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World’s First Stem Cell Study for Cystic Fibrosis

Researchers in the United States have performed the world’s first adult stem cell transplant on an adult with cystic fibrosis. They hope that the transplanted stem cells will reduce the amount of inflammation caused by the disease.

A 39-year-old Ohio man named Bob Held was the first recipient of the treatment.  The research is being performed at the University Hospitals Rainbow Babies & Children’s Hospital in Cleveland in Ohio.

Mr Held received a transplant of allogeneic human mesenchymal stem cells (hMSCs), which were donated by a non-related adult.  Bob has had cystic fibrosis since he was 16-months old and hopes that the treatment will reduce his symptoms.

Cystic fibrosis is an inherited autoimmune condition that affects certain cells in the lungs.  It causes these cells to swell and produce a thick, sticky fluid.  This fluid clogs up the tubes and passageways within the lungs.

The symptoms relating to this condition include shortness of breath, coughing, reduced respiratory function, lung infections, and an inability to gain weight.  It is a life-threatening condition and there is currently no cure.  The average life expectancy of people with cystic fibrosis is 41 years.  It is caused by the immune system over-reacting to the presence of bacteria.

This initial study will look at the safety and tolerability of hMSCs for people with cystic fibrosis.  The lead researcher, Dr. James Chmiel, described the project: “This is an early-phase trial, and the most important thing is to ensure safety. This study consists of a single infusion of stem cells. We will follow the study participants for a year to make sure it’s safe.”  It is a small-scale study with 15 participants from the United States.

The donated stem cells are screened and then given to study participants via an intravenous injection.  The stem cells then travel throughout the body — ideally reducing inflammation and moderating the immune system’s response to bacteria.

Source: World’s First Stem-Cell Study for Cystic Fibrosis

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