Pediatric Cancer Awareness in Washington D.C.

Pediatric Cancer Awareness in Washington D.C.

Last week was an eye opener for me as I travelled to Washington, DC, to attend several events in honor of Pediatric Cancer Awareness Month.  I say this because when I first got involved with the cause in 2007 after Max was diagnosed, there was relatively little attention given to childhood cancer – except of course if you happened to be among those with a child afflicted with the disease, a disease that strikes 15,780 children each year in this country and accounts each year for more deaths by disease in children than any other illness.

Pediatric cancer is not a single disease but rather, there are more than 16 major types of cancers in children, with more than 100 subtypes, each requiring different treatment options, one type of leukemia having a survival rate as high as 90%, with many having survival rates less than 50% and indeed for some, the disease is terminal with all children afflicted dying within the five year period following diagnosis (a child is considered a survivor if he/she is alive 5 years after diagnosis).  The overall 5-year survival rate, on average, is 83%.  But that does not tell the whole story.  There are 380,000 childhood cancer survivors in the United States (who survived the 5-year period) and that, in actuality, for those who survive, it is not the end of the journey, but rather the beginning of yet another potentially tortured road.

Thanks in part to the efforts of the bipartisan Congressional Caucus on Pediatric Cancer formed by Congressman Michael McCaul (R., Texas) in 2009, currently co-chaired by Congressmen McCaul and Chris Van Hollen (D., Md), childhood cancers have begun to be recognized more than at any time in the past.

The Caucus appears to have been the stimulus, with the aid of many individual childhood cancer foundations plus collaborative organizations such as the Coalition Against Childhood Cancer (CAC2) and the Alliance for Childhood Cancer (Alliance), that has caught the attention of the decision makers in Government who recognize that more attention must be given to the cause.  This is evident to me in that over the three days I was in DC last week I heard speakers from the National Institute of Health (Director Dr. Francis Collins), from the National Cancer Institute (Director Dr. Harold Varmus) and from the White House (Paulette Aniskoff, Deputy Assistant to the President and Director, White House Office of Public Engagement, moderated the 3-hour invitational meeting attended by high level folks from different branches of the White House and from the Department of Defense).

On Thursday, September 18th, I attended the Hyundai Hope On Wheels Thought Leaders’ Summit and Luncheon which was followed that evening by the Golden Toast at a local facility hosted by The Carson Leslie Foundation, in honor of Congressmen McCaul and Van Hollen.  It was very well attended by childhood cancer advocates from around the country.  This was followed by a private showing of the animated film, Henry & Me, which tells the story of a young boy with cancer who, with the help of Yankee Magic, battles the disease with courage and bravery, demonstrating how important it is for children who face obstacles to have a positive mental attitude.

The first event I attended, by invitation only, was the Hyundai Hope On Wheels Summit.  Three leading oncologists from St. Jude Children’s Hospital, from Children’s Hospital of Philadelphia and from the National Cancer Institute presented to the group, discussing the current state of research into childhood cancers, particularly emphasizing the role of T Cell Immune Cell Therapy that was taking the country by storm and indeed, according to one speaker I heard during my visits, might result in the scientist who first discovered this revolutionary treatment getting the Nobel Prize for Science.  This was of specific interest to me because Max Cure Foundation (MCF) provided the seed money for the development of the Immune Cell Therapy Lab at Memorial Sloan-Kettering Cancer Center in New York City (MSK).  To date, MCF has contributed $860,000 to the Max Cure Fund at MSK for the purpose of funding the laboratory (MCF’s long term goal is to contribute $5.0 million to the lab).

Science Magazine has stated that Immune Cell Therapy is the leading scientific breakthrough of 2014.  Currently, it has shown significant positive results on certain forms of leukemia and lymphoma, with the hope that in the future, based on the work being done in this area by the leading cancer hospitals in the country, including MSK, such therapy will produce positive results on other forms of childhood cancers.  The patients that are treated with T Cell Therapy are those that did not respond to standard chemotherapy treatments and those whose cancers had relapsed. The standard treatments would not have positive effects in either case.  Dr. Paul Meyers, who happened to be Max’s oncologist at MSK, in the February issue of the internal publication, “Center News”, was quoted as saying, in part, referring to the work being done in the Immune Cell Therapy Lab, “Kids who have failed every known therapy get treated with these genetically modified T cells and go into remission.  It’s an amazing accomplishment.”  We are told that at MSK, 65% of the patients treated with T cell therapy have gone into remission or otherwise had their tumors dramatically reduced in size.

Also speaking at the Hyundai Hope On Wheels Summit was Dr. Greg Armstrong from St. Jude Children’s Hospital.  Dr. Armstrong’s specialty deals with the children that survive the five years from diagnosis and identified as “survivors”.  But as noted above, after being designated a “survivor”, a child’s journey down the dark road of pediatric cancers is far from over.  In fact, one of the advocates at one of the meetings I attended suggested that a separate disease designation ought to be given for those that survive the five years and must confront the medical issues that inevitably follow for more than 95% of those that survive the five years from diagnosis.  There are 380,000 childhood cancer survivors in the United States.  Based on statistics developed by the American Cancer Society, 18% will die within 25 years following five years after diagnosis (35% of children with cancer will die within 30 years of diagnosis so that as to the 83% that survive the five years, 18% will die within the following 25 years).  Those that survive the five years have an 8 times greater mortality rate due to the increased risk of heart and liver disease, plus reoccurrence of the cancer or the occurrence of a secondary cancer.  Dr. Armstrong in his talk emphasized the substantial increased risk of death due to heart disease for the survivors, especially those that underwent a certain form of chemotherapy.  Finally, if that were not enough, greater than 95% of children that survive the five years will experience long term side effects, one-third of them being severe and chronic, one-third being moderate to severe and one-third being slight to moderate.  Dr. Armstrong made clear that survivors should be monitored regularly and that parents as well as the children as they mature must recognize “they are far from out of the woods” (my words, not Dr. Armstrong’s) when they celebrate with others that they are included in the population known as childhood cancer survivors.

Richard and Ken MochOn Friday morning I attended the 5th Annual Summit held by the Congressional Caucus in the Congressional Auditorium, Capitol Visitors Center, the same location where in 2011, Max, at age 8, having completed his chemotherapy treatments, at the invitation of Congressman McCaul, spoke to over 500 legislators, scientists, industry representatives and childhood cancer advocates in support of the Creating Hope Act (helping kids with cancer and other rare diseases) which was signed into law by President Obama in July, 2012.  I was honored to be accompanied to the Summit by Kenneth Moch, former CEO of Chimerix, the small biotech firm from North Carolina that ultimately delivered the drug to 7-year old Josh Hardy in March of this year that saved his life. Following the Summit I attended the luncheon co-hosted by CAC2, the Alliance and Children’s Cause for Cancer.  Max Cure Foundation was one of the sponsors of the luncheon.  This, too, was well-attended by many in the pediatric cancer community.  Thereafter, I was honored to be one of those invited to the meeting at the White House to discuss pediatric cancer issues, particularly that which our community thought needed to be done to better protect the children diagnosed with cancer.

The Congressional Caucus Summit on Friday, September 19th, focused on a presentation by Dr. Francis Collins, Director, National Institute of Health, which was followed by presentations on the issue of Compassionate Use waivers issued by the FDA giving a manufacturer of experimental drugs the ability to give the drugs to terminally ill patients when those patients had exhausted all appropriate treatments that had been FDA approved.  Those discussing the Compassionate Use issue were Dr. Ronald DePinho, President of MD Anderson Cancer Center, and Dr. Amrit Ray, Chief Medical Officer, Johnson & Johnson – Janssen Pharmaceuticals.  Congressmen McCaul introduced Drs. DePinho and Ray and in his remarks, indicating that the issue of Compassionate Use was brought to the forefront earlier this year through what has come to be known as the Josh Hardy matter.  In that case, a 7-year old boy who had suffered from kidney cancer was in need of an experimental drug to live (he had a bone marrow transplant followed by being infected with adenovirus- in that his immune system had been compromised, without the drug, the doctors stated he would surely die).  Josh’s family utilized social media and network media, not to mention print media, to pressure the company to give Josh the drug.  In cooperation with the FDA, the drug company, Chimerix, established a phase 3 clinical trial for children, with Josh being the first patient. In the prior two years before Josh received the drug, Chimerix had turned down 300 requests for the drug from folks seeking the drug under a compassionate use waiver.  Many of those present at the Summit were advocating for Josh to get the drug, including me.  The CEO of Chimerix at the time, Kenneth Moch, had been asked to resign (and did so soon after the drug was given to Josh), presumably because he violated company policy in agreeing with the FDA to establish the clinical trial, with Josh being the first patient.  Mr. Moch in my opinion was “collateral damage” to what turned out to benefit not only Josh (his life was saved) but also Chimerix in that it was able to establish the clinical trial for children much sooner than otherwise would have been the case and the Chimerix stock increased in value.   However, I detected criticism of Mr. Moch by Congressman McCaul in his introductory remarks and from Dr. Ray in his comments about Compassionate Use and the Josh Hardy case.  During the question and answer period, I made the following comment:

“I detected both from Congressman McCaul and from Dr. Ray criticism of the CEO of Chimerix. In my opinion Josh Hardy would not be alive today if it were not for the actions of a person with us today in this auditorium, the former CEO of Chimerix, Kenneth Moch.  I invited Mr. Moch to join me this week in attending certain of the events, including today’s Summit.  While I was going head to head with Mr. Moch trying to get Josh the drug, unbeknownst to me and others involved in the SaveJosh Campaign, Mr. Moch was working behind the scenes with the FDA to establish the phase 3 clinical trial for the drug for children, the first patient being Josh Hardy.  Josh is alive today due to the foresight and courage of Mr. Moch to apparently bypass the directive of his company and form the phase 3 clinical trial.”

I say “apparently” because according to the press reports Mr. Moch resigned.  I assumed he was asked to resign by the Board of Directors over the Josh Hardy matter in that he appears to have violated company policy in getting the drug to Josh and saving his life.

When I concluded my remarks, the audience, in unison, clapped to acknowledge what Mr. Moch had done for young Josh, resulting, as noted, in his having lost his job.  I was later told by Mr. Moch that it took a lot to make him blush, but my comments did so.  He may have lost his job, but somewhat less important to him I am sure, he gained a friend, me.  In fact, from comments made to me by others in the audience following the Summit, they thanked me for my comments, stating they had no idea that Mr. Moch had done what he accomplished and was a hero, rather than a villain, in the Josh Hardy matter.

Left to Right: Joe Baber, Richard Plotkin, Donna Carmical, Tom Pilko. aken at the White House Office of Public Engagement program for Childhood Cancer on 9/19/2014.

Left to Right: Joe Baber, Richard Plotkin, Donna Carmical, Tom Pilko. Taken at the White House Office of Public Engagement program for Childhood Cancer on 9/19/2014.

On Saturday, September 20th, I was invited to attend the advocacy meeting hosted by Alliance and moderated by the American Cancer Society (ACS).  The purpose of the meeting was to discuss the goals of the pediatric cancer community and to discuss how best to achieve those goals.  Two of the primary issues were additional funding for pediatric cancer research from NCI and Compassionate Use and Expanded Access relative to the requests for terminally ill patients of experimental drugs, whether as part of a clinical trial or as an individual patient.  The four-hour meeting touched upon all significant aspects facing the pediatric cancer community, including the often forgotten needs of those children that survive five years from diagnosis.  ACS will be issuing a summary of the meeting within the near future.

Following the luncheon immediately after the Summit concluded, some of us travelled to the White House for what was described as a Briefing and Discussion with Pediatric Cancer Advocates.  The Keynote Address was by Dr. Harold Varmus, Director of NCI.  Panel discussions followed the question and answer period at the conclusion of Dr. Varmus’ comments.  It was apparent that the White House wanted to get engaged in issues involving childhood cancers and wanted to learn as much as it could as to the needs of those children afflicted with the disease.  The panel discussion and the give and take following each speaker’s presentation among the advocates and the White House representatives, including the Department of Defense person who was identified as Director of Congressionally Directed Medical Research Programs for the Department of Defense, made it quite clear to me that those in the Executive Branch of Government were engaged in our cause.  Subsequent meetings are to be scheduled.  My only comment was as follows as I noted that President Obama issued a Proclamation proclaiming September, 2014, as National Childhood Cancer Awareness Month, encouraging all Americans to join him in reaffirming “our commitment to fighting childhood cancer”:

“I very much appreciate President Obama’s issuing the Proclamation and also appreciate the interest shown by the White House in convening this meeting with advocates for kids with cancer.  However, I wish to note that in the proclamation, the President asked to remember those children who died and to support those who are battling the disease every day.  What is missing from this Proclamation is a recognition that 379,000 pediatric cancer survivors are struggling every day with the remnants of the disease.  It is important in my opinion to recognize that 18% of the survivors will die within 30 years of their being diagnosed (17% having died over the first five years from diagnosis – 18% within the next 25 years); those that survive have an 8 times greater mortality rate; and over 95% of the survivors will suffer long term side effects from severe and chronic health problems to slight to moderate health issues.”

I hope my comments made an impact and that in next year’s Proclamation by President Obama, he will acknowledge the survivors.