Don Clarke, a student working with Dr. Michelina Iacovino out of the Child Health Research Center took top honors at a state-wide student research competition.
Clarke, a student at California State University, Los Angeles, presented his lab research on Development of a Stem cell/gene therapy approach to treat Sanfilippo syndrome type B at the competition’s final round held on the campus of California State University, Bakersfield.
Clarke won the biological and agricultural sciences section of the competition. For Clarke, the research is what makes it all worthwhile.
“We’re developing a stem cell gene therapy to treat MPS3-B,” he says. “So we’re using corrected neural stem cells to quantify their ability to produce the enzyme that causes the disease. The hope is that we can get these engineered cells into patients that are lacking this enzyme. It’s the idea that these cells will be able to engraft and continue to supply the body with these enzymes.”
Their initial work is simply ensuring that the cells that they’ve made are capable of secreting enzyme that itself are capable of being taken by the cell.
“It’s the very base of this project but as it moves forward, we’re going to move to the mouse model and see if these cells are capable of doing their job.”
There is currently no treatment for MPS3-B. The enzyme replacement therapy that works on other MPS disorders does not work on MPS3-b. People who suffer from MPS3-B have profound intellectual disabilities. Really bad behavioral disturbances. But mostly a neurotic disposition.
Clarke couldn’t be happier with the experience he’s gained while working in the Child Health Research Center.
“It doesn’t feel like a job,” he says. “The translational research is undoubtable. It’s challenging and it makes you want to cry sometimes but I love doing it. It’s great.”