Three Baylor College of Medicine researchers received support totaling $500,000 from the nonprofit organization Alex's Lemonade Stand Foundation for their work to find more effective treatments for childhood cancer.

Innovation grants were awarded to Dr. Margaret Goodell, professor of pediatrics ­ hematology/oncology, and Dr. Leonid Metelitsa, associate professor of pediatrics ­ hematology/oncology. Dr. Meenakshi Hegde, a clinical postdoctoral fellow in pediatrics ­ hematology/oncology, received a Young Investigator Award.

"We're grateful for the support of Alex's Lemonade Stand. They are an important partner in helping us reach our goal of finding better, more effective ways to treat childhood cancer that could eventually lead to a cure," said Dr. David Poplack, professor of pediatric oncology at BCM and director of the Texas Children's Cancer Center.

Role of gene LYL1

The grant funding to Goodell will support her research on the role the gene LYL1 plays in T-Cell ALL, one of the most frequent types of childhood leukemia. Researchers hypothesize that the overexpression of LYL1 plays a key role in generating T-ALL precursor cells. The research will examine in mice the mechanism through which LYL1 exerts its effects, with the hope to eventually develop strategies to interfere with the role of the gene in malignancy development.

T (NKT) cells

The grant to Metelitsa will help fund his ongoing research to understand how special cells called natural killer T (NKT) cells can be used to suppress neuroblastoma tumor growth. The project will use NKTs from neuroblastoma patients and genetically modify them with two proteins that have been shown in mice to protect and enhance the NKT cells' anti-tumor effectiveness.

"Neuroblastoma is a common solid tumor in children. About 60 percent of children with high-risk neuroblastoma are not cured, so new treatment strategies based on the understanding of body's immune system are needed to treat this disease," Metelitsa said.

Targeting two proteins

The Young Investigator Award to Hegde will support research that targets two proteins simultaneously that play a role in recurrent glioma brain tumors. Researchers will genetically engineer T-cells to become both HER2 and IL-13Ra2-specific and test in the lab and in animal models, if this results in better tumor control over targeting one protein only. High-grade gliomas constitute nearly 20 percent of all childhood brain tumors and even with aggressive therapy do not respond well to treatment. Expected survival rarely exceeds two years, and less than 25 percent of patients survive five years from diagnosis, Hegde said.

"It has always been our goal to find better treatments and cures for all kids with cancer by funding the best research possible," said Jay Scott, co-executive director of the foundation.