Category Archives: Clinical Research

Method to predict drug stability could lead to more effective medicines

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Researchers have developed a new method to predict the physical stability of drug candidates, which could help with the development of new and more effective medicines for patients.

Researchers from the UK and Denmark have developed a new method to predict the physical stability of drug candidates, which could help with the development of new and more effective medicines for patients. The technology is being developed for use in the pharmaceutical industry in order to make medicines that are more easily released into the body.

The researcher’s method solves an old problem: how to predict when and how a solid will crystallise. Using optical and mechanical measuring techniques, they found that localised movement of molecules within a solid is ultimately responsible for crystallisation.

Solids behave differently depending on whether their molecular structure is ordered (crystal) or disordered (glass). Chemically, the crystal and glass forms of a solid are exactly the same, but they have different properties.

Molecules in the glass form are more readily absorbed by the body because they can dissolve more easily

One of the desirable properties of glasses is that they are more soluble in water, which is especially useful for medical applications. To be effective, medicines need to be water-soluble, so that they can be dissolved within the body and reach their target via the bloodstream.

“Most of the medicines in use today are in the crystal form, which means that they need extra energy to dissolve in the body before they enter the bloodstream,” said study co-author Professor Axel Zeitler from Cambridge’s Department of Chemical Engineering & Biotechnology. “Molecules in the glass form are more readily absorbed by the body because they can dissolve more easily, and many glasses that can cure disease have been discovered in the past 20 years, but they’re not being made into medicines because they’re not stable enough.”

After a certain time, all glasses will undergo spontaneous crystallisation, at which point the molecules will not only lose their disordered structure, but they will also lose the properties that made them effective in the first place. A long-standing problem for scientists has been how to predict when crystallisation will occur, which, if solved, would enable the widespread practical application of glasses.

“This is a very old problem,” said Prof Zeitler. “And for pharmaceutical companies, it’s often too big of a risk. If they develop a drug based on the glass form of a molecule and it crystallises, they will not only have lost a potentially effective medicine, but they would have to do a massive recall.”

In order to determine when and how solids will crystallise, most researchers had focused on the glass transition temperature, which is the temperature above which molecules can move in the solid more freely and can be measured easily. Using a technique called dynamic mechanical analysis as well as terahertz spectroscopy, Prof Zeitler and his colleagues showed that instead of the glass transition temperature, the molecular motions occurring until a lower temperature threshold, are responsible for crystallisation.

These motions are constrained by localised forces in the molecular environment and, in contrast to the relatively large motions that happen above the glass transition temperature, the molecular motions above the lower temperature threshold are much subtler. While the localised movement is tricky to measure, it is a key part of the crystallisation process.

Given the advance in measurement techniques developed by the Cambridge and Copenhagen teams, drug molecules that were previously discarded at the pre-clinical stage can now be tested to determine whether they can be brought to the market in a stable glass form that overcomes the solubility limitations of the crystal form.

“If we use our technique to screen molecules that were previously discarded, and we find that the temperature associated with the onset of the localised motion is sufficiently high, we would have high confidence that the material will not crystallise the following manufacture,” said Prof Zeitler. “We could use the calibration curve that we describe in the second paper to predict the length of time it will take the material to crystallise.”


Magnetic nanoparticles prevent internal bleeding

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Scientists have found a way to effectively stop internal bleeding by magnetically-driven nanoparticles containing thrombin.

Scientists have found a way to effectively stop internal bleeding by magnetically-driven nanoparticles containing thrombin. A drug based on these nanoparticles can be injected intravenously and delivered straight to the site of a vascular injury. It can accelerate local clot formation and reduce overall blood loss by 15 times. The nanoparticles are not toxic to humans and can potentially be used for safe treatment.

In a new study, scientists from ITMO University suggested using magnet-driven nanoparticles to solve this problem. The particles consist of two key components. The first is thrombin, an enzyme responsible for blood clotting. It interacts with the protein called fibrinogen and triggers clot formation in order to block the damaged vessel. The thrombin is wrapped into a special porous matrix made of magnetite. This mineral is the second main ingredient and allows for precise control of the movement of particles inside the body using an external magnetic field.

Magnetic nanoparticles with thrombin have low activity and do not cause blood clotting if they are evenly distributed in blood vessels. Therefore it is possible to inject a solution of particles intravenously and localise them where needed using a magnet. When the patient receives an extra portion of fibrinogen, thrombin particles around the site of injury interact with it and the bleeding stops faster.

“We tested the nanoparticles’ efficiency on human blood plasma samples and a special vessel model,” says Andrey Drozdov, member of SCAMT Laboratory at ITMO University. “After the first experiments with plasma, we found out that thrombin in our nanoparticles is less active compared to its free variant. Yet we went on with the tests and ran additional experiments on a model of the bloodstream. We were able to observe how nanoparticles behave when the vessel is damaged. It turned out that magnetic localisation compensates for lower activity. Nanoparticles reduce the clotting time by 6.5 times and can reduce total blood loss by 15 times.”

“Synthesising these nanoparticles is not easy,” said the head of the laboratory Vladimir Vinogradov. “It is important to keep their size down to 200 nanometers; otherwise they will not be suitable for injection. In addition, mild synthesis conditions are required so that the thrombin molecule does not break down and lose its activity completely. Finally, we could only use biocompatible components. We checked the toxicity of our particles with human cells and made sure they are completely safe even during prolonged exposure.”

This work is part of a larger project aiming to create hybrid nanomaterial-based hemostatic drugs. Scientists are currently planning to test the drug based on the obtained material on animal models and, in case of success, run clinical trials. Researchers hope to create a nanoparticle-based hemostasis system that will be able to quickly and efficiently stop internal bleeding.

The results were published in Scientific Reports.


Amryt looking to expand rare disease franchise

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UK-based specialist Amryt Pharma is looking to expand in 2018 after acquiring two rare disease treatments.

A relative newcomer to the niche rare disease market, Amryt Pharma was founded by former Astellas medical director Joe Wiley and financier Rory Nealon in August 2015, who spotted an opportunity to develop products in under-served orphan disease therapy areas.

The Irish duo in-licensed their first product, Lojuxta from Aegerion in December 2016, and have since increased its revenues by more than 50%, as well as building a corporate team and European operations.

Lojuxta is licensed to treat the rare, life-threatening disease called Homozygous Familial Hypercholesterolemia (HoFH), and Amryt has marketing rights for the drug in the EEA region, as well as Israel, Turkey and MENA markets.

The company also has in its pipeline AP101, a promising product candidate currently in a pivotal trial for epidermolysis bullosa, a rare and debilitating skin condition.

Amryt expect to bring the product to the US and European markets by 2020, and believe the global market to treat the condition is worth around $1 billion annually.

“This is an exciting time of growth and expansion for us. Rare disease companies often start up in the US and then enter the European market, but our origins mean we have a deep understanding of what’s required and the nuances of doing business in the pharmaceutical space in Europe,” said Joe Wiley.

“I believe we’re proving this with the significant growth we have delivered in the space of one year with Lojuxta.”

The privately-held company is expected to look for further growth this year, and this includes interest in further in-licensing deals.

Meanwhile, the company is planning a paediatric study for Lojuxta and is currently awaiting sign off for its study design from the EMA.

The company is also pursuing additional licences for AP101.

“We’re planning to study other severe partial thickness wound conditions, such as Toxic Epidermal Necrolysis (TENs) / Stevens Johnson Syndrome (SJS), a rare, serious disorder of skin and mucous membranes. These are being planned in parallel in order to maximise the potential value of the AP101 asset for Amryt, our shareholders and most importantly for patients.”

Plans to enter US market

As for expansion into the US, the company says it already has its first “boots on the ground” in the US and in Latin America, its teams there establishing the opportunities in the market and meeting with relevant stakeholders.

“The EB market is particularly appealing in the US – there are approximately 20 centres focusing on EB, therefore it makes it easier for a small, agile company like Amryt Pharma to work closely with them,” says Wiley.

“We can offer patients what they need as we seek to improve their quality of life, with more effective wound care.”

The design of the global trial for AP101 has already been agreed with the FDA, and the company is currently completing the pre-clinical package and preliminary safety data set the US regulator has requested in order to open the IND specific to the US.

Amryt won’t have the EB market to itself, however, and a number of companies are developing novel treatments for the condition.

These include US-based Fibrocell Science, which has just got the green light to begin phase 2 trials of its gene therapy candidate FCX-007 for recessive dystrophic epidermolysis bullosa (RDEB).

Another company in the field is Netherlands-based ProQR Therapeutics, which is working on an RNA-based therapy for dystrophic epidermolysis bullosa (DEB).

Interim analysis of the data from Amryt’s EASE trial will be available later in 2018, followed by top-line analysis.

The company expects to compile its submissions to the FDA and EMA shortly thereafter, with marketing authorisation in Europe and US anticipated late 2019, or possibly early 2020.


Blood test could predict response to breast cancer drug early

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A new study has found that a blood test for cancer DNA could predict if a woman is responding to the new breast cancer drug palbociclib, months earlier than current tests.

Scientists from The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust, say the test could detect in two to three weeks whether the drug is working, although they caution that the results need replicating before they are used clinically.

The research, published today in the journal Nature Communications, was largely funded by the Medical Research Council (MRC). The researchers tested women with oestrogen receptor positive breast cancer – the most common kind – who were taking part in a clinical trial of palbociclib for advanced breast cancer.

In November 2017, palbociclib was approved for use on the NHS by NICE for women with previously untreated advanced breast cancer.

Currently, women must wait two to three months to find out if palbociclib is working, via a scan.

The new blood test instead looks for circulating tumour DNA – fragments of DNA shed by the cancer that have entered the bloodstream. The DNA mutations associated with the cancer can be detected in these samples.

The researchers found that they could predict if the palbociclib treatment would work by comparing the amount of a gene PIK3CA detected in a blood test before treatment and 15 days after starting treatment. In the study, 73 women had the PIK3CA mutation and were given blood tests before and after starting palbociclib treatment.

In these women, the researchers found that those who had a small decrease in PIK3CA circulating DNA at 15 days had a median progression-free survival (the length of time the patient survived and the cancer did not get worse) of only 4.1 months, compared to women with a large decrease in PIK3CA, who had a median progression-free survival of 11.2 months.

The test could allow the women in the first group for whom the treatment is not as effective to be identified early, so that they could consider altering their treatment.

Professor Nicholas Turner, senior author and Professor of Molecular Oncology at The Institute of Cancer Research, London, and Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, said: “Palbociclib is one of a new class of drugs that delays cancer progression for patients with advanced breast cancer, but it’s not effective for everybody. The problem is we have to wait for two to three months before doing a scan to see if the therapy is working.

“Our new study found that a blood test for cancer DNA in the first two weeks of treatment indicated whether the drug was likely to be effective. Having an early indication of how likely a treatment is to work might allow us to adapt treatment – switching some patients to an alternative drug that is more likely to benefit them.”

Dr Nathan Richardson, Head of Molecular and Cellular Medicine at the MRC, said: “This study provides early evidence that might help us understand sooner when a drug is successfully treating breast cancer, and if not, it can be discontinued and better approaches pursued.”

The research also received funding from the charity Breast Cancer Now and the pharmaceutical company Pfizer.


Gilead takes ‘HIV eradication’ combo into clinic

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Gilead has become a victim of its own success in hepatitis C, as it has pioneered a series of blockbuster combination drugs that are eradicating the disease, but are depleting the revenue-creating pool of infected patients.

Faced with pressure from competitors such as Merck & Co in hepatitis C, the company is now turning back to its old stomping ground – HIV, for which it has developed an arsenal of drugs that can stay the disease’s progression into full-blown AIDS.

Unlike hepatitis C, HIV is currently incurable, as drugs from Gilead and competitors like GlaxoSmithKline and Pfizer can only suppress it so that infected people can live much longer lives than in the past.

HIV creates a latent reserve within a patient’s body, lying dormant within infected CD4 cells, which can become activated and lead to the disease progressing into AIDS.

Only by tackling this reserve of infected, but dormant, T-cells can the disease be cured – and scientists have been trying for years to find ways to do this.

Gilead thinks it may have found a solution to the complex problem of tackling the infected dormant T-cells so that patients can control the disease without need for drugs.

Data from a trial in rhesus monkeys infected with simian-human immunodeficiency virus (SHIV) show that an oral toll-like receptor 7 (TLR7) agonist antiviral drug, and a broadly neutralising antibody (bNAb) could be an effective HIV eradication strategy for tackling this latent reserve.

Data from a subset of SHIV infected monkeys on suppressive antiretroviral therapy (ART) demonstrated a combination of a TLR7 called GS-9620, and a bNAb called PGT121, resulted in viral suppression after ART therapy stopped.

In the proof-of-concept study, 45% – five out out of 11 – of animals receiving both GS-9620 and PGT121 did not demonstrate viral rebound for at least 168 days after stopping ART. The other six animals rebounded but then began re-suppressing the virus without ART.

The data published at the Conference on Retroviruses and Opportunistic Infections showed that in a placebo arm 11 out 11 animals rebounded, nine out of 11 treated with only PGT121 rebounded and 10 out of 11 treated with GS-9620 rebounded.

Gilead is calling the combination therapy an “HIV eradication strategy” and will test it in phase 1 trials, in people who are HIV suppressed on approved ART therapies.

Gilead’s chief scientific officer, Norbert Bischofberger, said: “GS-9620 is currently in a phase 1b dose-escalation study in ART-suppressed people living with HIV and we have advanced GS-9722, a derivative of PGT121, into phase 1 testing.”

Gilead licensed in PGT121 from Theraclone Sciences under an agreement signed in 2014, although details of the deal were not disclosed at the time.

It was part of a portfolio of broadly neutralising antibodies discovered in collaboration with the International AIDS Vaccine Initiative and The Scripps Research Institute, Florida.


Rethinking the communication of clinical trial results

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If clinical trial results were more quickly and openly accessible, medical improvements would be implemented more swiftly too.

So what can be done to ease this process? What isn’t working and how can we change it?

It’s clear that people are continually adapting and evolving how they consume information, and businesses need to continually evolve to reflect this – just look at how social media and websites such as Craigslist have changed the newspaper businessBut is scientific publishing keeping up with this change? And, if not, what other options can be used in parallel to communicate clinical trial results data?

Results from clinical trials are shared for two fundamental reasons – to enable clinicians to use the knowledge gained to advance clinical care, and because patients take part in clinical trials with the expectation that the results will be used to improve treatments for themselves and others.

Clinical data therefore need to be communicated quickly and openly. Publishing them in scientific journals will continue to play a key part in this process, but it is important to consider any limitations and how to overcome them.

Challenges with the current model

Frustrating delays: Authors often comment that the communication of trial data, based on publication in conventional, peer-reviewed scientific journals, can be slow. The time taken from submission to publication of a manuscript, typically 6 to 12 months, means that important scientific research is not shared quickly enough in today’s era of rapid communication.

Can’t see the wood for the trees: Publication of results in one of many thousands of journals, that readers often have to pay to access, also means that data are difficult to find and may not reach the relevant audience.

Lack of innovation: The limited format of standard journal articles, often restricted to a certain number of pages and figures, makes it difficult for readers to gain a complete picture of the trial’s findings, while the static nature of these publications discourages iterations, creativity and interactivity. Crucially, this also means that journals may not reflect how readers prefer to consume information in an increasingly connected, and socially integrated, world.

Bias: As humans we naturally prioritise exciting, positive or surprising findings over the negative or mundane, and it’s no surprise that this results in bias, unconscious or conscious, in the study data that are selected for publication. Everyone contributes towards this bias: authors, reviewers, journals and even readers. Although vehicles do exist to publish less ‘exciting’

data, it’s almost impossible to eliminate bias completely and so we need alternative ways of sharing the totality of data.

‘Although the publication of clinical trial data in scientific journals will remain the gold standard, it is important to consider how this can be improved to ensure all audiences have quick, easy and open access’

How can the model change?

The International Committee of Medical Journal Editors (ICMJE) published the following requirements last year, showing that there may be light at the end of the tunnel:

  • From 1 July 2018, clinical trial manuscripts submitted to ICMJE journals must contain a data-sharing statement that explains what patient data will be shared (e.g. patient-level data), what additional documents will be made available (e.g. clinical study reports or protocols), how these can be accessed, and when this will happen.
  • Clinical trials that start enrolling patients after 2019 need to include a data-sharing plan in the trial’s registration.

These requirements are not as stringent as might have been expected, and do not make data sharing mandatory, but they certainly signal a shift towards greater openness.

Of course the website mandates interventional trials are registered there to report results on the site within one year of completion. However, an audit in 2012 established that a remarkable 88% of studies registered on had failed to do so.

Some journals have developed digital platforms in the hope of overcoming the challenges of traditional scientific publications. They offer features such as very fast publication, no length restrictions, links with social media, open peer review and pre-peer review publication. These are gaining some recognition, as shown by the Wellcome Trust’s launch of an in-house journal to ensure rapid publication of data from its funded research, to encourage transparency and reproducibility.

There is also the option of using a pre-print server such as bioRxiv to share publications and gain comments before submission to peer-reviewed journals. However, although these are being more widely used, they have yet to gain broad acceptance for sharing clinical trial manuscripts.

Platforms such as these are starting to improve the sharing of clinical data, but have yet to significantly change the behaviour of most investigators and authors.

‘Advances towards more transparent and effective data sharing include proposals by the ICMJE and new digital publishing platforms, but more needs to be done’

What should clinical trial data communication look like in the future?

Groups such as Alltrials have been working to ensure that results from clinical trials are fully reported, which is obviously a great aim. The downside, though, is that the pharmaceutical industry has sometimes been portrayed as being reluctant, over-cautious and opaque when it comes to communicating clinical data.

There is, however, a clear opportunity for pharmaceutical companies, and forward-thinking medical communications agencies, to be leaders in shaping how clinical data are shared with the wider medical community. We strive for a future where trial investigators have open access to all clinical data, and use this to prepare compelling educational communications that provide an overview of a clinical development programme, or place new data into more relevant context that reflects the real world in which patients live and HCPs practice.

These communications would be published rapidly in openly accessible scientific journals that provide opportunities for innovation and interaction, backed by easily available full datasets from every study, including completed trials. Crucially, this also enables the development of innovative approaches to harness, analyse and activate the wealth of clinical data that are currently hidden to provide new advances into diagnosis and treatment.

Making changes in how clinical data are communicated and shared will not necessarily be easy, but the need to incorporate a transparent, efficient and consistent method of sharing trial data is increasingly recognised across academia, industry and public organisations. Together, pharmaceutical companies and medical communications agencies should take the opportunity to be at the forefront of this move towards excellence in sharing data with the wider medical community.

This will ensure that the right information is quickly and efficiently communicated to the right audience using the most appropriate format and channel, and with valuable context, so building trust and, ultimately, making a positive change to patients’ lives.

About the author:

Mike Thompson is a Senior Scientific Director at Complete HealthVizion. He is an experienced medical communications professional who provides editorial, scientific and strategic insight into communications and publications across a range of therapy areas and clients.


Big pharma could turn to viruses to boost cancer immunotherapies

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Merck & Co’s $394 million acquisition of Viralytics has stoked interest in oncolytic viruses, a class of drugs that have been in the shadow of the checkpoint inhibitors and CAR-T therapies that are helping to set the standard of care in cancer.

But Merck’s deal to acquire Viralytics suggests this could be about to change, according to one of the Australian company’s close competitors, Norway’s Targovax.

Representatives of Targovax interviewed by pharmaphorum said the marketplace is set to get increasingly crowded in the future as pharma tries to use the approach to improve efficacy of checkpoint inhibitors.

There is only one FDA-approved oncolytic virus, Amgen’s Imlygic (talimogene laherparepvec) in 2015, but sales have been disappointing.

But with another 10 oncolytic viruses in clinical development, and another 40 in labs, the hope is that oncolytic viruses could be used to ‘prime’ tumours before treatment with checkpoint inhibitor immunotherapy, which has a response rate as low as 20% in some cancers.

Oncolytic viruses work by injecting genetic material into cancer cells, which modify them and make them visible to the immune system, which moves in to destroy the.

The issue with Imlygic is that it is approved as a monotherapy – and the thinking is that oncolytic viruses are likely to be more effective when used in combination with other drugs, notably immunotherapy drugs such as checkpoint inhibitors.

This is the approach taken by Viralytics, and by Targovax, which is looking to find partners as it progresses its two oncolytic viruses through the clinical trial process.

Now that Viralytics is effectively part of Merck & Co, this leaves Targovax as one of the most advanced independent biotechs working with oncolytic viruses.

Bristol-Myers Squibb has also done a big oncolytic virus deal, acquiring rights late last year to an “armed virus” targeting cancer from the UK biotech, PsiOxus Therapeutics, worth almost $900 million if the project is successful.

Merck deal ‘no suprise’

Targovax’s chief medical officer, Magnus Jaderberg, told pharmaphorum in an interview that the Viralytics deal came as no surprise following strong phase 2 results from its Cavatak, based on the Coxsackievirus, back in 2015.

Jaderberg said: “As soon as they released the data we felt that somebody would go after them and we were right. We were not surprised about the deal and we think it is very positive for us.”

He expects more interest in oncolytic viruses from Roche and Genentech, which so far have not done a big oncolytic virus deal, perhaps to boost their immunotherapy Tecentriq, which has produced some mixed results in trials.

Already partnered with Medimmune on one deal, Targovax is planning a phase 2 trial of its adenovirus-based drug TG01, targeting resected pancreatic cancer in combination with Merck’s Keytruda (pembrolizumab).

This could be extended to a phase 3 trial leading to registration in this disease, where there has been no significant progress for the best part of two decades CHECK.

Targeting RAS mutations, the drug could be used in 30% of all cancers that express this biomarker, suggesting a sales potential well in excess of the $400 million a year the company forecasts if approved in early pancreatic cancer.

Also in the Targovax pipeline is ONCOS-102, an oncolytic virus, which is in phase 1b/2 trials for mesothelioma and could produce an early readout in the coming weeks.

It is also developing ONCOS-102 in partnership with AstraZeneca’s MedImmune unit and the Cancer Research Institute for ovarian and colorectal cancer.

Jaderberg has been busy tapping up his contacts in big pharma having previously worked as chief medical officer for Bristol Myers Squibb in Europe.

He has already been in talks with manufacturers of checkpoint inhibitors to try and get licensing deals in place to fund further development.

Wiklund said: “It is tough as a small biotech to lift a phase 3 programme – it is obvious we are interested in licensing out one or both of our programmes.”

But Targovax’s CFO Erik Digman Wiklund was cagey about the prospects of a big buyout as seen in the case of Viralytics. “It is incredibly unpredictable, it is difficult to make any comment,” he said.


UK to crack down on 237m annual medication errors

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A new study commissioned by the Department of Health and Social Care has revealed that 237 million medication errors are estimated to be occurring within the UK’s healthcare system each years.

Within these figures, it is predicted that 700 deaths are directly as a result to medication errors and a potential 22,300 further deaths could be as a result of related mistakes.

Errors covered by the study included the wrong medications being given to patients, incorrect doses being delivered or a delay in medication being received.

The study did note that the vast majority, three quarters of cases, would have no impact on the health of patients. However, the cost of these errors is estimated to be worth around £1.6 billion to the NHS each year.

There are a total of 1.15 billion prescriptions made each year, with approximately 91% of these being made by GPs. So, even though the report suggested 71% of errors were made in primary care, it is not GPs that are disproportionately making mistakes but simply due to writing a larger percentage of prescriptions.

Responding to Health and Social Care Secretary Jeremy Hunt’s proposed measures to reduce medication errors in the NHS, Professor Helen Stokes-Lampard, Chair of the Royal College of GPs, said: “Identifying and prescribing the drugs that are most likely to benefit a patient is a core part of a GP’s training and daily practice.

“GPs work hard to avoid making mistakes in the prescribing process, and our patients should be reassured that in the vast majority of cases, prescriptions are made appropriately and correctly. But as well as being highly-qualified medical professionals, doctors are also human, so medication mistakes can and occasionally do happen.”

In response to the study, Jeremy Hunt is expected to announce changes further strengthening linked-up care between primary and secondary care services.

This will involve the wider introduction of electronic-prescribing systems, which will then be checkable by doctors on hospital admission – to ensure no errors has been made with prescription that led to the hospitalisation.

In practice, this will begin with ensuring NSAIDs given patients were delivered alongside a protective to the digestive system, as a test case.


AI predicts heart attack risk factors from retinal scans

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Artificial Intelligence can be used to predict heart attack risk using retinal images, according to new research backed by Google.

The researchers trained deep learning algorithms on data from thousands of patients recorded in a massive UK study, which was used with retinal scans to produce a program that can identify risk factors from the scan information alone.

This predicted cardiovascular risk factors not previously thought to be quantifiable using retinal images: these included age, gender, smoking status, gender, systolic blood pressure and major adverse cardiac events.

Reporting results in the journal Nature Biomedical Engineering, the team used a dataset from the UK’s Biobank, a study where 500,000 were recruited between 2006 and 2010, and agreed to have certain health measurements recorded.

Health outcomes such as hospitalisation, mortality and cause of death were also logged. Smoking status was obtained via survey using a touchscreen interface in the research backed by Google and Verily, Alphabet’s healthcare subsidiary.

Participants were also asked to identify whether they are a current smoker, former smoker, or had never smoked, and blood pressure readings were also taken.

A further 67,725 had paired images of their retina fundus taken, along with a second group that was used to create a training dataset with known risk factors.

The researchers asked a neural network to make an output prediction based on the fundus image.

It was able to analyse images from the group with unknown risk factors, compare it with the training data set and after the process was repeated, was eventually able to predict cardiovascular risk factors from new images.

This is just the latest in a series of studies showing the potential of AI to predict healthcare outcomes, saving lives and reducing costs by ensuring patients receive timely and sometimes life-saving treatments.

Early last month, the government’s life sciences tsar, Sir John Bell, said that similar techniques could save the NHS billions.

A team at Oxford’s John Radcliffe Hospital is using AI to identify abnormalities in ECG read-outs that could be missed by the human eye, for instance.

The hope is that this system could be used in hospitals across the country to prevent unnecessary hospitalisations caused by false positives, and prevent heart attacks where at-risk patients are sent home because doctors have failed to spot problems.

Another AI system outperformed a panel of experts when asked to diagnose breast cancer based on stained tissue samples, in a separate study published in the Journal of the American Medical Association last month.


Priority review for Shire’s HAE drug

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US regulators are undertaking a speedy review of Shire’s lanadelumab (SHP643) for the prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE).

HAE is a rare, genetic disorder that causes debilitating, painful and sometimes life-threatening swelling in the body.

Shire’s application for lanadelumab, an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein, is supported by data from four clinical trials.

Data from the pivotal Phase III HELP study showed that subcutaneous administration of 300mg lanadelumab once every two weeks resulted in an 87 percent reduction in the mean frequency of HAE attacks.

In addition, an exploratory endpoint, which will require further confirmatory studies, showed that during the steady state stage of the trial (day 70-182) a 91 percent attack reduction was achieved with eight out of 10 patients reaching an attack free state.

In this study, no treatment-related serious adverse events or deaths were reported, the most common side effecting observed being injection site pain (29.3 percent placebo vs. 42.9 percent across lanadelumab arms).

“Physicians as well as patients in the HAE community are excited to see lanadelumab moving forward for FDA review because there is now the real possibility of having a new way to prevent HAE attacks,” said Aleena Banerji, Massachusetts General Hospital, Boston, MA, and clinical trial investigator.

The US Food and Drug Administration has granted the application a priority review, signaling its belief that the drug has the potential to provide a significant treatment benefit to patients. As such, the regulator’s assessment should be completed within eight months as opposed to 12 months, meaning that a decision is due by August 26.