Children’s Cause Welcomes ‘Moonshot’ Pursuit of Cancer Cure, Urges Prioritization of Children

Moonshot_SOTU

Photo Credit: White House

President Obama announced a “new national effort” to cure cancer, to be led by Vice President Joe Biden, in the President’s last State of the Union address.  We thank President Obama and Vice President Biden for their leadership and their commitment to accelerating progress in the fight against cancer.

“Last year, Vice President Biden said that with a new moonshot, America can cure cancer. Last month, he worked with this Congress to give scientists at the National Institutes of Health the strongest resources they’ve had in over a decade,” President Obama said. “For the loved ones we’ve all lost, for the family we can still save, let’s make America the country that cures cancer once and for all.”

The President’s call to action is timely, given the rapid advances in cancer medicine. We urge the Administration to put children with cancer at the forefront of this movement. As a parent who has lost a son to cancer, we know Vice President Biden has a very personal understanding of the acute need for new therapies to treat cancers that cannot be cured with standard chemotherapies.

Vice President Biden’s own statement about the Moonshot initiative highlighted the need to translate science into cures: “Innovations in data and technology offer the promise to speed research advances and improve care delivery. But the science, data, and research results are trapped in silos, preventing faster progress and greater reach to patients. It’s not just about developing game-changing treatments – it’s about delivering them to those who need them.”

Vice President Biden is kicking off his ‘moonshot’ by meeting with leading physicians, researchers and policymakers this month. As this initiative progresses, the Children’s Cause will seek every opportunity to make the unique needs and perspectives of children with cancer heard in this important national dialogue.

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A Good Week for NIH

HACCongressional appropriators reached a tentative agreement this week on a $1.1 trillion omnibus spending bill, H.R. 2029, to fund the government through the end of the fiscal year (9/30/16) and avoid a government shutdown.

We are pleased to note that the bipartisan compromise bill would increase funding of the NIH to $32 billion, a historic high. That would amount to a $2 billion increase over current levels, a much-needed boost after a dozen years of flat funding and 2013’s sequestration cuts.

A vote on the spending bill is expected Friday, just before Congress adjourns for their holiday break.

20151216-strategic-planAlso this week: The NIH released a 5-year Strategic Plan: Turning Discovery Into Health. The plan focuses on core objectives that will drive its priorities and lays out 14 “bold predictions” for 2020, including “enhanced survival” for cancer patients as a result of advances in precision medicine.

“We are faced with extraordinary opportunities that demand exceptional attention if their promise is to be fully realized. Implementing these objectives will enable the more than 300,000 researchers supported by NIH across the nation and around the world to drive science further and deliver cures faster.”

– NIH Principal Deputy Director Lawrence A. Tabak, D.D.S., Ph.D.

This strategic plan was requested by Congress last year and is the first such agency-wide plan in more than two decades. Learn more: NIH Press Release

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Recent Drug Price Hikes Pose Threat to Kids with Cancer

DRUG PRICE HIKES (3)Children’s Cause has been carefully following troubling reports of excessive price hikes for prescription drugs, including medicines used to treat children with cancer and other highly vulnerable populations.

This week, the Senate Special Committee on Aging held a bipartisan hearing to investigate these hikes in off-patent drugs. Health care experts testified about the harm done by these price spikes, and Committee leaders strongly criticized four pharmaceutical companies for exploiting a decrease in market competition upon their acquisition of certain off-patent drugs.

The Committee highlighted the particularly egregious example of the drug Daraprim, manufactured by Turing Pharmaceuticals. The cost of Daraprim, used to treat toxoplasmosis, recently increased by 5,000 percent. For people with weakened immune systems – a population that includes children receiving cancer treatment – toxoplasmosis can be fatal.

Dr. David Kimberlin, a pediatric infectious disease physician at the University of Alabama, testified about his personal experience scrambling to find affordable treatments for infants suffering from toxoplasmosis. A course of treatment that used to cost $1,200 now costs $69,000, he told the committee.

“Babies’ lives literally hang in the balance here,” Dr. Kimberlin testified.

Successful treatment for children with cancer is threatened when essential drugs are hard to access because of exorbitant cost or short supply. Evidence indicates that alternative drugs substituted in original treatment regimens are not as effective as those in standard treatments.

More Congressional hearings around this topic are expected in 2016, and we will continue to monitor this dialogue and engage on behalf of children with cancer.

Read on for more coverage on this week’s Senate hearing:

 

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All aboard for #GivingTuesday!

GivingTuesday_Twitter_ModernSociety

Today is #GivingTuesday, and it’s also the launch of our “Full Speed Ahead” year-end campaign. This December, we’re asking you to hop aboard with us to turn 2015’s progress into 2016’s reality of improving the lives of children with cancer. With your donation on this national day of giving, you fuel our mission and our work heading into a critical year.

We’ve got momentum on our side: Sweeping childhood cancer legislation is gaining new Congressional cosponsors every week. Precision medicine is becoming a household term. More children are surviving cancer than ever before.

But we can’t slow down now. The stakes are simply too high. We’re blazing full speed ahead toward a brighter future, and we need you on this train with us

Maximize your impact this holiday season:

  • Help us earn matching donations through GoodWorld’s Million Dollar Challenge! Become a Social Giving Superhero this December by commenting #donate $20 (or any other amount!) on our Facebook page or tweeting out:  #donate $20 @childrenscause. Once you post those magic words on social media, Good World will send you a link to a 60-second form to securely process your donation – and their matching gift!
  • giving_tower_share_fbHelp build this year’s CrowdRise Giving Tower! If you make a donation to us here, a brick will be added to the interactive, global virtual giving tower. There’s an app available on iTunes and Google Play so you can follow along and watch the tower grow in real time — an especially great way to get kids excited about this giving season!
  • Still have items sitting in your Amazon online shopping cart from yesterday? If you do your holiday shopping through AmazonSmile and select Children’s Cause for Cancer Advocacy as your charity of choice, Amazon will donate a portion to us – at no extra cost to you!

Giving-Tues-Logo.jpgYour gift matters now more than ever, and there’s no better day than today – #GivingTuesday – to make your year-end donation. Your support enables us to be aggressive advocates for new pediatric cancer therapies that are more effective and less harmful.

Thank you for helping us usher in a new era of progress and hope for our children.

GIVE TODAY.

 

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Thank you, New York!

2015 Honoree Renzo Canetta, MD, with CCCA President Susan Weiner, PhD

2015 Honoree Renzo Canetta, MD, with CCCA President Susan Weiner, PhD

We’d like to take a moment to acknowledge and thank everyone who supported our recent cocktail reception and silent auction in New York City. It was a beautiful evening in a beautiful space, and we are so pleased to report that we have surpassed our fundraising goals!

Friends and supporters who joined us at the Payne Whitney Mansion in late October heard updates on Children’s Cause activities and recent policy achievements. Attendees also heard from Renzo Canetta, MD, our 2015 Honoree. Dr. Canetta is recently retired from Bristol Myers-Squibb, where he served as Vice President of Global Research & Development Oncology Policy.

We’d like to give special thanks to Bristol-Myers Squibb for their event sponsorship and to the many dedicated vendors and auction items donors for their generosity. We especially want to recognize Susan and Stephen Scherr, event co-chairs, and all the event committee members for making this terrific event possible year after year.

Finally, we send our thanks to Sarah Merians Photography & Video Company for capturing the evening so beautifully. Click a photo below to enlarge it.

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A STAR Act Milestone: 100 HOUSE CO-SPONSORS!

100_cosponsorsWe reached a big milestone this week:  There are now 100 House co-sponsors signed on to support the Childhood Cancer Survivorship, Treatment, Access and Research Act of 2015 (“STAR Act”).

THANK YOU to everyone who has helped us get this far. Every new co-sponsorship takes this important legislation one step closer to becoming law. Please take a moment to check the current House list and Senate list to see if your Congressmen are signed on. If they aren’t yet supporters, now’s the time to send a message and get them on board!

Please note: If you would prefer to email your Member’s office directly instead of using our action alert form, here’s a sample letter for your convenience.

The Children’s Cause, along with other advocacy leaders in the childhood cancer community, is working hard to secure additional support for this legislation. We’re reaching out to advocates in targeted districts to back up our Hill meetings and our communications with staff in those offices. If you get a personal email from us asking for your help, please know we really need you!

We can’t do this alone. We need your voice – and the voice of your friends, family, and neighbors – to continue to build on this momentum at this critical point. 100 Representatives and 12 Senators won’t be enough to advance the bill. We particularly need the support of more Republicans, so if you’re from a Republican-led district, please help us spread the word locally to get more emails, phone calls and tweets to your Member’s office!

Thank you for joining us in this important movement to help children with cancer live longer, healthier lives.

TakeAction_House    TakeAction_Senate

P.S. — This is also our 100th blog post! Thanks for following along. Let us know in the comments if you have suggestions for upcoming topics you’d like to see us cover.

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New Developments for Precision Medicine Initiative

In an exciting update, NIH Director Francis Collins announced last week that the Precision Medicine Initiative (PMI) is now entering the implementation phase, with a clear and visionary first goal:  creating a one-million-member strong “research cohort” within 3-4 years. Voluntary enrollment is slated to begin in 2016.

Slide from an NCI Stakeholders Briefing on the PMI Report, 9.25.15

Slide from an NCI Stakeholders Briefing on the PMI Report, 9.25.15

The just-released PMI Working Group report (PDF) describes detailed design plans, which includes recommendations on the cohort’s formation, engagement of participants, collection of data and specimens, and more. The Working Group recommends that the cohort be made up of volunteer participants from all life stages, including this special note about children:

The Working Group gave special consideration to including children [and select others]. There are scientific, ethical and policies issues surrounding these populations that warrant further discussion. Therefore, the Working Group recommends that NIH consider the safeguards necessary to ensure the appropriate enrollment, retention, and protection of these groups into the PMI cohort.

The million+ volunteers in the cohort will agree to share their electronic health records, take surveys on lifestyle habits and environmental exposures, undergo a baseline exam, and provide a blood sample. In return, participants will have access to the study’s findings (both individual and aggregate) and will be provided with “tools to make sense of the results.” [Press Release, 9.17.15] Dr. Collins has repeatedly emphasized that although genomics will play a large role, it is not solely a genomics project: “This is a project on health. This is trying to understand all of the factors that play out in health or disease.”

Slide from an NCI Stakeholders Briefing on the PMI Report, 9.25.15

Slide from an NCI Stakeholders Briefing on the PMI Report, 9.25.15

Precision Medicine & Children with Cancer:

Precision medicine – the potential to deliver the right treatment at the right time, every time – aligns with our mission of achieving less toxic and more effective therapies for children with cancer. We’re seeing early promise from analyzing the genomic profile of children with cancer and tailoring treatment to match the molecular make-up of their tumors.

JAMAIn a study published earlier this month in JAMA, researchers analyzed the genetic material of 91 children and young adults with refractory, relapsed, and rare cancers and discovered “potentially actionable findings” in 23 cases (25%). In some cases, the discovery of a genetic mutation led doctors to change the course of therapy. Two young patients even had their diagnoses altered after the discovery of previously unknown chromosomal abnormalities.

These findings “strongly suggest that precision medicine enhanced by genetic evaluation may improve the outcomes of children with cancer,” according to the authors. The study “represents an important contribution to the care of children with cancer. It makes clear that approaches that are rapidly evolving in adults are applicable to the care of children with cancer.”

Challenges for a Unique Population:

“We’ve known now for several years that the mutations in pediatric cancers are much more rare than they are in adult tumors… Neuroblastoma has the lowest mutation rate. That means it’s going to be the hardest to find mutations that we can have actionable drugs for. But we need to look, and we need to look a lot harder than we have so far.” – Lee Helman, MD

The keynote speaker at the 6th Annual Childhood Cancer Summit on September 18th was Dr. Lee Helman, the Acting Director of NCI’s Center for Cancer Research. Dr. Helman discussed precision medicine and the Pediatric MATCH Trial, slated to launch next year. He also outlined the following personalized medicine challenges that are unique to pediatric cancers, as seen in the slide below.

LeeHelman_Summit_PMI Challenges

The Children’s Cause will continue to engage with policymakers and other stakeholders to seek solutions to these challenges and ensure that children with cancer continue to benefit from advances in precision medicine during this exciting time of scientific discovery.

More on PMI: The Precision Medicine Initiative was first announced by President Obama in January at his State of the Union address. The president’s FY2016 budget includes $215 million for PMI, with $130 million of that total allocated to NIH to build the research cohort. Learn more here.

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Live from Washington: #StepUp with Us Today!

stepup-9We’re on Capitol Hill today for the 6th Annual Childhood Cancer Summit, followed by the Alliance for Childhood Cancer’s Luncheon & Art Reception, and we are looking forward to an inspiring and impactful day of events.

We’ll be pressing Members of Congress for their support of the Childhood Cancer Survivorship, Treatment, Access and Research Act of 2015, and we hope you’ll join us from home.

Please consider taking a moment to add your voice to the community’s joint efforts by asking Congress to #StepUp and Co-Sponsor the Childhood Cancer STAR Act.

Take Action Button

If your Senators and Representative haven’t heard from you yet this September, TODAY is the day to reach out. Write a letter, send a tweet, or pick up the phone. There is power in numbers!

We thank the Congressional Childhood Cancer Caucus for hosting this morning’s Summit on Progress and Opportunities in the Fight against Pediatric Cancer. (LiveStream available here). We’re looking forward to hearing from some great speakers, including keynote speaker Dr. Lee Helman, Acting Director of NCI’s Center for Cancer Researcher. And we’re pleased to have the opportunity to honor Congressional champions and educate additional Members and staff about the STAR Act at the post-Summit luncheon. Children’s Cause is proud to be a Gold Ribbon sponsor of the luncheon, and we applaud our colleagues in the Alliance for working together to make it a successful event.

Follow along with us on Twitter for live updates on what promises to be an exciting day in Washington!

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#RallyMedRes : Ask Congress to Increase Funding for NIH

Rally_Hill_Post_2015Today is the Rally for Medical Research Hill Day, and we’re proud to be one of over 300 partner organizations supporting this important Day of Action. Advocates on Capitol Hill are asking Congress to make funding for the National Institutes of Health (NIH) a national priority and thanking Members of Congress for their willingness to explore innovative ways to increase the NIH budget.

Thanks to NIH-funded research, children with cancer are living longer, healthier lives. But there is much more to be done, as childhood cancer remains the #1 disease killer of children in the United States and many childhood cancers continue to be terminal upon diagnosis or relapse.

We cannot allow flat funding or additional budget cuts to undermine progress in medical research at a time when we are on the cusp of exciting new advances. If Congress fails to make NIH funding a national priority, thousands of researchers across the country will be at serious risk of losing research grant funding, and with it the ability to carry out lifesaving research.

We hope you’ll join the #RallyMedRes effort today, in the middle of Childhood Cancer Awareness Month, by asking Congress to increase funding for the NIH with budget increases that are robust, sustainable and predictable in Fiscal Year (FY) 2016 and beyond.

Take Action Button

RallyMedRes2015

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Childhood Cancer Awareness Month is HERE!

september - VOICE

Ava_September_croppedIt’s here! Follow along with us all month on Facebook and Twitter as we honor and celebrate our brave survivors, recognize childhood cancer champions in Congress, and light our little corner of the world gold for National Childhood Cancer Awareness Month.

And join us as we remember with eternally heavy hearts every child that cancer took from us far too soon.

As childhood cancer advocates, there are a few things in particular we want to highlight as we look at the month ahead — plus, there are some great ways you can help strengthen our impact over the next 30 days:

  • stepup-6The #StepUp campaign runs all month long and we need YOUR help to make it a success. All interested organizations and individuals are invited to take part in this coordinated community effort to make childhood cancer a national priority. Learn how to #StepUp with us and make our voice stronger. Get started with something easy: Put a #StepUp Twibbon on your social media profile photo!

    Want to do more? Ask your Congressmen to #StepUp & co-sponsor the Childhood Cancer STAR Act. Check to see if your Senators and Representative are already co-sponsors. If so, tell them thank you. If not, send them a tweet or a write a personal message.

  • The 6th Annual Childhood Cancer Summit, hosted by the Congressional Childhood Cancer Caucus, is taking place September 18th. Immediately following the Summit, the Alliance for Childhood Cancer is hosting a Luncheon Reception to honor Congressional champions of the Childhood Cancer STAR Act and to educate other Members and staff on the bill. We are looking forward to attending the Summit & Luncheon to press Members for their support of this important legislation, which contains provisions CCCA has been championing for years.

    Is your child an artist? The Alliance is seeking submissions of artwork from children with cancer or childhood cancer survivors to feature at the Reception. Find out more details and submission guidelines.

  • CUREFEST logoCureFest is a family-friendly Walk and Festival on the National Mall taking place Sunday, September 20th. Festival entertainment will include inspiring dance, poetry, and musical performances, including an appearance by The Plain White T’s. The full schedule of events includes educational speakers and a butterfly release tribute. Over 75 childhood cancer foundations, including the Children’s Cause, will have exhibit tables at the event.
  • #GoGold: We’re excited to see gold t-shirts, gold pins, and gold landmarks bringing childhood cancer to the attention of the general public. Boston’s TD Garden and Fenway Park will #GoGold today (9/1) — don’t miss it! Times Square will #GoGold on September 17th, and “A Night of Golden Lights” on September 19th is a candlelight vigil in front of the White House. If you’re going gold in some way this month, be sure to use the #GoGold hashtag on social media so we can all follow along.

P.S. – Did you know there’s an easy way to see if there’s an event happening near you this month? Check out the Coalition Against Childhood Cancer’s Event Calendar. If you’re hosting an event in your area, add it to this community calendar to spread the word!

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