Risperdal, an anti-psychotic drug, has just been approved by the Food and Drug Administration to treat the symptoms of autism in children and adolescents ages 5 to 16.
It’s the first time the FDA has approved any drug to treat behaviors such as aggression, deliberate self-injury and severe temper tantrums associated with autism in children. [1001 Great Ideas for Teaching and Raising Children with Autism Spectrum Disorders]
“This approval should benefit many autistic children as well as their parents and caregivers,” said Dr. Steven Galson, director of the FDA’s Center for Drug Evaluation and Research. He calls Risperdal “a welcome addition” with “an appropriate risk-benefit profile when tested in children.”
According to Peter Bell, president and chief executive of Cure Autism Now, a national organization committed to accelerating the pace of autism research, the approval is “an extremely positive sign.”
A call for caution
It signals, Bell said, that the pharmaceutical industry is looking at autism as a future market.
“Risperdal is not going to cure every child, and it’s not going be appropriate for all,” Bell said, “but when used appropriately it could make a significant difference in a child’s life.”
But others are sounding more cautious notes.
Some doctors warn that the drug should be used only after other treatments are tried that don’t involve medication. And the National Autism Association, an advocacy group for families of autistic children, has serious concerns about Risperdal.
Wendy Fournier, the association’s president, said medications such as Risperdal mask symptoms. She likened its use to prescribing pills for a headache without addressing the cause of the headache.
The association is particularly concerned about the side effects of Risperdal, which can include drowsiness, fatigue, constipation and weight gain. There also are rare reactions, such as extreme weight gain, the seeping of a milklike substance from nipples in both girls and boys, and a neurological disorder causing involuntary movements, which according to the drug manufacturer can sometimes be permanent.
“The choices parents of autistic children have to make regarding medications are very difficult and very important. We don’t fault any parents for doing what they need to do for their kids. But this particular drug appears to have some very serious side effects. It’s a nightmare,” said Rita Shreffler, executive director of the association.
Risperdal, which is manufactured by Janssen, a subsidiary of Johnson & Johnson, has been around for more than a decade. It’s been used since 1993 to treat adults with schizophrenia and since 2003 to treat adults with bipolar disorder. The drug exceeded $3 billion in sales in 2005.
The FDA’s new approval for the use of Risperdal for autistic children comes after two eight-week placebo-controlled trials in 156 patients ages 5 to 16, 90 percent of whom were ages 5 to 12.
Autism is a neurodevelopmental disorder that usually begins before age 3. It manifests itself in a variety of ways, which may include impaired social interaction; diminished communication skills; rigid, repetitive patterns of behavior and self-abuse. The cause is not known, and there is no known cure. According to the association, it affects 1 in 166 children. Others say that number is more like 1 in 250 and that it depends on how broadly one defines autism. Risperdal will do nothing to eliminate the underlying condition, doctors say.
Dr. Andrew Adesman, chief of developmental and behavioral pediatrics at Schneider Children’s Hospital in New Hyde Park, agrees medication should not be part of any initial treatment plan for autistic children. [Let Me Hear Your Voice: A Family's Triumph over Autism]
“Far and away, the interest is in avoiding medications with this group if possible,” Adesman said. “There is a concern here that people will reach too quickly for the drug and not provide other treatment services. Risperdal should be used reservedly and not as the first line of treatment.”
Intervening to thwart injury
He said, however, if intervention without medication is not successful in changing the child’s behavior and if the child is doing such things as banging his head or biting himself, then “maybe there is a place for pharmacologic intervention.” It has to be “a balanced approach,” Adesman said.
Adesman said doctors should have a “healthy respect” for the side effects of Risperdal, but, he added, the severe side effects are rare. He said that when a child’s behavior does pose a threat to himself or others, people should not focus on the side effects while ignoring the benefits of the medication.
“These are heart-wrenching cases, and you want to help in any way possible,” Adesman said, “but you have to avoid the temptation to reach for what somebody sees as an easy answer.”
The association’s Shreffler said her worry is that some doctors will give in to that temptation. “The scary possibility is that since the FDA has said it’s OK to use with children, doctors across the country may well say ‘We’ll just try this and see what happens,’” she said.
“It’s frightening,” she added, “because a lot of these kids are getting better through treatments [without drugs] that address what’s underlying these behaviors rather than masking them with psychotropic drugs.”