CAC2 Advocacy Conference Call Notes from April 8



The fourth CAC2 Advocacy call was held on Tuesday, April 8, 2014 at 12:00 Noon. Beth Anne Baber was the moderator with approximately thirty-one participants.


Beth Anne Baber, The Nicholas Conor Institute ( opened the meeting.


Nancy Goodman, Kids V Cancer, explained that by utilizing bi-monthly calls, the mission of the advocacy call is provide a venue to inform each other about efforts we are all taking in the areas of legislative or regulatory reforms at the state, national or local level. She suggested that instead of having a call in June that we have a live meeting during the CAC2 Annual Meeting in June.  After some discussion, it was decided to let everyone know via email if that would be possible. (


Upcoming Events and Meetings:


CAC2 Summit & Annual Meeting – June 24 & 25, 2014: 

Joy Cruse, Team Connor Childhood Cancer Foundation, stated the annual meeting would be held on June 25, 2014.  Check in will be at 8:00 AM at the Microsoft Building on 901 K Street, Washington, DC.  The meeting will end at 4:30. A short board meeting will be held after the annual meeting. A Survey Monkey was sent out to obtain information on desired speakers and topics. So far the top areas of interest are: NCI Barriers on Drug Development, Congressional Childhood Cancer Caucus, what is it, what does it do?, Childhood Cancer Advocacy Committee, what is it, what does it do?, 2014 ACS Childhood and Adolescent Cancer Statistics.  A reception will be held on the evening of June 24th. Options for hotels are: Henley Park Hotel on Massachusetts Ave, if you are planning on only attending the CAC2 meeting. If you are planning on attending the Childhood Action Days we recommend staying at the Residents Inn, Marriott in Old Town Alexandria. Detailed information will be provided on the CAC2 website ( and in the forthcoming save the date newsletters. (


Curefest – September 21, 2014

Mike Gillette, The Truth 365, stated that Curefest would be on the National Mall on September 21st. Sixty-nine people supported the idea on a survey sent out by CAC2.

Mike stated that the event does not belong to a single organization but to all of the participating organizations. Eighty-five foundations have already committed and he believes we could expect a total of 150 to 200 foundations participating. 300 volunteers have signed up. A website will be set up for everyone to use to register, volunteer and get information. Efforts are being made to attract people from outside the childhood cancer community. A walk will be one of the central events. Teams may raise money for themselves in conjunction with the walk and direct it to any childhood cancer foundation.  1,000 dancers have committed to come and there may be a flash mob dance during the Curefest. Mike stated that they are looking at getting a larger stage and have some bands, which would increase the number of people as well. Plans by ten local organizations are working on hosting a family oriented event on the night before Curefest at National Stadium, which will complete the weekend. (


Childhood Cancer Action Day – June 23 & 24, 2014 

Maureen Lilly, Alliance for Childhood Cancer and CCCA.  Maureen stated this is the fourth Action Days planned for the Alliance for Childhood Cancer, an alliance composed of 30 childhood cancer advocacy and clinical organizations, which decided to merge the various organization’s advocacy activities together under Childhood Cancer Action Days in Washington, DC.  Registration is capped at 150 advocates and details for registration are on the Alliance for Childhood Cancer website ( Please register today, as there are not many slots available. If you are registered and cannot attend, please notify the Alliance and they will allow someone else to attend from their standby list. (


Congressional Caucus on Childhood Cancer – September 2014

Due to meetings on the Hill, Andy Taylor, LD, Rep. Mike McCaul ( and Erica Appel, LA, Rep. Chris Van Hollen ( were not able to attend and asked Beth Anne Baber to relay the following information: The date for the caucus has not been determined as yet. They welcome any ideas or suggestions from the advocates for the meeting. A call may be planned with a group of advocates in the near future to brainstorm on ideas for a successful caucus. They wanted to inform advocates that both Congressmen summited a joint letter to the appropriations committee on Labor HHS requesting an increase in funding for NIH to support critical

Research to prevent and treat diseases including pediatric cancer.  The letter was signed be several members of the caucus. The caucus website is in the process of being updated.


Rally for Medical Research Hill Day – September 18, 2014 

Jon Retzlaff, Director of Science Policy, AACR (, and Mary Lee Watts, Director of Government Relations, ACCR ( held the discussion. Jon mentioned that one year ago today they held a Rally for Medical research with over 10,000 people attending. Also last September they held another rally in September. Again this year, on September 18th, they will hold another rally. They have reserved the Hyatt Regency on Capitol Hill for the kickoff meeting in the morning. Soapbox will be scheduling all of the meetings with congressional members. Last year there were over 225 organizations that participated in both events. Mary Lee mentioned there were over 200 offices visited by rally members in support of medical research and there were 80 member level meetings on Sept 18th last year. Jon invited any organization that would like to support the Rally to contact the AACR. This year and 2015 will see restrictions on funding NIH and he would urge everyone to help get funding increased.


Update: Current Legislative Issues and Initiatives:


Recent NCI Cuts to COG and the Pediatric Pre-Clinical Testing Program

Beth Anne Baber, TNCI ( There have been some deep cuts to NCI, COG, and the Pediatric Pre-Clinical Testing Program.  Beth told everyone to expect a conference call with NCI’s Office of Advocacy Relations. Information on the call was received shortly after our meeting and is included below:


From: NCI Office of Advocacy Relations <>

Date: April 8, 2014 2:19:08 PM PDT

Subject: April 22 Teleconference on Pediatric Cancer Research


Dear Colleagues:


Please join the NCI and the Director’s Consumer Liaison Group in continuing  the discussion on how to move forward pediatric cancer research.  Dr. Jeff Abrams, Director for Clinical Research, Division of Cancer Therapy and Diagnosis, and Associate Director, Cancer Therapy Evaluation Program, and Dr. Malcolm Smith, Associate Branch Chief for Pediatric Oncology, Cancer Therapy Evaluation Program, will provide updates and answer questions about the Children’s Oncology Group and NCI’s pediatric cancer portfolio.


Date/Time: April 22, 10am EST

NEW Number: 1 -888-989-4986

NEW Passcode5890051


National Cancer Institute, Office of Advocacy Relations



Caroline Pryce Walker Conquer Childhood Cancer Reauthorization Act (HR: 2607/ S: 1251)

Maureen Lilly, CCCA ( stated the bill was introduced last summer in the House and Senate with bipartisan support and currently has 45 congressional co-sponsors and 13 Senate co-sponsors. The purpose of the bill is to fund a registry and create a bio-repository of tissues, data and demographics in a centralized location to aid researchers. Last Friday the Alliance for Childhood Cancer held a very informative congressional briefing for Hill staff about the importance of registries and bio-repositories. Your help in getting more co-sponsors would be greatly appreciated.


Patient Choice Act (HR: 2090)

Jonathan Agin ( and Karen Jaffe ( Due to the recent news of Josh Hardy and his family’s efforts to obtain compassionate use of clinical trail drugs, the Patient Choice Act has been given some press.  Karen said with bipartisan support in the house, the bill allows for availability of investigational drugs that have pasted phase I and II (safety) trials to be given or sold to patients with terminal or life threatening illnesses with out having to go through the compassionate use program, but with advised consent. It provides a parallel provisional approval pathway almost identical to what has been passed by MHRA in Europe. The bill would allow the pharmaceutical companies to potentially gain a little revenue and for the pharma-economic system to work while the drugs get to market with a full NDA.   Jonathan Agin related his situation with his daughter who died of a brain tumor, and may have benefitted from a bill like patient choice if it had been available rather than having to navigate the compassionate use program. Karen stated that anyone who is interested in obtaining more information to please contact her.


The MODDERN Cures Act (HR: 3116)

Joe Baber, TNCI, Four Square Clobbers Cancer, ( Joe stated he met with Congressman Leonard Lance in February and discussed the MODDREN Cures Act (acronym for: Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network). While the bill was not written a childhood cancer bill, it provides many benefits for children with cancer. The bill provides incentives for manufacturers in the form of increased patent protection for those who produce drugs or treatments for illnesses that have unmet medical needs. The term “unmet medical needs” applies to any medical condition or disease where no cure has been found or the cure rate is very low.  Many, if not most, childhood cancers could qualify under this definition.  The bill would allow 15 years patent protection for a new or dormant drug from the time it is passed by the FDA. Under normal circumstances, many companies lose half their patent protection years before their drug get passed by the FDA.  While very few exist today, the bill would stimulate creation of valuable companion diagnostics allowing for better precision medicine. The bill will reform diagnostics reimbursement which hasn’t been done since the late 1980’s and it will incentivize companies to bring forward better, but more expensive, diagnostics that will actually save money by eliminating the need for patients to have treatments for which they will not benefit.  The bill has 40 bipartisan co-sponsors and COB estimates the cost of the bill to only be $20 million over 10 years. More information can be found at


Good News to Share: Passed Bills, Advocacy Events, etc.


Compassionate Use trial and waiver: Josh Hardy & Zachary Bernstein:

Richard Plotkin, Max Cure Foundation, Grandparents In Action, (

Richard stated that Zachary Bernstein, 11 years-old, was diagnosed with DIPG and Richard connected the family with Jonathan Agin and Dr. Oren Becker who recommended a combination of drugs, from Merck and from Novartis and within two days, after phone calls made on their behalf by Richard, got a first ever Compassionate Use Wavier for a combination of drugs for a pediatric patient from the FDA. Unfortunately, Zachary passed away on March 12th, the same day that Josh Hardy received his life saving drug.  In the case of Josh Hardy, Vickie Buenger was contacted on March 5th and contacted Richard and others who were told that unless seven year-old Josh would die from an infection the following weekend if he did not get a particular drug in clinical trails and manufactured by Chimerix. Richard worked with Bill Burns, Josh’s uncle, and within days, through their various contacts, they got Josh’s mother on CNN, Fox and Friends and other networks and started a social media campaign and got 1.1 million Facebook views and over 400,000 tweets. Richard made an appeal on Fox and Friends on Tuesday, March 11th; he personally spoke with the President of Chimerix several times. Before the end of the week, rather than issue a Compassionate Use Waiver, the FDA and Chimerix, the drug manufacturer, established a phase 3 clinical trials for 20 children to be given the drug, with the first patient being Josh Hardy. Today Josh is making good progress.


President’s Office of Engagement

Tony Stoddard, A Day of Yellow and Gold (, stated that three years ago, after his son passed away from neuroblastoma, he and his wife became disturbed in September after seeing how little gold was showing and how little the awareness was toward childhood cancer awareness month, they decided to change that. Tony reached out to others to help increase awareness. He reached out to elected officials at the local, state and federal levels.  He became close to his Congresswoman and Senator who wrote letters to President Obama on his behalf after efforts to light the White House gold. He was contacted by the White House to begin conversations on how the President through various people in his office can help improve the childhood cancer situation. The first call with the White House Staff and a group of Childhood Cancer Advocates with be today at 2:00 PM. It is hoped that through these calls that many advancements can be made


Creating Hope Act

Nancy Goodman, Kids V Cancer (, began by inviting all that were on the call who were not CAC2 members to consider joining CAC2 so that they would be able to participate and benefit from calls such as this and others in the future.  Nancy stated the Creating Hope Act is doing well. A large, broad group of stakeholders are very interested in bringing something similar to Europe. She has been working with them and at the present time, they are investigating what such an act would look like in Europe. Domestically, she thinks draft FDA guidance on the Creating Hope Act may come out this summer. The FDA is currently trying to determine the best definition of pediatrics and what qualifies where this bill is concerned.  Nancy hopes to have three vouchers completed this year.



Gabriella Miller Kids First Research Act

Ellyn Miller, Smashing Walnuts Foundation ( explained that her daughter, Gabriella who had been diagnosed in 2012 with DIPG, did an interview with The Truth 365 two weeks before she passed away. The interview where 10 year-old Gabriella urged the lawmakers to “stop talking and do something,” and “talk is Bull Shit, we need action,” was seen by Eric Cantor who asked for permission to rename HR:2019 to the Gabriella Miller Kids First Research Act which would redirect funds from presidential elections in to a special pediatric research fund and NIH. The fund would be used for all types of pediatric research including cancer. Within six months of Gabriella’s passing, President Obama signed the bill into law last week. The bill will move $126 million over 10 years to pediatric research.  What we need to do now, Ellyn said is to make sure that they actually appropriate the funds as intended. We need to work together to make sure they move the money into the NIH special fund as promised.



Cancer Prevention Research Institute of Texas (CPRIT)

Annette Leslie, Carson Leslie Foundation ( related how she lost her son in 2010, Carson, and became an advisory board member of CPRIT. CPRIT is making available $3.2 million to be used exclusively for pediatric brain tumors. While the research has to stay in Texas, the results can be used around the world. CPRIT has just announced last week more opportunities for funding general cancer research specifically in the pediatric and adolescent arenas. These are great victories in the fight against childhood cancer!



Rare Disease Week

Rob Whan, Caleb’s Crusade Against Childhood Cancer (

This was my second consecutive year at the Rare Disease Legislative Advocacy Days and the experience did not disappoint. RDLA has the same challenges that we do with rare populations, some of them being FDA reform, low or nonexistent funding and lack of drug development. The atmosphere at RDLA is very comfortable for the childhood cancer advocate. We are welcome, as part of the team and it seems that they recognize that our challenges are much in line with theirs. Statistically, the childhood cancer community fits within the scope of rare disease with the guideline being less than 200,000 diagnoses per year and includes 7000 other diseases. There is a lot that this group does well but there are a few in particular that the childhood cancer community can embrace and aspire to. The 2 things that I wish to highlight from the experience are education and access.


The schedule surrounding the RDLA week was a busy one. At some times choices were offered of topics at different venues to fit your interest. We attended as many as we could and the education that was offered through the panel discussions and presentations were unprecedented in my 6 years of advocacy experience. The opening night was a documentary on “progeria” that educated us about this disease that had no treatment. The documentary is a case study of how a single family rallied around this disease and pushed a drug through to market that is actually adding about 5 years to these kids life expectancy through drugs that improve their vascular strength.


After the documentary the panel discussion opened with introductions, panelists included Dr. Francis Collins, Director, NIH. I am proud to announce that the 5 attendees from the childhood cancer cause in the audience lead the Q & A session with Dr. Collins and no one was holding anything back but we were all respectful at the same time.  I actually had a short conversation with him after the event and he was very receptive to our challenges and assured me that our advocacy efforts are necessary to get the changes we need for our patient population, for our kids.


In conclusion, I can attest that the first year I was a bit star-struck. This year I knew what to expect and was ready to engage. The experience that was led by RDLA is one that all serious advocates should attend to shape their advocacy tools and (experience) how another well organized cause does things. I encourage everyone to get involved with RDLA and they will open doors for our community.


Maureen Lilly mentioned that CAC2 is planning a Webinar about the third week in May that will be of interest to all on the call today and others that are interested in advocacy.

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