Pulmonary Fibrosis Foundation Issues Caution Concerning Stem Cell Therapies for IPF Treatment

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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The Medical Advisory Board of the Pulmonary Fibrosis Foundation (PFF) recently released an announcement regarding therapies based on stem cells for the treatment of idiopathic pulmonary fibrosis (IPF).

IPF is a progressive, fatal lung disease in which the alveoli and the lung tissue are damaged, becoming thick and scarred (fibrosis), leading to severe breathing difficulties and compromising oxygen transfer between the lungs and the bloodstream. IPF is characterized by a shortness of breath that gradually worsens, with respiratory failure being the main cause of death associated with the disease. There is no cure for IPF and it is estimated that 128,000 individuals in the United States suffer from the disease, with approximately 48,000 new cases diagnosed every year. IPF has a poor prognosis and around two-thirds of the patients die within five years after being diagnosed.

Late in 2014, the FDA approved two new therapies for the treatment of IPF, Ofev (nintedanib) and Esbriet (pirfenidone). However, patients are still seeking out novel therapeutic solutions for the disease, and stem cell therapy has recently become a popular, albeit controversial, emerging treatment approach for a range of diseases, including IPF.

Stem cell therapies are based on the use of stem cells, which are undifferentiated cells that have the remarkable potential to develop into several different specialized cells of the body, such as cells from the skin, bone, muscle, etc. Stem cells are also capable of proliferating and spreading into other tissues. The main sources of stem cell therapies are the bone marrow, but also umbilical cord blood.

In addition to early stem cell initiatives raising some ethical controversy in terms of abortion politics and human cloning, the new wave of stem cell therapy based on adult stem cells is prompting disease advocacy groups like the PFF to encourage patients to tread carefully with the still-untested treatment.

“We know very little about the impact of such therapies in humans, and rigorous clinical trials have not been conducted to test the role (or adverse effects) of stem cells in the management of IPF. Furthermore, few studies have been conducted in animals or humans for the treatment of any lung condition,” stated the PFF Medical Advisory Board in their announcement.

According to the Board, based on the potential regeneration of scarred lungs through stem cell therapies, patients are becoming increasingly eager to undergo such therapeutic interventions. However, “patients need to be aware that the treatment regimen with stem cell/infusions and its safety and efficacy need to be determined in ongoing studies that are still in early phase 1 and 2 clinical trials. At present, there are only a small number of approved clinical trials evaluating cell-based therapies in the United States, Europe, and other countries.”

The PFF Medical Advisory Board emphasizes that due to the promising therapeutic benefits of stem cell therapies, studies using these cells are being conducted in patients outside of approved clinical trials. Medical tourism for stem cell therapy is in fact becoming a reality, and the potential risks associated with it led the PFF to advise patients to use caution.

“The IPF community should be proud of the advancements made in IPF research and patient care. These advancements have been possible through the careful implementation of rigorous scientific efforts, including randomized clinical trials,” noted the Medical Advisory Board. “To provide the best care for our patients with pulmonary fibrosis, or any illness for that matter, a rigorous evaluation of any therapy in controlled clinical trials is the only way to assess the benefits and potential harm. This includes rigorously evaluating cell-based therapies. We owe it to our patients.”

The PFF went on to note that both Ofev and Esbriet were approved only after rigorous, carefully planned and executed clinical trials proved their safety and efficacy.

“Until the safety and effectiveness of such therapies have been rigorously documented, the Medical Advisory Board of the Pulmonary Fibrosis Foundation believes that patients should be skeptical about the usefulness of stem cell therapies and should engage only through participation in approved clinical trials, in which patients are closely monitored and the potential benefits and harm can be objectively assessed,” concluded the PFF Medical Advisory Board.

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