What were we learning?  How were we doing in school?  Did we understand the physics of building a house?  These are the questions my grandfather used to challenge us with every Sunday, as our family gathered for breakfast.  When the conversation turned towards history, I would daze off as my grandfather, father and brother would ramble onward.  But, twice a year, I was lucky enough to have the opportunity to show him my grades — the gleaming smile on his face was priceless.  He would say he expected it, but I knew by his smile how much it meant to him.  I used to revel in this.  Then, every time it was his birthday, he would say, “No… it’s not my birthday; it’s my anniversary.  I was born on August 15, 1929!”  “You’re a fart smeller,” was another classic line.  His playful demeanor defined what it meant to have Witt Humor.

then,

In early 2006, my grandfather was diagnosed with non-small cell lung cancer. His prognosis was initially very good; the cancer was caught at an early stage (it hadn’t spread), and he was set to have surgery to remove the mass.  Everything was hopeful.

Suddenly, we were startled…!  His surgery, set to begin shortly after my high school graduation, was taken off the table!  He had suffered a stroke while going off his blood thinners in preparation for the operation.  The gold standard was no longer an option and deemed “unsafe.”  His prognosis suddenly drastically diminished.  He began chemotherapy and radiation treatment.

I went off to school, as he began the chemo.  At this point, hope remained prevalent; but, as time progressed, so did his tumor.  It was around then that we began researching for alternative treatments which might benefit him / give us any bit of hope.

Nearly seven months later, we found a study that resulted in an 80% reduction, in mice, in both the size and weight of the same tumor type as his. This reduction in size and weight occurred just two days after the therapy’s administration! The result was astounding. And, while only performed in mice, it provided him with the best hope and opportunity possible. By this time, his cancer had gone into remission and recurred (stopped growing and started once more); he had done away with the option of continuing therapy, which was most often chemotherapy, as it left him nauseated and in poor physical condition.

Explaining to him that this therapy was not like his other therapies, that it was targeted therapy and shouldn’t have these same side effects, he agreed to try it. Understanding and accepting unknown consequences and risks involved, our family contacted the researchers who performed the study to plea for them to give my grandfather one last chance at life (access to their experimental treatment). They denied, citing the therapy was not approved by the FDA. We expected this (it is the law, right?), and pushed for another possible avenue of access. The doctor said one part of the therapy (it was two parts) was approved in China, and the other was available in a clinical trial in Texas.   This was our last hope.  With summer coming up in a few weeks, we decided to wait until then to attempt to obtain these therapies.

He died one week after summer began.

The problem

The problem here is subtle.  I never knew it until three years later.  Death will come to us all; rather the issue lies in that there is a method to obtain experimental therapies not yet approved by the FDA — we did not have to go to China, nor to a clinical study in Texas.  All we had to do was show he had exhausted his other options, did not meet the eligibility standards for the clinical trial, and get approval from the researcher, his doctor, and the FDA.   But, there was no mention of this option, known as Expanded Access / Compassionate Use, by his physician or this researcher.

The reason our family wasn’t told about this particular option might have been because 1) the FDA rarely approves therapies for this use prior to phase II clinical trial’s completion (this study was in pre-phase I stage of study), 2) the researcher may simply not have known about this option, 3) the researcher did know about the option but neglected to mention it, consciously or unintentionally.  Whatever the reason, the dilemma and issue our family faced remains…

This was my grandfather’s last opportunity at life, there were no other options, and our grandfather was going to die anyway.  Most disheartening, to me, is that he accepted unforeseen risks and unknown benefits; but was not given the opportunity to take his last stand, because he did not know a particular option existed.  This is an injustice that I cannot pass over, not if I am to be sure that anyone else I love or that my friends or family or anyone else in the world who might be a patient some day, we will know their options as patients.  Certainly, I can’t pass this up until it’s fixed, if we have the right to preserve our life, using all options possible when facing the biggest battle we may ever face – our battle for life.

Sincerely,

Ryan W

Founder

Innovation and Choice

ryan.witt@innovationandchoice.com