Intellectual Disability Treatment: New Down Syndrome Possibilities

intellectual-disability-treatment-new-down-syndrome-possibilities.jpgOne of the fundamental questions in research for complex neurodevelopmental disorders such as PWS is – can the cognitive challenges of the disorder ever be improved using drugs or supplements?  A decade ago, most scientists would have argued that the brain is permanently “set” early on, and any later use of medications, interventions, etc., wouldn’t change a thing. 

That perception has changed as researchers have used animal models of neurodevelopmental disorders like Down syndrome and Rett syndrome to define the specific pathways that are disrupted, and show that cognitive deficits in the brain might be overcome with the right medication, even later in life.  Showing these things in a mouse is different than showing that it works in humans, though.

In Down syndrome, there are a couple of ongoing studies testing whether the beneficial effects of such drugs might be applicable to humans, and one of them has reported some encouraging resultsScience Daily reported on the study and its implications

Using an extract of green tea (epigallocatechin gallate, EGCG), which is known to impact a cellular pathway important in intellectual disability in Down syndrome, researchers tested the supplement in combination with a cognitive stimulation training program in adults with Down syndrome (age 16-34).

They report significant and sustained improvements in some measures of memory and executive function. Not all measures showed benefit, so this is by no means a cure for intellectual disability in Down syndrome, but it is an important step in showing that cognitive disability might be treatable in complex neurodevelopmental disorders. The study also provides important lessons on the challenges of successfully measuring changes in intellectual disability in the setting of a clinical trial, which can be applied to future studies in Down syndrome and for disorders such as PWS. 

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Topics: Research

Theresa Strong

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Theresa V. Strong, Ph.D., received a B.S. from Rutgers University and a Ph.D. in Medical Genetics from the University of Alabama at Birmingham (UAB). After postdoctoral studies with Dr. Francis Collins at the University of Michigan, she joined the UAB faculty, leading a research lab focused on gene therapy for cancer and directing UAB’s Vector Production Facility. Theresa is one of the founding members of FPWR and has directed FPWR’s grant program since its inception. In 2016, she transitioned to a full-time position as Director of Research Programs at FPWR. She remains an Adjunct Professor in the Department of Genetics at UAB. She and her husband Jim have four children, including a son with PWS.

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