Category: Cancer

Categories : CancerTags :

Previously Untreatable Blood Cancer Has a New Gene Therapy

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Patient Alyssa Tapley

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at University College London and Great Ormond Street Hospital (GOSH), has shown promising results in helping children and adults fight a rare and aggressive form of blood cancer.

Using base-editing technology, the pioneering gene therapy (BE-CAR7), has been used to treat a previously untreatable type of blood cancer called T-cell acute lymphoblastic leukaemia (T-ALL). Base-editing is an advanced version of CRISPR technology, that can precisely change single letters of DNA code inside living cells.

BE-CAR7 was designed and developed by a team of researchers, led by Professor Waseem Qasim (UCL Great Ormond Street Institute of Child Health), who is also an Honorary Consultant at Great Ormond Street Hospital (GOSH), and the treatment is the first in-human application of base-edited cells. 

In 2022, Alyssa Tapley, from Leicestershire, (then aged 13) became the first person in the world to receive the treatment as part of a clinical trial at GOSH. Doctors had exhausted all other treatments for her and she and her family say the innovative therapy saved her life.

Now, a further eight children and two adults have undergone the treatment as part of the trial.

The trial’s results have now been published in the New England Journal of Medicine. Key findings from the study include:

  • 82% of patients achieved very deep remissions after BE-CAR7, enabling them to proceed to stem cell transplant without disease
  • 64% remain disease-free, with the first patients now three years disease-free and off treatment
  • Anticipated side effects including low blood counts, cytokine release syndrome and rashes were tolerable, with the greatest risks arising from virus infections until immunity recovered

Professor Qasim, a Professor of Cell and Gene Therapy at UCL, who led the research, said: “A few years ago this would have been science fiction. Now we can take white blood cells from a healthy donor and change a single letter of DNA code in those cells and given them back to patients to try to tackle this hard-to-treat leukaemia.

“We designed and developed the treatment from lab to clinic and are now trialling it on children from across the UK – in a unique bench-to-bedside approach.”

Alyssa, now aged 16, who has a brother, said: “It is incredible how much my life has changed. I went from four months straight in GOSH to now only coming back for medical appointments once a year. It is amazing how much freedom I have now.

“I am really grateful for all the opportunities the gene therapy treatment has given me. I feel like I have been able to help everyone else who went on the clinical trial after me. 

“I’ve now been able to do some of the things I thought earlier in my life it would be impossible for me to do. I really did think I was going to die and that I wouldn’t be able to grow up and do everything that every child deserves to be able to do.”

That has included going sailing and spending time away from home doing her Duke of Edinburgh Award.

Her dad, James, said: “We are eternally grateful. We’ve gone from being completely hopeless to where we are now. We could never have imagined that then.

“Back then we were at the point where we thought we were going to be a three-person family. Now we’re all making the most of family life. It is the little things you don’t take for granted anymore. Alyssa is amazing.

“The scientists at UCL and GOSH have been incredible. It has been really powerful seeing them in the laboratory developing the treatment and hearing the stories including about the difficulties they faced in getting this far.

“In terms of the timing of the trial, it aligned perfectly for us. Other families weren’t so lucky.”

New technology

Immunotherapy using CAR-T cells has recently become available to treat several types of blood cancer. This therapy uses immune cells, called T-cells, and modifies them to have specific proteins on their surface called chimeric antigen receptors (CARs).

The CARs can recognise and target specific ‘flags’ on the surface of cancer cells, and the T-cell can then destroy that cancer cell. Developing CAR T-cell therapy for leukaemia which itself has arisen from abnormal T-cells has been challenging. 

BE-CAR7 T-cells are engineered using base editing, a new-generation of genome editing that avoids cutting DNA, reducing the risk of chromosomal damage. 

Very precise chemical reactions were carried out using CRISPR guidance systems to change single letters of DNA code in order to modify the T cells. These complex DNA changes generate storable banks of ‘universal’ CAR T-cells that can find and attack T-cell leukaemia when given to patients.  

The ‘universal’ CAR T-cells in this study were made from healthy donor white blood cells and engineering steps were undertaken in a clean room facility using custom made RNA, mRNA and a lentiviral vector in an automated process previously developed by the research team.

These steps were:  
1.    Removing existing receptors so that T-cells from a donor can be banked and used without matching the recipient– making them ‘universal’.    
2.    Removing a ‘flag’ called CD7 that identifies them as T-cells (CD7 T-cell marker). Without this step, T-cells programmed to kill T-cells would simply end up destroying the product through ‘friendly-fire’. 
3.    Removing a second ‘flag’ called CD52. This makes the edited cells invisible to one of the strong antibody drugs given to patients to subdue their immune system.  
4.    Adding a Chimeric Antigen Receptor (CAR) which recognises the CD7 T-cell flag on leukemic T-cells.  A disabled virus added extra DNA code into the cells so they become armed against CD7 and recognise and fight T-cell leukaemia. 

When base-edited CAR T-cells are given to the patient they rapidly find and destroy all T-cells in the body, including leukemic T-cells.
If the leukaemia is eradicated within four weeks, the patient’s immune system is then rebuilt from a bone marrow transplant over a period of several months.   

Professor Qasim added: “Many teams were involved across the hospital & university and everyone is delighted for patients clearing their disease, but at the same time, deeply mindful that outcomes were not as hoped for some children. These are intense and difficult treatments – patients and families have been generous in recognising the importance of learning as much as possible from each experience.”

Dr Rob Chiesa, Study investigator and Bone Marrow Transplant consultant at GOSH said: “Although most children with T-cell leukaemia will respond well to standard treatments, around 20% may not. It’s these patients who desperately need better options and this research provides hope for a better prognosis for everyone diagnosed with this rare but aggressive form of blood cancer.

“Seeing Alyssa go from strength-to-strength is incredible and a testament to her tenacity and the dedication of an array of small army of people at GOSH. Team working between bone marrow transplant, haematology, ward staff, teachers, play workers, physiotherapists, lab and research teams, among others, is essential for supporting our patients.”

Dr Deborah Yallop, consultant Haematologist at KCH said “We’ve seen impressive responses in clearing leukaemia that seemed incurable – it’s a very powerful approach.”

Source: University College London

Categories : Cancer DermatologyTags :

Lower Doses of Immunotherapy for Skin Cancer Give Better Results

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Photo by Bermix Studio on Unsplash

According to a new study, lower doses of approved immunotherapy for malignant melanoma can give better results against tumours, while reducing side effects. This is reported by researchers at Karolinska Institutet in the Journal of the National Cancer Institute.

“The results are highly interesting in oncology, as we show that a lower dose of an immunotherapy drug, in addition to causing significantly fewer side effects, actually gives better results against tumours and longer survival,” says last author Hildur Helgadottir, a researcher at the Department of Oncology-Pathology at Karolinska Institutet, who led the study.

The traditional dose of nivolumab and ipilimumab is the one that is approved and established. Due to the extensive side effects, Sweden has increasingly begun to use a treatment regimen with a lower dose of ipilimumab, which is both gentler and cheaper. Ipilimumab is the most expensive part of this immunotherapy and causes the most side effects.

“In Sweden, we have greater freedom to choose doses for patients, while in many other countries, due to reimbursement policies, they are restricted by the doses approved by the drug authorities,” says Hildur Helgadottir.

Lower dose is more effective

The study included nearly 400 patients with advanced, inoperable malignant melanoma, the most serious form of skin cancer. The study shows that the regimen with the lower dose of ipilimumab is more effective, with a higher proportion of patients responding to treatment, 49%, compared to the traditional dose, 37%.

Progression-free survival, the time the patient lives without the disease worsening, was a median of nine months for the lower dose, compared to three months for the traditional dose. Overall survival was also longer, 42 months compared to 14 months.

Serious side effects were seen in 31% of patients in the low-dose group, compared to 51% in the traditional group.

“The new immunotherapies are very valuable and effective, but at the same time they can cause serious side effects that are sometimes life-threatening or chronic. Our results suggest that this lower dosage may enable more patients to continue the treatment for a longer time, which is likely to contribute to the improved results and longer survival,” says Hildur Helgadottir.

There were some differences between the two treatment groups, but even after adjusting for several factors such as age and tumour stage, the better outcome for the lower dose of ipilimumab remained. The study is a retrospective observational study and therefore it is not possible to definitively establish a causal relationship.

Source: Karolinska Institutet

Categories : CancerTags :

Tough Enough to Save a Life? SA Athletes Challenge Men to Register as Stem Cell Donors

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Jaco Pretorius was inspired by his best friend’s life being saved by a stem cell transplant.

With South African men having a 20-50% higher incidence rate of blood cancers than women, sports icons Jaco Pretorius, Seabelo Senatla, and Temba Bavuma are leveraging their platforms to challenge more men to join the stem cell registry and help rewrite the odds for patients in need.

The challenge confronts a widespread public disconnect from the issue. “Many people seem to be disinterested, until one of their own is diagnosed,” notes Senatla. “People tend to be nonchalant when things don’t pertain to them; they have this attitude of ‘not my problem’.”

Pretorius agrees that a lack of awareness is a major hurdle. “My experience is that people are not aware of the great need in our country and the simplicity of the process. But we’ve seen so many times how sport has the power to unite South Africa. When athletes from different backgrounds set an example, I believe people will follow, and together we can make a real difference.”

His advocacy is rooted in his own firsthand experience. Motivated to register after his best friend’s life was saved by a transplant, he was later called upon to donate. He hopes sharing his story will dismantle common fears. “The perception is that it is a painful procedure which carries personal risk. My experience was the complete opposite.”

A concern Bavuma often hears about is the time commitment, especially for those with demanding jobs or family responsibilities. “But if you do get the call to donate, those few days potentially add years, even decades, to someone else’s life. That’s a trade any of us should be willing to make.”

Tackling a common myth, Senatla says, “One of the biggest myths I’ve had to debunk is people having the notion that since stem cells are taken from them, they’ll be left with fewer stem cells. The body of a healthy person is constantly producing stem cells. You’re not in danger of having too little if you donate some to someone.”

For him, the motivation to act is deeply personal. “I grew up in an environment in which I was made to understand that your gifts are not only for you. Other people must benefit as well. That’s what’s in practice here.”

Addressing men who might be hesitant, Senatla points out that they aren’t losing anything by registering. “Rather, they’re affording someone who is ill a second chance at life.”

Bavuma challenges the passive mindset. “In cricket, you can’t field, thinking someone else will take the catch. The same goes for this. Too many people assume there are enough donors already, or that someone else will register.”

Pretorius adds, “I would encourage other people to immediately take action. The process is pain-free, professional, non-invasive, and there are no financial implications – only your time and commitment.”

The outcome of his simple act was profound. “I received communication from the stem cell recipient that the transplant was successful, and the person is healthy and well. That was one of the best feelings – to know that through such a simple action, someone else’s life was saved.”

Palesa Mokomele, Head of Community Engagement and Communications at DKMS Africa, highlights the impact of these role models. “While men currently make up the majority of registered donors in South Africa, the overall pool of donors is critically low compared to the national need. Having respected public figures like Jaco, Seabelo, and Temba lead this conversation is invaluable. When men see other men stepping up, it directly challenges hesitation, shifts perceptions, and ultimately helps save lives.”

Senatla offers a reminder of our shared humanity. “You’re never too important to help, and you should help because you can. Being in a position to help is an absolute privilege.”

“This isn’t a spectator sport; everyone who can help needs to get in the game,” concludes Bavuma.

South Africans aged 17 to 55 who are in good health can register as potential stem cell donors. The process is simple and starts with an online registration and a cheek swab.

Register today at https://www.dkms-africa.org/save-lives.

Categories : CancerTags :

Scientists Develop One-product-fits-all Immunotherapy for Breast Cancer

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Breast cancer cells. Image by National Cancer Institute

Triple-negative breast cancer is one of the most aggressive cancers. The name tells the story: It lacks the three main targets that make other types of breast cancers more treatable with powerful therapies.

UCLA researchers have developed a novel therapy that could fundamentally change the treatment plan for this deadly disease. In a study published in the Journal of Hematology & Oncology, the team details how this new type of immunotherapy, called CAR-NKT cell therapy, could attack tumors from multiple fronts while dismantling their protective shields.

“Patients with triple-negative breast cancer have been waiting far too long for better treatment options,” said senior author Lili Yang, a professor of microbiology, immunology and molecular genetics and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. “To finally have a therapy that shows superior cancer-fighting ability – and to be just one step away from clinical testing – is incredibly exciting.”

The therapy uses engineered immune cells called CAR-NKT cells, which can be mass-produced from donated blood stem cells and stored ready-to-use. This off-the-shelf approach offers an immediately available treatment option at a fraction of the cost of current personalized cell therapies, which can soar into the hundreds of thousands of dollars.

A triple threat against a triple-negative cancer

CAR-T cell therapies have transformed treatment for certain blood cancers by turning patients’ own immune cells into precision weapons. However, these therapies have struggled against solid tumours like breast cancer, which employ sophisticated defence mechanisms and constantly evolve to evade treatment.

To tackle these hurdles, the UCLA team’s cell therapy harnesses a rare but powerful type of immune cell called invariant natural killer T cell, or NKT cell. When equipped with a chimeric antigen receptor, or CAR, targeting mesothelin (a protein found on triple-negative breast cancer cells) these potent tumour-fighting cells gain the ability to recognise and destroy cancer through three distinct mechanisms.

The first mechanism uses the engineered CAR to target mesothelin, which is associated with more aggressive, metastatic disease. The second leverages the cells’ natural killer receptors that recognize more than 20 molecular markers, making it nearly impossible for tumours to evade all of them. The third employs the cells’ unique T cell receptor to reshape the tumour microenvironment by eliminating immunosuppressive cells.

“We’re not just targeting one molecular marker on cancer cells — we’re identifying dozens of them simultaneously,” said first author Yanruide (Charlie) Li, a postdoctoral scholar in the UCLA Broad Stem Cell Research Center Training Program. “It’s like attacking a fortress from every direction at once. The cancer simply can’t adapt fast enough to escape.”

When the research team tested the novel therapy on tumour samples from patients with late-stage metastatic breast cancer, the CAR-NKT cells successfully killed cancer cells in every single sample tested, while also eliminating the immunosuppressive cells that tumours recruit as protective escorts.

Engineering universal accessibility

Beyond its multipronged cancer-fighting capabilities, the CAR-NKT platform addresses critical barriers that have limited cell therapy access: manufacturing complexity and cost.

Current cellular immunotherapies require collecting each patient’s immune cells, shipping them to specialised laboratories for genetic modification, then returning the customized product into the patient weeks later — a process that can cost six figures and create dangerous delays for patients with aggressive cancers.

Yang’s team takes a fundamentally different approach. Because NKT cells naturally work with any immune system, they can be mass-produced from donated blood stem cells using a scalable system. A single donation could generate enough cells for thousands of treatments, reducing costs to approximately $5,000 per dose.

One product to tackle multiple cancers

The therapy’s promise extends beyond triple-negative breast cancer. Since mesothelin is also highly expressed in ovarian, pancreatic and lung cancers, the same cell product could potentially treat multiple cancer types that remain difficult to address with current immunotherapies.

“This is really a platform technology,” said Yang, who’s also a member of the UCLA Health Jonsson Comprehensive Cancer Center.

With all preclinical studies complete for both triple-negative breast cancer and ovarian cancer, the team is preparing to submit applications to the Food and Drug Administration to begin clinical trials.

“We’ve walked 99 steps to get here,” Yang said. “We’re missing just one final step to begin clinical testing and demonstrate what this promising therapy can really do for patients.”

Source: University of California – Los Angeles

Categories : CancerTags :

Many Men May Not Need Long-term Hormone Therapy for Prostate Cancer

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Wild type human prostate cells from an organoid. Credit: National Cancer Institute, National Institutes of Health

A study co-led by investigators at the UCLA Health Jonsson Comprehensive Cancer Center found that most of the benefits of androgen deprivation therapy (ADT) for prostate cancer occur within the first 9 to 12 months. Extending therapy beyond that provides only a small additional protection and increases the risk of other health problems, such as heart or metabolic issues. Results show that the ideal length of ADT depends on cancer risk:

  • Low-risk patients may not need ADT.
  • Intermediate-risk patients benefit most from 6 to 12 months.
  • High-risk patients may benefit from up to 12 months, while very high-risk patients may require longer therapy.

The study was published in the journal JAMA Oncology. 

ADT is a type of hormone therapy that is commonly given alongside radiation to slow the growth of prostate cancer by lowering testosterone. While effective at controlling the disease, long-term ADT can cause side effects, including bone loss, muscle loss and cardiovascular problems. Current treatment guidelines generally recommend 4 to 6 months of ADT for intermediate-risk patients and 18 to 36 months for high-risk patients, but the optimal duration has been unclear.

Researchers conducted a Meta-Analysis of Randomized Trials in Cancer of the Prostate (MARCAP) Consortium using data from 10 266 men across 13 international clinical trials. They assessed outcomes including overall survival, cancer-specific survival and deaths from other causes, comparing different ADT durations. 

The findings highlight the importance of personalised treatment plans for men with prostate cancer. Shorter courses of hormone therapy may be sufficient for many patients, reducing side effects while maintaining effectiveness. Physicians can now use patient-specific factors, including cancer risk, overall health, age and preferences, to make more informed decisions about ADT duration, improving both safety and quality of life.

Source: University of California – Los Angeles

Categories : CancerTags :

UK Launches World’s First Trial of Lung Cancer Vaccine

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Image of a lung lobe showing cells expressing the basal cell marker Krt5 spreading. Credit: UCL.

In the UK, people at high risk of lung cancer will soon be able to receive the first ever experimental vaccine designed to prevent the disease, in a world-first clinical trial led by researchers at UCL and the University of Oxford.

The research team has been awarded up to £2.06 million from Cancer Research UK, supported by the CRIS Cancer Foundation, to run a clinical trial of LungVax over the next four years.

This phase I trial will investigate the best dose of LungVax to give to people at high risk of lung cancer, as well as looking for any potential side-effects from different doses of the vaccine.

The trial is expected to begin in summer 2026, subject to regulatory approvals.

Professor Mariam Jamal-Hanjani, co-founder and lead for the LungVax clinical trials, from UCL Cancer Institute, UCLH and the Francis Crick Institute, said: “Fewer than 10% of people with lung cancer survive their disease for 10 years or more. That must change, and that change will come from targeting lung cancer at the earliest stages.

“The LungVax clinical trial is the crucial first step in bringing this vaccine to people at the highest risk of the disease. We will be looking carefully at how people respond to the vaccine, how easy it is to deliver, and who might benefit from it most in the future.

“Preventative vaccines will not replace stopping smoking as the best way to reduce the risk of lung cancer. But they could offer a viable route to preventing some cancers from emerging in the first place.”

Lung cancer cells are different from normal cells. They have ‘red flag’ proteins made by cancer-causing mutations within their DNA. These are called neoantigens and tumour associated antigens and appear on the surface of cells at a very early stage of lung cancer formation.

The LungVax vaccine carries a series of genetic instructions which train the immune system to recognise these tumour antigens on the surface of abnormal lung cells. In trialling the vaccine, the aim is to get the immune system to recognise these early abnormal cells, and kill them before they start to become cancer. The vaccine uses technology developed by the University of Oxford during the COVID-19 pandemic to deliver these instructions to the immune system.

Professor Sarah Blagden, co-founder of the LungVax project from the University of Oxford, said: “Lung cancer is lethal and blights far too many lives. Survival has been stubbornly poor for decades. LungVax is our chance to do something to actively prevent this disease.

“Years of research into the biology of cancer, understanding the fundamental changes which occur in the very earliest stages of the disease, will now be put to the test. This funding means that, for the first time, we hope that people will be able to receive LungVax in clinical trials from next year.” 

To find out how safe and effective the vaccine is, the trial will initially focus on people who have been diagnosed with early-stage lung cancer and have had it successfully removed but are at risk of it returning. The vaccine will also be tested in people who are undergoing lung cancer screening as part of the NHS Lung Cancer Screening Programme in England.

If the trial delivers promising results, the vaccine could then be scaled up to larger trials for people at risk of lung cancer.

There are around 48 500 cases of lung cancer every year in the UK. Around 72% of lung cancers are caused by smoking, which is the biggest preventable cause of cancer worldwide.

Graeme Dickie, 55, from Kilbarchan in Renfrewshire, is helping the scientists prepare for the LungVax clinical trial. In 2013, aged 42, he was diagnosed with stage II lung cancer. By 2017, it had progressed to stage IV. He has never smoked. Over the years, he’s undergone surgery to remove part of his left lung, and more than 80 rounds of chemotherapy. When those treatments stopped working, Graeme began a new targeted treatment drug, mobocertinib, that he continues with today.

Graeme said: “I am proof that research saves lives. I have been able to enjoy many more happy years with my family thanks to scientists working hard, year after year, to bring new tests and treatments.

“For me, research is vital. I won’t be able to benefit directly from LungVax personally, but I know that my story will help others to access better interventions at an early stage.”

  • Image of a lung lobe showing cells expressing the basal cell marker Krt5 spreading. Credit: UCL.

Source: University College London

Categories : CancerTags :

Focused Ultrasound with Chemotherapy Improves Survival for Glioblastoma Patients

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Image credit: University of Maryland School of Medicine

Patients with glioblastoma who received MRI-guided focused ultrasound with standard-of-care chemotherapy had a nearly 40% increase in overall survival in a landmark trial of 34 patients led by University of Maryland School of Medicine (UMSOM) researchers. This is the first time researchers have demonstrated a potential survival benefit from using focused ultrasound to open the blood-brain barrier to improve delivery of chemotherapy to the tumour site in brain cancer patients after surgery.

“Our results are very encouraging. Using focused ultrasound to open the blood-brain barrier and deliver chemotherapy could significantly increase patient survival, which other ongoing studies are seeking to confirm and expand,” said study principal investigator Graeme Woodworth, MD, Professor and Chair of Neurosurgery at UMSOM and Neurosurgeon-In-Chief at the University of Maryland Medical Center (UMMC).

The findings of this groundbreaking safety, feasibility, and comparative trial involved glioblastoma patients who were given focused ultrasound to open their blood-brain barrier before getting chemotherapy; they were matched to a rigorously selected control group of 185 glioblastoma patients with similar characteristics who received the standard dose of the chemotherapy drug, temozolomide, without receiving focused ultrasound. Trial participants were initially treated with surgery to remove their brain tumour, followed by six weeks of chemotherapy and radiation, and up to six monthly focused-ultrasound treatments plus temozolomide.

Results were published in the journal Lancet Oncology and show that trial participants had nearly 14 months of median progression-free survival, compared to eight months in the control group. In terms of overall survival, trial participants, on average, lived for more than 30 months compared to 19 months in the control group.

The study builds on more than a decade of intensive research to test the safety and feasibility of opening the blood-brain barrier using focused ultrasound first in animal studies and then in patients. It was led by Dr Woodworth and was conducted at UMMC and four other university-affiliated clinical sites. “We also demonstrated that this could be a useful technique that enables us to better monitor patients to determine if their brain cancer has progressed,” said Dr Woodworth, who also serves as Director of the Brain Tumor Program at the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center (UMGCCC).

He and his team demonstrated that opening the blood-brain barrier facilitated the use of a “liquid biopsy,” which is a blood test that detects cancer biomarkers, which can include DNA fragments, proteins and other components from the liquid environment surrounding the tumor site.

Such biomarkers have been used in other cancers to determine whether the tumor has remained stable or has the potential to progress or even metastasize. Up until now, however, these tests have not been utilized in brain cancer patients since most components can never pass into the bloodstream from the brain due to the blood-brain barrier.

“These liquid biomarkers were found to be closely concordant with the patient outcomes over time, progression-free survival and overall survival,” said Dr Woodworth.

While temozolomide is the standard treatment for glioblastoma, the drug typically gets blocked by the blood-brain barrier with studies showing that less than 20 percent reaches the brain in patients. This study did not determine the exact amount of temozolomide to reach the brain in each patient, but earlier studies have shown that opening the blood-brain barrier before delivering chemotherapy can dramatically increase the amount that gets to the original tumor site.

Glioblastoma is the most common and deadliest type of malignant brain tumour. The five-year survival rate is only 5.5%, and patients live an average of 14 to 16 months after diagnosis when treated with surgery, radiation, and chemotherapy when appropriate. The malignancy nearly always recurs even after it is removed due to residual infiltrating cancer cells that remain after treatment.

The blood-brain barrier is a specialized network of vascular and brain cells that acts as the brain’s security system to protect against invasion by dangerous toxins and microbes. It can be opened temporarily using a specialised focused ultrasound device. This process starts with injecting microscopic inert gas-filled bubbles into the patient’s bloodstream. Guided by an MRI, precise brain regions are targeted while the injected microbubbles are circulating.

“Upon excitation under low-intensity ultrasound waves, the microbubbles oscillate within the energy field, causing temporary mechanical perturbations in the walls of the brain blood vessels,” said Pavlos Anastasiadis, PhD, an Assistant Professor of Neurosurgery at UMSOM who is an expert in ultrasound biophysics.

Prior studies led by Dr Woodworth and this trial’s co-investigators showed that opening the blood-brain barrier temporarily can be safely and feasibly performed in brain tumour patients. He and his team conducted this procedure in the first brain cancer patient in the US in 2018 at UMMC after the US Food and Drug Administration (FDA) approved the inaugural clinical trial.

Future trials could use focused ultrasound alongside other chemotherapy agents to test the effectiveness of drugs never used in brain cancer due to their ineffectiveness at crossing the blood-brain barrier.

Source: University of Maryland School of Medicine

Categories : Cancer NeurologyTags :

Study Links Food Insecurity to Tumour Growth in Paediatric Neuroblastoma

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How food insecurity may biologically intensify neuroblastoma growth, bridging social determinants of health and cancer biology 

Image Credit: Justine Ross, Michigan Medicine

Neuroblastoma remains one of the deadliest childhood malignancies, accounting for a disproportionate number of paediatric cancer deaths worldwide.

Despite major therapeutic advances, survival rates remain lower for children from socioeconomically disadvantaged families, a pattern long observed and poorly understood at the biological level.

Extending earlier National Institute of Health’s Children’s Oncology Group findings that linked poverty to poorer survival in paediatric cancers, investigators at University of Michigan Health C.S. Mott Children’s Hospital set out to develop the first experimental model to test how social determinants might influence tumour biology itself.

The team led by Erika Newman, MD, Section Head of Pediatric Surgery and Associate Director for Health Equity at the Rogel Comprehensive Cancer Center developed an innovative murine cancer model that simulated food insecurity by intermittently varying chow access, mirroring the unpredictable nutrition many families experience.

The study, recently published in Communications Biology, used established neuroblastoma validated xenograft models to observe how this stressor affected tumour growth and biologic responses.

The results were striking: the experimental group exposed to food insecurity developed significantly larger and bulkier tumours, accompanied by persistent elevation of stress hormones (corticosterone) and activation of tumour survival pathways.

“Our work builds on decades of clinical evidence linking poverty and food insecurity to poorer cancer outcomes,” said Newman.

“We set out to define the biology behind those disparities, to show how social conditions can become embedded in the body and influence how tumours grow.”

The work provides a translational framework linking social determinants of health to molecular pathways of cancer progression, paving the future for studies that explore how interventions addressing nutrition and stress might improve treatment response.

“This model gives us a scientific bridge between social context and cancer biology,” stated Newman.

“It shows that the environments our patients live in, access to food, stability, and safety are not background conditions. They are part of the biology we must confront if we want equitable outcomes.”

The research arrives at a moment of renewed concern over federal nutrition programs, with potential SNAP benefit interruptions amid government budget negotiations.

Newman emphasises that these findings reinforce the urgency of policies ensuring consistent food access for vulnerable children and families.

Newman stresses that health care must account for the realities in which families live.

She calls for systematic screening of social determinants like food insecurity and economic strain within paediatric and oncology practices, ensuring that medical care addresses both biologic and social drivers of outcome disparities.

Source: University of Michigan Medicine

Categories : CancerTags :

Could a Liquid Biopsy Test Speed up Cancer Diagnoses?

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Study shows it would lead to increases in stages I–III diagnoses and a large decrease in stage IV diagnoses.

Photo by National Cancer Institute on Unsplash

Routine screening is limited to only a few cancer types. New research indicates that routine liquid biopsy testing (multi-cancer early detection testing) could substantially reduce late-stage cancer diagnoses, allowing patients to receive treatment at earlier cancer stages, which are more likely to respond to interventions. The findings are published by Wiley online in CANCER, a peer-reviewed journal of the American Cancer Society.

Currently, routine screening is only recommended for four types of cancer, leaving approximately 70% of new cancer cases to be detected only after symptoms appear, often at an advanced stage when survival rates are lower. Multi-cancer early detection tests offer a revolutionary approach by screening for multiple cancer types simultaneously from a single blood draw.

To evaluate the impact of one such test, Cancerguard, investigators used epidemiological data from the Surveillance, Epidemiology, and End Results database and developed a simulation model of 14 cancer types, which account for nearly 80% of cancer incidence and mortality. The researchers simulated 10-year disease progression for 5 million US adults aged 50–84 years and assessed the effects of incorporating an annual blood-based multi-cancer early detection test into standard care.

The model estimated that over 10 years, supplemental multi-cancer early detection testing would lead to a 10% increase in stage I diagnoses, a 20% increase in stage II diagnoses, a 30% increase in stage III diagnoses, and a 45% decrease in stage IV diagnoses, relative to standard care. The largest absolute reductions in stage IV diagnoses were in lung, colorectal, and pancreatic cancers. The largest relative reductions were in cervical, liver, and colorectal cancers.

“Our analysis shows that multi-cancer blood tests could be a game changer for cancer control,” said Jagpreet Chhatwal, PhD, the study’s lead author and Director of the Institute for Technology Assessment at Massachusetts General Hospital and Harvard Medical School. “By detecting cancers earlier – before they spread – these tests could potentially improve survival and reduce the personal and economic burden of cancer.”

Source: Wiley

Categories : CancerTags :

Single-dose Radiation Before Surgery Can Eradicate Breast Cancer

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These two magnetic resonance imaging (MRI) scans were taken 10 months apart. On the left, the blue arrow points to the edge of a breast tumour, and the red arrow locates a biopsy clip, which appears as a black dot. The MRI on the right, which includes the biopsy clip, shows the tumour is gone after a single, targeted dose of radiation and antihormone therapy.

A single, targeted high dose of radiation delivered before other treatments could completely eradicate tumours in most women with early-stage, operable hormone-positive breast cancer, according to a study led by UT Southwestern Medical Center researchers. The findings, published in JAMA Network Open, could shift the paradigm for patients with the most common form of breast cancer, who typically undergo surgery before a regimen of radiation therapy.

“This is a major advance in the field,” said study leader Asal Rahimi, MD, Professor of Radiation Oncology. “This treatment protocol provides patients a significant time savings, spares a lot of their tissue from irradiation, and allows them to still undergo any type of oncoplastic surgery they may choose, all while very effectively treating their disease.”

Like patients with other forms of cancer, those with breast cancer are typically treated with a combination of surgery to remove tumours, medications such as hormone blockers, chemotherapy, and radiation, often in that order. In addition, many patients choose to have breast reconstructive surgeries before radiation treatment.

Having targeted radiation prior to surgery has several benefits, including a more than 100-fold smaller volume of tissue being irradiated compared with whole breast radiation; one day of radiation compared with up to 6.5 weeks of radiation, creating a huge time savings for patients; and more options for patients seeking reconstructive surgery, explained Dr Rahimi.

Early-stage, hormone-positive breast cancer accounts for 60–75% of all breast cancers. Seeking a more time-efficient way to treat these patients, Dr Rahimi and her colleagues tested a strategy in which 44 patients started treatment with a single dose of targeted radiation. While typical radiation therapy protocols call for 1.8–2.67Gy per day for 16 to 33 days, the researchers divided the study participants into three groups and gave each patient a single dose of 30, 34, or 38Gy. The volunteers then went on hormone-blocking drugs and waited a median of 9.8 months until they underwent surgery to remove any residual tumour tissue.

In 72% of study participants, the surgeons found no residual tumor left, indicating that patients had a “pathological complete response.” An additional 21% of patients had a “near complete response,” meaning that their cancer was more than 90% eliminated.

Further analysis showed that time to surgery was the best predictor of response. The longer patients waited to undergo surgery, the more likely their tumours were to disappear, regardless of the radiation dose or tumour size. These results were probably due to the time it takes cells to die or be removed by the immune system after radiation therapy, Dr Rahimi explained.

This new treatment protocol could hold significant advantages over the current gold standard, said Marilyn Leitch, MD, Professor of Surgery. For example, being able to wait to schedule surgery will allow patients to plan for the disruption it brings to their lives. The radiation course lasts a single day rather than weeks. Plus, in the future, this new approach may eliminate the need for surgery in some patients.

“Much of the current research in breast cancer is looking at ways to reduce the extent of surgery, radiation, and/or medical therapy that is required to completely treat early-stage breast cancer. It is very exciting to be part of innovative research that can improve the quality of life of our cancer patients and minimize the extent of treatment they require,” Dr Leitch said.

The research team is currently enrolling patients in a phase two clinical trial. “If the results mirror the ones from this study, an initial targeted dose of radiation could become a new treatment option for patients with small, early-stage, hormone-positive breast cancer,” Dr Leitch said.

Source: UT Southwestern Medical Center