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Action Days on Capitol Hill

May 4, 2017

I have recently returned home from my 3rd year participating in Childhood Cancer Action Days.  This advocacy trip has become an annual trip for me and one that provides clarity and great motivation to continue raising money and awareness for childhood cancer through Wade’s Army. Action Days provide childhood cancer advocates including individuals, foundations, oncologists, social workers, nurse practitioners, and survivors the opportunity to unite and, with one voice, apply pressure to our Members of Congress to protect the rights of cancer patients and refuse the notion that the problem of childhood cancer has been solved.

Cancer remains the number one cause of disease-related death for children in the United States and many childhood cancers still carry a dismal prognosis upon diagnosis and with relapse little chance of survival.

In the past 30 years only three new drugs have been FDA-approved for childhood cancer, while during the same time frame over 60 new drugs have been approved for adult cancers. Currently there are almost 900 drugs in the adult cancer pipeline and only a handful of those are in development for children. This innovation gap is due to the incentives companies have to focus on adult treatments and, as a result, the difficulties researchers have accessing promising new drugs for children.

Two-Thirds of childhood cancer survivors suffer from late effects of their disease or treatment, including secondary cancers, developmental disabilities, and organ damage. Survivors and their families continually struggle to find a facility that provides comprehensive survivorship care and we need to improve collaboration among providers so doctors are better able to care for their patients as they age.

Ever since Wade’s death, I have been trying to turn our experience into something productive. I’ve come to the conclusion that change requires collaboration between our leaders in government, government agencies, industry, pediatric oncologists, lab scientists, advocates, and private foundations that fund science. Action Days are about working together to create the environment that encourages all these entities to work collaboratively to make change.

We have gained some traction on Capitol Hill and are in a good position to generate change. As a part of the greater good, Wade’s Army will continue to focus on raising money and awareness for research but also continue to participate in the broader childhood cancer discussion so we can make the most of all of our efforts. In case you were wondering, here is a brief overview of our request to Congress and why:

  1. Childhood Cancer STAR Act (Survivorship, Treatment, Access and Research) is the most comprehensive childhood cancer bill ever taken up by Congress. It would expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences and enhance the quality of life for all childhood cancer survivors. It also ensures pediatric expertise at the National Institutes of Health (NIH) by requiring at least 1 pediatric oncologist on the National Cancer Advisory Board. 
  2. RACE for Children Act (Research to Accelerate Cures and Equity for Children Act) provides that the FDA would have the authority to require any new cancer drug to be studied in pediatric cancers for which the molecular target of the cancer drug is relevant. Now, cancer drugs are developed by molecular target (e.g., a PD1 drug, a EGRF drug, An ALK inhibitor drug), instead of by organ (e.g. lung cancer, breast cancer). While children’s cancers don’t start in the same organs as adult cancers , they do share the same molecular targets  (e.g. an ALK inhibitor that treats adults with lung cancer also treats kids with neuroblastoma). 
  3. Support an increase of $2 billion for the NIH (National Institutes of Health) including a proportional increase for the and NCI (National Cancer Institute) to ensure that promising childhood cancer research can be conducted.  Additionally, provide $300 million for the Cancer Moonshot Initiative at the NCI as authorized in the 21st Century Cures.

I sincerely thank you all for being a part of Wade’s Army and affording us the opportunity to continue Wade’s battle by financially serving other families and contributing to the most promising research of today.

With a Warm and Grateful Heart,

Heather DeBruin
Wade’s Mom

INTERNATIONAL CLINICAL TRIAL PROVIDES TARGETED THERAPY TO CHILDREN WITH NEUROBLASTOMA

INVESTIGATIONAL 3RD GENERATION TARGETED DRUG TO BE STUDIED AS A TREATMENT FOR PEDIATRIC PATIENTS WITH RELAPSED OR REFRACTORY ALK-DRIVEN NEUROBLASTOMA

March 30, 2017 – NEW YORK, NY:  Lorlatinib, an investigational drug candidate currently in late stage clinical development for the treatment of lung cancer may also prove effective for the treatment of the pediatric cancer neuroblastoma. Solving Kids’ Cancer is leading an international effort of like-minded nonprofits to provide nearly $400,000 of collaborative funding for an innovative clinical trial to bring a potentially life-saving therapy to kids.

The phase I trial will be co-led by Dr. Yael Mossé at Children’s Hospital of Philadelphia (CHOP) and Dr. Araz Marachelian at Children’s Hospital of Los Angeles (CHLA), and will be conducted in 14 hospitals in the United States and Canada through the New Approaches to Neuroblastoma Therapy (NANT) consortium, as well as two European sites including Royal Marsden in London and Institut Curie in Paris.  The study focuses on a subset of neuroblastoma patients with mutations in the anaplastic lymphoma kinase (ALK) gene. Abnormal ALK genes are found in 15 percent of high-risk neuroblastoma patients, and portend a very poor prognosis.

The primary objective of this study is to identify a recommended Phase II dose and assess the anti-tumor activity as a result of administering this investigational drug candidate in pediatric patients with ALK-driven neuroblastoma. Lorlatinib is a next-generation ALK/ROS1 tyrosine kinase inhibitor that has been recently reported to show very compelling responses in lung cancer patients with ALK aberrations. The drug was specifically designed to inhibit tumor mutations that drive resistance. One important feature of this drug is that it penetrates the blood brain barrier and may be effective in patients with relapsed neuroblastoma in the central nervous system.

 

“Lorlatinib has shown dramatic results in the lab against ALK+ neuroblastoma tumors in mice,” said Dr. Yael Mosse, the physician-scientist who discovered ALK mutations in neuroblastoma and led the preclinical investigations at the Children’s Hospital of Philadelphia.  “If this trial proves successful, we expect the drug to quickly become part of frontline therapy in children with ALK-driven neuroblastoma – which is a tremendous accomplishment in cancer research, overall.”

The hope is that this trial will provide a foundation for future work, which could lead to approval by health authorities for this use. Only three drugs have ever been approved for pediatric cancers, and this would set a remarkable precedent for rapid introduction of a new drug.  

"This clinical trial could be revolutionary, and we are thrilled to know that it will be expanded to continental Europe so we can reach more families," said Heather DeBruin from Wade’s Army. "Through collaboration, we're committed to helping neuroblastoma families wherever they are." 

 

Childhood cancer is the leading cause of death by disease in the United States, and neuroblastoma is a pediatric cancer that strikes mostly infants and young children. The aggressive cancer originates in the sympathetic nervous system and manifests as tumors in the neck, abdomen and pelvis.

“We are excited to see such an innovative clinical trial come to fruition, and we are grateful to our partners who helped fund the gap so that we can continue in our joint mission to improve the overall survival rates for children diagnosed with neuroblastoma,” added Scott Kennedy, Executive Director, Solving Kids’ Cancer US.  “This study represents an unprecedented collaboration between academic researchers, industry and charities to rapidly advance an exciting new agent, and provide access to the children who need it.”

WADE’S ARMY is committed to fighting Neuroblastoma through research and supporting families through the fight.

COLLABORATIVE PARTNERS SUPPORTING THIS STUDY:

Solving Kids’ Cancer US/EUR
The Band of Parents
The Ronan Thompson Foundation
The Children's Neuroblastoma Cancer Foundation
Wade’s Army
The Catherine Elizabeth Blair Memorial Foundation
The Evan’s Victory Against Neuroblastoma Foundation

Also supported by community funds from families including Harrison Bate and Ellen’s Friends and Family!