Category: 8. Health

A new vaccine aimed at a common cancer gene mutation could help stop aggressive pancreatic cancers from coming back, a small clinical trial suggests.

Pancreatic cancer is one of the most lethal cancers, with a five-year survival rate of about 13%, according to the American Cancer Society.

Further, up to 80% of cases return after treatment, the National Institutes of Health says.

“If you were to ask me what disease most needs something to prevent recurrences, I’d say this one,” Dr. Zev Wainberg, a leader of the trial, told NBC News. He’s co-director of the University of California, Los Angeles gastrointestinal oncology program.

The experimental vaccine targets KRAS gene mutations, which are found in about 25% of all cancers, the University of Texas MD Anderson Cancer Center says. This includes up to 90% of pancreatic cancers and roughly 40% of colon cancers.

While these mutations have long been considered impossible to treat with drugs, researchers are finding new ways to target them.

The vaccine, called ELI-002 2P, uses small chains of amino acids called peptides to train the immune system to spot and destroy cells with KRAS mutations.

Unlike many cancer vaccines that are custom-made for each patient, this one is designed to be off the shelf, meaning it doesn’t require the tumor to be sequenced before it’s used, NBC News reported.

The Phase 1 study — reported Tuesday in Nature Medicine — included 20 people with pancreatic cancer and five with colon cancer. All had KRAS mutations and had already undergone surgery and chemotherapy.

Blood tests after surgery showed microscopic evidence of residual disease – cancer cells too small to see on scans. These leftover cells can cause the cancer to spread and return.

Post-surgery, participants received up to six priming doses of the vaccine, with 13 also getting booster shots. In all, the process took six months.

Here’s what the results showed:

85% (21 of 25 participants) had an immune response to the KRAS mutations.

About two-thirds of those had a strong enough response to help clear lingering cancer cells.

Nearly 70% developed immunity to other tumor targets not included in the vaccine.

A few “super-responders” had exceptionally strong immune reactions and the best outcomes.

In the pancreatic cancer group, patients survived for an average of 29 months, staying recurrence-free for more than 15 months after vaccination.

“That far exceeds the rates with resectable [surgically removable] cancers,” Wainberg said.

Cancer vaccines have been difficult to create because cancer cells share many proteins with healthy cells, making safe targets hard to find. Advances in mRNA technology and faster gene sequencing are now making more effective cancer vaccines possible.

The peptides in this vaccine also have a unique “tail” that helps them stay in lymph nodes, where immune cells are activated — a feature past peptide vaccines didn’t have, said Stephanie Dougan, an associate professor at Dana-Farber Cancer Institute in Boston, who was not involved in the study.

More research is needed to confirm the findings, and a Phase 2 trial is now underway to compare the vaccine with standard care.

“The fact that the long-term survival really correlated with T-cell response suggests that the vaccine caused this,” Dougan said, referring to the specific immune cells activated by the vaccine. “The idea that you can target KRAS is really exciting.”

More information

The Mayo Clinic has more on pancreatic cancer.

Copyright © 2025 HealthDay. All rights reserved.

As autism diagnoses continue to grow and remain a topic of nationwide debate, new research reveals that autistic individuals are facing mental health challenges at a major turning point in their lives – when they go to college.

According to a new study led by researchers at Binghamton University, State University of New York, autistic college students face dramatically higher rates of anxiety and depression compared to their non-autistic peers. 

Psychologists at Binghamton University examined data from the National Survey of Student Engagement (NSSE), which included 342 universities and 149,783 undergraduate student respondents. Of the questions posed, students can report being autistic and also whether or not they have a diagnosis of anxiety or depression. The researchers analyzed the data to determine the rate of anxiety and depression for those who also reported being autistic. 

What we found is really staggering – autistic individuals endorse much higher rates of anxiety and depression compared to their non-autistic peers.”

Diego Aragon-Guevara, lead author on the paper and PhD student in psychology at Binghamton

Aragon-Guevara, whose main research focus is improving the quality of life for autistic adults, said that autistic college students are an underrepresented, under-researched population.

“We wanted to sort of fill that gap in the research and find out how they are doing,” he said. “What are some challenges that they’re having, specifically around mental health, since mental health in college is really such an important topic,” he said. 

2021 marked the first year that autism was an endorsable category in the survey. According to paper co-author Jennifer Gillis Mattson, that added representation allows researchers to more readily conduct research on autistic students and compare them with their non-autistic peers. 

“We were really excited to see what the data would tell us. It was a big opportunity to be able to do this,” said Mattson, professor of psychology and co-director of the Institute for Child Development at Binghamton University. 

The study highlights the need for more mental health support for autistic students, said the researchers. 

“We want to provide the best support for them and to make sure that they have a college experience, where they get a lot out of it, but also feel comfortable,” said Aragon-Guevara.

For example, support personnel might address an individual’s autism and, in the process, overlook their mental health issues. More care needs to be put into addressing that nuance, said Gillis-Mattson.

“We’re shedding some light on the fact that if you have autistic college students in your college population – and we know the number of autistic college students continues to increase every single year – then we really do have an obligation to support these students,” said Gillis-Mattson. “And to know how best to support these students and we need to look beyond just autism, if you will. That there are these other mental health conditions, such as anxiety or depression, where people need to be able to acknowledge and understand that additional supports may be needed.”

Aragon-Guevara said that this new research is a starting point – confirmation that there is an issue regarding mental health in autistic college students. The researchers next want to identify the specific factors that influence these mental health challenges, whether it’s social dynamics, support from faculty, accessibility, etc. 

“There are so many elements that go into being comfortable in the new environment that is college, so we want to look into that and see if there are any deficits in those areas that autistic college students are experiencing, so that we know where we can help support them, or create institutional things to help improve quality of life as a whole,” said Aragon-Guevara.

This study is part of a broader research effort at Binghamton to better understand and support autistic students in higher education. Hyejung Kim, an assistant professor in the Department of Teaching, Learning and Educational Leadership, noted that there is still much to explore about this population. 

“This population often skews male, and interactions between personal factors and conditions such as anxiety and depression may shape overall well-being in college, an area that warrants further study,” she said. “Autistic students are also more likely to pursue STEM fields, and many report different experiences with faculty and staff across institutional settings. We still have much to learn about how these and other contextual factors relate to mental well-being.” 

The research team plans to examine these factors more closely, in collaboration with campus partners, to inform targeted supports that help autistic students thrive.

A new study from researchers at the University of Illinois Chicago reveals how the blood-brain barrier gets leakier with age, contributing to memory deficits. The study, published in Cell Reports, uncovered the molecular mechanisms behind this process and could provide new therapeutic targets to address cognitive decline earlier in the aging process.

The blood-brain barrier is a layer of cells lining the brain’s blood vessels that keep viruses, bacteria and toxins out while allowing helpful nutrients and chemicals in. A key structure of the blood-brain barrier are tight junctions that act as bridges between cells, restricting entry of molecules. A protein called occludin helps fulfill this essential role.

It’s a highly regulatable process that allows some molecules to go through and others to remain in circulation. Basically, it’s a mechanism that separates the central nervous system from everything else.”

Yulia Komarova, UIC associate professor in the department of pharmacology and regenerative medicine at the College of Medicine and senior author of the study

But like many physiological processes, the blood-brain barrier starts to malfunction as we age – it gets leakier. This can lead to memory changes as early as middle age, Komarova said. Exactly how this occurs is unclear.

In previous research, Komarova and her colleagues tested what happened if they deleted a protein called N-cadherin from the cells lining blood vessels. This made the vessels leakier in the lungs – and in the brain.

In the new study, Komarova teamed up with Leon Tai, associate professor of anatomy and cell biology in the College of Medicine, to see if this leakage had any effect on memory. Mice without functional N-cadherin could learn tasks as well as normal mice, but they quickly forgot what they learned.

A closer look at the brains of these mice showed that the issue was linked to a protein called occludin, which helps form tight junctions in the blood-brain barrier. Both aging brains and young brains lacking N-cadherin had fewer occluding junctions, resulting in a leakier barrier. Molecular experiments showed that when N-cadherin proteins on neighboring blood vessel cells interact, they trigger a signaling pathway that stabilizes occludin, helping to maintain the integrity of the blood-brain barrier.

Komarova collaborated with Dr. Jeffrey Loeb, head of neurology and rehabilitation in the College of Medicine, to examine human brain tissue from the university’s NeuroRepository collected during epilepsy surgeries. Comparing samples from younger patients (late teens to 20s) with those from middle-aged patients (40s to 50s), they found that the older group had reduced levels of both N-cadherin and occludin, mirroring the findings in mice.

The study is the first to look at how the signaling activated by N-cadherin controls organization of the tight junctions implicated in blood-brain barrier permeability, she said.

Because these deficits start to show in middle age, fairly early in the cognitive aging process, it’s not “too late in the game to start treatment,” said Komarova. Her team is now investigating if steps in the signaling pathway activated by N-cadherin could be therapeutic targets.

“This paper shows that actually there might be a much bigger therapeutic window for treatment of any age-related cognitive decline condition,” she said.

Other UIC co-authors on the paper include Quinn Lee, Wang Ching Chan, Shuangping Zhao, Harry Hailemeskel, Riya Thomas, Mohsin Zafar, Fozia Mir, Peter Toth and Kamran Avanki.

US Centers for Disease Control and Prevention Director Susan Monarez vowed to rebuild trust with the American public after the agency was the target of a deadly shooting last week, but some staff were frustrated that she didn’t offer more information on safety.

The gunman who shot nearly 200 rounds into four buildings and two security guard stands on the CDC campus Aug. 8 outlined his frustration with Covid vaccines before the rampage, according to the Georgia Bureau of Investigation. The attack occurred as medical misinformation is rampant in the US, fueling everything from political positioning to consumer purchases — and increasingly violent attacks.

SEOUL, South Korea, Aug. 12, 2025 /PRNewswire/ — A newly published post-hoc analysis of the landmark FUEL (Fontan Udenafil Exercise Longitudinal) Trial demonstrates that udenafil, a PDE5 inhibitor, significantly improves peak oxygen consumption (peak VO₂) in adolescents with single ventricle congenital heart disease (SV-CHD) who have undergone the Fontan procedure and have reduced exercise capacity.

Published in the Journal of the American Heart Association: “Revisiting the Effect of Udenafil on Exercise Performance in Patients With Fontan Circulation: Results of a Post Hoc Analysis of the FUEL Trial”

The original multinational, randomized, placebo-controlled Phase 3 FUEL trial demonstrated statistically significant improvements in key secondary endpoints – such as VO₂ at ventilatory anaerobic threshold (VAT) and myocardial performance index (MPI) – but narrowly missed statistical significance for the primary endpoint of peak VO₂ in the overall population. Recognition of a high performing “Super Fontan” subgroup prompted further analysis stratifying participants by baseline peak VO₂ (<80% vs. ≥80% of predicted) to assess whether initial exercise capacity influenced treatment response.

Key findings from the post-hoc analysis:

• In the 302 patients with baseline peak VO₂ <80% predicted (80% of the evaluable cohort), udenafil significantly improved peak VO₂ compared to placebo (p=0.021).
• Significant improvements were also observed in VO₂ at VAT (p=0.023), work at VAT (p=0.032), and MPI (p=0.007).
• A significant interaction between baseline exercise capacity and treatment effect for peak VO₂ (p=0.036) suggests that including high-functioning patients, whose physiologic ceiling may limit measurable gains, could have masked the full treatment effect in the original trial.

“These results provide a possible physiologic explanation for the outcomes of the FUEL trial and support further investigation of udenafil in Fontan patients with reduced exercise capacity,” said Dr. David Goldberg, lead author and pediatric cardiologist at Children’s Hospital of Philadelphia. “This analysis highlights the need for a confirmatory clinical trial targeted to the large majority of Fontan patients who are most likely to demonstrate benefit using peak VO₂ as the primary endpoint.”

“Importantly, the absence of a detectable change in peak VO₂ among higher-functioning Fontan patients may reflect– what may be termed a ‘ceiling effect’ – rather than a lack of therapeutic response,” added Dr. Bryan H. Goldstein, senior author and Director of the Cardiac Catheterization Laboratory at UPMC Children’s Hospital of Pittsburgh. “These patients still achieve meaningful improvements in submaximal exercise measures such as VO₂ at VAT and MPI. Moreover, as these younger, high-performing individuals age and are further exposed to ongoing Fontan circulatory stresses, their exercise capacity with decline and therefore, the need for targeted pharmacotherapy will likely increase.”

The findings highlight the importance of selecting appropriate endpoints and stratifying populations in rare disease trials. Informed by FUEL, the ongoing confirmatory phase 3 FUEL-2 trial focuses on Fontan patients with reduced baseline exercise capacity – those in whom changes in peak VO₂ are most reliably measurable.

“This focused design increases the likelihood of demonstrating efficacy on the primary endpoint, while still capturing udenafil’s broader therapeutic potential,” said Dr. Rahul Rathod, Global Principal Investigator for FUEL-2 and Director of the Single Ventricle Program at Boston Children’s Hospital. “Given the strong link between peak VO₂ and long-term outcomes in Fontan patients, a +1.13 mL/kg/min improvement over six months is clinically meaningful and may influence disease progression.”

The FUEL trial was supported by the National Heart, Lung, and Blood Institute (NHLBI) and Mezzion Pharma Co., Ltd., the regulatory sponsor of udenafil development for Fontan patients.

About Mezzion

Mezzion Pharma Co., Ltd. is a biopharmaceutical company developing therapies for rare and orphan diseases. Udenafil is currently being evaluated in the Phase 3 FUEL-2 trial for improving exercise performance in adolescents and young adults with Fontan circulation.

For more information, please visit www.mezzion.com and www.FUEL2Study.com.

Media Contacts:
John Presser, Chief Business Officer – [email protected]
Sung-Il Noh, Chief Financial Officer – [email protected]

SOURCE Mezzion Pharmaceuticals, Inc

US Centers for Disease Control and Prevention Director Susan Monarez vowed to rebuild trust with the American public after the agency was the target of a deadly shooting last week, but some staff were frustrated that she didn’t offer more information on safety.

The gunman who shot nearly 200 rounds into four buildings and two security guard stands on the CDC campus Aug. 8 outlined his frustration with Covid vaccines before the rampage, according to the Georgia Bureau of Investigation. The attack occurred as medical misinformation is rampant in the US, fueling everything from political positioning to consumer purchases — and increasingly violent attacks.

BBC

Diagnosis within an hour

ELI-002 2P, a lymph node–targeting amphiphile peptide–based vaccine, improved relapse-free survival (RFS) in patients with KRAS-mutated pancreatic ductal adenocarcinoma (PDAC) and colorectal cancer (CRC) correlated to a tumor biomarker, according to results from a phase 1 study published in Nature Medicine.¹

At a median follow-up of 19.7 months, the median overall survival (OS) was 28.94 months, and the median radiographic RFS was 16.33 months in patients who received ELI-002 2P after locoregional therapy. Crucially, investigators identified that patients with a T-cell response above a 9.17-fold threshold had significantly superior outcomes to those who did not.

Targeting the KRAS G12D and G12R mutations, ELI-002 2P is designed to deliver peptide antigens and amphiphile-adjuvant CpG-7909 to the lymph nodes to enhance KRAS-specific T-cell activation and amplification. Earlier reporting from the phase 1 AMPLIFY-201 trial (NCT04853017) established the safety and immunogenicity of ELI-002 2P.²

Twenty-five patients at 7 centers in the United States were enrolled to receive ELI-002 2P with fixed doses of the antigen peptides and escalating doses of Amph-CpG-7909, 20 of whom had PDAC and 5 of whom had CRC. Patients had high-risk stage I, II, III, or oligometastatic stage IV PDAC or high-risk stage II, III, or oligometastatic stage IV CRC. All patients had positive minimal residual disease (MRD) or after locoregional therapy. The primary end point was safety and secondary end points included reduction in circulating tumor DNA (ctDNA) and/or serum tumor antigen levels.

Patients received 6 subcutaneous doses of ELI-002 2P over 8 weeks followed by 3 months of observation, then 4 weekly doses of ELI-0002 2P. They were followed for up to 2 years after the first dose of ELI-002 2P.

No dose-limiting toxicities were observed and a recommended phase 2 dose of 10.0 mg of Amph-CpG-7909 was determined. At the initial data cutoff with 8.5 months’ median follow-up, 21 of 25 patients had mutant KRAS (mKRAS)-directed T-cell responses, with CD4+ and CD8+ T cells in 71% and responses to all 7 mKRAS antigens were present in 57%. All patients who received the 2 highest dose levels had T-cell responses.

With longer follow-up, post hoc analyses of immunogenicity and clinical outcomes were reported.¹ In the PDAC cohort, median RFS was 15.31 months, and median OS was 28.94 months, which was consistent with the full cohort that also included the 5 patients with CRC.

Using an exploratory receiver-operating characteristics analysis, investigators identified the 9.17-fold threshold in T-cell activation that separated patients with better or worse outcomes, after previously reporting outcomes based on the median of 12.75-fold change from baseline. With the new analysis, 17 patients (68%) had better outcomes and 8 (32%) did worse. All 8 of the patients with T-cell change below 9.17-fold experienced radiographic progression and 7 had died. However, 11 of the 17 (65%) with greater change in their T cells had no radiographic progression, 5 required no subsequent therapy and 6 received subsequent chemotherapy based on tumor biomarker increase but remained free from disease progression. There were 6 patients in this group who had complete ctDNA clearance.

Investigators calculated an increase in relative risk of radiographic progression or death of 2.96 for the below-threshold group. The median RFS was not reached vs 3.02 months for the above- and below-threshold groups (HR, 0.12; P = .0002). The median OS was not reached in the above-threshold group vs 15.98 months in the below-threshold group (HR, 0.23; P = .0099). The investigators compared these outcomes favorably to the historical progression of patients with MRD-positive PDAC after resection.

The immunogenicity analyses also showed antigen spreading patient-specific neoantigens not included in ELI-002 2P. T-cell response to nonvaccine antigens were expanded from baseline levels in 67% (6 out of 9 patients) of evaluated patients, and 13 of 52 evaluated neoantigens showed increased T-cell responses. Five of 6 patients with antigen-spreading responses were those with responses above the 9.17-fold threshold.

No new safety signals were reported at the time of this analysis; common treatment-related adverse events previously observed included fatigue, malaise, diarrhea, abdominal distention, and abdominal pain.

A 7-peptide formulation, ELI-002 7P, is also under investigation to target a greater number of KRAS mutations. It showed robust responses in the phase 1 AMPLIFY-7P trial (NCT05726864) and a randomized phase 2 trial is underway in the adjuvant setting for PDAC.

“The updated phase 1 AMPLIFY-201 data further demonstrate that the AMP platform has the potential to provide durable benefit to [patients with] PDAC in the adjuvant setting,” said Chris Haqq, MD, PhD, chief medical officer of Elicio Therapeutics, in a press release.³

_References:

_{1. Wainberg ZA, Weekes CD, Furqan M, et al. Lymph node-targeted, mKRAS-specific amphiphile vaccine in pancreatic and colorectal cancer: phase 1 AMPLIFY-201 trial final results. Nat Med. Published 11 August 2025. doi:10.1038/s41591-025-03876-4}

_{2. Pant S, Wainberg ZA, Weekes CD, et al. Lymph-node-targeted, mKRAS-specific amphiphile vaccine in pancreatic and colorectal cancer: the phase 1 AMPLIFY-201 trial. Nat Med. 2024;30(2):531-542. doi:10.1038/s41591-023-02760-3}

_{3. Elicio Therapeutics announces publication of ELI-002 updated AMPLIFY-201 phase 1 follow-up data in nature medicine for minimal residual disease (“MRD”) positive, adjuvant-stage patients. Elicio Therapeutics. News release. August 12, 2025. Accessed August 12, 2025. https://elicio.com/press_releases/elicio-therapeutics-announces-publication-of-eli-002-updated-amplify-201-phase-1-follow-up-data-in-nature-medicine-for-minimal-residual-disease-mrd-positive-adjuvant-stage-patient/}

Despite increasing evidence linking alcohol consumption to cancer, little is known about the biological mechanisms behind the association. A new study, published Aug. 12 in Cellular and Molecular Gastroenterology and Hepatology, suggests that inhibiting a cellular molecule called CREB might thwart pancreatic tumor development in response to alcohol.

Our model serves as an important platform for understanding how chronic inflammation related to alcohol consumption accelerates the development of pancreatic cancer.”

Siddharth Mehra, Ph.D., scientist at Sylvester Comprehensive Cancer Center, and first author on the study

Chronic, high alcohol use damages acinar cells in the pancreas, specialized cells that produce digestive enzymes. The damage in turn causes the cells’ enzymes to increase inflammation in the tissue, exacerbating damage to the pancreas.

Over time, precancerous lesions can develop, increasing the risk for full-blown pancreatic cancer, one of the deadliest types of tumors. Previous studies have implicated CREB, a DNA-binding protein that regulates gene activity, and associated molecules in helping to mediate this process.

Progression to cancer also generally requires other cellular events, such as a mutation in a pro-cancerous gene called Ras, which commonly occurs in pancreatic tumors.

In the new study, the researchers developed a model that recapitulated alcohol-induced inflammation, the development of pre-cancerous lesions and progression to cancer. The model contained Ras mutations in acinar cells, and it also had an intact CREB gene that could be experimentally knocked out in these cells.

The researchers found that exposure to alcohol and a pro-inflammatory molecule caused the development of symptoms similar to alcohol-induced pancreatitis, an inflammatory condition. Inflammation in turn prompted the development of precancerous lesions and, later, cancer. Consistent with previous studies, CREB was highly activated throughout this transition process.

The researchers next knocked out CREB and found that they could quell the development of precancerous and cancerous lesions, even in the continued presence of alcohol. Knocking out CREB also relieved damage to acinar cells.

The findings hint that inhibitors of CREB might have therapeutic potential in people who have high alcohol use. Such inhibitors could potentially relieve damage to the pancreas and thwart tumor development, said the researchers.

“We found that CREB is not just a mediator of inflammation; it is a molecular orchestrator that permanently converts acinar cells into precancerous cells, which ultimately progress to high-grade neoplasia,” said senior author Nagaraj Nagathihalli, Ph.D., associate professor of surgery and assistant director of the Sylvester Pancreatic Cancer Research Institute at the University of Miami.

Future studies should help provide additional information about how alcohol use promotes pancreatic cancer development.

Questions include whether similar events occur in human cells and tissues and what other molecules and cells play a role in the process. CREB activation may also be involved in other alcohol-linked cancers, speculates Nagathihalli.

He and colleagues are also leveraging the model to investigate the potential of CREB inhibitors, which are under development as potential cancer therapeutics.

“We believe this study lays the groundwork for future translational efforts targeting CREB as a therapeutic vulnerability in inflammation-associated pancreatic cancer,” said study co-author Nipun Merchant, M.D., Sylvester associate director of translational science and chief of surgical oncology.

The U.S. surgeon general recently declared alcohol the third leading preventable cause of cancer.

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