Investigators at Arkansas Children’s Research Institute (ACRI) and the University of Arkansas for Medical Sciences (UAMS) will use a $3.2 million award from the National Institutes of Health to lay the foundation for a new treatment that could transform quality of life for children with lymphatic malformations.
A type of vascular anomaly of the lymphatic system, these malformations often cause breathing and feeding difficulties for children. Patients also frequently experience pain, infections, and disfigurement.
Graham Strub, MD, a pediatric otolaryngology-head and neck surgeon at Arkansas Children’s and an associate professor of otolaryngology-head and neck surgery at UAMS, will lead the research team during the five-year study. He and members of his laboratory have been collecting tissue and blood from lymphatic malformation patients with the goal of developing new treatments to reverse their growth and development.
Using the novel approach of comparative multi-omics, Strub’s laboratory discovered the abnormal expression of several genes that appear to drive lymphatic malformation growth. Multi-omics is a holistic approach that combines data from genomics, transcriptomics, epigenetics and proteomics to understand molecular changes that contribute to normal development, cellular response and disease.
Collaborating with Robert Griffin, MD, a professor of radiation oncology at UAMS, they will study how microRNAs, which are small molecules that silence the expression of specific genes, can reverse this abnormal gene expression.
The teams are hopeful this novel technology will improve treatment outcomes, reduce side effects, and expand the understanding of the epigenetic regulation of the lymphatic system.
“Current treatments for lymphatic malformations have many limitations and often require multiple interventions over a long period of time,” Strub said. “The development of transdermal microRNA therapeutics that silence the genes responsible for lymphatic malformation growth could significantly improve the quality of life of these children.”
With this NIH award, Strub is a graduating research projectlLeader from the Center for Translational Pediatric Research, an NIH-supported Center of Biomedical Research Excellence at ACRI.
This research is supported by the National Heart, Lung and Blood Institute of the National Institutes of Health under Award Number R01HL173107.