The Clinical Trial System is failing America’s Children

Amanda Haddock, DragonMaster Foundation on Medium 8-20-2019

That may seem like a bold headline, but it isn’t click bait. This is a real problem facing many Americans (adults and children) in their most dire moments of need.

The best way I can illustrate this is by telling you a story. It’s not fictional. This story is playing out right now, as I type.

There is a 20 year old young man, named Jace, who was just diagnosed with a highly aggressive form of brain cancer called DIPG. There is no known cure for this type of brain cancer, and the life expectancy is very short. Basically, Jace has no time to lose in finding an innovative treatment to save his life. (It shouldn’t be up to Jace to find his own treatment, but that is a story for another day.)

So Jace and his family scour the internet to try and find a treatment that might a) extend or even save Jace’s life and b) help the next kid in line with this horrible disease. They call doctors and researchers literally all over the world. They find a drug that is new and showing some promise in clinical trials — BUT — Jace doesn’t qualify for those clinical trials.

There must be a good reason for that, right? Well, no, not necessarily. The drug Jace wants to go on, known as ONC201, actually has two open trials, one for adults, and one for children. Since Jace is 20, he falls between the gaps of those two trial designs.

Let me break that down a little further for you. The adult trial has been going on for a little while, and due to the nature of adult treatments and tumor progression, this trial was designed for patients with recurrent disease. That means it can’t be used as the first line of defense. (Never mind that some of those patients won’t respond to the first line of defense… that, too, is a discussion for another day.) Jace doesn’t qualify for the adult study because his tumor is newly diagnosed.

The pediatric trial is for newly diagnosed patients (yay!), but Jace doesn’t qualify for it either. Why not, you ask? Well, he is 20, and in the eyes of science, that is no longer a child. Never mind the fact that he has a “pediatric” tumor and most of the cases they would compare him to are for kids below the age of 12.

So even though he qualifies in all other aspects, Jace doesn’t match either trial’s guidelines completely. So no trial for Jace. But wait. Isn’t that what compassionate use is for?!? This has to be the perfect scenario, right?!?

It would appear to be a textbook case for compassionate use, so the family appeals to the drug company. They get approved. YAY!!! Now all they have to do is get a treating hospital to agree. That should be simple, since he is so close to the qualifying criteria, right? As it turns out, no.

So far, two hospitals have turned Jace down for compassionate use. One because their governing board couldn’t reach a consensus and one because they “aren’t doing any more compassionate use” right now. I really wish I was making a typo there. I’m sure it was said with more compassion than I can assign to that statement, but that is a shoulder shrug in the face of a patient whose very life hangs in the balance.

Hospitals are businesses, and as a foundation, we spend a lot of our time trying to negotiate things to paint them in the best possible light. We want to be good partners. We want to highlight the successes and minimize the failures so that people will focus more on what we can do to work together. But this kind of thing has to be changed. NOW.

I’m writing this story in hopes that a hospital of merit will step forward and do the right thing here. I truly believe that one of them will, but I’m a little ashamed that we are having to make a widespread public plea. I could help the family call all the hospitals we think might help, but that will take days, maybe weeks, and Jace doesn’t have any time to lose.

In this moment, we are failing Jace, but we don’t have to. In this instance, the doors are all unlocked. We are just in need of a person who will open it and let him in. Please share this story to help draw attention to this problem in general, and Jace’s specific need right now. Together, we can bring about the awareness that could easily translate to the changes needed to offer these patients hope.