Rich’s Story: My Unconventional Battle with GIST
by Rich Klein

CANCER – after the shock and horror of the diagnosis comes the treatment. The doctors usually follow a script of  “take this chemo because it seems to work in 80% (or 50% or 60%, etc) of the people that have your cancer.” 

But what about the 20% it doesn’t help? Why?

Peel the onion back a layer and it may be because the genes that are broken within you causing uncontrolled growth of the tumor are different than the 80%. This is where genetic mutation analysis came into play for me. 

I have something called a Gastrointestinal Stromal Tumor or GIST for short. It is very rare. I started treatment 6 years ago in the standard way I described – an 80% drug called Gleevec.

By way of explanation: These photos have been inserted to show I have a life too!

It didn’t work, so I started on the next drug on the list, Sutent. But now I wanted to know more. I had a mutation analysis on my tumor sample done. It showed clearly I was in the 20% category but still did not pin down what was broken. It said I was “Wildtype”, a label they put on something when they cannot pin down the specific mutation type.

That’s when I turned to the National Institute of Health’s GIST Wild Type Clinic for help and was lucky enough for them to look at my case. They did further mutation testing and determined I had a mutation in something called the BRAF gene. This was present in many cancers; being common to about 60% of Melanoma, approximately 10% in Colorectal Cancer but very very rare in GIST. Statistically there are only about 50 or 60 cases a year specifically diagnosed as BRAF GIST in the United States. 


For me it made sense to start going “off script”.  If I had a BRAF mutation, didn’t it make sense for me to take drugs specifically “designed” to fight BRAF mutations themselves. Since Melanoma had such a high percentage of cases attributed to BRAF, from that point, I started to target my treatment using Melanoma BRAF drugs. This is not the easiest thing to do since insurance companies and clinical trials still look at primary diseases (eg, melanoma) and not other patients with unique mutations (eg, GIST with BRAF).

My first 2 trials were unequivocal failures for me, even though those 2 drugs continue to show good success in fighting other people’s cancers. Quite discouraging. These were at OHSU and Memorial Sloan Kettering in Portland, OR and New York City respectively.  Pressing on I found a BRAF drug in trials at M.D. Anderson in Houston. This gave me my very first clearly effective drug to treat MY GIST. It held the beast at bay for 8 months! During this period I also was following a second drug; Vemurafenib, but there were no trials available for me.

It got FDA approval for BRAF Melanoma and I knew I would need this drug at one point or another in my fight.


After drug resistance occurred in Houston, I had my third surgery and I also convinced Dr Heinrich at OHSU to write a prescription for Vemurafenib off label. He was reluctant; not thinking it would be approved and not wanting me to waste my time and energy.  At a cost of $10,000 per month there was no way I could afford this treatment without insurance coverage.

I went forward and right on queue, Medicare Part D drug care promptly denied me. “Vemurafenib was for Melanoma. You do not have that disease.”  The insurance company denied me initially and through 2 appeals. 

For the third level of appeal, without the aid of a lawyer, I went to Federal Court and I won! Through peer reviewed literature, letters from several leading cancer researchers to the court and testimony from Dr. Heinrich and myself, I convinced the judge that this drug was designed to provide targeted therapy addressed specifically toward all cancers that harbor BRAF mutations. It was not only intended forMelanoma. 


I started this new direction, searching out trials and drugs designed to target my mutations by chasing BRAF mutant Melanoma. Even though these drugs have not been permanent solutions, they have shrunk and stabilized my disease for finite amounts of time. This was true for trial #4 in Los Angeles (effective for 4 months) and trial #5 in Portland (lasted 5 months). Along the way, I found out that besides BRAF, one of the tumor samples examined from my third surgery also showed mutation of the PIK3CA gene.

For my sixth and current trial I found and started on a dual drug trial. It was being offered at 3 different cancer centers. Two of the  three denied me admission because even though the criteria allowed for all solid tumors with the BRAF mutation, the drug combination in their minds was targeting Melanoma patients. The Principal Investigator at the University of Pennsylvania in Philadelphia, thank God, understood the distinction between targeting BRAF versus the erroneous targeting of Melanoma only. 

One of the drugs was Vemurafenib, the drug I fought so hard for. The trial does not pay for this drug. As I said earlier, I knew I would need it in my arsenal sooner or later. Now was the time to take advantage of my court case insurance win. It is being supplied to me by my Medicare Part D provider for the drug trial under normal Medicare pricing points. The first prescription I filled promptly took me into the Medicare “donut hole” and back out into catastrophic coverage levels; a cost initially of about $2500 and then $500 per month thereafter. It’s still very expensive but no where close to $10,000 per month.

The second drug is called PX866. It is being combined with Vemurafenib to see if it can improve the long term effectiveness of the BRAF inhibitor, putting off disease progression. It is a PIK3 inhibitor and as I just stated before, I have newly found knowledge that I have this mutation type as well. When I found this trial I knew I needed to get into it.

For the past nine months this drug combination trial has shrunk my tumor load by over 30% and then has kept them absolutely unchanged and stable.

I continue to look for drug combinations that would target my specific genetic disposition while seeing the world one cancer center at a time. I live my life in a different sort of “normal” than others. I’ve a great support group of friends and family and I have an amazing wife. In closing I add that we are currently preparing to spend a month vacation time in Australia and New Zealand. Not bad for normal.