St. Baldrick’s Foundation Statement: The Budget Sequester – A Threat to Children with Cancer

The St. Baldrick’s Foundation calls on Congress to act now to prevent budget sequester cuts that threaten the more than 13,500 children who are diagnosed with cancer each year in the United States, the tens of thousands of infants, children, teens and young adults currently in treatment, and the 350,000 survivors of childhood cancers that live in our country.

The Realities of Childhood Cancer
Collectively, the dozen malignancies that are known as childhood cancers make up the #1 disease killer of children in the United States. Every 4 minutes, one of every five children diagnosed with cancer dies.

While progress in treatments for childhood cancers has largely been for one disease, many have cure rates of 5% or less. Young cancer patients endure chemotherapy, radiation and toxic therapies that leave two out of three of them with learning disabilities, hearing loss, heart disease, infertility, secondary cancers and other lifelong problems. Much more work is needed to reduce the long-term and life-threatening side effects of the existing standard of care.

At this time, researchers have done all they can with the drugs and therapies currently available to children with cancer. Patients, families, and researchers are hopeful that new treatments will be developed and approved soon. Now, children with cancer need advancements that are only available through research.

Much about cancer in general has been learned and can still be gained from continued, sustained investment in pediatric, adolescent, and young adult research. Many of the common therapies available to adult cancer patients today were pioneered in the treatment of children with cancer, including chemotherapy and bone marrow transplantation.

The Landscape of Childhood Cancer
As members of Congress debate the budget sequester, we ask that you all consider the entire landscape of childhood cancer research. Today, the incidence of childhood cancer is on the rise and threatening to offset the gains made over time. Pediatric oncology research is funded primarily by the National Institutes of Health (National Cancer Institute) and private philanthropy (including the St. Baldrick’s Foundation.) As it stands, a mere 4% of the NCI’s total annual cancer budget (and only some of those funds are available for new programs) is allocated to all pediatric, adolescent and young adult cancer research.

In comparison, cancer research focused on adults, median age of 66, receives 96% of the annual NCI budget with 60% of total funding for adults coming from the pharmaceutical industry. Childhood cancer research receives no funding from industry, as it is not profitable for pharmaceutical companies to develop and manufacture pediatric oncology drugs. The “playing field” is vastly uneven for childhood cancer research, even without the budget sequester. As Eugenie Kleinerman, M.D., Chair of Pediatrics at the MD Anderson Cancer Center, says, “Curing childhood cancers is equivalent to curing breast cancer, in terms of patient years of life saved.”

How the Budget Sequester Threatens Children with Cancer
Ninety percent of all children treated participate in the clinical trials provided by the Children’s Oncology Group, the NIH-funded pediatric cancer cooperative group. These clinical trials are young patients’ best hope for a cure. The COG is an innovative research engine that unites more than 8,000 highly dedicated pediatric oncology physician researchers who donate their time at more than 200 children’s hospitals, universities, and cancer centers. The world’s largest organization devoted exclusively to pediatric and adolescent cancer research, COG provides front-line therapies to the children and adolescents enrolled in more than 100 clinical trials at any given time. As a result, the COG has become the model for other NCI cooperative groups.

• Disastrous Domino Effect for the Children’s Oncology Group
  The planned budget sequester cuts of $2.4 billion to the NIH would hit kids with cancer extremely hard — now and in the future — by setting off a disastrous domino effect for the COG that would impact childhood cancer research for years to come. NIH funds for childhood cancer research would be slashed, causing enormous downstream effects. COG research geared up for 2012 would be halted, meaning that projects slated for 2013, 2014, and 2015 and beyond would be cut. Highly-trained COG staff members who work with researchers to coordinate clinical trials would be laid off. As a result, the intricate workings of the COG would freeze. If this were to happen, it will take years to restore the COG to the current pace of operations. Congress will have effectively denied the youngest, most vulnerable cancer patients the cutting-edge therapies they depend on to survive.

• FDA Drug Development and Approval Compromised
  Children with cancer depend on the innovations available through research and through the drug development process. The wide reach of the budget sequester would not only devastate childhood cancer research, but also the Food and Drug Administration, with $318 million in cuts slated for the agency. The FDA’s ability to review and approve drugs and therapies for patients of all ages would be severely compromised. Children with cancer are especially vulnerable, as they receive oncology drugs originally developed for adult patients. The toxicity of these drugs causes severe side effects and extreme long-term effects in these young patients, including heart disease, secondary cancers, infertility, and disability, among other problems. New, highly-targeted therapies are desperately needed to treat pediatric cancers. Added to this challenge is the fact that pharmaceutical companies often shy away from developing childhood cancer drugs because of their low profit levels (only two drugs have been approved by the FDA in the last 20 years to treat children with cancer). Further delay of the FDA’s review and approval of childhood cancer drugs and therapies will result in more children dying of cancer.

An Entire Generation of Childhood Cancer Patients Needs Congress’ Help
Robert Arceci, M.D., PhD, King Fahd Professor of Pediatric Oncology at Johns Hopkins University School of Medicine, co-director of the Michael J. Garil Leukemia Survivor’s Program, and Chair of the St. Baldrick’s Scientific Review Committee, describes the sequester by saying, “…the real lives of present and future children and adults are at stake by continuing to strangle research, science and translation of knowledge for the betterment of humanity. A sequester would be devastating.”

An entire generation of childhood cancer patients and survivors is watching Congress, wondering if they will continue to have access to the most promising treatments and therapies available if new, less toxic, more effective treatments will remain in the research pipeline. For young cancer patients, the budget sequester is not about public policy, it is a direct threat to their hope for cure, a direct threat to their lives.

Congress: Please Act Now to Stop the Sequester
On behalf of all children and teens with cancer, we appeal to every member of Congress to take the necessary steps to prevent the budget sequester, or at the very least, protect NIH/NCI and FDA funding from these devastating cuts.