Protein Gone Rogue
NIH announces a $350,000 grant to help two EWU biochemistry professors tackle a deadly disorder.
The itpa protein plays a crucial role in several metabolic processes that are essential to human life. Severe defects in ITPA are uncommon, but the results can be devastating.
Infants born with a rare ITPA abnormality, for example, face the risk of a lethal neurological condition known as Developmental and Epileptic Encephalopathy 35. Few diagnosed with the disorder live past their third birthday. Even patients with less severe ITPA deficiencies can experience dangerous side-effects to medications prescribed for other conditions — including life-threatening conditions.
Nick Burgis, professor and chair of chemistry, biochemistry and physics at EWU, is perhaps the nation’s leading authority on the ITPA protein and the genetic processes it regulates. Together with his colleague Yao Houndonougbo, also a professor of chemistry and biochemistry at Eastern, Burgis is using that knowledge to pursue development of first-generation molecular-level treatments for ITPA-related disorders.
Burgis’ and Houndonougbo’s efforts recently received an important boost from the National Institutes of Health, which last month announced a three-year, $350,000 “research enhancement” award that will extend the agency’s previous support for the two scientists and their students.
The focus of the work supported by the grant, says Burgis, is to identify a molecule that can restore the ITPA’s proper functionality. To do this, he, Houndonougbo, and their students will deploy a battery of digital and biochemical tools to look for promising molecules among a virtual and physical library of 300,000 molecules housed at UCLA.
As part of this effort, Burgis — along with two, yet-to-be-named Eastern undergraduate researchers — will travel to Southern California to conduct the molecular hunt with Robert Damoiseaux, the professor of molecular and medical pharmacology who directs UCLA’s Molecular Screening Shared Resources Laboratory.
The hoped-for outcome, Burgis says, is a molecular-drug therapy that would benefit all at-risk populations: both infants with encephalopathy as well as older populations with less severe forms of the ITPA deficiency.
The project is funded by the NIH’s National Institute of General Medical Sciences.
