Stem cell combo therapy extends survival of mice with brain tumors

Glioblastoma multiforme--Courtesy of NIH

A new treatment that combines neural stem cells with chemo-radiotherapy drugs could provide a better way to combat glioblastoma, the most common and lethal form of adult brain cancer.

A team at the University of Chicago headed by Dr. Maciej Lesniak, director of neurosurgical oncology and neuro-oncology research at the Brain Tumor Center, has tested the new gene therapy procedure in animals with glioblastoma multiforme (GBM).

The researchers developed a neural stem cell line, called HB1.F3 NSC, which is derived from human fetal cells. The stem cell line acts as a carrier for an oncolytic virus--a virus that infects and destroys cancer cells while leaving normal cells intact. In animal studies, researchers found that mice survived significantly longer following treatment with the novel stem cell therapy than with conventional treatments alone. Combined with standard-of-care chemo-radiotherapy, the stem cell therapy was even more effective. The research appears in STEM CELLS Translational Medicine.

The stem cell line used in the research, HB1.F3 NSC, was recently approved by the FDA for use in a Phase I human clinical trial.

Glioblastoma is the most aggressive kind of brain tumor, and even with intensive surgery, radiation and chemotherapy, the cancer is fatal. But scientists have yet to figure out how to tap viruses to fight cancer, and so far, their use has been limited in clinical trials.

"When you inject a virus into a tumor alone (without a carrier, like NSC), the virus stays at the site of the injection, and does not spread. Moreover, our immune system clears it. By using NSCs, we can achieve a widespread distribution of the virus throughout the tumor mass, since the NSC travel. Also, they act like a stealth fighter, hiding the virus from the immune system," Lesniak said in a statement.

The Chicago team is aiming to begin a human clinical trial, in which a novel oncolytic virus will be delivered via HB1.F3 NSC neural stem cells to patients with newly diagnosed glioblastoma, in early 2014.

"Treatment of GBM depends on novel therapies," said Dr. Anthony Atala, editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine, in a news release. "This study establishes that a combination of conventional and gene therapies may be most effective and suggests a protocol for a future clinical investigation."

- here's the study abstract
- read the press release