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by +Richard Holbrooke – UM Miller School professor of medicine and surgery Marilyn K. Glassberg, M.D., has won approval from the U.S. Food and Drug Administration to conduct the first U.S. clinical trial of mesenchymal stem cells as a potential therapy for patients with idiopathic pulmonary fibrosis.

Issued 30 days after she applied for an investigational new drug trial, the FDA approval gives Glassberg her first real hope of extending or improving the lives of patients with the progressive and fatal lung disease that has no known cause or treatment, other than a risky transplant. About half the patients die within five years of diagnosis.

“Most of these patients walk into my office with their burial plots ready to go, and want to know how many more months they have,” said Glassberg, who has conducted most of the available studies on potential therapies for idiopathic pulmonary fibrosis. “Wouldn’t it be nice if I could say, ‘You know what? You’re going to get a lot more months now.’ If stem cells work, it’s a new lease on life.”

Joining Glassberg in testing the therapeutic potential of intravenous mesenchymal stem cells for the disease characterized by progressive shortness of breath and irreversible loss of lung function is Joshua Hare, M.D., the Louis Lemberg Professor of Medicine and Director of the Interdisciplinary Stem Cell Institute (ISCI). Glassberg’s application to the FDA, which authorized a Phase 1 trial with nine patients who will receive escalating intravenous doses of donor mesenchymal stem cells during a brief hospital stay, was inspired by Hare’s pioneering research in cardiac stem cell therapies. While reading his 2009 study in the Journal of the American College of Cardiology that demonstrated the capacity of mesenchymal stem cells to repair hearts damaged by myocardial infarction, she was struck by one of its findings: mesenchymal stem cells infused into the heart noticeably improved lung function, too.

Curious about the extent, she contacted Hare. When the pair met, they discussed the available cardiac data, and the fact that the lungs are the first stop for injected stem cells. It didn’t take long for the researchers to agree they should try stem cell therapy for the desperate pulmonary disease that has frustrated Glassberg for more than a decade.

“It is well documented that the cells go to the lungs first before passing into the heart. That is the reason we decided to try it,” she said. “We think they could provide factors that are needed to stimulate other cells into making new tissue. The lung can’t repair itself so that is the only hope.”

Calling idiopathic pulmonary fibrosis (IPF) “dreadful,” Hare said the disease “seems to have all of the features and characteristics that would make it responsive to cell therapy. And since we have very significant experience with these cells, and understand their safety profile, dosing and manufacturing, it seemed very worthwhile to partner with Marilyn to come up with this very exciting program of cell therapy for IPF.”

The FDA and the NIH apparently agree. With vital assistance from ISCI’s investigational new drug team and Rebecca Toonkel, M.D., a senior pulmonary fellow at the time, all the necessary application documents, including Glassberg’s previous IPF studies and ISCI’s extensive experience with mesenchymal stem cells, were assembled and submitted.

The proposal quickly passed ISCI and FDA reviews. Concurrently, the NIH’s National Heart Lung and Blood Institute convened a comprehensive review of all issues of cell therapy in lung diseases and invited Glassberg and Hare, along with other national experts, to attend the November 13-14 meeting of its new working panel.

The researchers also plan to submit an RO1 grant proposal to the NIH for the second phase, a double-blind, randomized study of 16 patients. With the nine patients already pre-screened, Glassberg hopes to complete the first phase within six months of Institutional Review Board approval.

“After 10 years, nothing has worked and it’s been really sad,” she said. “But, finally, I have a lot of hope for these patients.”

6 Comments

  • Veronica Brooks says:

    My husband Danny has had IPF going on over 5 yrs now .The last year it has really started progressing. We have tried the Predisone & other various drugs but nothing seems to be helping.He is taking breathing therapy now & is on 3 liters of oxygen 24/7. Nothing seems to be working for him so far.we even w went to Mayo clinic in Jacksonville but he did not qualify for any of the clinical trials or treatment or lung transplant.he also is diebetic & has had 4 heart attacks with a stent placement. His Dr ,Sergio Balingit said the only thing left to try was stem cell treatment but also said insurance would not pay for these services. Is there hope that maybe he would qualify for a stem cell trial for his lungs that would be paid for by research? If not can you tell me about the appromiate cost and what it involves? Thank you for your help. Veronica

  • Ricky Chjarles says:

    i have a close friend that is diagonsis with pulmonary fibrosis, i am intrested in this research. please leave me news of the trials with stem cells.

  • Richard Petrillo says:

    To: Dr. Marilyn Glassberg:

    I was diagnosed with IPF by Dr. John Fowler of the Vancouver, WA clinic in June of 2008.

    I have also been involved in the Boehringer Ingelheim Pharmeuticals Clinicaltrial.gov NCT01619085 using the drug BIBF1120 and conducted out of the Portland OR clinic, for almost two years. Since involved in that trial my breathing capacity has not changed. I’m told that I have about 68% of my lung capacity which has held steady for about a year now.

    Obviously, I am extremely interested in your experiment with stem cells in the treatment of IPF.

    I anxiously offer myself as a participate in your next trial, and can be available (with bags packed and airline ticket in hand) on a moments notice.

    Please let me know what I would need to do to participate. I anxiously await your answer.

    Richard Petrillo

  • Richard Petrillo says:

    I was diagnosed with IPF in June of 2008 by Dr. John Fowler of the Vancouver clinic in Vancouver, WA.

    I have also been participating in the Boehringer Ingelheim Pharmaceuticals trial NCT01619085 using the drug BIBF1120, conducted from the Portland OR clinic, for almost two years.

    I’m told their has been no deterioration of my breathing capacity for the last year, since I have been involved in this trial. I am also told my lung capacity is holding steady at about 68%.

    Naturally, I am extremely interested in your experiment with stem cells in treating IPF.

    I wish to offer myself as a participant in you work with stems cells to treat IPF.

    I can be ready at a moments notice (with bags packed and airline ticket in hand.

    Please let me know what I need to do to participate?

    Anxiously awaiting your reply.

    Richard Petrillo

  • Connie Applegate says:

    I was diagnosed by Dr.Limper at the Mayo Clinic, Rochester, Dec. 2005 with IPF and had a lung biopsy. I am being seen by Dr. DeAndrede at UAB now. I have had 3 infusions of stem cells in the past 3 years and they have helped. The infusions were not in country and not encouraged by Dr. DeAndrede. I know the infusions have extended my life. However, I would like to participate in your stem cell research if that is possible. I had mesenchymal with growth factor the last infusion as well as adipose stem cells. My oxygen level is at 4, for sleeping, 5 if I’m sitting and 6 if I’m busy in the kitchen etc.. Please contact me at my e mail address or call me at 850-651-0848. I live in Shalimar, Fla.

  • Marty Basil says:

    I have ipf – diagnosed in October 2013 – confirmed in Jan. 2014 at UPMC – I would be interested in trial.

    I am 55 years old.

    Marty

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