Improving Hematological Cancer Care Through Clinical Trials

The therapeutic landscape in oncology has changed dramatically in the last few years with the development of targeted therapies that have improved outcomes for many subtypes of cancer. In my field, the lymphoid malignancies, the recent approval of ibrutinib represents a major step forward for patients with chronic lymphocytic leukemia and several forms of non-Hodgkin lymphoma.

The recent approval of ibrutinib represents the culmination of years of effort of many committed people, and is an example of the need for continued emphasis on clinical investigation. Breakthroughs like this are only capable through well-designed clinical trials. Sadly, however, many patients who are potential candidates for such trials never enroll on study. 

During my training, I was fortunate enough to be at a cancer center that enrolled some of the first patients on study with ibrutinib. As a fellow, I would eagerly present this option to patients with relapsed disease, many of whom did not have other good options for therapy. While some patients were quick to sign up, thankful for the opportunity to participate in an experimental therapy that might improve their outcomes, if not the lives of future patients, many patients were quite reticent, suspicious of the clinical trial and our motivations. 

Many patients had been warned prior to coming to us that academic cancer centers “experimented” on patients and treated them like “guinea pigs”. Despite being presented with the details regarding the safeguards in place to protect all patients, many simply couldn’t get past the idea of receiving an unproven treatment as part of a clinical study.

As a new clinical investigator in the B-cell malignancies, I see several reasons that patients do not receive therapy through a clinical trial. Some are easily understood: the distance to travel is too great, the patient is not eligible due to comorbidities, or there is an effective, well-tolerated alternative. However, in all too many cases, patients are either never made aware of the trial or they are counseled against serving as a “guinea pig” for experimental therapies, often by well-meaning but misinformed friends or family members. 

While clinical trials could still successfully accrue in the past despite these challenges, enrollment has become much more challenging in the era of targeted therapies. Identifying a patient with a specific mutation or biomarker means that the pool of potential candidates is decreased. As a result, it is even more important that those patients be offered the opportunity to participate if eligible.

It is very disappointing to me when patients decline enrollment on a clinical study as a result of fear of being an experimental subject. While I recognize that history is unfortunately littered with episodes of abuse by physicians conducting clinical studies, it is important for patients, families, and referring physicians to be made aware of all of the steps that are taken to ensure patient safety, even in the most early phase studies. 

Prior to the enrollment of the first patient, a clinical trial has generally been reviewed by a scientific review committee, an institutional review board, a pharmaceutical company review board, and often the FDA. Clinical trials are written as a specific protocol which requires close attention and numerous staff, which are employed for each trial to ensure adherence to the protocol and patient safety. As a result, I find that enrollment on a clinical trial is, in many cases, safer than receipt of standard therapy. Patients on trial typically receive close follow-up, more frequent labs and scans, and a heightened response to reported symptoms.   

Clinical studies are performed with the goal of addressing an unmet need, where we know that the standard approach is not effective or may be toxic. Unfortunately, none of our therapies are 100% effective without toxicity, so the brightest minds in our field strive to improve outcomes for more patients and make therapies more tolerable. 

Enrollment on a clinical study offers a patient access to state of the art care with close safety monitoring and allows patients to contribute to our understanding of the management of their malignancy. Many of our newly available and upcoming targeted therapies are only available due to the participation of prior patients on these studies. The next breakthrough will be accelerated through collaboration between clinical investigators and treating physicians, and all oncologists should consider referrals for clinical trials as a standard component of their routine practice. 

What tips can you provide that may help boost cancer clinical trial enrollment?