Stem Cell-Derived Therapy Successfully Treats Glioblastoma

Findings from a new study published by Science Translational Medicine demonstrate that stem cells may be able to kill brain cancer cells.

These findings may lead to significant advances for patients with brain cancer who have few effective treatment options.

Previously, the team engineered mouse skin cells to stem cells that are able to target and destroy human brain cancer cells. The investigators found that this method increased time of survival 160% to 220%, depending on the type of tumor.

In the new study, the authors observed that this technique is effective in humans, and can work quickly enough to treat patients who otherwise would have survived an average of 18 months.

“Speed is essential,” said lead researcher Shawn Hingtgen, PhD. “It used to take weeks to convert human skin cells to stem cells. But brain cancer patients don’t have weeks and months to wait for us to generate these therapies. The new process we developed to create these stem cells is fast enough and simple enough to be used to treat a patient.”

The rate of survival after 2 years is only 30% for these patients, indicating a strong need for speedier treatments.

Current treatment methods include surgery, radiation, and chemotherapy. While these methods have been in place for more than 30 years, they have been seen to offer limited benefits.

Glioblastoma recurrence is seen in nearly all patients within months. This time, the cancer sends out tendrils into brain tissue, making it impossible to remove all of the cancer, since they cannot be seen or reached by drugs, according to the study.

“We desperately need something better,” Dr Hingtgen said.

The novel treatment is contingent on creating neural stem cells derived from skin cells, a process called skin flipping. For this approach, the researchers harvested fibroblasts from the patient, and reprogrammed the cells to become induced neural cells, according to the study.

These stem cells have the natural ability to target glioblastoma cells. However, the stem cells cannot kill the cells on their own, but rather bump into them. To remedy this, the investigators engineered the stem cells to carry a drug to kill the cancer cells.

The engineered stem cells carry a protein that activates a prodrug that is administered to the patient. The cells then create a halo of drug around the stem cell, and kills the surrounding cancer cells. Since the drug is not administered throughout the body, the patient will likely not experience widespread adverse events, according to the study.

“We’re one to two years away from clinical trials, but for the first time, we showed that our strategy for treating glioblastoma works with human stem cells and human cancers,” Dr Hingtgen concluded. “This is a big step toward a real treatment — and making a real difference.”