FOLLISTATIN GENE TRANSFER - Follistatin Gene Transfer to Patients with Becker Muscular Dystrophy


Frequently Asked Questions (FAQs) about this research:

What stage is this research?

  • This Phase 1 trial is ongoing but not recruiting participants.


Where is this research being performed and who is funding this research?

  • This research is being led by Dr. Jerry Mendell at Nationwide Children’s Hospital Research Center in Columbus, Ohio. It is being funded by PPMD’s GIFTED Program.


What is the goal or purpose of this research?

  • Follistatin is a muscle growth-stimulating Glossary Link protein. This research is intended to build upon preliminary studies in mice with muscular dystrophy and in non-human primates which demonstrated that the follistatin Glossary Link gene, when injected into muscles, can cause significant increases in the size of injected muscles and improvements in the strength of injected muscles. If successful, the investigators can potentially prolong a patient's ability to walk.
  • The gene will be carried into the muscle by a virus called adeno-associated virus (AAV). This virus occurs naturally in muscle and does not cause any human disease.


What do participants have to do in this study?

  • Participants with either of these diseases (Becker Muscular Dystrophy or Sporadic Inclusion Body Myositis) will have shots of the follistatin gene injected directly into their thigh muscle on one or both legs (one time only). A muscle biopsy will be done during the screening visit and 180 days following the gene delivery. Once both muscle biopsies are completed, the biopsies will be looked at closely to see if the muscle fibers are bigger. At day 180, participants will undergo testing to see if their muscle strength has improved. Between the time of the gene transfer and the muscle biopsy at day 180, participants will be carefully monitored for any side effects of the treatment. This will include an MRI of the thigh muscle before treatment and at day 180 following treatment. Blood and urine tests, as well as physical examination will be done on the participants during the screening visit and several times throughout the study to make sure that there are no side effects from the gene injections. 


What are the future plans for this research?

  • If this study is successful, the investigators will expand the research to a phase 2 study and will also make plans to test it in patients with Duchenne muscular dystrophy.


Where can I learn more about this research?

(Updated 3/31/2017)