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, doesn’t 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 Guzman’s 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 UM’s 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 don’t know how to repair themselves,” she explains. “Our hope is that the stems cells will curb the acceleration.”
The trial’s 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 Hare’s 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.
Now, with FDA approval, Glassberg will conduct the first phase of the clinical trial in the next few months with nine patients who will receive escalating intravenous doses of donor stem cells during a hospital stay. Guzman will be one of the nine.
The second phase will involve 16 patients, some of whom will receive the stem cell therapy while others will receive a placebo. The third and final phase is a larger study with about 300 patients worldwide.
Like Glassberg, Hare, director of the Interdisciplinary Stem Cell Institute at UM, said he is optimistic about stem cells as a treatment for lung diseases.
“Idiopathic pulmonary fibrosis seems to have all of the features and characteristics that would make it responsive to cell therapy,’’ Hare said. “Since we have very significant experience with these cells, and understand their safety profile, dosing and manufacturing, this clinical trial seems very worthwhile.”
From his tests with heart patients, Hare said he found the stem cells led to a scar tissue reduction in the heart. To him, it would make sense that the same effect would take place with fibrosis in the lungs.
“We did see lung function improve in heart patients. We don’t know why, but we’re going to find out.”
Hare says he finds this clinical trial exciting because of the possibility of using stem cells in multiple organs. He likens the potential to the discovery of antibiotics for multiple uses.
“It’s a transformative area in medicine and could lead to a new class of treatments,” he said.
Dr. Lorraine Ware, associate professor of medicine at Vanderbilt University, said she, too, sees potential for stem cells to be “a multi-modal therapy.” In the past, treatments have focused on single pathways or proteins, she noted.
“These stem cells can influence multiple pathways that lead to sites of injury,” she said.
Ware believes the medical field could be in the early stages of a breakthrough: “I think this represents quite a bit of promise for treating acute and chronic lung conditions.”
Ware, a lung researcher, says she is encouraged by previous trials with stem cells that indicate they are safe and show hints of efficacy.
Guzman, a grandfather of five, exercises regularly with a physical therapist, cares for his wife with diabetes, and says he looks forward to a possible treatment that will allow him to extend his life with his family.
“Dr. Glassberg’s goal is to find a cure for pulmonary fibrosis and I want do what I can to help her find it.”