May Bulletin: Action Day brings 200 advocates to DC

RSVP Now: Capitol Hill Briefing on June 23

Children's Cause will host a Congressional briefing with childhood cancer survivors and experts on June 23rd from 12:00-1:30PM.

The afternoon briefing will illustrate and address the access and coverage issues faced by childhood cancer survivors, including recent research findings and best practice recommendations for managing long-term care.

Speakers will include Dr. Smita Bhatia and Dr. Julie A. Wolfson of the University of Alabama, Dr. Anne Reilly of CHOP, and Dr. Monica Gramatges of Texas Children's Hospital.

Learn more here or RSVP to jkean@childrenscause.org.

Childhood Cancer Action Day Propels STAR Act to a New Milestone

The 5th Annual Childhood Cancer Action Days, hosted by the Alliance for Childhood Cancer, took place this month, bringing our community together in Washington to advocate for the Childhood Cancer STAR Act and increased NIH funding.

Approximately 200 advocates attended this year's event, spending a full day in advocacy training followed by a day of pre-arranged visits with Congressional offices. Around the country, advocates joined in from home by asking Congress to #StepUp for the #STARAct.

Your Action Day efforts helped us reach a big milestone: more than half of the House of Representatives now supports this life-saving legislation!

One advocate in attendance, Stacie Ritter, shared her take on the experience with us. Read our blog for Stacie's perspective on this year's Childhood Cancer Action Day.

Children's Cause Endorses ACE Kids Act

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The Children's Cause for Cancer Advocacy is proud to be supporting the Advancing Care for Exceptional (ACE) Kids Act, which would improve care for approximately two million children with medical complexities in the Medicaid program. 

Children with cancer, like children with other medical complexities, often see multiple specialists and a variety of physicians.  There are over 30,000 children with cancer covered by the Medicaid system and the specialized care they require often takes them across state lines. Under the current Medicaid system, parents of children with cancer and other multiple, life threatening disabilities struggle to coordinate the complex, multi-state care of their kids. Only federal legislation can fix the fragmented system for children with medical complexity.

We are honored to join the nation’s leading children’s hospitals, along the Children’s Hospital Association, to support this legislation, which is voluntary for states, families, children’s hospitals and other providers. 

Learn more about this bill:

Quick Links: Recommended Reading

  • Keeping Cures on the Shelf: Our own George Dahlman, CEO of Children's Cause, wrote a perspective piece on drug shortages in this month's Journal of Clinical Pathways.

"Ultimately, the mitigation of drug shortages will be the result of vigilance on the part of all stakeholders. Maintaining that awareness and managing a quick response system requires that doctors and families become involved in the policy process that makes it work and the rapid reporting of individual instances." (Journal of Clinical Pathways / May 2016)

  • Rare Tumors May Cause ADHD-like Symptoms: A new study suggests that some kids diagnosed with a hyperactivity disorder who also have high blood pressure may instead have a rare tumor - pheochromocytoma or paraganglioma - causing ADHD-like symptoms. (NIH, May 16, 2016)
  • Poliovirus Therapy May Treat Glioblastoma: The FDA granted 'breakthrough' designation to a treatment modified from poliovirus that shows promise for treating advanced glioblastomas. The therapy is currently being investigated in adults but researchers at Duke hope to expand their trials to pediatric brain tumor patients by the end of this year. (OncLive, May 16, 2016)

Childhood Cancer in the Spotlight:

Children's Cause Achieves GuideStar Platinum Status

Children's Cause is proud to be one of the first organizations to receive the Platinum Seal of Transparency from GuideStar, the world's largest source of nonprofit information. GuideStar recently launched the Platinum participation level to help nonprofits celebrate their results with new metrics to report on our progress.

We're proud to use GuideStar Platinum to share our full and complete story with the world. We achieved Platinum by completing GuideStar's preceding participation levels of Bronze, Silver, and Gold. Each provided stakeholders with increasingly detailed organizational information ranging from basic contact information to programmatic details.

Check out our updated #GuideStarPlatinum profile here: 

http://www.guidestar.org/profile/11-3485631

Jessica Kean
April Bulletin: FDA hosts forum; CCCA announces Hill briefing

FDA Hosts Childhood Cancer Forum

An FDA-hosted Childhood Cancer Advocacy Forum last week provided opportunity for groups including the Children's Cause to engage with agency representatives around current drug development challenges and opportunities. The Forum included an update on oncologic drugs approved for pediatric use and clinical trial progress, as well as presentations on BPCA and PREA, compassionate use, pediatric Patient-Reported Outcomes, and more.
 
Among the highlights from the event:

  • On PREA: In a presentation about the effectiveness of the Pediatric Research Equity Act (PREA), the FDA's Dr. Margaret Thompson reported that 15 new oncology drugs were approved in 2015-2016. Of those, only one was approved for treatment in pediatrics: dinutuxinab for neuroblastoma. As Dr. Thompson noted: "This was actually unusual. Most years, all new drugs would be for adult indications." Of the 14 drugs approved for use in adults, none have had the PREA requirement applied. This has led various stakeholders, including Children'sCause, to question how PREA can be improved to make it more effective. Dr. Thompson discussed several possibilities for improving PREA, which include changing the pediatric study requirement from indication-based to mechanism-of-action based.
  • On Trial Eligibility: Dr. Christy Osgood discussed one way to increase pediatric access to adult clinical trials by expanding eligibility criteria to include adolescents in adult trials when the disease and/or target is the same. Opportunities might be also identified where alternative trial designs could allow a younger cohort in early phase trials for older patients. Dr. Osgood said logistical challenges can arise because adults and children are treated in different settings. But Dr. Osgood affirmed that the FDA is "supportive of models that include pediatric patients earlier in trial designs."
  • On Expanded Access: Another member of the FDA team, Dr. Ashley Ward, discussed expanded access or "compassionate use." Benefits to expanded access include the potential to offer hope to children who have exhausted all other options. Barriers mentioned included the additional resource burdens on the manufacturer (time, paperwork, and sometimes limited drug availability). The biggest barrier identified is the manufacturer's fear that an adverse event might disrupt or derail the drug's path to approval. Dr. Ward commented that this fear is largely unfounded based on FDA experience, and stakeholders need to work together to increase manufacterers' confidence in the expanded access process.

Children’s Cause President Susan Weiner co-chairs the Alliance for Childhood Cancer task force on PREA and BPCA and is working with the Alliance on a package of BPCA and PREA recommendations for this year’s reauthorization of the Prescription Drug User Fee Act (PDUFA).

Save the Date: Capitol Hill Briefing & Reception on June 23

Children's Cause will host a Congressional briefing with childhood cancer survivors and experts on June 23rd, followed by a Capitol Hill reception.
 
The afternoon briefing will illustrate and address the access and coverage issues faced by childhood cancer survivors, including recent research findings and best practice recommendations for managing long-term care. At the evening reception, Members of Congress, staff and the health care community will be invited to join us in honoring the leadership of the Childhood Cancer Caucus and learning about new Children's Cause initiatives.
 
More details will be coming soon, as we finalize the program and speakers.
 
If you are in the National Capitol area and have personal experience with survivorship access issues, we want to hear from you!
Please email us at jkean@childrenscause.org if you're interested in helping us make this event a success.

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There's Still Time to Register for Action Days!

It's not too late to register for the 5th Annual Childhood Cancer Action Days in Washington on May 16 & 17. This two-day Alliance for Childhood Cancer event brings our community together to advocate for important childhood cancer issues currently before Congress. Register here.
 
A strong showing of advocates at this year's Action Days is more crucial than ever, as the Childhood Cancer STAR Act is nearing the critical committee stage in the legislative process. There are now 200 House cosponsors of this bill! Sharing your story in Washington this spring will help us tip the scales toward securing passage of this historic, bipartisan legislation. 
 
Issues and Advocacy Training will take place Monday, May 16th at the Holiday Inn on Capitol Hill, followed by pre-arranged meetings with Members of Congress and their staff on Tuesday, May 17th.
 
Visit the Alliance for Childhood Cancer website for more information and to register.
 

Moonshot Update: Pediatric Oncologists Appointed to Blue Ribbon Panel; NCI Accepting New Ideas

The National Institutes of Health (NIH) announced a Blue Ribbon Panel this month to guide and inform Vice President Biden's National Cancer Moonshot Initiative. We were pleased to see some pediatric experts named to this distinguished panel, including Dr. Peter Adamson of Children's Hospital of Philadelphia and Dr. James Downing of St. Jude.
 
"This Blue Ribbon Panel will ensure that, as NIH allocates new resources through the Moonshot, decisions will be grounded in the best science," said Vice President Biden. The panel will serve as a working group of the National Cancer Advisory Board and will be focused on how to drive advances in several key areas targeted by the Moonshot. Childhood cancer has been specifically identified as one of those key areas of focus.
 
NCI is asking the health community for scientific ideas and suggestions to support the Moonshot. One of the categories the NCI is specifically seeking input around is pediatric cancer.
 
If you have a suggestion, visit the NCI's online platform Cancer Research Ideas before July 1. We'll be weighing in, and we hope you will, too.
 
Related reading: 

Guest Blog: Why DIPG Needs an Awareness Week

This month we featured a guest blog from Janet Demeter, founder and CEO of Jack's Angels Foundation. Janet recently secured the introduction of a House Resolution (H.Res.586) that would designate the fourth week in May as DIPG Awareness Week.
 
An excerpt: 
 
"H.Res.586 was introduced in January by Congressman Steve Knight (CA-25) and calls for elevated consideration for low-surival-rate cancers and years-of-life-lost in the research grant process with the NCI. [This resolution] is the first legislation to directly recognize DIPG but our goal is to raise awareness for all the hundreds of types and sub-types of pediatric cancer. By calling attention to one type of childhood cancer, we aim to elevate the profile of all childhood cancers."
 
Read on and then take action.

Remembering Leila Polintan, Former CCCA Staff

We are deeply saddened to announce the passing of Leila May Polintan, 39, a former Children's Cause staff member who battled breast cancer for the past several years. Leila managed programs and development for the Children's Cause before moving on to become Communications Director for Grantmakers in Health. Leila is remembered by those who love her as an involved community member, an avid reader, a devout Catholic, and beloved wife, daughter, sister and friend.
 
We remember Leila with affection as a delightful, charming and passionate young woman who played an integral role in the formative years of the Children's Cause. Our thoughts are with her family and loved ones during this difficult time.
 
Leila's obituary is available here.
 

Are you ready for mother's day?

Mother's Day is quickly approaching, and we want to take the opportunity to remind you of a few ways you can support Children's Cause when you shop online:

If you're shopping on Amazon, considering using AmazonSmile instead. You get the same item at the same price, but we get a donation from Amazon!

Shopping somewhere else online? Check out Goodshop first to see if your shopping merchant is one of the thousands of stores in their network. Goodshop's site will help you find great coupons -- and then donate a percentage to us!
 
>>> Sending flowers? Use Goodshop and they'll donate 7-8% of your 1-800-Flowers or ProFlowers total to us!

When you make a purchase through our Bravelets page, Children's Cause receives a $10 donation. Products including jewelry, watches and scarves are available in yellow or gold (the colors of childhood cancer awareness) and enscribed with the words "be brave." Shop Bravelets.

As always, you can make a donation directly to the Children's Cause in honor or memory of a loved one. Let us know it's a Mother's Day gift in the comment field of your donation, and we'll be sure to let the honored woman know. Thank you!
 

Jessica Kean
March Bulletin: Mobilizing Childhood Cancer Advocates

KIDS ACTION NETWORK: NOW ACCEPTING MEMBERS!

We are excited to have a new grassroots advocacy program in development called the Kids Action Network (KAN). As we prepare to launch this new program, we invite you to become a founding member.  Members of KAN will receive tools and training to become engaged and effective advocates in Washington, in local communities, and online.

To promote and recruit this new program, we've spent some time this month talking about the power of advocates working together to find safe, effective, affordable treatments and cures for our kids:

CURE Magazine profiled our work through the lens of one family advocating on behalf of their daughter, a three-year-old survivor of a rare and typically fatal brain tumor: Children's Cause Gives Families a Voice in Pediatric Cancer Policy Debates.

George Dahlman, Children's Cause CEO, appeared on Childhood Cancer Talk Radio this month to discuss current policy issues in Washington and the power of collaboration in the childhood cancer community. A short clip is at right, and the full hour-long podcast is available here.

We have exciting plans in store for the KAN program and we are grateful for these opportunities to invite more passionate people to join our growing ranks of childhood cancer champions. As this program fully takes shape over the coming weeks and months, we look forward to keeping you posted on its first activities and achievements! 

Become an Advocate: Sign up now to become a founding member of the Kids Action Network!

CHILDHOOD CANCER STAR ACT: TURNING THE MAP GOLD

The Childhood Cancer Survivorship, Treatment, Access & Research (STAR) Act continues to gain bipartisan support in Congress with nearly 200 House and Senate co-sponsors, but we need even more in order to advance this bill to the Committee phase of the legislative process.

We've made it easier for you to engage around this bill with an interactive map and write-to-Congress tools all on the same page. If you see a red telephone icon in your district, then your Congressman sits on the critical Energy and Commerce Committee and needs to hear from you! We were glad to help bring this new tool to life and we hope it helps all of you engage around this bill.

Related: The Huffington Post Parents Blog asked their readers to think about the terrifying reality of childhood cancer in a piece titled "Childhood Cancer - How You Can Help." The post linked to our action alert, bringing brand-new supporters to our cause and new voices to Congressional offices. Thank you, Huffington Post!

TAKE ACTION:  Check the map, then write your Rep!

NIH AND NCI FUNDING: ALLIANCE WEIGHS IN

Through the Alliance for Childhood Cancer, we submitted comments this month to Appropriations Committee leadership on Fiscal Year 2017 funding levels and priorities. In this letter, we call for $34.5 billion for the National Institutes of Health (NIH) and $5.9 billion for the National Cancer Institute (NCI) in FY2017.

We also call for childhood cancer to become a greater priority within Congress and the NIH. Toward that aim, we included specific requests for report language around the Pediatric-MATCH Trial, pediatric immunotherapy advances, and more.

Read the full letter here.

REGISTER NOW FOR CHILDHOOD CANCER ACTION DAYS

There's still time to register for the 5th Annual Childhood Cancer Action Days in Washington on May 16 & 17. This two-day Alliance for Childhood Cancer event brings our community together to advocate for important childhood cancer issues currently before Congress. Register here.

Issues and Advocacy Training will take place Monday, May 16th at the Holiday Inn on Capitol Hill, followed by pre-arranged meetings with Members of Congress and their staff on Tuesday, May 17th. A block of rooms has been secured at the training location with a group rate deadline of April 15th.

Visit the Alliance for Childhood Cancer website for more information on registration, scholarships (still available!), and hotel reservations.

RESEARCH ROUND-UP

  • FERTILITY: Male survivors of childhood cancer are three times more likely to suffer infertility as a late effect of their treatment compared to female survivors. The new research, which examined the impact of 14 commonly used chemotherapy agents, found that 50 percent of male survivors would later become fathers compared to 80 percent for their siblings. The study's authors reinforced that boys diagnosed post-puberty should bank their sperm. Female survivors studied were significantly less likely to suffer infertility problems, but problems with fertility accelerated after age 30. (The Lancet Oncology)

Related: Cincinnati Children's is offering a Live Q&A on Fertility Preservation for children with cancer on April 20. Learn more and register.

  • HEART HEALTH: A two-decade clinical trial has proven the effectiveness of the cardioprotective agent dexrazoxane when administered following doxorubicin therapy (associated with toxic cardiac effects) for children with certain blood cancers. The study followed 600 patients and found that children who received dexrazoxane during their treatment had normal hearts, while many of those who did not receive dexrazoxane had significant cardiac late effects. (Journal of Clinical Oncology)
  • MEDULLOBLASTOMA: Early research in mice has identified a new and less toxic combination therapy to potentially treat the most aggressive form of medulloblastoma (Group 3 tumors). Patients with Group 3 medulloblastoma have just a 40 percent survival rate. Researchers hope to quickly advance this novel drug combination to a clinical trial (Cancer Cell)

ADVANCING HOPE ACT PASSES OUT OF SENATE HELP COMMITTEE

The Advancing Hope Act, a provision to reauthorize a pediatric priority review voucher program, passed out of the Senate's Health, Education, Labor & Pensions Committee this month by a vote of 18-2. This legislation is part of the broader Innovation for Healthier Americans Act, which is the Senate response to the House's 21st Century Cures bill (H.R.6, passed in July 2015).

The Senate Committee voted to extend the Advancing Hope Act program by ten years, which would give the program a 2022 sunset, with a 2027 extension for drugs and biologics that receive a rare pediatric disease designation by 2022.

SURVIVOR SPOTLIGHT: MEET AIDEN

In recognition of March as National Kidney Month, we highlighted a young survivor of Wilms' tumor to help spread awareness that children get kidney cancer, too. This March also marked three years since Aiden, now 6, was diagnosed with the disease.

"Over the course of the last 3 years, I have met many child patients, or warriors, and their families. I count them among some of the bravest people I know. Many of their stories don't end happily. We need to do more. More for these kids, and their families," his mom writes. "I can tell you that there is not a day that goes by that I don't feel a flash of terror at the thought of his cancer returning, even though the statistics say it won't. I have seen too many beautiful children succumb to these disease and become the wrong statistc."

Thank you for reading and sharing.

Have a story of your own? We want to hear from you.

HELP US UPDATE OUR RESOURCES

As part of a recent website relaunch, we are conducting a full review and update of our online resources. We want to hear from you: What organizations, publications, links or other resources would you recommend to families navigating childhood cancer? 

We're looking for resources that span the spectrum of care, from diagnosis and clinical trials to palliative care, transitions of care, survivorship and more. Thank you for helping us provide information that is current, comprehensive, and useful to families!

Leave your suggestions here, or email us at jkean@childrenscause.org.

Jessica Kean