- IMI Scientific Committee experts - How Europe can get better at treating rare diseases
- New vaccine technologies wouldn’t be possible without collaboration – an opinion piece by Pierre Meulien
- Latest COVID-19 vaccine based on technology tested in IMI Ebola projects
- Treatment tracker: IMI COVID-19 therapeutics projects make progress
- Alzheimer’s: visual assessment of PET scans can reveal presence plus extent and location of amyloid
- Are there differences in the brains of autistic men and women?
- We’re living longer. What does that mean for existing vaccine strategies?
- A recipe for the next disaster: a new, pan-virus methodology for ramping up vaccine production
- A quick, cheap and non-invasive way to measure osteoarthritis
IMI Scientific Committee experts - How Europe can get better at treating rare diseases
In June 2020, the IMI Scientific Committee issued a set of recommendations that call for a new way to address problems in rare disease research. In an interview with the IMI Programme Office, two of the authors, patient advocate Claas Röhl and Prof. Olivier Blin of Aix-Marseille University, discuss the issue.
Both agree that patient advocates are essential in rare diseases. ‘Rare disease patients are often completely lost,’ says Röhl, whose daughter has a rare disease. ‘Once a diagnosis is made, they leave the doctor’s office and are completely on their own. The advocacy groups are complementary to the work of the clinics.’
This disconnect not only cuts patients off from real support, it also makes it harder for researchers and others to know what’s really going on with these diseases. In the recommendations, the committee proposes that digital tools – like smartphones, apps, wearables, and online platforms – can go a long way towards bringing patient closer to the science and the decision-making process.
The pair also highlight the need to look at the costs of not treating a patient and how these accumulate over a lifetime. ‘Getting more familiar with the real-life trajectory is really key to determining the burden and the need, and for determining what might be ‘fair’ value of a new treatment or device,’ says Prof. Blin.
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New vaccine technologies wouldn’t be possible without collaboration – an opinion piece by Pierre Meulien
Every few weeks, news of a new COVID-19 vaccine hits the headlines. While the world now mostly focusses on the practicalities of mass distribution and immunisation, it’s useful to take a step back and consider the scale of collaboration that is necessary along the way to producing these vaccines, some of which are based on brand new technologies, writes IMI Executive Director Pierre Meulien in a new opinion piece published on the IMI website.
IMI’s extensive vaccine portfolio has already delivered results in a wide range of areas. For example, The Janssen/Johnson & Johnson COVID-19 vaccine is based on the vaccine technology platform AdVac® that also forms the basis of their Ebola vaccine, which was in turn supported by IMI through the Ebola+ programme.
However, most IMI projects do not focus on product development but on broad challenges in vaccine development. For example, IMI projects are working on correlates of protection (i.e. signs that indicate a vaccine is working), vaccine safety, and vaccines in older people.
‘IMI is an enabler,’ Dr Meulien concludes. ‘Our projects are focussed on areas of common concern to pharma companies who want to work together on a problem that poses difficulties for them all. This doesn’t usually directly translate into new products on the market. Rather, these collaborations are useful for breaking an impasse, allowing the science to move forward.’
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Latest COVID-19 vaccine based on technology tested in IMI Ebola projects
Data on the safety and immunogenicity of the technology behind the Johnson & Johnson COVID-19 vaccine was gathered during IMI-supported clinical studies into the company’s Ebola vaccine.
The new COVID-19 vaccine is based on the company’s unique and proprietary AdVac® technology. Through the EBOVAC projects, IMI funded Phase 1, 2 and 3 clinical studies that generated data on the safety of the AdVac® vaccine platform, providing evidence upon which the European regulator based its decision on the COVID-19 vaccine. The European Commission granted conditional marketing authorisation for the single-dose COVID-19 vaccine on 11 March.
‘The EBOVAC studies have made an important contribution to our global safety database for the platform technology,’ said a Johnson & Johnson representative. ‘Studies specifically supported by EBOVAC played an important role supporting our successful licensure application for the Ebola vaccine with the European Medicines Agency. Additionally, they gave the company confidence to use the AdVac® platform to initiate the development of COVID-19 vaccine.'
Like the Ebola vaccine regimen (which received marketing authorisation last summer), the Johnson & Johnson COVID-19 vaccine is compatible with standard storage and distribution channels, which makes it easy to transport to and deploy in remote areas.
The company is also using the AdVac® platform to develop preventive vaccine candidates for HIV, Zika and RSV (respiratory syncytial virus). Meanwhile IMI projects are continuing to support Ebola vaccination programmes in Africa.
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Treatment tracker: IMI COVID-19 therapeutics projects make progress
IMI’s COVID-19 therapeutics projects, launched in the wake of the pandemic outbreak, are starting to deliver results.
The CARE consortium are trying to identify candidates among existing drugs that could be effective as treatments against COVID-19. In the latest project update, it was revealed that the screening of clinically-approved drugs in a SARS-CoV-2 cell-based assay did not provide a promising drug that can quickly be positioned towards clinical development, although some repurposed compounds are still undergoing pre-clinical proof of concept testing. They have identified advanced antibodies, compared to clinical antibodies, and their full profiling is ongoing. The first in vivo testing has started, with the first in human study to start in the second half of 2021.
IMPENTRI is tasked with repurposing and rapidly developing an existing therapy, imatinib, as a new and improved therapeutic approach for acute respiratory distress syndrome (ARDS) caused by infection with SARS-CoV-2. Recruitment for the first clinical trial, a phase 2 trial of orally-administered imatinib in COVID-19 infected and hospitalised patients before they develop ARDS, has been completed. Recruitment for a second trial is set to commence.
MAD-CoV 2 is working on developing COVID-19 antivirals against SARS-CoV-2 by targeting the ACE2 receptor, the virus’s passageway into human cells. Several state-of-the-art advanced platforms for validation of antivirals against SARS-CoV-2 have been developed and some antivirals using this platform have been validated.
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Alzheimer’s: visual assessment of PET scans can reveal presence plus extent and location of amyloid
Deposits of beta amyloid protein in the brain are a common sign of Alzheimer’s disease. IMI’s AMYPAD project is studying the value of using positron emission tomography (PET) imaging to scan people’s brains for beta amyloid deposits. The ultimate aim is to get better at detecting Alzheimer’s early, as future therapies may be effective in treating the disease.
Doctors can use [18F] Flutemetamol PET scans to spot the presence of amyloid in brain tissue, which helps to make a diagnosis of Alzheimer’s. Going further, however, researchers from AMYPAD wanted to know if a visual read can tell the evaluator more about where the amyloid is, and how much of it there is. The answer, according to the authors, is yes.
The research is published in the European Journal of Nuclear Medicine and Molecular Imaging. The next step will be to validate the results using data from the two ongoing AMYPAD clinical studies and also investigate the possible clinical value of the findings.
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Are there differences in the brains of autistic men and women?
Around three times as many males are diagnosed with autism than females, which suggests that sex-related biological factors play a key role in its development. However, little is known about the ways that autistic males and females differ in terms of brain structure and function.
AIMS-2-TRIALS researchers, in collaboration with colleagues from the Child Mind Institute, explored this further using resting-state functional magnetic resonance imaging (R-fMRI) to study brain function in three large samples of autistic males and females and a control group of non-autistic people. They used one sample for discovery of new information and two additional large samples to see if those findings could be repeated (i.e. replicated).
Across these three samples, the researchers found that both neurotypical males and autistic people showed reduced resting-state brain function in the so-called ‘default network’, a network that is active when we engage in social cognition or thoughts about ourselves. Additionally, in the discovery sample and in one of the largest of the two replication samples, it was shown that connections crossing between the two halves of the brain in the visual cortex are reduced in autistic females, while autistic males are not different from males who are not autistic. The results suggest that many autistic people may have different interactions between the two hemispheres of their brain when compared to non-autistic people. This reflects a combination of effects, including some that appear to be unrelated to sex, and some in which there is an interaction between sex and autism diagnosis. Each of these effects appears specific to a different system in the brain.
Until now, lack of replication of imaging findings has hampered brain imaging research in autism. This study, which included replication analyses, highlights that autistic people may have non-typical interactions in the connections between their brain’s hemispheres. Further, these findings may shed light on the mechanisms underlying sex-differences in autism. The research is published in the journal Molecular Autism.
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We’re living longer. What does that mean for existing vaccine strategies?
IMI vaccine project VITAL is tallying the burden of infectious diseases in ageing adults to figure out the best strategies to keep people healthier for longer. In an interview with the IMI Programme Office, project coordinator Debbie van Baarle of at the University Medical Centre, Groningen sets out the project’s objectives and progress so far.
‘Ageing adults are more exposed to infections than other age-groups except children. Moreover, when they have an infection, they suffer more than others and therefore they may have more difficulties in recovering well promptly,’ she explains. ‘This has been exemplified by the current COVID-19 pandemic.’
The major objective of VITAL is to improve vaccination strategies by understanding better which infectious diseases affect ageing adults the most, why they respond worse to infections and vaccinations, and what strategies could assist in improving heathy ageing in this group.
The project is already making progress. ‘One milestone we already achieved is in obtaining insights into the perspective of older people on vaccination,’ says Professor van Baarle. ‘This will help in communicating about vaccination. Furthermore, we are analysing and comparing vaccination responses between older and younger people to understand the deficits underlying immune protection in elderly.’
Asked how the project will affect the lives of older people, Professor van Baarle is clear. ‘If the project is successful, we will have a coordinated and efficient vaccination programme for ageing adults akin to what exists today for children,’ she explains. ‘The final goal is to have a more efficient and sustainable preventative programme in place that can ensure healthy ageing in European adults.’
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A recipe for the next disaster: a new, pan-virus methodology for ramping up vaccine production
One way of dealing with the threat of another pandemic like COVID-19 is investing in plug-and-play vaccine technologies that can respond to a wide variety of virus types. IMI’s ZAPI project has created a new methodology for quickly designing and manufacturing a vaccine in the event of a new disease outbreak in humans or animals that consists of the assembly of only two components. The only variable is the immunogen, which will change depending on the target pathogen.
Manufacture of a so-called ‘ZAPI-like vaccine’ can take place anywhere. That’s because a guiding objective in the design of the manufacturing process was to keep it very simple by limiting the number of steps involved. Having multiple manufacturing sites is one of the key factors in responding as quickly as possible in the event of a new pandemic as it enables to expand production capacity close to the needs everywhere in the world.
‘We have demonstrated up to a significant scale that this vaccine approach can be readily and rapidly industrialised to supply the large number of vaccine doses which are needed,’ explains ZAPI coordinator Jean-Christophe Audonnet. ‘This is something that everyone can appreciate when you see that SARS-CoV-2 vaccine manufacturers are suffering from shortages or significant capacity reductions compared to the amounts that had been promised initially in the vaccine supply agreements.’
ZAPI-based vaccines are currently being developed for SARS-CoV-2. 'Chinese, UK and US teams are actively working with this MPSP display-based vaccines, most of them using exactly the same MPSP [multimeric protein scaffold particle] as in the ZAPI project, coupled to SARS-CoV-2 RBD subunit,' concludes Dr Audonnet.
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A quick, cheap and non-invasive way to measure osteoarthritis
The APPROACH project has found that a commercially-available motion-measuring technology could be used to gauge the extent of osteoarthritis as an alternative or add-on to existing diagnostic options.
There are a number of ways that osteoarthritis (OA) progression can be measured, including x-rays, MRIs, and questionnaires, but all come with drawbacks. APPROACH studied whether a commercially-available technology called GaitSmart® could offer added value in the evaluation of knee osteoarthritis as a non-invasive, inexpensive and flexible alternative or add-on to help doctors make a diagnosis. The GaitSmart® system involves attaching six sensors to a person’s body and having them walk 15-20m at their own speed. The sensors feed data to a laptop which analyses the results. The whole process takes about 10-15 minutes and is easy to transport and set up.
GaitSmart® was found to measure different features of OA as well as offer more information than what can be gathered through a survey. It was also found that GaitSmart® can offer additional information on the severity of tissue damage as seen on conventional x-rays.
According to the project, GaitSmart® could ultimately be used in a primary care setting, where a patient presenting with suspected knee osteoarthritis could have the test carried out by a healthcare assistant, with a report made available to the GP prior to consultation. The system also allows patients to be more invested in their progress as the changes in the report are easy to identify.
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