Many advanced cancers are invisible or silent, ravaging internal organs behind the scenes until they have spread far enough to make an early death inevitable. This was the case for both Don and Mike, but they didn't give up:
- If you'd bumped into 56-year-old Don on a typical day in late 2011, you might not have even suspected that he was sick. A bluegrass musician, Don spent his time strumming his guitar to Willie Nelson's "Mountain Dew," walking his dog Charley, or spending quiet time with his family. By all signs, he was a mellow, happy guy with everything to live for. Yet looks can be deceiving. At the same time Don was enjoying life, he was facing nearly certain death from pleural mesothelioma, an asbestos-triggered cancer. He was committed to living every day to the fullest and not letting the mesothelioma dictate how he lived: "Keep yourself busy," he said. "Just do something that keeps your mind from thinking about what could happen." For Don, that included enrolling in a clinical trial.
- In 1999, Mike was vigorous and active, with a particular love of biking. After returning from a grueling Latin American trip, at age 58 he went to the doctor thinking he'd picked up a bug that was making him feel "punk." Instead, he received a Stage IV pancreatic cancer diagnosis, with the prediction that he would only survive 4-6 weeks if treated with standard-of-care drugs. "Pancreatic cancer is like a big tractor-trailer barreling down the street right at you," his wife explained. Mike was determined to extend his life to the degree possible, so he embarked immediately on the first of a series of clinical trials.
Both Don and Mike confronted the dilemma that many thousands of patients face when diagnosed with an advanced, invisible, and usually fatal cancer: What do I do if I've exhausted standard treatments? For them, clinical trials may be the only hope for extending life and may contribute to breakthrough cures for diseases that are today incurable.
Invisible Cancers: Greatest Needs for New Therapies
Mesothelioma is relatively rare in the U.S., with only 2,500-3,000 new documented cases per year and over 2,600 deaths. It rarely grows as a solid, surgically removable mass. Rather, for many people spreads along the linings of lungs and chest along organ, abdominal, and chest cavity surfaces and along nerves and blood vessels. As a result, it may be impervious to the surgery or radiation therapies typically used in treating many other cancers. In this regard, mesothelioma is like many other somewhat rare, advanced mucinous (fluid) variants of other cancers (colorectal, lung, appendiceal, ovarian, and so on) for which chemotherapy may be the only treatment option.
Mesothelioma's detection challenge is that it doesn't become apparent until 20-40 years after a person has inhaled asbestos fibers on the job (especially for construction, auto repair, firemen, and military veterans) or at home, or through secondary exposure when the fibers are unwittingly brought home on shoes and clothes. By the time it's discovered, it's too far advanced to cure.
In contrast, pancreatic cancer is over 14 times more prevalent than mesothelioma, with over 46,000 new cases a year and almost 40,000 deaths. Its causes are unknown but are suspected to include smoking, excessive alcohol consumption, and mutations in patients' DNA. Like mesothelioma, pancreatic cancer survival statistics are grim, with a higher proportion of diagnoses coming late and only 15 percent of patients surviving one year after Stage 4 diagnosis.
Neither of these two deadly cancers has a therapy proven to drive advanced disease into remission, which is why new drug development and associated clinical trials are so critical. Such trials can give patients access to cutting edge treatments while buying patients time.
What is a Clinical Trial?
Clinical trials are prerequisites before any new treatment can be approved for widespread use. A clinical trial is a test of a new potential prevention, detection, or treatment product or process. Trials are conducted by the National Cancer Institute (NCI, within the National Institute of Health) or by a potential pharmaceutical manufacturer under the oversight of the U.S. Food and Drug Administration. No new treatment can be approved in the U.S. without passing such formal tests in animals and then in people to prove its efficacy and safety to treat a targeted disease. Without volunteers to try new treatments, no new therapies can move forward, however urgent the need.
Trials consist of four phases:
- Is it safe?
- Does it work against the targeted disease?
- Does it work better than existing therapies?
- Is it safe and effective over the long term?
For the most part, a patient's oncologist is the gatekeeper for clinical trials, finding and recommending them to patients for whom standard treatment regimens are no longer providing curative benefits. The earlier the trial, the less certain its benefits, but for patients whose standard options have run out, there is often little to lose.
To participate in a trial, volunteers must meet the study's qualification parameters and then go through an informed consent process. Informed consent ensures that the patient understands how the new treatment differs from the standard of care, what risks might be involved, what doctor visits will be required, and what tests will be needed to determine whether the treatment is working. Information about who pays what for clinical trials can be found on the NCI website.
Trials are generally safe: if the treatment isn't working or if severe or life-threatening side-effects arise, patients may leave the trial or the trial may be cancelled.
If My Prognosis is Poor, Why Participate in a Clinical Trial?
Deciding whether to participate in a clinical trial is a very personal decision. For some patients, a clinical trial can produce a near miracle, but for others it may represent a dead end.
The decision to participate is not automatic. Participation in one clinical trial may preclude some other treatment options at a later date. Further, it takes an average of 15 years for an experimental drug to progress from lab to patient. Since participation in a clinical trial can last over a year and it may take even longer to analyze the results, any individual patient with a terminal disease may feel that the trial is unlikely to change the course of his disease.
Low levels of participation in clinical trials pose a particular challenge for patients experiencing a relatively rare and incurable cancer:
- A Harris survey in 2001 showed that 85 percent of patients were either unaware of or unsure that participation in a clinical trial was an option at the time of their cancer diagnosis. Seventy-five percent said they would have been willing to enroll if they had known that such trials were available to them. Almost 90 percent said they would be willing to volunteer if their initial treatment failed.
- Only 2-3 percent of oncology patients participate in clinical trials. Dr. Axel Hanauske, board co-chair for the Mesothelioma Applied Research Foundation, believes that a low voluntary patient participation rate has caused a particular scarcity of new mesothelioma drugs.
Despite the patient recruiting challenges, researchers are advancing science and often improving the quality of patients' lives. For many patients who enroll, the trial gives access to treatments that would not otherwise be approved. In addition, even if the disease can't be reversed, the new treatment in trial may extend survival time and eventually help bring a new life-saving treatment to market:
- Don's early stage mesothelioma wasn't halted by surgery, radiation, or standard-of-care chemotherapy, so he enrolled in a Phase 2 mesothelioma trial in late 2012. He died 21 months after diagnosis, the median survival time for patients diagnosed at the early stage. His daughter calls it a "blessing" because the trial was able to keep him relatively comfortable longer than many patients who received standard treatments.
- Several sequential clinical trials and simultaneous palliative care (for symptom relief) allowed Mike to enjoy his wife, kids, and new grandchild and to extend the initial 4 -to 6-week prognosis to 27 months. When he died at home, Mike was free of serious pain, nausea, or anxiety. His wife calls it a "victory with a sad ending."
Increased volunteer participation in trials offers patients access to cutting-edge treatments and contributes to the development of better treatments to prolong life and eventually drive those cancers into remission.
There are at least 87 mesothelioma clinical trials and 117 pancreatic cancer trials planned or underway in 2014. These numbers change daily, but some are currently recruiting patients. Clinical trials for all types of cancers are listed on line both in the National Cancer Institute registry and on the websites of the 41 comprehensive cancer centers that have been certified by the National Institutes of Health. There are also matching services (some dedicated to specific cancers, like Mesothelioma Applied Research Foundation, MesotheliomaHelp, and PanCan) that help patients find appropriate specialists and clinical trials and others (like CenterWatch) that help clinical study teams to find appropriate patient recruits.
If you've been diagnosed with an incurable advanced cancer and are no longer responding to standard treatments, ask your doctor about trials that might help you live better and possibly longer while advancing development of new therapies. You'll be doing yourself and those who follow in your footsteps an immeasurable favor.