Novartis new drug

Posted by john schell , 2012/06/09, 14:45:55 Post Reply   Main Forum
I received the following response from My son Dr. Michael Schell . He is a neuroscientes and researcher. He answered my inquiry regarding research being conducted by Novartis Drug Co. on their drug BYM338 being studied for use in IBM patients. The Co is working with The Children s Hospital in Columbus Ohio who are also involved in the "Gene Therapy Trials" Although this effort is not related to the current ongoing "Gene Therapy trials being conducted under Dr. Mendel", apparently they are going to use some of the information gathered by Children's Hospital in terms of measurements taken by the Hospital on a number of us who volunteered to have our strength abilities tested. At this point of time no trials are in process, but hopefully having the tests available will speed up the drug being for human trials more quickly. I think this is a positive sign and good approach. For this reason I submit the following from my son.

Hi Dad,

It took me a while to figure out how that Novartis drug BYM338 is supposed to work. It is a type of monoclonal antibody that blocks a receptor in the body called activin receptor IIB (ActRIIB). The reason this is relevant to IBM is that some other researchers studying the process of wasting (cachexia) in cancer discovered that blocking the receptor prevented the weight loss and muscle loss in a mouse model for cancer-induced wasting. You can read all about it at the link below.

The related audio interview is especially good, and it is free:

There are some underlying mechanisms shared by muscle wasting in cacechia and wasting in IBM. Because BYM338 will probably prevent myostatin signaling, it should promote muscle growth. The effect should be pretty fast (weeks). What else blocking this receptor (i.e., unwanted side effects) will do to the body is unclear. BYN338 is a monoclonal antibody, so it will have t be injected and it is expensive. One promising thing is that small molecule blockers of the same receptor are already in development, which could in theory lead to a cheaper oral alternative.

Bottom line: this is a promising line of research. The pros are:

1. The trial is placebo-controlled double-blind. So, about half of the subjects will get a placebo injection. This is the best design to be confident about the study's outcome.
2. The clinical assays of outcome (MRI and a walking test are good.
2. The hypothesized improvement in muscle function should occur fast--on the order of 8 weeks. Thus, they should obtain good information about this approach to treat IBM pretty quickly.

The cons and risks:

1. BYM338 is unlikely to be specific for promoting muscle growth. It may trigger all sorts of other things, or have gonadal hormone-like side effects, or unanticipated effects on the immune system. The follistatin gene therapy approach going on in Ohio targets muscle growth more specifically than would BYM338.

2. Requires Injections of expensive monoclonal antibody--how long or how many, temporary or for the rest of your life--unknown.

So for those considering participating in the trial, they need to weigh the risks against potential benefit and also understand that if they do participate, they have a 50% chance of being assigned to the placebo control group. Regardless of outcome, participation in the trial may also lead to more pioneering research in this area. If the outcome is promising, it may point to better strategies to target the ActRIIB signaling pathways more specifically with drugs or genes, thus reducing side effects and improving dosing.

Love Mike John 37

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