Brian A. Van Tine, MD, PhD
Assistant Professor of Medicine
Division of Medical Oncology, Department of Medicine
Washington University School of Medicine
St. Louis, MO
One of the privileges of working in an academic medical center is the luxury of being able to subspecialize in the treatment of sarcoma. Nationwide, there may be 40 to 50 medical oncologists who focus on the treatment of adult soft tissue and bone sarcoma. With most of the specialists concentrated in just a few centers on the East and West coasts, access to teams with multidisciplinary experience in the treatment of sarcoma is sometimes difficult.
The National Comprehensive Cancer Network (NCCN) guidelines state that, “All patients (with sarcoma) should be evaluated and managed by a multidisciplinary team with expertise and experience in sarcoma, prior to the initiation of therapy.”1
The reasons for this statement are many. First, the average medical oncologist in private practice sees only 1-2 cases of sarcoma a year. Since mesenchymal tumors (ie, sarcoma) include more than 50 histological subtypes with varying natural histories, genetics, prognostic factors, and sensitivities to treatment, the specific treatment planning requires an in-depth knowledge of sarcoma biology. Though sarcoma is often referred to as a chemotherapy-resistant disease, it may be more correct to say it is chemotherapy-selective cancer. Thus, this necessitates the participation of a sarcoma expert with extensive experience in selecting agents for the specific subtype of sarcoma to best benefit a patient. For example, while ifosfamide is highly active in synovial sarcoma (now called X:18 sarcoma), it is much less responsive in the treatment of leiomyosarcoma. Second, the surgical and radiation planning and chemotherapy sequencing for many of the tumors is complex and requires multidisciplinary planning. Third, and most importantly, the pathology of a sarcoma is best reviewed by a pathologist with extensive experience in the diagnosis of sarcoma.2
Histology drives not only treatment decisions but also clinical trial opportunities; therefore, having the most accurate diagnosis is imperative.
It must be clearly pointed out that NCCN guidelines say patients should be evaluated and managed by a sarcoma specialist—not treated. As most sarcoma specialists would agree, standard-of-care regimens are easily given by referring physicians and most likely should be. Otherwise, we would each be treating 400-500 patients, and that is not the role for an academic physician. The major role of the academic sarcoma medical oncologist is the design and implementation of clinical trials that allow for rapid enrollment to move the field forward. Due to the rarity of the disease, most clinical trials for sarcoma patients are limited to the larger academic centers for financial reasons. As such, there is an even greater need to partner with the community oncologist to give the standard-of-care agents.
Important Clinical Trials Are in Progress
One of the greatest successes of the sarcoma field has been in the treatment of gastrointestinal stromal tumors (GISTs).3
The successful treatment of this tumor, once named gastrointestinal leiomyosarcoma, with imatinib necessitated a name change, since imatinib is not effective in other leiomyosarcomas. As most community oncologists are aware, first-line treatment of GIST is imatinib, the second line is sunitinib, and the third line is regorafenib, with other tyrosine kinase inhibitors being used in specific circumstances.
Since the GIST population is now commonly treated in the community with standard-of-care agents, our ability to enroll this patient population in a clinical trial has become more difficult. For example, there is a phase III second- line trial of sunitinib versus masitinib (NCT01694277) that is very slow to enroll because most patients have been treated with sunitinib prior to either visiting or returning to an academic center (Table
). Also, there is an international trial of first-line imatinib versus masitinib (NCT00812240) that is now opening. The only way this trial can accrue rapidly is through a partnership with community oncologists making sure that GIST patients are aware of it.