After several promising treatments in Panama using stem cell technology developed by Medistem Panama Inc. at the City of Knowledge in Panama, a 6 year-old Duchenne’s muscular dystrophy patient received his first umbilical cord tissue-derived mesenchymal stem cells in the US earlier this year following FDA approval of a second application for a single patient, investigational new drug (IND) for compassionate use.
Umbilical Cord Stem Cell Treatment
Duchenne muscular dystrophy (DMD) is a rapidly progressive form of muscular dystrophy that occurs primarily in boys. It is caused by an alteration (mutation) in a gene, called the DMD gene, which causes the muscles to stop producing the protein dystrophin. Individuals who have DMD experience progressive loss of muscle function and weakness, which begins in the lower limbs and leads to progressively worsening disability. Death usually occurs by age 25, typically from lung disorders. There is no known cure for DMD.
This trial marks the second time the FDA has granted an investigational allogeneic stem cell IND for Duchenne’s in the United States.
Ryan Benton, the first DMD patient to be treated in the US with umbilical cord stem cells just celebrated his 30th birthday, a landmark age for any Duchenne’s patient. The FDA recently approved a request to increase Ryan’s treatments from two to three times per year. Since his treatments began in September 2014, Ryan’s condition has stabilized and there have not been any adverse side effects reported.
The new subject had traveled to the Stem Cell Institute several times for treatments similar to Ryan’s. Encouraging results and news of Ryan’s compassionate use trial prompted his parents to seek out a similar trial for him in the US, which was recently granted by the FDA.