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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.

5|25: Celebrating Five Years of Autism Science Day 22: Combined Therapies Hold Promise for More Effective Treatments

February 22, 2010 Leave a comment

In honor of the anniversary of Autism Speaks’ founding on Feb 25, for the next 25 days we will be sharing stories about the many significant scientific advances that have occurred during our first five years together. Our 22nd item, Combined Therapies Hold Promise for More Effective Treatments, is from Autism Speaks’ Top 10 Autism Research Events of 2009..

Just over three years ago the FDA’s landmark approval of risperidone for the treatment of ASD represented a significant breakthrough for the autism community. Since then other large-scale autism studies have sought FDA approval for drugs that target core or associated symptoms for autism, but unfortunately few of these trials have proven successful. In 2009, taking a cue from other disorders such as ADHD where a combined effect of both medication and behavioral therapies has proven fruitful, researchers published the first successful combined randomized controlled trial for ASD. The paper in the Journal of the American Academy of Child and Adolescent Psychiatry demonstrated that combined pharmacological and behavioral treatments was more effective than pharmacological treatment alone for reducing challenging behaviors.

Risperidone is approved for reducing aggression and irritability in children and adolescents with autism. However, its use still presents a number of challenges to clinicians. Like other atypical anti-psychotics it can have adverse side effects including weight gain, potentially leading to increased risk for obesity, and GI symptoms such as diarrhea and constipation, which can already be problematic for children with ASD. Clinicians must therefore balance the benefit of treating the problem behaviors with the potential for creating new health challenges for the child. On the other hand, behavioral therapies have been shown to be one of the most reliably effective treatments for improving problem behaviors with limited side effects. Combination therapies create a synergistic therapeutic environment in which medication allows a child to get more from behavioral therapies and, at the same time, the benefits of behavioral therapy may mean lower doses of medication are required.

A new multi-site study by the Research Units on Pediatric Psychopharmacology Autism Network, the same group that conducted the pivotal studies leading to the approval of risperidone, investigated whether combining risperidone treatments with a simultaneous behavioral intervention would be more effective than medication alone. Their 24-week study of 124 children ages 4-13, compared a treatment regime of risperidone alone with a combined treatment regimen of risperidone and a parent training program that followed the principles of applied behavioral analysis. While both the combined and medication-only treatments reduced the severity of non-compliant behaviors, the combined therapy resulted in a significantly greater reduction while using lower doses of risperidone. The combined therapy was also better at reducing other challenging behaviors, such as irritability and hyperactivity.

This study provides hope for a wider range of available treatments and greater flexibility for clinicians who should be encouraged to use combined approaches in cases where medications or behavioral interventions are not effective on their own. Confirming the effectiveness of coordinated treatments that take full advantage of the benefits of both pharmaceutical and behavioral approaches also demonstrates the continued need to support research establishing the most effective treatments in all realms. Finally, the vast majority of clinical trials conducted to date have only addressed how an individual treatment compares to a placebo. Very few studies have been conducted that make head-to-head comparisons of two or more treatments as was done here, so the success of this trial will also serve to highlight the utility of “comparative effectiveness trials” for determining the best treatments for ASD.

Did you know?: Autism Speaks’ funded Interactive Autism Network (IAN) is a web-based family registry and social network that brings together thousands of families with autism research and provides a forum for families to report information about their experiences.  In a recent study on over 5000 children in IAN, 35% of parents reported that their children were taking at least one psychotropic medicine and the use of these drugs increased with age.  The incidence of a comorbid condition such as seizures, ADHD or anxiety increased the likelihood of medication use.  The IAN authors also reported on correlations between insurance access and use of multiple medications, noting that those children using public insurance plans (such as Medicaid) tended to be on more medications, possibly due to an inability to get coverage for behavioral therapies.

5|25: Celebrating Five Years of Autism Science Day 6: FDA Approval of Risperidone

February 6, 2010 Leave a comment

In honor of the anniversary of Autism Speaks’ founding on Feb 25, for the next 25 days we will be sharing stories about the many significant scientific advances that have occurred during our first five years together. Our sixth item, FDA Approval of Risperidone, is from Autism Speaks’ Top 10 Autism Research Events of 2007.

In late 2006 the U.S. Food and Drug Administration (FDA) approved the first medication indicated to treat certain symptoms associated with autism, making 2007 the first year an approved drug for autism was available. The drug, risperidone sold under the brand name Risperdal, is manufactured and marketed by Janssen, L.P., a subsidiary of Johnson & Johnson.

First introduced in 1993 as an atypical antipsychotic to treat schizophrenia, Risperdal can now be marketed as a treatment of irritability associated with autistic disorder in children and adolescents aged 5-16 years. This includes symptoms of aggression towards others, deliberate self-injuriousness, temper tantrums and quickly changing moods. Importantly, Risperdal does not treat the core symptoms of autism such as communication problems and trouble with social interactions. However, J&J used two clinical trials to demonstrate the benefits of the drug in treating the associated behavioral disturbances that can interfere with school, learning and family life.

This event not only sets precedence for gaining FDA approval for medications that treat autistic symptoms, it also shows that autism is considered a viable market for the pharmaceutical industry which will hopefully lead to the development of new compounds that will benefit the quality of life for those living with autism.

Update since this story was first run: On November 20, 2009 the FDA approved Abilify (aripiprazole) for the treatment of irritability associated with autism, making it the second medication to receive an autism indication. Specifically, the drug is approved for children 6-17 to help alleviate symptoms of “aggression towards others, deliberate self-injuriousness, temper tantrums, and quickly changing moods.” Aripiprazole, which is manufactured and marketed as Abilify by Bristol-Myers Squibb, is an atypical antipsychotic medication used to treat schizophrenia, bipolar disorder and depression. Although an FDA approval for a medication addressing the core symptoms of autism is still lacking, the approval of Abilify will offer additional options for families and clinicians.

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