2017年5月27日星期六

First Duchenne MD Patient to Be Approved for Adult Stem Cell Treatments in US Turns 30

Ryan Benton, the first patient to be approved in the United States for allogenic adult stem cell transplantation as a Duchenne muscular dystrophy (DMD) treatment, turned 30 this year — largely exceeding his original life expectancy.

DMD is a genetic disease that affects 1 out of every 3,600 male infants, leading to progressive muscle degradation. The average life expectancy for people with this disease is about 25 years. Benton, of Wichita, Kansas, was diagnosed with DMD at age 3, and doctors predicted he would live only into his late teens to early 20s.

At age 22, his health began to seriously deteriorate. At the time, research indicated that adult stem cell therapy might reverse DMD progression, so Benton met with Neil H. Riordan, PhD, the founder of the Stem Cell Institute in Panama City, Panama, and Medistem Panama.

Although he knew that there was no guarantee, Benton decided in 2009 to receive the stem cell treatment provided by Dr. Riordan’s team of physicians at the Stem Cell Institute. However, due to the laws that restricted adult stem cell therapy in the U.S., he had to travel to Panama. Shortly after the first treatment, he started feeling stronger and experienced no adverse side effects. He ended up making a total of seven trips to Panama for treatments, and after five years was granted approval by the Food and Drug Administration (FDA) for stem cell therapy inside the United States.
But the FDA’s approval was limited to his treatment exclusively, and only allowed for twice yearly treatment for a maximum of three years. Although the treatments improved lung capacity and muscle mass, increasing the ability to breathe and physical strength, the cells started to lose their effectiveness three to four months after each treatment.

In January 2016, the FDA granted approval for Benton to undergo an additional treatment per year, allowing for a more effective reversion of the disease’s progression. Additionally, the regulatory agency approved the therapy for another patient, a 6-year-old boy also showing benefits from previous treatments in Panama. The boy has already received his first treatment in the U.S., potentially paving the way for future patients.