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Deciding to Enroll in a Phase I Clinical Trial for Our CLL (chronic lymphocytic leukemia)

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Deciding to Enroll in a Phase I Clinical Trial for Our CLL (chronic lymphocytic leukemia)

I recently read an insightful commentary in the New York Times by a skin cancer patient on clinical trials. Mr. Collender describes both the reasons for the unfortunate lack of participation in clinical trials (approximately 3 percent of all cancer patients in the US join a study), and his reasons for choosing to enroll in one.  He suspects that both doctors and their patients do not know about the availability of studies, and that patients fear them.  He identifies with these concerns and thoughtfully describes how he overcame them.

Mr. Collender’s commentary is very relevant to CLL patients, and certainly one that I related to. As a patient of Dr. Thomas Kipps, at UCSD Moores Cancer Center, I have participated in two clinical trials, the most recent of which was the Phase I trial of cirmtuzumab, a new and highly promising monoclonal antibody. And although I’ve had previous experience with a Phase II clinical trial, and more importantly, I know that Dr. Kipps and his team would not recommend a treatment or study for which they did not feel I was well suited,  I must admit that enrolling in a Phase I study gave me reason to pause.

The National Institute of Health has a good primer on clinical trials, including a description of the different phases here . Basically, as a drug is developed it moves through the phases, starting with a limited number of patients in Phase 1, thereafter enrolling more and more patients in later stages, through Phases 2-4, as the efficacy and safety of the drug is established. Phase 1 can mean ‘first in human’. By Phase 3, much is known about how patients respond and tolerate and the goal is to confirm the response in a large population of patients.

My primary concern was based on an outdated perception that Phase I studies are for patients who don’t have other options – last ditch efforts that offer little hope of a response, for patients who don’t have much left to lose. I was reassured then to find a recent article in the Journal of Clinical Oncology: American Society of Clinical Oncology Policy Statement Update: The Critical Role of Phase I Trials in Cancer Research and Treatment. The article defines Phase I trials and makes recommendations for increasing enrollment.  But the section I found most relevant, and reassuring, was the detailed description of the evidence for clinical benefit even at the initial study stage.

As suggested by Mr. Collender, the ASCO article offers strong support that clinical trials, including Phase I trials, offer patients control over their disease and treatment, and a sense of making a contribution to the development of new treatments that may help future patients. Phase I patients are also watched carefully and receive excellent palliative care, contributing to a high quality of life.

But here’s what I found most helpful: Phase I patients have the “prospect of a direct medical benefit”. Response rates are often as high as the response rates to standard of care, FDA approved drugs, particularly as cancer treatments become more and more targeted to the individual patient and doctors can better predict who is likely to respond. Furthermore, experimental treatment-related deaths have decreased to one-tenth of what they were 25 years ago, in part also because of the targeted agents but also because of new trial designs that minimize risk.

Eight weeks after completing the cirmtuzumab trial, I can agree with the conclusions in the ASCO article and Mr. Collender’s experience. I received a dose that was meant to determine potential response and possible toxicity, without exposing me to significant risk. I was watched very carefully by a team of the world’s experts on CLL, who clearly had my best interests at heart. I felt fortunate to collaborate with this team. At no time did I feel like a lab rat.  I did not feel anxious or concerned for my well-being, or that the treatment did not offer hope of a clinical benefit. In fact, cirmtuzumab offers the potential for a response in not only CLL patients, but also ovarian, pancreatic and lung cancer patients, and I feel extremely fortunate to contribute to its development. It has been an opportunity that I am grateful to have been offered. And I will be happy to participate in another clinical trial, should one be recommended.

Cynthia Havstad

July 7, 2015