Recognizing National Neuroblastoma Awareness Day

Floor Speech

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Mr. McCAUL. Mr. Speaker, I rise today to recognize September 20th as National Neuroblastoma Awareness Day. Neuroblastoma is a deadly pediatric cancer that primarily strikes infants and young children. Of the 15,780 new cases of pediatric cancer in the U.S. each year, approximately 700 are neuroblastoma diagnoses. About half of these children will have an advanced-stage, high-risk form of disease. Even with aggressive treatment, only 40 to 50 percent of high-risk patients will survive. Neuroblastoma is the most common extra-cranial solid tumor among children and the most common cancer in infancy. The cause of the disease is unknown but leads to abnormal cell growth during the development of the sympathetic nervous system.

I am pleased to inform my colleagues that we have seen significant progress this year in the fight against this devastating disease. In March, the Food and Drug Administration approved the first drug ever to treat children with high-risk neuroblastoma. In August, the same product received regulatory approval in the European Union. The drug, Unituxin (dinutuximab), is marketed by United Therapeutics Corporation. United Therapeutics was also granted a Pediatric Rare Disease Priority Review Voucher by the FDA. This innovative voucher program was established by the Creating Hope Act--legislation that I sponsored with my colleagues Congressman CHRIS VAN HOLLEN (D-MD) and Congressman G.K. BUTTERFIELD (D-NC). Enacted into law in 2012, the Creating Hope Act is designed to incentivize the pharmaceutical industry to invest in new therapies for rare childhood diseases.

Approval of this groundbreaking therapy is the result of a unique public-private partnership over many years. Originally developed by Dr. Alice Yu, University of California San Diego, the drug was tested in high-risk neuroblastoma patients in clinical studies conducted by the Children's Oncology Group through support from the National Cancer Institute (NCI). Manufacturing of the complex chimeric antibody was conducted by the NCI at its biopharmaceutical laboratory in Frederick, Maryland. In 2010, United Therapeutics entered into a Cooperative Research and Development Agreement with the NCI where the company assumed responsibility for manufacturing the drug and moving it through the regulatory approval process.

According to Dr. Malcolm Smith, Associate Branch Chief, Pediatrics in the Cancer Therapy Evaluation Program at NCI, ``The FDA approval of dinutuximab represents the culmination of a remarkably productive collaboration between researchers of the NCI-supported Children's Oncology Group, the manufacturing and clinical research groups of NCI, and the oncology team at United Therapeutics. Children with neuroblastoma will benefit from this collaboration, and the drug development pathway blazed by dinutuximab will likely be followed in the future to develop other novel agents directed against pediatric cancer therapeutic targets.''

Mr. Speaker, I have the privilege of co-chairing the Congressional Childhood Cancer Caucus with Congressman VAN HOLLEN. Each September, the Caucus commemorates National Childhood Cancer Awareness Month by hosting a Childhood Cancer Summit on Capitol Hill. This event features pediatric cancer patients, advocates, physicians, industry partners and other key stakeholders. As part of this year's Summit on September 18th, we will hear from Casey and Lesley Ryan, the parents of Rex Ryan, a young neuroblastoma patient from my home state of Texas. We will also hear from Roger Jeffs, PhD, President and Co-Chief Executive Officer of United Therapeutics, Dr. Lee Helman from the National Cancer Institute, Dr. Michael Link of the Stanford School of Medicine, Dr. Amy Fowler of the Dell Children's Medical Center, and Danielle Leach of the St. Baldrick's Foundation.

As we recognize the progress that has been made in neuroblastoma treatment, we remain focused on the many challenges that remain and the toll this disease has taken on so many families. One such family is the Lindbergs from Germantown, Maryland. Wendy and Gavin Lindberg lost their 7 year-old son Evan to neuroblastoma in 2010. He was their only child. Diagnosed at the age of 3, Evan waged a four-year battle against Stage IV neuroblastoma that defined courage. Evan was a remarkable little boy who inspired everyone he met with his bravery, compassion and joyful approach to life.

In his memory, Wendy and Gavin established The Evan's Victory Against Neuroblastoma Foundation to promote awareness of the disease, fund much-needed research, and support patient wellness programs for children in treatment. Since Evan's passing, the Foundation bearing his name has made and continues to make a real difference in the lives of children and families suffering from neuroblastoma. There are many other organizations doing wonderful philanthropic work in memory of children lost far too young to this terrible disease. Their strength in the face of adversity compels us to do all we can to help families facing the unthinkable.

So Mr. Speaker, I am proud to rise in recognition of September 20th as National Neuroblastoma Awareness Day and encourage my colleagues to join in the fight against all pediatric cancers. Our children's future depends on it.

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