You may remember the #SaveJosh news articles in the spring of 2014 where little Josh Hardy’s family mounted, as a last ditch effort, a social media campaign to get him into a clinical trial that St. Jude’s doctors said was his last chance. He had only a few weeks left or he was going to die, there was nothing else that would save him. It seems the entire childhood cancer community came together at once and with the help of several key players, social media, and TV news and talk shows, within 10 days, Josh got his medicine and it literally saved his life. You may also remember Judson Shepard, a little boy with a similar situation needing the same drug. This time the little boy died because it took too long to get the medicine. These two incidents occurred and can you believe it, the FDA has a compassionate use program in effect that “should have worked” in both cases. It did not work and it is not working in another case at this very moment. Frankly, for the most part, it’s not working for children with cancer.
I am sure most of the readers of this blog know Ellyn Miller, Gabriella Miller’s mom, and founder of Smashing Walnuts Foundation. She and her husband Mark set up the foundation to honor Gabriella’s wishes to find a cure for brain cancer. Her efforts have resulted in the passing of the Gabriella Miller Kids First Research Act to repurpose Presidential Election and Campaign Funds into research for pediatric disease and childhood cancer. She is now working very hard to ensure that the bill gets funded.
Ellyn, wrote and posted the article below on the Smashing Walnuts Facebook page. In drawing attention to the current case of fifteen year-old Nathalie Traller and the barriers to compassionate use for kids, Ellyn sums up the problems as no one else can… a mother who has pursued every avenue of compassionate use to try and save her dying child.
Smashing Walnuts Facebook Page July 30, 2014 by Ellyn Miller
I have attached a link to a blog written by the dad of a 15-year old girl who has been told that his daughter cannot participate in a promising clinical trial because she is a minor. This girl has to wait three (3) more years until she is considered to be an adult.
The problem is that cancer does not wait for someone to come of age. Trust me on this one. It didn’t wait for my daughter. When Gabriella’s cancer started to regrow our options for clinical trials were limited. That didn’t stop me though. I WAS FIGHTING FOR MY DAUGHTER’S LIFE!!! I called all around the country, all around the world, speaking with doctors/researchers. I spoke to everyone that had any promising brain cancer treatments. NIH, Hopkins, Sloan Kettering, Duke, UCSF, the UK, Australia – to name a few… I was told – over and over again – that Gabriella wasn’t a candidate because of her age, she was too far post radiation, her tumor had metastasized … You name it, I heard it. I also heard, “Maybe the trial will open for your daughter in six months or a year. Well, dammit, my daughter didn’t have that long. I needed, my daughter needed, treatment NOW.
But, I have to tell you that this “age” thing (not being old enough for a drug trial) just makes me see RED! Are you kidding me!?! Not old enough!?! So, dear sirs that make up these laws, let me ask you this…WHY are we giving our children drugs that were created for adults? WHY are we giving our children drugs that are 30, 40, 50+ years old? WHY are we giving our kids drugs that are so toxic that the nurses have to wear gloves to administer them? WHY are we giving our children drugs that have known side effects such as causing secondary cancers? And, you’re telling me that you won’t/can’t give our children a new, promising drug because “they are too young”?!? I simply can’t understand that.
My daughter died! She was only ten-years old. Ask any parent of a child dying from cancer if they would let their child try a promising new drug, despite their young age. THE ANSWER IS YES, YES, YES!!! The alternative is too horrible to comprehend. The alternative is a nightmare. Our children die! Do you know how many of us parents are living in that nightmare? Way too many. And, the number grows everyday.
Don’t use age as the reason not to treat our kids. Their age should be the reason why we DO treat them. They have so much to live for…
If I were a member of the FDA, NIH, NCI or Pharma, I certainly would not want to face Ellyn Miller and try to explain why the current compassionate use policy is good for children with cancer.
Authors: Ellyn Miller and Joe Baber
Four-Square Clobbers Cancer 
Pingback: The Fault in Our Systems | Four-Square Clobbers Cancer
Hi, the compassionate use system is a royal mess and I don’t have the answers as to a fix. However, for any patient with cancer (any age or any tumor type), the best course of action is typically treatment in a well-designed clinical trial. That age-18 cutoff is extremely arbitrary and a big part of the problems facing childhood cancer drug development. I think there should be a revisiting of rational age criteria in the current era of these novel investigational anticancer therapies, which are very different than traditional chemotherapy. I don’t see why at least some early-phase clinical trials, which are typically conducted in populations of patients with a mix of different advanced and difficult-to-treat cancer types, can’t include pediatric subgroups and adult subgroups in the same trial. The trial designs could be tweaked should differences in dosing exist. Improving the trial designs to meet current needs, not excluding children with poor prognosis tumors simply because they are children, should be the answer.
Laurie O – You have nailed it. We are stuck with an outdated clinical trial model. Lining up the kids for last on promising therapies shouldn’t wash anymore. The UK has passed legislation where trial age cut-offs must be justified in science.
Our children deserve so much more! If there is a viable option, use it! For goodness sake! They are the future. Ellyn & Joe, great post. I know that Gabriella is proud of your efforts, as are we! We do what we do for all the children ( regardless of age )!