Simone, Joseph V. MD
One could argue convincingly that pediatric oncology, my own subspecialty, has been the most successful force of modern oncology in treatment successes, lives saved, and clinical research productivity. At least three quarters of all children treated for cancer in the last decade are long-term survivors, and nearly that many are cured of cancer.
How did this happen? The main reason is biology. Pediatric tumors predominantly arise from embryonic tissue and are inherently more sensitive to current therapy. Adult tumors are overwhelmingly carcinomas that arise from epithelial tissue, and most are relatively insensitive to current therapy.
But there has been another contributing factor.
The system of care for children with cancer differs significantly from the care for adults with cancer. Most children are treated at childhood cancer centers, all of which participate in clinical trials. Consequently, a large fraction, perhaps 50% or more, of all children with cancer are treated according to a peer-reviewed protocol.
Although many of the protocols consist of empirical variations of prior studies rather than dramatically novel therapies, this mix of approaches has proved effective. An example is childhood acute lymphoblastic leukemia.
The cure rate has increased progressively from about 10% to 15% in the mid-1970s to nearly 80% today, despite the lack of any new main line chemotherapy agents in that time. At the same time, childhood ALL has been a hotbed of immunologic, cytogenetic, and molecular innovation.
Perhaps an even greater effect of the widespread use of protocols has been that standards for pathology, surgery, imaging, radiation therapy, and chemotherapy were established in these institutions and soon were applied to patients whether or not they were treated according to a protocol, thus weaving a fine cultural fabric of research and care.
This engendered an atmosphere of high standards, cooperation, and enthusiastic participation. However, there are worrisome signs that the fabric of that culture is fraying.
First, we are victims of our own success. As overall cure rates approach or exceed 75% in some cancers, launching radical innovations is more difficult for fear of endangering sure cures.
But substantive innovation not only drives research, it also powers enthusiasm and optimism and attracts top trainees interested in research. Caution is warranted; reticence is not.
Second is the troubled merger of the Pediatric Oncology Group and the Children's Cancer Group to form the Children's Oncology Group.
To date the COG has made little if any progress in attaining the primary objectives of the merger— i.e., greater efficiency and productivity, lower cost, and more patients on trials.
Furthermore, the spirit of optimism and cooperation certainly has suffered. Some former POG members have said that CCG effectively engineered a coup d'etat to control COG. Whether true or not, at best it reflects a feeling of dissatisfaction that certainly doesn't enhance the collaborative spirit.
Some former CCG and POG members have complained that the COG structure allows fewer opportunities for leading studies and suffers from a painfully slow process for getting protocols designed and approved. These problems may fade with time, but meanwhile another movement is gaining steam.
“It would be a tragedy if pediatric oncology were to decline as a vibrant, innovative scientific craft because of insufficient attention to strategic and organizational issues.”
Groups of five or more pediatric institutions have been forming research consortia for the study of neuroblastoma, brain tumors, Hodgkin's disease, acute myeloid leukemia, and others. With the smaller membership, meetings can be more intimate, agreement quicker, meaningful participation virtually assured, and the opportunity for innovation greater. The larger the committee, the less likely it will innovate.
Thus, pediatric oncology, “Quo Vadis?” (Where are you going?)
Is this a time that a new strategic model, such as a federation of smaller groups of institutions, is needed for this changed environment?
For example, two or three neuroblastoma groups might be better, because they would generate independent research vectors and also would have the virtue of a built-in peer review system for collaborative competition. The COG as a whole would continue to focus on large-scale Phase 3 studies. An open discussion would certainly generate other models to consider.
“Groups of five or more pediatric institutions have been forming research consortia for the study of neuroblastoma, brain tumors, Hodgkin's disease, and AML. With the smaller membership, meetings can be more intimate, agreement quicker, meaningful participation virtually assured, and the opportunity for innovation greater.”
Pediatric oncology has been the innovator for many facets of oncology. Its institutional and cooperative group leaders have an opportunity to reconsider and debate its direction and structure to assure its leadership role in the future.
It would be a tragedy if pediatric oncology were to decline as a vibrant, innovative scientific craft because of insufficient attention to strategic and organizational issues.
We are delighted to introduce this new column by pediatric cancer pioneer Joseph V. Simone, MD. Dr. Simone, a long-time member of OT's Editorial Board, is Clinical Director Emeritus of Huntsman Cancer Institute, Professor Emeritus of Pediatrics and Medicine at the University of Utah, and now President of his own consulting company (www.SimoneConsulting.com).
From 1992 to 1996 he was Physician-in-Chief of Memorial Sloan-Kettering Cancer Center, and before that, was a leading researcher at and eventually Director of St. Jude Children's Research Hospital. In his years there, he was engaged in clinical research efforts to improve therapy for children with cancer and played a leadership role in the development of curative treatments for childhood leukemia and lymphoma.
Dr. Simone has also served as Medical Director and Chairman of the National Comprehensive Cancer Network and as a member of the Board of Scientific Advisors of the NCI. Currently he is Chairman of the National Cancer Policy Board of the Institute of Medicine.
He has received numerous awards and honors, including the Richard and Hinda Rosenthal Foundation Award of the American Association for Cancer Research and the Distinguished Service Award for Scientific Excellence from the American Society of Clinical Oncology.
Dr. Simone welcomes suggestions for topics for this column, which can be e-mailed to him at OT@LWW.com
© 2003 Lippincott Williams & Wilkins, Inc.