Inclusion Body Myositis

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.

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.

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?

I do think that’s a possibility. But would insurance companies pay for it? I can’t answer that one…

Absolutely! They don’t help everyone, but they are worth trying.

Please see my previous answer about stem cell therapies…

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.

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.

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).

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.

Unfortunately, not in the very near future (i.e., the next few years). However, there may be very promising trials during this time.

Massage is probably OK. But there is no data that testosterone will help – and it can have side effects.

I do not think that NIH has any current or planned trials for sporadic IBM.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

No, sporadic inclusion body myositis is not considered to be a dystrophy. Dystrophies are a category of genetic muscle diseases.

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.

In my experience, some, but certainly not all, IBM patients do suffer from chronic muscle pain.

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.

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.