PBTC

This article was written for The Childhood Brain Tumor Foundation,Germantown, MD 20876

Pediatric Brain Tumor Consortium

Roger J. Packer, MD

In April of 1999, the Children's National Medical Center was named as one of eight institutions in the United States selected for membership in the newly formed Pediatric Brain Tumor Consortium (PBTC). This consortium, supported by the National Institutes of Health and the National Cancer Institute, was developed to perform innovative, technologically challenging studies for children with brain tumors, studies designed to improve survival and quality of life. The eight institutions were chosen after a competitive review of their past experience in the care of children with brain tumors, the depth of their pediatric brain tumor program, the clinical resources of the institution, and their ability to perform innovative research. In 2003, a 9^th institution was added. In April of 2004, PBTC was re-approved for another five years of operation. The chosen programs are listed in Table One.

Dr. Roger J. Packer, Executive Director of Neuroscience and Behavioral Medicine and Chairman of Neurology at the Children's National Medical Center (CNMC), is the principal investigator for CNMC. He is joined by Drs. Tobey MacDonald, Brian Rood, Gilbert Vezina, Robert Keating, Derek Bruce and Rita-Maria Santi, and nurse Debbie LaFond, in directing the CNMC effort. As part of the CNMC selection for the PBTC, working relationships were documented with the Imaging Branch of the National Institutes of Health (NIH). The Children's National Medical Center's Brain Tumor Program has worked closely with other oncology programs in the region to develop the best possible comprehensive care for children with brain tumors. Part of the reason that CNMC's Brain Tumor Program was awarded this grant by the National Cancer Institute is the strong regional working group headed by the CNMC, which includes Georgetown University (Dr. Gootenberg), Fairfax Hospital (Drs. Greenberg and Horn), Kings Daughter of Norfolk, Virginia (Dr. Bevan), Geisinger Medical Center (Dr. Shah), University of Virginia at Charlottesville, Virginia (Dr. Jane), DuPont Institute and Delaware Children=s Hospital (Dr. Walter), and Hershey Medical Center (Dr. Neely). The members of the regional neuro-oncology program have agreed to participate with the Children=s National Medical Center in the selection of appropriate patients for PBTC studies. All patients who are candidates for these new, innovative studies will be treated at the Children=s National Medical Center, however much of the supportive care can be delivered at the referring institution.

The mission of the Consortium is to perform innovative types of clinical trials, for children with brain tumors, which cannot be completed at any one single institution and which are so new and challenging that only a select group of institutions can perform the trials. The PBTC will work closely with already established national groups, such as the Children's Cancer Group, the Pediatric Oncology Group, and the Pediatric Oncology Branch of the National Cancer Institute, to perform studies. It is planned that the studies to be completed by the PBTC will include those investigating new methods of delivery of anti-cancer agents to the region of the brain tumor and the area of brain where the tumor may have infiltrated; innovative biologic agents, such as antiangiogenesis agents; pharmacological studies of new drugs; innovative means to evaluate the efficacy of treatment; and innovative approaches to brain tumor management. Monies have been set aside to do important correlative biologic studies.

Over the first five years of operation, the Pediatric Brain Tumor Consortium began multiple studies. The studies included: 1) innovative approaches for infants with malignant brain tumors, including the use of intrathecal chemotherapy (chemotherapy being given directly into the cerebrospinal fluid; 2) studies investigating the use of toxin treatment labeled to a brain tumor specific receptor for children with recurrent malignant cortical tumors (the initial study utilizing convection-delivered therapy is chaired by Dr. Packer and centered at CNMC); 3) evaluations of anti-angiogenic agents, including one study chaired by Dr. MacDonald; 4) cell-cycle disrupting agents, including an oral farnesyl transferase inhibitor; 5) trials of anti-growth factor agents; 6) approaches designed to overcome drug resistance; and 7) new approaches for children with newly-diagnosed brain stem gliomas.

The PBTC, which was strongly supported by Dr. Malcom Smith and his co-workers at the National Cancer Institute, is an important avenue to improve the care of children with brain tumors and hastens the integration of innovative approaches in the management of children with brain tumors. The program is designed to supplement, but not in any way replace, the efforts already ongoing on a national and regional basis for children with brain tumors. The Children's National Medical Center program will meet on a semi-yearly basis with the members of the regional working group to update members of the working group on the progress of the Consortium. Families interested in the activities of the Pediatric Brain Tumor Consortium can contact Debbie LaFond (202-884-3569) at the Children's National Medical Center for more information.

Table One

Members of the Pediatric Brain Tumor Consortium

Baylor Children's Hospital, Houston, TX (S. Blaney, MD, P.I.)

Boston Children's Hospital/Dana Farber Cancer Center, Boston, MA (M. Kieran, MD, P.I.)

Children's Hospital of Philadelphia, Philadelphia, PA (P. Phillips, MD, P.I.)

Children's National Medical Center, Washington, DC (R. Packer, MD, P.I.)

Duke University, Durham, NC (H. Freeman, MD, P.I.)

Pittsburgh Children' Hospital, Pittsburgh, PA ( I. Pollack, MD, P.I.)

St. Jude Children's Hospital, Memphis, TN (L. Kun, MD, P.I.)o

Seattle Children's Hospital, Seattle, WA (R. Geyer, MD, P.I.)

University of California at San Francisco, San Francisco, CA (M. Prados, MD, P.I.)