This article describes a myeloma patient’s experience participating in a clinical trial. It is part of a series of articles intended to help clarify the clinical trial process.

Don was diagnosed with multiple myeloma in 2004. At the age of 69 and despite living with a cancer that attacks his bones, Don inspires fellow myeloma patients by running in marathons. He has completed three marathons so far this year, is running in one tomorrow, and plans on participating in three to four more marathons just this year. Additionally, Don maintains a blog in which he shares his myeloma experiences with fellow patients, including details about his participation in a clinical trial.

With progressive stage 1 myeloma, Don decided to participate in a Phase 2 clinical trial at the Mayo Clinic Cancer Center that studied the effects of pomalidomide plus dexamethasone (Decadron) for patients with relapsed or refractory multiple myeloma. His doctor suggested this trial to him because it seemed to be less toxic and possibly more effective than other therapies he had tried, such as Revlimid (lenalidomide), thalidomide (Thalomid), and Velcade (bortezomib).

Despite the main advantage of trying a new therapy (that hopefully works), Don had some concerns about trying a relatively new treatment. In discussing his primary fear about the trial, Don said, “I have an ongoing concern that we may eventually discover a late-developing side effect from pomalidomide.”

Finding a clinical trial was an easy process for Don. The trial was recommended to him by his doctor at the Mayo Clinic who was also the principal investigator for the study. As Don’s myeloma specialist, she was able to recognize and recommend a trial best suited to Don’s needs.

In addition, Don did research about potential treatment options on his own that enabled him to make a more educated choice about his treatments.

“By then, I knew enough about the various treatments to believe in her choice,” said Don. “She gave me a choice between Revlimid and pomalidomide, and I chose pomalidomide.”

Enrolling in the trial was also an easy process. “I had no trouble getting into the trial,” he said. “At that time, eligibility required: at least one prior therapy, ‘measurable’ disease (any of several criteria), and otherwise being healthy enough to participate.”

The transition into the trial was also made smoother by the ease of determining eligibility through tests for myeloma. Myeloma patients are already frequently tested for several blood markers and other symptoms and given X-rays, bone marrow biopsies, and PET scans. Eligibility can be determined by the results of those tests.

Don’s tests confirmed his eligibility and also showed stage 1 bone lesions, which for many patients like Don, are reason enough to seek out new therapies in hope of relief or prevention of further bone damage.

Throughout the trial, Don has been fully aware of exactly what medications he is taking. “It would be hard to conceal it, actually, because I take the little pills every night, and I have the side effects that everyone attributes to pomalidomide and dexamethasone.”

The treatment regimen allows patients to take the pills by themselves, once a day, so Don never needed in-clinic treatment.

Don goes to the Mayo Clinic once a month for testing. He described the routine as follows: “I drive 90 minutes to the Mayo Clinic every 28 days. They do a blood draw at 6:30 a.m., followed by an electrocardiogram on every third visit. Sometimes there are more tests. Later that same morning I will see the doctor or her nurse practitioner, and most of the test results will be ready for that meeting. By about noon, usually, I will pick up the next 28-day supply of pomalidomide and head home or wherever we're going.”

Don sees his doctor once a month during the trial. “If I were not in a trial, I suppose I might not see the doctor quite as often,” Don explained.

When asked about his experiences with the clinical trial, Don replied, “I am very glad that I am in this trial. The drug (pomalidomide) is free, and my insurance company doesn't have to pay the very high price commanded by Revlimid, nor do I have to pay the co-pays. More important, I believe that pomalidomide is a better drug and has, for me, fewer side effects.”

Don pointed out that no myeloma treatment is without serious side effects. However, in Don’s case, the pros outweighed the potential cons. “Pomalidomide with dexamethasone brought my M-Spike down from 2.7 to 1.0 g/dL within a few months, holding it mostly stable since then. I have been on the drugs for two years now. I discontinued dexamethasone in December because of current side effects and other potential effects of long-term treatment.”

Don described the side effects he experienced from pomalidomide as “modest” and “fewer than most.” They included:

• Slight neuropathy in feet (tingling and numbness)
• Slightly reduced heart rate
• Declining white blood cell count, especially neutrophils (this is the primary reason why a blood check is done every 28 days).

The pomalidomide trial has changed Don’s life by providing a new treatment for him that allows him to experience a pretty high quality of life. “I am a runner. Running stops when a runner breaks a bone. Because of my Mayo Clinic doctors and pomalidomide [and dexamethasone], I have continued running, with no broken bones,” he added.

Don had the following advice for fellow myeloma patients looking into clinical trials, “If I hadn't participated in the trial, I would have gone with Revlimid. But Revlimid is only available because other people participated in trials for it. My most immediate concern is my own health, of course, but I'm glad to be able to ‘pay it forward’ in this way. It's a good feeling.”

For more information about Don and his clinical trial participation, please visit his blog, “Myeloma Hope: Don’s Myeloma Diary.” To learn more about clinical trials, please read the previous articles in the series and check The Myeloma Beacon in the coming weeks for the final article in this series.