We are pleased to introduce Dr. Andrew Mammen today to answer your questions about IBM. Dr. Mammen, a neurologist and member of TMA's medical advisory board, was one of the founding directors of Johns Hopkins Myositis Center. He currently has a laboratory studying myositis at the National Institutes of Health. He's the author of many peer-reviewed publications on myositis. He's on TMA's Medical Advisory Board and is a long-time friend of TMA. He'll speak at TMA's Annual Patient Conference in September in New Orleans.
Many of you have already submitted your questions, but feel free to submit more as the discussion progresses. Dr. Mammen is not able to diagnose or treat your particular illness, and will give priority to questions of interest to all of those with IBM.Ask a Question
I've read in several journals that some improvement has been noted in IBM patients treated with anabolic steroids. Why is treatment with the anabolics never offered or suggested?Dr. Andrew Mammen:
I am not aware of convincing studies demonstrating a benefit of these drugs, which can have significant side effects, for patients with IBM. That’s why most clinicians do not recommend them.
Promising Clinical TrialParticipant:
With BYM338 out of the picture for now, which current clinical trial is the most promising at this time? If all criteria are met, when might this protocol reach the U.S. market?Dr. Andrew Mammen:
Some experts are optimistic about a study using arimoclomol for IBM. It was found to be safe in humans. However, it may be some time before the trial testing its efficacy in humans will be completed.
While some of us are fortunate to be on the slower end of the muscle-wasting trajectory, and can still lift-weights, play golf, dash about as though life is normal, are short periods (1-2 weeks) of extreme exhaustion a common element of IBM?Dr. Andrew Mammen:
Many people with IBM do experience exhaustion. However, 1-2 week periods of extreme exhaustion with no exhaustion in between these episodes would be less common.
My husband had a biopsy at Columbia Presbyterian Hospital on 4/28/16. The surgical report concludes "a reasonably high suspicion for inclusion body myositis" but the report also refers to " an absence of rimmed vacuoles and amyloid deposition."
As a layman it seems to me that another physician should look at his slides to confirm what sounds like a somewhat questionable diagnosis. Would you be willing to review his slides or recommend the appropriate resource?Dr. Andrew Mammen:
If the diagnosis of inclusion body myositis is in doubt, it is reasonable to seek a second opinion. Since the biopsy slides alone are not sufficient to definitively diagnose IBM, this will involve a detailed physical exam and perhaps other testing (i.e., more than just reviewing the slides.) For referrals to the Johns Hopkins Myositis Center, please fax records to 410-550-3542.
Supplements to Slow ProgressionParticipant:
Dr. Mammen, Are you finding in your research that there are a few supplements such as Coq10 that do have benefit in slowing progression of IBM, and if so what other supplements might be helpful?Dr. Andrew Mammen:
To the best of my knowledge there is no data suggesting that supplements such as CoQ10 slow disease progression in IBM. Most clinicians treating IBM patients do not currently recommend these supplements for IBM.
Electric Muscle Stimulation MachineParticipant:
With IBM can we benefit with the use of electric muscle stimulation machine with exercises?Dr. Andrew Mammen:
These devices may play a role in those who have certain kinds of nerve diseases (although I’m not an expert on this). However, I am not aware of any reason to think this would be helpful in patients with muscle disease.
I was disappointed in the reported lack of success with BYM338 and the subsequent hold status in the Regeneron myostatin inhibitor trial. Is this avenue of treatment dead in the water, or is there still some hope for benefit from this strategy of treatment?Dr. Andrew Mammen:
Although the trial failed, these myostatin inhibitors may still have some promise, perhaps also in other muscle diseases. However, I have not heard whether the companies intend to keep moving forward with them.
Can you comment on the status of the clinical trial of bimagrumab? In spite of its not reaching the first end-point, is it likely ever to be offered to the public? If so, when?Dr. Andrew Mammen:
I do not have inside information about bimagrumab or the FDA. That being said, my understanding is that the study was not able to demonstrate a benefit of bimagrumab in IBM. Consequently, I don’t think the FDA will approve this drug and make it available to the public. If a future study showed it to be beneficial for IBM (or something else) it could someday be available. But I do not think this is likely to happen anytime soon, if at all.
Leg Pain and RestlessnessParticipant:
Judging by discussions on Facebook, an increasing number of IBM patients are reporting leg pain and restlessness. I have not found anything that helps relieve that pain and calm the restlessness. Are there any treatments that could help?Dr. Andrew Mammen:
It depends what the cause of the symptoms is. For example, if pain is due to neuropathy, there are medications for that. Similarly, different medications are appropriate for restless leg syndrome. You should see consider seeing neurologist to have the cause(s) diagnosed and the symptoms managed.
Exposure to Agent OrangeParticipant:
I am a Vietnam veteran with exposure to Agent Orange and have had the IBM diagnosis since 2013...I know of 2 others veterans in Maine with similar history and diagnosis. Have you noticed any connection more than average between the two?Dr. Andrew Mammen:
To the best of my knowledge, there is nothing published on an association between Agent Orange and IBM. We have seen a couple of IBM patients with Agent Orange exposure. As the generation who served in Vietnam becomes older, it may be possible to determine whether this is truly a risk factor for IBM.
Testosterone and BimagrumabParticipant:
I am a 68-year-old recently diagnosed (by muscle biopsy) IBM patient. I have been on testosterone for lab confirmed low testosterone since before the IBM diagnosis. I noticed that at least one clinical trial excluded anyone on testosterone. If I want to get into another clinical trial, should I stop the testosterone now? There seems to be a 6 month "off" criteria.
With the recent discouraging news about bimagrumab failing to meet it's primary endpoint, is it still possible that it will proceed toward FDA approval? Given the fact it attained Fast Track and Orphan Drug status, it seemed like the "best hope" out there. Do follistatin and arimoclomol or others hold similar hope in the near future?Dr. Andrew Mammen:
Given that there is a good medical reason for you to be on the testosterone, I would think twice about stopping it to join a trial.
Regarding the trial: I don’t have any inside information on this. However, I think it’s highly unlikely that bimagrumab will be given FDA approval based on the recently completed trial, which did not demonstrate a definite benefit.
How common is it for people with IBM to have chronic pain?Dr. Andrew Mammen:
In my experience, some, but certainly not all, IBM patients do suffer from chronic muscle pain.
Going Off MedicationsParticipant:
Thank you, Dr. Mammen, I have had four neurologists and three rheumatologists over ten years as I lose them to research. Five have been adamant that I have polymyositis from the biopsy and from the fact that I manage better with taking Celcept and having Rituxan. The last two doctors I've seen feel sure I have IBM and just now had me get a blood test to see. I have not yet gotten the results. I am so concerned as I know I do better with the medication. If the test indicates IBM, will it be absolutely necessary to go off all meds? I have a sister with dermatomyositis and a brother with IBM. He is very depressed with no treatment. I hope I don't have to go that route. I appreciate your comments.Dr. Andrew Mammen:
Your case sounds very complicated and I am unable to give definite advice about whether you should go off medications. But I would make the following general comments. First, there is no blood test that can be used to make a definite diagnosis of IBM. For example, the anti-NT5C1a test is positive in ~60% of patients with IBM, but is also positive in ~20% of patients with dermatomyositis. Second, if siblings both have muscle disease, it’s very unlikely either has polymyositis or sporadic IBM; this is more likely to be a genetic muscle disease.
Leg Swelling and Burning SensationParticipant:
Context: I have sIBM, 20 years, wheel chair bound, age 75. Problem: I have foot, ankle, and leg swelling, and burning sensation on skin up past the knees. Diagnosed as veins with leaky valves. Compression hose help, but not a solution. Is this a side effect of IBM? If so, is there a mitigating treatment.Dr. Andrew Mammen:
Leg swelling is not usually a direct effect of IBM. However, being in a wheelchair can aggravate this. Also, skin burning sensation sounds suspicious for a nerve problem (e.g., small fiber neuropathy). This may or may not be related to the IBM; some studies suggest IBM patients have more neuropathy than one would expect. Your doctors will need to figure out the cause of these symptoms and only then make recommendations about appropriate treatments.
Distal Muscular DystrophyParticipant:
Is a diagnosis of inclusion body myositis the same as distal muscular dystrophy?Dr. Andrew Mammen:
No, sporadic inclusion body myositis is not considered to be a dystrophy. Dystrophies are a category of genetic muscle diseases.
Stem Cell TreatmentsParticipant:
I've heard of some successful stem cell treatments for IBM. Are there any resources for stem cell treatments you could recommend? Thank you!Dr. Andrew Mammen:
For a variety of reasons, I would be very wary of trusting anecdotal reports of benefit from stem cell therapies. For now, there is no data that stem cell treatments are helpful in IBM. They also happen to be very expensive and often enrich those who provide them. Consequently, the TMA Medical Advisory Board unanimously feels that stem cell transplant approaches have no place in the treatment of IMB unless they are offered in a legitimate clinical trial.
Thank you for talking with us today. I have IBM with all the classic conditions of weakness, falling often, hands dropping things, etc. My quadriceps are wasting away on both legs. I am still walking but only for short distances. I have had the illness since 2010 when I was 50. My question is, I have heard that IBM is not genetic, but my mother had the illness (my body is doing exactly what hers did in relation to atrophy, weakness, etc.), and I had a sister who was diagnosed with polymyosits. Have you ever heard of the illness running in families like this and there not be a genetic trait? I have two sons and worry about their contracting it in their future.Dr. Andrew Mammen:
If more than one person in a family has muscle disease, it’s very unlikely either has polymyositis or sporadic IBM. It is more likely they share a common genetic muscle disease. I would consider consulting with a neuromuscular doctor with expertise in diagnosing patients with genetic muscle disease.
Stem Cell ApplicationParticipant:
Dr. Gary Steinberg, the Chair of Neurosurgery, Director of the Stanford Moyamoya Center is enjoying some success with stroke victims by injecting stem cells into the brain. Is there some possible application of stem cell application to IBM, of which I have been a patient now for 10 years?Dr. Andrew Mammen:
Please see my previous answer about stem cell therapies…
Prepare for FallsParticipant:
Is there a way to help train my body for when I fall, it's like I lose all muscle control and I am not able to protect my head when I go down.Dr. Andrew Mammen:
Falling is not allowed. If you are falling and not using an assistive device (e.g., rolling walker), then it’s probably time to consider an assistive device. If you fall with the assistive device, then it’s probably time to consider a wheelchair or scooter.
I have sIBM. Diagnosed three years ago, however, I have probably had sIBM for over 16 years based upon lab work and symptoms. (I was not one to go to the doctor and complain about difficulty walking, standing up, and falling down.) I am wondering if a course of medication should be tried to help with the symptoms. Such as prednisone, cylosporin, etc. and IVIG was swallowing issues. I had my throat stretched five years ago for dysphagia and scleroderma was ruled out.Dr. Andrew Mammen:
No therapies have proven effective for treating IBM. However, whether or not therapies should be tried remains a very controversial topic even among those who are experts. You will need to discuss with your doctor and weigh the potential benefits of each medication (unknown) against the potential side effects, which can be significant (e.g., stroke or heart attack with IVIG). In my opinion, if used at all, these medications should only be continued in IBM patients if there is a significant objective (i.e., measurable) improvement in muscle strength when they are taken.
AFOs and KFOsParticipant:
Should AFO's and KFO's be considered to help with leg/knee weakness and ankle/foot drop.Dr. Andrew Mammen:
Absolutely! They don’t help everyone, but they are worth trying.
If BYM338 gets approved for sarcopenia, will it possibly be prescribed for sIBM.Dr. Andrew Mammen:
I do think that’s a possibility. But would insurance companies pay for it? I can’t answer that one…
IBM and DementiaParticipant:
Is there a relationship between IBM and dementia and memory changes?Dr. Andrew Mammen:
There may be some connection between the disease processes that cause dementia and those that cause IBM. And there are some genetic diseases that can cause dementia and an IBM-like muscle disease (for example mutations in the VCP gene). That being said, the vast majority of patients with IBM do not seem to be at increased risk for developing Alzheimer’s or other forms of dementia.
Substitute for a Muscle BiopsyParticipant:
What is the validity of the blood test for IBM...is it a viable substitute for a muscle biopsy? Yes, I'm very concerned about having an invasive procedure like the muscle biopsy...it seems that in many instances the muscle biopsy gives questionable results, or am I missing something? Not so sure about the EMG either...I apologize if I sound like a 'cynic', but I sincerely believe that we have to be our own 'best advocates'. These invasive tests are most often painful as well as very expensive, especially considering that there is no "end game" following the tests, at least to date, so to speak. THANKING YOU in advance for your response to my cynicism. Editorial comment: believe it or not, my colleagues most always refer to me as a "pollyanna"...if only they could see me now...guess I'm feeling passionate about this. Actually, I'm surprising myself, but this is 'critically important stuff' for so many...thanks again for even taking the time to read this epistle.Dr. Andrew Mammen:
This is a good question and you would probably get 5 different answers from 5 different IBM specialists. So this is just my opinion. But if a patient has a classic history and pattern of weakness for IBM, an elevated CK, and an EMG showing features consistent with myopathy, you could make an argument that neither the biopsy nor the blood test is absolutely needed. But I would recommend the EMG. However, it’s not always 100% clear what a patient has and in these instances the anti-NT5C1a test and muscle biopsy are often helpful to increase confidence that you have the right diagnosis. The more things that are consistent with IBM, the more confident you are about the diagnosis. Is there a relationship between IBM and dementia and memory changes?
What kind of exercises are best for IBM?Dr. Andrew Mammen:
In many cases, both muscle strengthening and aerobic exercises are appropriate for patients with IBM. However, the appropriate exercises may depend on other health issues and the severity of the weakness. The best approach is to work with a physical therapist who has experience taking care of patients with IBM or genetic muscle diseases (as with IBM, these are progressive).
Can IBM directly affect the vocal cords? Since the tongue is composed of skeletal muscle, can it also be affected by IBM? What would be the early signs? What is your opinion of using Vital Stim (electrical stimulation) to treat dysphasia vs. conventional esophageal dilatation? My doctor said that the muscles that control my eyelids are weak.... what will that mean to me down the road?Dr. Andrew Mammen:
IBM does not typically affect the voice or tongue, except perhaps in very advanced cases. I don’t have much experience with Vital Stim and I’m not aware of any published trial comparing this approach with esophageal dilation. Some people with advanced cases of IBM cannot completely close their eyes when they sleep at night. They need some special treatments for this (e.g., to keep the eyes moist). But this is pretty rare I think.
I have IBM and finger contraction with both hands. Can you recommend a series of exercises that can help me regain use of my fingertips for gripping a glass, etc?Dr. Andrew Mammen:
There may be exercises to help with the contractions. However, these would be tailored to your specific condition and this is not something that can be done in this format. Rather, you should consider seeking the help of a hand physical therapist. These are highly specialized PT’s who focus on rehab issues related to the hands.
Foods to AvoidParticipant:
With IBM are there foods that we should avoid 100%? Also what about protein powders?Dr. Andrew Mammen:
As far as we know, there are no foods that specifically need to be avoided in patients with IBM. That being said, a healthy diet probably makes a difference.
Genetic Muscle DiseaseParticipant:
Hi, I was diagnosed with IBM in 2013 by muscle biopsy. My father was having very similar symptoms , but more advanced. One year ago he was hospitalized for aspiration pneumonia. I asked to have a muscle biopsy perform (on me) and it came back positive for IBM just 3 days before my father passed away. I was able to obtain the last tube with his blood from the hospital and sent it to Columbia University for testing. My doctor said that he did not find any genetic predisposition from his blood and mine. I would like to know if any other tests can be performed to figure out why we both have sIBM. Can we test my children to find out if they are at risk to have sIBM in a future and where tests can be performed?Dr. Andrew Mammen:
If more than one person in a family has muscle disease, it’s very unlikely either has polymyositis or sporadic IBM. It is more likely they share a common genetic muscle disease. I would consider consulting with a neuromuscular doctor with expertise in diagnosing patients with genetic muscle disease. If testing for specific genes associated with IBM (e.g., VCP) is negative, then it may be reasonable to consider whole exome sequencing. Interpreting the results of this testing can be tricky and again, you probably need to find someone that has extensive experience with this.
Upcoming Clinical TrialsParticipant:
Are there any current or upcoming clinical trials at NIH for IBM patients?Dr. Andrew Mammen:
I do not think that NIH has any current or planned trials for sporadic IBM.
Do you see any promising treatments in the very near future available for IBM patients?Dr. Andrew Mammen:
Unfortunately, not in the very near future (i.e., the next few years). However, there may be very promising trials during this time.
I have PM with lung involvement. I am losing muscle and weight rapidly(about 10 pounds in 6 months). I know that exercise is good at slowing this muscle loss. Questions: Is there any objective evidence that testosterone will help or aggravate my muscle loss? Also what about light massage of the muscles? - ThanksDr. Andrew Mammen:
Massage is probably OK. But there is no data that testosterone will help – and it can have side effects.
Is there a genetic component to S-IBM? Could my kids inherit it?Dr. Andrew Mammen:
Excellent question. It’s becoming clear that some people who currently have a diagnosis of sporadic IBM actually have a genetic muscle disease (and there’s not just one gene responsible for this). And there may be genetic risk factors for developing what we consider to be true sporadic IBM. There is a lot of work going on right now to try to answer this question. Stay tuned.
This concludes today's discussion. TMA would like to extend a special thank you to Dr. Andrew Mammen for spending the time to answer your questions. Thanks to all the members who participated.Dr. Andrew Mammen:
Thanks - it was my pleasure. I would be happy to answer more questions if I see you at the TMA Conference in New Orleans!