- IMI welcomes proposal to launch Innovative Health Initiative
- IMI launches new projects on artificial intelligence, advanced therapies and cancer
- Nature Microbiology letter puts spotlight on IMI's Ebola and COVID projects
- Treating rare diseases: can new business models solve equity of access issues? An opinion piece by Pierre Meulien
- Lab results show novel drug combo fights SARS-COV-2 on two fronts
- Algorithmic advances, triage models and getting the ethical all-clear: IMI COVID-19 research efforts advance
- Neuronet Knowledge Base showcases 18 IMI neurodegeneration projects
- TRIC-TB starts clinical trial of anti-TB drug booster
- Viruses can be hijacked to help cure rare diseases, but the immune system keeps fighting back
- Regulators clear study of gut microbiome ‘protector’ in cancer patients
- Hope that a cancer drug can stop FOP, one of the rarest diseases in the world
- Mobilise-D and IDEA-FAST team up on digital health technologies
IMI welcomes proposal to launch Innovative Health Initiative
IMI has welcomed the proposal to launch a new public-private partnership (PPP) for health under Horizon Europe, and hopes that the Innovative Health Initiative (IHI) will build on the successes and lessons learnt from IMI.
IHI is set to be a true cross-sectoral partnership involving COCIR, EFPIA, EuropaBio, Medtech Europe and Vaccines Europe as full partners. Their input, combined with the support of the European Commission, will allow IHI to focus on preventive strategies as well as early diagnostics, treatments and care, in order to contribute to a holistic, citizen-centric model of health care that is fit for purpose.
‘Part of IMI’s added value lies in our ability to act as a neutral platform where all the stakeholders involved in health research and health care, including patients, smaller companies and regulators, can come together to collaborate on major challenges,’ said IMI Executive Director Pierre Meulien. ‘IHI will keep this ‘neutral platform’ approach, and we are confident that this will ensure that IHI makes a meaningful contribution to achieving the goals of Horizon Europe, including the cancer mission, while also strengthening the European Research Area (ERA).’
Looking to the future, we hope that the new partnership will build on what has worked well in IMI, learn from the difficulties we have faced, and most importantly, continue to deliver high quality, impactful results that will advance research and ultimately, improve patients’ lives.
- Read IMI’s statement
- Read the European Commission's press release
- Read the joint statement by COCIR, EFPIA, EuropaBio, MedTech Europe and Vaccines Europe
IMI launches new projects on artificial intelligence, advanced therapies and cancer
In the last few weeks, three new IMI projects have got underway, all of them from IMI2 – Call 18.
BIGPICTURE – Many diseases are still diagnosed, monitored and studied in part on the basis of biological samples that have been mounted on a glass slide and studied under a microscope. Today, that analysis is still carried out by a human pathologist, but what if the slides could be scanned and analysed using artificial intelligence (AI)? And what if they could be placed in a repository and made available to the scientific community? These are the questions the BIGPICTURE project hopes to answer. The project aims to create a repository of digital copies of around 3 million slides covering a range of disease areas. This repository will then be used to develop artificial intelligence tools that could aid in the analysis of slides.
SISAQOL-IMI – In drug development, it is important to find out how treatments affect how patients feel and function in their daily lives. This information is also essential when weighing up the benefits and risks of a medicine. In practice, it is rather difficult to obtain this information and communicate it clearly and simply. The aim of SISAQOL-IMI is to develop recommendations on how to analyse and interpret data on health-related quality of life (HRQOL) and patient reported outcomes (PROs) in cancer clinical trials.
T2EVOLVE – T cells are an important part of the immune system, and in recent years scientists have succeeded in creating ‘engineered’ T cells designed specifically to seek out and destroy cancer cells. Scientists are working on a number of cancer treatments based on T cells, but their efforts are hampered by a number of challenges. The aim of T2EVOLVE is to develop an innovation ecosystem that will accelerate the development of engineered T cell therapies in the EU.
Nature Microbiology letter puts spotlight on IMI's Ebola and COVID projects
In a newly published letter in the journal Nature Microbiology, IMI Executive Director Pierre Meulien sets out how IMI’s Ebola+ programme has delivered concrete results, including significant contributions to the development of vaccines that have since received regulatory approval, and the development of rapid diagnostic tests. Furthermore, the experience of rapidly launching the first Ebola projects helped IMI to quickly launch projects on treatments and diagnostic tests for COVID-19, Dr Meulien adds. Meanwhile, many other IMI projects have applied their knowledge, skills and resources to tackling the COVID-19 outbreak.
‘Thanks to the long-term investments of a wide range of funders, the world is now much better placed to prevent and deal with an Ebola outbreak,’ Dr Meulien concludes. ‘Moreover, the experience of responding to the Ebola outbreak has helped to shape our response to the current coronavirus pandemic.’
The letter is particularly timely given the recent announcement of a new Ebola outbreak in Guinea in west Africa.
Find out more
- Read the letter in full
Treating rare diseases: can new business models solve equity of access issues? An opinion piece by Pierre Meulien
From setting standards for genetic newborn screening to helping pharma create a shared platform to treat thousands of rare diseases, IMI is ready to help drive the science forward on rare diseases, writes IMI Executive Director Pierre Meulien in an opinion piece published on the IMI website.
A rare disease, by definition, affects relatively few people. But taken collectively, they affect millions of patients in the EU alone. Defective genes cause the majority of these diseases, and gene therapy in particular has offered a lot of hope to patients and their families in their search for cures. However, developing gene therapies is far from easy. In 2020, IMI launched the ARDAT project with the aim of tackling some of the key challenges in the development of gene therapies, with a focus on rare diseases caused by a single mutation.
IMI also has a project on fibrodysplasia ossificans progressiva (FOP), one of the rarest diseases in the world. FOP causes a defect in a gene related to bone metabolism. ‘What I find very interesting is that this gene is implicated in many other more common diseases, including certain cardiac malformations, cancers of the nervous system and Parkinson’s disease,’ writes Dr Meulien. ‘What I would like to know is: what can we learn about the link between those specific genes and other more common diseases? I suspect that our understanding of this link would be extremely beneficial, and it’s exactly the kind of pre-completive research question that IMI specialises in.’
Finally, IMI is finalising a project on genetic newborn screening, which should get underway this year.
‘Science has tended to neglect diseases that affect small numbers of people, but that has been changing,’ concludes Dr Meulien. ‘Europe has really been a leader in this area, and IMI stands ready to help drive the science forward.’
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Lab results show novel drug combo fights SARS-COV-2 on two fronts
Scientists from IMI’s MAD-COV 2 project have shown that a low dose combination of the antiviral remdesivir and a drug called APN01 (hrsACE2) can stop the SARS-CoV-2 virus which causes COVID-19 from multiplying in cells.
Remdesivir is an antiviral that has been approved for use in COVID-19 patients. It works by stopping the virus from multiplying. However, higher doses can cause damage to the liver and the lungs. ‘Human recombinant soluble ACE2’, also known as hrsACE2, is a genetically modified variant of the angiotensin converting enzyme 2 (ACE2), a cell membrane protein that the coronavirus hijacks in order to enter our cells.
Previous laboratory studies have shown that hrsACE2 lures the coronavirus to attach itself to the enzyme copy, hrsACE2, instead of to the actual cells, thereby reducing the viral load in cells. In this study, the researchers tested combining remdesivir and hrsACE2 in cell cultures from monkeys, human liver spheroids and human 3D kidney replicas, so-called organoids grown from stem cells. The use of low doses of each substance was able to reduce toxicity, making the drugs safer to use.
By targeting different aspects of the viral cycle simultaneously, the researchers have shown that effectiveness of the treatment can be boosted while reducing the risk of side-effects. They hope their findings will pave the way for clinical trials.
The study was published in the journal EMBO Molecular Medicine.
Find out more
- Read the article in full
Algorithmic advances, triage models and getting the ethical all-clear: IMI COVID-19 research efforts advance
Algorithmic advances, triage models and getting the ethical all-clear: the diagnostic projects launched by IMI in 2020 are making steady progress.
COVID-RED is working on developing a wearable device that can be used for remote early detection of SARS-CoV2-infections. Findings from the project’s feasibility study have proven very encouraging; the team have produced an algorithm capable of indicating potential Covid-19 infection more rapidly than the current symptom-based system. The team have also identified participants and sites for a large study that will include 20 000 individuals. They are currently waiting for approval from local ethical authorities before starting recruitment.
DECISION is working on a miniaturised, disposable molecular point-of-care diagnostic device. The electronic base of their small instrument has been successfully designed and a micro controller mainboard and the firmware developed. They are currently working on optimising the workflow, and the development of a compatible chemical/enzymatic, thermal lysis.
DRAGON is developing diagnostic and prognostic models based on imaging, combined with molecular profiling by AI-enhanced analysis deployed by federated machine learning networks. The team have created a model for triage of COVID-19 patients, a platform for predictive models which enables doctors to supplement their judgment with patient-specific predictions, and supports researchers in showcasing their work.
RAPID-COVID is working on high-throughput automation and point-of-care identification of COVID for a diagnostic test that can detect SARS-CoV-2 as well as 30 other common respiratory bacteria and viruses, to ensure patients are quickly isolated and that all patients receive the right treatment. Preliminary testing of the point-of-care prototype instrument started in December 2020 on known samples, with clinical testing due to start in March 2021. The project is making good progress in optimising the robotic system to be used for the clinical study.
Find out more
- Read the article in full
Neuronet Knowledge Base showcases 18 IMI neurodegeneration projects
IMI’s Neuronet project has launched a Knowledge Base which brings together in one place information on 18 IMI neurodegeneration projects. The comprehensive resource is an integral part of Neuronet’s endeavour to boost collaboration across the research portfolio by identifying gaps, multiplying the portfolio’s impact, and enhancing its visibility.
As well as providing an overview of the IMI neurodegeneration research programme through its interactive dashboard, the Knowledge Base acts as a one-stop shop to explore the diverse projects and outputs of the programme. In addition to links to over 380 publications and 350 publishable deliverable reports, the Knowledge Base includes an ‘asset map’ showing the different assets resulting from the projects, such as genetic datasets, clinical cohorts, and data platforms.
Furthermore, the Knowledge Base offers access to a regulatory, health technology assessment & payer engagement Decision Tool to help researchers identify the key processes and procedures for engagement with these stakeholders at key points in the development of an asset.
‘The Neuronet Knowledge Base tool intends to bring the “better together” team concept to life and overcome the inherent fragmentation often found in a project-based research landscape,’ said Neuronet coordinator Carlos Díaz of SYNAPSE.
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TRIC-TB starts clinical trial of anti-TB drug booster
The TRIC-TB project has started a clinical trial with BVL-GSK098, a drug that is designed to boost the infection-fighting ability of the tuberculosis (TB) drugs ethionamide (Eto) and prothionamide (Pto). Although Eto and Pto are potent weapons in the fight against TB, they can cause severe side effects. If approved for use, BVL-GSK098 could allow doctors to reduce the efficacious dose of Eto or Pto, and along with it the side effects they cause. Importantly, BVL-GSK098 has shown in preclinical studies to overcome pre-existing resistance to Eto and is active against multi-drug resistant (MDR)-TB.
Last year, the US Food and Drug Administration (FDA) gave Qualified Infectious Disease Product (QIDP) designation to BVL-GSK098 in a fixed combination with Eto, a move that incentivises clinical development with faster development times, by allowing for fast track and priority review designations, and offers 5 years of additional market exclusivity in the USA.
Now, BioVersys, the Swiss SME behind the drug, has announced the start of a Phase 1 clinical trial of BVL-GSK098 in collaboration with GSK. The trial, which involves healthy volunteers, aims to assess the safety and tolerability of BVL-GSK098 as well as its behaviour in the body.
‘More than 1.5 million people die every year from tuberculosis through a lack of efficacious treatments and access to medicines,’ said BioVersys CEO and co-founder Dr Marc Gitzinger. ‘At BioVersys we remain committed to developing innovative and life-saving treatments for patients suffering from drug-resistant infections, and the combination of BVL-GSK098 and Eto or Pto has the potential to improve patient outcomes, reduce treatment times, and even replace isoniazid in first-line TB therapy.’
‘GSK is committed to the discovery of novel treatments for tuberculosis including the drug-resistant forms of Mycobacterium tuberculosis,’ said Dr David Barros-Aguirre, VP and Head of Global Health Pharma Research Unit, Global Health Pharma R&D, GSK. ‘Entering clinical trials is an important milestone in our successful collaboration with BioVersys as we develop BVL-GSK098 within the IMI2 TRIC-TB programme, towards a potential treatment to optimise the beneficial effects of ethionamide.’
Find out more
- Read the BioVersys press release
- Visit the TRIC-TB web page on the AMR Accelerator website
- Visit the BVL-GSK098 web page on the website of the Working Group on New TB Drugs
Viruses can be hijacked to help cure rare diseases, but the immune system keeps fighting back
Advanced therapy medicinal products (ATMPs) such as cell and gene therapies could overhaul the way we treat genetic diseases, but it’s still extremely difficult to predict who will respond well to treatment and who will suffer serious side effects. IMI’s ARDAT project aims to answer some of the biggest questions surrounding ATMPs in a bid to get us closer to new treatments for rare diseases.
In an interview with the IMI Programme Office, the project leaders explain the many scientific and regulatory challenges facing the project, and how they plan to address them. If successful, the project could help to change the lives of people living with rare diseases.
‘The goal of gene and cell therapy is to provide, with a single treatment, sustained levels of therapeutic gene expression or cell activity, with potentially lifelong duration,’ they said. ‘For rare genetic diseases, this can provide a chance for patients to have a cure for their severe, debilitating disease state. It could free them from weekly intravenous infusions of clotting factor, free them from being confined to a wheelchair, or it could allow others to live who would otherwise would succumb to their disease.’
Find out more
- Read the interview in full
Regulators clear study of gut microbiome ‘protector’ in cancer patients
COMBACTE-NET partner Da Volterra has been given a green light to move forward with a phase 3 trial of an innovative product to protect the microbiome of cancer patients from antibiotic-induced disruption.
The microbiome of cancer patients is constantly assaulted by numerous prescribed drugs, in particular life-saving antibiotics. The consequences of this include a higher risk of picking up infections, lower efficacy of anti-cancer treatments, and even potentially lower survival due to interference with the immune system. There is now hope for change.
In a world first, French biotech Da Volterra, a partner in the COMBACTE-NET project, has been granted authorisation to proceed to phase 3 trial to get evidence on the efficacy and safety of its product, DAV132, in patients with hematologic malignancies (cancers of the blood, bone marrow and lymph nodes). The product, they claim, will protect the microbiome of these patients in spite of massive antibiotic use.
The MICROCARE study will enrol 900 patients mainly in Europe and the USA. The objective is to show that DAV132 contributes to decreasing the occurrence of life-threatening complications of hematologic malignancies in people who are undergoing chemotherapy.
The study is a major milestone. It is a testament to the capacity of the quality clinical and laboratory research network set up by COMBACTE-NET and its ability to successfully conduct the first-ever phase 3 study of the project, thus facilitating market access of novel products to combat life-threatening conditions and antimicrobial resistance (AMR).
Find out more
- Read the article in full
Hope that a cancer drug can stop FOP, one of the rarest diseases in the world
STOPFOP is trialling the cancer drug saracatinib, which has shown promise in treating people with fibrodyplasia ossificans progressiva (FOP), a rare and devastating disease in which muscles, tendons and ligaments slowly turn to bone. People with FOP gradually get ‘locked in’ their bodies, making it difficult to move and breathe. It’s caused by a mutation in a single gene, called ACVR1, which encodes for the protein kinase ALK2. The mutation causes ALK2 to become overactive, causing muscles and connective tissues turn into bone. There is no drug to treat this disease.
STOPFOP was launched to trial an experimental drug from AstraZeneca called saracatinib, originally developed as a cancer drug. The project team has shown that it blocks the activity of ALK2 kinase in mice, preventing ectopic bone formation and keeping the joints mobile. We know enough about the drug’s safety and efficacy thanks to trials in humans with cancer as well as healthy volunteers, so researchers are now proceeding to trial the drug on people suffering from FOP.
The drug is taken orally, as a once-daily dose. The first patient is now progressing through the STOPFOP study, with the randomised control trial period due to be completed within six months. Success will be measured on the ability of saracatinib to show less increase in bone formation outside the skeleton.
‘We hope to stop the formation of bone, which will hopefully preserve the movement they have,’ says Marelise Eekhoff of Amsterdam UMC, referring to the patients enrolled in the trial. ‘A 100% effect given the complexity of the clinical picture is almost impossible. We have given the definition of a success at least more than 50% reduction in bone formation outside the skeleton compared to controls. But in this disease even any effect would be useful.’
Find out more
- Read the article in full
Mobilise-D and IDEA-FAST team up on digital health technologies
Mobilise-D and IDEA-FAST, two IMI projects that aim to develop real-world digital outcomes, have announced their plan to collaborate, to bring synergy and increase the impact and benefits of the digital health technologies developed by both projects. The collaboration will exploit common themes and approaches across the projects, to develop digital biomarkers in mobility, sleep and fatigue, including large observational studies involving diverse patient cohorts. The topics of collaboration include areas such as ethics, principles of external data-sharing, regulatory approaches, dissemination activities, training and education, and working with stakeholders.
Professors Lynn Rochester and Wan-Fai Ng from Newcastle University, the coordinators of Mobilise-D and IDEA-FAST respectively, state: ‘We are delighted with this collaboration which cements the intended relationship seen from the inception of the projects, and look forward to the extended impact and reach possible by formalising our ongoing collaboration.’
For their part, the projects’ EFPIA leads, Dr Ronenn Roubenoff (Novartis, Mobilise-D) and Dr Frederic Baribaud (Janssen, IDEA-FAST) identify the future benefits of the inclusion of digital biomarker approach in clinical trials to develop and evaluate pharmaceutical interventions.
Professor Walter Maetzler, who participates in both projects commented: ‘What could be more advantageous than to consistently combine the strengths of both projects and thus increase the impact for patients and medicine?’
Mobilise-D focuses on mobility assessment in chronic obstructive pulmonary disease, Parkinson’s disease, multiple sclerosis and proximal femoral fracture and IDEA-FAST on the assessment of fatigue, sleep disturbances and instrumental activities in neurodegenerative disorders (Parkinson’s disease, Huntington’s disease) and immune-mediated inflammatory diseases (inflammatory bowel diseases, lupus, rheumatoid arthritis, Sjogren’s syndrome). Both projects aim to develop and validate digital biomarkers towards regulatory approvals.
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