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Posts Tagged ‘medicines’

Are there effective medicines for treating core autism symptoms?

December 9, 2011 12 comments


This week’s “Got Questions” answer comes from Joseph Horrigan, MD, Autism Speaks assistant vice president, head of medical research.

First, it’s important to note that medicines for treating autism are most effective when used in conjunction with behavioral therapies. Ideally, medicines are a complement to other treatment strategies.

Medicines for treating autism’s three core symptoms—communication difficulties, social challenges and repetitive behavior—have long represented a huge area of unmet need. Unfortunately, few drugs on the market today effectively relieve these symptoms and none of the options most often prescribed by practitioners work well for every individual.

In fact, while the Food and Drug Administration (FDA) has approved two drugs for treating irritability associated with the autism (risperidone and aripiprazole), it has yet to approve a medicine for treating autism’s three core characteristics. Nonetheless, medicines such as risperidone and aripiprazole can be beneficial in ways that can ease these core symptoms, because relieving irritability often improves sociability while reducing tantrums, aggressive outbursts and self-injurious behaviors.

The good news is that the range of medication options may soon change, thanks to recent advances in our understanding of the biology that produces autism’s core symptoms. This has made it possible for researchers to begin testing compounds that may help normalize crucial brain functions involved in autism. Early experiments suggest that several compounds with different mechanisms of action have great potential for clinical use, and many are now in clinical trials. [This link takes you to the search engine of the NIH clinical trial network, with results under the search term “autism.”]

Although these developments are exciting and hold real promise for bettering the lives of people with autism, we will have to wait at least a few more years before we know if any of these drug studies produce enough information on safety and effectiveness to merit FDA approval for the treatment of core symptoms.

Today, most medicines prescribed to ease autism’s disabling symptoms are used “off label,” meaning that their FDA approval is for other, sometimes-related conditions such as attention deficit hyperactivity disorder (ADHD), sleep disturbances or depression. Such off-label use is common in virtually all areas of medicine and is usually done to relieve significant suffering in the absence of sufficiently large and targeted studies.

An example in autism would be the class of medicines known as selective serotonin re-uptake inhibitors (SSRIs), including fluoxetine. Several of these medicines are FDA-approved for the treatment of anxiety disorders and depression, in children as well as adults. Although large clinical trials have yet to demonstrate their effectiveness, parents and clinicians have found that they can  ease social difficulties among some people with autism. However, it has proven to be difficult to predict which medicines in this class may produce the greatest benefit for a given patient with autism. Similarly, determining the best dose can be quite challenging.

Another example would be naltrexone, which is FDA-approved for the treatment of alcohol and opioid addictions. It can ease disabling repetitive and self-injurious behaviors in some children and adults with autism.

These medicines do not work for everyone, and all medicines have side effects. And as noted above, each person may respond differently to medicines. In addition, changes in response to a medicine can occur as time goes on, even when the dose is not changed. Over time, some people develop tolerance (when a drug stops being effective) or sensitization (when side effects worsen).

Because using these medications in children and adolescents can be a difficult decision for parents, you may find it helpful to use our Medication Decision Tool Kit, a guide for actively working with a physician to find the approach that fits best with your values and goals. You can download it free here.

These are exciting times in the development of new medicines for relieving autism’s most disabling symptoms, and Autism Speaks is increasing its funding and focus in this promising area, while placing great emphasis on ensuring the safety of promising new medicines. Please stay tuned!

Read more science news and perspective on the Science Page.

Autism Speaks Participates in FasterCures “Crossing the Valley of Death” Forum

September 23, 2010 2 comments

As more and more is learned about the biology of autism, the development of novel medicines to treat its core symptoms is no longer just a hope.  Therefore Autism Speaks was pleased to be invited to participate in the 5th annual FasterCures-Esquire Leadership Forum, devoted this year to the topic of translational research, that is, how to translate knowledge gained from basic research into products that can provide real healthcare benefits for patients.

FasterCures, a Washington D.C. think tank focused on accelerating medical solutions, is based on the founding principle that saving time saves lives.  Since 2005 they have brought together a cross-disciplinary group of innovative thinkers to explore novel approaches that hold the potential to accelerate the search for new treatments and cures for diseases.  With representatives of biotechnology, pharmaceutical and patient advocacy organizations in attendance, the theme for this year’s small workshop was to determine action items to achieve innovation in drug development.   Attendees agreed that in order for this to happen as fast as possible, there needs to be better integration between all stakeholders – patients, financiers, academic researchers and for-profit companies alike.  The meeting participants called for the identification of new business models and research paradigms to achieve this integration.

Full of buzz words such as “open innovation,” “collaboration,” and “entrepreneurship,” the discussions focused on identifying the organizational and cultural barriers that influence the interface between traditional academic research and industry-related drug development.   This interface has long been called the “Valley of Death” because of the enormous difficulty in translating research discoveries into viable medical solutions.   While the government has begun several initiatives to push biomedical research forward across the Valley of Death, for-profit organizations such as pharmaceutical and biotechnology companies are beginning to unite to address the challenges that make it inefficient and, at times, impossible, to carry forward such risky and costly research on their own.  Some of the challenges identified at the meeting include the many knowledge gaps that remain in our basic understanding of human biology, the lack of financial incentives to work in some disease areas, and the constraints created by business policies that are meant to provide competitive advantages but instead often hinder progress.

The short meeting highlighted lessons learned from other industries and several new models of collaboration.   At its conclusion the participants agreed that streamlining the drug development process will involve not just procedural improvements but much more partnering than has ever been attempted before, including between individual pharmaceutical companies and between pharmaceutical companies and academia.  This opens a role for non-profit advocacy organizations such as Autism Speaks in bringing together the many diverse players, each with their own motivations and incentives.   Towards this end, Autism Speaks has recently formed a Translational Research Initiative that aims to enhance communication between academic scientists and industry representatives and nurture our renewed hopes for successful translational research in autism and new healthcare solutions.

Read more about translational research from our Chief Science Officer, Dr. Geri Dawson.

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