AUG 08, 2018 6:31 AM PDT

Cholesterol Pill For Treating A Brutal Lung Disease

WRITTEN BY: Nouran Amin

A brutal lung disease called Pulmonary Alveolar Proteinosis (PAP) is caused by a thick substance known as surfactant that clogs up the air sacs in the lungs. Now, researchers report in Nature Communications that they figured out why this clogging occurs and show that a cholesterol pill referred to as statin may effectively treat this brutal disease. The study serves as hope for PAP patients because current standard treatments are invasive and usually are repeated every time a patients lungs clogs up, essentially affecting their quality of life.

But, investigators at Cincinnati Children's Hospital Medical Center found that the issue is cholesterol. More specifically, the research team have discovered that early studies PAP was linked to a disruption of cellular regulation by a molecule known as granulocyte-macrophage colony stimulating factor (GM-CSF). GM-CSF is needed for the development of mature macrophages that are crucial for removing surfactant. Led, the team found out years ago that PAP is linked to disruption of cell regulation by a molecule called GM-CSF is crucial for the development of fully mature macrophage cells in the lungs, which are needed to clear away used surfactant -- a substance important to lung function -- so that people can breathe.

In the study led by Bruce Trapnell, MD, director of the Translational Pulmonary Science Center, researchers examined PAP patients and mouse models of the disease and uncovered a critical reason behind disruption of cell regulation by GM-CSF which triggers PAP. This is because cell dysregulation reduced the function of macrophage cells to process and clear out cholesterol, causing the levels to build up. This build-up has adversely effects as it contributes to the accumulation of surfactant leading to PAP. "Showing that the pathogenic driver of the disease is disruption of cholesterol homeostasis in these patients will change thinking in the PAP field," explains Trapnell. "Now that we know cholesterol in macrophages is a target for therapeutic development, repurposing statins is a pretty straightforward pharmacological approach for treating people with PAP." A clinical trial is currently being planned.

The research study was supported from the National Institutes of Health/National Heart, Lung, and Blood Institute (R01085453, HL118161, HL136543), and a National Center for Advancing Translational Science grant through the National Heart, Lung, and Blood Institute (HL127672).

 

Source: Cincinnati Children's Hospital Medical Center

About the Author
  • Nouran earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
DEC 05, 2019
Drug Discovery & Development
DEC 05, 2019
New Injection that Treats Peanut Allergy
Peanut allergies affect between 1 and 3% of the US population. Associated with a heightened risk of severe anaphylactic reactions, oral immunotherapy is th...
DEC 19, 2019
Technology
DEC 19, 2019
Can a 'Lemon' help develop new drugs?
Although artificial intelligence has many advantages in the drug discovery process, challenges still remain--one being the ability for machine learning to ...
DEC 23, 2019
Drug Discovery & Development
DEC 23, 2019
Eye Solution Approved for Surgical Application
As announced by the Dutch Ophthalmic Research Center in a press release, a new eye solution was recently approved by the US Food and Drug Administration fo...
JAN 15, 2020
Drug Discovery & Development
JAN 15, 2020
FDA Approves Drugs Faster than Ever- But is That Good?
The Food and Drug Administration (FDA) is approving new drugs for patients on less and less evidence thanks to special programs that streamline their appro...
JAN 15, 2020
Drug Discovery & Development
JAN 15, 2020
A New Drug Target for Substance Abuse
Research carried out by the University of Minnesota Medical School indicates a new drug target for treating a drug addiction. Researchers note that the dru...
FEB 13, 2020
Cell & Molecular Biology
FEB 13, 2020
Study of Early-Onset Parkinson's Reveals Potential Therapeutic
Around 500,000 Americans are diagnosed with Parkinson's disease every year, and the rate of the disease is rising....
Loading Comments...