Sep 262013

You can get the full news release from the Novartis website. See link at bottom of article

When I was diagnosed with sporadic inclusion body myositis (sIBM) in 1996, my doctor and I agreed that it was pointless to try any of the drugs available at that time as trials had shown them to be ineffective and experiences have shown that their side effects could be very serious. Once in a while new ideas would be brought forth, but I always evaluated them from two criteria: 1. Did their mode of action seem to make sense for this particular disease, based on what we knew about its behavior? 2. Had any studies been done that produced empirical data supporting their claims?

Of course there were numerous anecdotal testimonials but I discounted those since IBM is such a slow-moving disease and since our physical performance can vary so much over time due to psychological influences and general health. In other words, placebo effects could easily fool people into thinking a regimen was working. There was also the gender factor. Although I have yet to see any data confirming this, my own observation has been that females tend to be more likely to benefit from treatments that are not of any use to males. My theory is that hormones must play a role in the disease one way or another.

So when the new drug from Novartis (BYM338) was given breakthrough status by FDA, I took notice. From what I have read, the mode of action seems to make sense. They believe they have identified an enzyme that restricts the development of new muscle cells and that it is an overabundance of that enzyme causing IBM patients to lose strength. BYM338 blocks the action of that enzyme, allowing muscle cells to reproduce unchecked. The drug has been through two stages of clinical trials including one that showed significant benefits when compared to a placebo. Now they are launching phase 3 which will be a much larger trial. Since the drug has received breakthrough status, a smaller proportion of trial participants will be receiving placebo, so if you are in the trial, there is a very good chance you would be receiving the real thing.

Now for the disappointment. My doctor at the MDA clinic called the researchers on my behalf to see if I could get into the trial. However, due to my advanced stage of disease, I am non ambulatory, and that is an exclusion from the trial. The reason is that they use the 6 minute walk test as a method of determining drug effectiveness. This is a standard test used in many different types of illnesses and thus it would be very difficult to persuade them not to use it. On the other hand, since one of the reasons they got Breakthrough Designation is that this illness is life-threatening in its later stages, I think it is a little unfair that they do not include a trial that focuses on those of us who are at those late stages and may not have time to wait for general availability of the drug.

Follow this link to all the study details.

As always, I caution you to do your own research about any medical topic I discuss. I am not a doctor, even though, like most of us with this disease, I may have more knowledge about IBM than most physicians other than those who received their education after 1990 and are interested in neuromuscular illnesses.

Here is the link to the Novartis website which is the best place to start for gathering information about the medication.

  42 Responses to “Novartis BYM338 (bimagrumab) – Promise and Disappointment”


  2. Hi all
    Its interesting news I have IBM for at least 10years that I can look back on. I can be very unsteady on feet at times but as long as the terrain is flat with no nasty suprises i can walk for 30 min. I also live in Australia and would not know where to start to try and get accepted or tested for this program. It is most likley not even available to us

    • Hi Ron, I too have IBM and live in Australia, Sydney to be precise, I attend the neuromuscular clinic at the RPAH, and will be getting them to follow up on the BYM338 trial. Feel free to contact me if you’re interested, I have never met or heard of Anyone else with IBM locally.

  3. Maybe Royal north shore Hospital at St Leonard’s Sydney.Neurologist Dr Christina Liang who has taken over Dr Merrilee Needhams position when she left,both are Specialising in Myositis.I don’t know if its possible for you to go there?Im on a list hopefully for Novartis but they are not saying yet.I go next month for my three monthly appointment so hope to find out then more and maybe when ,I’ve been on list for quite a while.Good luck it all sounds great as long as we can keep our legs long enough!

  4. I have IBM ABOUT 10 YEARS,can still walk about 1/8 mile on flat surface,would like to here from others my email is, hope this drug is the real thing, its hard to find out and get to go on clinical trails,I hope our canadian grov. can look into this , for this being not a very common illness, it would be great to have little more support.thank you

  5. As a physician treating patients with degenerative diseases I share too often the disappointment associated with new treatment trials implementing “draconian” inclusion criteria. It appears that survival beyond a certain point, or deterioration beyond a certain mark prevents many of my patients from entering the study.
    For me, as a treating physician, rather than a scientist designing a study, it is painful. We always tell our patients that there is hope, that medicine takes big strides these days in finding treatments for rare illnesses and we are right. Unfortunately the too advanced patients don’t have any longer easy measurable parameters that could show favorale response on outcome scales. The scientists should develop more sensitive and measurable tools to assess the disease progression and/or response to treatment.
    For example, Alzheimer’s disease. In addition to using the cognitive scales, one can measure brain volumes on MRI’s and even more, the cerebrospinal fluid tau protein (neuronal death marker) and amyloid beta (the accumulation of which “chokes” the neurons).
    I believe that measurable parameters can be identified for each disease, thus allowing the scientist derive usefull information from each trial participant. This goes beyond the obvious, of allowing any patient to experiment a new drug, when scientific merit is demonstrated. Identifying acurate and measurable outcome markers beyond the clinical scales will allow the scientific community to collect useful data from each and every patient receiving experimental treatment and will open up such treatment to whoever interested, once standard precautionary measures are taken.

    • Thank you Dr. Katz, for such an informed reply. As you can imagine my friends and family bring up the very same questions. I think one problem is that because this is a relatively new disease, there have been very few longitudinal studies. Many doctors seem to presume that this is strictly a disease of mobility distress, whereas it actually has significant impact on respiration as it progresses. I just came upon a Norwegian study from Brain. (I have added the link to this reply). They took in 64 IBM patients carefully monitoring all their parameters, and followed them for 12 years. At the end of the study, 46 patients had died, and of those they were able to obtain more data about 15. There is lots of good information there among other things confirming the rate of progression of muscle weakness, and the fact that, for this particular population, IBM did not demonstrate any life shortening. However, there was a dramatic difference between IBM patients and the general population regarding cause of death. In their general population, 4.4% of deaths in the early eighties were due to respiratory conditions. But among the same age group with IBM, 28.3% died of respiratory failure.

      This is a strong indication that IBM begins affecting the respiratory muscles in its later stages. A few days ago I had a few pulmonary function tests which showed that my MIP was about 30% of normal and my MEP about 20%. (I am 72 years old, diagnosed in 1996.) It seems that this could be one fairly good late stage marker for people with advanced inclusion body myositis. I am sure there are many others.

      I encourage all of my readers to download the study as it is one of the first I have seen that tries to give a big picture view of the progress of this illness.

      • Good points. And it’s not just respiratory muscles that are affected. My Dad has developed other conditions such as Dysphagia as his IBM has rapidly progressed over the last two years.

        • (writing from New Mexico, USA)–you are so right, Bairet. Swallowing problems persist for many of us. As do difficulties with breathing. And pain; some IBM patients have a great deal of pain despite what some sites claim.

          Here’s a little Hx thats somewhat atypical, with a bit of hope perhaps for a few among the IBM community.

          I had three “episodes” of rapid-onset muscle wasting a little less than 10 years apart. Good recovery followed the 3 month duration of the 1st occurrence, moderate (80%, over a year’s time) the 2nd, and very slow recovery (about half of the prior 80%) over the last 8 years: leaving me now with about 4/10ths of the strength I had originally.

          I was not diagnosed with anything for 20 years until, in 2007, a neurologist decided that these “episodes” were recurrent Guillain-Barre syndrome (which has so many varying causes that its not too useful a Dx). Then in 2012, intensive neuromuscular testing at Mayo, MN, followed by a biopsy of thigh muscle, finally established IBM.

          At which point I was informed that there was nothing to be done about it. And just last month, my rheumatologist happily informed me, “It doesn’t kill you;” this was reluctantly followed by an admission that well, yes, choking and/or ceasing to breathe can be hazardous to your health. (Hence, our interest here in BYM338 as long as it does not prove to be a risk for other disease states.)

          Meanwhile, since the literature has made it clear that physical therapy has been the only (long term) non-risky, somewhat helpful approach, here’s what I’ve been doing since 2007– long before I knew this was IBM: 1) bit by bit, relocated my household items-used-daily to more
          challenging spots–a little more reach up or down, a little more
          stretch before grasping, a few more steps to get to items;
          2) backed off temporarily when anything started hurting, as connective tissues are not designed to take the stresses of musculature and so sometimes ligaments can start to tear;
          3) following a swallowing study, experimented with the Dr.’s advised techniques until my swallowing ability improved a bit and I choked less often (also, at times my throat muscles won’t initiate a swallow so food gets stuck at the back of my throat; I try very, very gently inhaling a bit of air through the nose, then expel that air forcefully through the mouth with a cough to dislodge the stuck food);
          4) did slow breathing exercises at bedtime, lying flat: a long, gentle breath in through the nose as deeply as possible, then out through pursed lips very slowly as though blowing out a candle (hint: if the chest muscles feel as though they can’t expand enough, try using the abdominal muscles for “belly breathing” as babies do);
          5) because no physical therapist who was well-trained and experienced in working with IBM patients was available in my area, ADL’s (activities of daily living) have had to substitute for professional assistance; a little book entitled “Limbo” was an incalculable help, too (I found it in a box of discarded books as I was leaving Arizona’s Mayo Clinic in 2008–where the doctors had suggested over a dozen possible diagnoses including “bizarre presentation”.)

          Clearly, many commenters at this site have done sufficient research into their condition to have arrived at the point where clinical trials into potential chemotherapies are of interest.

          If any readers of this site either 1) know of someone else with muscle loss suggestive of IBM, or 2) are not yet entirely incapacitated themselves, it is my hope that everyones’ posts here will continue to inspire others to keep searching for improvements in both treatment and personal functioning. You certainly have given me a good, strong nudge. Thank you.

  6. I have IBM for 7 years and i have difficulties with stairs. I walk 2 to 3 miles a day and exercise daily. I am from Canada. I am very happy to hear about this new drug . Hope it helps a lot of people.

    • Hi fellow Canadian,I have been dealing with polimyositis for about seven years. They are now calling it IBM. I think they put me in this category because there are no Tx that they can offer me anymore. I was put through all the usual suspects ( ie. methotrexate, Prednasone IGIV ) Nothing seems to have helped or worked if anything my health was made worse by these drugs. What has been your experience with the medical society concerning your illness? Have you had any favourable treatments to help with your IBM? Thank you for any info you can relay too me!

      • Hi Anne,

        I am taking methotrexate but my medication is being reduced every year. I do exercise every second day, and I stay positive. I am now retired, but when working I would force myself to climb the stairs rather then using the elevators. I am 58 years young and hope someday there will be help for all us.

  7. It is good to see the helpful communication on this disease. It makes dealing with it easier when we all share our experiences. I was diagnosed 2 years ago but have been struggling with the effects for about 6-8 years. Have any of you tried the anti-immune drug called Cellcept? I’m wondering if it could be helpful. Thank you in advance for any comments you’d care to share….Fred.

    • Hi Fred

      I was prescribed Cellcept by the UCL Hospital in London, as my IBM showed quite a lot of inflammation. I stayed on the drug for around 18 months but it had no effect, as the drug is pretty toxic I came off it as you never know if it may be doing more damage to other parts of the body. Hoping to get on the next lot of trials as this sounds a lot more hopeful.

      • Thanks, Diane.
        I appreciate your reply and wish you well in being able to be included in a future trial. I’m hopeful that bym 338 will be good news for us. Blessings…Fred

      • Hi Diane,
        I too have IBM diagnosed about seven years ago. I am now 68.
        I walk with two sticks, and have problems with stairs, getting off the toilet etc. my legs are very weak, and I have had quite a few falls.
        I cycle with the aid of a battery bike, and swim for half an hour. I am better in the water than on land!
        I also have a fold up mobility scooter which we take on planes, busses and trains. A new trial is about to commence, but because I have had a renal transplant, I may not be able to participate.

      • Hi Diane,
        I too have IBM diagnosed about seven years ago. I am now 68.
        I walk with two sticks, and have problems with stairs, getting off the toilet etc. my legs are very weak, and I have had quite a few falls.
        I cycle with the aid of a battery bike, and swim for half an hour. I am better in the water than on land!
        I also have a fold up mobility scooter which we take on planes, buses and trains. A new trial is about to commence, but because I have had a renal transplant, I may not be able to participate.

    • Cellcept can be helpful but it puts patients at a high risk for cancer. My Dad has gotten cancer five times since he was first perscribed. A tough decision many IBM patients have to make is whether the risks outweigh the rewards. Though the battles with cancer weren’t fun (albiet brief) the day-to-day improvement was, and has been, worth it. Just be aware of the risks.

    • Fred; I have had IBM for 5 years and am 75 now. In 2010 my Neurologist wanted me to try Cellcept. I was to take 3 pills a day, and after taking 2 pills I developed severe pains in my chest. I spent three days in the hospital because my liver enzymes were off the chart. The official diagnosis was drug induced hepatitis due to the Cellcept. Therefore, I would be very careful if you decide to try Cellcept.

  8. Thank you for your information.I have IBM, and have had it for over seven years now.
    four years ago, I landed in the hospital, with clots in the lungs, and two years ago I fell
    on my head, and have not been able to walk since then, I got so week from muscle loss.

    I do not think I have much hope now, I am a young 73 years old, and I would think the help would go to some one much younger. That is OK as long as there is some cure in the future.

    Gilbert Ruiz


  9. hi,
    im a pharmacists..currently part of a research team. you writing is amazing,its great. and you ,you are full of life and hope and ingenious. neurodegenerative diseases has took and taking over the life of my dearests. please stay strong ..please stay steady and firm..even though your whole world is shaking up and its unexpected and massive consequences..please stand it..even though standing is the most difficult choice..and it really does..but we will make a different..there..somewhere at some point we will make it better.

  10. I would like to enrolled in the trial being held in Houston, Texas. I have been unable to a contact number.

  11. Hi
    I am 52 years old and had been diagnosed with IBM about three months ago, although I had been struggling with walking and running for a few years already. In the original news release by Novartis, they mentioned that the results of the study (phase II), will be made available at a Neurology Conference in the USA in October, and that it will be published in a scientific journal later in the year. I am unable to find any information on the results. Any idea where to start looking?

    • As far as I know the results have not been published yet. However the main researcher presented the information that he had available to The Myositis Association national conference last month. Here is a link to the YouTube video of his presentation:

      You really should join the myositis Association. There is no membership fee and it is a tremendous source of information.

  12. Soy portador de una IBM,todavía puedo andar por terreno llano con ayuda de un bastón,edad 68 años.Vivo en Valencia (España).Me gustaría que alguien me indicara como puedo acceder al Tto. con BYM338. Muchas gracias.

  13. I have tried to get into the clinical trial in Kansas City. They are only taking five people. I called Mayo Clinic same there. The doctor told me it’s the same everywhere, there will only be 240 people world wide in this trial, 5 people at each trial site.

  14. Oxandrolone does not alleviate IBM symptoms?

  15. Neurology. 2002 Apr 9;58(7):1081-7.

    A pilot randomized trial of oxandrolone in inclusion body myositis.

    Rutkove SB1, Parker RA, Nardin RA, Connolly CE, Felice KJ, Raynor EM.

    Author information



    Inclusion body myositis (IBM) remains without effective therapy. As anabolic steroids have myotrophic properties, the authors studied whether a synthetic androgen, oxandrolone, would have efficacy in IBM.


    A double-blind, placebo-controlled, crossover design was used. Patients received oxandrolone or placebo for 12 weeks followed by a minimum 2-month washout period, followed by 12 weeks of the alternative treatment. Maximal voluntary isometric contraction testing (MVICT), manual muscle testing (MMT), and functional performance testing were obtained before and after each treatment period, with the whole-body MVICT score as the primary outcome measure.


    Of 19 patients enrolled, 16 (14 men, 2 women; median age 68.5 years) had complete data for at least the first treatment period, with 13 completing the entire study. Whole-body MVICT improved by a median of 15.5 kg with drug and 4.1 kg with placebo (p = 0.06), whereas MMT demonstrated a median increase of 2.0 Medical Research Council points with drug and 0.9 point with placebo (p = 0.33). Upper-extremity MVICT demonstrated a significant treatment effect, with strength increasing a median 6.3 kg with drug vs 2.5 kg with placebo (p = 0.006). Stair climbing also increased a median of 1 step on average with drug versus no change with placebo (p < 0.001). Minimal adverse effects occurred.


    Oxandrolone had a borderline significant effect in improving whole-body strength and a significant effect in improving upper-extremity strength as measured by MVICT. Given these findings, further study of this drug, possibly in combination with an immunomodulating agent, is warranted.

  16. Has anyone read the case study of the 70 year old woman that they put on low dose IVIG instead of high dose? Have they had any success with anyone else on low dose? I tried to get my doctor to put me on low dose version, he wouldn’t even consider it. He said if it was going to work it would work on high dose, but he’s never had any success with IVIG. He put me back on eight weekly infusions of prednisone again that seem’s not to be working also. My question is why are doctors so reluctant to try anything, if it doesn’t work you can always go back to prednisone. I guess maybe I’m just too frustrated with being told there’s nothing they can do. I say step out of the box, what do we have to lose.

  17. Hello,

    So I found out today that we are having a baby boy!
    Along with this exciting news, I made the all important sweep of telling everyone.

    I eventually came across my granny, who now lives in her small room at the old folks home after gramp died ofcourse.

    She has one leg with a muscle the size of a walnut supporting it.

    She has had this problem for around a year and I am hoping someone can point us in the right direction.


  18. I was 35 yo when on a routine physical I noted that my Creatinine Kinase was elevated.It was shortly thereafter I noticed I had a frequent foot drop while walking. Over the next 23 years I have suffered just about all of the symptoms that have been described by so many of you. I too have tried all of the “anectodotal” medicines from Prednisone, Methotrexate, IVIG, until finally realizing there was nothing that would help my situation.I too have pushed myself with a kind of self prescribed physical therapy as the cycle of strengthening and then wasting seem to at least keep me on an even keel.
    Suffice to say,I now take solace in the fact that I am not alone, and I never realized that there are so many that are struggling with this disease. I also am now hopeful that Novartis will continue to push through with this very promising drug and possibly offer all of us an effective treatment, I won’t bother you with a long list of my symptoms, nor my struggles in getting a definitive diagnosis, but if anyone wants to know more about my individual experience, feel free to contact me by email.

    • Hi Thomas,

      I was diagnosed in Oct 2015 with IBM. I can’t imagine your struggles but would like to know how you have coped with the disease for 23 years.

      • I suspect your journey will be quite different from mine because there should be some kind of treatment available in the next few years. For me, my life has just slowly changed as I have made adjustments and adaptations to the loss of various muscles. It hasn’t been that unpleasant for the most part, just different. I wrote a book about it all covering the time from pre-diagnosis to 2014. It is titled “Rolling Back: Through a Life Disabled,” and is available on Amazon in Kindle or paperback.


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