Clinical Trials

Fictional Case Study

Nichelle, age 53 (Nichelle is not a real patient.)

Diagnosis: Multiple Myeloma

After months of fatigue and back pain that would not go away, Nichelle scheduled a visit with her primary care physician, who ordered an MRI and blood tests. The MRI showed a cracked vertebra and lesions on her ribs. Nichelle also had higher than normal levels of calcium as well as a low red blood cell count. Her doctor referred her to a hematologist who specializes in treating blood problems.

The hematologist, who is also an oncologist, reviewed Nichelle’s MRI and blood test results. He suspected Nichelle had multiple myeloma, so he ordered a bone marrow biopsy and aspiration to confirm it. Bone marrow is the soft, sponge-like tissue in the center of most large bones that produces white blood cells, red blood cells and platelets. Results showed a high proportion of plasma cells in her bone marrow and a high level of the M-protein. The results confirmed a diagnosis of multiple myeloma. Nichelle was surprised because she had never heard of this type of cancer before and had no family history of cancer at all.

She asked her hematologist for more information about multiple myeloma and the M-protein. He explained it is a type of hematologic (blood) cancer that begins when the blood’s plasma cells multiply uncontrollably. When abnormal plasma cells grow out of control, they can weaken the immune system by preventing healthy plasma cells from producing antibodies, which can affect the body’s ability to fight infection. Myeloma cells produce too much of the same abnormal antibody called the M-protein, which is not needed by the body and does not fight infection. The M-proteins accumulate in the blood and urine and can lead to damage of the kidneys or other organs. Because myeloma cells were found in more than one area, Nichelle’s diagnosis is multiple myeloma.

What was Nichelle’s treatment plan?

A friend of Nichelle’s had a clinical trial for another type of cancer, and she asked if that was a possible treatment option for her. Her hematologist agreed that a trial could be a possibility, but after discussing her treatment goals, he recommended they try a particular therapy first. His treatment plan included drug therapy followed by an autologous stem cell transplant, which would use Nichelle’s own stem cells. He started with a three-drug regimen to knock down the myeloma cells. The drug regimen consisted of a targeted therapy, an immunomodulator and a steroid.

Over time, her treatment was able to reduce the abnormal plasma cells. When her hematologist felt it was time for the autologous stem cell transplant, he prescribed medication to increase blood cell production. Then her stem cells were harvested and she began chemotherapy in preparation for the transplant.

Several months after the transplant, tests showed Nichelle’s myeloma cells had decreased enough that her hematologist considered that a complete response. To keep the multiple myeloma from returning, he suggested a maintenance therapy of the immunomodulator she previously took. Nichelle agreed and started this therapy.

What happened when Nichelle’s therapy stopped responding?

Nichelle’s blood counts remained at good levels for three years. When she noticed a lot of pain in her shoulder, she called the hematologist, who ordered an MRI. The scan showed multiple spots on her clavicle and more on her vertebrae. She received radiation therapy and started bisphosphonate therapy to protect her bones. Her hematologist also recommended a different targeted therapy along with a steroid. This treatment regimen kept the multiple myeloma at bay for 18 months, but follow-up testing showed the cancer was no longer responding to treatment.

At this point, Nichelle and her hematologist revisited the idea of a clinical trial. He recommended one that was testing a new type of immunotherapy called a bi-specific T-cell engager (BiTE), which contains monoclonal antibodies that have two separate targets — the T-cell and the cancer cell. This treatment directs the patient’s immune system to link its T-cell to a myeloma cell and kill it.

He connected Nichelle with Diane, the clinical trial coordinator at the cancer center, so she could learn more about this potential option. Nichelle met with Diane, who explained the trial and its eligibility requirements, which included that her multiple myeloma must be relapsed or refractory and that she had received other lines of treatment.

Diane gave Nichelle the Informed Consent form, which included details about the trial, such as the therapy being tested, potential side effects, risks and benefits. She let her know that as with any cancer treatment, a clinical trial presents potential risks and side effects. It may also require more medical appointments and/or tests than she would ordinarily have scheduled. Diane explained the goal of the trial, how Nichelle would be monitored, the safeguards in place, and how to withdraw from the trial if she chose to.

Nichelle took the Informed Consent form home to read it thoroughly and discuss it with her family. Together, they researched clinical trials using a list of trusted resources from Diane.

To find out the costs of the trial that would be covered and which she would be expected to pay out of pocket, Nichelle called her health insurance provider. Her provider explained that her plan would cover routine clinical trial costs such as office visits and tests. Diane confirmed that the trial’s sponsor would cover the research-related expenses, including the experimental therapy as well as any procedures or tests that would need to be completed for the study.

Nichelle’s decision: Move ahead with the clinical trial

Nichelle felt confident this would be the right treatment approach for her. After she signed the Informed Consent form, Diane provided the schedule of appointments, introduced her to the clinical trial team, gave her contact information for reporting any side effects and reminded her that she can leave the trial at any time for any reason.

Nichelle is excited to participate and contribute to cancer research for future patients. And she is hopeful. If this therapy stops being effective, Nichelle is confident she and her doctor will find other options to consider.

 

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