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The largest research grant ever given for neurodevelopmental conditions has been awarded by the Innovative Medicines Initiative to an international consortium academically led by the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King’s College London.

The €115 million grant, titled Autism Innovative Medicine Studies-2-Trials (AIMS-2-Trials), will increase our understanding of autism and help develop new therapies to improve health outcomes and quality of life for autistic people.

More than 1 in 100 people are autistic. In addition to the core features of autism, many autistic people struggle with co-occurring conditions such as epilepsy, anxiety and depression, and life expectancy for autistic people can be reduced by up to 30 years.  However, the causes of autism and its associated difficulties remain largely unknown and there are very few effective and autism-appropriate therapies.

The Oxford Translational Molecular Neuroscience Group will undertake the stem cell disease modelling for this consortium to understand gene/environment interactions in autism. The goal is to identify biomarkers and targets that may enable stratified clinical trials and personalised medicine in autism.
- Associate Professor Zameel Cader, Nuffield Department of Clinical Neurosciences

AIMS-2-Trials brings together autistic people and their families, academic institutions, charities and pharmaceutical companies to study autism and provide an infrastructure for developing and testing new therapies. In line with the autism community’s priorities, the consortium will also focus on why some autistic people develop additional health problems that severely impact both quality and length of life.

Professor Declan Murphy, the project academic lead and Director of the Sackler Institute for Translational Neurodevelopment at the IoPPN at King’s says: ‘Many autistic people face extremely poor health outcomes, yet autism research receives far less investment than other conditions which also limit life expectancy and quality of life, such as cancer or dementia. This grant will allow us to bridge the gap between basic biology and the clinic by offering personalised approaches that address problems which really impact autistic people’s lives.’

All autistic people are different which makes identifying and testing new therapies challenging. AIMS-2-Trials will take a precision medicine approach aimed at tailoring therapies to a person’s biological profiles. Achieving this will require developing tests that can predict how a person’s autism may progress throughout development and their likelihood of developing additional mental health problems.

AIMS-2-Trials will create the first European clinical trials network for autism, as well as allowing for international collaborations with charities, government agencies and industry to rapidly determine if therapies are effective. Partnership with autistic people, their families and carers will be a crucial part of developing therapies that achieve the outcomes which matter most to autistic people.

Dr James Cusack, Director of Science at Autistica, a UK charity supporting AIMS-2-Trials, says: ‘Autistic people deserve an equal right to a long, healthy happy life.  To deliver on that vision, we're working together to understand why autistic people are different from each other. We're excited to be joining this consortium to ensure that the views of autistic people and their families are considered throughout the project.’

Professor Louis Reichardt, Director of the Simons Foundation Autism Research Initiative (SFARI) says: ‘The development of new and effective therapies for autism is a daunting task that requires the talents, efforts, and resources of public, private, and non-profit sectors. The scientists at SFARI will be working with AIMS-2-Trials investigators on many of the project's initiatives, and we look forward to the project's success.’

Through the Innovative Medicines Initiative, European Union funding matches in-kind contributions from autism charities and the pharmaceutical industry, with nearly €60 million provided by charities, and €2.5 million from the European Federation of Pharmaceutical Industries and Associations (EFPIA).

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 777394. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme, EFPIA, Simons Foundation, Autism Speaks, and Autistica. 

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