Last month while in a small plane over Belize, I received this email.

Tony:

I’m sorry for reaching out to you if this is inappropriate, but I thought you might have some experience or knowledge that I could tap to help fight a denial of insurance coverage for my current maintenance plan.

As you know, I’m a high risk patient and Dr. [redacted] has recommended aggressive post-transplant treatment with Kyprolis, Pomalyst, and dex (KPD). My insurance administrator, [redacted], denied coverage on the basis of “medical necessity” arguing that combination therapy is not supported for maintenance in the NCCN. We’ve processed a Level 1 appeal and provided documentation regarding combination therapy for high risk patients, but I receive word this morning the appeal was denied.

I feel like I need to do more than just say, “the specialists at [redacted] know more than the general oncologist at [redacted] reviewing this.” Do you have any suggestions, any research, anything that might assist me?

Thank you.

MM-8

MM-8 and I have a few things in common: middle age, gender, long term residency in the Pacific Northwest, myeloma, and unique numbers assigned to us by All4Cure’s Knowledge Sharing Platform, where myeloma patients allow their detailed medical histories to be viewed by participating clinicians, researchers and other patients.

MM-8’s initial appeal letter and a supportive letter from his nurse practitioner laid out the key points: 1) He has high risk disease as evidenced by the presence of a chromosome 17p deletion; 2) He was initially treated with revlimid, velcade, and dexamethasone (RVD) in November 2015, which didn’t work. 3) He was then switched to KPD which did work, and proceeded to an autologous stem cell transplant (SCT) in July 2016; 4) He resumed KPD as maintenance therapy in September 2016, which has continued to keep his myeloma at bay; 5) The supportive letter also cited a paper by Nooka and colleagues in which high response rates and prolonged progression free survival were observed in 45 high risk myeloma patients treated with a three drug maintenance regimen (RVD) compared to historical results using a single drug or no maintenance therapy (Leukemia, 28:690–693, 2014). The use of this citation suggested a leap of inference: “Because one 3 drug regimen, RVD, produced good results as maintenance therapy in high risk myeloma, another 3 drug regimen, KPD, makes sense when RVD doesn’t work.” The insurance company’s denial of this first round of appeals suggested that they weren’t prepared to jump.

Terra Firma?

With an ever-increasing focus on value-based care, how can an insurance company tell when a clinical decision regarding expensive treatments has gone off the rails? If expert guidelines and the peer-reviewed literature are terra firma, then the alarm sounds when a patient’s treatment falls outside these consensus-defined boundaries.

All4Cure wrote a letter on MM-8’s behalf, which included a graph adapted from his All4Cure dashboard showing all of this treatments and responses. Figure 1 shows how MM-8’s treatments correlate with blood levels of a protein that originates from his myeloma. These “M-Spike” measurements provide a facile way of gauging treatment responses.

Figure 1 graphically depicts the points made previously: that MM-8’s initial treatment with lenalidomide (revlimid), bortezomib (velcade) and dexamethasone (RVD) was ineffective, that switching to carfilzomib (kyprolis), pomalidomide and dexamethasone (KPD) worked, and that resumption of KPD following his autologous SCT was associated with a further decline in his M-spike to undetectable levels. Switching from an effective treatment to something unproven in MM-8 was hugely undesirable.

We also provided information from another patient enrolled in All4Cure’s Platform (Unique ID number MM-77) who, like MM-8, is also receiving KPD as maintenance therapy following an autologous SCT. As can be seen in Figure 2, KPD has yielded a further decline in M-spike levels for MM-77 following autologous SCT, as was seen for MM-8.

Does MM-77’s experience strengthen the case for MM-8? What should determine whether a particular regimen is used in a particular patient? Instead of grounding decisions in consensus guidelines, what if we could ground them in our collective experience, or “experiencia firma”? Results assembled across dozens or hundreds of patients receiving the same treatment would provide an incredibly potent resource for determining which regimens work best in the real world. Combined with measurements of cost, experiencia firma would provide a common foundation upon which both clinical and reimbursement decisions could be based.

Might MM-8’s appeal have been even more compelling were it possible to share the experiences of dozens or hundreds of similar patients? Of course more data is better, but in this case one was enough — his appeal was granted.

Cozy, but not quite the digs to which I’d grown accustomed at the University of Washington. Having requested a leave of absence from my day job as a Professor of Medicine to focus on my startup, All4Cure, I’ve been advised that the ethics rules for Washington State employees preclude any use of public resources for my private company. That includes my beautiful 5th floor lab, overlooking the Allen Institute, the Fred Hutchinson Cancer Research Center and beautiful South Lake Union, dotted with shells crewed by UW teams and seaplanes departing for the San Juans. Here’s a picture taken just outside my lab, of me flanked by two former trainees, Zhijun Duan, PhD (now Associate Professor at UW) and Hui Zeng, MD, PhD (now Professor at the Institute of Infectious Diseases of the Capital Medical University in Beijing). This picture was taken in September 2015, just ahead of my first stem cell transplant.

I began All4Cure between two stem cell transplants. The first took place in October 2015 and was “autologous”, meaning that stem cells were taken from and later put back into me. A few months later, I had a second stem cell transplant that was “allogeneic”, meaning that the stem cells were taken from someone else — namely my brother Scott. Although known throughout adult life by his first name Matt, those of us that were there in the beginning still call him by his childhood name. A Methodist minister in Zanesville, Ohio, Scott likes to say that he infused me with the Holy Spirit.

Since being diagnosed with myeloma a little more than two years ago, I’ve discovered a few upsides to having cancer, including: #1 Instantaneous elevation to near sainthood status. Now I’m almost always spared the ire of friends and family, protected by an invisible shield that deflects blame to those around me. #2 Cancer is a supreme driver of focus. As an ever-present reminder that the future is not guaranteed, I am focused on make a lasting contribution to the world through All4Cure.

All4Cure is based on the premise that cancer patients will do better if they take control over their disease. Taking control means that they need to think of their cancer as a potentially solvable problem, understand cancer as an evolutionary process, and have access to the tools and expertise needed to help them achieve the best outcome possible. After enrolling thousands of patients we can learn from each other. As a patient I can compare the genetic profile of my tumor with that of thousands of other myeloma patients, find patients whose tumors are most closely matched to mine, learn which treatments worked for them, and have my own experience add to this ever improving body knowledge. Patients can make an enormous impact on the rate at which cancer treatments improve, and All4Cure can help them get there.

All4Cure is launching a Kickstarter campaign in September. A 4 minute draft video put together for our campaign is provided here.

Please let me know if you’d like to be contacted when our campaign launches. You can reach me at tony@all4cure.com.

The genomics revolution has revealed that cancer is more complex than previously imagined. Every cancer is unique, built upon a combination of genetic changes different from any other cancer that has ever existed or ever will exist. Compounding this complexity is that each of the >1 billion malignant cells that invade a cancer patient can acquire additional changes, some of which may promote treatment resistance, relapse and eventually, death. Although evolutionary theory is still disparaged in some quarters, all of us have witnessed this display of evolution in loved ones lost to cancer, and these countless personal experiences affirm that evolution is a formidable opponent.

In October 2013, dozens of my colleagues, my wife (a community oncologist specializing in breast cancer), and I began a clinical trial for patients with an exceptionally aggressive form of breast cancer. In our trial we extensively characterize the molecular features of our patients’ tumors, enlist experts from diverse scientific disciplines to interpret findings and predict effective therapies, and allow these predictions to be tested in the patients themselves, learning from their outcomes to help future patients.

Deploying the same infrastructure I have transitioned from investigator to a subject of investigation. We found that a drug developed for some forms of leukemia might work for me, and though we won’t act on this information now, it’s empowering to think several steps ahead.

Today, I am 18 days into an allogeneic stem cell transplant I received from the bone marrow of my younger brother, Scott.

My cancer is called myeloma, and in a twist of fate, it’s a disease I have become well acquainted with in my past twenty years as a doctor and scientist.

Multiple Myeloma: A cancer of plasma cells, a type of white blood cell normally responsible for producing antibodies.

I am a hematologist/oncologist at the University of Washington, where I have specialized in bone marrow transplantation for blood disorders, leukemia, lymphoma, multiple myeloma, and myelodysplastic syndrome.

In the past few years, I’ve shifted gears into the individualized role of cancer care. I have been examining my own experiences as a doctor, scientist, and now as a patient to re-define what it means to “do our best” for patients with advanced cancer, focusing on a type of breast cancer called “triple negative” breast cancer.”

To learn more about what I’m working on, see the video of my TEDxTacoma talk below.

Back to the myeloma. I have been fortunate enough to hear opinions from some of the smartest myeloma experts in the country about how to approach my condition.

Many of these experts think that an allogeneic stem cell transplant for my condition is a bad idea.

Allogeneic Transplant: A procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor. This is often a sister or brother, but could be an unrelated donor.

But I’m resigned to the reality that almost no matter what decision I take, “some expert somewhere” will be convinced that I’m messing things up.

In the following posts I will attempt to describe how I came to this decision and how I am trying to tackle my disease.

I fervently believe that the sharing of information between patients, researchers and clinicians represents an incredibly important way of advancing our collective ability to understand and treat cancer.

I hope that other cancer patients will find some of the concepts and specific information provided here to be useful in thinking about their own situations. In fact, I fervently believe that the sharing of information between patients, researchers and clinicians represents an incredibly important way of advancing our collective ability to understand and treat cancer.

But first, let me begin at the beginning of my myeloma.

January 2015 — First signs

I began to notice some left hip pain and back pain at the beginning of 2015, but I attributed it to a new, firmer mattress.

Returning to the previous mattress didn’t help, and in the following weeks my hip pain became pretty prominent with walking.

Now there were aspects of this “hip pain” that didn’t make sense. It didn’t actually hurt when I moved my hip, rather the pain was in the left anterior superior iliac spine, a region which, when struck by an opponent’s football helmet, causes an injury called a “hip pointer”.

Another clue that this wasn’t ordinary hip pain was that my zingy “hip pointer” became unbearably painful with a cough or sneeze.

Over time it hurt more and more when I walked, but I eventually learned that Advil helped a lot, and so with more Advil than common sense I was able to keep going until my doctor, a terrific Seattle internist, Sam Warren, recommended a hip X ray.

That changed everything.

Thursday April 9, 2015–8:00am, Diagnosis

Only as I laid on the X-ray table that next day did it occur to me that this might be a little more complicated than degenerative hip disease.

The X-ray technician left the room, the machine clicked, and an instant digital rendering of my hip appeared on a screen to my left.

In medical school they teach you to compare the left and right sides of an X-ray to look for symmetry. I thought my hips looked okay, but as I leaned back to do the symmetry check, my eyes transfixed on an enormous flattened egg shaped hole with ragged edges in my left iliac wing (on the right of the screen below). This monster threatened to break through the outer rim of my cortex, with only a tiny margin of ratty looking bone remaining.

Within 10 seconds of “the click”, I knew I had cancer.

3:30pm

It was a brilliant, sunny afternoon in Seattle.

I called Sam Warren and told him that I was sure that he would be getting a call from the radiologist telling him that I have cancer, and he called back a few minutes later telling me that the radiologist thought that I had some type of metastatic cancer (i.e. cancer that had spread, i.e. bad).

I was due to pick up my 10-year-old daughter Melisa from choir practice at 6:30 pm and so rather than going back to the lab, I stopped in a coffee shop in Capitol Hill. I tried to call Sibel but she was swamped with patients and couldn’t answer. My mom happened to be in Sibel’s clinic for followup of her own breast cancer, and when she answered Sibel’s phone I told her that there was nothing urgent and we could talk later.

Why was I in such a hurry to screw up everyone’s life?

6:30pm

I picked up Melisa and we went to a restaurant at her favorite dining spot — Seattle’s University Village, where we had a magical dinner (followed by ice cream) before driving home to our new collective reality.

9:30pm

By the time we got home Sibel and my mom had just arrived themselves, and I immediately took Sibel upstairs to our bedroom and told her what had happened. A few minutes later we told Melisa and mom and called our older kids Benny (27), Manya (22) and Elif (20).

Tears, disbelief, tears.

10:30 pm

Sibel called two of our friends — Jeni Spokely the coordinator for Sibel’s clinic in Puyallup, and her partner and fellow hematologist-oncologist, Andrea Veatch, who immediately agreed to meet us at her office at 7:30 the next morning for a bone marrow biopsy.

Bone Marrow Biopsy: The removal and examination of marrow from inside bone. Bone marrow is the soft tissue inside bones that helps form blood cells. It is found in the hollow part of most bones.

Without knowing which type of cancer I had, it was entirely plausible that I might not survive more than a few months.

Sibel examined me and thought my liver was enlarged — suggesting liver mets, further adding to the sense that my time had likely come.

That night was surreal.

As described in a TEDx talk I gave in 2014, “Cancer feels like a punch, that leaves you breathless.”

April 10, 7:30pm

The next morning, Sibel and I arrived in her office in Puyallup for a bone marrow biopsy, where we were greeted by a throng of friends and coworkers, all in tears.

Within a few minutes, Andrea placed an 18 gauge needle in the same side of my pelvis that contained the tumor, hoping to draw out the cells that would provide a diagnosis.

That was followed by lots of tubes of blood taken by the best phlebotomist I have ever encountered — another “Tony”, known for his skill, demeanor and ridiculously contagious laugh. Next we wanted to assess the metabolic activity of my pelvic lesion and look for tumor elsewhere, and this required a test called a PET-CT.

Getting a PET CT typically requires several days of advanced planning for insurance approval and to prepare the radio-labeled glucose.

Being the miracle worker that she is, Sibel got phone approval from the insurance company within the hour and Tacoma radiologist Dr. Joe Sam tracked down a supply of radioisotope, completing my PET CT by noon.

Joe told us to grab lunch and he would be ready for us when we returned. When we came back he told us that he was virtually certain that I had myeloma.

It was the best news we could have possibly hoped for.

On the drive back home we learned that I had an IgA kappa myeloma. Once we got home Sibel called our dear friend and colleague, Pam Becker, a fellow hematologist oncologist at the Seattle Cancer Care Alliance. Pam epitomizes the concept that appearances can be deceptive.

Although mild in demeanor, Pam Becker is a tiger. When Sibel told Pam that I had myeloma, Pam responded incredulously “My Tony?”

That evening she arrived at our house with her husband, Marc Stewart, medical director at the Seattle Cancer Care Alliance, with a bottle of red wine and a Boston Creme pie. That night Pam repeated her mantra that I would be OK, that I would get through this.

As will be apparent here and in the posts to follow, a recurring theme of my experience with myeloma is that I have benefitted enormously from the very best expertise and care the world can provide.

Through much better-than-average connections, income and knowledge, I have enjoyed advantages available to very, very few other cancer patients.

While this circumstance is certainly unfair, and I can’t yet say whether improved access and resources will lead to an improved outcome, I hope that this blog can play a small role in helping to level the knowledge playing field.

While many renown experts for whom I have the deepest respect will disagree with some of my choices; there are few unambiguously right choices, I hope this blog launches a discussion that is educational for all.