[Congressional Record (Bound Edition), Volume 161 (2015), Part 10]
[Extensions of Remarks]
[Page 14561]
[From the U.S. Government Publishing Office, www.gpo.gov]




            RECOGNIZING NATIONAL NEUROBLASTOMA AWARENESS DAY

                                 ______
                                 

                         HON. MICHAEL T. McCAUL

                                of texas

                    in the house of representatives

                      Thursday, September 17, 2015

  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.
  

                          ____________________