Trial to determine if stem cells can help with rare lung disease

Jose Guzman began gasping for breath while using his treadmill on the lowest setting and knew something was wrong. His search for answers led him to the diagnosis of a rare lung disease with no cure.

Guzman, 72, who arrived from Cuba penniless and built a thriving Miami travel agency, doesnt give up easily. He has signed on to be one of the first patients to participate in a clinical trial being launched at the University of Miami. Dr. Marilyn Glassberg has obtained approval from the Food and Drug Administration to launch the first U.S. clinical trial that will test whether mesenchymal stem cells given intravenously could be a therapy for patients with Guzmans rare lung disease, known as idiopathic pulmonary fibrosis. The disease strikes mostly men who are 55 and older and ex-smokers.

Glassberg, a pulmonologist and professor of medicine and surgery and director of the pulmonary division at the Interdisciplinary Stem Cell Institute at UMs Miller School of Medicine, has studied rare lung diseases for 20 years. She says this is her first real hope of extending or improving the lives of her patients who have this progressive and fatal lung disease, which often leads to death within five years of diagnosis.

Glassberg says it if successful, stem cells could be applied as a potential therapy for other, more common lung diseases such as asthma or emphysema.

We believe moving cell-based therapies to diseases like these make sense, Glassberg says.

For the clinical trial, Glassberg says she chose to first focus on a lung disease with the worst prognosis.

Once fibrosis is present, it is the end stage because the damaged cells dont know how to repair themselves, she explains. Our hope is that the stems cells will curb the acceleration.

The trials results could change the way doctors manage lung disease for patients and get them away from drugs that have not been effective, she said.

The idea for using intravenous mesenchymal stem cells as a treatment for lung disease came from the work of several researchers, including a UM colleague who had used stem cell therapies on cardiac patients, Glassberg says. While reading a report on Dr. Joshua Hares 2009 study that used stem cells to repair heart damage, Glassberg said she was struck by one of its findings: the cells infused into the heart noticeably improved lung function, too.

The probability made sense to Glassberg because the lungs are the first stop for injected stem cells, regardless of where they are targeted. She became convinced she should try to apply this therapy to the pulmonary disease that has frustrated her for more than a decade.

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Trial to determine if stem cells can help with rare lung disease