Children's Cause for Cancer Advocacy

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The Virtual Pediatric Oncology Drug (vPOD) Enterprise

What's the Need?

Cancer remains the leading cause of death by disease for U.S. children under 15. Despite the decrease in death from cancer from 1975 to 1998, the decline in mortality has stalled in recent years. Some cancers, such as brain tumors, remain incurable. Further, the aggressive treatments responsible for improved survival rates come with a dark side. Drugs used to treat children have serious long term and late effects on health and development. Scientific advances now document that the molecular and biological characteristics of most childhood cancers differ substantially from those of adults. Using select molecular abnormalities as starting points in the search for more precisely targeted drugs, new therapies can be discovered that will be more effective and less toxic than current regimens.

What's the Problem?

Drug discovery and drug development for children with cancer is fraught with challenges. Perhaps most important is the small market size for any particular pediatric cancer, which are numerous and varied. Because the number of children affected can be in the hundreds, this represents poor opportunities for pharmaceutical industry investment. Industry also does not screen their chemical libraries against molecular targets specific to childhood tumors, limiting knowledge about the number of compounds that might be of benefit in treating pediatric malignancies. Academic efforts to develop agents are limited by access to industry libraries and by expertise in key aspects of drug development (e.g. medicinal chemistry). Finally, treatment successes thus far have created the misperception that childhood cancer has been "cured," blunting any sense of public urgency for greater efforts.

What's the Solution?

The 2005 Institute of Medicine (IOM) report, Making Better Drugs for Children with Cancer, recommended the creation of a new coordinating entity for the discovery and development of novel pediatric cancer therapies. The virtual Pediatric Oncology Drug Initiative (vPOD) will operate with a non-profit business model to manage collaboration among research institutions, industry, government and patient advocates to develop new agents to treat pediatric cancers.

What will vPOD Do?

vPOD will be an independent, sustainable entity to drive a rational, efficient and more rapid process for developing novel pediatric cancer therapeutics. Pediatric oncology research already has established networks for clinical testing through the National Cancer Institute and academia, capable of studying new therapies in children. vPOD will work on those steps in the discovery and development pipeline prior to bringing a new agent into children.

vPOD will:

Coordinate diverse national resources and activities from academic medical centers, pharamaceutical/biotechnology firms, government agencies, advocacy groups and others in support of specific drug discovery and development projects
Count the facilitated entry of an agent into Phase 1 pediatric trials a success
Have a scientific advisory committee to advise the entity on project priorities and to help identify resources and contribute to the overall mission
Have a board of directors to oversee vPOD's structure, management and fundraising
Engage administrative and management staff to implement vPOD projects
Be governed by transparency and inclusiveness in its operation and activities.

Where is vPOD Now?
A Steering Committee, starting with representatives from the six leading academic medical centers, industry and patient advocates was created at an Institute of Medicine workshop in 2006. A team is currently creating a business plan and start-up cost estimates. Broader financial support is now being sought to recruit an executive team and create a sustainable revenue stream.

What Can You Do?

Serve on an advisory committee to help identify scientific resources available for a particular project
Suggest a new validated target for a childhood tumor
Identify people in industry and academic who might be interested in working on a project
Suggest a new strategy or technology to accelerate pediatric drug discovery and development
Identify financial or in-kind support for a project.

For more information, contact Susan L. Weiner, President and Founder of the Children's Cause for Cancer Advocacy at slweiner@childrenscause.org.