May 05 - Cord Blood Stem Cells for Cystic Fibrosis

May is Cystic Fibrosis Awareness Month. In this article, we’ll explain how cord blood stem cells may be used to treat cystic fibrosis and share some recent research findings.

What Are Cord Blood Stem Cells?

Cord blood is blood that remains in the umbilical cord and placenta after childbirth.  Researchers have discovered that it is a valuable source of stem and progenitor cells, which can be used to treat a many different medical conditions.

Most of the stem cells found within cord blood are hematopoietic stem cells (HSCs). HSCs are blood-forming stem cells that use a process called haematopoiesis to turn into white or red blood cells.

Currently, there are more than 80 stem cell treatments that use HSCs. This includes treatments for conditions like leukaemia, lymphoma, sickle cell disease, Fanconi anaemia (FA), and Wiskott-Aldrich syndrome.

Cord blood also contains Mesenchymal Stem Cells (MSCs). MSCs are multipotent stromal cells that can differentiate into various types of cells including adipocytes (fat cells), chondrocytes (cartilage cells), osteoblasts (bone cells), and myocytes (fat cells).

MSCs are a particularly valuable resource for researchers in the field of regenerative medicine. This field studies the “process of replacing, engineering or regenerating human cells, tissues or organs to restore or establish normal function”.

Researchers have discovered that MSCs can:

  • Help the body repair various types of tissue
  • Form new blood vessels in damaged tissue
  • Reduce inflammation
  • Slow the progression of autoimmune diseases
  • Prevent transplant rejection, and much more.

These attributes could make MSCs useful for treating a many different conditions including osteoarthritis, bone injuries, heart disease, and cancer.

What is Cystic Fibrosis?

Cystic Fibrosis (CF) is a common genetic disorder caused by mutations in the gene encoding of the CF transmembrane conductance regulator (CFTR). This gene is responsible for creating a protein that regulates how salt and water move into and out of cells.

This mutation causes the CFTR protein to be be produced in insufficient quantities or to be malformed. As a result, the mucus, sweat, and digestive juices produced by the body are thick and sticky, when they should be thin and slippery. This is particularly damaging to the respiratory and digestive systems. This thick, sticky mucus clogs up the passageways, ducts, and tubes in certain organs.

There are a variety of symptoms associated with cystic fibrosis including persistent coughing, frequent lung infections, breathlessness, and constipation. It is a very dangerous condition because it can lead to complications like respiratory failure and diabetes.

There’s at least 70,000 people worldwide living with CF and there is currently no cure for the condition. The average lifespan of a cystic fibrosis sufferer is only 37, with the most common cause of death being lung complications.


How Could Cord Blood Stem Cells be Used to Treat Cystic Fibrosis?

Scientists around the world have been studying how stem cells could be used to treat cystic fibrosis for a number of years. Some of the potential treatments they have developed are designed replace or regenerate damaged tissue in the airways, to improve the respiratory function of cystic fibrosis sufferers. Other research projects seek to fix the malformed CFTR genes that cause cystic fibrosis to occur in the first place.

Most of the current research projects use either cord blood stem cells or induced pluripotent stem cells (iPSCs). iPSCs are generated from a human subject’s own cells in a laboratory. They are powerful multipotent cells capable of turning into any type of cell, including lung cells. This makes iPSCs useful for improving the condition of the lungs in people with cystic fibrosis.

One of the most exciting discoveries involving the use of cord blood stem cells has come from the University of Adelaide in Australia. A study performed by scientists from this university found that cord blood stem cells could be used to restore lung function. Researchers will soon test the treatment on a patient using stem cells donated by their sibling. The trial will be held later this year.

Another upcoming study, also from the University of Adelaide, uses a combination of stem cell therapy and gene therapy. The researchers will harvest adult stem cells from the lungs of CF patients, correct them with gene therapy, and then reintroduce those cells back into the patient. The re-introduced cells won’t have the mutation that causes cystic fibrosis. If successful, the treatment could completely cure cystic fibrosis.

Researchers in the United States are testing the use of stem cells to reduce lung inflammation associated with cystic fibrosis. They believe that the anti-inflammatory capability of mesenchymal stem cells could reduce cystic fibrosis symptoms, extending the lifespan of people with the condition.

Another study from the United States used stem cells to convert immature lung cells into mature cells, to improve lung function. This treatment could help people suffering from many types of lung diseases. Clinical trials are expected to commence in the coming years.

Stem cells are also being used to help researchers develop new drugs for cystic fibrosis.  One research project from the School of Women’s and Children’s Health & University of NSW (UNSW) uses patient stem cells to determine the precise type of CFTR gene mutation they have. They can then test drugs that specifically address this mutation, restoring production of the CFTR protein and halting the progress of the disease.