Day: August 31, 2026

Categories : CancerTags : Leave a comment

Targeted Therapy for Metastatic Pancreatic Cancer Receives Approval in US

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Pancreatic cancer cells. Credit: NIH

The US Food and Drug Administration has approved daraxonrasib, to be sold under the brand name Rasonque, for the treatment of adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy. The approval follows results from an international phase 3 study co-led by UCLA that showed the targeted therapy significantly extended overall survival and reduced the risk of death by 60% compared with standard chemotherapy.

In the study, patients who received daraxonrasib, an oral RAS(ON) multi-selective inhibitor designed to block active RAS signaling, one of the primary drivers of pancreatic cancer, lived a median of 13.2 months compared with 6.7 months for those who received investigator’s choice chemotherapy.

The FDA approval provides a new treatment option for patients with metastatic pancreatic cancer, a disease that remains among the most lethal cancers and has historically had limited effective therapies.

The results were published in the New England Journal of Medicine and presented earlier this year at the annual meeting of the American Society of Clinical Oncology in Chicago.

“This FDA approval represents an important milestone for patients with metastatic pancreatic cancer, who have needed new and more effective treatment options,” said Dr. Zev Wainberg, professor of medicine and investigator at the UCLA Health Jonsson Comprehensive Cancer Center and co-first author of the study. “The results of this trial demonstrate that targeting RAS can meaningfully extend survival and improve disease control, and it is exciting to see these findings translated into an approved treatment for patients.”

More than 90% of tumours are driven by alterations in the RAS signalling pathway, particularly mutations in KRAS, a gene that helps regulate cell growth. When mutated, the gene can lock cells into a constant growth state, fuelling tumour development. Despite decades of research, RAS proteins have proved notoriously difficult to target with drugs.

Unlike earlier targeted therapies that focused on a single mutation subtype, daraxonrasib is part of a new class of therapies designed to inhibit multiple RAS mutations, including G12, G13 and Q61 alterations that dominate pancreatic cancer.

The study involved 500 patients with metastatic pancreatic cancer whose disease had already progressed after one previous treatment from 60 clinical sites across six countries. Participants were randomly assigned to receive either daraxonrasib orally once daily (248 patients) or standard chemotherapy chosen by their doctor (252 patients). About 92% of patients had RAS G12 mutations.

In addition to improved overall survival, patients treated with daraxonrasib experienced significantly longer disease control. Median progression-free survival was 7.2 months compared with 3.6 months for chemotherapy, effectively doubling the time before cancer progression, in the overall study population.

Tumour shrinkage was also more frequent in the daraxonrasib group, with approximately 30% of patients achieving an objective response in the overall study population compared with about 11% in the chemotherapy group. Patients receiving the targeted therapy also experienced slower worsening of pain and better preservation of quality of life over time.

“While most patients had RAS G12 mutations, the benefit appeared generally consistent across different patient groups and mutation types,” said Wainberg, who is also the co-director of the UCLA GI Oncology Program. “These findings support the idea that blocking active RAS signaling will become an important treatment strategy for pancreatic cancer.”

The most common side effects of daraxonrasib include rash, diarrhoea, stomatitis, nausea, fatigue, vomiting, abdominal pain, enema, decreased appetite and haemorrhage. The prescribing information also includes warnings and precautions for dermatologic and soft tissue toxicity, stomatitis and oral disorders, diarrhoea, gastrointestinal perforation, interstitial lung disease or pneumonitis and embryo-foetal toxicity.

Rasonque is manufactured by Revolution Medicines.

Source: UCLA Health

Categories : OphthalmologyTags : Leave a comment

Cortisone Eye Drops can Save the Sight of Extremely Premature Babies

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Photo by Hush Naidoo on Unsplash

Researchers at the University of Gothenburg and Sahlgrenska University Hospital have conducted the first randomised clinical trial in the world to test cortisone eye drops to slow the progression of serious eye disease in extremely premature babies. The results suggest that the intervention can reduce the need for invasive treatment for Retinopathy of prematurity (ROP), a disease that causes blindness in 35 000 children worldwide each year.

ROP occurs when the blood vessels in the retina develop abnormally in very premature babies. In severe cases, laser treatment or injections into the eye are currently required to prevent vision loss and blindness. These treatments are invasive and destructive, and children who require treatment are at the greatest risk of blindness and visual impairment.

The Swedish multicentre study DROPROP involved 100 children born before 30 weeks of pregnancy. The children had a severe but not yet treatment-requiring form of ROP and were randomised to receive either dexamethasone eye drops or placebo.

Large clinical effect

The results showed that 20 percent of the children who received dexamethasone developed treatment-requiring ROP, compared with 38 percent in the placebo group. This corresponds to a 47 percent reduction in relative risk. The difference did not reach statistical significance but showed a large clinical effect.

“The results are very promising because the treatment is simple, inexpensive and non-invasive. In a significant proportion of children, we were able to reduce the need for laser and ocular injections in very fragile premature babies”, says Ann Hellström, professor at the University of Gothenburg and chief physician at Sahlgrenska University Hospital.

The researchers also closely monitored any side effects. No clinically significant difference in complications was seen between the groups. No clear signs of serious hormonal or metabolic side effects were observed.

Long-term follow-ups

The study, funded by the Swedish Research Council, was conducted at six university hospitals and eight county hospitals in Sweden between 2022 and 2025 and is the first double-blind randomized clinical trial of dexamethasone eye drops in ROP.

The researchers are now planning long-term follow-ups of the children to investigate vision development and possible late effects of the treatment.

“There is a great global need for simpler treatments for ROP, especially in parts of the world where access to specialist care is limited. If eye drops can prevent severe ROP, it could save the sight of many children around the world”, says Ann Hellström.

Study: Dexamethasone Eye Drops to Prevent Treatment-Requiring Retinopathy of Prematurity: The DROPROP Trial (JAMA Pediatrics)

Source: University of Gothenburg

Categories : NeurologyTags : Leave a comment

Brain Activity Patterns may “Tag” Experiences for Later Memory Making During Sleep

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Theta oscillations during learning predicted which experiences were remembered after sleep

Photo by Cottonbro on Pexels

The brain may “tag” experiences during learning to better consolidate the memory of them during sleep, according to a study published August 27th in the open access journal PLOS Biology by Dan Denis from the University of York, United Kingdom, and colleagues.

Most people do not remember every experience they’ve ever had. Instead, the brain consolidates some experiences – but not others – into long-term memory during sleep, suggesting that some experiences are “tagged” during learning for later encoding into memory.

To better understand how the brain selected experiences for later memory consolidation during sleep, the authors of this study collected data from 31 participants using electroencephalography (EEG) to measure patterns of brain activity. The participants learned sets of object-word pairs and were tested on them immediately and after a two-hour break. During one visit, participants were allowed to sleep, and in the other, they remained awake. As they learned and rested, the scientists analysed their EEG readings, matching the repetitive oscillations of brain activity during learning to those that were remembered after the participants’ sleep period.

The authors found that word-object pairs associated with theta oscillations between 3-8Hz during learning were more likely to be remembered after a period of sleep, but not after staying awake. The theta activity during learning predicted more coupling between slow neural oscillations during sleep and sleep spindles – bursts of brain activity. The slow oscillations and the sleep spindles were in turn associated with better memory performance after sleep. While the study only tested memory after two hours, and further studies would be needed to determine if memories persisted, the authors suggest that theta oscillations may indicate a mechanism by which the brain identifies experiences to consolidate into memories during sleep. 

The authors add, “How the brain decides what is important to remember and what can be forgotten is still a mystery. These new findings help to answer that question, by uncovering for the first time a signature of neural activity which instructs the brain which experiences to process during sleep and form into long-term memories.

“If we remembered everything that we experienced, our brains would quickly reach system overload. By selectively prioritising important events in our lives, be they emotionally salient or important for the future, we are able to use our past experiences to help guide our interactions in the world.

“Although it is typically adaptive to prioritise certain kinds of information, there are cases where this becomes maladaptive. For example, in depression, individuals tend to over allocate attentional resources to negative information, whilst disregarding or downplaying more positive experiences. Our new findings shed new light on the brain processes that dictate what is ultimately remembered, and may open new avenues for understanding and treating common mental health problems such as depression.”

Provided by PLOS

Categories : Ageing Cardiovascular DiseaseTags : Leave a comment

Statins Cut Heart Attack and Stroke Risk in Older People by 30%

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Photo by Kampus Production

A world-first clinical trial conducted by Monash University researchers has shown that cholesterol-lowering medication can reduce heart attack and stroke in people aged over 70 years.

Statins are commonly prescribed to manage high cholesterol in people up to 75 years, and are known to reduce the risk of heart attack and stroke. But until now it was unclear whether they were effective in older people.

New research from Monash University, published in the New England Journal of Medicine and presented concurrently at the European Society of Cardiology Congress 2026, found the medication decreases the risk of a first major cardiovascular event by 30 per cent for adults aged over 70 living independently.

The Statins in Reducing Events in the Elderly (STAREE) Trial is the first randomised controlled trial of statin therapy to examine whether the drugs could help in older people who have historically been overlooked in clinical trials.

This landmark, ten-year trial analysed the cardiovascular outcomes of almost 10 000 participants while they took either a statin or placebo.

Lead researcher Adjunct Professor Sophia Zoungas, from the Monash School of Public Health and Preventive Medicine, said the findings will change the way clinicians manage cardiovascular risk in older people.

“What we hope to see, now that we have provided such strong evidence, are updated treatment guidelines to help clinicians make use of this new finding,” Professor Zoungas said.

The global prevalence of cardiovascular disease is expected to almost double from 600 million in 2025 to more than one billion affected adults in 2050. An estimated 2 ,000 older Australians suffer from major cardiovascular events, like heart attack and stroke, each year. The risk increases with age, so people aged over 70 are automatically in a high risk category regardless of lifestyle or other factors.

“The risk of heart attack and stroke is a major concern for older people,” Professor Zoungas said. “Knowing there is a safe and effective measure for lowering that risk will be a huge reassurance to older people and their families.

“Anyone who has seen a loved one impacted by cardiovascular disease will know just how important prevention is.”

Co-author Professor Mark Nelson, a prominent general practitioner, Adjunct Professor at the Monash School of Public Health and Preventive Medicine and Research Fellow at Menzies Institute for Medical Research at the University of Tasmania, said this is critical evidence to support GPs in their decision-making around cardiovascular health in older people.

“This was a particularly important trial because it was conducted in the community, with more than 3400 GPs taking part across Australia,” Professor Nelson said. “In light of this new evidence, older patients should have a conversation with their GP about whether statins are an appropriate treatment for their cardiovascular health to help them remain fit and healthy.”

Read the research paper: http://doi.org/10.1056/NEJMoa2607314

Source: Monash University

Categories : Respiratory DiseasesTags : Leave a comment

Doctors Often Disagree on Auscultation Findings in Paediatric Pneumonia

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Photo by cottonbro studio

For generations, a stethoscope has been one of the most essential tools for diagnosing pneumonia. But a new study suggests that what doctors hear through it may depend on who is listening.

In this cohort study, US investigators who are part of the Pediatric Emergency Care Applied Research Network analysed data from children and teens diagnosed as having community-acquired pneumonia (CAP). The findings showed that clinicians often disagreed about their findings on physical exam, including decreased breath sounds, crackles (wet, bubble-likes sounds when air flow is blocked), and rhonchi (abnormal breathing that sounds like snoring or rattling) – symptoms frequently associated with pneumonia. 

Current US guidelines recommend diagnosing CAP based on clinical findings rather than chest X-rays in children who are treated as outpatients. 

Same patient, different findings

For the study, published in JAMA Network Open, researchers analysed data from 252 participants, ages 3 months to 17 years, diagnosed with CAP at one of seven academic paediatric emergency departments across the United States. Two clinicians independently examined each patient within 60 minutes of one another and recorded their findings.

None of the physical exam findings met the researchers’ predetermined threshold for acceptable interrater reliability, a measure of how much different clinicians agree when analysing the same data or event. Wheezing (kappa value [κ], 0.50) and chest retractions (κ, 0.49) were the exam findings clinicians agreed on the most. Even so, agreement was modest.

Agreement was significantly lower for decreased breath sounds and crackles, which both had κ values under 0.25.

The findings raise questions about how heavily clinicians should rely on listening to the lungs when diagnosing pneumonia. 

“This variability is not a trivial concern,” write Susan Lipsett, MD, of Boston Children’s Hospital, and Mark Neuman, MD, MPH, of Harvard Medical School, in an accompanying commentary. When examination findings vary depending on the observer, “their utility as diagnostic anchors is diminished” and their ability to appropriately guide treatment is compromised.

“If physicians cannot consistently agree on the presence of auscultatory findings, treatment decisions may hinge more on examiner interpretation than underlying pathology,” Lipsett and Neuman write. “This variability may contribute to well-documented differences in antibiotic prescribing and chest radiograph use across institutions.”

Why lung sounds can be hard to interpret

Several factors could explain the disagreement. Accurately interpreting lung sounds can depend on background noise, patient cooperation, and clinician experience—factors that “become even more challenging in a busy emergency department,” write Lipsett and Neuman. Children also have more flexible chest walls, which may make it harder to determine exactly where the sounds originate. What’s more, clinicians may also use terms like “crackles” to refer to slightly different things.

The commentators argue that the results strengthen the case for objective risk-assessment tools that combine factors such as fever, oxygen saturation, demographics, and selected clinical findings rather than relying on individual lung sounds. 

“By quantifying risk and reducing dependence on subjective auscultatory interpretation, clinical prediction tools may mitigate interobserver variability and promote more consistent decision-making regarding imaging and antibiotic therapy,

By Laine Bergeson

Source: University of Minnesota