Intro by Jonathan Agin, Section Editor Childhood Cancer Awareness & Advocacy
The childhood cancer world is filled with all kinds of journeys and fights. Children diagnosed with cancer fight on a daily basis for survival. Some are fortunate enough to have viable treatment options. Even still, those treatments do not always provide a “cure.” And then there are those who, on top of fighting against the underlying diagnosis, are faced with other incremental and peripheral battles that are direct outgrowths of the cancer and the treatments used to provide the “cure”.
The following is the story of how one of those very battles evolved. It is the story of strange bedfellows who ultimately grew to respect one another significantly. The story reads more like a work of fiction than the true to life reality that unfolded and has brought the community to this point of reevaluation of the policies and procedures of expanded access, or as it’s more commonly referred to, compassionate use. In the end, the efforts of the author, Richard Plotkin, numerous other advocates and one unlikely “hero” successfully saved the life of a boy who was almost certainly going to die within days. The twists and turns provide for a roller coaster ride of life and death. Thankfully, this has kick-started high-level conversations with the goal of reforming the manner in which dying children receive experimental drugs.
There are always struggles within the childhood cancer community. Fighting to obtain a drug that means the difference between life and death should not be one of those struggles.
At the ripe old age of 69 I made a new friend, Kenneth Moch. At one time I considered him to be my # 1 enemy who I believed did not care if a 7-year-old boy, Josh Hardy, attempting to defeat his cancer, died. But, he later became the main hero in saving that boy’s life.
The story is told from his point of view in a new article entitled “Rescue Me: The Challenge Of Compassionate Use In The Social Media Era“, (The Article), which he wrote with NYU Professor Arthur Caplan (who also played a role in the Hardy case). I have attached the article. I believe this article should (and hopefully will) be the basis for changing how industry, patients, health care providers and the FDA treat requests by terminally ill patients for experimental drugs and other experimental devices that could potentially save or otherwise prolong their lives.
The co-author of this article, Arthur Caplan, Ph.D., is Director, Division of Medical Ethics, NYU School of Medicine, NYU Langone Medical Center. He has, with a group of his colleagues, begun an investigation into how, if at all, the current system regarding the granting of compassionate use waivers by the FDA and industry needs to be changed. I am privileged and humbled to have been selected by Dr. Caplan to be among those on the Patient Advocacy Committee seeking to assist Dr. Caplan in his investigation and look forward to working with those on other committees consisting of industry representatives, including Kenneth Moch, Institutional Review Board (IRB) members, which is a research ethics review committee, physicians, as well as other noted representatives in various fields brought together by Dr. Caplan to accomplish his goal.
But first, I want you to know how I got to the point of being chosen by Dr. Caplan as the sole representative from the childhood cancer community to be selected for this panel (as the representative to represent the children with cancer and their families – otherwise known as the “little guys”). And also, how my former adversary, Ken Moch, has become my friend and someone I admire greatly.
For 38 years, until my retirement in 2008 from my law firm as a trial lawyer, my practice focused primarily on representing industry “against the little guys”. I fought for auto manufacturers in defense of claims by those that claimed they bought defective automobiles; I fought for banks and financial service firms against claims by individual customers that they lost money due to the alleged unlawful acts of my clients; and I supervised 54 law firms in 48 states plus the District of Columbia on behalf of a company which manufactured products containing asbestos over a five-year period during the height of the asbestos litigation in the late 1970’s/early 1980’s. Plaintiffs claimed my client’s products resulted in their asbestos related diseases, including cancers. I attempted to demonstrate through expert witness testimony that my client’s products did not cause the diseases allegedly afflicting plaintiffs.
I was at the height of my career in 2007 when my grandson, Max, then age 4, was diagnosed with a rare form of cancer, stage 4. I soon thereafter retired from my law firm, left behind a very successful career in the law, and with my family formed The Max Cure Foundation, Inc., whose initial mission was to fund research and to assist low income families battling cancer in their children. I had left my advocacy skills behind – that is until early 2014 when, unbeknownst to me, I obtained for a terminally ill 11-year old boy with brain cancer the first compassionate use waiver ever issued by the Federal Drug Administration (FDA) for a child for a combination drug therapy consisting of an experimental drug manufactured by a client of my former law firm combined with two drugs already on the market. I made a few phone calls, which resulted in the granting of consent by my former firm’s client that led to the granting by the FDA of the compassionate use waiver. When I was told I had broken ground and was congratulated by a colleague in the childhood cancer community, that was the first I heard the term “compassionate use.”
Then came Josh Hardy and Max Cure’s involvement in the #SaveJosh Campaign that led ultimately to saving the life of 7-year old Josh, who had fought several bouts of cancer since he was 9 months old. Following a bone marrow transplant at St. Jude Children’s Hospital in Tennessee in January, 2014, Josh developed a virus (identified as adenovirus) that was threatening to take his life if his immune compromised body did not receive an experimental drug known as brincidofovir (the “Drug”). The Drug was manufactured by Chimerix, a small biotech firm in North Carolina. St. Jude had been involved in a phase 2 clinical trial of the Drug as it was administered to children. The doctors at St. Jude concluded after Josh developed the adenovirus infection that without the Drug, he would die.
I was contacted on March 6th by the President of the Coalition Against Childhood Cancer (CAC2) and told of the above and that if Josh were not given the Drug by the following weekend, March 15th, Josh would most certainly die. I was asked to lead the charge for Josh, with several other members of CAC2 assisting me behind the scene.
Chimerix had previously made the Drug available to over 400 patients under “compassionate use” protocols, but had stopped doing so about two years earlier in order to focus its efforts on a Phase 3 clinical trial needed to secure FDA approval for the Drug. Chimerix had not only denied the requests by St. Jude to give Josh the Drug, but over that two year period prior to the requests by St. Jude, I later learned that 300 or so similar requests were also denied. If the manufacturer of an experimental drug like brincidofovir agrees to give the drug to the patient requesting it, the FDA then considers whether to grant a compassionate use waiver permitting the drug to be administered to the patient. I was told that if a manufacturer agrees to dispense an experimental drug, it almost always follows that the FDA grants the request for the waiver.
I knew time was of the essence and immediately reached out to Chimerix to understand why it had declined the request to save Josh’s life. I did so on Friday March 7th and spoke to Chimerix’ President, Ken Moch, which was the first of three conversations between Mr. Moch and me. This led to my appearance on Fox & Friends on Tuesday March 11th. During my television appearance I made an appeal to the Chimerix Board of Directors to overturn the decision of Mr. Moch who I believed unjustifiably refused to give the Drug to Josh, stating without the Drug, Josh would die by the weekend (and repeated that plea in an email to the Board when I returned to my office).
By March 11th, Max Cure through its contacts in the media assisted in getting Josh’s mom, Aimee Hardy, on CNN (March 9th), FNC (March 10th) and CBS (March 11th). The goal was to gather public support behind the #SaveJosh Campaign to force Chimerix to give the Drug to Josh. Over the 4-day period between Saturday March 8th and Tuesday March 11th the Max Cure social media campaign reached 1.1 million people on Facebook and 400,000 on Twitter. The campaign resulted not only in media coverage throughout the United States but also in Brazil, France, London, Germany and the Mid-East, among perhaps other countries and geographic areas.
Mr. Moch told me in our conversation on March 10th that as a result of the social media campaign, he and others at Chimerix had received death threats, causing the company to hire security for those involved. We certainly did not, and do not, condone the death threats or threats of any nature, which we did not encourage.
Based on my conversations with Mr. Moch, I did not think I would be able to change his position in time and Josh would die by the weekend. While all this was going on, other CAC2 members were working their contacts behind the scene to assist the #SaveJosh Campaign.
Ultimately, though, I received a call at about 6pm on Tuesday, March 11th from an unnamed source, who told me to please call the person I then considered my number one nemesis, Ken Moch. I thought he was kidding. By that time I thought it was inevitable that Josh would die within a matter of days unless the Board of Directors of Chimerix overruled Mr. Moch’s decision.
I nonetheless called Mr. Moch. I was told that I should be on the lookout for an announcement, which might be released by Chimerix in a matter of an hour or so. At 6:57pm on March 11th, the announcement appeared that Chimerix had been working actively with the FDA outside of the public view to establish a new Phase 3 clinical trial to consist of 20 children with the same adenovirus that was afflicting Josh, the first patient being Josh Hardy. This was not a single patient “compassionate use,” but rather a way of meeting the needs of what Mr. Moch describes as “the many Joshes.”
By the time the press release was issued, Chimerix had already arranged for the Drug to be delivered to St. Jude. Josh was given the Drug on March 12th with immediate positive impact on his virus. Josh survived and hopefully will live a long and healthy life due to the heroic efforts of Mr. Moch and Chimerix, aided by the FDA.
Little did I know then how the experience with Josh Hardy would impact my life and would lead The Max Cure Foundation in a direction it had not previously laid out formally, i.e., to add advocacy to its mission. I also did not know that I would also through this effort establish a relationship that I would cherish going forward – a friendship with Ken Moch. It should also be noted that on April 10th it was announced that Chimerix had replaced Mr. Moch as President of Chimerix. Mr. Moch and I had planned on meeting in New York City on April 22nd during which I would toast him for his and Chimerix’ efforts that saved Josh’s life. That meeting was cancelled but I am hopeful, especially after reading the Article that he will allow me to make that toast at a later date.
Following March 12th, when Josh was given the Drug which saved his life, through CAC2 I again was asked to get involved in helping 3 other children with cancer obtain experimental drugs. I then realized how arbitrary the system is relative to the granting of compassionate use waivers by the FDA and what an incredible burden is placed on those of us in the advocacy arena trying to help save the lives of children with cancer. Given what CAC2 was able to assist in accomplishing for the Hardy’s, I realized we had within our power the ability and burden to help decide which children would be given a chance to live and which would continue on the path to premature death. Of the three children referenced above, I elected not to get involved for two and did not call upon the Max Cure Foundation’s social media team and its supporters with media contacts to seek compassionate use waivers. It appeared to me, a layman and former lawyer with no medical training, that the drugs sought had no chance of saving their lives (both children have since died). As to the third child, who is 15 years old and currently fighting for her life, I elected to get involved. In my limited world, I was making a decision as to who should live and who should die – a very uncomfortable position to be in. After reading the Article by Kenneth Moch and Arthur Caplan and after attending the first meeting last month of the patient advocacy group established by Dr. Caplan, I realized that something MUST be done to establish a better system to determine who shall live and who shall die relative to the issuance of compassionate use waivers. Those with access to the media advocating for cute photogenic kids ought not have their children be given a better chance to live when compared to others that, for among other reasons, do not have media access because they are not sympathetic children. Do not get me wrong, I have said that of all my professional accomplishments, the one I am most proud of is that I participated in the process that saved Josh’s life and am hopeful that I will have been able to play a similar role with the 15-year-old girl referenced above. I look forward to working with Dr. Caplan and Kenneth Moch in accomplishing what the attached article suggests needs to be done, namely, establish a better system.