A drug derived from a natural compound used in traditional Chinese medicine may work against malignant brain tumors, a study has found.

The researchers have found a derivative of the natural compound indirubin, found in indigo plants used in Chinese medicine, to be effective in treating glioblastoma in mice. The study will pave way for future clinical trials with human participants.

Glioblastoma (GBM) is a fast-growing and aggressive brain tumor that can be fatal if left untreated. It occurs most often in the cerebral hemispheres of the brain, especially in the frontal and temporal lobes, and invades the nearby brain tissue.

After diagnosis, the average life expectancy of a glioblastoma patient is 14 to 16 months. However, approximately 1% of patients survive at least 10 years.

Treatment for GBM often faces several challenges due to the localization of tumors in the brain, resistance to conventional therapy, migration of tumor cells to nearby brain tissue, tumor capillary leakage and tumor-induced seizures.

Indirubin is a natural product that constitutes Dang Gui Long Hui Wan, a mixture of plants used in traditional Chinese medicine to treat chronic diseases such as inflammation, gastrointestinal diseases and some forms of cancer.

"The interesting thing about this drug is that it targets a number of important hallmarks of the disease. That's appealing because this type of cancer keeps finding ways around individual mechanisms of attack. So if we use multiple mechanisms of attack at once, perhaps that will be more successful," Sean Lawler, lead author and researcher at the Legorreta Cancer Center of Brown University, said.

Earlier studies showed indirubin slowed the growth of glioblastoma tumors in mice, but they could not explain the reason behind it. The researchers also had issues working with the modified drug as it was difficult to test dosage levels and efficiently deliver it to the tumor.

The team used a pharmaceutical formulation of indirubin, called 6'-bromoindirubin acetoxime (BiA), for the study.

BiA was found to be effective in slowing the growth and proliferation of tumor cells and was easier to use as an injectable cancer treatment. The drug also improved survival via effects on important immunotherapeutic targets.

"The drug impacted the immune system in these mouse experiments in a way that we think could enhance clinical immunotherapy in humans," Lawler added.

Further testing will continue to study the interactions with chemotherapy and radiation, so that they can be used in clinical trials, researchers said.

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