Clinical Trials for Bladder Cancer
SSometimes, numbers can tell a story but they sometimes obscure it. According to recent data from the National Cancer Institute (NCI), the bladder cancer death rate has not decreased in 30 years. But experts who treat the disease tell a different and more hopeful tale—and they credit a surge in clinical trials for much of it. “In just the last five or six years, there has been quite a bit of work looking at new therapies for patients with bladder cancer, and we’re seeing unprecedented responses with some of these new drugs,” says Dr. Stephen A. Boorjian, professor and chair of the department of urology at Mayo Clinic in Rochester, Minn.
One class of drugs known as immune checkpoint inhibitors has been instrumental in the treatment of bladder cancer. They shift a person’s immune system activity in ways that help it fight cancer cells. “These have been paradigm-changing,” Boorjian says. “The way we treat bladder cancer is different than it was just five years ago.”
Many others working in this field share a similar tale. The past 50 years have seen the development of many promising treatments for bladder cancer patients, both nonmetastatic and metastatic. While some of these treatments are revolutionary, others provide incremental improvements that make a significant difference in patient care. All of these advances depend on the clinical trials that are conducted and the participation of those involved.
“Clinical trials are how we got here,” says Dr. Guru Sonpavde, director of the bladder cancer program at Dana-Farber Cancer Institute and associate professor of medicine at Harvard Medical School in Boston. “All the new drugs we have coming out that have changed the outcomes for patients with bladder cancer—all of this has come from clinical trials.”
A clinical trial is a carefully planned research study that takes place in a medical environment. It helps experts find new and better ways to diagnose or prevent disease. “Participating in a trial is often the only way to get access to new and promising drugs, or to therapeutic alternatives that may be better than standard of care,” Sonpavde explains.
While most types of cancer are under investigation, bladder cancer could be a hot area for clinical trials. Bladder cancer is sixth most prevalent cancer in America, accounting for more than 85,000 new cases each year. There was another reason: until recently there had been little progress in the treatment of bladder cancer. “We had some chemotherapies, but this was a cancer where we hadn’t seen many advances until about five years ago,” Sonpavde says.
Boorjian highlights, like Boorjian’s, the revolutionary effect immune checkpoint inhibitions have on the treatment and prevention of bladder cancer. This and other advancements have led to an explosion in new research—and with it, a need for more people to participate in clinical trials.
This need is unfortunately not being met. “There are so many drugs and therapies being explored in this space that in some cases there hasn’t been enough patient participation to validate study results,” says Dr. Ashish Kamat, an endowed professor of urologic oncology and director of bladder cancer research at MD Anderson Cancer Center in Houston. “To obtain reliable results, trials need lots of patients, and until we have greater participation it’s going to be hard to move the needle as much as we’d like.”
Where to look for clinical trials
Kamat says that there is a problem in that many bladder cancer patients may not realize they have the right to participate in a clinical study. Cancer doctors—especially those not affiliated with major research institutions—often fail to bring up these opportunities with their patients. “I’ve given educational talks to patient groups, and people have come up to me and said they had no idea this option existed,” he says. “In some cases they’ve ended up coming to Houston and participating.”
His advice is to have patients talk about the matter. Asking your doctor whether clinical trials are worthwhile could help get things started. Sites such as ClinicalTrials.gov (maintained by the U.S. National Library of Medicine) provide current information regarding research trials. Search for current clinical trials within your city or state to find them. Many major research and health institutions have their own websites that provide information about clinical trials for patients. Online resources can be found at MD Anderson and Dana-Farber as well as Mayo Clinic.
A clinical trial is not for everyone who has bladder cancer. Some patients may find the standard of care the best treatment. “For a patient that has low-grade bladder cancer, a resection”—that is, surgical removal of the tumor—“has a very high cure rate,” Kamat says. “For this -patient, it may not be advisable to participate [in a trial] because the standard of care is so effective.”
On the other hand, the prognosis for those with metastatic bladder cancer—that is, a cancer that has spread to other parts of the body—may make the prospect of a trial more appealing. “Unfortunately for patients in this space, existing therapies aren’t providing durable long-term survival,” he says. They may have the best chance to surpass those averages by participating in clinical trials. The trials could also offer alternative options to surgical procedures. Kamat says that among those with non-muscle-invasive bladder cancer—a group that makes up about 75% to 80% of new bladder cancer -patients—some don’t respond well to the current drugs. This group of patients must normally have their bladders surgically excised. “This is a life-changing invasive procedure, and clinical trials may provide another option,” he says. Of course, every case is different and needs to be evaluated by a patient’s physician.
It is possible that some people are hesitant about signing up for this experiment. To ensure participants don’t face undue risk, clinical trials have strict regulations. Dana-Farber’s Sonpavde says clinical trials are always built on a foundation of prior work that suggests the intervention—that is, the new treatment or other thing being studied—is at the very least not excessively risky compared with the current standard of care, and that there is evidence it may be beneficial. “Once a trial happens, you already have basic research showing the intervention is promising,” he says. There are always risks. However, there are potential rewards such as accessing better care for your cancer.
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What it’s like to participate in a clinical trial
Let’s say you and your care team have identified a clinical trial that could be a good fit. You’ll next meet with someone affiliated with the trial who will make sure that you understand exactly what the experiment entails. You’ll get a thorough explanation of the study’s design, as well as any potential risks or drawbacks.
If you decide you want to proceed—that is, if you give your informed consent—you’ll likely undergo some type of screening process to ensure you meet the trial’s criteria. It may involve additional testing—scans, biopsies, blood tests—as well as an examination of your medical records. “Sometimes we start doing these evaluations and something comes out that renders the patient ineligible,” Sonpavde says. You may have a genetic condition that is not compatible with study guidelines or be on medication that could interfere with treatment. But if screening goes well, you will likely begin the study soon—usually within one to three weeks.
It’s important to highlight that, in many clinical trials, some people do not get the new drug or novel treatment. In other words, they’re part of a “control” group that will help the study team assess whether the new intervention provides a benefit. “Generally, the trial will be comparing the new treatment to the standard-of-care treatment,” Sonpavde explains. In other words, even if you don’t get the new treatment, you’ll generally be no worse off than if you hadn’t enrolled.
Another benefit of participating in clinical research is that in some cases—whether you’re getting the new intervention or the control intervention—the cost of your treatment will be covered by the trial. Outside of a clinical-trial setting, some cutting-edge drugs would cost thousands of dollars—or they may not be accessible at all.
However, most clinical trials will not pay for lodging and transportation costs. This can be a major barrier for those who don’t live in cities or near research institutions. Participants are often not paid.
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What’s happening now
Nearly every type of bladder cancer has been the subject of clinical research. However, there are a few areas that have been particularly studied.
Boorjian claims that non-muscle invasive bladder cancer is one of the fastest-growing areas of research. Adenoviral vector therapy was something he recently conducted. “This involves instilling the novel medication into the bladder to stimulate the immune system to attack the bladder cancer,” he says. His trials showed promising results and his team is currently looking for participants to similar studies. Boorjian said that a lot is being done in the meantime to find new, more effective ways of delivering treatment. Some trials look at time-released drug delivery methods, which could offer benefits over intravenous injections. “We’re looking at better ways to administer medications to minimize the risk of toxicity,” he says.
A hot topic in bladder cancer research, is personalized treatments for metastatic cancers. Precision medicines are directed to specific tumor targets. Boorjian mentions some new classes of therapies that are designed to target specific genetic mutations within a person’s cancer cells. “We’re starting to target therapies to those tumors, and this is another move-the-field-forward change,” he says.
Change the story
National Cancer Institute predicts that more than 80,000 bladder cancer patients will be diagnosed this year. More than 17,000 patients with bladder cancer will not survive to 2023. These figures can be improved by clinical trials, and those who take part in them.
“The only way we make further advances is for patients to take part in this work,” Sonpavde says. There are risks involved in participating in trial participation. Not everyone is a good match. But you lose very little—and stand to gain a lot—by exploring your options with your care team.
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