February 16th, 2015

Living with Stage IV Appendix Cancer/Clinical Trial-Immunotherapy

PMP/Appendix Cancer, Peritoneal Surface Malignancy, Peritoneal Carcinomatosis Immunotherapy Trial Winter 2015

My name is Cindy, and I am the advocate and caregiver for my husband Paul.

I am glad to be able to write a little about our experiences over the past 32 months since my husband's cancer diagnosis in the hopes that it might help someone else that is going through similar circumstances. Paul was diagnosed with an aggressive form of Appendix Cancer, Adenocarcinoma moderately differentiated, a form of PMP, Pseudomyxoma Peritonei in late June of 2012 after he asked for a CT scan from his local GP. That scan radically changed the course of our lives. It showed extensive abdominal metastatic appendiceal cancer. My husband was an active, robust, 51 year old New York State law enforcement officer assigned to the training academy for the DEC at that time. We were both at the apex of our careers, with two college aged children and busy and active lives. He had been experiencing some mild but mysterious symptoms of electrical shooting pains for a couple of months that would travel across the surface of his lower abdomen then disappear. He began to call these pains, "Zingers". He also had some lower back pain which we attributed to his lower discs in his spine that he had had some issues with in the past. We were never anticipating the diagnosis we received, Stage IV terminal cancer that may be able to be treated, but would likely not be survivable. The words 3 to 6 months floated in the air between the doctor and us in that surreal consult. 

The first surgical oncologist we were sent to in NY turned us away and told us that while systemic FOLFOX chemotherapy may soften and shrink down the extensive tumor, my husband's case was considered unresectable at that time. We left that office feeling defeated and terrified, unsure where to turn. Even though our local oncologist warned us that there was a lot of misinformation on the Internet, in desperation I began to do research and found the PMP Research Foundation and a group called PMP Pals. From that group I was turned to a grass roots support group on Facebook that is closed to the public, but exists for PMP Appendix Cancer patients and their caregivers for emotional support and assistance. This group, the PMP Appendix Cancer Support Group, literally to date, has saved my husband's life and set us on a course seeking survivorship for Paul, and seeking treatment at the highest level of care for my husband, instead of staying rooted in our local area. That was a very scary decision for us, but one we are very glad we have been able to make. After being told my husband's tumor was inoperable he began his local chemotherapy in the city near where we live. I felt defeated as his advocate that I had not been able to connect my husband to a higher level of care or to find a better solution for him in his battle with this rare and often life limiting cancer other than off the shelf systemic chemotherapy that is really intended as a treatment for colon cancer, which is a different form of cancer entirely. Appendiceal cancer does not even have its own chemotherapy developed to date. As a rare, (older stats say it's a one in a million cancer), very very little research has historically been done on PMP or its more aggressive cousin, adenocarcinoma of the appendix. 

I made dozens of calls to the large well known cancer centers, the ones our insurance calls the "Centers of Excellence", only to be turned away and told that they had no specialists in Appendix Cancer, PMP at those facilities. The American Cancer Society had only sparse info and proved to be another blind alley. But the grass roots support groups on Facebook, the PMP Appendix Cancer Support Group and the Pseudomyxoma Survivor.org, PMP Research sites shared information and offered us direct support on many levels and helped us begin to have a deeper understanding of what medical options were out there for Paul. These FB closed groups (for increased confidentiality) also provided me as Paul's caregiver, support and guidance so that I could go forward as his seeker in confidence that we were on the right path. We learned that there are expert surgical oncologists in large cities that had proven track records doing hundreds, and in some cases thousands, of a special kind of surgery called Cytoreductive Surgery (CRS) with HIPEC, Hyperthermic Intraperitoneal Chemotherapy, a method developed by a famous surgeon, Paul Sugarbaker, MD in Washington, DC at the Medstar Hospital. Sometimes referred to as the "Shake and Bake" surgery named after Dr. Sugarbaker, this surgery offered an extension of my husband's life. CRS HIPEC refers to a surgeon opening a PMP Appendix cancer patient with an incision from stem to stern midline and methodically removing all visible tumor seedlings inside a person's body cavity from where it has spread stemming from a few malignant cells that form a neoplasm (tumor) that begins in the appendix. As these malignant cells multiply they burst the soft walled appendix and leak out into the patient's abdominal cavity spreading and attaching to the surface of many organs. The nature for many of these tumors is to have a gelatinous sticky mucinous texture. Thus the term "jelly belly" was coined in the past to describe PMP Appendix cancer. Within the general term, PMP,  Appendix Cancers represent a handful or more of actual subsets of types of PMP Appendix cancerous tumors. Some, like most PMP tumors, are slower growing and mucinous in texture. Others, like my husband's tumor,are fast growing and aggressive and not mucinous. Signet ring, goblet cell and endocrine based tumors like carcinoid tumors are all other kinds of PMP Appendix Cancer tumors, we learned. In fact, we learned a lot during those first weeks of exploration, post diagnosis, both on the Internet and by making phone calls. We learned that pathology and histology of the tumor is key to predicting outcome and that the completeness of the CRS HIPEC is an indicator of overall progression free survival. 

By being an aggressive advocate I was able to set up a consult with a world renowned surgical oncologist in July of 2012. My husband stayed on his FOLFOX regimen for 7 cycles in hopes of reducing his tumor burden in advance of the surgery slated for December of 2012. Paul underwent his first CRS HIPEC that December in 2012 and had a 10 hour long incomplete surgery. He lost many organs or parts of organs to that surgery, including his appendix, cecum, omentum, gallbladder and sections of his large and small bowel. The kind of surgery that he underwent at that time is sometimes called a right hemi-colectomy. People can live with a good quality of life without a lot of the organs that we are born with. The surgeon stated that there was an additional area in Paul's pelvis that could not be reached during that first big surgery and that a second surgery would need to be scheduled for six months after Paul was able to recover. Adjuvant chemotherapy was administered to Paul as he recovered with no complications over that Winter and Spring of 2013. We made the painful decision in 2013 to retire.  Even though that was a hard decision, it was the right one for us, given Paul's state of health. Paul's second surgery in June of 2013 repaired mesh that had been left by three previous inguinal hernias. We learned that for some appendix cancer patients, having multiple hernias can be a red flag that something is wrong in the peritoneal cavity that is stressing the integrity of the abdomen. In my husband's case it was this tumor that had been silently growing for possibly as long as five years before it was found by CT. Throughout 2013, Paul was carefully monitored by his surgical oncologist and local oncologist with CT scans every three months and blood work every few weeks. We had made a connection with a special medical oncologist on the west coast that did a specialized kind of tumor assay to better predict the hopeful outcome of potential chemotherapies that might have a better response (killing cancer cells) to Paul's individual tumor. 

During these many months, Paul continued to marshall his energy and to enjoy most all of his regular daily life. Fishing, boating and ice fishing are some of his favorite pastimes and for me as his wife and advocate, being able to watch him engage in the things he loves with our young adult children was my inspiration to continue to push for a vigilant standard of medical care at the very highest levels. Then in late winter of 2014, March, Paul experienced a sudden bowel obstruction. He was in a lot of pain and the local hospital and his local oncologist could offer no solutions. We traveled in an emergency mode to his surgical oncologist seven hours away, who performed a 5 hour surgery and found massive scar tissue, adhesions and recurrence of Paul's cancer. During this third major surgery Paul lost almost five feet of small intestine to alleviate the bowel obstruction, a common symptom of PMP Appendix cancer patients with adhesions/scar tissue, (a common consequence of any type of surgery,including CRS HIPEC). After Paul was diagnosed with recurrence of his tumor I began to explore clinical trials. This was in the spring of 2014. At this time I also made a connection with Marcia Horn at ICAN, the International Cancer Advocacy Network. Marcia was a true Godsend to us. She provided us with a wealth of information and resources including how to look for a clinical trial. Having Marcia and ICAN as a part of our care team for Paul made us feel connected to what were emerging treatment options and the very best and most hopeful care providers, clinical trials, and the best that science had to offer. Marcia said to us in our first phone call, "ICAN and I will never give up on Paul!" Her confidence and caring manner made us feel we were not alone! Armed with ICAN's newly shared resources, I began to make a volley of phone calls and using the www.clinicaltrials.gov site, in the late summer of 2014 I found a clinical trial that would be starting enrollment in 2015 that seemed very hopeful. This trial was not about more or novel regimens of chemotherapy. By this time Paul had endured more than 25 rounds of three regimens of chemo and while some had proven very helpful in shrinking and softening his tumor to aid the surgeons in removing the tumor, other regimens had only made his quality of life and the condition of his health very poor. The trial we were interested in was based on immunotherapy. This very word for us conjured up notions of future-science. We learned that one of the ways cancer proliferates is because the immune system (T cells, white blood cells that protect us from invaders like viruses and bacteria) can't recognize, (see) tumors. The tumor cells literally have a cloak of invisibility that shields them from the little soldiers of our immune system. Another foundational condition of cancer in general is the presence of inflammation in the body. The trial we were interested in attempts to stop the PMP Appendix tumor by stripping away its shield at the cellular level so that the T cells can see the cancer cells and go after them. This trial also attempts to ramp up the patient's own immune system using drugs that have long been approved by the FDA in immune boosting and reducing inflammation in the body but in this case, using those FDA approved drugs in new ways to fight the PMP cancer. The trial was a Phase 1 and 2 trial--with the goal of the first phase to establish a standard dose of the medicines that would be administered. The goal of the second phase of the trial was to continue with the immune therapy to see if this combination of medicines could have a positive effect in overall progression-free survival for the trial participants. 

My husband is only 54 years old. We have two young adult children who we love dearly and desire to live long enough to see them married someday. Paul has a lot of motivation to want to live with the best quality of life he can have, for as long as he can. In the late summer of 2014 we consulted with the surgical oncologist at this large medical center and Paul was tentatively accepted into the program as a trial candidate into the Phase I and 2 trials.  In the late Fall of 2014 Paul underwent a second CRS HIPEC that was 12 hours long to remove tumor down to the 1 mm size. The heated chemotherapy administered through closed perfusion would hopefully kill any remaining cancer cells. Tumor cells taken from this surgery were added to Paul's T cells taken though as process known as Leukophoresis prior to the surgery and added together to create a vaccine that would later be given to Paul in clinic over the course of a 27 week trial. Paul entered the trial after a four week healing period and to date has completed the first half of this clinical trial. Other than feeling fatigued some weeks when in the clinic he has experienced no significant side effects from the trial. It is too soon to know if this clinical trial is helping Paul or not. At this time Paul is grateful to be a part of the trial that may in fact help his overall survival and may also help other patients in the future. Targeted therapies based on molecular profiling, immunotherapies, and customized cancer care is, Paul and I believe, the pathway for a cure for patients with PMP Appendix cancer. We pray for a not too distant future where his kind of cancer becomes adopted by more research facilities and more hopeful treatments can be developed. In the interim, CRS HIPEC has proven to be the standard of care for PMP Patients under an expert surgical oncologist. These pioneer surgeons are breaking ground in training new surgeons in this complex and specialized technique and traveling to other countries to share this method of battling not only PMP Appendix Cancers but other forms of abdominal and thoracic cancers. 

While my husband has endured four major surgeries and over two dozen rounds of standard chemo and now, is part of a clinical trial and his long term future is not yet known, he has had the past 32 months of life to be with our kids and catch fish and walk in the woods most days with his much beloved black Labrador, Bear. For today that is good enough for him. We remain resolved to fight for year three post diagnosis and for every day after that. As Stuart Scott, of ESPN, who recently passed of his PMP Appendix cancer after a long fight of many years said in his speech at the ESPY Awards, "You beat cancer by how you live, why you live, and in the manner in which you live." His powerful words push us on in our fight. Paul's years since his diagnosis have inspired in us a drive to give back and to pay it forward to others that are recently diagnosed. We live in a state of quiet hopefulness, and we make time to connect with others who fight this same fight to offer support and kindness. We have been offered so much kindness and help though this battle. From our oncologist locally, to the surgical oncologists in two major medical centers, to our, friend and advocate, Laurie Johnson-Todd, "The Insurance Warrior" and insurance strategist who wrote our appeal to fight our health insurance provider and won our appeal quickly and resoundingly, making it financially possible for Paul to have his first surgery (when it would otherwise been deemed out of network).To the ICAN team and Marcia Horn who continue to provide us so much support and access to resources and our Clinical Trial team. To the many hundreds of fellow patients and caregivers who have opened their hearts to us and offered emotional support, advice, love and prayers on our PMP support groups, we thank you all and are so grateful we have found you. 

Cancer is a raw, cruel, unrelenting and unforgiving disease, but we have also seen, over the past 32 months, the very best in people, our friends and family, our support group network of family, selflessness, kindness and love, even from strangers who become friends and a kind of extended family, though our journey. We thank each one of those people from the bottom of our hearts. You can live, with a good quality of life with Pseudomyxoma Peritonei, Appendix cancer if you have the right people by your side and on your team!

Cindy and Paul, 12:00 noon, February 17th, 2015