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.
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.