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Calls to Action

Spurring Development of New Medicines

Posted by Joe Horrigan, M.D., Autism Speaks assistant vice president and head of medical research 

I am feeling particularly hopeful about the pace of new drug development to treat autism. In recent years, we’ve seen a rapid series of scientific breakthroughs and leaps in our understanding of the biology of autism. The moment is right to accelerate the development of medicines to treat the core symptoms of autism spectrum disorder (ASD). 

I recently returned from an exciting meeting of the EU-AIMS, short for the European Autism Interventions – A Multicentre Study for Developing New Medications. EU-AIMS, funded in part by Autism Speaks, is one of the largest collaborations of academic researchers, pharmaceutical companies and advocates working together to develop new medicines for ASD. I was so impressed by the level of collaboration, harmony and communication among these groups, and I believe this reflects the urgency that we all feel to make the most out of this unique alignment of energy and resources.

I have long been concerned about the lack of safe and effective medicines to treat ASD. Currently, there are only two approved medications in the United States, both to treat autism-related irritability. None are approved to treat autism’s core symptoms. Moreover, many individuals with ASD have a complexity of disorders such as autism plus anxiety and/or insomnia and/or seizures and/or gastrointestinal issues. Clearly this makes their care more challenging.

Because of the lack of FDA-approved medications for autism, doctors are often obliged to prescribe drugs off label when treating persons with autism. This means that the drugs have been approved for other uses but not for autism-related indications. This is far from ideal, especially from a safety standpoint.

It is important to remind ourselves that drug development not only requires the investments of pharmaceutical companies, but is dependent on individuals with autism participating in the drug trials. This year, more trials are being launched, and several of these studies are quite large and may contribute to the approval of new medicines for the treatment of ASD and its related symptoms.

Part of my mission at Autism Speaks is to both encourage family participation and help ensure the safety of those who do choose to partake in clinical trials.

To that end, I have talked with pharmaceutical companies about medicines that may have potential benefit for individuals with ASD. These discussions have involved both drugs in development and those already available for other therapeutic uses. Our discussions have addressed study designs, outcome measures and safety practices that may enhance the value of the clinical trials while also attending to the best interests of the participants.   

From an advocacy perspective, another critical aspect of my work is to provide patients and families with a thorough understanding of the potential benefits of participating in drug trials as well as helping them to understand and assess the potential drawbacks and safety issues.

For these reasons, I have helped to develop Autism Speaks A Participant’s Guide to Autism Drug Research, a road map for patients and families who are considering participation in a clinical drug trial. You can read more about the guide and download it here.

There are real benefits to being a part of a trial. They include interacting with expert clinicians, having potential access to novel medicines and contributing to science and the greater good. But there are also risks and burdens. It’s important to understand how roles change when an individual shifts from being a patient to being a research subject. The Participant’s Guide describes how to assess the pros and cons of these different roles, and it walks families through several important considerations that will enhance their experience of participating in a research study.

Our collective efforts to assure that better medicines are made available for individuals with ASD depend on high-quality research that provides insight into the potential benefits and risks of these medicines. This essential knowledge is made possible by the thoughtful participation of individuals with ASD and their families. I and my colleagues at Autism Speaks are motivated to help in your decision-making around this important issue.

Families who want to consider a clinical trial can find more information on the Participate in Research page of our website. You can also visit www.clinicaltrials.gov, a searchable database of trials.

Thanks to all our families and volunteers for their support of this important work.

The Autism Speaks blog features opinions from people throughout the autism community. Each blog represents the point of view of the author and does not necessarily reflect Autism Speaks' beliefs or point of view.