Tag: Medication

Posted on

STAT+: Kyverna Therapeutics plans to submit cell therapy for stiff person syndrome for FDA approval

A one-time, personalized cell therapy from Kyverna Therapeutics improved mobility and reduced disabilities in patients with stiff person syndrome, a rare, neurological autoimmune disorder, according to study results presented Tuesday.

Kyverna intends to submit the treatment to the Food and Drug Administration by the middle of the year. If approved, it would become the first treatment for stiff person syndrome and the first personalized CAR-T therapy for an autoimmune disease of any kind to reach the market. 

Currently, CAR-T treatments are approved only for blood cancers, but using engineered T cells to deplete B cells — essentially performing an immune system reset inside a patient — has pushed a growing number of biotech companies to shift their CAR-T focus to autoimmune diseases. 

Continue to STAT+ to read the full story…

Posted on

STAT+: At AACR, more strong results for Revolution Medicine’s KRAS drug, plus assurance from NCI’s director

You’re reading the web version of STAT’s popup newsletter, AACR in 30 seconds, your guide to what’s happening at the American Association of Cancer Researchers’ annual meeting. Sign up here.

We’re nearing the end of a big AACR. We hope to see everyone at our live event on Tuesday night. Clearly, Revolution Medicines and KRAS have been the big topic of the meeting. Last year, AACR was dominated by big concerns over what cancer research funding would look like in the Trump administration. This year, the new head of the NCI tried to allay researchers’ fears. Read on!

Strong results for Revolution Medicines’ KRAS drug 

Last week, researchers working with the biotechnology firm Revolution Medicines presented stunning news: the experimental drug daraxonrasib more than doubled survival in second-line pancreatic cancer compared to chemo — although that only meant increasing median survival in this terrible disease by six months.

Continue to STAT+ to read the full story…

Posted on

AACR 2026: MRI-ctDNA Combo Informs HPV-Related Throat Cancer Treatment

At the 2026 annual meeting of the American Association for Cancer Research (AACR), researchers from Memorial Sloan Kettering Cancer Center (MSKCC) presented new evidence that a blood-based biomarker, combined with advanced imaging, could enable real-time adjustment of cancer treatment in patients with HPV-related throat cancer. Findings from this clinical study (NCT03323463) highlight a potentially important shift in care, particularly for HPV-associated oropharyngeal cancer, a disease with generally high cure rates but ongoing efforts to reduce treatment-related toxicity. Rather than waiting until therapy is complete, clinicians may be able to tailor treatment intensity based on early indicators of response.

Circulating tumor DNA (ctDNA) has already shown promise for detecting minimal residual disease (MRD), but its role in guiding treatment decisions during therapy remains largely unexplored. To address this gap, a research team led by Bill H. Diplas, MD, PhD, a radiation oncology fellow at MSKCC, investigated whether serial ctDNA measurements, paired with weekly MRI scans, could provide a more precise and dynamic view of tumor response. In collaboration with Labcorp and Biocartis, the MSKCC researchers developed a personalized ctDNA assay that combined two strategies: detection of patient-specific tumor mutations and quantification of DNA from high-risk HPV strains, particularly HPV-16 and HPV-18, using anchored multiplex PCR and high-throughput sequencing.

The study enrolled 158 patients with HPV-associated oropharyngeal cancer who had undergone primary tumor resection followed by risk-adapted chemoradiotherapy guided by hypoxia assessment. MRIs were performed pretreatment and weekly following treatment to determine tumor volume, and blood samples were collected before treatment and weekly during therapy, yielding nearly 1,000 samples from 119 patients (mean 8.2 samples/patient) up to 126 weeks.

At baseline, ctDNA was identified in 93.9% of patients—outperforming either mutation-based (89.4%) or HPV-based (80.3%) methods alone. ctDNA levels also correlated with tumor size and biological features such as cell death and viral load. Notably, ctDNA emerged as a faster and more sensitive indicator of treatment response than imaging. Changes in ctDNA levels appeared earlier and across a broader dynamic range than tumor size reductions observed on MRI. By the second week of therapy, ctDNA measurements could already distinguish patients likely to require more intensive treatment.

The identification of patients with high-risk disease was significantly improved by combining on-treatment ctDNA assessment with imaging in a multimodal model, outperforming any modality alone. These results underscore the complementary nature of molecular signals in blood and structural changes seen on imaging.

Similar multimodal strategies that integrate ctDNA with imaging have been explored in other cancers, including breast and lung, primarily in research settings. Studies suggest that combining these approaches can improve prediction of treatment response and enable earlier detection of resistance. Broader analyses across colorectal, lung, and breast cancers further support the value of integrating molecular and imaging data to refine models of response and survival. However, most of these approaches remain investigational, and the use of ctDNA to guide real-time treatment decisions is only beginning to be tested in prospective trials.

Although further validation is needed, this study establishes a framework for real-time, personalized treatment in oropharyngeal cancer. If translated into clinical practice, such an approach could accelerate the shift toward adaptive therapy—where decisions are guided not only by how tumors appear on imaging, but by how they respond at the molecular level throughout treatment.

The post AACR 2026: MRI-ctDNA Combo Informs HPV-Related Throat Cancer Treatment appeared first on Inside Precision Medicine.

Posted on

This tool could show how consciousness works

How does the physical matter in our brains translate into thoughts, sensations, and emotions? It’s hard to explore that question without neurosurgery. But in a recent paper, MIT philosopher Matthias Michel, Lincoln Lab researcher Daniel Freeman, and colleagues outline a strategy for doing so with an emerging tool called transcranial focused ultrasound.

This noninvasive technology reaches deeper into the brain, with greater resolution, than techniques such as EEG and MRI. It works by sending acoustic waves through the skull to focus on an area of a few millimeters, allowing specific brain structures to be stimulated so the effects can be studied.

The researchers lay out an experimental approach that would use the tool to help test two competing conceptions of consciousness. The “cognitivist” concept holds that brain activity generating conscious experience must involve higher-level processes such as reasoning or self-reflection, likely using the frontal cortex. The “non-­cognitivist” idea is that specific patterns of neural activity—more localized in subcortical structures or at the back of the cortex—give rise to subjective experiences directly.

“This is a tool that’s not just useful for medicine, or even basic science, but could also help address the hard problem of consciousness,” Freeman says. “It can probe where in the brain are the neural circuits that generate a sense of pain, a sense of vision, or even something as complex as human thought.” 

Posted on

GABA-Based Target for Alzheimer’s Therapy Identified

Researchers at the University of Galway have found evidence that targeting inhibitory signaling in the brain may help address cognitive dysfunction in Alzheimer’s disease (AD), a finding that runs counter to current therapeutic approaches that focus on influencing excitatory pathways. The research, published in Neuropharmacology, identifies how modulation of gamma-aminobutyric acid (GABA) signaling can restore disrupted neural balance and improve memory-related function in AD disease models.

“Given the ever-increasing burden of Alzheimer’s disease, the urgent need for the identification of novel targets for the development of disease-modifying therapy is clear,” said senior author Andrea Kwakowsky, PhD, associate professor of pharmacology and lead researcher at the School of Medicine, University of Galway.

Alzheimer’s disease is characterized by progressive cognitive impairment and is associated with hallmark pathological features including β-amyloid (Aβ) plaques and neurofibrillary tangles. In addition to these, disruption of the brain’s excitatory/inhibitory (E/I) balance has gained traction as a central mechanism contributing to memory loss. Today, most approved therapies for AD target excitatory neurotransmitter systems such as cholinergic and glutamatergic pathways, but “the symptomatic relief provided by these therapies is only marginal, and the progression or underlying causes of the disease are not addressed,” the researchers noted.

For their work, the University of Galway team instead focused on the inhibitory side of this balance, specifically the role of gamma-aminobutyric acid (GABA), the brain’s main inhibitory neurotransmitter. GABA regulates neuronal activity and is essential for maintaining stable network function and memory processes. In AD, however, E/I balance becomes dysregulated with increased extracellular GABA—triggered in part by Aβ—leading to overactivation of certain GABA receptors, particularly α5-containing GABA type A receptors (α5-GABA ARs), which are abundant in the hippocampus. The result is a dampening of neuronal signaling and which impairs learning and memory.

“Our research is significant in that it demonstrates that if we block this GABA receptor activity in nerve cells we can reverse Alzheimer-like effects caused by amyloid beta and improve cognitive performance,” Kwakowsky said.

To test whether blocking a5-GGABA A could help restore E/I balance, the team investigated α5IA, an α5-GABA AR-selective inverse agonist. α5IA works by reducing the activity of α5-GABA ARs, which decreases excess tonic inhibition. The data showed that in experimental models of AD, the compound improved long-term potentiation (LTP), a mechanism of synaptic plasticity and memory, reduced abnormal inhibitory conductance, and restored spatial memory performance.

Mechanistically, α5IA appears to act by restoring physiological levels of inhibition in the hippocampus which is critical for memory formation. By reducing excessive tonic inhibition, it rebalances E/I signaling, which allows neuronal circuits to function more effectively. “The data presented here suggest that in both ex vivo and in vivo AD models, α5IA improves cognitive function by restoring CA1 tonic inhibition, thereby re-establishing E/I balance and ameliorating the abnormal hippocampal network activity induced by Aβ1-42,” the researchers wrote.

This new study is the latest to indicate that targeting inhibitory neurotransmission could be an effective treatment approach for AD. Earlier research has shown that α5-GABA AR modulation enhances memory and reduces inhibitory signaling in both animal models and humans. But most of these studies have not directly examined the effect of α5IA in chronic neurodegenerative disease models.

The researchers noted there are some limitations to their work, pointing out that while α5IA improved cognitive outcomes, it did not reverse neuronal loss in vivo, suggesting that its effects may be primarily functional rather than neuroprotective at later stages of AD. Also, variability in drug exposure and timing may influence outcomes. Finally long-term use of α5IA has also been associated with safety concerns at high doses, including renal toxicity, so further research is needed to determine toxicity and dosing regimens and limits.

Nonetheless, the implications of this research indicate there is potential to develop new AD therapies that directly target network dysfunction rather than focusing solely on amyloid accumulation or excitatory signaling. By restoring E/I balance, this approach shows the potential to improve cognitive function even when AD pathology has taken root. The findings could also benefit diagnostic methods, as biomarkers of inhibitory dysfunction or altered GABA signaling could help identify patients who would benefit an approach that rebalances E/I signaling.

The post GABA-Based Target for Alzheimer’s Therapy Identified appeared first on Inside Precision Medicine.

Posted on

Sepsis Fast Diagnostics Could Save Lives and Billions in Costs

Fast diagnostics could improves patient outcomes and save billions in healthcare costs if used systematically to identify sepsis in hospitalized adults at risk due to bloodstream infections, according to an independent report.

The research encompassing all G7 countries revealed multiple benefits from earlier intervention for time-critical infections through the extensive application of fast identification and antimicrobial susceptibility testing (Fast ID/AST).

The Value of Fast Diagnostics in Time-critical Infections report by the independent Office of Health Economics showed how systematic use of fast diagnostics would lead to far fewer sepsis-related deaths and significantly decrease long-term post-sepsis complications, thereby improving patients’ quality of life.

The multi-country economic evaluation, commissioned and funded by bioMérieux, also showed major savings in healthcare costs each year, with the magnitude dependent on country size, incidence, and cost structures.

“While the magnitude varies by country, the direction is consistent: the model demonstrates that early diagnostics reduce the likelihood that high-risk patients progress to sepsis,” said Julien Textoris, PhD, vice president of EMEA medical affairs at bioMérieux.

“Preventing cases of sepsis could therefore reduce the risk of long-term complications after hospital discharge, including recurrent infections, cognitive decline, psychological effects, and organ-specific complications.”

Sepsis is a life-threatening reaction to an infection responsible for 21 million deaths worldwide each year. The initial hours of management are crucial, with targeted antibiotic treatment a key predictor of survival.

However, conventional methods for diagnosis takes at least a couple of days deliver results and nearly one in five bloodstream infection patients receive an inappropriate initial treatment.

In a model-based health economic analysis, Shaheer Hassan and co-workers at the OHE examined what would happen if fast ID/AST were systematically used early in the care pathway before clinical deterioration occurs.

The study encompassed Canada, France, Germany, Italy, Japan, the United Kingdom, and the U.S. and used expert-validated clinical inputs, country-specific cost data, and conservative assumptions.

Results revealed consistent cost savings regardless of the structure or financing of the healthcare system.

More than half of all savings—between 53% and 83%—would occur during the initial hospitalization, when the clinical and economic consequences of deterioration are most evident.

This is because early diagnostic information would prevent the chances of patients progressing into one of the most resource intensive stages of sepsis care.

The savings per patient ranged from €500 in Canada to €3,800 in Japan. This was primarily driven by fewer admissions to the intensive care unit, shorter hospital stays, and educed management of severe complications.

Annual national savings ranged from €26 million in Canada to €2.5 billion in the U.S. and reflected both cost savings in the acute phase and from reduced long-term complications.

“To realize these benefits, hospitals must address structural and workflow barriers so fast results translate into faster therapy, alongside broader system reforms to correct the persistent undervaluation of diagnostics,” the report maintained.

“Overcoming the underutilization and undervaluation of diagnostics will require coordinated system-level action, with the G7 countries included in this analysis well-positioned to lead.”

 

 

 

The post Sepsis Fast Diagnostics Could Save Lives and Billions in Costs appeared first on Inside Precision Medicine.

Posted on

Exposure to negative physical and social factors accelerates brain aging

Nature Medicine, Published online: 21 April 2026; doi:10.1038/s41591-026-04348-z

In a study that included 18,701 people from 34 countries, 73 aggregated physical and social exposomes exhibited nonlinear, synergistic effects that accelerated brain aging. In some cases, these effects were comparable to or stronger than those of mild cognitive impairment and dementia. Environmental inequities might shape brain aging and demand multisectoral and structural policy responses.