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31st October 2019
Dupixent is indicated as an add-on therapy with intranasal corticosteroids for the treatment of adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control.
CRSwNP is a chronic disease of the upper airway that obstructs the sinuses and nasal passages. It can lead to persistent breathing difficulties, nasal congestion and discharge, reduced or loss of sense of smell and taste, and facial pressure or pain.
“People living with severe CRSwNP, are often desperate to find new treatment options given that current standard treatments such as intermittent courses of systemic corticosteroids or sinonasal surgery are associated with disease recurrence,” said George D Yancopoulos, MD, PhD, President and Chief Scientific Officer at Regeneron. “Dupixent significantly improved the signs and symptoms of severe CRSwNP, and also eliminated the need for further surgery or corticosteroid use in approximately three-quarters of patients. Today’s approval provides patients in Europe with the first biologic treatment to address the type 2 inflammation that underlies most CRSwNP. This is the third type 2 disease in which Dupixent has been approved, and we continue to investigate Dupixent in a broad range of type 2 inflammatory diseases.”
“Many patients with CRSwNP have co-morbid asthma, and those patients tend to have more severe disease that is often more difficult to treat,” said John Reed, MD., PhD, Global Head of Research and Development at Sanofi. “These particular patients may have an increased risk of asthma attacks, high symptom burden and a substantial adverse impact on health-related quality of life. Nearly 60% of the patients in the CRSwNP trials had asthma, and the data showed Dupixent provided an additional benefit of improved lung function in these patients.”
Truncated proteins (nonsense mutations) can give rise to hereditary diseases and different types of cancer. To keep the number of truncated proteins to a minimum, human cells recognise and remove RNAs with nonsense mutations through a quality control process known as nonsense-mediated mRNA decay (NMD).
To better understand the effect of NMD on human disease, researchers built NMDetective, a tool describing every possible nonsense mutation that can occur in the human genome. Developed by large-scale statistical analyses based on machine learning, the algorithm identifies which mutations in the genome are susceptible to NMD.
As described in Nature Genetics, scientists used NMDetective to analyse thousands of genetic variants that give rise to hereditary diseases in humans. “We were surprised to observe that, in many cases, NMD activity was predicted to lead to a greater severity of the disease,” says Fran Supek, ICREA researcher, head of the Genome Data Science laboratory at IRB Barcelona and leader of the team that built the tool.
The results of the study suggests that pharmacological NMD inhibition could slow the progression of many different genetic diseases. To distinguish which patients would benefit from this therapy, it is necessary to apply a precision medicine approach to determine the mutation responsible for the disease and the effect of NMD on this mutation, and this is precisely where NMDetective comes into play.
Researchers also studied the role of NMD in cancer and the interaction between the tumour and the immune system and found that NMD hides mutations that would otherwise trigger the immune system. Therefore, NMDetective can be used to analyse the mutations present in the tumour, in order to better distinguish between cancer patients that respond to immunotherapy from those who do not respond to immunotherapy.
“Tumours are riddled with genetic mutations that should make all sorts of weird proteins. The immune system should pick these up, identifying the cells gone wrong and destroying them,” says Ben Lehner, a researcher at the Centre for Genomic Regulation who also took part in the study. “But many of these weird proteins never actually get made because of NMD.”
“This algorithm can distinguish which mutations will and won’t trigger this error-checking system. What’s exciting is that drugs that block NMD already exist, which could be used in conjunction with other treatments to help the immune system better recognise tumour cells,” explains Lehner.
“Here we see an example where publicly available cancer genomics data can be ‘reused’ through machine learning approaches to better understand biological processes such as NMD,” says lead author Rik Lindeboom, a researcher at Radboud University in the Netherlands. “What makes this study especially exciting, is that we could directly translate this fundamental research into insights that are relevant for clinicians and patients.”
Their study suggests that blocking a signalling molecule known as TAK1 can suppress inflammation caused by gout.
Elevated uric acid levels can lead to the formation of monosodium uric acid (MSU) crystals that accumulate in joints. In gout, the immune system will perceive these crystals as a threat and launch an immune response against them that increases the production of interleukin-1-beta (IL-1-beta), a cytokine protein that causes inflammation and triggers the intense pain and swelling people experience during gout attacks.
“It’s kind of a vicious cycle that starts with these crystals, which cause IL-1-beta to be produced, inducing inflammation and activating a lot of other proteins to produce more inflammation,” said Salah-Uddin Ahmed, a professor of pharmaceutical sciences in the WSU College of Pharmacy and Pharmaceutical Sciences and senior author on the study.
“We already knew that MSU crystals activate what is known as the inflammasome pathway, which produces IL-1-beta,” Ahmed said. “However, our study found that MSU crystals also use an alternate pathway that triggers inflammation through TAK1, which is a new finding related to how gout develops.”
Next, they showed that the use of a chemical that inhibits, or blocks, TAK1 could completely suppress any inflammation caused by MSU crystals, both in healthy human macrophage cells and in a rodent model of gout.
Ahmed said their discovery has opened the door towards the development of new treatment strategies for gout.
One current treatment he said scientists have experimented with is Anakinra, a drug that blocks the binding of IL-1-beta to its receptor. Though it has shown promise, Ahmed said the drug is not clinically used for gout, because it is given by infusion – which requires hospitalisation; its effectiveness is limited; and it comes with a potential risk of infections when used long term. Developing TAK1 inhibitor drugs that could be taken by mouth would allow patients with gout to manage flare-ups of the disease at home.
The team’s next goal is to confirm their findings in cells taken from patients with gout. They are currently pursuing federal funding for this project, which they plan to conduct in collaboration with clinical scientists at the University of Alabama Birmingham and the University of Michigan Ann Arbor. If their findings hold up, this may eventually lead to clinical trials to test TAK1-inhibitors in patients.
Ahmed said their finding could also eventually be tested in other diseases that involve IL-1-beta mediated inflammation, such as multiple sclerosis, inflammatory bowel disease, and type 1 diabetes.
In the study, published in The Journal of Allergy and Clinical Immunology, researchers tested samples from patients who suffer from peanut allergy with peanut oral immunotherapy (POIT) and found that it does not stop them being allergic just suppresses the reaction.
In this study, the researchers tested samples from peanut allergic patients who underwent POIT and found that although POIT does suppress allergic reactions, when the protective antibodies were removed, the allergic cells are still as reactive as before treatment. These findings support the concept that while POIT can provide some protection it does not ‘cure’ the allergies.
Lead author, Dr Alexandra Santos, King’s College London and Evelina London Children’s Hospital commented “Peanut oral immunotherapy can confer some protection to accidental exposure to peanut as a result of the so-called ‘blocking antibodies’ and shown by the reduction in the reaction of allergic cells after treatment but if we remove these ‘blocking antibodies we could see that the cells are still as reactive as before, confirming that the patients were still allergic and need to keep going with the POIT regimen to maintain the protection.”
The authors used a test known as mast cell activation test (MAT) to confirm whether the patients are still allergic after POIT and need to continue taking peanut as per POIT regimen or are no longer allergic and can eat peanuts freely.
The next steps in the research are to validate these findings in other cohorts of peanut allergic patients treated with POIT and confirm that the MAT is useful to monitor patient’s response to this treatment.
Dr Santos said: “Definitive treatments for peanut and other food allergies are highly needed. Currently, immunotherapy is the only treatment option we can offer peanut allergic patients, mostly through clinical trials as this is not yet available on the NHS. Immunotherapy can be delivered by ingestion (oral immunotherapy), under the tongue (sublingual) and applied to the skin as a patch (epicutaneous immunotherapy). To a greater or lesser extent, the different forms of peanut immunotherapy can make allergic patients less sensitive, which can protect them from accidental exposure, but patients remain allergic.”
24th October 2019
In the study, 136 patients were enrolled and 131 were randomised (66 to CT-P13 SC and 65 to CT-P13 IV). After loading doses of IV 5mg/kg at weeks 0 and 2, patients were randomised at week 6 to receive either CT-P13 SC 120mg (<80kg) or 240mg (≥80kg) every 2 weeks in the CT-P13 SC arm, or CT-P13 IV 5mg/kg every 8 weeks in the CT-P13 IV arm.
The Phase I pivotal study demonstrated that CT-P13 SC showed non-inferiority in PK to CT-P13 IV as determined by the trough concentration, pre-dose level (Ctrough) at week 22. In terms of efficacy, both clinical response and remission in patients were induced and maintained, and the overall efficacy results of CT-P13 SC were comparable. The combined clinical remission rates for CD and UC patients at week 30 were comparable between CT-P13 IV and CT-P13 SC (66.7%, 44 out of 66 patients in CT-P13 SC arm and 54.7%, 35 out of 64 patients in CT-P13 IV, p=0.1620). The safety profile of CT-P13 SC was comparable to CT-P13 IV. In addition, fewer patients were found to have positive anti-drug-antibodies with CT-P13 SC compared to CT-P13 IV.
A subcutaneous formulation of CT-P13 has the potential to enhance treatment options for the use of biosimilar infliximab by providing high consistency in drug exposure and a convenient method of administration.
“CT-P13, a biosimilar of infliximab, has already been thoroughly characterised as a prime choice for intravenous infliximab therapy in the past with dedicated trials demonstrating clinical similarity in comparison with the originator molecule in both RA and IBD. The new, highly innovative SC formulation of infliximab has been specifically developed to address unmet needs in maintenance therapy. Our results from the ongoing development program for SC CT-P13 infliximab are promising. CT-P13 SC infliximab has the potential to become the most innovative biosimilar treatment – improving convenience and allowing patients to have more control of their treatment in addition to direct clinical benefits,” said Professor Stefan Schreiber, Director of the Clinic for Internal Medicine at Kiel Campus of the University Hospital Schleswig-Holstein in Germany and first author of the study.
“Not without reason the IV/SC sequence therapy that has been implemented into most development paths for novel molecules.” “A new subcutaneous formulation of infliximab has the potential to change the way patients manage their condition on a day-to-day basis, offering them greater choice and convenience in the long term. Furthermore, offering patients the option to self-administer will reduce demand on healthcare systems, by lessening patient time spent in hospitals, keeping patients out of clinics and providing clinicians with additional time to spend with other patients,” said Mr. Hyoung-Ki Kim, Vice Chairman at Celltrion Healthcare.
“This new formulation could create a new treatment paradigm with IV/SC sequence therapy making treatment affordable, convenient and personalised.”
An insomnia medication may be effective in preventing alcohol intake and relapse in alcohol use disorder, and a new clinical trial has been launched to investigate.
‘Alcohol use disorder presents significant social and economic burden worldwide,‘ said Professor Andrew Lawrence from The Florey Institute, Australia. ‘Outside of Europe, no new treatments have been approved since Campral (acamprosate) more than 20 years ago.‘
The Florey Institute and St Vincent’s Hospital in Melbourne, Australia, will trial the treatment of suvorexant (brand name Belsomra) in 128 participants with co-occurring insomnia and alcohol use disorder.
Led by Professor Lawrence, researchers at The Florey Institute have been studying the brain mechanisms underlying alcohol use disorder for over 15 years. His team was the first in the world to publish evidence on the role of a peptide system in the brain – known as the orexin system – in driving relapse to seek and consume alcohol.
‘The orexin system demonstrates just how complicated addiction can be. We know that chronic intake of alcohol greatly disrupts sleep and wake patterns which, in turn, can drive the brain to further seek and consume alcohol,‘ said Lawrence.
Belsomra, which is manufactured by MSD, is understood to block the binding of orexin in parts of the brain involved in addiction. The medication also targets the amygdala, a brain region associated with mood disorders, including depression and anxiety.
The study will examine sleep measures in trial participants, as well as looking at alcohol withdrawal symptoms and ongoing alcohol use. In the double blinded study, participants will receive either Belsomra or placebo treatment daily for seven to 10 days in hospital before continuing treatment for up to six months with regular follow up.
17th October 2019
Reason Digital is teaming up with Parkinson’s UK, the Stroke Association, Muscular Dystrophy UK and the MS Society to develop the project, which is set to transform the way medical advice and information is delivered to almost half-a-million people in the UK.
The Digital Health Assistant (DHA) will use machine learning to develop an understanding of the person being supported and continues to adapt to their needs over time based on interactions. This allows DHA to provide emailed content and support specific to the individual’s needs, making it more effective than current alternatives.
Alison Butt, 52, a health visitor living with Parkinson’s who tested the assistant, explained: “When I first heard about an AI health assistant I thought it sounded innovative and wanted to take part in a project to help others with Parkinson’s.
“I have been living with the condition for the last year after noticing a tremor in my right hand. Following diagnosis by a neurologist Iwas given a hefty folder of information which was overwhelming. I refused to Google my condition and instead found the Parkinson’s UK website very helpful. I joined a local support group and spoke to my friend who also has the same condition.
“I’ve found there’s an ongoing process of coming to terms with the condition. The DHA trial was a real eye-opener, I liked the idea of receiving weekly bite-sized tips and this influenced me to find new ways to slow the condition down. I discovered specially designed exercises for those with Parkinson’s such as PD Warrior, which helps improve functionality and prolong quality of life. The weekly classes I attend are motivating, run by neuro physios and help correct posture and strengthen your core.”
Richard Nash, 54, a physiotherapist living with Parkinson’s who trialled the system, added: “After being diagnosed in January I was asked to take part in the DHA pilot. It was great as there is a real need for two-way information sharing and up-to-date content that’s relevant to each stage of your condition. When it’s developed DHA will be a reliable and relevant portal that’s efficient and accessible. It will act as a gatekeeper that can be accessed at any time online, so users won’t have to spend hours researching from unofficial sources online.
“The main challenge I’ve encountered since diagnosis is a psychological one, as I’m concerned about keeping my job and thinking about the future. It’s been hard coming to terms with how things will be. I found researching Parkinson’s online hard as there’s no filter and some sites are untrustworthy. DHA is a real breakthrough as it’s convenient, can be accessed at home, is official and guides you every step of the way. ”
Matt Haworth, co-founder of Reason Digital, said: “Understandably there’s a lot of fear around the implications of AI for society. The reality is that whether it empowers us or oppresses us simply depends on who’s using it. That’s why with DHA, we’re putting the power of AI in the hands of people who need support for their long-term health conditions.
“Diagnosis of a serious health condition such as Parkinson’s, Muscular Dystrophy or multiple sclerosis, or experiencing a stroke, naturally generates an abundance of questions for the person receiving the news. Online research so often results in unanswered questions and out of date, generic information which just adds to the stress and anxiety of an individual’s situation. What people want is curated information and updates from a trusted source.”
The tool has been designed to tackle common issues pervading chronic conditions in the UK, including the lack of tailored and up to date information available for people who’ve recently been diagnosed, and staff shortages. The four charities involved expect it to dramatically improve the quality of practical and emotional care on offer to individuals following a diagnosis and, longer-term, support them in successfully managing their condition.
The technique involves attaching small microphones to knees, and detecting high frequency sounds from the joint components as people perform sitting standing movements.
The signals, known as acoustic emissions, are computer-analysed to give information about the health of the knee. The analysis is based on sound waveforms during different movement phases.
The latest study, published in PLOS ONE, is the first to investigate the use of the technique in a large local cohort of people previously diagnosed with knee OA. The results show that the technique can distinguish between healthy and OA knees, and that it works well both in general practice and hospital settings.
The research was conducted by a large multi-disciplinary team led by Lancaster University with partners from the Universities of Central Lancashire, Manchester and Cumbria, as well as the NHS in Lancashire and Cumbria. It also involved two businesses working on leading edge technologies for acoustic sensing and cartilage measurement.
Emeritus Professor Goodacre of Lancaster University, who led the project, said, “This work is very exciting because it involves scientists and clinicians working together as a team to develop an entirely new approach. Potentially, this could transform ways in which knee OA is managed. It will enable better diagnosis, and will enable treatments to be tailored more precisely according to individual knee condition. It will also enable faster, bigger and better clinical trials of new treatments“.
Researchers are now developing a non-invasive portable device which could be used by health professionals to see whether patients’ knees are changing or responding to treatment. This will provide a quicker, cheaper, more convenient and more refined assessment than currently-available methods.
15th October 2019
One-year results from the ongoing Global ETNA-AF (Edoxaban Treatment in routiNe clinical prActice) registry, providing a snapshot of characteristics and outcomes from a broad range of NVAF patients receiving edoxaban in routine clinical care, were presented at the Great Wall International Congress of Cardiology (GW-ICC) 2019, in Beijing, China.
Overall, results from ETNA-AF at one year showed low rates of bleeding (major and gastrointestinal [GI]), intracranial haemorrhage (ICH) and ischaemic events during the first year of edoxaban therapy. Per year, in the 12,574 patients from 825 sites in Europe:
Rates of systemic embolic events (SEE) and myocardial infarction (MI) were generally low. Per year in Europe:
Additionally, mortality rates were low. In total, per year in Europe:
“In routine clinical practice, the rates of major and clinically-relevant non-major (CRNM) bleeding in elderly edoxaban-treated patients were lower than those observed in clinical trial settings. Additionally, rates of all other cardiovascular events were also low in these patients,” said Professor Raffaele De Caterina, Professor of Cardiology, Institute of Cardiology at the University of Pisa, Italy. “Atrial fibrillation is the most common form of arrhythmia in patients over the age of 65, and its prevalence increases with age. As such, it is critical that we understand the safety and efficacy profile of edoxaban in elderly populations.”
The wider Global ETNA-AF programme, which at present is the largest and most comprehensive repository of routine clinical practice data on the use, effectiveness, and safety of a single NOAC in patients with atrial fibrillation (AF), collected data from 24,962 patients in 2242 sites in Japan, Korea/Taiwan and Europe.
“These positive results provide great insight into the safety and efficacy profile of edoxaban in NVAF patients, particularly in those who are elderly and/or those with comorbidities, who are considered at higher risk of cardiovascular events,” said Wolfgang Zierhut, MD, Executive Director Medical Affairs and Head Thrombosis and Cardiovascular at Daiichi Sankyo Europe. “Findings from ETNA-AF demonstrate that Phase III efficacy and safety results from the ENGAGE AF-TIMI-48 clinical study are being confirmed in regular routine clinical care across the region.”
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