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