National Center for Advancing Translational Sciences at NIH

February 3, 2011

Francis S. Collins, M.D., Ph.D.
Director
National Institutes of Health
9000 Rockville Pike
Bethesda, Maryland 20892

Dear Dr. Collins,

The Children's Cause for Cancer Advocacy (CCCA) has followed with interest the debate and decision making around the establishment of a new National Center for Advancing Translational Sciences at NIH. We are pleased to lend our support to this bold endeavor.

CCCA is a national non-profit organization founded in 1999 to communicate with public and professional communities on legislative and regulatory issues of concern to childhood cancer patients, survivors, and their families. Today, CCCA is the leading national advocacy organization that works to facilitate development of less toxic, more effective pediatric cancer therapies; to expand resources for childhood cancer research and specialized care; and to address the unique needs and challenges of childhood cancer survivors.

The story of childhood cancer is two-fold: it is one of scientific progress, medical success and hope; and at the same time, it is a story of pain, loss, and frustrating stagnation. In the last 30 years, survival rates for some pediatric cancers have significantly increased and are approaching 80 or even 90 percent, yet for other cancers mortality rates remain high and have scarcely budged in decades. Despite our progress for some patients, cancer remains the number one cause of death by disease in our children, claiming the lives of some 2,500 children annually. Further, for those who do survive, the threat and reality of serious long-term medical, social and emotional effects as well as premature death due to their disease and current toxic treatment regimens is ever present. New therapies are sorely needed to improve treatment outcomes and avoid the negative long-term effects.

There are many obstacles to development of these treatments for kids such as: small patient populations that offer little market incentive or human resources for research; ethical considerations around research in children; and long delays between the time testing is begun in adults, for whom the drugs are developed, and in children. Testament to these hurdles is the fact that only one oncology drug has been developed for pediatric cancer in the last 20 years. While the advance of genomics has led to significant and exciting scientific discovery related to cancer in recent years, children with cancer are not reaping the benefits. We need a new approach.

CCCA has long recognized that relying on the private sector to develop new pediatric oncology drugs is not adequate and there must be a role played by other entities, including the federal government, to identify and prioritize promising therapeutic targets and "de-risk" drug development for children with cancer. For this reason, we believe that a re-ordering of our nation's premier research programs and centers to maximize new scientific discoveries with the goal of facilitating access to better treatments and cures for childhood cancer and other diseases that have been neglected is wholly appropriate. We cannot let rapid scientific progress be stymied by organizational inefficiencies, and we believe that a re-tooling of the NIH to help bridge the gap between basic research and clinical application is a necessary step. We are hopeful that the creation of a National Center for Advancing Sciences at NIH will promote synergies among the various programs that are geared toward translational research and effectively leverage resources to facilitate therapeutic development for patients in need.

CCCA wishes you success as you take on the task of this ambitious reorganization. Thank you for your commitment to a vision of NIH that supports greater scientific collaboration and public-private partnerships that benefit Americans searching for hope.

Sincerely,

Lori A. Salley
Executive Director