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Stem Cell-Conventional Treatment Combo Offers New Hope in Fighting Deadly Brain Cancer

Durham, NC (PRWEB) August 07, 2013

A new type of treatment that pairs neural stem cells with conventional cancer fighting therapies is showing promise in animal studies for the most common and deadliest form of adult brain cancer — glioblastoma multiforme (GBM). The details are revealed in a groundbreaking study led by Maciej Lesniak, M.D., that appears today in STEM CELLS Translational Medicine.

“In this work, we describe a highly innovative gene therapy approach, which is being developed along with the NIH and the FDA. Specifically, our group has developed an allogeneic neural stem cell line that is a carrier for a virus that can selectively infect and break down cancer cells,” explained Dr. Lesniak, the University of Chicago’s director of neurosurgical oncology and neuro-oncology research at the Brain Tumor Center.

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

GBM remains fatal despite intensive treatment with surgery, radiation and chemotherapy. And while cancer-killing viruses have been used in clinical trials to treat therapeutically resistant and infiltrative tumor burdens throughout the brain, “there were major drawbacks,” Dr. Lesniak explained.

“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.” By using NSC loaded with a novel oncolytic adenovirus that selectively targets GBM, along with standard of care that includes chemo-radiotherapy, the team was able to overcome these limitations.

Using mice that had GBM, the research team showed how their neural stem cell line, which is derived from human fetal cells, could significantly increase the median survival time of the mice beyond conventional treatments alone. The addition of chemo-radiotherapy further enhanced the benefits of this novel stem cell-based gene therapy approach.

“Our study argues in favor of using stem cells for delivery of oncolytic viruses along with multimodal chemo-radiotherapy for the treatment of patients with GBM, and this is something that we believe warrants further clinical investigation,” Dr. Lesniak concluded.

The team is completing final FDA-directed studies and expects to start a human clinical trial, in which a novel oncolytic virus will be delivered via NSCs to patients with newly diagnosed GBM, early in 2014.

Treatment of GMB depends on novel therapies,” said Anthony Atala, M.D., Editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. “This study establishes that a combination of conventional and gene therapies may be most effective and suggests a protocol for a future clinical investigation.”

The full article, “The Timing of Neural Stem Cell-Based Virotherapy Is Critical for Optimal Therapeutic Efficacy When Applied With Radiation and Chemotherapy for the Treatment of Glioblastoma,” can be accessed at

About STEM CELLS Translational Medicine: STEM CELLS TRANSLATIONAL MEDICINE (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

About AlphaMed Press: Established in 1983, AlphaMed Press with offices in Durham, NC, San Francisco, CA, and Belfast, Northern Ireland, publishes two other internationally renowned peer-reviewed journals: STEM CELLS® (, in its 31th year, is the world's first journal devoted to this fast paced field of research. The Oncologist® (, also a monthly peer-reviewed publication, in its 18th year, is devoted to community and hospital-based oncologists and physicians entrusted with cancer patient care. All three journals are premier periodicals with globally recognized editorial boards dedicated to advancing knowledge and education in their focused disciplines.

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