News from the internet.

Aug 312015

The interest level and expectations surrounding BYM338 are evidenced here.

The interest level and expectations surrounding BYM338 are evidenced here.

All of us with inclusion body myositis are waiting anxiously for some sign of results from the Novartis BYM338 trials. After all, until now there has been no proven treatment available for this relentlessly progressive muscle wasting illness. The critical stage III worldwide trial is wrapping up later this year and so far all we have are anecdotal remarks from participants. Of course they can only guess because the study is blind, with a fourth of the participants getting a placebo and another fourth getting a dose that is very low. The good news is that some people are reporting results that to them seem significant enough they believe they must be getting either the middle or highest dose and it is working.

However there are places we can look to find real data. For example, a study report published in Current Rheumatology Reports and made available through contains some actual results from the phase IIa trial on patients. This was a small trial of 14 participants and of them 11 received the drug. It states that the participants who received the drug were given a dose of 30 mg per kilogram of body weight in a single infusion. The results were then measured at two months and again at three months. After two months, the average person receiving the drug gained about 7 percent of thigh muscle volume. Walking distance in six minutes was measured another month later and the average drug recipient gained 15 percent in walking speed. Both results were statistically significant. The dose was three times greater than the maximum dose being used in the current stage IIb/III dose-finding trial, (although it was only a single dose while the trial consists of monthly doses.) According to the Brookings institute, these results were what prompted the FDA to grant Breakthrough Status to BYM338.

The results of an early study of BYM338 are reported here.

The results of an early study of BYM338 are reported here.

Another place we can look to obtain hints about the success of BYM338 is in the other trials that Novartis has been conducting. The website lists eight studies that have been completed or are ongoing plus one that was withdrawn. Among the conditions for which the drug is being tested are IBM, sarcopenia, and cachexia related to chronic obstructive pulmonary disease, lung cancer and pancreatic cancer. Sarcopenia is the skeletal muscle wasting that occurs in most people beginning at age 30 and accelerates as we age. It is classified as an illness when the muscle wasting exceeds two standard deviations from the norm. Cachexia is the wasting of muscle plus fat that results from many serious illnesses. Considering the high cost of conducting all these trials, it is clear that Novartis has high hopes for the drug. As do we!


1. In the Novartis quarterly report presentation for Q2 of 2015 they continue to state that FDA submission of BYM338 for sporadic inclusion body myositis will be in 2016. My physician was told by Novartis that they were accepting no requests for compassionate use for this drug prior to FDA approval. Might that policy change once the submission to FDA has been made? (Compassionate use, also called extended use, is an FDA program to make drugs available to seriously ill patients when no other medication is available. FDA states that compassionate use is available for drugs that have not been formally approved nor proven effective, so it is a mystery why Novartis has chosen to withhold this particular drug from the program.)

2. As I understand the mode of action of BYM338, it prevents myostatin from signaling muscle cells to stop growing. This allows the cells to resume their normal process of regeneration (myogenesis). Although it doesn’t cure the IBM, it hopefully will allow the body to build new muscle cells as fast or faster than IBM destroys them. Of interest to those of us where the disease process has resulted in near paralysis, how much muscle fiber must be remaining for myogenesis to take place?

3. The trial with mechanically ventilated patients was withdrawn. Does this mean there was some side effect related to mechanical ventilation? Unfortunately, some IBM patients may become too weak to breathe without mechanical assistance.

Feb 132014

The paperback version of Rolling Back: Through a Life Disabled

The paperback version of Rolling Back: Through a Life Disabled

Rolling Back has been published in paperback and is available on Amazon for $6.99 ($6.64 for Amazon Prime members). There is also the Kindle version that costs $2.99. I have provided links to each of them below.

Writing and publishing Rolling Back as been a personally rewarding experience that I wouldn’t trade for anything. Several people have urged me to write another, and I will probably try. However I think I’m ready for a change of pace and may attempt a fiction novel next. I’d be interested to hear your thoughts.



Feb 042014

This is the cover for my new book. The art is a slightly modified version of one of my late wife's paintings.

This is the cover for my new book. The art is a slightly modified version of one of my late wife’s paintings.

My book, Rolling Back: Through a Life Disabled, has been published and is available as a Kindle version on Amazon. You don’t need a Kindle to read it, you can read it on any computer or any tablet for smart phone using the free Kindle app. Kindle owners who are Amazon Prime members can borrow it for free.

Rolling Back will be available as a paperback in a few weeks. Right now it is only in the Kindle format, but will be expanded to include other e-readers in three months. The price for the Kindle version is just $2.99. If cost is an issue I hope to be able to offer it free for five days on Amazon. When that happens, I will let everyone know.

Oct 192012

My daughters and I have spent the last few days working on Beth’s obituary. This is a task that should not be put off until the death of a loved one. There is so much we would want to tell people about my wife, their mother. But there is also the reality. To begin with, people reading the newspaper and scanning the obituaries are generally not likely to want to read a long story about how much someone meant to you. What’s more, at $10 a line, indulging in excess sentiment could quickly become very expensive–money that should be better used. So when it came right down to it, we realized the important facts of her life trumped the depth of our loss and the breadth of our love. Here is the finished product as it will appear in Sunday’s paper.

Obituary for Elizabeth Shirk

Obituary for Elizabeth Shirk as it will appear Sunday, October 21, 2012.

Apr 282011

The Outlook Spring 2011, Page 6

The Outlook, a quarterly publication edited by Theresa Curry for The Myositis Association (TMA) devotes nearly two pages of its Spring 2011 issue to tell its readers about the Life! disabled blog. TMA is a nonprofit organization located in Alexandria, Virginia and dedicated to raising and distributing research dollars in search of cures for the various myositis diseases. In addition to being mailed to patients, caregivers, physicians, researchers and opinion leaders, the Outlook is also available online to members of The Myositis Association. One way to support the important research they do is to join TMA. The dues are very reasonable and the work they do is extremely valuable, especially to those of us who suffer from such rare, difficult and disabling illnesses. Please visit their website:

Sep 072010

Welcome to Life! (disabled). And note the emphasis. Despite having a difficult disabling illness, I still find a lot of pleasure in living. Much of that pleasure stems from finding – and sharing – new ways to cope as my illness progresses. In this blog I will share some of the things Inclusion Body Myositis has taught me. I am not a medical professional, so please regard this information as personal observations and not medical opinion.