Today's astonishing disease-fighting drugs and therapies are giving hope—and the possibility of a long, healthy life—to thousands of patients. Go behind the scenes with oncologists, drug companies, and the science of optimism.
Carol Turtur, 55, would rather be skiing. Or better yet, heading down the Danube by boat. But this January morning in 2006, all vacation plans are on hold. The former flight attendant is reporting to the University of Texas M.D. Anderson Cancer Center in Houston to test an experimental cancer treatment. As one of the patients in a preliminary trial, she will be helping doctors and a drug company determine whether human milk fat globule-1 antibody is safe and, if so, at what dose. She is also hoping for a miracle. For the past ten and a half years, Turtur has been battling breast cancer. “You keep waiting for the one treatment that is going to work. You want to be the one,” she says, caught up in the enthusiasm of science's new high-tech push to find a cure.
A look at Turtur's treatment itinerary shows she has tried mightily to be that pioneer. In 1995 she underwent a double mastectomy and six months of chemotherapy. But just shy of the five-year mark, a routine chest X-ray revealed inoperable breast cancer that had spread to her lungs. She refused more chemotherapy, so Francisco Esteva, MD, PhD, her physician at M.D. Anderson, treated her first with tamoxifen, the warhorse drug that inhibits estrogen's ability to promote breast cancer cell growth, and when that didn't work, with two aromatase inhibitors to lower estrogen levels. After a few months on each, with little progress against the disease, Esteva put her on a new medication called Herceptin.
Herceptin, introduced in 1998, was a revolution in the treatment of cancer—arguably the first “targeted” drug on the market. While radiation and chemotherapy indiscriminately destroy cancerous and healthy cells alike in a scorched earth policy against disease—often leaving the patient miserable and struggling into remission if she is lucky enough to live—targeted drugs elegantly block specific pathways involved in cancer cell growth. Herceptin, for example, binds to HER-2, a protein that, when present in excessive quantities on the surface of certain cells, causes a particularly virulent form of breast cancer. The drug works by attaching to those cells and halting their growth while, theoretically, healthy cells remain intact. For Turtur it had an immediate effect, reducing her cancer by half in only two months.
About six months passed before the cancer began progressing again, but this time so slowly, she didn't feel the symptoms for almost five years. In July 2005, with more than one-third of her lungs affected, she started getting winded climbing stairs. Luckily, a therapy that slows the growth of tumors by targeting their blood supply had been approved just a year earlier for colorectal cancer. At Esteva's suggestion, Turtur began taking the drug, Avastin, with Herceptin to see if it might hobble her cancer as well. This time, too, the results were astounding. By November 2005, her tumors had noticeably diminished.
Last year, when the cancer started advancing again, she hoped for the same results with human milk fat globule-1 antibody (designed to get the immune system to start attacking breast cancer cells). Unfortunately, that didn't work; nor did a second experimental targeted drug called RAD001. So last fall, Turtur started back on chemo (with Herceptin), but in a new formulation that uses nanoparticles and “is a little more targeted, if you will,” says Esteva. Amazingly, her tumors have shrunk again, her symptoms vanished.
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