In 1938, American doctor Dorothy Anderson noted a condition in which individuals suffered damage to the pancreas, calling it cystic fibrosis. Other physicians of her time and even earlier noted symptoms such as salty skin and thickened mucus, making it hard to breathe and function normally. While in recent decades, scientists have gained a much better understanding of what cystic fibrosis does to the body, they haven’t had nearly as much luck in finding an effective course of cystic fibrosis treatment. Luckily, that may soon change.
In this article:
- Cystic Fibrosis in Children
- Cystic Fibrosis: A Brief Overview
- Cystic Fibrosis Treatment
- Stem Cells: A 21st Century Promise
- Stem Cells and Cystic Fibrosis: Match Made in Heaven?
- Widespread Use: On the Horizon
Stem Cells for Cystic Fibrosis Treatment
Cystic fibrosis treatment is in high demand. It is the most common inherited disease for caucasian people, and one in 2,500 babies are born with some level of the disease.
Cystic Fibrosis in Children
While cystic fibrosis prognosis is getting better, it is still only at 37.5 years for children who receive a cystic fibrosis diagnosis at birth. Cystic fibrosis in children has devastating consequences for health and ability, and cystic fibrosis life expectancy for milder forms of the disease is still significantly shorter than those without it.
Luckily, new stem cell research shows significant hope for cystic fibrosis treatment. It seems this disease, which has haunted humanity for millennia, may be nearing a cure.
Cystic Fibrosis: A Brief Overview
Cystic fibrosis is a genetically transmitted disease. It passes down from family members, and many people are born with it, though others don’t show symptoms until later in life. The disease affects the exocrine glands, which in turn causes the body to produce very thick mucus. This substance is normally used to lubricate the body, but when it becomes too viscous, it instead blocks up pathways. These include lung bronchi, intestines, pancreatic ducts, and more. Breathing difficulty and respiratory infection often ensue.
So, what are the symptoms of cystic fibrosis? They include:
- shortness of breath
- persistent, mucus-laden coughing
- inability to exercise
- lung infections
- stuffy nose
- poor weight gain
- intestinal blockage and constipation
In recent years, awareness of cystic fibrosis has expanded dramatically. Since the 1950s and 1960s, when research started bringing its mechanisms to light and a number of famous people were revealed to suffer from the disease, it has taken center stage as a source of research and scientific inquiry. Today, doctors are busy diagnosing cystic fibrosis in adults, categorizing it (e.g., delayed onset cystic fibrosis and cystic fibrosis in babies) and determining the causes of cystic fibrosis.
All of these steps are likely to lead to an effective cystic fibrosis treatment sooner or later, and with the new research happening today, probably sooner.
Cystic Fibrosis Treatments
Sadly, there is not a cure for cystic fibrosis. However, there are a number of cystic fibrosis treatments that help to alleviate the symptoms and consequences of the disease.
Currently, cystic fibrosis is treated with:
- antibiotics to prevent and remedy chest infections
- medicines to thin mucus within the lungs
- medicines to widen the airways
- medicines to reduce inflammation
- medical devices to clear mucus from the lungs
- medicines that help patients absorb food, diet modifications and supplements
For patients with severe cystic fibrosis, a lung transplant may even become necessary.
Stem Cells: A 21st Century Promise
Stem cells are providing emerging hope for cystic fibrosis treatment. Stem cells are the body’s “master cells,” capable of becoming more specialized versions to meet the demands of the body. There exist many multipotent stem cells in the body, which are capable of becoming a range of cells in a specific category. Multipotent blood cells can become dozens of different types of specialized blood cells. While pluripotent stem cells can become any cell in the body, they unfortunately are typically sourced from embryos.
Thankfully, scientists discovered a way to “induce” adult cells to become pluripotent in nature. These cells are known as induced pluripotent stem cells.
The problem with the body normally is that not all cells can reproduce, so damaged cells don’t get replaced. Even in the ones that can normally reproduce, such as the lung cells affected by cystic fibrosis, the genes responsible are damaged. Stem cells can help in two ways:
- Stem cells can replace the affected cells with healthy ones.
- Stem cells can carry new genetic codes into the body, replacing the defunct codes that dictate the production of over-thick mucus.
Researchers could potentially accomplish this in a number of ways, including modifying genes and their expression, as well as transplanting healthy cells into an individual, where they will grow and replace the unhealthy cells currently there. Typically, they use multipotent adult stem cells (cells derived from living adults), obviating the need for embryonic sources.
Stem Cells and Cystic Fibrosis: Match Made in Heaven?
New research shows that cystic fibrosis treatment could manifest sooner than we think. Studies from the faculty members at the University of Melbourne show that umbilical cord blood may have the potential to treat the disease and restore healthy lung function. For now, the research is exploring the use of stem cells from sibling cord blood, which may counteract the dysfunction and overproduction of mucus in damaged bodily systems.
The Cystic Fibrosis Trust has also funded a project to correct “premature stop codon mutations in cystic fibrosis using genomic editing of human Induced Pluripotent Stem Cells (iPSCs).” Cystic fibrosis is caused by a genetic mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, so this approach will use a gene editing technique known as CRISPR to correct the mutation.
In addition to exploring a gene-edited cell therapy approach to the treatment of cystic fibrosis, the researchers involved with this project are creating a valuable population of cells to be used for testing new drugs for cystic fibrosis.
Additionally, a search of the world’s largest Clinical Trial Database (ClinicalTrials.gov) reveals 7 clinical trials have investigated the use of stem cells for cystic fibrosis treatment.
Only one of these trials is currently active, titled “Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis” (NCT02866721). The collaborators on this study are Case Western Reserve University and the Cystic Fibrosis Foundation.
For now, it seems that stem cells could provide promise for those suffering from cystic fibrosis. Because the problem stems from unhealthy cells, the ability to replace those cells – and have the new healthy cells continue to thrive over time – could potentially mitigate symptoms or cure the disease.
Cystic Fibrosis Treatments On the Horizon
Stem cell and cystic fibrosis testing have come a long way in the last few decades. Researchers have examined the disease from many angles, including cystic fibrosis in children and cystic fibrosis in adults newly diagnosed. They have examined mild cases and severe, long-term conditions and complicating factors.
To search for a stem cell trial for cystic fibrosis, watch this video:
While there aren’t yet many treatments available for widespread use, the momentum is accelerating. For now, the best source of information is a primary care provider or specialist, so for patients and loved ones looking for answers, start there.
Questions about Your Treatment Options for Cystic Fibrosis?
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For this reason, please contact GIOSTAR, a global stem cell company that has treated a large number of patients, with your medical questions. You can reach them at this link to schedule a consultation or ask them your questions.
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