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Attacking Cancer on All Fronts

The bench-to-bedside approach to research is not just a catchphrase. When science is at its best, it can happen quite often, although sometimes the connections emerge only after time.

For example, in the mid-1980s, Lewis Cantley and colleagues in Boston made the seminal discovery of a new enzyme, called phosphoinositide-3-kinase or PI3K. PI3K is present in all cells and plays a critical role in a variety of cellular functions, as well as in insulin signaling and many types of cancer. Dr. Cantley's research has helped establish that PI3K gene mutations are often present in various cancers, particularly those that affect mainly women, including breast, cervical, ovarian, and uterine cancers. Recently, he has been investigating which patients are likely to benefit from targeted therapy with PI3K inhibitors.

After joining Weill Cornell in 2012 to head our new Meyer Cancer Center, Dr. Cantley has been collaborating with some of our clinical researchers on PI3K inhibitors, including hematologist and oncologist Richard Furman. A recent clinical trial led by Dr. Furman tested an experimental PI3K inhibitor called idelalisib in patients with relapsed chronic lymphocytic leukemia, a type of blood cancer. These patients could no longer be treated with chemotherapy and were given standard treatment with or without idelalisib, which is taken as a twice-daily oral pill. After 6 months, cancer had not progressed in 93% of patients given idelalisib, as compared to 46% receiving placebo. Idelalisib also improved survival, so the study was ended early and all patients were given the experimental drug.

Although not yet approved, idelalisib is currently being tested for other types of blood cancer. While it does not offer a cure, it may help patients avoid the toxic effects of chemotherapy and transform their disease into a chronic condition that can be managed with a simple pill.

The trajectory of this story--stretching from Cantley's original discovery of the PI3K enzyme to the development of PI3K inhibitors and their subsequent testing by clinical researchers like Furman--epitomizes for me the power of translational research to improve patients' lives.

Posted April 10, 2014 1:07 PM | Permalink to this post