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Progress in Developing First Pediatric Cancer Treatments in Decades Highlights 4th Annual Childhood Cancer Summit

September 19, 2013

Progress in Developing First Pediatric Cancer Treatments in Decades Highlights 4th Annual Childhood Cancer Summit

WASHINGTON, D.C. – Progress into research and development of the first new pediatric cancer drugs in nearly 30 years was announced at the 4th Annual Congressional Childhood Cancer Summit on Capitol Hill today.

Memorial Sloan Kettering Cancer Center announced it has taken steps under the Creating Hope Act, legislation passed last year, to develop new treatments for rare types of brain tumors and other malignancies in children. 

The Creating Hope Act, passed by Congressmen Michael McCaul and G.K. Butterfield and which took effect less than one year ago, established a voucher system within the U.S. Food and Drug Administration that allows companies to receive faster approval of a more lucrative drug to offset the high cost of development of a pediatric cancer treatment that is otherwise cost-prohibitive. 

“Industry has become increasingly interested in working with pediatric oncologists” because of the Creating Hope Act, Dr. Crystal Mackall, Chief of Pediatric Oncology, National Cancer Institute, stated during today’s Cancer Summit.  The FDA confirms three additional applications for vouchers under the new law.

“Finally, now pharmaceutical companies can have a reasonable rate of return,” said Nancy Goodman, founder of Kids v. Cancer.

“The reason we formed the caucus was to give children a voice who don’t have one,” said Congressman McCaul, founder and co-chairman of the Congressional Childhood Cancer Caucus.  “At the end of the day we have to ask whether we’re making a difference in the lives of others, and I think with the Creating Hope Act we are.”

“I’m committed to working with Congressman McCaul and my colleagues on a range of initiatives to support children with cancer and their families. We know that investment in biomedical research is critical to treatments and cures for childhood cancer,” said Congressman Van Hollen, co-chairman of the Congressional Childhood Cancer Caucus. “That is why I’m working to replacing the deep, immediate cuts in the sequester, which are undermining lifesaving research at places like the National Cancer Institute and the National Institutes of Health.”

The pharmaceutical industry has approximately 900 adult cancer drugs in the development pipeline and almost none for children's cancers.  According to the Institute of Medicine, two out of three pediatric cancer survivors will develop at least one complication—many of them life threatening, including secondary cancers —due to their harsh therapy originally developed for adults.  Pediatric cancer is the number one disease killer of kids, yet research is profoundly underfunded. 

Another key research need emphasized during the summit was preparing for future advances in technology.  One critical way to address this need mentioned by Dr. Peter Adamson is to expand biorepositories to ensure that materials are available for testing as new tools are developed.  He pointed to the Caroline Pryce Walker Conquer Childhood Cancer Reauthorization Act (HR 2607/ S 1251) as important pending legislation to accomplish this goal.

Dr. Patrick Leavey of Children’s Medical Center Dallas highlighted an additional concern for the childhood cancer community, that is the necessity of improving survivorship care.  Citing a figure of 60% of childhood cancer survivors who will experience significant late effects, Dr. Leavey emphasized the importance of cancer treatment summaries to be made available to all patients and urged participation in national survivorship studies and pointed to the Childhood Cancer Survivors’ Quality of Life Act (HR 2058/S 1247).

Also participating in today’s Summit were Dr. Peter Adamson, Children's Hospital of Philadelphia and Chair of the Children's Oncology Group; Dr. Kim Kramer of Memorial Sloan-Kettering Cancer Center; Dr. Patrick Leavey, Children’s Medical Center Dallas and Dr. Andrew Litt, Chief Medical Officer, Dell Healthcare & Life Sciences.

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