Tammy Kielian, Ph.D., professor of pathology and microbiology, has received a two-year, $500,000 National Institutes of Health grant for pre-clinical testing of therapeutic compounds for Juvenile Batten disease.
Dr. Kielian’s lab is testing two classes of drugs that have anti-inflammatory activity, which may delay some of the pathology associated with the disease.
Juvenile Batten, or juvenile neuronal ceroid lipofuscinosis, is an inherited, autosomal recessive, neurodegenerative condition resulting in an abnormal amount of protein and lipids becoming trapped inside the cells. Accumulation in brain cells results in neuron death. It leads to loss of vision, motor coordination and cognition. The condition is eventually fatal.
Dr. Kielian’s research will study mouse models with the equivalent of Juvenile Batten, to see if their outcomes are improved following treatment.
“We’re trying to target what we believe to be deleterious inflammation in the brain, in the cells that support the neurons,” Dr. Kielian said. “If we could do that perhaps we could prolong the survival of the neurons.”
Dr. Kielian, who has been honored as a UNMC Distinguished Scientist and with UNeMed’s Emerging Inventor Award, has investigated immune responses in the brain for more than a decade. She first realized her research interest might be applicable to Juvenile Batten when her niece, Olivia, was diagnosed with the disease.
For more on Olivia, and how she inspires Dr. Kielian’s work, read this story.
Awesome Dr. Kielian!!!! Congrats!