Cystic fibrosis, a genetic disease that leads to progressive lung infections and limits a person’ t ability to breathe, is a deadly disease that impacts more than thirty, 000 people in Americand 70, 000 people worldwide. Just 70 years ago, people born with CF rarely also survived through elementary school, but medical advancements today allow those with CF to survive into their 40s. Still, a substantial amount of progress is still needed to find a cure for cystic fibrosis.
Cystic Fibrosis: A Complex Disease of the Lungs
People diagnosed with cystic fibrosis live with the risk of lung infections caused by thick, sticky nasal mucus that accumulates in the lungs. As the mucus builds up, bacteria thrive and reproduce. The same phenomenon can occur in the pancreatic and liver as well, leading to poor nutrient absorption plus liver disease. This means that people with CF must complete demanding treatments on a daily basis to stay safe. These treatments include cleaning their airways of mucus, inhaling medicines to open the particular airways, and taking enzyme supplements to help the body more proficiently absorb critical nutrients.
The World’ s First CF Stem Cell Research
The application of stem tissues to treat and cure a myriad of diseases over the past years provides inspired James Chmiel, MD, and others at the Cystic Fibrosis Therapeutics Development Center at UH Rainbow Babies & Children’ s Hospital to launch a new research study making use of stem cells to treat cystic fibrosis. “ This is an earlier phase trial, and the most important thing is to ensure basic safety, ” Dr . Chmiel explained. “ The study consists of an individual infusion of stem cells. We will follow the study individuals for a year to make sure it’ s safe. Before using any therapy on a broad basis, we want to make sure that it’ s safe. ”
A 39-year-old from Ohio, Bob Held, launched the particular investigation as the very first patient to receive the infusion associated with mesenchymal stem cells (hMSCs). Healthy volunteers supplied the particular bone marrow from which Held received the hMSC shot. Dr . Chmiel utilized hMSCs in particular because they possess a lot of properties that can treat inflammatory and degenerative diseases. Although this phase one study is simple meant to ensure protection, the researchers hope they will also see a reduction in the particular inflammation in Held’ s lungs.
Dr . Chmiel explained, “ Once within the patient’ s body, the stem cell tracks towards the area where there’ s a significant amount of inflammation, and so they take up residence there. The stem cells then react to the environment, and hopefully reverse some of the abnormalities. We hope within future studies to demonstrate that the stem cells reduce the disease and inflammation and return the lungs to a lot more normal state. ”
As the early phase trial commenced, Held explained to reporters that his participation was inspired by his past due wife Michelle, who also suffered from and ultimately passed away from cystic fibrosis. “ I am hoping the future generations associated with CF patients can get better treatments and that eventually a remedy will be found for them, ” he said. If most of goes as planned, Held will honor his wife’ s memory by being the first participant in what proves to become a groundbreaking research study toward a cure for cystic fibrosis.