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IOWA CITY - A University of Iowa clinician and neuroscientist has received an $18 million grant to continue a decade-long study on how a potential treatment for Huntington's disease may affect children's brain development.
Dr. Peg Nopoulos, chairwoman of the University of Iowa department of psychiatry, was awarded a five-year grant by the National Institution of Neurological Disorders and Strokes, part of the U.S. Institutes of Health.
'These are really high-in-the-sky questions,” she said. 'We're just boots on the ground, trying to understand how this affects children at risk for Huntington's disease.”
Huntington's disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells, diminishing thinking skills and emotions and disrupting fine motor function. If a parent has Huntington's disease, there is a 50 percent chance her or his child will develop the same disease.
There is no cure for Huntington's, but an emerging gene therapy in clinical trials has presented promising findings for slowing its progression, Nopoulos said. The gene therapy has been given only to individuals who have Huntington's disease in an attempt to slow the progressive breakdown.
Nopoulos said the next step is to give the therapy to individuals before the genetic disorder takes effect, to test whether it could prevent it altogether.
However, she said this possibility presents a key question - as the gene that causes the disease also is important for the growth and development of an individual's brain
'Our study is really examining how this gene affects brain development,” Nopoulos said. 'And in that context, we're examining how we would deliver a gene therapy and how that would impact brain development.
'We certainly don't want to prevent the disease only to cause problems for brain development.”
The gene that causes Huntington's, called HTT, is key for an area of the brain called the striatum, which helps control voluntary movement, among other functions. Through their study, Nopoulos and other researchers on the project hope to understand what consequences could result from the therapy on development.
'If we were to give the gene modifying therapy too early, it could prevent maximum brain function,” she said.
Nopoulos has been studying this question for 10 years at the UI and will continue that work under the newly funded project, called Children to Adult Neurodevelopment in Gene-Expanding Huntington's disease.
The $18 million grant from the national institute is a renewal of the same study from the UI, allowing Nopoulos and other researchers to expand the scope and size of the original study by including five times as many participants from five sites across the United States.
In addition to the UI, those sites will include the Children's Hospital of Philadelphia, Columbia University in New York, the University of California, Davis and the University of Texas in Houston.
All subjects in the study, who are between the ages of 6 and 30, have a parent with Huntington's, which means they are 50 percent at risk for developing the genetic disorder sometime in their lifetime.
Nopoulos said the test subjects don't know if they carry the genetic marker for the disease.
'We've been doing this for 10 years, and I'm always amazed at how strong these kids are,” Nopoulos said. The study 'gives them meaning and an opportunity to help gain knowledge about the disease their parent is going through.
'A lot of these children really take on the altruism of wanting to give back to the community, even when they know they're at risk themselves,” she said.
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