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If breakthroughs take time, how can research help families today?

February 9, 2012 10 comments

Posted by Autism Speaks Chief Science Officer Geri Dawson, Ph.D.

Dear friends,

Recently, someone posed a question that made me think hard about the immediate relevance of our research to those affected by autism. I had been explaining Autism Speaks’ new focus on developing medicines that, one day, will target autism’s core symptoms in ways that reduce disabilities and improve learning abilities. Someone commented that this would likely take years to accomplish. I had to agree. His follow-up question: So, how does research help families today?

For the answer, I found myself thinking about how the Cystic Fibrosis Foundation faced this same question decades ago. Like Autism Speaks, the Cystic Fibrosis Foundation was grappling with a disorder in its medical infancy. Cystic fibrosis was defined as a medical condition in 1938. The Foundation followed in 1955. At that time, the median age of survival for those affected by the disorder was just ten years.

The leadership of the Cystic Fibrosis Foundation knew they were grappling with a complex disorder that would take years to fully understand. So they developed parallel research efforts. One focused on the immediate development of improved diagnosis and treatments that could ease symptoms. The other focused on basic science with the goal of ultimately revolutionizing treatment with therapies that target the disorder’s root causes.

Their short-term efforts included support for a network of clinical care and research centers, a patient registry and studies that focused on improving treatment of chronic symptoms and associated medical conditions. Within a relatively short time, diagnostic methods improved and physicians began adopting new gold-standard practices, including new methods for fighting lung infections and improving lung function – all made possible through research that the Cystic Fibrosis Foundation helped support. The median age of survival jumped from 10 years to 37 years!

Meanwhile, long-term research efforts focused on understanding the causes and biology of cystic fibrosis. In 1989, scientists made major breakthroughs in genetic understanding. This, in turn, led to tremendous insights into the disorder’s underlying biology. Then, just last week, the FDA approved the first drug to treat the underlying cause of cystic fibrosis, rather than its symptoms. One doctor described how his patient was able to “shovel snow for the first time.” Not coincidentally, the Cystic Fibrosis Foundation had contributed millions of dollars to the development of this drug (Kalydeco). Its early funding had been essential to convince drug companies to make the larger financial investment needed to bring any successful drug to market. In the process, the foundation negotiated a deal to earn drug royalties, which will now be reinvested in further research advancements. Just as exciting, other “disease-modifying” cystic fibrosis drugs are moving through the research pipeline.

This is the same strategy that Autism Speaks is taking with investments in both research that improves quality of life in the short term and longer-term research that promises to transform how autism is treated.

Here are just a few examples of funded research projects with the potential to improve quality of life in the near future:

  • Identification of preventable environmental risk factors for autism spectrum disorder (ASD)
  • Validation of questionnaires that pediatricians can use to screen babies for ASD and, so, offer earlier intervention that will improve outcomes
  • Biomarkers (e.g. immune alterations) that could identify infants at risk for ASD
  • Development of effective early interventions for babies before the full syndrome develops
  • Support of technological inventions to enhance communication in nonverbal persons
  • Development of physician guidelines for assessment and treatment of medical conditions associated with ASD
  • Development of more effective treatments for associated conditions, including sleep disturbances, GI disorders, seizures and anxiety
  • Development of interventions to improve employment success and relationship skills in adults
  • Development of cognitive rehabilitation interventions for adults

Even as we support the development of these improved services, we are also investing in research that can identify the most effective ways to broadly implement new gold-standard practices to produce positive changes in community healthcare, education and support services for all persons who struggle with autism. This type of “dissemination research” also tells us how to best target limited resources.

Meanwhile, our long-term investments are advancing the understanding of autism’s underlying biology and the genetic and environmental factors that contribute to its development. These investments are exploring the role of the immune system, brain signaling pathways and the GI system, among other topics. Over the last five years, tremendous progress in these areas has advanced research to the point where we are now collaborating with industry to develop novel drugs with the potential to ease severe and disabling core symptoms – in adults as well as children. Fortunately, the tools we have available today will make drug discovery and development much faster than before.

Connecting the dots
At Autism Speaks, the research we fund interconnects with all parts of our mission, including awareness, advocacy and family services. Our awareness campaign, for example, is shaped by research that has revealed great disparities in access to services by communities such as ethnic-minority and low-income families.

Our advocacy of insurance reform, in turn, critically depends on research that demonstrates how early intervention improves outcomes. Research also plays a critical role in bolstering our advocacy for adolescents and adults. For example, a recent study demonstrated that adults with ASD face greater challenges in employment and social participation than do adults with other common disabilities. More importantly, this same study suggests that providing transition services immediately after high school is the most cost effective way to improve outcomes. We can use this information to advocate for improved services during the transition from high school to adulthood. Other currently funded studies promise to help us advance insurance reform to assure coverage of other interventions with proven benefits for school-age children and adults.

Similarly, Autism Speaks is funding research aimed at determining the real-world effects of proposed changes in the diagnostic criteria for autism. Will these new criteria exclude people previously diagnosed with ASD? Will they affect access to vital services? These answers will be crucial to our ability to advocate for any necessary changes in the proposed criteria.

While we see our research improving lives now, we remain committed to our long-term goals of revolutionizing treatment of ASD. I know in my heart that someday we will be making the kind of breathtaking announcement that we heard from the Cystic Fibrosis Foundation last week. The day is coming. In the meantime, we will ensure that our scientific mission remains relevant to our families today.

Warmest wishes,

Geri Dawson
Chief Science Officer, Autism Speaks

How common are anxiety disorders in people with autism, and are there effective treatments?

January 6, 2012 14 comments

This week’s “Got Questions” answer comes from Rob Ring, PhD, Autism Speaks vice president of translational research.

Without question, anxiety is a real and serious problem for many people on the autism spectrum. We hear this from parents, teachers and doctors, as well as from adolescents and adults with autism spectrum disorder (ASD). This disabling anxiety can take the form of one or more disorders, including panic disorder and phobias.

A recent review of scientific studies on autism and anxiety revealed that we have no clear gauge of how commonly anxiety disorders overlap with autism. A few small, relatively short-term studies have produced starkly different results: from 11 percent to 84 percent. (For comparison, the prevalence of anxiety disorders among the general population is about 18 percent.) A reliable estimate will require a study that tracks many more individuals with autism over longer periods of time and that considers the distinctive way that anxiety oftentimes expresses itself in those affected by ASD.

Fortunately, Autism Speaks is funding the Autism Treatment Network, which collects systematic data on a wide range of medical conditions, including anxiety disorders, in children with ASD.  This data will help us better understand the proportion of people with ASD who are suffering from anxiety symptoms.

Meanwhile preliminary studies have provided insights. They suggest, for example, that adolescents with autism may be particularly prone to anxiety disorders, while younger children on the spectrum may not differ at all from the average population. Some studies likewise suggest that high-functioning individuals on the spectrum experience higher rates of anxiety disorders than do lower-functioning individuals. Still we must emphasize that these results are preliminary. We don’t know nearly as much as we should about how anxiety disorders affect those with autism.

recent review of studies found that behavioral interventions can help many children and adolescents with autism who also struggle with anxiety. Along these lines, some studies  suggest that cognitive behavioral therapy can be particularly helpful for high-functioning adolescents and adults with autism and anxiety. We will explore behavioral interventions further in a future “Got Questions?” blog. My own expertise is in the medical treatment of anxiety in persons with ASD.

Currently, we have no medications approved by the Food and Drug Administration (FDA) expressly for the treatment of anxiety in children, adolescents or adults with autism. Some classes of drugs commonly prescribed for treating anxiety disorders in the general population likewise help some of those on the autism spectrum. These include the selective serotonin reuptake inhibitors (SSRIs) such as Prozac. For those with autism, anxiety drugs are best used in combination with behavioral interventions. Among high-functioning individuals, they may be particularly effective when combined with cognitive behavioral therapy.

However, some doctors report that anti-anxiety medications seem to be less effective overall in people with autism spectrum disorder than they are in the general population. This observation needs to be verified with controlled research. It suggests the possibility that the biological root of anxiety in those with autism may differ from the “norm” and, as a result, may respond best to different treatments.

At Autism Speaks, we are actively supporting research into anxiety disorders and other medical conditions frequently associated with autism. This includes both basic research on the underlying biology of autism and the safe development of drugs that can relieve disabling symptoms and improve quality of life.

If you are considering anti-anxiety medication for a child with autism, our recently published Medication Decision Aid can help you work with your child’s physician to sort through the pros and cons in the context of your values and goals for your child. You can learn more about the medication tool kit and download a free copy, here.

Got more questions? Send them to GotQuestions@autismspeaks.org. And bring them to  our next webchat with Autism Speaks Chief Science Officer Geri Dawson, Ph.D., and  Autism Speaks assistant vice president and head of medical research Joe Horrigan, M.D. More information on their monthly webchats here.


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.

Can my taking medication during pregnancy cause autism in my baby?

September 2, 2011 49 comments

This week’s “Got Questions?” response comes from Alycia Halladay, PhD, Autism Speaks’ director of research for environmental science

Last month, a group of California researchers reported an increased risk of autism among babies whose mothers took a certain catergory of antidepressant medications–selective serotonin reuptake inhibitors (SSRIs)—during the first trimester of pregnancy. You may know these drugs by such brand names as Prozac, Effexor, Paxil, and Celexa.

So what do these results mean for pregnant women? First, caution is needed before rushing to judgment. The study was relatively small, and the increase in the risk of autism was modest. So more study is clearly needed to confirm the link and clarify how great a risk, if any, is associated with a mother using this type of antidepressant during pregnancy.

Further caution is needed because the effects of a mother’s anxiety and depression during pregnancy and early infancy are well known. In fact, it’s not clear whether the autism risk associated with taking antidepressants during pregnancy is, in fact, related to the women’s depression rather than the drugs themselves.

For these reason, many doctors have argued that the benefits of SSRIs outweigh concerns about risks that SSRI exposure may pose to a fetus or infant during pregnancy and nursing. Clearly, more research is needed.

Beyond SSRIs, researchers have looked at several other medications to see if their use during pregnancy increases the risk that a baby will go on to develop autism. Among the most thoroughly researched is the anti-seizure medication valproic acid (U.S. brand name Depakote). Studies show that, as a group, children whose mothers take valproic acid during their first trimester of pregnancy are more likely to develop an autism spectrum disorder (ASD) than are children who are not exposed.

Autism Speaks has supported research into how valproic acid might contribute to the development of ASDs. Through the study of donated brain tissue, for example, we have learned that individuals with autism share some “neuropathologies,” or altered brain features, with those who were exposed to valproic acid before birth. In addition, several studies show that exposure to valproic acid during critical periods of brain development can produce autism-like behaviors in animal models.

So the good news is that our research has deepened understanding about how valproic acid during pregnancy can contribute to the development of ASDs. The bad news is that it can be quite dangerous for women with epilepsy to stop taking this medication during pregnancy—owing to their increased risk of seizures. As a result, such decisions should be made carefully with a physician can discuss alternative drugs.

Findings are still emerging with other medications given during pregnancy. For instance, relatively small studies (such as this one) suggest an increased risk for ASD in babies whose mothers were given the medication terbutaline to stop premature labor. Another small study suggested increased risk of autism related to women taking high doses of the anti-ulcer drug misoprostol early in pregnancy. (This drug is also used to induce labor later in pregnancy.) But in many cases, such preliminary research has yet to move past the “interesting” stage to reach enough certainty to change medical practices.

Other, larger studies hint at an increased risk of autism in the babies of women who take certain broad classes of medications such as antipsychotics or mood stabilizers during pregnancy. Still the question remains: Is the autism risk due to the medications or to the underlying medical conditions that the drugs are being used to treat?

Beyond medications, studies have revealed a number of other pregnancy complications and events that appear to contribute to the risk that a baby will go on to develop autism.  These include the pregnant mother’s exposure to toxic chemicals, infections such as flu, and her diet and nutrition at the time of conception as well as during pregnancy.

Autism Speaks continues to fund a number of important studies looking at autism risk factors during pregnancy. If you have at least one child already diagnosed with an ASD, find out more about participating in the EARLI study (link at left) before or at the start of your next pregnancy. Or consider enrolling your child and family in the CHARGE study, which looks at risk factors before, during, and after your child’s birth.

We will be continuing to update you on the science as it emerges.  If you have any concerns about the medications you are taking during pregnancy, please discuss them with your doctor. For more resources, we also recommend the Organization of Teratology Information Specialists.

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