53rd Edition – January 2016

EUPATI launches educational toolbox on medicines R&D

IMI’s patient education project EUPATI has launched an online educational toolbox on medicines research and development (R&D). Designed for patients, patient groups, and anyone interested in learning more about medicines R&D, the freely-accessible toolbox includes materials covering drug discovery, clinical development, regulatory affairs, medicines safety, pharmacovigilance, and health technology assessment. The toolbox is available in seven languages: English, French, German, Spanish, Italian, Polish and Russian. It was designed by patients in collaboration with experts from other stakeholder groups such as academia and industry. Birgit Bauer, who has multiple sclerosis and recently graduated from the EUPATI Patient Expert course, welcomed the publication of the new website. ‘From my perspective, the EUPATI toolbox provides an objective, reliable and up-to-date resource for patient education on medicines R&D,’ she said. ‘The multilingual materials deliver a wealth of patient awareness materials and an understanding on the opportunities patient advocates have to strengthen their voice and to get actively involved in the process.’ The need for the new toolbox is backed up by recent EUPATI research which reveals that pharmaceutical industry personnel believe patients can improve medicines research and development. The study is published in the journal BMJ Open.

• Watch a video about the new toolbox.

EU-AIMS paves way for clinical studies on autism

The approach of the EU-AIMS project to carrying out clinical studies on autism and assessing the effectiveness of new treatments has gained the support of the European Medicines Agency. This development, announced in an article in Nature Reviews Drug Discovery, will facilitate the development of new treatments for autism spectrum disorders (ASD). EU-AIMS is running a Europe-wide study of people with autism, in which participants undergo genetic tests, brain scans, cognitive tests and clinical assessments to identify biological markers that could be associated with different subtypes of autism. The project has been working with regulators for some time to ensure the results of their studies will be accepted as reliable by regulators. ‘It’s critical for researchers to engage with the drug regulators early on, to figure out exactly what will be required to prove that a treatment is safe and effective for persons with autism,’ said Paul Wang of project partner Autism Speaks. ‘The new publication shows how the EU-AIMS group has succeeded in starting this dialogue, and it represents a key step toward translating autism science into autism treatment.’

• Read the Autism Speaks press release
• Read the Roche press release

DDMoRe platform to help scientists share info

The IMI project DDMoRe has launched a new, open source platform that allows researchers to simply and reliably share drug and disease models with one another. Scientists often use computer-based tools to simulate both diseases and the impacts of drugs on them. DDMoRe’s new interoperability framework uses new standards and connectors for many commonly-used programming tools and languages. As such, the interoperability framework provides a consistent environment in which researchers can work, using modelling tools they know, while opening up their work to others through a standard language for describing models. The framework is free to download and the project is keen to get feedback from users. ‘We are excited to see how new opportunities for collaboration will develop now that this platform is available for stakeholders such as academia, industry and health authorities,’ commented DDMoRe Scientific Officer Wendy Aartsen.‘ We are very keen to involve anyone with an interest in the use or further development of the platform.’ To complement this release, the DDMoRe repository enables access to curated and shared knowledge for the benefit of model-informed drug discovery, development (MID3) and therapeutic use. As of today, more than 60 models are available to the public; all are free to download and ready to be used.

PREDECT takes lab-based cancer models to the next level

IMI’s PREDECT project is making progress on its goal of improving lab-based cancer models to make them better, more reliable tools for use in medical research and drug development. Studying cancer in the laboratory is challenging; tumour samples in petri dishes simply do not behave in the same way as tumours in the body. PREDECT is working to improve these over-simplistic models by designing complex three-dimensional models that also include cells from the body’s connective tissues which interact with the tumour. Analyses of the models reveal that these models’ behaviour is much closer to that of cancer in the body. Scientists in the project are now using these models in their research. Another group of researchers in the project is looking at improving ways of studying slices of tumours in the laboratory. Tumour slices provide a lot of information on the architecture and make-up of complex tumours. However, the act of creating a slice and keeping it alive can affect the cells’ behaviour. Scientists from PREDECT have found ways to get round some of these issues to create samples that give more reliable results. The models are all the result of collaboration between scientists from small companies, universities, and large pharmaceutical companies. Details of the models are published in open access papers. The new models offer a number of advantages. Most notably, their complexity means scientists will be able to study tumours in the lab in unprecedented detail, and this will reduce their reliance on animal models. ‘There is a growing wave of concern that an over-dependence on reductionist models is one factor contributing to limited success in drug discovery in all therapeutic areas,’ commented project coordinator John Hickman of Servier. ‘To go to more complex systems that model pathology usually means using more animals. This is also expensive. We are attempting some compromise solutions: complex in vitro models.’

• Details of the papers can be found on the PREDECT website

QuIC-ConCePT scientists make recommendations on cancer clinical trials

An international team of experts, including scientists from IMI cancer project QuIC-ConCePT, have put forward a framework to improve the way imaging is used in clinical trials of cancer drugs. Writing in The Lancet Oncology, the team explains that improvements in imaging methods are making them increasingly popular in clinical trials of cancer drugs. Used well, imaging can show whether or not a patient is responding to treatment with days of the treatment starting. However, there are issues with the use of imaging in clinical trials; for example, the quality of data derived from imaging is variable. The researchers propose a practical, risk-based framework and recommendations on the use of imaging as a marker of how well a treatment is working. They recommend carrying out a risk assessment plan before the study starts, and reviewing and updating the risk plan throughout the trial. ‘In cancer clinical trials, we are always trying to strike the right balance between maximising data quality and minimising cost. Here, risk management can be an extremely helpful tool, because it can help us to prioritise, reduce costs, and decrease attrition rates,’ said the lead author of the paper, Dr Yan Liu of the EORTC. ‘In our study, we used a quality risk management approach to help us outline a consensus framework for imaging biomarker driven trials. This approach recognises that other stakeholders such as regulatory bodies, pharmaceutical companies, and patients also play essential roles in the conduct of these trials.’

EBOVAC2 clinical trials underway in France

IMI Ebola project EBOVAC2 has launched a campaign in France to recruit around 300 volunteers for a trial Ebola vaccine regimen. The goal of this study is to assess the safety and efficacy of a novel prime boost preventive regimen against Ebola virus disease. The vaccine regimen under investigation has two parts – a ‘prime’ vaccine to stimulate the immune system and a ‘boost’ vaccine to strengthen and extend the immune response. Additional volunteers are being recruited in the UK and in Africa. ‘Participants in this trial cannot become infected with the Ebola virus,' said EBOVAC2 project coordinator Rodolphe Thiébaut of INSERM. ‘Only synthetic proteins or parts of proteins are used in the various vaccines being tested. They cannot in any way cause infection. This is based on the same principle as many existing vaccines for infectious diseases.’

• Read the INSERM press release on EBOVAC2.