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Gene Therapy May Offer New Treatment for Brain Tumors

ST. PAUL, MN (May 20, 1998) ñ A gene therapy technique may provide a new treatment for fatal, cancerous brain tumors, according to a study published in the May issue of Neurology, the scientific journal of the American Academy of Neurology.

Researchers infected human brain-tumor cells with a tumor-supressor gene which made genetically engineered retinoblastoma (Rb) protein. The Rb-treated tumor cells were placed in mice. After 30 days, tumor cell growth was suppressed and tumors were unable to form.

"These findings suggest that restoring normal Rb activity in brain tumors, know as gliomas, may have therapeutic effects," said neurologist Alfred Yung, MD, of the University of Texas M.D. Anderson Cancer Center in Houston, TX.

Yung said more research is needed before the therapy becomes available to patients. Human trials may be possible within two or three years.

Malignant glioma is brain tumor that is highly resistant to conventional therapies such as surgery, radiotherapy and chemotherapy. Over the past decade, research has improved the understanding of brain cancer; however, with few exceptions, the prognosis remains grim.

"This preliminary study provides initial steps toward future improvements in the treatment and care for patients," according to an accompanying editorial written by Lisa M. DeAngelis, MD, of Memorial Sloan-Kettering Cancer Center in New York.

Rb protein controls cell growth. When Rb is inactive or abnormal, tumor cells move into the next phase of the cell cycle, leading to uncontrolled cell growth and eventually cancer.

Gene replacement requires a vehicle, usually a viral vector, to deliver the therapeutic genes. In this study, researchers infected mice with an adenovirus, the vehicle in which Rb gene was delivered to cells.

Gene therapy has limitations in treating human gliomas, according to DeAngelis. "Because people have vast differences in their tumor genetics, not all gliomas will respond therapeutically to Rb treatment," she said.

Yung said his future research would focus on identifying other abnormal genes, replacing them and learning more about their possible therapeutic effects.

The American Academy of Neurology, an association of more than 15,000 neurologists and neuroscience professionals, is celebrating its 50th year of improving patient care through education and research.

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