study enables a more refined understanding of drug tolerability, moving beyond population averages to individualized risk profiles. 

As with many large-scale genetic studies, statistical power was greatest among individuals of European ancestry, reflecting broader imbalances in genomic datasets. However, the key findings were consistent across multiple ancestral groups, supporting their generalizability.

“We’re not seeing fundamentally different genetic effects across populations,” Auton said. Still, increasing diversity in genetic research remains essential to ensure equitable advances in precision medicine. As digital tools continue to integrate genetic, clinical, and self-reported data, this participant-driven model may play an increasingly central role in biomedical discovery. 

Putting pharmacogenomics in patients’ hands 

Identifying genetic variants is only the first step, of course. The larger goal is to translate those discoveries into tools that can guide real-world decisions. To that end, the 23andMe Research Institute scientists developed predictive models that combine genetic information with clinical factors to estimate treatment outcomes. 

The vision is straightforward: before starting a GLP-1 drug, a patient could receive a personalized profile indicating likely weight loss and risk of side effects. “People are making decisions about whether these medications are right for them,” Auton said. “Can we give them information to help with that decision?” 

Such tools could have immediate clinical applications. A patient with a high predicted risk of nausea, for example, might start at a lower dose or follow a slower titration schedule. Another with a favorable genetic profile might be reassured about expected benefits. 

For now, these findings are unlikely to immediately change prescribing practices, as clinical guidelines will require further validation through prospective studies. However, the trajectory is clear. In the near future, patients considering GLP-1 therapies may undergo genetic testing as part of routine care, with treatment decisions—such as drug choice, dosing, and expectations—guided in part by their DNA. For a class of drugs already transforming millions of lives, this approach could further enhance both efficacy and tolerability, underscoring that responses to GLP-1 therapies are shaped not only by pharmacology but also by the subtle variations of the human genome. 

The broader significance of the study lies in its contribution to precision medicine: the idea that treatments should be tailored to individual biology rather than applied uniformly. In fields like oncology, this approach is already standard. But precision obesity treatment is in far earlier stages.  

Auton is quick to re-emphasize that genetics is only one piece of the puzzle. Lifestyle, environment, treatment adherence, and underlying health conditions all shape outcomes. Still, even a partial predictive signal could be transformative in a field where trial-and-error prescribing is common. 

As researchers continue to study GLP-1RAs, their potential appears to extend far beyond weight and blood sugar. Early evidence suggests benefits in cardiovascular health, inflammation, and even neurological conditions. Some studies are exploring their role in addiction and compulsive behaviors. “There’s an increasing literature that they’re beneficial in multiple areas,” Auton said. 

This expanding scope makes understanding variability even more important. If GLP-1 drugs are to be used to treat a wide range of conditions, predicting who will benefit and who may be at risk becomes one of the most important, if not the most important, challenges.

What about sequencing? 

Throughout our conversation, there was at least one elephant in the room. One is that this is not the first study to identify genetic variants influencing responses to GLP-1 drugs, as prior research has also implicated rs10305420. Slovenian researchers showed that genetic variability in GLP1R is associated with inter-individual differences in the weight-lowering-lowering potential of GLP-1 drugs in obese women with polycystic ovary syndrome (PCOS) in 2015, at a time when the main GLP-1 drug was liraglutide, which required daily injection.

More provocative is that the directionality of the variants’ effect reported in the Nature paper is the opposite of these previous studies. Auton’s team writes that such discrepancies may stem from differences in disease context, smaller sample sizes, limited statistical power, and variations in drug type, cohorts, and analytical methods.

Additionally, the GIPR variant rs1800437 (p.Glu354Gln) is already a known partial loss-of-function mutation, previously identified in a study of Chinese type 2 diabetes patients in 2019. 

Perhaps the more significant issue is the question of sequencing. It’s not a space that 23andMe has completely avoided, as their premier consumer kit employs exome sequencing. But the cost of whole genome sequencing (WGS) direct-to-consumer products is now often priced lower than 23andMe’s premier kit, which goes for $499. 

When asked about employing WGS, Auton revealed little of the calculus behind why 23andMe hasn’t added WGS to its arsenal of tools for interrogating genomes. “We’re very excited about that space,” Auton said. “Our focus has always been on what we can do in a direct consumer framework. There’s always been a price question there for WGS. It’s great. But when it was $1,000, it wasn’t obvious that that was going to be a compelling consumer offering. The pricing has reached its current level. It’s an area we’re very excited about and we’ll continue to look at.”

With studies like this, 23andMe 2.0 is making a case, perhaps its strongest yet, that its true value lies in something far more consequential: the ability to predict how individuals will respond to medicine before they ever take it. If that vision holds, the implications extend well beyond GLP-1 drugs. It suggests a future where prescribing a medication without first consulting a patient’s genetic profile feels incomplete, even irresponsible. 

The post 23andMe Reports Genetic Predictors of Response to GLP-1 Drugs for Obesity appeared first on Inside Precision Medicine.

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STAT+: Steve Ubl to step down as CEO of PhRMA

WASHINGTON — Steve Ubl is stepping down as CEO of the Pharmaceutical Research and Manufacturers of America after more than a decade leading the brand drug industry’s main trade group.

Ubl plans to depart by the end of the year and will remain in his position until a new leader is found, according to a PhRMA statement.

Ubl led the organization during tumultuous times that included the Covid-19 pandemic and aggressive political attacks on drug prices. Democrats passed a law directing Medicare to negotiate drug prices, and the Trump administration struck voluntary deals with individual drugmakers aimed at lowering U.S. prices to levels in other high-income countries. 

Continue to STAT+ to read the full story…

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Base Editing Shows Early Promise for Treating Beta Thalassemia

The Chinese biotech CorrectSequence Therapeutics, also known as Correctseq, reports good results from a Phase I study of its technology involving editing a person’s hematopoietic stem cells to treat beta thalassemia.

The trial, published in Nature, included five patients with transfusion dependent beta thalassemia who were able to stop red blood cell transfusions, the standard treatment for the condition, after receiving the base-edited treatment CS-101. The participants continued to have good levels of hemoglobin with no serious side effects during follow-up.

Beta thalassemia is a rare inherited condition affecting around one in 100,000 people in the U.S. Mutations in the beta‑globin gene HBB reduce or stop production of the beta chains of hemoglobin, leading to chronic anemia that varies in its severity.

There are already several therapies on the market for beta thalassemia. The most common treatment is still regular blood transfusions to treat the anemia, but recently the genetic therapies Zynteglo, a lentiviral gene therapy developed by Bluebird Bio, and Casgevy, a CRISPR edited therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics were approved by the FDA.

Casgevy works by boosting fetal hemoglobin levels to treat the anemia seen in thalassemia patients. It uses CRISPR–Cas9 to cut both strands of DNA at the BCL11A enhancer site, which relies on error‑prone repair and can theoretically generate insertions, deletions, and larger rearrangements.

Correctseq is also aiming to raise fetal hemoglobin levels with CS-101, targeting the same site, but is only changing individual bases without making a full cut, which should reduce risks linked to double‑strand breaks, such as large deletions or chromosomal translocations.

In this study, CS-101 was given to five patients with beta thalassemia, previously treated with blood transfusions. The process involves extracting their stem cells, reactivating fetal hemoglobin production using base editing, giving the patients chemotherapy to clear existing stem cells and make way for the newly edited population, and finally injecting the patients with the edited stem cells.

All five patients were able to stop red blood cell transfusions and had maintained good levels of hemoglobin at three months. These levels stayed at a similar level through a median follow up period of 23 months. No deaths or reported cancers due to the chemotherapy treatment were observed and the safety profile so far is acceptable.

Although these results are promising, this trial is just a small initial study and further work is needed to confirm safety and efficacy of CS-101.“The planned Phase II/III trial will be crucial for evaluating a larger and more genetically diverse patient population across multiple centers,” write the authors.

“Extended follow-up will be required to enable comprehensive analyses of chimerism and clonality, which will facilitate more definitive assessment of long-term safety, engraftment dynamics and clinical benefit.”

One Correctseq’s main competitors is U.S.-based Beam Therapeutics, which is developing a similar base edited treatment. Beam is behind Correctseq in developing its edited therapy for beta thalassemia, but ahead with its therapy for sickle cell disease, something Correctseq are also targeting using a similar pathway.

The Chinese biotech industry is currently on an upward trajectory. Correctseq is one of many Chinese biotech companies currently working to produce competitors for gene therapies like Casgevy and Zynteglo at a more affordable price than those seen in the U.S.

The post Base Editing Shows Early Promise for Treating Beta Thalassemia appeared first on Inside Precision Medicine.

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The Download: water threats in Iran and AI’s impact on what entrepreneurs make

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

Desalination plants in the Middle East are increasingly vulnerable 

As the conflict in Iran has escalated, a crucial resource is under fire: the desalinization technology that supplies water in the region. 
 
President Donald Trump has threatened to destroy “possibly all desalinization plants” in Iran if the Strait of Hormuz is not reopened. The impact on farming, industry, and—crucially—drinking in the Middle East could be severe. Find out why. 

—Casey Crownhart 

This story is part of MIT Technology Review Explains, our series untangling the complex, messy world of technology to help you understand what’s coming next. You can read more from the series here. 

AI is changing how small online sellers decide what to make 

For small entrepreneurs, deciding what to sell and where to make it has traditionally been a slow, labor-intensive process. Now that work is increasingly being done by AI.   

Tools like Alibaba’s Accio compress weeks of product research and supplier hunting into a single chat. Business owners and e-commerce experts say they’re making sourcing more accessible—and slashing the time from product idea to launch.  

Read the full story on how AI is leveling the path to global manufacturing. 

—Caiwei Chen 

The gig workers who are training humanoid robots at home 

When Zeus, a medical student in Nigeria, returns to his apartment from a long day at the hospital, he straps his iPhone to his forehead and records himself doing chores.  
 
Zeus is a data recorder for Micro1, which sells the data he collects to robotics firms. As these companies race to build humanoids, videos from workers like Zeus have become the hottest new way to train them.   
 
Micro1 has hired thousands of them in more than 50 countries, including India, Nigeria, and Argentina. The jobs pay well locally, but raise thorny questions around privacy and informed consent. The work can be challenging—and weird. Read the full story.  

—Michelle Kim 

This is our latest story to be turned into an MIT Technology Review Narrated podcast, which we’re publishing each week on Spotify and Apple Podcasts. Just navigate to MIT Technology Review Narrated on either platform, and follow us to get all our new content as it’s released. 

The must-reads 

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology. 

1 Anthropic’s new model found security problems in every OS and browser 
Claude Mythos has been heralded as a cybersecurity “reckoning.” (The Verge)  
+ Anthrophic is limiting the rollout over hacking fears. (CNBC) 
+ It’s also launching a project that lets Mythos flag vulnerabilities. (Gizmodo) 
+ Apple, Google, and Microsoft have joined the initiative. (ZDNET) 

2 Iranian hackers are targeting American critical infrastructure 
Their focus is on energy and water infrastructure. (Wired) 
+ They’re targeting industrial control devices. (TechCrunch)  

3 Google’s AI Overviews deliver millions of incorrect answers per hour 
Despite a 90% accuracy rate. (NYT $) 
+ AI means the end of internet search as we’ve known it. (MIT Technology Review) 

4 Elon Musk is trying to oust OpenAI CEO Sam Altman in a lawsuit 
As remedies for Altman allegedly defrauding him. (CNBC) 
+ Musk wants any damages given to OpenAI’s nonprofit arm. (WSJ $) 

5 ICE has admitted it’s using powerful spyware 
The tools that can intercept encrypted messages. (NPR) 
+ Immigration agencies are also weaponizing AI videos. (MIT Technology Review) 

6 Greece has joined the countries banning kids from social media 
Under-15s will be blocked from 2027. (Reuters) 
+ Australia introduced the world’s first social media ban for children. (Guardian) 
+ Indonesia recently rolled out the first one in Southeast Asia. (DW)  
+ Experts say they’re a lazy fix. (CNBC) 

7 Intel will help Elon Musk build his Terafab in Texas 
They aim to manufacture chips for AI projects. (Engadget) 
+ Musk says it will be the largest-ever semiconductor factory. (Engadget) 
+ Future AI chips could be built on glass. (MIT Technology Review)  

8 TikTok is building a second billion-euro data center in Finland 
It’s moving data storage for European users. (Reuters) 
+ Finland has become a magnet for data centers. (Bloomberg $) 
+ But nobody wants one in their backyard. (MIT Technology Review) 

9 Plans for Canada’s first “virtual gated community” have sparked a row 
The AI-powered surveillance system has divided neighbors. (Guardian) 
+ Is the Pentagon allowed to surveil Americans with AI? (MIT Technology Review) 

10 The high-tech engineering of the “space toilet” has been revealed 
Artemis II is the first mission to carry one around the world. (Vox) 

Quote of the day 

“This case has always been about Elon generating more power and more money for what he wants. His lawsuit remains nothing more than a harassment campaign that’s driven by ego, jealousy and a desire to slow down a competitor.” 

—OpenAI criticizes Musk’s legal action in an X post. 

One More Thing 

USWDS

Inside the US government’s brilliantly boring websites 

You may not notice it, but your experience on every US government website is carefully crafted. 

Each site aligns an official web design and a custom typeface. They aim to make government websites not only good-looking but accessible and functional for all. 

MIT Technology Review dug into the system’s history and features. Find out what we discovered. 

—Jon Keegan 

We can still have nice things 

A place for comfort, fun and distraction to brighten up your day. (Got any ideas? Drop me a line.) 

+ Rejoice in the splendor of the “Earthset” image captured by Artemis II. 
+ Meet the fearless cat chasing off bears. 
+ This document vividly explains what makes the octopus so unique. 
+ Revealed: the rhythmic secret that makes emo music so angsty. 

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Opinion: What American hospitals can learn from India about waste

The first time we stepped into an operating theater in the United States as medical students, we were shocked by the sheer amount of waste produced from just one surgery. In fact, health care is responsible for nearly 10% of total carbon emissions in the United States, contributing approximately 5 million tons annually. Thirty percent of that waste comes from operating rooms, much of it due to disposable gowns, drapes, instruments, and plastic packaging.

As we continued with our medical training, we grew used to the idea that high-quality care inevitably produces high waste. But reading several published articles on the resource efficiency of hospitals in India forced us to question that assumption. How were they providing thousands of surgeries a day with a fraction of the waste — and no compromise in safety?

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Posted on

Judge refuses to block sending abortion pill by mail for now, but says FDA must finish review

A federal judge Tuesday refused to block filling prescriptions for the abortion pill mifepristone by mail across the U.S. — at least for now — in a setback to Louisiana’s effort to stifle groups that send it into states where abortion is banned.

U.S. District Judge David Joseph, who sits in Lafayette, Louisiana, ruled against Louisiana Attorney General Liz Murrill, who asked that U.S. Food and Drug Administration rules that allow mifepristone to be dispensed through the mail be paused while a challenge to those 2023 regulations moves through the courts.

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