New Target for Treating Glioblastoma May Increase Survival Time

Researchers from the Peter O’Donnell Jr. Brain Institute and Harold C. Simmons Comprehensive Cancer Center at the University of Texas (UT) Southwestern may have found the ultimate drivers of glioblastoma cell proliferation.

Glioblastoma is an aggressive brain cancer that has not seen large gains in survival over the past 3 decades of research. However, researchers believe they have discovered a new target for treating glioblastoma. Their findings were published in Cell Reports.

According to co-senior author Robert Bachoo, MD, PhD, of the UT Southwestern Medical Center, the findings change the understanding of the molecular basis of glioblastoma. The findings represent a set of critical genes shared across nearly all glioblastomas that can be targeted by drugs.

“Our work shows that the gene mutations which the pharmaceutical industry and clinicians have been focusing on are essential only for starting tumor growth,” Bachoo said in a statement. “Once the tumor has advanced to the stage where patients seek treatment, these mutations are no longer required for continued tumor growth; they are in effect redundant.”

The current standard of care for glioblastoma is surgery followed by chemotherapy and radiation. This regimen increases median survival by just 4 to 6 months, on average. Bachoo and colleagues identify a protein that drives the growth of brain tumors and believe that targeting it may inhibit the growth of gliobastoma.

According to the researchers, master proteins regulating the activity of genes during brain development are reactivated and drive the growth of glioblastoma. However, the authors believe that these transcription factors can be inhibited through the use of mithramycin, a chemotherapy drug that is known to cause liver toxicity in some patients and has not been in clinical use for years. Repurposing the drug with safer treatments could take years, but it does present some hope.

“Our discovery has the potential for the development of a new therapy that may increase survival time for glioblastoma patients,” said co-senior author Ralf Kittler, PhD.