Stories about: developmental disorders

A Manhattan Project for the brain, at age 50

Formation of the IDDRCs in the 1960s launched a Manhattan project for the brain.
Landmark federal legislation in JFK’s final days launched an explosion of neuroscience research. (PHOTO ILLUSTRATION: NANCY FLIESLER/ADOBE STOCK)

On October 30th, 2018, Boston Children’s will be marking the 50th anniversary of the founding of its Intellectual and Developmental Disabilities Research Center.

As the African-American civil rights movement was flowering in the 1960s, a less visible civil rights movement was dawning. And so was a revolution in science that may outshine that spurred by the U.S. space program.

It was a time when children with what is now called intellectual disability (ID) or developmental disability (DD) were “excused” from school and routinely abandoned to institutions. “Schools” like the Fernald Center in Massachusetts and the Willowbrook State School in New York housed thousands of residents.

Some participated in research, but not the kind you might think. At Willowbrook, children were deliberately infected with hepatitis to test a new treatment. At Fernald, they were deliberately exposed to radiation in an experiment approved by the Atomic Energy Commission. Institutional review boards did not then exist.

In 1962, President John F. Kennedy convened a panel to propose a “National Action to Combat Mental Retardation,” at the strong urging of his sister Eunice Kennedy Shriver. Three weeks before JFK’s assassination, the first legislation passed. It changed the course of history.

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Rett syndrome sees glimmer of hope in Phase I trial

Hope for Rett syndrome?This post is the first in a two-part series on clinical trials in autism spectrum disorders. Read part 2.

In the world of neurodevelopmental disorders, an exciting trend is the emergence of specific molecular targets and treatments through genetic research. A case in point is IGF-1 therapy for Rett syndrome, a devastating disorder in girls that affects their ability to speak, walk, eat and breathe. It causes autism-like behaviors, intellectual disability and repetitive hand-wringing movements—a hallmark of the disorder.

A Phase I trial, published this week in the Proceedings of the National Academy of Sciences Early Edition, has modest but consistent results suggesting improvements in some salient features of the disorder.

Current treatments for Rett syndrome address only the symptoms and comorbidities, such as seizures, anxiety and scoliosis, but not the disease itself. But in 2007, findings in a mouse model (which even replicated the hand-wringing) changed how scientists think about Rett and other neurodevelopmental disorders, previously thought to be untreatable.

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