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Arnie’s Rebounding World: Is The Multiple Myeloma Treatment Glass Half Full Or Half Empty?

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Published: Mar 11, 2014 2:56 pm

Hardly a day goes by when I am not inundated with the news of incredible advances that have been made in the treatment of multiple myeloma.

Survival rates have gone from three years to five years and now to eight to ten years.

Five new drugs have been approved for the treatment of myeloma over the last decade.  A couple of dozen more are in various stages of clinical trials.

We are tantalized by the promises of “breakthroughs” just on the horizon.

Immunotherapy hopes to harness the body’s own immune system to fight myeloma.  Genomics holds the potential of uncovering the genetic mutations that trigger multiple myeloma, allowing researchers to design drugs that specifically target these mutations.

I suspect that as exciting as these things are, they are going to be much more complex and take much longer to work out than we are often led to believe.

The history of cancer research has been littered with broken promises and unmet expectations.  In the early 1970s, President Richard Nixon declared a war on cancer.  Yet, here we stand over 40 years later.

To be sure, there is much to be thankful for.  For example, I just reached the eight-year anniversary of my diagnosis.

There is no doubt that I would not be alive today without the advances that have been made.  But is it good enough?

I am refractory to all of the U.S. Food and Drug Administration (FDA)-approved drugs and have exhausted all the standard treatments.

Multiple myeloma is still considered an “incurable disease.”

So, is the glass half full or half empty?  It depends on how you look at it.

I recently had the pleasure of attending a wonderful dinner for multiple myeloma patients.  It was organized by fellow Myeloma Beacon columnist Pat Killingsworth.

Each person got up and briefly told their “myeloma story.”

Now, granted, this was a self-selected group that may not have been completely representative, but I was struck by the wide spectrum of stories and how well most of the people were doing.

One woman had been on just thalidomide (Thalomid) for 10 years and was doing fine.  Many had achieved complete responses and were stable on nothing or their current regimens.

For many of them, it might seem as though the myeloma treatment glass is half full.

Very few seemed to be in my situation with advanced, aggressive disease.  I began to think that these people don’t even have the same disease as me.

It is apparent just from reading the various other Myeloma Beacon columns how varied the disease course and response to treatment is.

Herein lies part of the problem.  Multiple myeloma is really a wide spectrum of diseases with different behaviors.  How one views the current state of myeloma treatment depends on where you are on that spectrum.

“Treatable but not curable” is how multiple myeloma is described.  But “treatable but not curable” can look like many different things.

Toenail fungus is treatable but not curable.  Diabetes is treatable but not curable.

But relapse is still the norm.  Even those who are stable and doing well have the constant specter of knowing that the disease can become refractory and turn nasty and aggressive at any time.

So what should we all make of the current state of multiple myeloma drug development and treatment?

The pace of drug development has been more rapid than in many cancers, and some of the improvements have been impressive.  But the advances are incremental. There has not been a ground-breaking, game-changing drug.  There is too much emphasis on modifying existing drugs, which results in drugs with new names and modest improvements.

There is, however, a promising pipeline of new categories of drugs with different mechanisms of action.

The monoclonal antibodies are on the forefront of this list.  Elotuzumab and daratumumab are leading the way, and at least one will likely be FDA-approved in the next year or two.

Drugs that target other pathways such as KSP inhibitors and HDAC inhibitors, among others, are in clinical trials and are showing promise, but again, they are not necessarily game changing.

Clinical trials are still too limiting, cumbersome, and time consuming.  Overcoming drug resistance is still a big issue.  The complexity and heterogeneity of the different multiple myeloma subtypes and which drugs work best for a given situation is likely many years away from being resolved.

Many try to just stay alive until the next drug.  But that next drug may not come fast enough.

We have come a long way, and that’s good.  Is it good enough?  It depends on where you are.

Is the myeloma treatment glass half full or half empty?

Arnold Goodman is a multiple myeloma patient and columnist at The Myeloma Beacon. You can view a list of his columns here.

If you are interested in writing a regular column to be published by The Myeloma Beacon, please contact the Beacon team at .

Photo of Dr. Arnold Goodman, monthly columnist at The Myeloma Beacon.
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15 Comments »

  • LibbyC said:

    Hi Arnie,
    It's always a good topic - Is the glass half full or half empty? It depends on what glasses the person has on when looking at the glass. Obviously the person wearing the old rose tinted glasses are going to see it half full. A scientist will see it as completely full - 1/2 liquid the other 1/2 gas. A representative of a drug company with no real understanding of the complexity of myeloma will see it as overflowing - "there have been so many advances"...

    At my diagnosis I was told that it was terminal but it could be delayed with treatment. As it became clear that my myeloma was chemo refractory - even to melphalan - my existence became dependent on the availability of "new" drugs. But, like you, they were not developed fast enough to continually keep the myeloma under control. I am extremely lucky that my allograft worked.

    If I put "Cynical Negative Nanny's" glasses on the glass is definitely half empty (somebody has probably taken a drink from it as well). This disease is so heterogeneous the mind boggles. New drugs will be developed that will be able to target the wider myeloma community - this is great. However, I can't see any research being put into developing drugs that specifically target a mutation that is present in only say 3% of myeloma patients. Using the new drug will delay the inevitable but the myeloma patient then becomes refractory to that drug.

    How much research is going towards understanding the myeloma cells from chemo refractory patients? What genes are involved? Who would pay for the research? Where are the drugs that target these pathways? I don't know if clinical trials test the patients genomics but they should. I could go on & on.

    My hope is eventually there will be individualised targeted therapy - I think there needs to be.

  • Nancy Shamanna said:

    Hi Arnie, thanks so much for the detailed column about the treatments available, and wondering if they are enough to keep the glass half full. I know it is a lot better than it used to be for myeloma patients, but we still have a long way to go before any patients can feel confident of having that elusive 'cure'. I hope that one of the monoclonal antibodies is approved soon in the US. That will at least let relapsed patients try the new type of therapy, and get the ball rolling for international approvals too.

    Wishing you and your family all the best and hope you can catch a break with treatments too!

  • Thomas said:

    Dear Dr. Goodman, the answer for me is clear: As long as myeloma is a disease that could kill (the word sounds cruel and it is) we should look at the glass as half empty. The rhetoric of cure sometimes overlay that we are not there yet. Optimism and hope is always good, but it should not hide the fact, that many of us patients will come or are in urgent situations, where better structures, agents and support is needed. No one should be left behind! Best regards and thank you for your inspiring columns. Thomas

  • Randy Strode said:

    I read an article recently in the Beacon from one of the leading myeloma doctors from the Arkansas treatment center. He brought up some of the same issues as you. Do you treat multiple myeloma patients like a chronic long term illness or should you pull out all the stops and attempt for a "cure". His approach is usually blast the heck out of it with everything you got while you can. I haven't reached that point yet in my treatment plan, however reading everything in sight is starting to help me form an opinion of our plan of attack.

    Hope you find that illusive treatment so you can get things back under somewhat of a controlled nature. And bring on the next round of drugs or treatments to keep us all alive until we do find that "cure". In the meantime, keep fighting, keep searching, and keep sharing your journey with others!

  • Jan Stafl MD said:

    Hello Arnie, thank you again for another thoughtful article. I am at at a similar point of myeloma treatment as you, having exhausted all major FDA approved MM meds. I have not had an allo transplant that you had last year, but have found an actionable mutation through genomics, BRAF. For the last four months, my BRAF inhibitor Zelboraf (approved for metastatic melanoma), with weekly dex and occassional IV Zometa have resulted in normal kappa free light chains, with a PET/CT scan confirming near resolution of previous multiple lesions.

    This therapy is only applicable in the 3% of MM pts. with this high risk mutation, but there are several other genomic based therapies available, and many more in development. So in high risk recurrent MM, genomic analysis can be helpful. Has that analysis given you any other options?

    This not a cure; no one knows how long a near complete remission will last, and there are dermatological and other side effects. But it is the best option for me. A MEK inhibitor may be added if needed. I am quite hesitant to consider an allo transplant. So I am a glass half full kind of guy, and I love life to the fullest while I can.

    Epigenetic effects on gene expression are always possible, not only with meds and supplements, but with exercise, mind body therapies, and energy medicine. Meditation and prayer (Thy Will be done) are helpful. Ultimately, surrendering to what is may be the best we can do. None of us are in control, but we can affect how we deal with our challenges, with the help of our loved ones. I wish you and yours deep healing.

  • Mike F. said:

    Thanks for another good column, Arnie.

    Being someone who was diagnosed two years ago at the age of 53, I find the glass still half empty. A ten year median survival is pretty good if you're 75, but not so great if you're 53. Being otherwise extremely healthy, I was planning on a long and active retirement. Knowing that I may be dead or incapacitated by treatment by the time I hit that age is not a good thing.

    On the other hand, I am extremely hopeful and consider myself among the more fortunate of the unfortunate. So far, the disease has responded well to an auto-SCT and Revlimid. Every month that goes by brings us closer to new forms of treatment. Every year that goes by brings new research on everything from immunotherapies to mechanisms of myeloma immunity to drugs. There is reason to believe that if I can make it another eight years, then treatment protocols will have progressed to the point that the disease can be kept permanently at bay with few side effects.

    As you say, though, it can take a long time to find treatments that work and much of what looks promising now may never pan out. So I see the glass as half empty, but it wouldn't take a lot to get it just past half full.

  • Steve said:

    "The pace of drug development has been more rapid than in many cancers, and some of the improvements have been impressive. But the advances are incremental. There has not been a ground-breaking, game-changing drug. There is too much emphasis on modifying existing drugs, which results in drugs with new names and modest improvements"

    Doctor, you raise an interesting point there, as one has to wonder that from a "business model" perspective, with comparatively so few MM patients vis a vis lung cancer, prostate, breast cancer etc., whether reserarch dollars, either private or public sector, will be dedicated to new MM drugs that are radically new in treatment pathways. If there is to be a cure, absolute or functional in nature, my guess is that it will derive from some kind of "happy accident", if you will, and given the heterogeniety of MM, both within and without the patient, it's not likely, imho, that whatever drug therapy that may become a MM breakthrough, will probalby only successfuly treat a smallish fraction of all MM patients. So until such an event may occur, I'm thinking that continued MM research and medical discovery will proceed along at an incremental pace of development as sadly, that is probably seen by researchers as the most economically vialble strategy. I'm not sure if that's a half empty or half full perspective?

    That said, a few evenings ago I was watching a late night show on one of the cable channels, it was called something like, "Small Stocks, Big Dollars" and believe it or not, the host was encouraging viewers to invest in Pharma companies, and in particular, companies researching MM drugs! He continued on to say that the MM drug development market will become a 6 billion dollar market by the year 2018! Well that's great, of course, but how many of us have 4 years left on our clocks? Just saying.

    But of course let's not forget, we've still got that big ol' Black Swan honking it's way under a cloak of....well...under a cloak of Black Swaniness....to our rescue! :) When,how, you ask? Apparently no one can tell us because then they'd have to kill us and that would kinda defeat the whole purpose, now wouldn't it! ;)

    Hey, I kid His Swaniness! ;)

    But I'm not kiddin' 'bout this: I wanna new drug....

    http://tinyurl.com/k6e8gk8

  • Arnold Goodman (author) said:

    Thanks everyone for your comments. Good to hear people on both sides I'm with you Steve, I wanna new drug. We are all hanging on hoping for the next drug. Jan, I've been thinking of you and am so glad to hear that you are still responding well to the BRAF inhibitor. Unfortunately, how to manage some of the other know mutations has not been well worked out yet, and they are not really ready for prime time. But, to be sure, a great deal of work is being done. How long and how complicated is difficult to say. For now, we all just keep swinging, and hopefully we will hit something.

  • asaryden said:

    Hi Arnie and thank you for sharing your thoughts on another interesting topic. I find your topics to be so spot on! The Bucket list, the "how are you"-dilemma, how to get hold of drugs not yet approved and now half full/empty cup.

    I had my allogeneic transplant 18 months ago and for the first time since diagnosis 3 and a half years ago I'm in sCR (so never give up!). That should be a reason for a half full cup approach but being on 15 mg Revlimid and 20 mg betamethasone affects my mood significantly and it feels more like half empty too often.

    I often wonder why MM treatments do not aim at molecular remission? Surely there are a lot of evidence by now that that gives outstanding OS?

    My very best wishes to you Arnie,
    Åsa

  • Arnold Goodman (author) said:

    Asa, Thrilled to hear you are in CR. I know that we had our allo transplants at about the same time and I have not been quite as lucky. I would take that as a glass more than half full. Keep it up

  • lys2012 said:

    I'm naturally an "optimistic" person so this would mean I see the glass as half full. My MM is "standard" responded well to my Velcade-based treatment + stem cell transplant, and I'm back to doing "normal" things like working and traveling a bit.

    But the flip side. The half empty side. I was 30 when I got really sick, took two years to diganose me, so 32 it was officially called myeloma. Most of my friends have new babies, new families, but this is probably not going to happen for us. I was told I can hope for 10 years remission, but I will be in and out of treatment the rest of my life. So this is obviously a huge stress. Prepare for the worst but expect the best? I hope I live to see 40. I really need hope for a cure, not just the same meds in different formulations.

  • tiziano28 said:

    Hi,

    I like Lys’ definition: “same meds in different formulations”. We have to be realistic. After the improvements due to the so called new drugs: the "thalidomide, bortezomib and lenalidomide triplet”, pomalidomide and calfilzomib are not showing that superiority. Overall survival has not improved in the last decades, apart maybe of a few months. (Please recall the IMWG recent statement: "7 years average survival for the standard risk mm"). I had started thinking I will have always been above the average, but I have learnt I am usually below. But I also think statistics are too optimistic. Remember however that 7 years probably means 2 years of terminal cancer.

    MABs (monoclonal antybodies) have started to delude (said from the best haematologist of my country). Transplants are based on melphalan, a drug invented in the Soviet Union in the sixties of last century. Not all hospital and countries are able to find a good donor (if ever a perfect match!).

    Horrendous conditions for many of us and long term damages come from drugs and b.m. transplant(s). An old man facing neuropathy may say: “Doctor, I cannot fasten the buttons of my shirt”. A man of forty or fifty in the same condition: “Doctor, I had to leave my job cause I could not use my hands properly”.

    This is my picture. Too simplistic? Maybe. But for sure it is by far less strange than the systematic mystification about progresses in mm treatments. Millions of words, smiles and celebrations in front of how many months of improvements in OS? Big Pharma’s interests and Big Research’s interests are too strong. Rarely a trial about a new molecule go beyond phase 2 and after that you do not know anything about for years. Something that seemed really promising disappears from the oncology field. What is the end of JQ1 molecule? Too simple to be true (or too strong to be put on the established market?).

    My MGUS was discovered when I was a boy of 34. From my disease status I had the opportunity to understand in the years (not so many to be honest) that men can be heartless or can be angels. I have been the guinea pig of 2 tandem ASCTs. I understood that in a hospital you are LESS important of your protocol. I REALLY have heard on the web the SAME doctors speaking the SAME words after 5 or more years “huge change”, “bla, bla, bla”….

    Now I do not see so many months in my future and I am sure am not the only one and I really do not understand WHO THEY are talking TO when they say that the future of mm is a brilliant one. Just not to loose this message I am telling it to my nephews. But I am doubtful for them too.
    Tiz.

  • Pat Killingsworth said:

    WOW! This may be the best "pros and cons" explanation of multiple myeloma therapy I've ever read -- and I've read a lot about it! And I'm not just saying that because you gave me a "shout out" in your column, Arnie. So, so glad you are still alive, able to spend time with friends and family -- and your profound and helpful thoughts with us. Thank you!

  • Ann Kosa said:

    Multiple myeloma is new in our family, and my spouse, unfortunately, has one of the rare types: IgD. So we assume that there is going to be minimal research done on that particular type, given the rarity. But this thread is certainly helpful in terms of a better understanding of current therapies and hopes for the future, nonetheless.

  • Paul said:

    Half empty unfortunately. I am 43 with three young children and have just discovered the persistent upper back pain I have seen multiple doctors about over the last two years is MM.

    Two weeks after diagnosis, I was admitted to hospital with difficulty breathing following a wedge fracture of my T4 vertebra and partial collapse of my right lung due to impingement from a mass on one of my ribs. Radiation helped and I have started chemo now, but I have been told I will not be able to return to work until my vertebra is fixed, which, in turn, can't happen until after chemo and stem cell transplant, so a minimum of six months.

    I have no idea what my prognosis is. I don't know if I will get better or worse. I don't know if I will see my children grow up. I don't know if I will respond to treatment. I don't know if I will ever be able to return to work. I don't even know what form or stage of the disease I have.

    To cap it all off, I have just discovered, when I went to claim, that my provider had lapsed all of my policies held with them without notice. This included all of my income protection insurance and a third of my death and disability cover. So now, instead of coming to grips with my diagnosis and concentrating on understanding what is happening and doing the best I can to ensure the best outcome, I am fighting a dishonest financial services company in the hope of safeguarding my family's future if I don't make it.