Investigational cancer drug prevents abnormal brain growth, reverses seizures in lab mice

(MEMPHIS, TENN--Nov. 13, 2003) Investigators at St. Jude Children's Research Hospital used an experimental anti-cancer drug to prevent or reverse abnormal brain cell growth that is caused by lack of the anti-cancer gene Pten. The study demonstrated that the runaway cell growth in the absence of Pten is triggered by a second gene called mTor.

The anti-cancer drug, called CCI-779, not only reduced runaway growth in certain parts of the brain in young mice, but also reversed abnormal growth in certain areas of the adult mouse brain. In addition, CCI-779 reduced the frequency of seizures and the death rate in adult mice that did not have cancer but suffered neurological deficits caused by abnormal growth of brain cells in the absence of Pten.

These findings are important because they show that mTor plays multiple roles in the brain from regulating the size of individual cells to more complex functions of neurons, such as signaling, according to Suzanne Baker, Ph.D., associate member of the St. Jude Developmental Neurobiology department. Baker is senior author of a report on this study that appears in Proceedings of the National Academy of Sciences.

In addition, CCI-779 is already undergoing clinical trials for the treatment of cancer, and the current study suggests additional uses for this drug.

"Our findings have direct relevance to understanding the biological basis of a variety of human disorders caused by Pten deficiency," Baker said. "Pten deficiency in the brain can cause disorders as varied as Lhermitte-Duclos disease, ataxia, seizures and glioblastoma multiforme."

Lhermitte-Duclos disease is caused by hypertrophy (enlargement) of the granule cells in the cerebellum, which results in headaches, movement disorders, tremor and other problems. Ataxia is a loss of muscle coordination which can be seen as the inability to walk normally. Glioblastoma multiforme is a cancer of the nervous system arising in cells called astrocytes, which sup

Contact: Bonnie Cameron
St. Jude Children's Research Hospital

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