March 16, 2015 | by Sumanta Kumar Pal M.D.
"Tonight, I'm launching a new precision medicine initiative to bring us closer to curing diseases like cancer."
These were the words of President Barack Obama on Jan. 20, 2015, during his State of the Union address. So what is precision medicine, and how close are we to making it a reality for patients?
Let’s begin with some definitions. Precision medicine entails two general tenets: (1) understanding the biology of an individual patient’s cancer, and (2) treating the patient according to this biology. When I was in medical school, both elements would have been perceived as a pipe dream. Now, they are slowly becoming a reality.
Comprehensive genetic tests that used to take days to weeks to run can now be done in minutes; these tests can point an oncologist toward specific medications that target proteins at the cancer cell surface, while sparing normal cells. In contrast to chemotherapy (which attacks cells indiscriminately), targeted therapies may potentially have fewer side effects.
One example of progress is in the treatment of bladder cancer. The disease doesn’t often garner headlines, but it has a substantial toll in the U.S., with an estimated 74,000 diagnoses and 16,000 deaths in 2015. When bladder cancer spreads, or metastasizes, to surrounding areas (e.g., the lungs or liver), it is invariably fatal. No drugs have been approved for metastatic disease in over three decades, and the average patient with metastatic disease lives for only one year.
So how might precision medicine change these grim statistics?
I recently presented genetic data from more than 400 patients with bladder cancer at the Genitourinary Cancers Symposium, a meeting of over 2,000 oncologists and surgeons in Orlando, Florida. These genetic data were obtained through use of a commercially available test called FoundationOne. For the first time in such a large series of patients, we were able to characterize common gene mutations in bladder cancer.
Remarkably, nearly a third of these mutations were “actionable,” meaning targeted treatments were available to treat them.
One of the cases detailed in my study was Jyoti Shah, a patient of my own. Ms. Shah remains healthy and active at the age of 61, although this had almost come to a screeching halt with her diagnosis of bladder cancer. The disease had spread deep into her pelvis, and the standard chemotherapy drugs were failing. While the survival prognosis in this scenario is just a few short months, we were able to detect mutations in her cancer that pointed us toward a drug called everolimus. With the addition of everolimus to her chemotherapy regimen, her cancer disappeared over a span of one year.
In Ms. Shah’s case, the lifesaving potential of precision medicine is already a reality. Within the next five to 10 years, investigators at City of Hope and around the world will increasingly band together to conduct clinical trials to vet this strategy.
Disclosure: Sumanta Pal receives consulting fees from Novartis Pharmaceuticals, the maker of everolimus.
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