A few years ago, early-stage breast cancer patients enrolled in clinical trials testing whether zoledronic acid could reduce breast cancer recurrence. When the trials ended, researchers concluded that this bone-preserving bisphosphonate, used to treat osteoporosis, could also help cure breast cancer in post-menopausal women — while causing minimal side effects.
Oncology clinical trials can yield amazing benefits. They provide long-term monitored care to patients, as well as another care option and access to new medications. They also contribute to scientific knowledge that helps us address cancer, plus its high health care costs and emotional toll.
If you are interested in enrolling in a trial, I commend you. If you are uncertain, I do not fault you: 3 of every 4 people don’t understand the process. Here is a primer on what to expect, and why enrolling is becoming a more viable — and important — care option:
[See: What Your Doctors Wish You Knew.]
The Lowdown
Oncology clinical trials test whether potential therapies can treat cancers, measuring their effectiveness and determining how they work. Many trials test new drugs, while some study drugs already approved by the Food and Drug Administration to mine for new benefits.
Consider a trial if you are not satisfied with the standard cancer treatments. Begin by consulting with your doctor. Many physicians will recommend a trial, but if they do not, ask for their take. Some doctors initially overlook trials because they fear losing your business or are unaware of all existing trials.
You can also locate and enroll in trials by visiting CenterWatch.com and ClinicalTrials.gov (and patients who see doctors in my medical group may also visit our research institute’s site). Apps like Apple’s ResearchKit now offer another option, but take heed: Although such apps let you enroll more easily than traditional methods, they do not account for key factors including demographics and patient-study match.
We have to take such factors into account. To qualify for a study, you must be diagnosed with the appropriate cancer type and be healthy enough (able to hold a job, for example). You should also ask your doctor whether you can stop chemotherapy for one or two months, so researchers can properly evaluate the study drugs.
Studies are formally phased; you will be enrolled in a Phase I, II or III trial. Phase I trials test for treatment safety and proper dose. Phase II trials evaluate how participants respond to the recommended dose established by Phase I, if the drug passes Phase I (7 in 10 drugs do). Only 1 in 3 treatments then advance past Phase II to Phase III, when we assess how participants taking the treatment fare compared to a control group receiving a placebo (or a placebo combined with the standard treatment). Drugs that pass Phase III may be submitted for FDA approval, and this whole process can take five to 10 years.
Software determines which patients are divided into which group in Phase III trials, not the researchers. If you are in the control group, which you will be unaware of, you still could benefit from the trial. Placebo-controlled trials are designed to maximize care for all participants, never using placebos alone when effective therapies or potential cures are available. I have also seen the placebo effect in action; the mind is a powerful thing.
No matter which group or phase you are participating in, do not expect to benefit from trials because they are just not predictable. Trials can last several years because that’s how long it takes to evaluate how therapies work long-term within the human body, and because researchers need to study thousands of people often across diverse populations. Before you even begin participating, you must be briefed, sign consent forms and have your shared information securely stored (as it will be throughout the process).
[See: How to Disinfect Germ Hotspots.]
The Issues
Now is a great time to take advantage of oncology clinical trials. Start with promising scientific advances taking hold with targeted therapies and immunotherapies. Enrolling lets you access these extremely expensive treatments.
Private donors and government sources are taking steps to make trials more available, efficient and productive. Johns Hopkins University recently announced it is founding a $125 million institute that will conduct trials, while Vice President Joe Biden’s $1 billion “moonshot” cancer initiative strives to streamline trial enrollment. In addition, the U.S. Senate is considering one bill (already passed by the U.S. House) aimed at strengthening and modernizing trials, and another to help enroll more women and minorities in National Institutes of Health trials.
These efforts are encouraging but also necessary. Minorities are especially underrepresented in trials; less than 2 percent of National Cancer Institute-funded trials from 1993 to 2013 included enough minorities to meet NIH specifications. And while the number of registered trials internationally has increased dramatically since 2004 (the 206,658 trials in 2015 were 16 times the 2004 figure), grant spending has leveled off: It increased by only 2 percent annually from 2008 to 2014, after increasing by 9 percent annually from 2002 to 2008, largely due to declining contributions from governmental sources like the NIH.
Because of these decreasing subsidies and skewed public attention, research into many cancers is severely under-funded. While breast cancer studies receive a lot of public funding, prostate and pancreatic cancer studies get much less.
Another issue: Trials are expensive and difficult to carry out, because they need numerous patient volunteers and because small mistakes can force scientists to start over. Due to essential study checkpoints, 7 in 10 trial volunteers fail prescreening, and another 1 in 3 then fail further vetting. Researchers struggle to recruit enough patients, and sometimes delay or fail to complete trials.
You can help counter these issues, in addition to helping yourself or somebody you care about. Support a foundation addressing an under-funded cancer. Write your representatives asking for funding. And if your doctor recommends it, please consider a trial — especially if you are a minority or over 65. Most participants are younger caucasians, but different ethnicities and ages respond uniquely to treatments, and most oncology treatments are approved only through trials.
I have heard the arguments against enrolling, especially patients telling me they don’t want to be a guinea pig. But wild guinea pigs do not live as long as domestic ones. You can be better off under the close supervision of a trial than without it.
But don’t just take my word, ask a patient who has been part of a trial. Ninety-eight percent say they would consider participating again. You don’t have to know what bisphosphonates are to interpret that statistic.
[See: Which Practitioner Do I See, and When?]
More from U.S. News
17 Ways Heart Health Varies in Women and Men
7 Things You Didn’t Know About Lung Cancer
The Value of Signing Up for Clinical Trials originally appeared on usnews.com
