CTOS Op Ed

CTOS Op Ed

The creation of a robust registry of sarcoma patients and biological resources

Paul A. Meyers, MD

Professor of Pediatrics

Weill Cornell Medical College

Vice Chair, Department of Pediatrics

Memorial Sloan-Kettering Cancer Center

[Editor's Note: This editorial is part of an ongoing series of Op Ed pieces written by one of the members of the Board of Directors of the Connective Tissue Oncology Society (CTOS). Dr. Meyers is the current president of CTOS. These editorials are intended to address important and controversial issues in the field. The “Questions, Comments & Counterpoint” column allows readers to express their opinions in response to these Op Ed pieces. Click here to send in an opinion.]

The Connective Tissue Oncology Society (CTOS) was founded by a small group of physicians and scientists with interest in tumors of the connective tissues. The great majority of these tumors are sarcomas. From its beginnings, CTOS was an inclusive organization, including physicians from every specialty involved in the care of patients with sarcomas and clinical and basic scientists carrying out research to improve our understanding and treatment of sarcomas. We have surgeons, orthopedic surgeons, medical oncologists, pediatric oncologists, radiation oncologists, diagnostic radiologists, pathologists, and basic scientists who focus on tumors of the connective tissues. Our annual meeting has grown to become the premier international forum to share new knowledge about sarcoma biology and treatment. CTOS members identified a need to create an organization to perform clinical trials of new agents in patients with sarcomas. We created the Sarcoma Alliance for Research through Collaboration (SARC). This group was developed to allow us to perform clinical trials of new agents and novel concepts at many centers around the world. SARC has a well deserved reputation for developing trials and making novel therapies available to patients with minimal delay. SARC has overcome difficult obstacles that impeded international collaboration in the past. SARC successfully completed important trials of imatinib for the treatment of gastrointestinal stromal tumor (GIST) and equally importantly, showed that imatinib was not helpful in many other sarcomas. SARC is currently conducting a critical trial of an inhibitor of the insulin-like growth factor 1 receptor (IGF1R) in a variety of sarcomas. Accrual to this trial is proceeding faster than initially projected because of the hard work and enthusiasm of the international community of investigators dedicated to sarcoma research.

Progress in understanding the biology of sarcomas and developing novel therapies has been slow. One of the most difficult obstacles to progress is the protean nature of sarcoma. Sarcoma is not one disease but dozens of diseases, each with its own clinical presentation and unique biological features. Even in aggregate, sarcomas are rare tumors. When we try to understand individual sarcomas, they become even rarer. This makes it very difficult to be sure that we have an accurate understanding of the usual behavior of some sarcomas, their response to therapy, and their biologic alterations which will give us the clues we need to develop therapies. For precisely this reason, we need international collaboration to aggregate adequate numbers of individuals with sarcoma. This allows physicians to provide accurate information to allow patients to make informed decisions about their care.

All of us in the sarcoma community would benefit from the creation of a robust registry of sarcoma patients and biological resources. A registry needs to incorporate rigorous measures to protect vigorously the privacy of any participants. We refer to this process as anonymization, and this is routinely accomplished in many registries. The registry needs to include demographic information such as age, gender and ethnicity. It needs to include precise diagnostic information. It needs to include staging information about the size, location, and extent of primary tumors, and the presence or absence of spread to regional lymph nodes or distant sites. Most importantly, it needs to include information about the evolution of disease including response to treatment. This aggregated information will allow the clinicians caring for patients with sarcoma access to reliable contemporary information which they can share with patients struggling with therapy decisions. It will identify cohorts of patients appropriate for novel therapies. The registry can also track the availability of key resources for scientific investigation such as tumor tissue or blood samples from patients with tumors. This is sometimes referred to as a virtual tumor bank. Researchers with questions about the biology of a specific sarcoma will be able to find appropriate resources and solicit collaboration which will allow them to answer the questions much more quickly. For basic science researchers, it is critical to be able to link the results of their scientific studies to the clinical course of individual patients from whom the biological specimens are acquired. This requires linkage to the demographic information and outcome of the patients in the registry, protected by the process of anonymization.

The idea of a patient and tumor registry is not a new one. This mechanism has a proven track record of success in a variety of more common cancers. What makes sarcoma a more difficult problem is the rarity of the individual tumors. No single institution can ever hope to accumulate enough patients or biological specimens to achieve the goals. We need to create a registry with the same international scope represented in CTOS. The history of progress in science reveals that good ideas often arise in several minds at the same time. There is already an existing registry for bone tumors in Europe. We recently held an international symposium on Ewing sarcoma at Dartmouth College in New Hampshire. The final session of the symposium was devoted to brainstorming ideas that could help us advance more rapidly. Our number one recommendation was to create an international registry of patient related clinical information and research oriented biologic specimens. The Sarcoma Foundation of America has initiated a registry focused on dermatofibrosarcoma protuberans, clear cell sarcoma, alveolar soft part sarcoma, myxoid liposarcoma or osteosarcoma. We should not tax our limited resources by duplicating the effort required to create a registry.

We need to work together. A successful registry requires resources, personnel, and commitment. The resources include hardware and software to store the information. We need personnel to enter the data and perform quality assurance audits to insure data accuracy. We need to support the work of data managers at participating institutions who acquire and submit the data. We need to insure that the information is continuously updated as changes occur. We need biostatistical support to analyze the information in the registry. Such analysis could include outcome information for a particular type of sarcoma to help patients and treating physicians. It would also include outcome data for laboratory investigators to help them determine if their biologic questions are relevant to the behavior of tumors.

This is an effort which is widely supported by patients, physicians, and scientific investigators. We need to work together with all the groups interested in sarcoma to create this registry to help us all in our joint goal, the control and cure of sarcomas.