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In an article in The Day, by Lee Howard, we learn that associate research fellow at Pfizer Diane Stephenson and her colleague Howie Mayer were able to bring to fruition their idea of forming a separate research unit focusing on autism.
Stephenson has two nephews and a niece with the neurological disorder; Mayer has two children with autism.
They were later joined by another colleague, Larry Fitzgerald. The group contacted key experts outside Pfizer who knew the latest breakthroughs in autism research.
According to Stephenson, science in the past years have started making significant headway in genetic research geared toward autism. Two years ago might have been too soon. But late last year, the colleagues felt they had enough science on their side to make their case.
“Most everyone told us we were crazy,” says Stephenson. The pharmaceutical industry — and Pfizer — has been in a downsizing mode lately. But senior management embraced the idea and launched the autism unit in January with 15 scientists.
Fitzgerald became the first head of the unit, but departed a few weeks ago, and mayer has also moved on, now working for another business unit at Pfizer. Stephenson is the remaining founder still working on-site.
The long range goal will be to prevent autism, but Pfizer plans to address short-term solutions at first.
Researchers plan to begin by targeting symptoms that appear to cross the spectrum of autism disorders: anxiety, agitation, sleep disorders, social deficits, language disabilities and repetitive behaviors. The focus is to be identification of medications which address symptoms.
A longer-range goal is to understand the neurobiology behind the disorder so that core symptoms can be treated.
One asset in the research process, according to scientists, is the fact that several markers of autism have recently been identified. One of these telltale signs is eye-tracking. While normal children focus on the eyes of a face, autistic children look away, toward the mouth.
This baseline awareness can help scientists see if a drug is having an effect.
Senior scientist Edward Guilmette, in the neuroscience unit labs, is starting to target certain genes that could have an effect on autism. In mice models, the effect of turning on or off various genes than is studied.
Fifteen researchers can seem like a big commitment, but Pfizer scientists say that number is small compared to the vast research that remains to be done. They have reached out to collaborators at MIT, the Yale Child Study Center, and NYU, to develop and expand their work.
The current work being done at the moment involves mostly biology and animal studies. But as specific small-molecule drug targets are developed, more chemists will be enlisted to help.
The first Pfizer autism medicines will likely come from its established drug portfolio. One possibility is the pain medication Lyrica, although Stephenson emphasizes that drug trials have yet to establish any clinical support for the hypothesis.
Finding uses for established drugs will be much less costly than the $1 billion price tag that would be involved in bringing new drugs to market. Current drugs have already been proved to be safe.
Another factor reducing costs for developing autism drugs is that several of the spectrum disorders, including Fragile X, currenly have no approved treatment. That means companies won’t have to prove to the FDA that their drugs are more effective than others on the market.
The long-term hope, according to Stevenson and her colleague Michael Tranfaglia of the FRAXA Research Foundation, is to actually reverse the course of autism — an idea that a few years ago would have seemed absurd, but is already being shown to be within the realm of possibility.
“The sooner you intervene, the better,” says Stephenson.
Tranfaglia contends, “You can actually normalize development. It’s entirely reasonable to think you could completely alter the course of the disease.”
my source was: www.courant.com; article on 10/25/09. See the entire article by Lee Howard at The Day: http://www.theday.com/article/20091012/BIZ02/310129965
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