Some feel as if they have nothing to lose. Some are determined to contribute to scientific knowledge. Some want access to promising, although unproven, new therapies.
For a variety of reasons, almost all of Upstate’s pediatric cancer patients and roughly 10 percent of its new adult cancer patients enroll in clinical trials.
“I virtually always recommend participation if they are eligible, because I think clinical trials provide state-of-the-art treatment, and may be the next best thing,” says oncologist Stephen Graziano MD.
“Most new treatments are not proven to be better than standard therapies. But that is precisely why they are so important. There needs to be strong evidence for a new therapy to replace an established one.”
He admits that “a lot of studies don’t pan out. But the treatments that we have today were really the clinical trials of five years ago.”
Upstate has about 50 cancer-related trials under way, mostly paid for by the National Cancer Institute or drug companies.
Because most of the hospitals that treat children are part of academic medical centers, most pediatric patients are enrolled in a clinical trial as they undergo treatment.
Among adults with cancer, Graziano estimates just 2 to 3 percent participate in a clinical trial. That rises to about 10 percent at academic institutions, such as Upstate, where research is part of the mission. Adult patients are more likely to have conditions that disqualify them from participating in trials.
At Upstate both Austin Baker, 7, and Joyce Compoli, 48, are participating in trials of medications that have already been proven safe and effective. Baker takes a medication to fight leukemia, and researchers are comparing the dosage he receives with three other dosing protocols. Compoli takes a shorter course of medication than is routinely used to reduce the risk of recurrence of colon cancer, with researchers track side effects and effectiveness.
“He asked me if I would be willing to try it,” Compoli recalls of Graziano’s mention of the trial. “I said ‘sure.’ I mean, what have I got to lose?”
Compoli of Chittenango was diagnosed with colon cancer on Mother’s Day last year. She developed stomach aches and was having trouble keeping food down, so she went to her doctor, who sent her for a colonoscopy. She went for the test the Friday before Mother’s Day, and did not come home until 10 days later – after surgery to remove part of her large intestine.
After that, she took chemotherapy every other Friday for three months. It was horrible.
“Every once in a while I would get nauseous,” Compoli recalls. “I would get really emotional with it. One minute I could be happy, and the next I could be crying all day.” She wore gloves to reach into her freezer or touch anything cold because cold things burned her. She had to drink warm liquids, instead of cold, for the same reason. She lost her sense of taste and most of her hair and a great deal of energy. “I would get tired walking from the house to the mailbox, which is only like 30 feet.”
During her surgery, five of 24 lymph nodes tested positive for cancer, so Compoli was recommended for chemotherapy.
Graziano says patients have a 60 percent chance of recurrence if colon cancer involves lymph nodes. Taking the combination of chemotherapy drugs called FOLFOX every two weeks for six months can reduce that chance to perhaps 40 percent. “A lot of us feel that’s a lot of treatment,” the oncologist says. So a study underway looks at whether three months on the drug is sufficient.
The hope is, too, that a shorter course of chemotherapy will result in less severe side effects. Many people taking FOLFOX struggle with neuropathy, numbness and tingling in the fingers and toes, which usually develops toward the end of six months.
“If we could cut the treatment time short, then maybe they would not have those symptoms,” Graziano explains. “The risk, of course, is that three months may not be as effective as six.”
Compoli feels victorious. Tests done in December revealed “no sign of cancer,” she says.
The family of Austin Baker of DeRuyter feels optimistic. Austin is fighting leukemia. His mom, Amber says in one month of chemotherapy, his cancer cells dropped from 85 percent to 1 percent.
He was diagnosed March 11, 2011, a Friday. Earlier in the week, he awoke with pain in his hip, which his parents figured was a result of horsing around with his brother the day before. But the pain intensified. Xrays and blood work did not explain the pain. The next morning, Austin could not walk.
An MRI scan at Upstate revealed seven lesions on his pelvis. The next Monday, he started chemotherapy. He’s been hospitalized on and off for treatment, allergic reactions, pancreatitis, pneumonia, fevers and sinus infections. His compromised immune system means he’s susceptible to whatever germs are going around.
Amber Baker says even though the cancer cells have been reduced, her son is in a trial comparing different amounts of the same cancer drugs, given at different times. He has blood drawn every month and undergoes a spinal tap every 12 weeks; some kids in the trial have them every month.
He takes from two to nine pills per day — cancer drugs every evening and every Tuesday, pneumonia preventative on the weekends and a week-long course of steroids every 12 weeks. He gets hungry when he’s on the steroids, and battles pain when he comes off them, Baker says.
But she’s optimistic. Austin is able to attend school. His only restriction is no contact sports, due to his medication port.
“He’s got maintenance until May 17, 2014,” his mom says. “The day before he turns 10, he’ll be done with everything.”
Graziano says he enrolls patient into clinical trials almost every week, and often they are the same trials underway at big-name cancer centers such as Memorial Sloan-Kettering and Dana-Farber.
Not all patients want to participate in trials, and Graziano says that’s fine. Even those who sign up initially may change their mind. That’s OK too. “If at any point you change your mind, you tell your doctor and you come off the study,” he says. “That’s very explicit in the informed consent process.”
Learn more about Dr. Graziano.
Read more about clinical trials at Upstate.