71st Edition – September 2017

AstraZeneca opens up preclinical safety data to scientific community

Part of AstraZeneca’s contribution to the IMI project eTOX was in the form of extensive data on the safety/toxicity profiles of (potential) medicines. Now, the company has decided to make the data provided to eTOX available to the wider scientific community through the company’s Open Innovation portal. ‘Following on from the success of the eTOX project, we are keen to further broaden access to our preclinical safety data in order to help advance the mechanistic understanding and prediction of drug safety and bring safer medicines to patients faster,’ said AstraZeneca’s Nigel Greene. In a statement on the project website, the eTOX team notes that the project ‘has signified a paradigm change in the pharmaceutical industry’s willingness to openly share legacy data for the benefit of the wider toxicology community’. The eTOX project resulted in a number of tools for toxicologists, many of which have been made available to the wider scientific community.

CHEM21 contributes to Manchester MOOC

IMI’s CHEM21 project has contributed strongly to a massive open online course (‘MOOC’) on industrial biotechnology run by the University of Manchester. The course has six modules – three on the core principles of industrial biotechnology, and three on specific applications. All modules comprise a mixture of recorded presentations, videos, reading material and multiple choice tests. CHEM21 is devising new, more environmentally friendly ways of synthesising medicines, and scientists from the project were heavily involved in the design of the course. As a result, the sustainability principles underpinning CHEM21’s work are integrated into the entire course, particularly in the module on pharmaceuticals. The course was launched in July and the organisers hope that the bite-sized, modular nature of the course will make it attractive to both undergraduates and existing professionals who are using it as part of their continuing professional development.

PRISM clinical study on social withdrawal gets underway

The PRISM project’s clinical study on the biological underpinnings of social withdrawal in Alzheimer’s disease and schizophrenia is now officially underway, as the first patients have had their first clinical visits. Social withdrawal is a common early symptom of both Alzheimer’s disease and schizophrenia. However, we do not know if the underlying causes of social withdrawal in the two diseases are the same or different. In this study, PRISM scientists will study over 200 people, including people with suspected Alzheimer’s disease, people with schizophrenia, and healthy people (the control group). Participants with the diseases under study will include people who show signs of social withdrawal as well as people who do not. Participants will undergo a range of tests, including brain scans, blood tests and questionnaires. They will also be offered the opportunity to use a smartphone app called BeHapp that measures people’s sociability and social exploration in their daily lives. ‘The ability to precisely link symptoms to underlying neurobiology would not only facilitate the development of better treatments, it would also allow physicians to provide patients and relatives with a better understanding of the complexities and management of their illness,’ said the project. ‘Moreover, there is a growing realisation that psychiatric and neurodegenerative disorders overlap by much more than previously thought, and that they may better be described as domains of trans-diagnostic traits rather than separable categories.’

Pharma-Cog results may speed up Alzheimer’s drug development

When it comes to Alzheimer’s disease (AD), most potential drug candidates fail during clinical trials, resulting in significant setbacks in the development of effective treatments. IMI’s Pharma-Cog project set out to change that. The project developed a matrix of biomarkers which can be used to study the effect of a drug candidate both in animals and humans, and has the potential to more accurately predict the success of future drugs in early stages of drug development. Pharma-Cog scientists also found a better way to stratify patients with early signs of Alzheimer’s disease, which may lead to more definitive clinical trials. All this has the potential to help pharmaceutical companies conduct more efficient and more precise clinical trials in the future, speeding up the development of AD drugs. At least one biotech company is already using the results of Pharma-Cog to test a promising new drug candidate. ‘We have attracted an SME which didn’t take part in our project’, explained scientific coordinator Régis Bordet, in an interview with the IMI Programme Office. ‘They saw the results of Pharma-Cog and they asked me to help them develop their compound which could be very interesting for AD treatment. The project is worth 3 million euros and has received support from the French government. By early 2019 we will know whether there is a sizeable effect of this compound on Alzheimer’s, and whether it is worth pursuing further in a clinical trial. Thanks to this cooperation, we will have a real-life demonstration that we have done in Pharma-Cog is useful for the development of new compounds for AD patients.’

RAPP-ID outputs could boost development of rapid diagnostic tests

Doctors treating infectious diseases still don’t have rapid diagnostic tests which could help them identify the exact strains of pathogens ailing their patients. The development of such tests is complex and expensive, but that may improve thanks to IMI’s RAPP-ID project, which developed some of the technologies and innovations needed to speed up the development of such tests in the future. For example, the project developed a prototype of a breath sampler for influenza – the first test which uses something as simple as breathing out to detect the presence of pathogens in patients. ‘So far in clinical practice, there are no rapid point-of-care tests for detection of infections that are based on something as non-invasive as breathing out,’ said project coordinator Jorge Villacian, in an interview with the IMI Programme Office. ‘Of course, we are aware of the fact that we’re not there yet – this is still in an early stage of the development process. But the technology itself may be useful for other fields (outside of infectious diseases) as well.’ Some of the technologies created under RAPP-ID are undergoing further development in new EU-funded projects.

BTCure insights could have huge benefits for over-burdened healthcare systems

Rheumatoid arthritis is a chronic disease set off by the body’s immune system not working properly. It causes pain and swelling, especially in joints, and affects about 1% of the population. While new treatments have helped to relieve the inflammation, almost a third of patients still respond poorly to these and scientists have no way of predicting who they will work on and why. This makes the choice between available treatments complicated and uncertain. IMI’s BTCure project has tackled this knowledge gap head on. It has created better tools, such as new biomarkers, animal models of the disease, and bio-informatic systems. It has developed testing procedures (assays) to measure these biomarkers for signs of disease, its progress and its responses to therapy. BTCure researchers also believe they have partly answered the question of why joints are targeted by the immune system in rheumatoid arthritis. They identified a link between ‘osteoclasts’ – cells that absorb bone tissue during growth and healing – and the offending antibodies (ACPAs). ‘Close interaction between our industrial and academic partners using a large set of bio-banked samples resulted in new ACPA-detection chip designs,’ said Martina Johannesson of Sweden’s Karolinska Institutet, one of BTCure’s scientific coordinators. ‘These are instrumental to the device that is now being developed to help clinicians diagnose rheumatoid arthritis much earlier.’ This advance could have huge benefits for over-burdened healthcare systems and boost productivity in businesses with much fewer workers on long-term leave.
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Mofina develops device for faster testing of deadly Ebola

Ebola is a deadly disease which spreads among humans through direct contact with bodily fluids of infected patients. There is currently no licensed treatment, and development of medications and vaccines is hampered by challenges including the time required for diagnosis. IMI's Mofina project researchers have developed a portable device to test in the field whether a person has caught the deadly Ebola disease. It gives reliable results in 75 minutes, which can help contain outbreaks and save lives.  ‘The quicker contacts of Ebola sufferers are tested, the easier it is to contain the outbreak,’ explains project coordinator Edmund Newman of Public Health England, an executive agency of the UK’s Department of Health. ‘MOFINA addresses the challenge of rapid testing and containment during an outbreak by providing a mobile testing capability that can be taken to the patient and followed up with appropriate action based on the result.’ The product is now commercially available. It is anticipated that potential buyers could include public health institutes, diagnostic services, the World Health Organisation (WHO), and non-governmental organisations tackling outbreaks. In the long term, the platform could be expanded to cover more pathogens. The project’s partners aim to develop and manufacture diagnostic tools for each of the WHO’s ‘priority pathogens’ – those viruses and bacteria posing the greatest threat to human health. ‘The consortium is now looking at ways to secure funding to further develop this technology so a wider range of infectious disease can be tested for in the one sample: not just Ebola strains but other similar diseases such as Lassa fever and hantaviruses,’ says Newman.
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EBiSC stem cell biobank keeps growing and could lead to new cures

In recent decades, shape-shifting stem cells have captured the imagination because of their potential to repair all kinds of damaged tissue. Today’s emerging research is exploring how to develop pluripotent cells – the immature form of stem cells – and direct them to grow and differentiate into healthy tissue for just about any organ or body part. IMI’s EBiSC project was set up to meet increasing demand for high-quality, research-grade ‘human-induced pluripotent stem cell’ (hiPSC) lines for targeted diseases – but also healthy ‘disease-free’ lines – as well as the data and services associated with delivering them. The project has been boosted by a so-called ‘hot start’ by securing and validating established cell lines from leading biotech and academic partners. This approach almost instantly yielded 36 hiPSC lines along with the necessary operational, administrative and executive tools and guarantees to reliably use them in the process of developing new therapies. The project has since boosted that number to more than 370 gender-balanced cell lines for some 20 diseases, including Alzheimer’s and Parkinson’s disease as well as metabolic and cardiovascular disorders, all available via its online catalogue. But EBiSC is aiming much higher than that. It eventually aims to process over 1 000 lines once reaching full-scale operations at the end of 2017. The project plans to continue as a sustainable not-for-profit bio-banking operation from 2021 onwards. In future, the international network aims to offer joined-up services such as standardised bio-sample procurement, cell-line creation and in-depth quality control. These may significantly boost the chances of new drug discovery and development, and improve the health and lives of people worldwide.
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