Jun 192015

There seems to be some optimism for the future of the new Novartis drug BYM338 currently under investigation for its efficacy and safety as a treatment of inclusion body myositis.There is nothing official, but anecdotal remarks from study participants and others who may have connections within the study point toward encouraging news.

I have heard some people speculate that even if BYM338 is proven effective, it is too late for those of us with more advanced disease. I would like to express a contrary opinion.

My inclusion body myositis has progressed quite far yet I still see potential benefit that could come from BYM338 should it prove effective. After nearly 30 years with IBM (19 since diagnosis) all of my muscles have been affected to some extent. The earliest affected muscles are the worst, such as the quadriceps and finger flexors, but all the rest are gradually growing weaker. At this point, each loss of muscle results in a significant loss of function. For example, the weakness in my diaphragm and core muscles is significantly reducing my ability to breathe and sit upright. The last time my inspiration and expiration strength were measured two years ago, both were only 30% of the low limit of normal. In the past few years, the additional loss of strength in my biceps took away my ability to feed myself using normal motions and forced me to adapt to a slinging motion combined with tilting back in my wheelchair. The point is that once IBM has progressed far enough to cause the ability to walk or stand to be lost, this is far from an endpoint with the illness. I still am able to recruit other muscles to accomplish most of my crucial activities of daily living beyond walking through creativity and use of mechanical aids.

I can also tell that even my most seriously affected muscles still have enough living cells within them to produce tiny movements. After all, a muscle consists of many bundles of muscle fibers which themselves consist of many muscle cells. The point is that even a slight gain in strength and muscle that has been mostly destroyed could still contribute to an adaptation that is important to the patient. Or a slight additional loss of strength could cause that adaptation to be lost.

In my own case, preserving or strengthening certain shoulder muscles could allow me to continue feeding myself indefinitely. Preserving or strengthening remaining healthy muscle fibers in the diaphragm and rib cage could allow me to avoid full-time ventilation. Each of these would be benefits that could easily justify an expensive medication.

If anyone knows how to get this observation in front of any researchers or Novartis executives, please do.

Feb 202014

This is a reenactment of my position when the caregivers got to me.

This is a reenactment of my position when the caregivers got to me.

I am supposed to tilt my wheelchair every couple of hours to relieve the pressure where I sit. This past Saturday was a beautiful day (sorry those of you in the rest of the US and UK), so I chose to get horizontal out in the garden. Before I knew it I had dozed off and was awakened when my right arm slipped off the armrest. It was time to get out of the sun anyway so I attempted to raise my arm to the seat controls. I got within an inch or two and then my arm collapsed. So I tried again. No luck. “Well,” I thought, “I’ll rest a few minutes and try harder.”

Still couldn’t get my arm high enough to grab the armrest. By now both arms were becoming sore from dangling and I realized I would never build up enough strength. There are usually a few people wandering around in the garden, but not this day. Surely someone would come soon. After another 30 minutes I realized it was wishful thinking. Then I noticed I was having trouble breathing. My weak diaphragm makes it more difficult to breathe when I am horizontal.

So I tried to yell for help. Now you’re probably thinking (and you would be right), “How can someone with weak breathing muscles do a good job of yelling for help?”

I decided to pace myself and yell for help two or three times every few minutes. After another 15 minutes, I heard lots of excited voices and was soon surrounded by caregivers who restored me to the upright position. It turned out a resident had been enjoying the sun on our patio about 200 feet away, heard me calling, alerted the staff and I was saved!

I wish I could say, “All’s well that ends well,” but not really. Now I know I can’t venture far from the facility on my own if there is any chance I might, through force of habit, tilt my wheelchair.

I often say that inclusion body myositis forces me to rewrite my life’s script. Lately, it seems it’s trying to force me into coming up with an ending.