First-in-human clinical trials reveals the potential of using mesenchymal stem cells to slow the rapid decline of lung function associated with pulmonary fibrosis.

What is pulmonary fibrosis?

Pulmonary fibrosis is a condition where tissues in the lung between the air sacs are scarred and thickened, making it difficult for the lung to function properly. Symptoms of the disease, which include shortness of breath, vary significantly between people. Some people get sick very quickly and decline rapidly. In contrast, symptoms do not a worsen for a long period of time in others.

In most cases, doctors cannot identify the cause behind the scarring, which is known as idiopathic pulmonary fibrosis. Rapidly worsening symptoms are treated with antibiotics, corticosteroids or other medications.

Why study idiopathic pulmonary fibrosis (IPF)?

According to researchers of this study, IPF is one of the most common forms of lung disease in older adults. Additionally, the prevalence of IPF has been increasing over the years.

This study involved patients from a specific category of IPF where lung function deteriorates fast. These patients have a very poor prognosis, where many patients do not survive more than two years after diagnosis.

Why can stem cells be used to restore lung function?

Stem cells are early-stage cells that have an unlimited capacity to divide and grow. Mesenchymal stem cells (MSCs) are one type of stem cells found in the body that has the capability to make many different cell types, including those of the lung.

In this first-in-human clinical trial, researchers looked at whether high doses of MSCs can prevent the rapid deterioration of lung function observed in patients with IPF.

What did they find?

The trial involved 20 patients randomly divided into two groups: one group received two doses of MSCs every 3 months and the other group received a placebo.

First, to note, patients who received stem cells were given a high dose of cells. The researchers were happy to see no significant side effects associated with administering these high doses. Thus it shows that high doses of MSCs are safe and tolerated if used as a treatment option for IPF.

Second, when they looked at the group that received MSC therapy, there was a significant increase in lung function. Those who received the placebo continued to experience deterioration of their lungs.

Optimism for the future

Given the poor prognosis of patients with IPF this clinical trial opens the possibility of exploring MSCs as a safe and promising method for reducing disease progression that increasing life expectancy.

Reference

Averyanov, A, Koroleva, I, Konoplyannikov, M. et al. (2019) First‐in‐human high‐cumulative‐dose stem cell therapy in idiopathic pulmonary fibrosis with rapid lung function decline. STEM CELLS Transl Med. 2020;9:616. doi: 10.1002/sctm.19-0037.