Tag: cancer treatment

Categories : CancerTags :

Long-term Results Suggest that Follicular Lymphoma Is Curable

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Long-term data suggests an overall cure rate of 42%

Photo by National Cancer Institute on Unsplash

Unlike some other forms of lymphoma, advanced stage follicular lymphoma is considered incurable. But a new analysis of long-term data on patients treated for the disease years ago with standard regimens of immunotherapy and a chemotherapy combination known as CHOP suggests that many of those patients can now be considered cured.

The analysis is just published in the journal JAMA Oncology. 

“A subset of advanced-stage follicular lymphoma patients can achieve cure with CHOP-based chemoimmunotherapy, as relapse rates decline over time,” said Wilmot Cancer Institute Director Jonathan W. Friedberg, MD, MMSc, at the University of Rochester Medical Center, who is senior and corresponding author on the paper.  

“This finding represents a paradigm shift in our understanding and approach to follicular lymphoma, with broad implications for initial patient discussions and future research strategies.” 

Historically, follicular lymphoma has been considered an incurable disease, with most patients experiencing relapses even years after initial treatment.

The JAMA Oncology paper reports on an analysis of follow-up data from patients with advanced follicular lymphoma who had been treated with a standard first-line chemoimmunotherapy regimen on a large clinical trial.

Roughly 70 percent of the patients remained alive 15 years after beginning treatment, and a statistical method known as cure modelling estimated that 42% of treated patients had been cured. 

Cure modelling incorporates background mortality rates in an analysis of patient survival data to estimate what fraction of a group of patients can be considered cured of a disease. Such modelling accounts for the fact that over time some patient deaths will occur that are unrelated to the given disease.

The researchers applied a cure model to 15-year follow-up data from the S0016 clinical trial conducted by the SWOG Cancer Research Network, a clinical trials group funded by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), with the participation of other groups within the NCI-funded National Clinical Trials Network (NCTN).

This phase 3 trial, which opened in 2001, enrolled patients with untreated advanced-stage CD20-positive follicular lymphoma and randomised 531 of them to one of two treatments, both of which were built around a core chemotherapy regimen known as CHOP (cyclophosphamide, hydroxydaunorubicin, vincristine, and prednisone). One arm treated patients with rituximab plus the CHOP combination (R-CHOP), while the other arm used CHOP followed by radioimmunotherapy (CHOP-RIT). Primary results of the S0016 trial were published in 2013 (Press, OW. J Clin Oncol. 2013).

The S0016 modeling, including cure analysis, was carried out by Michael LeBlanc, PhD, a biostatistician at Fred Hutch Cancer Center and director of SWOG’s Statistics and Data Management Center (SDMC), and Hongli Li, MS, also at Fred Hutch and the SWOG SDMC.  

It showed that, with a median follow-up time of 15.5 years after a patient had begun treatment, the rate of disease relapse dropped substantially over time, falling from 6.8% of patients relapsing in the first 5 years to only 0.6% relapsing between years 15 and 20.

Fifteen years after starting treatment, about 70%of patients remained alive. The analysis also showed no statistically significant difference between the two treatment arms in the rates of 15-year overall survival.

Based on their work, the authors conclude that a substantial subset of patients with advanced-stage follicular lymphoma can, when treated with a standard regimen that includes immunotherapy and chemotherapy, achieve a functional cure – defined as having no chance of lymphoma recurring during the patient’s expected lifespan.

“These results reinforce that front-line chemoimmunotherapy remains an important option – particularly for appropriate patients – because it can deliver long-term disease control after a time-limited course of treatment, ” said first author Mazyar Shadman, MD, MPH, of Fred Hutch Cancer Center. Shadman is medical director of cellular immunotherapy at Fred Hutch, where he holds the Innovators Network Endowed Chair.

“As we bring novel agents into the first-line setting, the durability seen here sets a high benchmark; new strategies should aim not only to improve short-term response rates but to match or exceed long-term remission and cure potential.”

The idea that many of these patients can be cured could change how newly diagnosed patients are counseled and could eliminate the need for indefinite oncology and radiologic follow-up visits after treatment, with patients eventually transitioning from oncology care back to a primary care team.

Source: SWOG Cancer Research Network

Categories : Diseases, Syndromes and ConditionsTags :

Ivermectin Was Touted as a Cure for COVID, Now it’s Being Tested for Cancer. But what can it Actually Treat?

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Photo by Halgatewood.com on Unsplash

Nial Wheate, Macquarie University

Ivermectin was originally celebrated as a revolutionary treatment for parasitic disease in humans and animals. It has since evolved into a focal point of misinformation and heated debate.

During the early part of the COVID pandemic, it was touted on social media as a miracle cure for the virus, despite a lack of robust evidence.

Now the United States National Cancer Institute is looking into the drug as a potential cancer treatment, with early human clinical studies underway.

But what can it successfully treat?

What is ivermectin?

The drug is a small organic chemical that can be extracted from the bacterium Streptomyces avermitilis. This bacterium grows in the soil, and was first found near the grounds of a Japanese golf course.

Ivermectin’s discovery in the 1970s was considered so important its discoverers were awarded the 2015 Nobel Prize in Physiology or Medicine.

It was first approved for use in animals in 1981 and in humans in 1987. It’s now available in various brands as tablets and creams you apply to the skin.

Assessing the evidence

Governments use human clinical trials to decide whether to approve a medicine for sale.

But clinical trials aren’t the highest level of evidence to inform best practice and guide decisions. For that, there are Cochrane reviews.

A Cochrane review brings together a panel of experts who collate and assess all the relevant evidence on a medication. It takes data from multiple clinical trials, and other studies, and evaluates it following clear and structured steps. It’s able to examine and critique study designs to identify bias and reject bad data.

Cochrane reviews are also regularly updated to take into account new information. The result is a summary that is considered the highest level of evidence to guide decision-making.

So what do Cochrane reviews say about ivermectin for different conditions?

What can and can’t ivermectin treat?

ConditionDoes it work?Notes
CancerUnclearStudies only just starting
COVIDNoDoes not prevent infection or treat
Gut and lymphatic wormsYesTreatment for various roundworms
MalariaUnclearNot enough evidence to decide
River blindnessUnclearNot enough evidence to decide
RosaceaYesUse the topical formulation
ScabiesYes, but with caveatsNot the most effective
Table: Nial Wheate Source: Cochrane reviews – variousGet the dataCreated with Datawrapper

Gut and lymphatic worms

Ivermectin is used to treat a variety of parasitic worm infections. These include the round worms Ascaris lumbricoides, Strongyloides stercoralis, Wuchereria bancrofti, and Brugia malayi.

The latter two worms cause the disease lymphatic filariasis (or elephantiasis) which causes severe swelling in the arms, legs, breasts and genitals.

When ivermectin is used to treat Strongyloides stercoralis, the Cochrane panel found it is better than albendazole and had fewer side effects than thiabendazole.

For Ascaris lumbricoides, the panel concluded ivermectin was as good as albendazole and mebendazole.

For treating lymphatic filariasis, a Cochrane review found ivermectin or diethylcarbamazine should be standard treatment in combination with albendazole.

Rosacea

The Cochrane review for rosacea evaluated 22 different treatments for this skin condition, including a variety of drugs, as well as light therapy, cosmetics and reducing the intake of spicy food.

It concluded that ivermectin applied to the skin was more effective than a placebo, and a bit better than the other standard medication, metronidazole.

Scabies

Cochrane has two reviews on the use of ivermectin for scabies. One specifically evaluated ivermectin and permethrin as treatments. The other evaluated all available treatments for scabies.

The first review concluded both permethrin and ivermectin were just as effective, regardless of whether the ivermectin was administered orally or directly onto the skin.

In contrast, the second review concluded ivermectin does work but topical permethrin appeared to be the most effective treatment.

Malaria

The Cochrane panel looked specifically at whether ivermectin could reduce transmission of the malaria parasite, rather than as a treatment.

Unfortunately there was just a single clinical trial to use as evidence. In that trial, residents of eight villages were given ivermectin and albendazole together, with follow up doses of just ivermectin. The researchers then looked at the rates of child infection over 18 weeks.

Even though the trial didn’t show ivermectin prevented infection, due to the high risk of bias in it, the Cochrane panel couldn’t conclude either way whether ivermectin worked or not.

River blindness

River blindness is caused by another parasitic worm called Onchocerca volvulus.

The Cochrane review concluded there was a lack of evidence either way to know whether it works to prevent infection-based visual impairment and blindness.

It evaluated the data from four clinical trials and two large community-based studies.

One of the reasons the panel was unable to make a firm conclusion was because it thought the drug may work differently against different strains of the parasite and in people of different ethnicity.

Cancer

There are no Cochrane reviews on ivermectin’s use for cancer because clinical interest in the drug for this condition is just starting.

There is a current clinical trial that is evaluating ivermectin in combination with antibody-based drugs for breast cancer.

Early results showed the combination of antibody drugs with ivermectin was safe to patients, but no efficacy data has been published.

COVID

The Cochrane panel rejected the data for seven clinical trials and included 11 other trials. Rejected trials included those which compared ivermectin against other drugs which were known to not be effective against COVID, such as hydroxychloroquine.

The review concluded there was no evidence to support the use of ivermectin for the treatment or prevention of COVID. In making that conclusion, it evaluated treatments that used invermectin or placebo in combination with standard care and whether treatment reduced death, illness, or the length of the infection.

Nial Wheate, Professor, School of Natural Sciences, Macquarie University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

Categories : Cancer GastrointestinalTags :

Faecal Transplant Pills Show Promise in Clinical Trials for Multiple Types of Cancer

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Two Canadian clinical trials show poop pills could help patients respond to immunotherapy while also reducing toxic side effects of cancer drugs

Faecal microbiota transplants (FMT), can dramatically improve cancer treatment, suggest two groundbreaking studies published in the prestigious Nature Medicine journal. The first study shows that the toxic side effects of drugs to treat kidney cancer could be eliminated with FMT. The second study suggests FMT is effective in improving the response to immunotherapy in patients with lung cancer and melanoma.

The findings represent a giant step forward in using FMT capsules – developed at Lawson Research Institute (Lawson) of St. Joseph’s Health Care London and used in clinical trials at London Health Sciences Centre Research Institute (LHSCRI) and Centre de recherche du Centre hospitalier de l’Université de Montréal (CRCHUM) – for safe and effective cancer treatment.

A Phase I clinical trial was conducted by scientists at LHSCRI and Lawson to determine if FMT is safe when combined with an immunotherapy drug to treat kidney cancer. The team found that customised FMT may help reduce toxic side effects from immunotherapy. The clinical trial involved 20 patients at the Verspeeten Family Cancer Centre at London Health Sciences Centre (LHSC).

“Standard treatment for advanced kidney cancer often includes an immunotherapy drug that helps the patient’s immune system tackle cancer cells,” says Saman Maleki, PhD, Scientist at LHSCRI. “But, unfortunately, the treatment frequently leads to colitis and diarrhoea, sometimes so severe that a patient must stop life-sustaining treatment early. If we can reduce toxic side effects and help patients complete their treatment, that will be a gamechanger.”

Separate Phase II lung and skin cancer studies were led by researchers at CRCHUM in collaboration with Lawson and LHSCRI. The studies found that 80 per cent of patients with lung cancer responded to immunotherapy after FMT, compared to only 39-45 per cent typically benefiting from immunotherapy alone. Similarly, 75 per cent of patients with melanoma who received FMT experienced a positive response to treatment, compared to only 50-58 per cent response in patients who receive immunotherapy alone. Twenty patients participated in the lung cancer clinical trial and 20 patients participated in the skin cancer clinical trial.

“Our clinical trial demonstrated that faecal microbiota transplantation could improve the efficacy of immunotherapy in patients with lung cancer and melanoma,” says Dr Arielle Elkrief, co-principal investigator and Physician Scientist, Université de Montréal-affiliated hospital research centre (CRCHUM). “The results also uncovered one possible mechanism of action of faecal transplantation – through the elimination of harmful bacteria following the transplant. Our results open up a novel avenue for personalised microbiome therapies, and faecal transplant is now being tested as part of the large pan-Canadian Canbiome2 randomised controlled trial.”

“Faecal microbiota transplantation in melanoma and lung cancer opens an entirely new therapeutic avenue, made possible by the exceptional commitment of our patients and the teamwork,” adds Dr. Rahima Jamal, Director of the Unit for Innovative Therapies (UIT) at CRCHUM. “At the Unit for Innovative Therapies (UIT) of the CRCHUM, we have had the privilege of translating laboratory discoveries into early phase clinical trials and witnessing their concrete impact on people living with cancer.”

Both studies use advanced, world-leading FMT capsules, also known as LND101, produced by Lawson in London, Ont. The research reinforces London’s place as a global leader in FMT innovation and treatment. The capsules are processed from healthy donor stools and ingested to help restore a patient’s healthy gut microbiome and treat different types of cancer.

“To use FMT to reduce drug toxicity and improve patients’ quality of life while possibly enhancing their clinical response to cancer treatment is tremendous, and it had never been done in treating kidney cancer before this,” says Dr Michael Silverman, Scientist at Lawson and Head of St. Joseph’s Infectious Diseases Program. “And none of this would be possible if not for this close collaboration: innovating the FMT capsules in Lawson labs and introducing them at LHSCRI and CHUM to advance vital research initiatives. Also, because LND101 comes from healthy donors, production can be scaled up to eventually help large numbers of cancer patients.”

The studies build on earlier London and CHUM-generated Phase I research showing FMT can safely augment treatment for people with melanoma. FMT is also being studied in people with pancreatic cancer and triple-negative breast cancer, and is already a well-established treatment for serious gut infections such as C. difficile, which can cause severe diarrhoea.

“Our hope is that our research will one day help people with cancer live longer while reducing the harmful side effects of treatment,” adds Dr Ricardo Fernandes, Scientist at LHSCRI and Medical Oncologist at LHSC. “We are world leaders in FMT research and we’re excited about its potential.”

Source: London Health Sciences Centre Research Institute

Categories : Cancer Diseases, Syndromes and ConditionsTags :

Repurposed Cancer Drug may Aid Recovery from Severe Malaria

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Red Blood Cell Infected with Malaria Parasites
Colourised scanning electron micrograph of red blood cell infected with malaria parasites (teal). The small bumps on the infected cell show how the parasite remodels its host cell by forming protrusions called ‘knobs’ on the surface, enabling it to avoid destruction and cause inflammation. Uninfected cells (red) have smoother surfaces. Credit: NIAID

A new clinical trial led by QIMR Berghofer, in collaboration with University of Sunshine Coast Clinical Trials Network has found a medication currently used for some blood disorders could help the body fight malaria more effectively.

The findings mean the drug, ruxolitinib, could potentially be used alongside standard treatment to boost recovery and strengthen people’s immune systems against future infections.

Malaria kills more than 600 000 people each year and three quarters of those deaths are in children under the age of five.

Current treatments for malaria work by killing the parasite that causes most malaria deaths, Plasmodium falciparum. However, even with these treatments, fatality rates from severe malaria remain high.

Furthermore, while patients develop some immunity after infection, this protection is often incomplete, leaving many vulnerable to reinfection.

Head of QIMR Berghofer’s Clinical Malaria Group Associate Professor Bridget Barber says the research overcomes a key hurdle.

“While antimalarial treatments are effective at killing the parasite, they don’t directly address the inflammation that contributes to severe illness and death. These findings suggest that we may be able to improve clinical outcomes by targeting the host inflammatory response as well as the parasite itself,” she said.

The research, published in Science Translational Medicine, looked at how the immune system responds to malaria via the body’s ‘early warning system’ known as type 1 interferon signalling.

To do this, researchers enrolled 20 healthy adult volunteers who had never been exposed to malaria. Participants were deliberately infected with Plasmodium falciparum under closely monitored conditions. Eight days later, all participants received standard malaria treatment (artemether-lumefantrine), while 11 were also given ruxolitinib. Three months later, participants were re-infected with malaria to test how their immune systems responded to a second infection.

The research revealed ruxolitinib was safe and well-tolerated, compared with the placebo group, and participants who received ruxolitinib showed a lower inflammatory response, and favourable changes in markers linked to disease severity.

QIMR Berghofer’s Program Director of Infection and Inflammation Professor Christian Engwerda says the results are encouraging.

“One of the biggest challenges in efforts to eliminate malaria is the limited efficacy and duration of protection provided by current vaccines. By boosting the immune system without causing detrimental inflammation with drugs like ruxolitinib, we may be able to overcome these challenges,” he said.

The researchers say it’s important to note that the study was conducted in healthy volunteers who did not live in malaria-endemic regions. Further studies in malaria-endemic regions will be needed to determine whether these findings translate into improved outcomes for patients most affected by the disease.

Read the scientific paper here: www.science.org/doi/10.1126/scitranslmed.aea2531

Source: QIMR Berghofer Medical Research Institute

Categories : Cancer NeurologyTags :

Electrotherapy may be a Promising New Glioblastoma Treatment

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Photo by Anna Shvets

Electrotherapy using injectable nanoparticles delivered directly into the tumour could pave the way for new treatment options for glioblastoma, according to a new study from Lund University in Sweden.

Glioblastoma is the most common and most aggressive form of brain tumour among adults. Even with intensive treatment, the average survival period is 15 months. The tumour has a high genetic variation with multiple mutations, which often makes it resistant to radiation therapy, chemotherapy and many targeted drugs. The prognosis for glioblastoma has not improved over the past few decades despite extensive research.

Electrotherapy – a new treatment method

Electrotherapy offers another strategy to combat solid tumours. Using short, strong electric pulses (irreversible electroporation), non-reversible pores are created in the cancer cells leading to their death. The body’s immune system is simultaneously stimulated. The problem is that surgery is required to place the stiff metal electrodes that are necessary for the treatment. In sensitive tissue, in the brain for example, this often entails a very difficult procedure, which has led to strict criteria regarding which patients can be treated. Johan Bengzon is a researcher in glioblastoma and adjunct professor at Lund University, and consultant in neurosurgery at the Skåne University Hospital. He regularly treats patients with glioblastoma and is frustrated by the limited treatment options.

“The short distance between the hospital and the University in Lund facilitates cooperation and that’s why I contacted research colleagues to find out if injectable electrodes could be an alternative solution in electrotherapy,” says Johan Bengzon.

Said and done. The research team, with Amit Singh Yadav, Martin Hjort, and Roger Olsson at the helm, had previously used nanoparticles to form injectable and electrically conductive hydrogels to control brain signalling and heart contractions. It is aminimally invasive method in which the particles are injected using a thin syringe directly into the body. The particles break down after the treatment and thus do not need to be surgically removed. Perhaps the same technology could be used to destroy tumour cells in glioblastoma. 

“After surgical treatment, unfortunately the glioblastoma tumour often returns on the outer edge of the area operated on. By drop casting the nanoparticles into the tumour cavity after an operation, we could electrify the edges while the immune system is also activated. In animal models the procedure, due to this irreversible electroporation, led to tumours being wiped out within three days,” says Roger Olsson, professor of chemical biology and drug development at Lund University, who led the study. 

Promising results – but a long way to the patient

The prospects are good and the researchers are very hopeful for the future, even though there is a long way to go before it becomes a clinical reality. The challenge is now to test the method on larger tumours. 

“We have seen that the electrode is well received in the brain. We have not noted any problems relating to side effects and after 12 weeks the electrode disappeared by itself as it’s biodegradable. The technology combines direct tumour destruction with activation of the immune system and can be an important step towards more effective treatment of glioblastoma,” concludes Amit Singh Yadav, researcher at Lund University and first author of the study. 

Source: Lund University

Categories : CancerTags :

Tec-Dara Combo More Effective for Relapsed or Refractory Multiple Myeloma

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Depiction of multiple myeloma. Credit: Scientific Animations

Patients with relapsed or refractory (R/R) multiple myeloma who received a combination of teclistamab, a bispecific monoclonal antibody, and daratumumab, a CD38-directed monoclonal antibody, were 83% more likely to be alive without disease progression compared with those who received standard second-line therapies at a median of nearly 35 months of follow-up, according to the results of a new trial presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition.

The trial is the first to test a bispecific monoclonal antibody as early as the first relapse after initial treatment for multiple myeloma. Based on the findings, researchers suggest the combination of teclistamab and daratumumab, known as Tec-Dara, could represent a new standard of care for R/R multiple myeloma.

“We were surprised by the efficacy data because we didn’t expect such a magnitude of benefit,” said lead study author María-Victoria Mateos, MD, PhD, physician in the haematology department and professor of medicine at the University of Salamanca in Spain. “These are the best data we’ve seen in patients with R/R multiple myeloma after one line of therapy. Patients will live longer overall and with no worsening of quality of life.”

Multiple myeloma is a cancer that causes excessive production of plasma cells, crowding out the production of other types of blood cells and harming the body’s ability to fight infections. It is most common in older adults. Patients who relapse or experience an incomplete response to initial treatment often have their cancer return after subsequent therapies, pointing to a need for improved second-line treatments.

Teclistamab is approved by the U.S. Food and Drug Administration for R/R multiple myeloma after at least four prior lines of therapy. Daratumumab is a therapy targeting the CD38 protein that has been approved for use in combination with other therapies for newly diagnosed and R/R multiple myeloma. Laboratory studies have suggested that teclistamab and daratumumab may work synergistically to eradicate cancer to a greater extent than either agent individually.

To test this hypothesis, researchers randomized 587 patients with R/R multiple myeloma to receive either Tec-Dara or standard second-line therapies. For patients in the control arm, treating physicians chose between two standard three-agent combination therapies which included daratumumab with dexamethasone plus either pomalidomide or bortezomib (known as DPd or DVd, respectively).

Study participants had a median age of 64 with a range of 25-88, and all patients were R/R after one to three prior lines of therapy. Participants remained on their assigned treatment regimen unless they experienced intolerable adverse events, and were followed for a median of nearly 35 months.

The 36-month rate of progression-free survival, the study’s primary endpoint, was achieved in 83.4% of participants who received Tec-Dara and 29.7% of those receiving DPd/DVd, a substantial improvement in favor of Tec-Dara. This benefit was consistent across subgroups of patients by age, prior treatment, tumor genetics, and other factors.

In addition to being a highly efficacious treatment for R/R multiple myeloma as early as the first relapse, researchers noted that the Tec-Dara combination could be more accessible than other second-line therapies for multiple myeloma as it could be delivered in community settings, not just academic centers.

Tec-Dara outperformed DPd/DVd in terms of the trial’s secondary efficacy endpoints as well as quality of life outcomes and had a safety profile comparable to the control arm. Patients receiving Tec-Dara were significantly more likely to achieve a complete response or better, which occurred in 81.8% of patients receiving Tec-Dara and 32.1% among the control arm. They were also more likely to test negative for minimal residual disease (MRD), a sensitive test for remaining cancer cells, with 58.4% of those in the Tec-Dara arm achieving MRD-negativity compared with 17.1% in the control arm. Overall survival was also higher in the Tec-Dara arm, with 83.3% of patients in this group being alive at 36 months compared with 65.0% in the control arm.

The results showed comparable rates of treatment-emergent adverse events, with 95.1% of patients in the Tec-Dara arm and 96.6% of those in the control arm experiencing grade 3-4 adverse events. Rates of serious adverse events and discontinuations due to adverse events were also comparable between groups, researchers reported.

The rate of infections was higher among those receiving Tec-Dara, with 96.5% of patients in this group experiencing infections compared with 84.1% in the control group. The onset of higher-grade infections decreased over time, and researchers noted that strategies for managing infections also improved over the course of the study. Low-grade cytokine release syndrome (CRS) was also common, with grade 1-2 CRS occurring in 60.1% of those receiving Tec-Dara.

One limitation of the study is that patients refractory to daratumumab were not included in the trial. However, some patients (5%) had received daratumumab as part of their first-line therapy and benefited equally from the Tec-Dara combination.

Dr. Mateos noted that future studies could help clarify how doctors might select which patients would benefit most from the Tec-Dara combination in comparison to other therapies. Trials involving other bispecific antibody combinations are also underway and could shed additional light on the optimal use of such combinations as early as the first relapse.

Source: American Society of Hematology

Categories : CancerTags :

‘Wondrous’ Drug to Treat Aggressive Leukaemia Gets UK Approval

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Killer T cells surround a cancer cell. Credit: Alex Ritter, Jennifer Lippincott Schwartz and Gillian Griffiths, National Institutes of Health (CC BY 2.0).

Adult patients with an aggressive form of leukaemia will be able to receive a breakthrough immunotherapy, which was invented by University College London researchers, on the NHS within weeks following approval for use by the UK’s National Institute for Health and Care Excellence (NICE).

The CAR T-cell therapy – known as ‘obe-cel’ and marketed as Aucatzyl – involves taking a patient’s immune cells and reprogramming them in a lab to identify and target their cancer, before returning them to the body as ‘living medicine’.

Obe-cel is a second-generation CAR T cell therapy invented by scientists from the UCL Cancer Institute, led by Dr Martin Pule, and has delivered promising results in treating patients with acute lymphoblastic leukaemia (ALL), an aggressive blood cancer.

The therapy has reduced immune toxicity and persists for longer in blood cancer patients, overcoming two common limitations of earlier CAR T cell therapies. Aucatzyl was taken through clinical trials and is manufactured by UCL spinout business Autolus, which was set up with the help of UCL Business, the commercialisation company of UCL.

The development of CAR T cell therapy has had long-standing support from the National Institute for Health and Care Research (NIHR) UCLH Biomedical Research Centre (BRC).

NHS England today announced that the personalised therapy would be available on the NHS within weeks through specialist centres.

Dr Claire Roddie, one of the team who developed the treatment from UCL Cancer Institute and UCLH consultant haematologist, said: “I am delighted to hear of NICE’s decision. Many more patients now stand to benefit from CAR-T cell therapy on the NHS. 

“We have been working on proving the safety and efficacy of this drug since 2017 and it has brought together clinical and research teams from UCL and UCLH, with support from government and arm’s-length bodies like the NIHR and the BRC as well as the pharmaceutical industry.

“The many, many people involved in this work can feel immensely proud of this achievement which will help save the lives of many more patients.”

Eligible patients will receive two doses of CAR-T therapy intravenously, ten days apart, with the treatment being delivered at specialist CAR-T centres across the country.

The treatment will be available to people aged 26 and over with B-cell acute lymphoblastic leukaemia which has returned or not responded to previous treatment. 

It is estimated that it could be administered to around 50 patients each year in England.

In a clinical trial, 77% of patients saw their cancer enter remission after treatment with obe-cel, with half of those showing no signs of detectable cancer after three and a half years. 

The treatment – which has been researched, developed and manufactured in the UK – was also found to have lower toxicity and was less likely to cause serious side effects than other CAR (chimeric antigen receptor) T-cell therapies.

Dr Anne Lane, UCL Business CEO, said: “This cutting-edge personalised immunotherapy has been on a 10-year journey starting with research by clinical academics in UCL’s Cancer Institute who, with the support of UCL Business, established Autolus, a spinout company dedicated to developing, trialling and bringing AUCATZYL® to market. That journey has required vision, tenacity and over £800m. Today that has hugely paid off and will benefit people across the UK. It’s an inspiring demonstration of what can be achieved when university academics, NHS hospitals and investors work together.”

Professor Peter Johnson, NHS National Clinical Director for Cancer, said: “This cutting-edge therapy has shown real promise in trials and could give patients with this aggressive form of leukaemia a chance to live free from cancer for longer – and, for some, it could offer the hope of a cure.

“This ‘living medicine’ boosts a patient’s own immune system and then guides T-cells towards the cancer to kill it – it is fantastic to have another pioneering option available on the NHS, adding to our range of CAR-T therapies which are helping people with blood cancers live longer, healthier lives.”

Harry, a 19-year-old student from Harrogate, was treated with obe-cel for B-cell ALL as part of a clinical trial in 2024. He said: “I feel so lucky to have had access to such a wondrous treatment. Not only did it work better than my doctors thought it would, it worked without many of the horrible side effects you can get from other treatments.

“I think it’s brilliant obe-cel is now available on the NHS for people over the age of 26. The biggest thing it offers is hope. When you’re facing a situation like mine, hope is the most valuable thing you can have.”

Health Minister Ashley Dalton said: “This pioneering treatment is excellent news for patients and their families, demonstrating how the NHS is at the forefront of medical innovation.

“Our 10 Year Health Plan is about harnessing our world-leading life sciences sector to deliver treatments like this – innovative therapies that save lives.

“By supporting new treatments with fewer side effects and shorter hospital stays, we’re building an NHS fit for the future whilst cementing the UK’s position as a global leader in medical research.”

Fiona Bride, interim Chief Commercial Officer and Director of Medicines Value & Access at NHS England, said: “This is a success story that’s made in Britain, and shows how collaboratively we can bring to life the ambition of the 10 Year Health Plan, showcasing how the UK’s competitive edge in life sciences can translate to better outcomes and treatments for NHS patients.

“The journey of obe-cel from scientific research in a UK university to a safe, clinically and cost-effective treatment set to be delivered through the NHS specialist CAR-T network is a remarkable one and I am grateful to colleagues who have played their part along the way.”  

Acute lymphoblastic leukaemia is an aggressive cancer in the blood and bone marrow, with around 800 people being diagnosed in the UK every year, around half of which are in adults.

Data shows patients with aggressive forms of the cancer receiving chemotherapy, the current routine standard of care, live for just 10 months on average after treatment.

The therapy will be fast tracked to patients more quickly than the standard 90-day implementation period thanks to interim funding from the NHS’s Cancer Drugs Fund.

Source: University College London

Categories : Cancer Cardiovascular DiseaseTags :

Old Blood Pressure Drug, New Tumour-fighting Tricks

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A Penn-led team has revealed a how hydralazine, one of the world’s oldest blood pressure drugs and a mainstay treatment for preeclampsia, works at the molecular level. In doing so, they made a surprising discovery – it can also halt the growth of aggressive brain tumours.

Reseachers from the Megan Matthews lab at Penn treated human glioblastoma brain tumour cells with hydralazine, one of the oldest-known blood pressure drugs and a first-line treatment for preeclampsia, for three days. At day three (imaged), more cells become enlarged and flattened – a hallmark of senescence. (Image: Courtesy of Kyosuke Shishikura)

Over the last 70 years, hydralazine has been an indispensable tool against life-threatening high blood pressure, especially during pregnancy. But despite its essential role, a fundamental mystery has persisted: No one knows its mechanism of action, which allows for improved efficacy, safety, and what it can treat.

“Hydralazine is one of the earliest vasodilators ever developed, and it’s still a first-line treatment for preeclampsia – a hypertensive disorder that accounts for 5-15% of maternal deaths worldwide,” says Kyosuke Shishikura, a physician-scientist at the University of Pennsylvania. “It came from a ‘pre-target’ era of drug discovery, when researchers relied on what they saw in patients first and only later tried to explain the biology behind it.”

Now Shishikura, his postdoctoral adviser at Penn Megan Matthews, and collaborators have solved this long-standing puzzle.

In a paper published in Science Advances, the team uncovered the method of action of hydralazine, and in doing so, revealed an unexpected biological link between hypertensive disorders and brain cancer. The findings highlight how long-established treatments can reveal new therapeutic potential and could help in the design of safer, more effective drugs for both maternal health and brain cancer.

“Preeclampsia has affected generations of women in my own family and continues to disproportionately impact Black mothers in the United States,” Matthews says. “Understanding how hydralazine works at the molecular level offers a path toward safer, more selective treatments for pregnancy-related hypertension—potentially improving outcomes for patients who are at greatest risk.”

Hydralazine blocks an oxygen-sensing enzyme

The team found that hydralazine blocks an oxygen-sensing enzyme called 2-aminoethanethiol dioxygenase (ADO) – a molecular switch for blood vessels contraction.

“ADO is like an alarm bell that rings the moment oxygen starts to fall,” Matthews says. “Most systems in the body take time; they have to copy DNA, make RNA, and build new proteins. ADO skips all that. It flips a biochemical switch in seconds.”

Hydralazine acts by binding to and blocking ADO – effectively “muting” that oxygen alarm. Once the enzyme was silenced, the signaling proteins it normally degrades – called regulators of G-protein signaling (RGS) – remained stable.

The buildup of RGS proteins, says Shishikura, tells the blood vessels to stop constricting, effectively overriding the “squeeze” signal. This reduces intracellular calcium levels, which he calls the “master regulator of vascular tension.” As calcium levels fall, the smooth muscles in blood vessel walls relax, causing vasodilation and a drop in blood pressure.

From preeclampsia to brain cancer: A common target

Prior to this study, cancer researchers and clinicians had begun to suspect that ADO was important in glioblastoma, where tumours often have to survive in pockets of very low oxygen, Shishikura explains. Elevated levels of ADO and its metabolic products had been linked with more aggressive disease, suggesting that shutting this enzyme down could be a powerful strategy, but no one had a good inhibitor to test that idea.

To see if hydralazine was a contender, Shishikura worked closely with structural biochemists at the University of Texas, who used X-ray crystallography to visualise hydralazine bound to ADO’s metal centre, and with neuroscientists at the University of Florida, who tested the drug’s effects in brain cancer cells.

They found that the ADO pathway that regulates vascular contraction also helps tumour cells survive in low-oxygen environments. Unlike chemotherapy, which aims to kill all cells outright, hydralazine disrupted that oxygen-sensing loop, triggering cellular senescence.

Unlocking the potential for other lifesaving treatments

Their findings highlight how long-established treatments can reveal new therapeutic potential and could help in the design of safer, more effective drugs for both maternal health and brain cancer.

They say the next step is to push the chemistry further building new ADO inhibitors that are more tissue specific and better at crossing, or exploiting weak points in, the blood-brain barrier so they hit tumour tissue hard while sparing the rest of the body.

Matthews is also working to continue engineering the next generation of medical solutions by revealing the mechanics of clinically tested, long-known treatments.

“It’s rare that an old cardiovascular drug ends up teaching us something new about the brain,” Matthews says, “but that’s exactly what we’re hoping to find more of – unusual links that could spell new solutions.”

Source: University of Pennsylvania

Categories : CancerTags :

Beyond the Diagnosis: The Financial Toll of Cancer in SA

On By ModernMedia

Cancer is one of the fastest-growing health challenges in South Africa, with over 100 000 new cases diagnosed annually, according to the National Cancer Registry. While most conversations focus on the physical and emotional impact, the financial strain of the disease often goes unspoken.

According to the South African Medical Journal, treatment costs for cancer can exceed R1 million per year, particularly when advanced therapies and prolonged care are required. This leaves many families facing difficult decisions that extend far beyond the hospital ward. With medical aid often falling short and with only 16% of South Africans covered by medical schemes, as reported by the Council for Medical Schemes the financial burden of cancer can be as devastating as the diagnosis itself.

“Medical aid alone often isn’t enough to cover the full cost of treatment, especially when it comes to critical illnesses like cancer,” says Matthew Green, Product Portfolio Manager at FNB Life. “We’ve seen firsthand how having the right insurance can make a real difference.”

The true impact of cancer is often measured in rands and cents: savings depleted, debt accumulated, and households forced to sacrifice essentials to pay for treatment. Myths about affordability and a lack of awareness mean that too many people enter this battle unprepared. The result is a financial shock that can be as devastating as the diagnosis itself. Beyond the direct medical expenses, families often face a range of additional costs from transport to and from treatment centres, specialised nutrition, home modifications, and caregiving support, to lost income due to time off work. Critical illness cover is designed to help bridge these financial gaps, providing a lump-sum payout that can be used not only for treatment, but also for these broader, often overlooked expenses that impact the entire household.

“Against this backdrop, insurers are under growing pressure to offer support that reflects the lived realities of ordinary South Africans. FNB Insure is among those stepping in to help close the gap – focusing on making financial protection more accessible, flexible, and relevant to everyday needs,” says Green.

Rather than positioning insurance cover as a luxury, the emphasis is on practical tools that help households navigate the rising costs of treatment and the economic strain that often follows a serious diagnosis. Whether it’s support during hospital stays, assistance with unexpected medical shortfalls, or a payout that enables immediate action after a diagnosis, the goal is to empower customers to focus on recovery – not financial survival.

This is evident from our customer feedback, where individuals have shared how timely access to cover helped them act quickly, avoid financial delays, and prioritise their health during some of life’s most difficult moments. “And its stories like this underline the importance of early financial planning and the role of accessible insurance in giving families space to focus on recovery rather than financial survival,” says Green.

With October marking Breast Cancer Awareness Month and November bringing the spotlight on men’s health through initiatives like Movember, FNB Insure is adding its voice to the broader call for awareness. “The message is clear: cancer doesn’t only affect health; it reshapes every aspect of life. Building resilience means preparing not just medically, but financially too,” concludes Green.

Categories : Ageing CancerTags :

Global Study Challenges Age-Based Treatment Decisions in Leukaemia

On By ModernMedia

Study of 2,800 patients suggests moving beyond chronological cut-offs in cancer care

SAG Leukaemia. Credit: Scientific Animations CC0

An international study conducted by the Alliance for Clinical Trials in Oncology and the Acute Myeloid Leukemia Cooperative Group reveals that age-based classifications in the treatment of acute myeloid leukaemia (AML) may be outdated and overly simplistic.

AML is a fast-growing cancer of the blood and bone marrow that disproportionately affects older adults. Historically, age has been a key factor in determining treatment intensity, eligibility for clinical trials, and access to targeted therapies. However, this new research suggests that age alone is not a reliable indicator of disease biology or prognosis.

“Our findings support a more flexible, biology-driven approach to AML treatment and trial design. Age alone should not be a gatekeeper to potentially life-saving therapies,” said Alliance researcher and lead author Ann-Kathrin Eisfeld, MD, associate professor of Internal Medicine and director of the Clara D. Bloomfield Center for Leukemia Outcomes Research at The Ohio State University. “Our results suggest reconsidering age-based eligibility criteria for treatments. By focusing on molecular and genetic profiles rather than chronological age, clinicians may better tailor treatments to individual patients, improving outcomes and expanding access to novel therapies.”

Published in Leukemia, the study analysed data from 2823 adult AML patients treated in the setting of large cooperative group frontline trials across the United States (CALGB/Alliance) and Germany (AMLCG), uncovering nuanced age-related trends in genetic mutations and survival outcomes that challenge current clinical practices. This research is the first large-scale, cross continental study to analyse the mutational patterns and outcomes among patients of all age groups with AML.

The analysis included patients treated with frontline cytarabine-based chemotherapy between 1986 and 2017. Molecular profiling was conducted using targeted sequencing platforms, and survival outcomes were assessed using the 2022 European LeukemiaNet (ELN) genetic-risk classification.

The study found no clear age threshold that could biologically or prognostically separate patients into distinct groups. Instead, genetic mutations and survival outcomes varied continuously across the age spectrum. This challenges the long-standing practice of using arbitrary age cut-offs, such as 60 or 65 years, to guide treatment decisions.

Survival outcomes also declined steadily with age, even among patients classified as having favourable genetic risk. For example, patients aged 18 to 24 with favourable-risk AML had a five-year overall survival rate of 73%, while those aged 75 and older had a survival rate of just 21%. This trend was consistent across all risk categories, indicating that age negatively impacts prognosis regardless of genetic classification.

“This research arrives at a critical moment in oncology, as precision medicine continues to transform cancer care,” added Dr Eisfeld. “Most targeted treatment options are still only available for patients above a certain age threshold that is dictated by corresponding inclusion criteria of pivotal clinical trials, even though patients outside of that age range might equally benefit from these often less toxic treatments.”

Source: Alliance for Clinical Trials in Oncology