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European consortium strives to spur autism drug development

by  /  21 May 2012

Cohesive unit: A unique partnership brings together researchers from different European countries, in different phases of research, and from both academia and the pharmaceutical industry.

A $38.7 million project in the European Union — the largest single grant for autism research in the world — aims to bring together academic labs and pharmaceutical companies, including Roche, Eli Lilly and Pfizer, to speed the move from basic to clinical research.

The effort, called European Autism Interventions – A Multicentre Study for Developing New Medications, or EU-AIMS, focuseson improving translational research, including developing stem cells, animal models and a European network for running clinical trials. Launched in April, it is led by Roche and researchers at King’s College London.

Half the funding for the five-year project comes from the Innovative Medicines Initiative, a European public-private initiative designed to help develop better and safer medicines, and half from the European Federation of Pharmaceutical Industry Associations. Autism Speaks, a U.S.-based research and advocacy organization that contributed $1 million to the project, is another collaborator.

“I think it’s a great first effort and will dramatically contribute to the understanding, treatment and de-stigmatization of autism in Europe,” says Randall Carpenter, president of Seaside Therapeutics, a Massachusetts-based biotech company testing drugs for autism and fragile X syndrome, and head of the grant review committee. “They have been visionary in their thought process and tried to integrate and learn from everyone else who has tried these projects.”

For example, the researchers plan to make the resources that emerge from the project, such as databases and a collection of biological samples, able to link with existing autism repositories. 

The products of the project are also considered ‘pre-competitive,’ meaning the information can be shared freely. “Pre-competitive applies to areas where the field is young and needs to progress to a critical point where companies can invest in proprietary research,” says Santarelli.

Trial training:

A central thrust of the EU-AIMS project is to create a network of centers of expertise for the various components of autism research.

“We want to develop better animal models, better understanding of the biology of disease, and better clinical scales and endpoints,” says Santarelli. “If you do that in isolation, you have less power than doing it as a team.”

One group aims to generate induced pluripotent stem (iPS) cells from people with autism to try to identify cellular abnormalities, and to develop new technology for drug discovery assays.

Researchers specializing in animal models plan to generate new strains that robustly show the core symptoms of autism: social deficits and repetitive and restricted behaviors. They also aim to develop reproducible behavioral assays and anatomical and functional biomarkers, such as differences in brain structure or behavior, that can ultimately be used to test drugs. 

Part of the translational effort will be to develop new molecular imaging techniques that can be used across different species.

“We want to put everything together and try to come up with a holistic view,” says Declan Murphy, professor of neurodevelopmental sciences at King’s College London, and the academic lead on the effort.

For example, he says, the researchers don’t just want to measure glutamate and GABA, two chemical messengers in the brain, in live animals. They instead hope to understand how these markers relate to autism-linked mutations and how they are modulated by different drugs.

On the clinical side, researchers plan to work closely with families, for example by studying infants who have siblings with autism and are therefore at higher-than-average risk of developing the disorder. They plan to follow approximately 300 of these so-called ‘baby sibs’, as well as 500 children with autism, in search of early biomarkers that can predict autism, and to assess whether those biomarkers change over time. 

“What they are doing is very comprehensive and commendable in terms of trying to integrate investigators and bringing in pharma to the table,” says Antonio Hardan, assistant professor of psychiatry and behavioral science at Stanford University in California, who is not involved in the project. Forty million dollars, he points out, “is a great catalyzer.”

One of the most important aspects of the project is the development of six clinical centers — with more planned — across Europe. “If we identify new treatments, we can roll them out to clinical populations immediately,” says Murphy.

The researchers also plan to work with Europe’s drug regulatory body, the European Medicines Agency, to determine the best outcome measures.

For a disorder as diverse as autism, it’s especially important to have access to large numbers of people for clinical trials. Most clinical trials in autism have been comparatively small, with 50 or fewer participants. That makes it difficult to identify subgroups that might respond to the treatment, says Hardan.

Given that the effort spans at least seven different countries and intends to include individuals who speak as many languages, the researchers face the unique challenge of developing and validating tools that work across the various languages and cultures. 

The U.S. has its own autism clinical trials network, known as the Research Units on Pediatric Psychopharmacology Autism Network, but it has few sites and does not incorporate a basic science component.

“No other effort [in the autism field] is bringing basic science and clinical trials under one umbrella,” says Hardan. “These countries have done it before politically, so they can probably [work] together here also.”


2 responses to “European consortium strives to spur autism drug development”

  1. DrLou1 says:

    I certainly can’t disagree with the reported statement that “forty million dollars a great catalyzer.” I would add, however, that with half of the amount coming from Pharmaceutical Industry Associations, this clinical research project will likely be less than comprehensive while more pursuing a singular agenda/outcome once again.

    Another way to view this 38 million dollar project is that it will further nurture the ongoing big money misdirection to authentic treatment, intervention, support and individual understanding of persons with autism and their families. That we first need to know each individual with ASD; their lives, family and world – one at a time – remains overlooked

    That ASD is a remarkably heterogeneous disorder and far more likely representative of an umbrella like category rather than a targeted genetic and/or neurological process is a first challenge to address before any attempt to ‘cure’ and, even more so, to believe it even possible – let alone ethical – to make in vitro ‘corrections.’ .

    That autism becomes integrated into the being; the individualism and strengths of so very many of those persons with this ‘diagnosis’ also means that the continued presumption that there could be a ‘unifying’ drug treatment or category for treatment..about any treatment, for that matter…already brings a substantial flaw and challenge to such a project’s internal validity…let alone any subsequently presumed external validity.

    In fact, the project’s title; ‘European Autism Interventions – A Multicentre Study for Developing New Medications’ indicates a massive endorsement of pharmaceutical companies who are already far too deeply entrenched in the autism ‘treatment’ model.

    Despite some ongoing and worthwhile clinical efforts and theory, we still don’t have stable data as to a much more likely ‘categorical’ presentation of ASD.

    We don’t know the degree to which tiny sibs of those older with an ASD diagnosis who are, themselves, starting to exhibit ASD characteristics are the result of genetics, social replication of their older sibs behavioral patterns or a blend of both.

    We’ve not taken seriously the profusion of more often pseudoscientific autism ‘treatments’ which, though being used endlessly in many places, never seem to actually generate the related and hypothesized outcomes while taking time away from what we know really can help.

    And we’ve also still not more comprehensively studied the impact of demographics on purported autism prevalence and how this ‘diagnosis’ is often given. It is these huge holes, among others, which need to be resolved before investing $38 mill towards some kind of ‘unifying’ neurological theory and/or drug treatment for those given an ASD diagnosis.

    The use of highly targeted and carefully titrated medications blended with instructional/behavioral best practice across some categories of autism may ultimately be found productive. But we still know far too little about the impact of medication on ASD; on global processing disorders even as the prescriptions continue to be written.

    Until above identified, and so many other, connected areas of related clinical and social research are more consistently pursued first, such a massive infusion of money towards an initially flawed pharmaceutical construct deeply concerns.

    Autism is not a ‘disease’ and persons with autism are not ‘diseased.’ Still more medication solutions then can only serve to falsely blame the person and those who care….distract from what we know truly are best practices. And ‘animal testing’ for ASD…as it is a remarkably heterogeneous disorder across humans…is anticipated to serve what purpose other than torturing animals?

    Imagine, instead, what we could do with even a reasonable percentage of 38 million dollars focused on instructional, environmental and family support; jobs and transition services; school based supports for instructional differentiation, relevant adaptations and professional training; community development; future-centered person (and family) planning…debunking so many autism myths…since the diagnosis of ‘autism’ does not – should not – ever presume anything.

    Imagine, too, what we could do with a reasonable percentage of that money towards an authentic focus on the ‘Hidden Crisis’ of autism; that is, the so many children and families who very often suddenly realize that about nothing is available when the child ages out of the current plethora of earlier childhood services towards and into adulthood.

    The need for real time transition service and supports; support for young adults with ASD whether they are moving towards college, jobs, increasing independence or other interests/preferences is where money and time needs to be spent. This current neglect of the dramatically growing numbers of young adults across the autism spectrum will not only continue to create a huge human and family toll but a financial one as well.

    Neurological research is a good and needed entity on behalf of those with ASD and their families but it should not be presented as a primary, immediate or presumptive resolution to ASD-based needs and challenges. It also cannot continue to dissect the autism from the person as if autism is but another medical ‘disease’ since doing so starts off any such project with an inherent and deep seated flaw.

  2. DrLou1 says:

    I certainly can’t disagree with the reported statement that “forty million dollars a great catalyzer.” I would add, however, that with half of the amount coming from Pharmaceutical Industry Associations, this clinical research project will likely be less than comprehensive while more pursuing a singular agenda/outcome once again.

    Another way to view this 38-million-dollar project is that it will further nurture the ongoing big money misdirection to authentic treatment, intervention, support and individual understanding of persons with autism and their families. That we first need to know each individual with ASD; their lives, family and world – one at a time – remains overlooked

    That ASD is a remarkably heterogeneous disorder and far more likely representative of an umbrella like category rather than a targeted genetic and/or neurological process is a first challenge to address before any attempt to ‘cure’ and, even more so, to believe it even possible – let alone ethical – to make in vitro ‘corrections.’

    That autism becomes integrated into the being; the individualism and strengths of so very many of those persons with this ‘diagnosis’ also means that the continued presumption that there could be a ‘unifying’ drug treatment or category for treatment; about any treatment, for that matter…already brings a substantial flaw and challenge to such a project’s internal validity…let alone any subsequently presumed external validity.

    In fact, the project’s title; ‘European Autism Interventions – A Multicentre Study for Developing New Medications’ indicates a massive endorsement of pharmaceutical companies who are already far too deeply entrenched in the autism ‘treatment’ model.

    Despite some ongoing and worthwhile clinical efforts and theory, we still don’t have stable data as to a much more likely ‘categorical’ presentation of ASD.

    We don’t know the degree to which tiny sibs of those older with an ASD diagnosis who are, themselves, starting to exhibit ASD characteristics are the result of genetics, social replication of their older sibs behavioral patterns or a blend of both.

    We’ve not taken seriously the profusion of more often pseudoscientific autism ‘treatments’ which, though being used endlessly in many places, never seem to generate the related and hypothesized outcomes while taking time away from what we know really can help.

    And we’ve also still not more comprehensively studied the impact of demographics on purported autism prevalence and how this ‘diagnosis’ is often given. It is these huge holes, among others, which need to be resolved before investing $38 mill towards some kind of ‘unifying’ neurological theory and/or drug treatment for those given an ASD diagnosis.

    The use of highly targeted and carefully titrated medications blended with instructional/behavioral best practice across some categories of autism may ultimately be found productive. But we still know far too little about the impact of medication on ASD; on global processing disorders, even as the prescriptions continue to be written.

    Until above identified, and so many other, connected areas of related clinical and social research are more consistently pursued first, such a massive infusion of money towards an initially flawed pharmaceutical construct deeply concerns.

    Autism is not a ‘disease’ and persons with autism are not ‘diseased.’ Autism is also not a ‘mental health disorder’ but fundamentally developmental in nature. Still more medication solutions then can only serve to falsely blame the person and those who care…distract from what we know truly are best practices. And ‘animal testing’ for ASD…as it is a remarkably heterogeneous disorder across humans…is anticipated to serve what purpose other than torturing animals?

    Imagine, instead, what we could do with even a reasonable percentage of 38 million dollars focused on instructional, environmental and family support; jobs and transition services; school based supports for instructional differentiation, relevant adaptations and professional training; community development; future-centered person (and family) planning…debunking so many autism myths…since the diagnosis of ‘autism’ does not – should not – ever presume anything.

    Imagine, too, what we could do with a reasonable percentage of that money towards an authentic focus on the ‘Hidden Crisis’ of autism; that is, the so many children and families who very often suddenly realize that about nothing is available when the child ages out of the current plethora of earlier childhood services towards and into adulthood.

    The need for real time transition service and supports; support for young adults with ASD whether they are moving towards college, jobs, increasing independence or other interests/preferences is where money and time needs to be spent. This current neglect of the dramatically growing numbers of young adults across the autism spectrum will not only continue to create a huge human and family toll but a financial one as well.

    Neurological research is a good and needed entity on behalf of those with ASD and their families but it should not be presented as a primary, immediate or presumptive resolution to ASD-based needs and challenges. It also cannot continue to dissect the autism from the person as if autism is but another medical ‘disease’ since doing so starts off any such project with an inherent and deep-seated flaw.

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