Hope is a precious commodity to the parents of DIPG children. It must be pursued and discovered, and then cherished and defended once found. For us, hope came from a team of doctors on the cutting edge of DIPG research.
As mentioned in the last post, one of the biopsy samples from Piper’s tumor was sent to doctors and researchers at the University of Michigan so they could determine the tumor’s full genetic profile. The doctors sent us swabs to obtain Piper’s normal DNA from the inside of her cheeks, and they compared the profile of Piper’s normal DNA against the DNA found in her tumor to identify the tumor’s specific mutations. Performing this task took many weeks but resulted in the discovery that Piper’s cancer is driven by two genetic mutations. These mutations, in turn, are targeted and treated by two existing FDA-approved medications called Panobinostat and Everolimus.
Armed with the knowledge that Piper’s tumor may well respond to these medicines, and that they would probably get into her brain, taking them was presented as the best option to prolong her survival. At last we had hope.
That hope, however, was quickly dashed. Although months of science had gone into discerning which drugs would work to treat Piper’s disease, our health insurer refused to pay for them on the grounds that they were experimental and investigational. Our doctors appealed, but the insurer automatically maintained its denial since the drugs had not been FDA-approved for the treatment of Piper’s specific disease. As a reminder, there are no drugs FDA-approved for the treatment of DIPG. We were crushed when we learned this, followed by the news that the drugs would cost $35,000 per month if we were to pay out of pocket.
Fortunately, I am an attorney specializing in insurance coverage and litigation. Having secured health and disability coverage for many others in similar circumstances, I knew how the internal appeal process worked and what documentation was necessary to get approval of the medicines. I scoured through our 128-page policy to find that while it potentially excluded experimental and investigational drugs, it covered off-label use of FDA-approved medicines. Against this policy basis, I called the insurance company every day for a week to gather and present information, I conferred with other lawyers to discuss strategy, and I met with Piper’s doctors and nurses to obtain medical articles and letters supporting that the medicines were safe and effective. After a second appeal, the insurer agreed to pay for the medicines.
A relief no doubt, but the process cost nearly a month of Piper’s precious time.
Last week Piper began using Panobinostat, taken in the form of two capsules three times a week, every other week. No small feat for a 3-year-old who practiced for an hour with M&Ms before she could learn to swallow pills. Pills that each cost $1,450, so it was a nervous undertaking for us all. But like everything, she has tolerated the strange new routine remarkably well. She will start Everolimus in the coming weeks, and we pray they will be the breakthrough she deserves.
We hope that Piper’s fight, and in turn, our struggle to obtain science-based treatment for her, paints a picture of the difficulties pediatric cancer families face every day. There are simply too few medicines for the treatment of cancer in children, and even when they exist, insurance companies will not willingly approve them.
So, in addition to caring for our child by day, and trying to maintain hope and normalcy, we were forced into a secondary fight in the off hours. Without the specific knowledge we were fortunate to have, we might not have this opportunity. That needs to change. Because in the world of DIPG, hope must be nurtured. Once you find it, you can never let go of it.
We still have ours, and for that we are grateful.
Nothing in this new reality makes sense. It’s a place where opposites rule and decisions – what few we have in this mess – must be made quickly.
Where else can words like “progression” be words to fear? Where else can phrases like “enjoy your time” be the polite equivalent of “sorry we can’t help you”? Where else can children go from full of life, to a life celebrated in memory in the same year?
Every year there are new technologies and slick advancements – money poured left and right for mindless entertainment. But the world of pediatric cancer research remains sluggish and unchanged. Yet in our experience, people DO want to help and WISH they could do more. Trouble is, there seems little to be done until more doors open up, or alternatively, too much to be done with too few resources.
Bottom line – it’s an impossible place, our reality. This is DIPG. But as parents, our instinct to keep trying, hoping, and fighting for Piper is as strong as our instinct to breathe, no matter the odds.
After Piper was diagnosed, we were presented with two up-front clinical trial options. We elected to participate in a phase zero study which entailed giving her one dose of Gemcitabine, a well-known chemotherapy drug, just before her biopsy, to see if the molecules could pass the blood/brain barrier (BBB) and reach her tumor. The BBB has proven an impenetrable fortress in countless trials before, but to everyone’s surprise the drug got in and was equally dispersed throughout her tumor tissue. But, given the early stage of research, we were told this wasn’t a viable therapy we could consider for Piper, as factors like dosing and side-effects were still untested.
Next we were presented with an opportunity to send some of Piper’s tumor collected at biopsy to the University of Michigan to undergo extensive genetic testing. This is something researchers have only begun to do now that DIPG biopsies are considered safe and routinely offered. Previously, tests like these were limited to post-mortem tissue when elected by the family. The goal of this study was to illuminate the specific biology of Piper’s tissue, and use that information to identify effective treatments.
After agreeing to those initial trials we focused on Piper’s six week radiation regimen (AKA the same and only standard treatment for DIPG unchanged in 60+ years). We were repeatedly told that radiation was palliative, and that it was not a cure for Piper’s DIPG. We were advised to seek second opinions only after this point, as radiation represents the “gold standard” in treatment and would be suggested by any/all reputable neuro-oncologists.
When the time came, we sought opinions from all the top pediatric cancer institutions including: Stanford, Dana Farber, St. Jude, and Memorial Sloan Kettering. This exercise resulted in nothing ground-breaking, only unanimous agreement of Piper’s diagnosis. It did give us the opportunity to consider a few additional trials that were not previously available, however we resolved to try a different path altogether.
With the genetic information obtained from the University of Michigan study, Piper’s care team will pursue a personalized treatment plan. We reasoned that if obtaining biopsy tissue and genetically testing it represents the newest technology in the world of DIPG, we should use what we learned. While this is not presented as a “cure” (a word decidedly avoided in these cases) it goes beyond just controlling symptoms. It aims to fight Piper’s tumor using a duo of chemotherapy drugs believed to represent her best chance. Unlike trials, this gives her the freedom to continue on Avastin in lieu of steroids (greatly improving her quality of life) as well as stay in Colorado for her care.
If we are successful in our insurance negotiations, Piper will start treatment this month, along with physical therapy to encourage her body to reclaim its independence. We are in uncharted territory. But it’s what we have – and its more than some families ever get. For our part, we will continue to “enjoy our time” and use it to laugh together, research like hell, advocate in between, and hope and pray that this treatment works for Piper.
The warning signs were not what you might imagine with such a weighty diagnosis. Piper’s symptoms presented in such a way that simple, rational explanations were enough to satisfy our early concerns.
After Nelson and I returned home from a 3-night trip to Chicago (our first travel without the girls) we began to notice changes in Piper. It started with her speech. Minor pronunciation issues that we attributed to her wanting to talk like her baby sister, Harlow, or simply acting out because we had been away. A couple days later we noticed she also seemed to have difficulty swallowing and chewing her food. Her saliva would gather in the right corner of her mouth and if she was laughing or looking down, it would drip out. But still this was all minor. Then came the calls from her school. Always watchful, they described a lack of energy and engagement uncharacteristic for Piper.
Friday, June 9, six days after our trip, we had an appointment with our family pediatrician in hopes of finding answers. The visit put all our concerns at ease… “it may be an ear, nose, throat issue” … “perhaps she has strep or swollen glands” …. “no neurological concerns”…. Piper had also been having night terrors and, paradoxically, sleep laughter over the previous six months (also early signs of DIPG) so the doctor felt that sleep deprivation might be playing a role. She scheduled a sleep study and told us to go to Target to get nasal spray to calm down what she thought was a swollen tonsil.
Then, late Sunday, June 11, Piper’s symptoms suddenly worsened. No longer nuanced changes, but clear signs that something bigger than an ENT issue was at hand. She lost her energy, began to trip and stumble, and her speech became so slurred we could hardly make out her words. Although she was talking as much as ever, she was clearly laboring to do so. Alarmed, we began to Google her symptoms but her eventual diagnosis never once came up in the search. At that late hour, reassuring ourselves it could not be neurological like the doctor said, we decided to let her rest and take her in first thing the following morning.
Monday, June 12, 2017, will forever be etched in our memories as the day the sky fell. The day our seemingly perfect world evaporated. At 6:30 a.m. it became clear Piper was not improving. Although she had slept comfortably in our bed all night, she was slow to wake and couldn’t even really get out of bed. Rather than wait for her doctor’s office to open, we rushed to the ER at Children’s Hospital South Campus. They wasted no time getting a CT scan in process. Even then, worried as I was, I believed the explanation would be something I’d at least heard of, or could be treated in the age of modern medicine. We waited for what seemed like hours, and although we had been given no information, we began to know that something was wrong. The nurses and staff who were so cheery when we arrived were now avoiding eye contact and conversation. After about half an hour, the ER doctor asked us to meet her in an empty room. The short walk to the next room was silent and foreboding. A box of tissues had already been placed on the table in front of where we were to sit, then the doctor bravely, and with obvious heartbreak, told us that the scan showed a mass in Piper’s brainstem. Everything echoed. “What kind of a mass?” we both asked. The doctor said nobody would know until Piper had an MRI at the Children’s Hospital Main Campus in Aurora. After we pressed the issue, she said the mass appeared to be cancerous. Before the conversation was over, an ambulance was ready for us.
Minutes have never felt so long. After we arrived, Piper’s condition began rapidly deteriorating. She couldn’t walk or even move much at all. She couldn’t talk, but just whimpered and uttered half-formed words. She wanted to tell us something, but she just couldn’t. The doctors explained they would image Piper’s brain and spine and that it would take about an hour and a half. We held and kissed her as they put her under general anesthesia so they could obtain a clear image.
Before the procedure was even complete we were asked to come to yet another ominous room. They had seen enough to make a diagnosis. Waiting for us was another pre-placed box of tissues, two neuro-oncologists, a medical student, and a social worker. It was there that we first heard the words Diffuse Intrinsic Pontine Glioma, or DIPG for short. These words came alongside others like “inoperable,” “incurable,” and “universally fatal.” The doctors said our beautiful 3-year-old would die from her brain tumor and that she might live 6-9 months if we elected palliative radiation, or just 7 weeks if we did nothing.
We held hands and cried until there were no tears left. Then each of us broke the news to our parents. Life has never been the same since. We spent the next week in-patient with Piper, encouraging her to talk, chew, and slowly walk again. Her tumor was biopsied (something they only started doing in the last five years) and confirmed it to be grade IV DIPG.
Every day since June 12, 2017, has been far from normal – even in the happiest of moments. Although our reality has been irreversibly altered, some changes have been positive. We understand as we never could have before that we should be grateful for each day. To take struggles one step at a time. To live for today. To love like there may not be a tomorrow.
Arvada, Colorado, USA