Stem cell therapy for ILD, where are we now?

Coline van Moorsel, PhD CV

Stem cell therapy for ILD, where are we now






The public health information series of the April 2017 issue of the blue journal carries the title “Unproven Stem Cell Treatments for Lung Disease—An Emerging Public Health Problem”.  Stem cell approaches are generally considered to be promising and may lead to new therapies. Particularly in IPF, as many patients show disease progression under current best treatment regimens.

The article informs readers on current status: there are no proven stem cell therapy for any lung disease and interested patients might consider participation in legitimate clinical trials.

First results of one such trial are presented in the may 2017 issue of Chest by Glassberg et al. They report on the relative safety of a single infusion of allogeneic human mesenchymal stem cells in 9 patients with mild-moderate IPF over a period of 60 weeks. While several reports have now shown relative safety of different procedures, stem cell therapy in IPF is still in its infancy. Basic information on optimal stem cell source (autologous vs allogeneic; umbilical cord, bone marrow, or blood derived) and delivery route (intravenous vs endobronchial) is missing.

Of six currently listed IPF stem cell trials, one clearly stands out: Clinical Efficacy and Safety of Autologous Lung Stem Cell Transplantation in Patients With Idiopathic Pulmonary Fibrosis (NCT02745184).

The study is based on the identification by Zuo et al (Nature, 2015) of specific distal airway stem cells (p63(+)Krt5(+)) that were experimentally confirmed to be essential for lung regeneration. In the current phase 1, 2 trial, autologous bronchus derived stem cells will be expanded in vitro and returned directly into the lung lesion after lavage.

Coming results from this and other studies will not only show if stem cell therapy has significant efficacy in IPF, but will also shed light on the capacity of the patient’s own lung cells to contribute to lung repair.

Further reading:

Unproven Stem Cell Treatments for Lung Disease-An Emerging Public Health Problem. Am J Respir Crit Care Med. 2017 Apr 1;195(7):13-4.

Glassberg et al. Allogeneic human mesenchymal stem cells in patients with idiopathic pulmonary fibrosis via intravenous delivery (AETHER): a phase I, safety, clinical trial. Chest. 2017 May;151(5):971-81.

Zuo et al. p63(+)Krt5(+) distal airway stem cells are essential for lung regeneration.  Nature. 2015 Jan 29;517(7536):616-20.