How Lab-Made "Mini-Organs" Can Treat Cystic Fibrosis

Over 1,000 individuals in the United States are diagnosed with cystic fibrosis every year. Fortunately, due to several advancements in its treatment, the life expectancy of this condition has been extended. Even though the disease cannot be prevented, researchers are focusing on new opportunities to develop a lifelong cure. One such example is lab-made “mini-organs” to treat cystic fibrosis. 

Cystic fibrosis is a severe, genetic condition that affects the respiratory and digestive systems and damages the lungs, liver, intestines, and pancreas. It causes a build-up of thick, sticky mucus, leading to symptoms such as a persistent cough, shortness of breath, wheezing, stuffy sinuses, and a stuffy nose. The enzymes produced by the pancreas become blocked in the passageway due to the abnormal mucus build-up, thus hindering the absorption of nutrients in the body. This causes constipation, bloating, foul-smelling stools, and loss of appetite. The symptoms may appear in childhood, or they may not develop until puberty or even late adulthood. Depending on the individual’s health and response to treatment, the symptoms may become better or grow worse. 

A single gene mutation induces cystic fibrosis; it causes the production of a protein called CFTR (cystic fibrosis transmembrane conductance regulator). This protein regulates the water and salt content in the cells that line the organs. Changes in the CFTR gene produce thick, sticky mucus, which can cause life-threatening problems if left untreated. So, early diagnosis and proper treatment are important to improve a patient’s quality of life. Although cystic fibrosis cannot be prevented, individuals with the disease or who have relatives affected by it can undergo genetic testing to determine their risk of developing the condition.

In order to develop a lifelong cure, researchers are focusing on new treatment opportunities. Elsa van der Heijden, a 53-year-old woman with cystic fibrosis, experienced difficulties while breathing due to the thick mucus build-up. Doctors could not find a cure for her rare form of cystic fibrosis, so they decided to try grazing a few cells from Heijden to grow a mini replica of her large intestine in a dish. They noticed that the mini-gut had a good response to treatment, so they believed a similar result could be obtained from the large intestine. In this experiment, a mini replica of the intestine was gown in order to get to the root cause of the symptoms and determine a suitable treatment. This approach is in the early stages of experimentation in laboratories worldwide. In a laboratory in the Netherlands, mini-guts have been grown in order to identify and understand the rare mutations associated with cystic fibrosis. Different types of expensive drugs are currently being tested on them. Mini-livers in the United States, mini-kidneys in Australia, and mini-brains in England have also been created. However, these experiments are very expensive. Drugs are administered to the custom-made organs in the laboratory and researchers then observe whether the mini-organs are able to properly balance water and salt. 

Heijden’s overall health has been significantly impacted by this research. She was diagnosed with cystic fibrosis during childhood, but in recent years, she has been able to regain her strength thanks to experiments like these. This approach can be used for gene therapy as well. Several patients with cystic fibrosis can continue to live long, healthy lives through such advancements. The main goal is to continue discovering new therapies in order to tackle the underlying cause of cystic fibrosis.