Muscular Dystrophy Sufferers Hope New Treatment Can Answer Prayers

Jemelle Holopirek, KAKE 10 News, May 4, 2010

Adult stem cell therapy has been used for many diseases including heart failure, liver failure, stroke, multiple sclerosis, and even drug resistant tuberculosis. The biological basis for how stem cell therapy works seems to be two-fold. On the one hand, the stem cells appear to have the ability to become new tissues, on the other hand, the stem cells produce various proteins that stimulate the body to heal itself. One condition for which stem cell therapy may offer great hope is Duchenne Muscular Dystrophy, a disease in which muscle cells deteriorate due to the presence of a mutated gene (dystrophin) whose protein produce is involved in muscle contraction.

Researchers from Cellmedicine in collaboration with Medistem has previously published a case report in the peer-reviewed literature demonstrating improvement in a Duchenne's patient treated with mesenchymal stem cells (Ichim et al. Mesenchymal stem cells as anti-inflammatories: implications for treatment of Duchenne muscular dystrophy. Cell Immunol. 2010;260(2):75-82). The patient described in the paper, Ryan Benton, was the subject of a previous news report which is available at http://www.youtube.com/watch?v=Jyt2LHayjcs.

Today a news report was published describing follow-up on Ryan Benton as well as another Duchenne's patient Ian Conner that was treated with stem cells by Cellmedicine.

Ryan and Ian have known each other all of their lives, having watched their condition progressively deteriorate. Last year Ian's condition substantially worsened.

"At the time, I didn't think I was going to live much longer," Conner said. His mother Laurie Conner stated "Last year, I thought it would be very soon that he would be dying. We needed to get ready, because he was so sick, in bed a lot and he felt terrible." However there was a glimmer of hope. Ian's mother told him about the response Ryan had after receiving stem cell therapy.

"We got a muscle biopsy back and it has produced dystrophin and it's producing normal amounts of dystrophin," Ryan said. He continued "The main difference I've noticed I've gained a lot of weight I was down to 77 pounds."

The treatment appears to show greater effects the more times the stem cells are injected. Both Ian and Ryan are hoping that stem cell therapy for Duchenne's will one day be approved in the United States so that they do not have to travel outside of the country.

Dr. Riordan has been in discussions with various organization and welcomes any input on collaborations that can be used to accelerate implementation of this approach through the Food and Drug Administration.