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.