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12th November 2019
Research presented at the 2019 ACR/ARP Annual Meeting shows that there is a profound ongoing need for additional medications to control the signs and symptoms of juvenile idiopathic arthritis (JIA), despite the availability of disease-modifying biologic drugs.
There are several biologics used for JIA treatment in the US including etanercept, adalimumab, abatacept, tocilizumab and canakinumab. Nevertheless, many children with JIA continue to have active arthritis despite the available medications and are treated with other medications off-label. Medications that have been proven to be safe and effective in adults with chronic inflammatory arthritis are not being universally studied in children with JIA. This study’s goal was to document the continuing medical need for additional, newly approved medications to treat children with JIA.
“The approved treatment options for JIA have expanded tremendously, but there are still significant proportions of children who do not respond to available therapies or who are receiving medications that have not been approved for JIA. We must demand that newly developed medications are studied for safety and effectiveness in children,” says Timothy Beukelman, MD, MSCE, associate professor, Division of Pediatric Rheumatology, at the University of Alabama at Birmingham, and the study’s co-author.
For the study, the researchers reviewed electronic medical record data for 1599 JIA patients treated at Cincinnati Children’s Hospital Medical Center (CCHMC) since 2008 for medication use and disease activity over time. In addition, they assessed 7379 JIA patients enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry for medication use and disease activity at their most recent registry visit.
The researchers defined ongoing medication need as active JIA despite sequential use of two or more biologics. They defined active JIA as either physician global assessment of JIA activity (on a scale of zero to 10 with zero as inactive disease) of three or higher, or three or more active joints, or a patient global assessment score (on a scale of zero to 10 with zero meaning very well) of three or higher. They only assessed medication failure for patients with complete data.
Use of biologics was common in both data sources (53% in CCHMC; 65% in CARRA registry), and ongoing medication need was assessed in 487 CCHMC patients and 1159 CARRA patients. Approximately 52% of CCHMC patients and 45% of CARRA patients had ongoing active JIA despite treatment with two or more biologics. Among all patients who received any biologic treatments, there was frequent use of medications that are not approved for JIA (37% CCHMC patients and 24% CARRA patients).
The study’s lead author Dr Hermine I. Brunner, chief of rheumatology and director Lupus Center at Cincinnati Children’s Hospital Medical Center, and scientific director of the Pediatric Rheumatology Collaborative Study Group (PRCSG), said: “There is clearly a need to increase the number and types of therapies available for the treatment of children with JIA. Only if FDA demands studies from the pharmaceutical companies as part of their drug development program, will paediatric rheumatologists have valid information about the proper dosing, efficacy and preliminary safety of new medications. Further, FDA approval greatly increases access of JIA patients to new medications.”
11th November 2019
“We used data from the National Health Interview Survey (NHIS) from 2012-2016 to track information on children who have been diagnosed with asthma,” says allergy and immunology fellow Sairaman Nagarajan, MD, lead author of the study. “Of the more than 57,000 children whose information we examined, more than 20% of those who had a family history of cancer had an asthma diagnosis.”
The children represented in the survey were 51% male and 49% female. The children with asthma were older (10 years vs 8 years) than the children without asthma.
“The NHIS survey reflects the US population, and because of the large number of those surveyed, the findings are significant for people across the country,” says allergist Rauno Joks, MD, ACAAI member and co-author of the study. “If a parent knows there is a family history of cancer, they should be sure to tell their paediatrician and allergist, as an extra effort at asthma screening could be valuable in diagnosing and treating childhood asthma.”
9th November 2019
This study focussed on the role of lactate in how the body’s immune system responds to inflammation.
Dr Valentina Pucino, of the University of Birmingham, said: “The recent discovery of the fundamental role of metabolism in immune cell biology is contributing immensely to our understanding of immune cell regulation.”
Dr Michelangelo Certo, of the University of Birmingham, added: “So far, most studies have focused on the role of metabolic pathways in the establishment of the immune response.
“Lactate has mainly been seen as a by-product of metabolism or as a biomarker in critical care at best, rather than a bioactive molecule, and its functional effects have thus been neglected for a long time.
“However, far from being inert, the accumulation of lactate in the cells, molecules and structures of tissue is found in both inflammatory disease and cancer.”
In this study, the researchers analysed the response of immune cells to lactate in chronic inflammation caused by rheumatoid arthritis.
In their research, which involved the use of mice, they also used blood from healthy patients and patients with arthritis, as well as joint biopsies, and tonsils removed following tonsillectomies due to the fact that inflamed tonsils share similarities with an inflamed joint.
Dr Claudio Mauro, of the University of Birmingham’s Institute of Inflammation and Ageing, said: “We identified the pathway initiated by lactate build-up in inflamed tissue that exacerbates the inflammatory response.
“We also now can provide evidence that molecules made by immune cells to transport sodium lactate – the sodium salt of lactic acid – could be a target for treatment to stop this lactate build-up in chronic inflammatory disorders.”
8th November 2019
The approval was based on positive interim results from the Phase III JAVELIN Renal 101 study, which demonstrated that Bavencio, a PD-L1 inhibitor in combination with axitinib (a VEGF inhibitor) significantly lowered risk of disease progression or death by 31% (HR: 0.69 [95% CI: 0.574–0.825; p<0.0001]) and nearly doubled the objective response rate (ORR; 52.5% [95% CI: 47.7-57.2] vs. 27.3% [95% CI: 23.2-31.6]) compared with sunitinib in patients with advanced RCC irrespective of PD-L1 status.
“Advanced kidney cancer has a devastating impact on patients’ quality and longevity of life and there is an unmet need to improve on the current available treatment and treat this disease with newer drugs,” said Dr Balaji Venugopal, Honorary Clinical Senior Lecturer, University of Glasgow. “Avelumab, a novel immunotherapy, in combination with axitinib has demonstrated clinically significant and meaningful improvements in efficacy by extending the time without worsening of cancer and causing tumour shrinkage, along with a generally manageable safety profile compared to currently available treatment. Notably this benefit is seen in all prognostic risk groups in patients with advanced renal cell carcinoma and can help healthcare professionals optimise their patients’ treatment. Today’s approval is a step in the right direction and it is exciting to be able to treat our patients first-line with this novel combination to improve outcomes for our patients.”
Since the early 1990s, kidney cancer incidence rates have increased by almost 87% in the UK and it is expected to be one of the fastest growing cancers over the next decade due to a range of factors including the UK’s aging population, smoking and the rise in obesity. More than 12,000 people are diagnosed with RCC in the UK each year and it is now the seventh most common cancer in British adults.
Approximately 40% of patients are first diagnosed with RCC at the advanced stage, and 30% of patients treated for an earlier stage go on to develop metastases. About half of patients living with advanced RCC do not go on to receive additional treatment after first-line therapy, for reasons that may include poor performance status or adverse events from their initial treatment. The five-year survival rate for patients with metastatic RCC is approximately 12%.
Nick Turkentine, CEO of Kidney Cancer UK said: “RCC is the most common type of kidney cancer, accounting for over 80% of all cases of the disease. Around 4500 kidney cancer deaths occur in the UK every year and people with advanced RCC have typically faced very poor outcomes. There is a considerable need for more treatment options, and we are pleased this new treatment for advanced RCC has been approved to be used in the UK. It’s important that it is now made available to patients who need the treatment as soon as possible.”
“Approval of the combination of Bavencio with axitinib for people with advanced RCC across all prognostic groups, allows patients to benefit from both an anti-PD-L1 immunotherapy combined with a VEGF inhibitor in first line,” said Belinda Byrne, Medical Director, Merck UK & Ireland “our focus now is to work with funding bodies across the UK to ensure patients have access to this much needed treatment option.”
“The European Commission approval of Bavencio in combination with axitinib has the potential to bring new first-line treatment options to patients living with advanced renal cell carcinoma,” Olivia Ashman, Oncology Medical Director, Pfizer UK. “Pfizer has been developing treatments for patients with kidney cancer for over a decade and, through the Alliance with Merck, will continue to help fight against advanced forms of this disease.”
The National Institute for Health and Care Excellence (NICE) technology appraisal for avelumab in combination with axitinib for untreated advanced or metastatic renal cell carcinoma is in development. The first committee meeting is scheduled for 15 January 2020, and the expected publication date for the final appraisal document is 08 April 2020.
Children with PFAS also suffer from seasonal allergies. PFAS (also known as oral allergy syndrome) is caused by cross-reacting allergens found in both pollen and raw fruits, vegetables or some tree nuts. Symptoms usually include itchy mouth, scratchy throat or swelling of the lips, mouth, tongue and throat.
“We surveyed 20 paediatric patients between the ages of 9 and 18 receiving allergy shots as treatment for mugwort, timothy and orchard grass allergies,” says allergist Alana Jones, DO, ACAAI member and co-author of the study, which was presented at the 2019 ACAAI meeting in Houston, US. “All 20 patients reported PFAS symptoms. Of the 20 surveyed, 11 (55%) described improvement or resolution of their symptoms. Four (20%) reported unchanged symptoms and five (25%) reported they hadn’t tried to reintroduce foods they’d previously reacted to.”
PFAS typically does not appear in young children. It is more common in older children, teens and young adults who have been eating the fruits or vegetables in question for years without any problems. In addition, young children under the age of 3 do not usually develop hay fever until after they are toddlers. Hay fever is associated with reactions to the pollens that cross-react with the foods. Those with PFAS typically have allergy to either tree, weed, and/or grass pollens.
“Research has shown that adults who are receive allergy shots see improvement in PFAS symptoms, but the effect hasn’t been widely shown in paediatric patients,” says Dr. Jones. “We think it’s an important finding, especially for kids who are receiving allergy shots and also suffering from PFAS.“
The data show that roxadustat significantly increased haemoglobin (Hb) levels in non-dialysis-dependent (NDD) and dialysis-dependent (DD) patients with anaemia from chronic kidney disease (CKD), respectively.
The OLYMPUS trial compared roxadustat to placebo while the ROCKIES trial compared roxadustat to epoetin alfa.
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “Anaemia is a common, serious condition among patients with chronic kidney disease. It occurs when the body has fewer healthy red blood cells than normal and low levels of haemoglobin, which may leave patients fatigued and short of breath. Results from OLYMPUS and ROCKIES reinforce the potential role that roxadustat could play in increasing haemoglobin levels and managing anaemia, which is often underdiagnosed and undertreated.”
Steven Fishbane, MD, Zucker School of Medicine at Hofstra/Northwell, Great Neck, New York, US and primary investigator on the OLYMPUS and ROCKIES trials, said: “These data demonstrated that roxadustat effectively increased haemoglobin levels for patients with anaemia from chronic kidney disease, including those who show signs of inflammation. Patients who experience chronic inflammation are often more difficult to treat than the overall chronic kidney disease patient population, emphasising the need for new treatment options.”
In the OLYMPUS trial, roxadustat demonstrated a statistically significant improvement in Hb levels from baseline, with a mean increase of 1.75g/dl averaged over weeks 28 to 52, compared to 0.40g/dl with placebo, the primary efficacy endpoint.
Roxadustat also improved Hb levels from baseline in a subgroup of patients with elevated high-sensitivity C-reactive protein (hsCRP) levels of greater than 5mg/l, with a statistically significant mean increase of 1.73g/dl, compared to 0.62g/dl with placebo, a secondary endpoint. hsCRP is a protein in the blood that increases when inflammation is present.
Overall safety findings are generally consistent with the NDD-CKD patient population. For all patients, the most frequently reported adverse events in the intent to treat analysis set were end-stage renal disease, pneumonia, urinary tract infection and hypertension. Additional serious adverse events reported were azotaemia, sepsis, acute kidney injury and hyperkalaemia.
In the ROCKIES trial, roxadustat demonstrated a statistically significant improvement in Hb levels from baseline with a mean increase of 0.77g/dl averaged over weeks 28 to 52, compared to 0.68g/dl with epoetin alfa, the primary efficacy endpoint.
Roxadustat also improved Hb levels from baseline in a subgroup of patients with elevated hsCRP levels of greater than 5mg/l, demonstrating a statistically significant improvement with a mean increase of 0.80g/dl compared to 0.59g/dl with epoetin alfa, a secondary endpoint. Patients treated with roxadustat used less monthly intravenous (IV) iron (mean = 59mg) compared to those treated with epoetin alfa (mean = 91mg) from week 36 to the end of the study.
Adverse events with roxadustat were generally similar to those seen in patients treated with epoetin alfa and commonly found in DD-CKD patients. In roxadustat treated patients, the most frequently reported adverse events were diarrhoea, hypertension, pneumonia, headache and arteriovenous fistula thrombosis. Additional serious adverse events reported were sepsis and acute myocardial infarction.
7th November 2019
But the underlying causes have been poorly understood: are hospitals really less safe on weekends or do other factors lead to a comparison-skewing weekday reduction of the risk of mortality?
A new study led by University of Cambridge researchers, based on nearly 425,000 emergency department attendances over seven years at Addenbrooke’s Hospital in Cambridge, confirms the weekend effect. This appears to be because junior doctors are more likely to admit patients with lower mortality risk during the week. The results are reported in the Emergency Medicine Journal.
The research found that junior doctors (qualified doctors still in training) based in the emergency department admitted less-sick patients at half the rate at weekends compared to weekdays, diluting the risk pool of weekday mortality and contributing to the weekend effect.
In contrast, the admitting patterns of senior doctors was the same on weekends and weekdays, and the data did not provide evidence of a weekend effect among patients admitted by senior doctors.
The researchers found that the weekend effect was associated with seniority of the physician working in the emergency department, that the case-mix of patients at the weekend was of a higher acuity and that junior doctors admitted fewer standard patients at the weekend than on weekdays.
“There has been previous research on how physician-level factors influence patient care, but our study instead focuses specifically on how seniority affects admitting patterns and in turn how this relates to the weekend effect,” said co-author Stefan Scholtes, Dennis Gillings Professor of Health Management at Cambridge Judge Business School. “It’s clear that the admitting patterns of junior doctors changes at the weekend.”
In a commentary about the new study, also published in Emergency Medicine Journal, the President of the Royal College of Emergency Medicine, Dr Katherine Henderson, said the study had “given us a lot to think about” – describing as “surprising” the finding that junior doctors admitted more relatively well patients on weekdays.
“The NHS needs to use its resources as effectively as possible,” she wrote. “We should only admit patients who need to be admitted. This paper suggests it would be a good idea to make sure we are using our senior decision makers where they can be most valuable – seeing sick patients and actively evaluating all borderline admission/discharge decisions.”
Although this is early research, the findings could ultimately lead to new ways to treat melanoma. The research is published in Cancer Research
Scientists have previously known that lower levels of vitamin D circulating in the body have been linked to worse outcomes for people with melanoma, but they haven’t fully understood the mechanisms that cause this.
Professor Newton-Bishop from the University of Leeds and her team wanted to see what processes were being regulated by vitamin D in melanoma cells, and what happens when there is a lack of a protein on the surface of the melanoma cells called a vitamin D receptor (VDR), which enables vitamin D to bind to the cell’s surface.
The researchers looked at the activity of the gene that makes VDR in 703 human melanoma tumours, and 353 human melanoma tumours that had spread from the initial site. The activity of the VDR gene was cross-referenced with other patient characteristics, such as the thickness of their tumour and how fast their tumor grew. They also wanted to see if the amounts of VDR in human melanoma cells were associated with genetic changes that happen when tumours become more aggressive. They then used mice to check whether VDR levels changed the cancer’s ability to spread.
The team found that human tumours with low levels of the VDR gene grew faster, and had a lower activity of genes that control pathways that help the immune system fight cancer cells.
They also discovered that tumours with lower VDR levels also had a higher activity of genes linked to cancer growth and spread, especially those controlling the Wnt/β-catenin signalling pathway, which helps to modulate a variety of biological processes within the cell, such as its growth.
In mice, the researchers found that increasing the amount of VDR on the melanoma cells reduced activity of the Wnt/β-catenin pathway, and slowed down the growth of the melanoma cells. They also found that the cancer was less likely to spread to their lungs.
Professor Newton-Bishop said: “After years of research, we finally know how vitamin D works with VDR to influence the behaviour of melanoma cells by reducing activity of the Wnt/β-catenin pathway. This new puzzle piece will help us better understand how melanoma grows and spreads, and hopefully find new targets to control it.
“But what’s really intriguing, is that we can now see how vitamin D might help the immune system fight cancer. We know when the Wnt/β-catenin pathway is active in melanoma, it can dampen down the immune response causing fewer immune cells to reach the inside of the tumour, where they could potentially fight the cancer better.
“Although vitamin D on its own won’t treat cancer, we could take insights from the way it works to boost the effects of immunotherapy, which uses the immune system to find and attack cancer cells.”
Martin Ledwick, Head Information Nurse at Cancer Research UK, said: “Vitamin D is important for our muscle and bone health and the NHS already recommends getting 10 micrograms per day as part of our diet or as supplements, especially in the winter months.
“People who have been newly diagnosed with melanoma should have their vitamin D levels checked and managed accordingly. If you are worried about your vitamin D levels, it’s best to speak to your doctor who can help ensure you are not deficient.”
Chiesi UK has announced that the first NHS funded patient with LSCD caused by chemical eye burns has been treated with its stem-cell based therapy, Holoclar, the first advanced therapy medicinal product containing stem cells to be approved in the EU.
In 2017, the National Institute for Health and Care Excellence (NICE) recommended Holoclar as an option for adults with moderate-to-severe LSCD after eye burns, if it is only used to treat one eye and in those who have already had a conjunctival limbal autograft (or there is not enough tissue for a conjunctival limbal autograft or it is contraindicated).
The therapy is a ‘tissue engineered product’ which consists of cells taken from the patient’s healthy limbus (at the edge of the cornea) during a biopsy. The cells obtained during the biopsy are then transported to the manufacturing facility at Holostem Terapie Avanzate in Italy, where they are prepared and grown in a unique culture to create a new layer of healthy tissue. After at least 50 days, this layer of healthy tissue is delivered back to the treating hospital and implanted by a surgeon into the damaged eye helping it to heal and repair the damaged corneal surface. In case of a partial bilateral problem, the healthy cells are taken from a spared portion of a patient’s less damaged eye.
Professor Francisco Figueiredo, Consultant Ophthalmologist at the Newcastle upon Tyne Hospitals NHS Foundation Trust and Professor of Ophthalmology at Newcastle University who treated the first NHS funded patient following NICE approval, said, “In Newcastle we are a leading centre with an international reputation in autologous limbal stem cell transplantation, and our significant scientific research and clinical experience in limbal stem cell deficiency has led to us having proudly performed the first Holoclar treatment on an NHS patient. This operation was the first of a series that may benefit a number of blind patients over the next few years, not only from Newcastle but from across the whole of the UK, helping to restore their sight and comfort”.
Chiesi’s UK Managing Director, Tom Delahoyde commented, “We are delighted that the first patient outside of a clinical trial has received this innovative and breakthrough medicine. This first NHS treatment marks a major milestone for those people with LSCD due to physical or chemical burns and we look forward to many more eligible patients benefiting from such a ground-breaking therapy. Chiesi would like to thank NHS England and the approved Trusts for their support on setting up this new service in the NHS over the last two years and reaching this milestone today.”
1st November 2019
New mortality data shows that there were around 5700 deaths from liver cancer in 2017 in the UK, which is the highest ever yearly number of deaths recorded. This has climbed from 3200 deaths in 2007. Of all cancer types, liver cancer has had the largest increase in deaths over the last decade and the most rapid rise in deaths since UK records began.
Experts at Cancer Research UK believe that death rates have risen so steeply because the number of people being diagnosed with liver cancer has also increased – by 60% in the last decade – and survival is typically low. It’s one of the hardest cancers to treat, and five-year survival can range from anywhere between 6% and 37% depending on age and gender.
The disease is difficult to treat mainly because it can be hard to spot at an early stage as it often doesn’t cause symptoms until it has progressed. Because of this, emergency presentations are the most common route to diagnosing liver cancer and surgery isn’t an option for many of these patients because the disease has already spread. Clinicians find that many patients also have chronic liver disease, so symptoms of cancer can be easily missed.
While there are several factors affecting liver cancer risk, being overweight or obese and smoking are two of the biggest preventable causes. A total of 23% of liver cancer cases can be linked to being overweight or obese, and 20% can be linked to smoking. Overall, around half of cases are preventable.
Cancer Research UK’s liver cancer expert, Professor Helen Reeves at Newcastle University, said: “Unfortunately, progress in treating liver cancer has been painfully slow and we desperately need more options for patients. Another problem is the rise in the number of people being diagnosed, which has meant we are losing more people to this disease than ever before.
“Rising levels of obesity and associated conditions like diabetes and non-alcoholic fatty liver disease have likely had a big role in this, although they aren’t the only factors.
“But the good news is that there are some potentially game-changing treatments on the way. Research looking at refining immunotherapy has been hugely promising. It doesn’t work in all patients just yet and we’re still looking at why that is, but it can add years to lives when it does.”
Every year, around 5900 people are diagnosed with liver cancer in the UK. And this number is projected to rise by 38% in the UK between 2014 and 2035. Although liver cancer can happen at any age, it is most common in older people over the age of 60. And it’s more common in Asian and Black people.
Cancer Research UK’s chief executive Michelle Mitchell said: “A lot of progress has been made saving lives from cancer, but it’s worrying to see deaths from liver cancer increasing at such an alarming rate. Far too many lives are being lost, which is why we’re funding more research into this area. And aiming to understand more about the biology of the disease to develop better treatments.
“There are things we can all do to make a difference to our cancer risk and it’s never too late to make a change. Keeping a healthy weight, not smoking, and drinking less alcohol will all help lower your chance of developing liver cancer.”
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