A LEAP forward for autism

The EU-AIMS LEAP study gains EMA support for its biomarkers, study criteria

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NEW YORK—With autism numbers on the rise, answers about autism spectrum disorder (ASD) are needed now more than ever, and organizations from all walks of industry, whether public or private, are working together in a massive translational effort in search of those answers. The Longitudinal European Autism Project (LEAP) is part of the European Autism Interventions–A Multicenter Study for Developing New Medications (EU-AIMS), the largest single grant for autism globally and the largest for any mental health disorder in Europe, and is on the hunt for biomarkers for autism.
“We now recognize the enormous diversity among people with autism spectrum conditions,” Eva Loth, EU-AIMS project science coordinator and deputy lead clinical researcher, said in an Autism Speaks press release. “We need to move away from the idea of a one-size-fits all treatment for autism. We believe that it will be more fruitful to develop treatments—medical and non-medical—for different persons with autism depending on their specific needs. To do this, it is vital to have clearly defined biomarkers, or biological measures, to identify subtypes of autism and match them with the treatments most likely to provide benefit.”
“The issue that we face in the development of medicines for autism spectrum disorder and the development of new treatments is that most measures that are used right now to diagnose and to measure outcome are very subjective,” adds Gahan Pandina, senior director and venture leader at Janssen Research & Development and one of the publication’s authors. “They’re clinician- or parent-reported rating scales. And what was identified is that although we know that there are new novel mechanisms in the brain that are being identified through pharmacogenomics research, this lack of objective measures of ways to stratify the population—that is, to create subgroups of the response to treatments—and also to measure change sensitively and objectively, is really hampering our ability to do clinical trials, and clinical trials are the way we test new medicines in clinical research.”
A December publication—”Identification and validation of biomarkers for autism spectrum disorders,” which appeared in Nature Reviews Drug Discovery—reported on an important milestone for the program, namely that the European Medicines Agency (EMA) had agreed on procedures for selecting study participants and evaluating the effectiveness of novel therapies for autism.
LEAP unites support organizations, industry members and academia, including Autism Speaks, Janssen, Roche, Eli Lilly and Kings College London. The study is slated to run several years, and consists of about 450 patients with ASD and roughly 350 control participants without autism or any other psychiatric conditions. Pandina says patients in LEAP are anywhere from six to 30 years old and are tested in 18-month increments, looking at changes in biomarkers, symptoms, co-morbidities, quality of life and adaptive behavior. Biomarkers for this study include neuroimaging (such as functional MRI and diffusion tensor imaging), eye tracking, cognition, EEG, brain chemistry and pharmacogenomics.
Researchers from LEAP reached out to the EMA, specifically the Committee for Medicinal Products for Human Use (CHMP), to see if the study’s biomarker findings would be accepted in regulatory decisions for future clinical trials. The article shared the results of their correspondence, namely that the EMA supported their proposed criteria for selecting study participants and the symptom improvements that should be used to measure therapeutic benefit, as well as the methods for identifying biomarkers for subtypes of autism.
“Autism spectrum disorder is a very heterogeneous disorder. In fact, we think it’s many disorders, and for that reason you can have very different symptom presentations, and the symptoms that people have can vary very much from subject to subject. So finding an objective means to measure the biology of the disorder itself that relates directly to those symptoms is very, very important,” Pandina explains. “Although there’s been an increase in interest and focus in autism spectrum disorder over the past decade, particularly with the rising prevalence of autism, the complexity of autism has made it difficult to study, and it’s been especially difficult to understand how we might develop new treatments given the complexity of autism.”
EU-AIMS, he notes, is “truly a public-private partnership.”
“[LEAP] is a very ambitious, exciting program,” he says, adding that “That comprehensive approach is going to jump our knowledge ahead substantially in the field, and has real promise to help us immensely in our quest to develop new therapies for autism.”

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