the party protein

We have mentioned the Follistatin Gene Therapy clinical trials at just about every KIT group meeting we’ve hosted, but often don’t have visual aids on hand. Thank goodness this blog is a visual aid! Because follistatin is kinda a big deal.


Follistatin looks like party streamers, right? When it comes to growing muscle, this is the party every myositis patients wants to get invited to!!!

Basically, follistatin is a muscle GROWING protein that fights myostatin (a muscle growing INHIBITOR).

Click here for Wikipedia’s definition of follistatin.

When you’re talking about myositis, a disease that breaks down muscle, anything that helps bulk muscle mass (and hopefully strength and endurance) is a good thing. That’s why we’ve been so excited to follow the progress of the Follistatin Gene Therapy trials! This is truly science fiction come to life: follistatin’s Superman to myostatin’s Lex Luthor.

Click here for a history of gene therapy.

We first heard about Follistatin Gene Therapy at the 2008 TMA Conference here in Denver (again, if you haven’t been to a TMA Conference, they are AMAZING!!! Click here for information about the 2014 TMA Conference). In 2008 follistatin research had been on mice and macaque monkeys, and we were shown pictures similar to the ones below.

Screen Shot 2014-03-07 at 6.43.12 AM

The blog post “Super Strong Genetically Engineered Monkeys” is a good interpretation of the macaque trial in 2009, click here. Or to read the full medical article of the early trials, Kota et al 2009 [from the National Center for Biotechnology Information (NCBI) and National Institutes of Health (NIH)] click here.

Screen Shot 2014-03-07 at 6.21.31 AMScreen Shot 2014-03-07 at 6.37.51 AM

With the success of the animal trials, in 2010-2011 the Follistatin Gene Therapy clinical trials moved on to human testing. Testing was done on both Becker muscle dystrophy (BMD) and sporadic (non-genetic) inclusion body myositis (sIBM) patients.

Parent Project Muscle Dystrophy (PPMD) and The Myositis Association (TMA) funded the Follistatin trials.

Investigators, led by Jerry Mendell, M.D., director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital with co-investigator Brian Kaspar, Ph.D, will inject a modified virus (vector) carrying the gene for the muscle growth-stimulating protein follistatin into the quadriceps muscles of volunteers with Becker muscular dystrophy and sporadic inclusion body myositis.  The goal of the study is to verify that the procedure is safe and to document any increase in quadriceps muscle size and function.  People with these diseases have overall muscle weakness but with particular weakness of the quadriceps muscle, which is important for standing and sitting.  Preliminary studies in mice with muscular dystrophy and in non-human primates demonstrated that follistatin delivered in this manner can cause significant increases in the size of injected muscles. Improvements in the strength of the mice and non-human primates were documented. – See more at:
“This is the first time a gene therapy approach has been used to supply genes that generically stimulate muscle growth rather than directly replacing missing muscle proteins, “ explains Sharon Hesterlee, Ph.D., PPMD Senior Director of Research and Advocacy, “Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse. “  – See more at:

TMA hosted a Live Discussion on their website, June 2013, to have Dr. Mendell answer questions about the Follistatin Gene Therapy trials, click here for transcript. And Dr. Mendell announced preliminary findings in October 2013, click here.

You can see why we are so excited about these advancements – Party On, Myositis Colorado!!!


Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s