Radically different
Have you ever wondered how many drugs are behind the counter at your local pharmacy?
According to Thomas Goetz — a journalist, entrepreneur, and host of the new podcast “Drug Story” — “there are over 3,000 drugs behind a typical pharmacist counter.” And behind each drug is a story.
Dr. Shinya Yamanaka is recognized for the generation of induced pluripotent stem cells (iPSCs) from fibroblasts by a combination of multiple transcription factors, and he won the Nobel Prize in Physiology or Medicine in 2012 jointly with Sir John B. Gurdon for this discovery. Twenty years after the discovery, the Cell Reports Medicine editorial team discusses with Dr. Yamanaka the scientific, technical, and translational milestones that have shaped the field of regenerative medicine. We also discuss the role of iPSCs in disease modeling and drug discovery, the interplay with genome editing, and ongoing issues that still prevent the widespread clinical application of iPSC-derived therapies.
Bellut et al. show that the NLRP3 inflammasome drives hyperacute inflammation and infarct progression during large vessel occlusion in stroke. Pharmacological inhibition reduces infarct growth in mice. NLRP3-positive cells in human pial blood before thrombectomy correlate with clinical outcome and highlight a potential therapeutic target prior to recanalization.
The Swiss drugmaker Roche on Tuesday presented the latest data for its experimental multiple sclerosis drug, setting the stage for the company to seek approval for a medicine that it believes can cut relapse rates and slow the progressive disability the disease causes.
Now the test is whether the drug, called fenebrutinib, can win the regulatory green light.
While three late-stage trials of the drug have shown it to be effective, analysts have homed in on some potentially worrying liver safety signals, an issue that previously prompted the Food and Drug Administration to reject an MS therapy developed by Sanofi. In data released Tuesday, researchers also disclosed that there were two drug-related deaths among patients who took fenebrutinib.
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.
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!
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.
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.
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.”