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A novel anti-cancer drug, CCI-779, has shown promise in safely producing anti-cancer responses in patients with some types of advanced cancers. (European Society of Medical Oncology, 2000).

Many different proteins exist within normal cells, that modulate chemical signals and pathways in order to maintain control of cellular proliferation (multiplication). The mammalian target of rapamycin, (mTOR), is one of these proteins, and is responsible for facilitating cellular proliferation through two known pathways.

In order to maintain control over excessive or constant cellular proliferation through the mTOR pathways, a suppressor protein is activated, which halts the activity of mTOR. Through the suppression of mTOR, cellular proliferation stops. A large percentage of cancer cells possess a genetic alteration (mutation) within the gene responsible for the mTOR suppressor protein. With a non-functional suppressor protein, the activity of the mTOR pathway remains constantly activated. This results in excessive, uncontrolled proliferation of cells – the hallmark trait of cancer.

CCI-779 is a drug that binds to a specific site on the mTOR protein. This binding action disables mTOR from facilitating the proliferation of cells. CCI-779 has shown promise in some cancers and is still in the initial phases of clinical trials. Presently, researchers are attempting to determine against which cancer types CCI-779 is most effective.

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One initial clinical trial was recently conducted in France to determine the safety of CCI-779 in 16 patients with different types of cancer. All patients tolerated the treatment well and reported only mild side effects. In addition, 6 patients achieved anti-cancer responses with CCI-779.

Researchers from the University of Texas Health Science Center also performed a trial to determine the safety and tolerability of CCI-779 in 51 patients with various types of cancer. These patients also tolerated the treatment well and nine of them achieved anti-cancer responses with CCI-779.

Two multicenter trials are currently underway involving patients with renal cell (kidney) cancer and breast cancer, as these cancer types consistently showed a response to CCI-779 in previous clinical trials. In addition, researchers are considering clinical trials that involve patients with prostate cancer and glioblastoma, as many patients with these cancers have mutated mTOR suppressor genes. Future clinical trials involving CCI-779 are crucial in order to understand which cancers respond well to this treatment and how CCI-779 can be used in combination with other treatments to produce optimal outcomes for these patients.

Patients with renal cell, breast, prostate or glioblastoma cancer may wish to speak with their physician regarding the risks and benefits of participating in a clinical trial further evaluating CCI-779.(The Lancet Oncology, Vol 1, No 4, pp 198-202, 2000)

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