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Hui Li, PhD, and Team Identify Potential Treatment for the Deadliest Brain Cancer

Hui Li, PhD
A team of UVA Comprehensive Cancer Center researchers led by Professor of Pathology Hui Li, PhD, has identified a small molecule that blocks the gene responsible for glioblastoma, offering hope of a new and more effective treatment for this aggressive brain cancer that is currently always fatal. The finding follows the groundbreaking 2020 discovery by Dr. Li and colleagues that the hyperactive AVIL gene is required for glioblastoma’s formation and spread, presenting a potential target for treatment. The new AVIL inhibitor molecule was not only found to slow tumor growth in lab models, but did so without damage to healthy tissue or other harmful side effects. It also is capable of crossing the body’s blood-brain barrier that thwarts some neurological treatments, and could be taken orally in a pill. Dr. Li and team are working to adapt the compound to be administered in potential human trials. Their research, funded by the National Institutes of Health and the Ben and Catherine Ivy Foundation, was published in Science Translational Medicine. Read more in the UVA Health Newsroom.
Cancer Center Team Awarded $13 Million to Lead National Study Seeking New Treatments for Acute Myeloid Leukemia
UVACCC Director Thomas P. Loughran Jr., MD, and Professors of Medicine/Hematology and Oncology Charles Chalfant, PhD, and David Feith, PhD, have been awarded a five-year, $13-million National Cancer Institute (NCI) P01 program project grant to lead a multi-institution, interdisciplinary study seeking new therapies for acute myeloid leukemia (AML). The deadliest form of leukemia, AML is diagnosed at a median age of 68 and its responsiveness to treatment varies between patients. Ten previous years of NCI-funded UVACCC research found that measuring sphingolipids in AML cells can help determine if patients are at greater risk of chemotherapy resistance and should pursue alternate therapies, including clinical trials. The new study seeks to further study and categorize sphingolipids in AML, and to develop and test new treatments delivered via nanotechnology to reduce ceramide metabolites that support the disease’s spread, and replace them with ceramides that will kill it. Read the full article about the grant.
‘Mismatched’ Transplants Now Safe for Blood Cancer Patients

Karen Ballen, MD, PhD
In a major advance for treatment of blood cancers, a study co-authored by Professor of Hematology and Oncology Karen Ballen, MD, found that patients can safely receive “mismatched” stem cell transplants by administering the drug cyclophosphamide to prevent graft-versus-host disease. Researchers saw one-year survival rates of 80 percent among 145 patients — similar to outcomes with fully matched donors. Just 10 percent developed moderate to severe complications. Dr. Ballen, UVA Health’s Chief of Hematology/Oncology and Medical Director of Stem Cell Transplants, said all eligible transplant patients at UVA last year were able to find a suitably matched donor thanks to this progress. The findings, published in the Journal of Clinical Oncology, could greatly expand access to lifesaving transplants for leukemia, lymphoma and myelodysplastic syndrome. Read the full article about the discovery.
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