May Bulletin

Formal Report Issued to FDA on Chemo-Induced Hearing Loss in Pediatrics

As one of the co-hosts of last fall's workshop on chemotherapy-induced hearing loss in pediatrics, the Children's Cause is proud to share the formal Voice of the Patient report submitted to the FDA this week: View Report.

Young adult survivors of childhood cancer and parents of children with cancer participated in this externally-led Patient Focused Drug Development meeting on September 13, 2018, in the DC metro area. This meeting was an incredibly empowering day for childhood cancer survivors and family members, giving them a long-deserved spotlight to share their very personal stories about the severe impact that hearing loss has had on their lives.

The formal report provides the FDA with patient perspectives to inform their regulatory decision-making and improve the drug development process. It is our hope that this report will be used to guide approvals of much-needed therapies to prevent - and ultimately treat - chemotherapy-induced hearing loss in survivors of childhood cancer.

Among the key points in the report:

  • Survivors with hearing loss report severe social isolation and anxiety as their single most challenging quality-of-life issue, describing difficulties in trying to keep up at school or interact with colleagues at work.

  • Although patients use a variety of devices, therapies, and adaptation strategies to manage the consequences of hearing loss, the effectiveness is limited and each modality has significant downsides, with 37% of respondents indicating that these interventions did not help at all.

  • Hearing aids are the most widely-used intervention but can have significant disadvantages, including discomfort, expense, reliance on battery life, and poor performance in noisy environments.

  • The number one issue that worries caregivers and patients the most about hearing loss is that its severity will worsen, impacting related issues like tinnitus and balance.

This FDA-approved, externally-led meeting was organized and presented by the Children’s Cause for Cancer Advocacy, Children’s Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology.

Learn more at

Promising Early Results from Pediatric MATCH Trial

Researchers convene tomorrow in Chicago to kick off the 2019 ASCO Annual Meeting, and one abstract session will be focused on encouraging early results out of the Pediatric MATCH Trial. 

Pediatric MATCH is a nationwide clinical trial led by the National Cancer Institute and the Children's Oncology Group, aimed at children and adolescents ages 1-21 with solid tumors that are not responding to treatment. The tumors of enrolled children are genetically sequenced to identify genetic alterations that 'match' an investigational targeted therapy. 

When Pediatric MATCH launched in 2017, it was estimated that there would be a match rate of about 10 percent, meaning one-in-ten participants were expected to be eligible to receive treatment with at least one drug. Early results, however, are much more promising: An analysis of 400 patients screened reveals a match rate of 24 percent. 

More from the ASCO News Release

  • 'Targetable alterations were detected in more than 40% of patients with brain tumors and more than 25% of patients with the other cancer types tested (other solid tumors, lymphomas, and histiocytic disorders) demonstrating the utility of tumor screening for children with both common and rare cancers.'

  • 'Pediatric MATCH is anticipated to enroll at least 1,000 patients. Study investigators plan to continue to add new targeted therapies to the trial in an attempt to further increase the number of patients who could be matched to drug treatments on the study – protocols for four additional drugs are currently under development.'

Appropriations Update: House Committee Approves NIH Funding Boost

Earlier this month, the House Appropriations Committee approved a $189.8 billion spending bill for fiscal year 2020 that includes $41.1 billion for for the National Institutes of Health (NIH). This amounts to a $2 billion boost for the NIH, including a $300 million increase for the National Cancer Institute and $50 million for childhood cancer research. 

The FY 2020 funding discussions are weaving through some arcane budget mechanics involving budget caps, the debt ceiling and priority setting. Read more about the specifics on our blog and then help us make sure an NIH funding boost gets through the lengthy appropriations process: Your Role in Childhood Cancer Research Funding.

Brain Tumor Awareness Month Comes to a Close

Brain tumors are the second most common type of childhood cancer (after leukemia) and the leading cause of cancer deaths among children and adolescents. Today, 3 out of every 10 childhood cancer deaths are caused by brain cancer. 

Despite significant research progress over the last several decades, survival rates for certain brain cancers remain perilously low. Every year, 200-400 children in the U.S. will be diagnosed with diffuse intrinsic pontine glioma (DIPG) - a brain cancer with a 0% survival rate. Most of these kids will live less than a year after a diagnosis of DIPG. Nothing has changed for DIPG in the last 40 years in the standard treatment nor in its terminal prognosis. 

Learn more about childhood brain tumors: 
National Cancer Institute Overview
Pediatric Brain Tumor Consortium

May is also Melanoma/Skin Cancer Detection and Prevention Month:

A recent study noted that childhood cancer survivors who had lived five or more years from their primary malignancy are at an increased risk of developing skin cancer compared with the general population.

The most common skin malignancies identified in the study group included basal cell carcinoma, melanoma, and squamous cell carcinoma. Of note, approximately 40% of the study group received radiation as part of their initial treatment.

The best line of defense for everyone - especially childhood cancer survivors: wear sunscreen and limit sun exposure. Examine carefully any moles or other spots on your child's skin. Pay particular attention to the area that received radiation if you are a childhood cancer survivor. Be sure to tell your doctor if you find any abnormal bump, mole or spot.

Take Action on the CLINICAL TREATMENT Act

Medicaid is the only major payer not required to cover the routine care costs associated with clinical trials -- a coverage gap that would be addressed by the CLINICAL TREATMENT Act (H.R.913). This month, we joined with ASCO in a community-wide push, asking lawmakers to support this legislation. You can still join in that effort, here: Learn more and take action.

At least one-third of childhood cancer patients depend on Medicaid coverage for their care. The CLINICAL TREATMENT Act (H.R. 913) would require Medicaid to cover routine costs associated with enrollment in approved clinical trials for those with life threatening conditions like cancer.

Most children are treated in the context of clinical trials, and their routine care - such as doctor visits and lab studies - is covered by insurance, while the research costs of a trial are covered by a sponsor (most often the National Cancer Institute). 

Unfortunately, Medicaid recipients cannot be certain that their care costs will be covered, because of the patchwork of coverage standards across state Medicaid programs. The CLINICAL TREATMENT Act will address the coverage gap for many Medicaid recipients and will eliminate a disparity in access to clinical trials that currently exists. 

Quick Links | Recommended Reading

  • T-Cell Therapy & Relapsed Neuroblastoma: "A new phase I clinical trial run jointly between Baylor College of Medicine and Texas Children's Hospital is evaluating [a] brand new treatment approach for children with relapsed neuroblastoma. The CAR-NKT cell treatment CMD-501, developed by UK-based cellular therapy company Cell Medica is the first of its kind to ever be trialed in any disease." (Forbes, 5.1.19 / Press Release, 5.30.19)

  • Mice Trials Advance DIPG Research: "Scientists at The Institute of Cancer Research, London, led research with an international team of colleagues, finding that the new drug class can kill brain cancer cells with mutations in the ACVR1 gene and shrink tumours in mice. Open science company M4K (Medicines for Kids) Pharma has taken on development of the ACVR1 inhibitor drugs -- and clinical trials in children with brain cancer are expected to begin in 2021." (Science Daily / Communications Biology, 5.9.19)

  • New Drugs Take 6.5 Years Longer to Reach Children: "Cancer drugs approved by the U.S. Food and Drug Administration took a median of 6.5 years to go from the first clinical trial in adults to the first trial in children, according to a study at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. ... In the U.S., the recent RACE for Children Act strengthens the requirement that new cancer therapies with potential biological relevance to pediatric cancers be evaluated in children. This study can serve as a benchmark as this new policy is enacted." (Dana-Farber, 5.21.19 / European Journal of Cancer)

Jessica Kean
April Bulletin

ACE Kids Act Signed into Law

The President signed the ACE Kids Act into law this month, as part of theMedicaid Services Investment and Accountability Act of 2019.

The Advancing Care for Exceptional Kids Act will improve care for approximately two million children with medical complexities in the Medicaid program. We estimate that approximately 1/3 of the nation's children with cancer are covered by Medicaid. 

The bill will provide more consistency for children with medical complexity as they often need to travel across state lines for care, which currently results in coverage issues since Medicaid coverage may differ from state to state. The Children's Cause joined the nation’s leading children’s hospitals, along with the Children’s Hospital Association, in supporting this legislation.

Providing children with complex medical conditions enrolled in Medicaid the best possible care is a national challenge, and passage of this legislation brings us closer to ensuring these vulnerable children receive the care they need.

Survivor Scholarship Recipients Announced

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We are excited to introduce the 2019 class of our College Scholars Program! We congratulate Amelia Corl and Isha Kunnath, chosen from among a competitive field of applicants.

About the College Scholars Program: Children’s Cause offers an annual opportunity for two childhood cancer survivors to gain valuable cancer advocacy experience. College Scholars, who receive a $2,000 academic scholarship, agree to complete a project of their choosing related to childhood cancer advocacy.

Meet Amelia: Amelia was an 18-year-old freshman at William & Mary when she suddenly found herself thrust into the world of cancer with a diagnosis of Hodgkin’s lymphoma. This May, Amelia graduates as a survivor and an advocate; this fall, she'll begin her graduate studies at George Washington University. For her College Scholars project, she plans to start up a new student organization for survivors of serious childhood illnesses like cancer and others managing chronic diseases like diabetes. Amelia’s organization will be a support group but also an advocacy network that works to tackle policies around medical leave, student accessibility, and student health resources — finding solutions to the same problems that plagued her as a patient and student.

About Isha: Isha was diagnosed with Pre-B Acute Lymphoblastic Leukemia in 7th grade. As a high school senior, she's taken courses in DNA and has a research-based internship under her belt, working toward the goal of becoming a biologist and cancer researcher. This fall, she’ll begin her undergraduate studies as a biology major at the University of Virginia. To combat social isolation faced by pre-teens and teens with cancer, Isha wants to create an online community for 10-18 year old patients and survivors.Isha’s program will include a website with message boards and chat rooms to connect kids with shared interests. In addition to making peer connections, she also wants to create a mentorship component that connects these pre-teens and teens with adult survivors of childhood cancer.

Find out more about Amelia and Isha and the projects they'll be taking on this summer and fall: Meet Our 2019 College Scholars.

Childhood Cancer Advocates Collaborate at AACR Annual Meeting

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Childhood cancer advocates, including our founder Susan L. Weiner, were among the thousands of oncology experts in attendance at this month's American Association for Cancer Research (AACR) Annual Meeting in Atlanta.

Susan was part of a major symposium panel on pediatric oncology focused on the global potential of the ACCELERATE multi-stakeholder platform. This session featured representatives from FDA, the Pediatric Cancer Working Group and international stakeholders.

The ACCELERATE program provides a transparent forum to tackle overarching issues in the development of innovative therapeutic medicines with the goal of improving global outcomes for children and adolescents with cancer. Dr. Weiner serves on the ACCELERATE Steering Committee, as one of just three committee members from the United States. 

The Steering Committee hopes to one day bring a Pediatric Strategy Forum to the U.S. — a goal made closer by this new exposure at AACR.

At the same session, Dr. Greg Reaman of the FDA described the RACE for Children Act and credited the childhood cancer advocacy community for working collaboratively over many years to achieve the 2017 passage of this landmark legislation, designed to increase kids’ access to new cancer drugs.

Other coalition achievements featured at AACR included a poster presentation by Alliance for Childhood Cancer Chair and St. Baldrick’s Advocacy Director Danielle Leach on the Childhood Cancer STAR Act, and a poster presentation by CAC2 President Vickie Buenger on the power of collaboration and cooperation in the childhood cancer community.

Take Action on NCI Funding

Congress returns to Washington this week after a two-week Easter recess and will resume budget talks for fiscal year 2020. Now is the time to emphasize to your Senators and Representative the life-saving importance of robust funding for the National Institutes of Health, including the National Cancer Institute (NCI). We've made it easy for you to do just that with the top action alert on this page.

Talking Points on NCI Funding:

  • The Trump Administration has called for a 17 percent cut to NCI funding for fiscal year 2020, which would have a devastating impact on cancer research and significantly delay progress toward reaching cures.

  • Cancer remains the number one disease killer of children, and some pediatric cancers remain terminal upon diagnosis.

  • The vast majority of childhood cancer research is funded by the NCI, which provides grants to researchers and supports cooperative groups that test new treatments in clinical trials.

Read more:
- "Experts applaud $500 million proposal for pediatric cancer research but say more is needed" (HemOnc Today, 4.8.19)
- "The power of pediatric cancer research" (Danielle Leach - The Hill, 4.12.19)

Children's Cause Can Help with Graduation

Instead of mailing graduation announcements this spring, consider sending them online. There are several great designs, and Children's Cause receives a donation for every one you send via EcoCards.

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We can also work with you to create a custom donation form for special occasions like graduations, birthdays or bar mitzvahs. Learn more.

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Quick Links | Recommended Reading

  • Air pollution and childhood cancer survivors: "The research...found a strong connection between those who had survived childhood cancer and those being hospitalized for breathing problems. Most of the episodes, the study found, came within three days of a major bad air event — primarily inversions, which impact the Salt Lake Valley in the winter months." (The Salt Lake Tribune, 4.23.19)

  • Global incidence rates in children under 5: "The study found in children under 5 years, cancer rates increased for both common types of childhood leukemia, one brain tumor subtype, neuroblastoma and hepatoblastoma." (University of Minnesota, 4.22.19 / JNCI Cancer Spectrum)

  • Proton therapy and neuroblastoma: "Researchers analyzed the largest cohort to date of pediatric patients with high-risk neuroblastoma treated with proton radiation therapy, finding both that proton therapy was effective at reducing tumors and demonstrated minimal toxicity to surrounding organs." (Children's Hospital of Philadelphia, 4.10.19)

  • Survivors and stroke risk: "In addition to the dose level of cranial radiation therapy that increases stroke risk up to 11 fold, variants on a chromosomal region called 5p15.33 increase risk approximately three-fold overall. ...This is the first study to link the chromosomal region with an increased risk of stroke." (St. Jude, 4.2.19)

Jazz in June Tickets on Sale


We invite all of you in Washington, DC, Maryland, and Virginia to join us on Monday, June 17th at the Hamilton in Washington, DC from 6pm - 8pm. It promises to be a terrific evening of music, food, fun and friends. And you don't want to miss our silent auction this year -- especially foodies, sports fans and adventurers!

Tickets are now available, at this link. Get yours today!

Jessica Kean
March Bulletin

President's Budget Proposes Cuts to NIH

Childhood cancer advocates were encouraged when President Trump announced a childhood cancer research initiative at the State of the Union address, proposed at some $500 million over ten years – or $50 million a year.

But the promise of that extra funding and any new initiatives was thrown into doubt this month with the release of the Administration’s budget proposal. It recommended that the National Cancer Institute (NCI) be cut by 17% or $897 million; theoretically dwarfing any increase in childhood cancer funding.

We break this down in more detail on our blog but here's what you need to know: Even in the most harmonious environment, the President's budget is merely a suggestion. In this case it’s more like ‘dead-on-arrival.’ Over the coming months, Congress will decide what is available for each agency and program in a deliberate process, with input from stakeholders, including us.

It will be critical as childhood cancer advocates, patients, families and supporters, that we appeal to our representatives — and we've made it easy for you to do just that with the top action alert on this page.

It’s imperative that our community stand shoulder-to-shoulder with other cancer groups to ensure that total NCI funding is increased. A rising tide lifts all boats. At the same time, our community needs to stress the urgent need that the promise of the STAR Act be fulfilled and that the President’s promise of childhood cancer is met – so that no child is left behind. Take action now.

College Scholars Program: Last Chance to Apply!

The deadline to apply for our College Scholars Program is just two days away -- March 31st at midnight EST. We're awarding two survivors with a $2,000 academic scholarship, along with the chance to gain valuable cancer advocacy experience.

Our College Scholars Program includes a requirement to complete a childhood cancer advocacy project of the scholar’s choice. Projects could be as extensive as a summer internship in our DC office to something more local, like organizing an awareness event in your hometown or mobilizing other students at your school in a letter-writing campaign. Your creativity is encouraged, and you’ll receive mentorship and support from the CCCA team toward completing your chosen project. 

Learn more and apply here. Award recipients will be selected and notified by April 15th.

Webinar Recording Now Available

We held a webinar this week to provide an update on progress since last fall's externally-led, FDA-approved Patient Focused Drug Development (PFDD) meeting on chemo-induced hearing loss. If you weren't able to join us, the webinar recording is now available:

We are working to finalize the formal "Voice of the Patient" report that will go to the FDA. If you want to be among the first to receive the final version of that report once it's ready, sign up on the project website:

Update Your Contact Information

We are in the process of updating our advocate and donor contact information. Can you help? Please take a few minutes to make sure we have your current phone number, email, and mailing address. If you are not sure we have your correct address information, please provide your information by completing the form at this link.

We also want to learn more about you so we can deliver the information you find most important. Are you a parent of a childhood cancer survivor or current patient? Are you most interested in new treatments, survivorship, or advocacy -- maybe all three or something else altogether? Please share that with us as well:

Reframing Medicaid : An ACS Initiative

If you have Medicaid or are in the health insurance gap, we want to hear from you! We're supporting an American Cancer Society initiative to collect stories that put a human face to Medicaid, to combat common misconceptions about Medicaid and the families who rely on it to keep their children healthy. If you're willing to open up about your Medicaid story - and focus attention on this issue - either send us a note at so we can touch base or fill out this form at the ACS website.

Results from our Two Year Patient Needs Survey

In both 2017 and 2018, we conducted an online survey in partnership with the Patient Advocate Foundation. We heard from nearly 450 caregivers and survivors, and now we're sharing a few top findings related to survivorship, financial hardship, and coverage challenges.

Key takeaways:

  • 60% of childhood cancer families we surveyed either don't have a written survivorship care plan or are unsure if they do. Follow-up care plans are a critical tool for transitioning care to adult providers and managing long-term side effects.

  • Among parent/caregiver respondents in both 2017 and 2018, 6-in-10 reported experiencing a financial hardship over the past 12 months due to the cost of their child's medical care. Of those who have experienced a recent hardship in both years’ surveys, approximately one-third categorized that hardship as extremely severe, unable to manage.

Among caregivers surveyed in both 2017 and 2018, one-third reported insurance denials for treatment. The impact of those denials ranged from forcing caregivers to spend time navigating their insurance plan to requiring out-of-pocket expenses or loans and interrupting or changing the course of their child’s treatment.

Read more here, including personal testimonials.

Save the Date for Jazz in June

We invite all of you in DC, Maryland and Virginia to join us on Monday, June 17th at the Hamilton in Washington, DC from 6pm - 8pm. It promises to be a terrific evening of music, food, fun and friends. And you don't want to miss our silent auction this year -- especially foodies, sports fans and adventurers!

Tickets are now available, at this link. Get yours today!

2018 Annual Report Now Available

We're proud to announce the release of our 2018 annual report, an electronic booklet filled with our most notable achievements and accomplishments from the 2018 calendar year, plus a financial reporting of our revenue and expenses.

Please visit and click on the cover image to flip through this document to see how your financial support in 2018 translated into real progress on behalf of children with cancer and the nation's 500,000 childhood cancer survivors.

Jessica Kean