On Thursday, February 13, 2020, in the Gold Room of the Rayburn House Office Building in Washington, DC, diffuse intrinsic pontine glioma (DIPG) patient and family advocates joined national experts in childhood brain tumor research as well as representatives from government and industry at a Congressional Briefing titled DIPG, Pediatric Brain Cancer and the Importance of H. Res. 114. Presented by the DIPG Advocacy Group in collaboration with Congresswoman Jackie Speier (D-CA), the event was held to urge the 116th Congress to pass U.S. House Resolution 114 (H. Res. 114).
H. Res. 114 calls for the designation of the 17th of May each year as “DIPG Awareness Day” to raise awareness and encourage research into cures for diffuse midline gliomas (including DIPG), and for childhood cancers in general; as well as to open opportunities for data-sharing, and to push for increased private and public funding for research and new clinical trials for treatment. Additionally, the resolution supports arming patient families and doctors with more knowledge from the date of diagnosis concerning experimental research, clinical trials, and/or treatment strategies.
Res. 114 is not a law, but an apolitical resolution with no funds allocated. It only requires a House vote to pass. At its heart, the resolution calls on Americans to focus attention on DIPG and all childhood brain cancers as urgent priorities in research. H. Res. 114 shines a spotlight on a disease that affects 200-400 children in the U.S. each year, with a five-year survival rate of less than 1% and an average life expectancy of 9 months, post-diagnosis. Brain tumors are the leading cause of cancer-related death in U.S. children, with DIPG and other diffuse midline gliomas being the 2nd most common form or malignant pediatric brain tumor.
In 2019, 32 states recognized May 17 as DIPG Awareness Day. H. Res 114 was introduced into the 115th Congress on February 8, 2019 with 17 original co-sponsors, including Congresswoman Jackie Speier (D-CA) and Congressman David Joyce (R-OH). As of March 2, 2020, 99 co-sponsors have signed on in support of the Resolution.
DIPG Advocacy Group member Janet Demeter of the Jack’s Angels Foundation opened the briefing and gave an overview of H. Res. 114. In her remarks, Demeter shared the sad fact that there has been no significant change in either the standard of treatment or survival prognosis for children with DIPG and other diffuse midline gliomas since astronaut Neil Armstrong’s daughter died from the disease in 1962. She went on to say that “40 years ago, Leukemia was also a death sentence. But we recognized that – we called it out by name. Since then, huge strides have been made in the treatment of that disease. Sadly, the same cannot be said for brain cancer in kids. That is why awareness is so important. We can do this [better treat DIPG] with increased attention and resources.
Since H. Res. 114 was first introduced in the U.S. House of Representatives in 2016, 1500 more children have died from diffuse midline glioma brain tumors.
Co-Project Lead of the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and Children’s Brain Tumor Tissue Consortium (CBTTC) Principal Investigator Dr. Sabine Mueller was among the research experts to lend support to the House Resolution at this event. She began her remarks by sharing the clinical realities for children diagnosed with DIPG and other diffuse midline gliomas. “DIPG is the deadliest tumor for children. Most who are diagnosed survive for less than one school year,” said Dr. Mueller. “We need true collaboration to change this outcome.”
Collaboration in fighting this disease is not limited to the laboratory or clinic. Dr. Mueller also informed the gathered attendees and congressional representatives that, currently, DIPG research is funded solely by philanthropy. “It is critical to raise awareness and funding for this deadliest of diseases,” she concluded.
Scientific Co-chair of the CBTTC and Scientific Chair of PNOC, Dr. Adam Resnick also spoke at the briefing. Dr. Resnick shared the value of DIPG research to science, industry, and data management, including what the scientific community is collectively learning about pediatric brain cancer and how increased attention to this research could not only improve our understanding of the human brain itself, but also lead to more effective treatments for all forms of cancer, ultimately saving more lives.
Additionally, Dr. Resnick stressed the importance of sharing data across disease types outside of the childhood cancer space. As an example, he noted that one fifth of all DIPG patents share a mutation with Fibrodysplasia Ossificans Progressiva (FOP) patents. “The key is to harness both the connectivity but also new practices around the speed that information is shared. Time is that variable around which we need to optimize,” Resnick said.
In total, seven panelists delivered remarks to the gathered supporters of H. Res. 114 and congressional representatives present at the briefing. Dr. Josh Allen, Senior Vice President of Oncoceutics, a biotech firm responsible for the promising diffuse midline glioma clinical trial ONC-201; and Dr. Charles Keller, the Scientific Director of the Children’s Cancer Therapy Development Institute (cc-TDI), both spoke about the challenges that exist in developing promising new therapies. They each repeated that it comes down to lack of awareness, investment, and collaboration in tackling DIPG and other childhood brain cancers.
“Some of the cancers I was taught 10 years ago were incurable are now treatable thanks to new drug therapeutics. We have not yet made those breakthroughs for DIPG,” said Dr. Allen. In his remarks, Dr. Keller expanded on this, reporting that, on average, there are 12 FDA-approved drugs per year for adult cancers. However, only 8 have been approved for all forms of childhood cancer since 1978. There have been no FDA-approved drugs for childhood brain tumors.
The most powerful remarks from the briefing came from Jace Ward, a 21-year-old college student who was diagnosed with DIPG brain cancer in May of 2019. Since his diagnosis, Jace has become a leading voice in pushing researchers, funders, public policymakers, and the community at-large to place a greater priority on breaking down barriers to discovering new treatment strategies, as fast as possible.
“According to estimates, I should be dead in four days. Any day past that, I’ll consider myself lucky. Please consider my request to pass H. Res. 114. I may not be here again next year,” Ward said. “DIPG is not rare. It’s just rarely talked about…As a nation, we are not aware. We are failing these patients and their families. Awareness will lead to more cures.”
Following the congressional briefing, gathered supporters of H. Res 114 were invited to attend the State of DIPG Summit, hosted by Covington & Burling, LLP and the Michael Mosier Defeat DIPG Foundation. At the summit, patient advocates joined medical researchers to share perspectives on the state of diffuse midline glioma research, drug development, data sharing and collaboration, how patient families can empower research efforts, how to navigate the treatment landscape, and the experience of living with a DIPG diagnosis.
Speaking at the summit, Amanda Haddock, mother of a child who passed away from DIPG brain cancer and co-founder of Dragon Master Foundation, spoke about the importance of collaborating, and of patents and families taking ownership of their medical data to drive discoveries that could lead to a cure.
“It’s important for families and policy makers to understand how open data fits into patient care. First and foremost, data belongs to the patient and they should have final say in where it goes and how it is used. A patient’s data is no longer just for research but will increasingly be used to guide their own care,” Haddock said.
“Brain cancer data is rare, making access to it all the more important. It also means that we need to make comparisons across brain cancers and across different types of diseases in order to identify new targets and stop wasting time on dead leads. I’ve heard international data sharing experts state that no single country can amass the data to solve this highly complex set of puzzles on their own,” she added.
As with the congressional briefing, the most powerful comments shared at the summit were given by three young people currently fighting diffuse midline gliomas: Jace Ward, Anjalie Bartee, and Katie Bedingfield. The three spoke eloquently on behalf of all children and young adults who are currently suffering and searching for a cure. Each of them urged our collective community to do everything possible to drive research into these diseases and bring awareness to the devastating impact that brain tumors have on children across the U.S.
“I want to point out that even though I was given a survival window of less than one year, it still took more than 12 months to get onto the ONC-201 clinical trial. While this trial is giving me hope for a longer life, many don’t have the same option,” said Katie Bedingfield.
“DIPG won’t wait until this is convenient. DIPG won’t wait until we are ready. While we have been ‘waiting’ to take a solid stand, DIPG has been taking the sight, the hearing, the speech, the ability to swallow, and eventually the breath of thousands of kids across this country,” said Jace Ward.
To see video recordings of the #Moonshot4Kids DIPG Awareness Congressional Briefing and Summit, visit the Michael Mosier Defeat DIPG Foundation on facebook.