Stem Cells Used to Deliver Drugs Directly into Brain Tumors

Stem Cells Used to Deliver Drugs Directly into Brain Tumors

Researchers at University of North Carolina Lineberger Comprehensive Cancer Center and UNC Eshelman School of Pharmacy may have found an potential new treatment for childhood brain cancers such as medulloblastoma – the most common type of malignant primary pediatric brain tumor.

When brain tumors are discovered in children they need to be surgically removed, although it is not usually possible to remove all of the cancerous tissue. Children therefore also have to undergo radiation therapy or chemotherapy; however, those treatments are not without considerable risks. Both options can be extremely toxic to the developing brain and can have lasting neurologic and developmental side effects. While treatments have dramatically improved survivability and extended the lives of children diagnosed with brain cancers, a new form of treatment is required that is more effective and less harmful to patients.

The researchers had previously demonstrated the natural ability of stem cells to find tumors and investigated how those stem cells could be used to deliver cancer-treating drugs directly into tumors. The stem cells for the study were derived from skin cells, which are easy to harvest from patients.

The harvested stem cells were genetically engineered to create a substance that is toxic to other cells. That substance is only released when the cells are exposed to another drug, which is administered once the stem cells have located the tumor.

The study was conducted on a murine model and the researchers demonstrated that following surgery, it was possible to decrease the size of the tumors by 15 times. Median survival time was extended by 133% after treatment. The researchers also used human stems cells and showed they could extend the lives of the mice by 123%.

The researchers also discovered that following the surgical removal of tumors, the cancerous cells that remained after surgery became much more aggressive.

The researchers are hopeful that their technique could be used to find and kill any cancer cells that were not removed during surgery and avoid having to treat children with radiation or chemotherapy, or at least reduce the amount of radiation or chemotherapy treatment that is required.

“This is a great avenue of exploration, particularly for the 30% of children who struggle or don’t make it with standard therapy. We want to nudge the needle even further.” study co-author Scott Elton, M.D., chief of the UNC School of Medicine division of pediatric neurosurgery.

The research has shown that the treatment is effective in the lab and is an important first step toward a clinical trial. The researchers hope that their technique can not only be used on difficult to treat brain cancers in children, but also for delivering cancer-killing drugs to treat glioblastoma – the most aggressive and deadliest brain cancer in adults.

The research is detailed in the paper – Intra-cavity stem cell therapy inhibits tumor progression in a novel murine model of medulloblastoma surgical resection – which was recently published in the journal PLOS One

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