Children's Cause for Cancer Advocacy is a strong advocacy and policy voice for children with cancer in national debates on research, new therapy development and health care reform. Our mission is to achieve a long, healthy life for every child with cancer.
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 last 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.
Recent news stories about hearing loss in pediatrics:
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, with generous support from the below sponsors.
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.
'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.'
As advocates in the childhood cancer community know all too well, funding for research is largely dependent on federal funding from the National Cancer Institute (NCI), which supports academic researchers and much of the clinical trials infrastructure that treats and cares for our kids.
The amount that NCI is able to devote to those programs – and other cancer programs - is, in turn, dependent on what Congress determines in its annual appropriations process. And much of that is determined by how hard advocates - like you – push their members of Congress to make it a priority.
In the past few years Congress has provided the NCI with some healthy increases: $293 million in FY 2018, and $179 million in FY 2019, along with some supplemental money from 21st Century Cures and the Cancer Moonshot Initiative.
With the passage of the milestone Childhood Cancer STAR Act last year, NCI could be provided with an additional $30 million annually — though that is dependent on annual funding. With those dollars made available this year, NCI has already issued a grant solicitation on survivorship and - more recently - begun a coordination effort on biorepositories. The community was heartened to hear the President propose an additional $50 million annually, and the NCI leadership has indicated any money provided for that purpose would be used to more comprehensively collect patient data for research.
The operative question is whether childhood cancer funding would receive that funding in addition to the STAR Act funding, or whether it - or other programs - might be cut to accommodate it. Advocates across the community have been seeking those assurances and much will depend on how vocal we are.
For fiscal year 2020, which begins on October 1, the process began with a dismal request from the President, suggesting a 17% cut at the National Institutes of Health (NIH), which would translate to a $897 million cut for the NCI. Of course: that’s not likely to happen. This was a starting point. And support for medical research is one area in which there is broad bipartisan agreement. The Alliance for Childhood Cancer and the rest of the cancer community is calling for at least a $378 million increase to NCI, for a total budget of $6.522 billion.
Beyond the NCI’s research agenda, progress in childhood cancer treatment is also dependent on the Food & Drug Administration (FDA) and its ability to review and approve new drugs. With the passage of the RACE Act last year, FDA now has the authority to require companies who are seeking approval for adult treatments to conduct clinical trials in children if the molecular method of action is similar. With disease increasingly defined by genetic markers, this is potentially groundbreaking for childhood cancer.
In FY 19, Congress committed more than $1.6 billion to support drugs, devices, biologics and other medical product elements of the FDA’s mission. This year, the patient community is asking for an additional $316 million to support the scientific capabilities of the agency.
As of this writing, the FY 2020 funding discussions are weaving through some arcane budget mechanics involving budget caps, the debt ceiling and priority setting. The good news is that the House has approved a $2 billion increase for NIH, including a $300 million increase for NCI and $50 million for childhood cancer research. The Senate, however, has not acted and probably won’t until later this summer. The final number will be determined when they negotiate a compromise.
This is where you come in. Members of Congress are now home for the Memorial Day recess. They will be at community events and holding meetings with constituents. This is your opportunity to tell your story and urge them to put childhood cancer research at the top of their agenda. Check their web site for events and availability and use our tools to prepare and make your pitch:
After your meeting, please take a moment to share with us here how it went. This information will remain confidential within Children's Cause staff and serves to help us improve our advocacy and refine our own work with these offices.
If you can’t meet with them, send a message using the action alert form provided here to urge them to support research funding.
If we all stick together and make our voices heard we’ll increase research, improve treatments, save lives and improve the outcomes for survivors.
Did you know Medicaid is the only major payer not required to cover the routine care costs associated with clinical trials? Please ask your lawmakers to support the CLINICAL TREATMENT Act requiring Medicaid to cover those costs.
Today, ASCO is leading a community-wide push for support of this legislation. Please join us in this day of action - especially if your Member(s) of Congress sits on the Senate Finance Committee or the House Energy and Commerce Committee:
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.
Clinical trials fuel treatment advances, including new drugs and other therapies, and also provide individual patients access to cutting-edge treatments through clinical trials enrollment. This bill would ensure that Medicaid recipients, including the roughly one-third of children with a cancer diagnosis who rely on Medicaid for their care, will see the financial barrier to clinical trials participation eliminated.
Please act now: Use the action alert below to ask your Members of Congress to support this bill.
We also encourage you to use social media to reach out to your Members of Congress. Here's a sample tweet: Medicaid is the only payer not required to cover routine care costs associated w clinical trials. @(insert your lawmaker’s twitter handle) please support HR 913 the CLINICAL TREATMENT Act, to ensure clinical trial access for Medicaid enrollees.
May is Brain Tumor Awareness Month and also Melanoma Awareness Month. To support awareness around these cancers, we bring you a few key points and resources:
BRAIN TUMOR AWARENESS MONTH:
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 standard treatment nor in its terminal prognosis.
Arielle was diagnosed with an aggressive brain tumor at just 8 days old. Today, she’s an energetic grade schooler. Read her story.
The signs and symptoms of childhood brain and spinal cord tumors are not the same in every child but can include:
Morning headache or headache that goes away after vomiting.
Frequent nausea and vomiting.
Vision, hearing, and speech problems.
Loss of balance and trouble walking.
Unusual sleepiness or change in activity level.
Unusual changes in personality or behavior.
Increase in the head size (in infants).
Resources for more information on childhood brain tumors:
Because exposure to UV light is the most preventable risk factor for all skin cancers, the American Academy of Dermatology encourages everyone to protect their skin from the sun’s harmful UV rays by seeking shade, wearing protective clothing, and using a sunscreen with a Sun Protection Factor of 30 or higher.
Melanoma symptoms vary from child to child. It’s a good idea to examine carefully any moles or other spots on your child’s skin.
When examining your child's moles, remember ABCDEF:
Asymmetry: Is one side of the mole different than the other?
Border irregularity: Are the edges ragged or irregular?
Color variation: Is the mole getting darker? Is part of it changing color or does it contain several colors?
Diameter: Is the mole bigger than ¼ inch?
Evolution: Is the mole growing in width or height?
Feeling: Has the sensation around a mole or spot changed?
Pediatric melanoma is rare. If your child has any of the above symptoms, they likely are caused by a less serious problem. However, be sure to tell your doctor if you see any abnormal bump, mole or spot on your child’s skin.
MELANOMA RISK FOR CHILDHOOD CANCER SURVIVORS:
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.
While Congress is on break, childhood cancer advocates press on for medical research funding. Join us:
Members of Congress are back home for a two-week district work period -- also known as Easter Recess. If you're interested in meeting with your Congressional representatives about childhood cancer issues, this is an ideal time to find your Senators and Representative somewhere near you!
Congress isn't due back to Washington until Monday, April 29th, and they're spending this time back home meeting with constituents just like you. If you'd like to meet with your Member of Congress to share your childhood cancer story, here's a good place to start: CCCA's Guide to Congressional District Office Visits. You may also want to check Town Hall Project to see if there's a town hall or "constituent coffee" event happening near you.
When Congress returns to Washington at the end of April, they will resume budget talks for fiscal year 2020. So 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).
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.
If you're not able to catch your Members while they're home this month, you can send them a note about NCI funding using the form at the bottom of this post.
If you do interact with a Member of Congress about NCI funding or another childhood cancer issue, please take a moment to share your experience with us via this Meeting Report form. This information will remain confidential within Children's Cause staff and serves to help us improve our advocacy and refine our own work with these offices.
Thank you for taking the time to share your story and our shared cause with lawmakers who have the power to create meaningful change for our kids.
Photo: Childhood cancer advocates gather at the 2019 American Association for Cancer Research. At far right is CCCA’s founder Susan L. Weiner, PhD.
Childhood cancer advocates, including our own founder Susan L. Weiner, were among the thousands of oncology experts in attendance at this week’s American Association for Cancer Research (AACR) Annual Meeting in Atlanta.
We’re particularly proud that 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. From the ACCELERATE site, these forums aim to “facilitate dialogue and provide an opportunity for constructive interactions between relevant stakeholders on topics requiring an open discussion on the development of medicines in the best interests of children and adolescents with cancer.” The 4th Pediatric Strategy Session is taking place this month in the Netherlands, on the topic of medicinal product development for acute myeloid leukemia in children and adolescents.
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.
Continue to follow this space for updates and developments on these collaborative efforts and achievements — and the impact they’re having on children with cancer and childhood cancer survivors.
Tom Coughlin is the Executive Vice President of Football Operations for the Jacksonville Jaguars and a two-time Super Bowl winning coach.
Most parents dream and save for their child’s future education; no parent dreams a child will be diagnosed with cancer, let alone saves for its financial chaos. But each year, thousands of children under the age of 15 will be diagnosed with cancer and their families will struggle with the impact the disease creates on their income and home life. The American Cancer Society estimated 10,590 children in the United States, alone, were diagnosed in 2018, and with this diagnosis comes a hefty price tag for the treatment. In fact, the price tag’s name and the aftermath it causes has quickly become a part of our lexicon and is known as financial toxicity.
When I was head coach of the football team at Boston College in 1991, a young player by the name of Jay McGillis was diagnosed with Leukemia. I visited Jay in the hospital on a regular basis and witnessed, firsthand, the impact this disease had on the entire family. Everyone rushed to his bedside. Family members stopped working to care for him. But the expenses continued to rise. It was soul-crushing to watch the family struggle. The football team wanted to do something to support the McGillis family so they organized a lift-a-thon and raised $50,000. It was awe inspiring how the community came together for Jay and his family. His battle brought out the best in humanity, but in the end, we lost Jay on July 3, 1992.
These statistics weren’t necessarily quantified 24-years-ago when I started the Tom Coughlin Jay Fund Foundation, but the need was with every family we met and helped.
Since its inception, my foundation has provided over $10 million in financial assistance to over 5,000 families. We help them keep a roof over their heads by paying the mortgage or rent. We’ve kept food on the table, the utilities going, and so much more for families facing the unthinkable, and we do it in the name and spirit of Jay McGillis. Our mission is to BE THERE for families so they can BE THERE for their sick child.
So, what can families do and where can they turn to when they reluctantly become a passenger on the roller coaster ride known as cancer? I always tell parents when people offer to help, take it. No one fights this disease alone. It takes a team of doctors, nurses, caregivers, family members, friends, and organizations who want to BE THERE for you during your child’s cancer journey. Talk to the social worker at the hospital; he or she is there to help.
When the Jay Fund met the Cabrera family, Magen was an eighth-grader just starting to learn about childhood cancers in science class. Her family was overwhelmed when doctors told them the then 13-year-old had acute-lymphoblastic leukemia (ALL). ALL requires between two and three years of treatment, followed by radiation. Magen’s mom and dad, along with her grandmother, either couldn’t go to work or work a full week when Magen was home from treatments.
Changes to their income and mounting medical bills became a side effect of this already devastating disease. A social worker introduced my team to Magen and her parents, and they quickly became part of our Jay Fund family. We helped them financially, as well as providing financial coaching to get their finances healthy and in shape for when Magen would be well and thinking about college. They came to the Giants training facility for our annual Sundae Blitz, which is really what I like to think of as a day off from cancer for the entire family. Magen and her family met the players and ran drills with them, toured the locker room, played games, and took a few moments to breathe and enjoy each other.
Today, Magen is a senior, getting ready to go to prom, graduate, and, yes, go to college in the fall. And the Jay Fund will BE THERE to support and celebrate this next phase of life with her and her family, as will the many other individuals and entities who were part of her team.
Jay McGillis’s story is not unique, but it is because of him so many patients and families going through the same thing have organizations like the Jay Fund on their team. Find your team and let them BE THERE for you.
A blog post by Children’s Cause CEO George Dahlman, father of a childhood cancer survivor:
Last month childhood cancer advocates were encouraged – and somewhat surprised – 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. Not long afterwards, officials from the Administration and the National Cancer Institute held a conference call to advise that they were planning a ‘big data’ project to identify and catalog biomarkers for every child diagnosed. Similar projects are underway by other organizations, and the NCI is now planning a consortium or ‘federation’ to coordinate all these data projects.
Certainly, a project of this type would dovetail nicely with a provision of the STAR Act supporting biorepositories and complement the $30 million that STAR is slated to receive each year.
But the promise of that extra funding and any new initiatives was thrown into doubt this week 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.
You may ask: Couldn’t childhood cancer programs still get an increase and be held harmless from any cuts? The answer: Not very likely.
First, Congress has the final say here and normally appropriates NCI funding in a block, allowing the NCI Director to determine program priorities. In the face of withering cuts to a range of other programs – many of which also benefit childhood cancer research – the Director would simply not have the resources to take on any new projects.
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.
Furthermore, when NCI tries to account for spending by disease, the math gets fuzzy. Many of their grants are for basic research that could apply to multiple conditions. Even accounting for increases in STAR funding and any new initiative, a budget cut of 17% across the board would more than eliminate any gains in one specific area.
The news in the Administration’s budget isn’t all bad. The Food and Drug Administration (FDA), which reviews and can accelerate treatments, would see an increase of some $260 million over the current level.
It will be critical as childhood cancer advocates, patients, families and supporters, that we appeal to our representatives — and you can do that right here with the action alert below.
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.
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 below we share a few key findings related to survivorship, financial hardship, and coverage challenges.
In our 2018 survey, we asked survivors to think back on their cancer treatment: “Were you involved as much as you wanted to be in decisions about your care and treatment?” While the majority responded favorably, one-in-five survivors answered No, and I would have liked to be more involved. By comparison, just 7 percent of parents/caregivers answered no.
When asked “In the last 12 months, how often did your general practitioner seem informed and up-to-date about the care your received from cancer specialists?” less than 40% responded that their doctor was usually, always or almost always well-informed about the survivor’s cancer care. 34 percent responded sometimes, and 28 percent responded almost never.
Alarmingly, six-in-ten survivors 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.
Too often, these hardships are going unaddressed. Over half - 55 percent - of families surveyed responded no when asked “at any point in your child’s care, were you asked about any financial hardships your family may be experiencing related to food, energy or housing?”
Families report a very wide range of out-of-pocket expenses from the past 12 months related to their child’s healthcare costs (not including monthly health insurance premiums). Several families report owing more than $100,000 in outstanding debt directly related to their child’s cancer treatment.
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:
INTERRUPTING OR CHANGING THE COURSE OF A CHILD'S TREATMENT: “One drug was never approved under compassionate use despite my daughter's desperate and urgent need for it to save her life. She lost her life at the age of 7 within a month after this drug was denied.”
NAVIGATION TIME & STRESS: "Our treatment hospital is in network for treatment but out of network for diagnostics requiring lots of back and forth and pre-approvals. I have lost a fair amount of sleep in the process and worried about the potential for big bills."
HIGH OUT-OF-POCKET EXPENSES: “The insurance only covered 7 prescription pills for nausea in a month but my child needed 2 pills every day. It was a new medication with no generic option, and a large expense.”
At the Children’s Cause, we’re using this data to support a variety of policy proposals and projects in the works. We thank all the families and survivors who are helping to inform our advocacy work by sharing their experiences and challenges. Your input makes us a stronger voice for our children in Washington and around the country.