By Kelly Cox

At the IMF, I run the Regional Community Workshop program and serve as a Regional Director of Support Groups. I frequently find myself travelling to patient and medical meetings across the country. My aim is to bring the IMF's mission directly to those affected by myeloma, as well as to the healthcare professionals who serve them.

In June 2011, I was attending the annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago, Illinois. While taking a short break from the IMF booth, I found myself walking the aisles of the convention hall. One of the booths I passed had no visitors, so I stopped to say a neighborly hello.

The DKMS Americas (dkmsamericas.org) representatives were at ASCO to promote their cause and to solicit potential bone marrow donors. The process was simple. A painless swab of the inside of my cheek and I was on my way to becoming part of DKMS's database. I didn't give it a second thought. I practically forgot this ever happened.

In August 2011, I received a call from the New York offices of DKMS. It seemed that I was a possible match for a woman in her early 20s who needed a transplant for acute aplastic anemia. The sample collected from me at ASCO matched all the preliminary selection criteria. Even at that point in time, the whole concept of being a donor remained pretty abstract for me.

Things started becoming a bit more "real" over the following 4-5 days when I started donating vials of blood for the next stage of the matching process. In total, 21 vials of my blood were collected at a local Los Angeles clinic. The next step could not take place in LA, so it was time for me to fly to the Alta Bates Medical Center in Berkeley, CA.

They were so nice to me. They took great care of me and my wife, Mary. My niece and Mary's best friend were with us for support. The day before the collection procedure, we walked around the Berkeley campus, had a sandwich and some gelato. It was a beautiful, sunny day. I felt so happy and so excited in anticipation of the next morning. I stayed up most of that night because I was too energized and giddy to go to sleep. The mere thought of what was to come gave me goose bumps.

At 7 a.m., as I was waking up from the anesthesia, my bone marrow was already aboard a plane heading for an unnamed destination on the East Coast where the recipient was waiting. 176 punctures in my back and pelvic bones yielded 1.5 liters of bone marrow, more than enough for the recipient's transplant. As I lay in the hospital bed recovering from the bone marrow "taps," watching the two pints of blood I had "banked" being re-infused into me, the magnitude of the experience began to sink in.

Now let me back up for a moment. I must confess that I read none of the materials given to me in preparation for the collection procedure. I just didn't want to know what I was getting myself into. In hindsight, maybe this was because I did not want to learn something that might have discouraged me from moving forward. I felt fully committed and I wanted nothing to stand in the way.

The only question I remember asking in advance was addressed to Dr. Morie A. Gertz of the Mayo Clinic in Rochester, MN.  Dr. Gertz is a member of the IMF's Scientific Advisory Board, a frequent faculty presenter at the IMF's patient education meetings, and an all-round great guy. He told me that a patient with acute aplastic anemia had a 95% chance of survival after a well-matched donor transplant. That was all I needed to hear.

Being a donor is a personal choice. While I certainly appreciated all the support I received from friends and family members throughout the process, I did not expect the outpouring of positive messages I received via my Facebook page, many of them from strangers or from people I had lost touch with over the years.

Oddly, the emotional impact of the entire experience hits home now, after the fact, more than it did before or during the donation process. I get choked up when I think of it and, especially, when I think of that young woman. I hope that the transplant was successful and that it has given her a fresh start. I catch myself wishing that she has a full and productive life and, whenever she is up for it, I hope that she will find a way to be of service to someone else who is in need of help.

The donation was anonymous. While I would love to meet my recipient, there is no telling if this will ever happen. In the meantime, I would want her to know a few things about me. I am 53 years old and I have spent most of my life trying to be of service, trying to make this world a better place. I work for a wonderful non-profit organization, alongside colleagues who spend each and every day making the lives of others better and healthier. I have a newfound empathy and admiration for the myeloma patients I work with who undergo transplantation as part of their cancer therapy.

I've been told I have a big heart and I really do I care about helping people. I feel honored and grateful to have had an opportunity to be a donor. If she ever needs it, I will be here for the same recipient again in the future. And I have gone on to donate blood and platelets through two additional organizations in hopes of helping more people.

Emotionally, spiritually, and intellectually I am absolutely blown away by the experience. And I am still struggling to wrap my mind around the concept of possibly saving a life. Without a doubt, this was one of the most important events and accomplishments of my life.

By Jim Omel, MD

All of us with myeloma were thrilled to get a resounding 11-0-1 vote in favor of carfilzomib at the FDA's Oncologic Drugs Advisory Committee (ODAC) meeting on Wednesday, June 20.  Prior to the meeting, the general mood was neutral at best for carfilzomib's chances, and many financial analysts even predicted a negative outcome.  The FDA had identified significant cardiac, pulmonary, and liver toxicities with the drug and clearly stated their doubts about carfilzomib's Risk-Benefit ratio in their briefing document.

When the FDA has strong sentiment for a new drug's approval they say so.  When they have doubts however, the agency calls in a panel of outside experts for advice.  ODAC is a standing FDA committee of 13 oncologists and biostatisticians, very few of which treat myeloma patients.  For every new drug application with an identified cancer indication, the committee also selects a patient representative (PR) with that specific cancer to join the committee for that meeting.  The FDA PR program involves about 125 individuals with a huge spectrum of first-hand disease experience.  There are two to three of us with myeloma who are PRs, and I was the one selected to represent our disease last Wednesday.

The meeting began with a presentation by Onyx Pharmaceuticals of their phase II trial results for relapsed and refractory myeloma patients and FDA official's enumeration of their significant concerns.  Committee members then asked pointed questions of the sponsor (Onyx), the tone of which sounded very negative.  Most of us in the room frankly had a sense that the drug would not be approved based on the sharp tenor and tone of the questions.  I badly wanted to comment on the drug's good qualities, but strict committee protocol only allowed sponsor questions at that point.

Following a scheduled break, nine members of the public were each allowed three minutes to comment on the value (or not) of the drug.  People from the DC area, the MMRF, and the IMF truly ruled the day and gave new breath to carfilzomib. From the IMF, Michael and Robin Tuohy and Diane Moran made exceptionally eloquent statements. The committee of breast cancer, prostate cancer, and childhood cancer experts saw firsthand that myeloma is NOT a disease of old peopleand other "typicals" they had been taught in residency.  From my vantage point at the table I watched them INTENTLY listen to all 9 speakers.  No one surreptitiously checked their cell phones, fidgeted with their ties, or yawned as if to say, "I've heard these meaningless anecdotal testimonials before."  They were mesmerized!

The formal meeting schedule finally allowed comments from panel members.  I quickly got the chairman's eye and indicated my desire to speak.  As he jotted my name I prepared my thoughts and waited my turn.  I used Dr. Durie's words and said that myeloma is "a sneaky disease."  When it appears to be stopped, its genetic expression changes and myeloma cells escape death (apoptosis) by utilizing metabolic "escape pathways."  Myeloma physicians need multiple drugs (like carfilzomib) to block multiple pathways to control this cancer.  Though carfilzomib will not cure myeloma, it will buy precious time to stay alive until research finally gives us a cure.  I made other comments which countered a panelist's earlier assertion that "only the best patients were pre-selected by this phase II trial by living 5 years."  I reminded him that many of us now live 5 years because of treatments developed since 2000.  I also pointed out that 85% of myeloma patients present with "standard risk" genetic profiles and 15% are classified as "high risk."  In the Onyx trial, fully 28% of the patients had high-risk cytogenetics.

Discussion around the table had a decidedly different tone after the 9 powerful public speakers, and I had great hope for a vote of approval.  Topics centered on the fact that various toxicities identified by the FDA would be expected in the heavily pre-treated patients of this trial.  When the time came we all cast our electric votes, and no one had access to anyone else's vote.   At last the tally appeared on the screen.  There it was:  11,0,1.  The one abstention was cast by a biostatistician who explained that he opposed trial design, not the drug.

A few of us shared broad smiles and later tight hugs.  The ODAC is only an advisory body and the FDA will make its own decision on approval or not.  By law (PDUFA) this decision must come by July 27 of this year.  I think the resounding 11-0 vote will heavily influence approval.  

Following the meeting, the FDA's official who organizes the PR program told me she "nearly cried" when the vote tally flashed on the screen and she considered the relief which all of us with myeloma felt.  Deb is a wonderful government official with warm humanity and empathy.

Next up is pomalidomide, an immunomodulatory drug (IMiD) with proven clinical activity in patients NOT responding to Revlimid® (lenalidomide).  It too is at the FDA right now awaiting approval based on a phase II trial.  A decision is not expected until late 2012 or early 2013.  I hope pomalidomide is so impressive that an ODAC evaluation will not even be necessary.  If it is however, bring it on!  Either I or another capable myeloma PR will gladly represent those of us with this horrible insidious cancer again.  With the IMF's help we WILL find a cure!

Last week I shared some hard-earned advice about how to deal with side effects caused by a variety of myeloma therapies.

My recommendations were broad and general. I hope they helped.

But I promised to share some more specific tips this week. What about the day-to-day side effects that many of us face once we begin myeloma therapy?

Inconvenient things like too much stomach acid, nausea, constipation and any number of significant side effects caused by the drug that we all love to hate: dexamethasone.

Speaking of that particular emotion, don't you hate when you begin reading an article touting ways to help you resolve any number of medically related issues, only to learn that it was so basic that it was a waste of time reading it? I don't want that to happen here!

So let's turn this topic into a multi-week discussion, shall we? That way we can really tackle some specifics.

This week I will cover complications from too much stomach acid. Next week nausea, followed by constipation and last but certainly not least, dexamethasone-related side effects.

Following my autologous (using my own cells) stem-cell transplant, I battled a long list of side effects for months: Fatigue, low -blood counts and nausea, to name just a few. But the longest lasting of these was excess stomach acid. 

Commonly a problem for a month or so while one's digestive system gets back on track following a stem cell transplant (SCT), mine persists today, 11 months later.

Although it wasn't ever diagnosed by a specialist, it is likely I suffered from auto graft/host disease. I didn't even know graft/host was an issue in auto patients.  But the most common area of concern for auto host/graft is the digestive tract, so my myeloma specialist feels it is likely that may have affected me.

For months I could hear and feel acid jetting out of control into my unprotected stomach. Not fun!

My doctors recommended I take Prilosec OTC--or a generic equivalent such as omeprazole magnesium--along with an over-the-counter antacid like Maalox.

A fellow transplant patient recommended the prescription drug, pantoprazole, which he felt was much stronger and worked miracles for him.

For some reason, my doctors weren't anxious to prescribe pantoprazole. I finally ended-up using omeprazole magnesium capsules because they were inexpensive and considered relatively safe. As a matter of fact, both my myeloma specialist and medical oncologist told me I could take it twice a day--morning and night--and at twice the recommended dose on the box.

Check with your doctor, but the twice-a-day approach helped get my acid rush under control. I still use omeprazole magnesium once each morning, a real life saver!

Another patient friend recommended taking a calcium supplement throughout the day. Another felt ginger helped her.

This topic quickly crosses over to include what I call a sour stomach and/or nausea. And these common side effects aren't reserved for those of us who have undergone a SCT. Dexamethasone can really mess with one's stomach, for example. So can VELCADE®.  Really? You probably have never noticed, since it is standard medical practice to include an anti-nausea drug in the same IV.

Since I would like to cover those issues in detail, let's continue our discussion about ways to improve treatment-related sour stomach and nausea next week.